SINGAPORE – A few months after releasing its phase 1 and 2 data, OSE Immunotherapeutics, which is based in Nantes, France, has announced positive results for its therapeutic vaccine to treat cancer. Following its promising findings concerning early-stage melanoma, pancreatic cancer, ENT cancers, and HPV-associated anogenital cancer, the company-funded phase 3 Atalante-1 trial has shown the benefits of the Tedopi (OSE2101) vaccine in treating patients with advanced non–small cell lung cancer who are on their second or third line of treatment.

The results suggest that Tedopi is the most developmentally advanced therapeutic vaccine for cancer.

The data from Atalante-1 were presented at the World Conference on Lung Cancer and were simultaneously published in Annals of Oncology.

Tedopi is composed of synthetic tumoral neo-epitopes (peptide fragments) that target five tumoral antigens, permitting the activation of tumor-specific T-lymphocytes for patients who are HLA-A2 positive. In 95% of cases, tumors express at least one of these five antigens. The aim of integrating these five antigens is to prevent immune escape. The technology uses the human leukocyte antigen (HLA) system, one of the keys for presenting antigens to T-lymphocytes. The vaccine is effective for patients who express the HLA-A2 gene, which is present in around half of the population. The HLA-A2 biomarker, detected via a blood test, can identify appropriate patients.
 

Study protocol

In the Atalante-1 trial, participants had locally advanced (unresectable and not eligible for radiotherapy) or metastatic (without alteration of the EGFR and ALK genes) non–small cell lung cancer that was resistant to previous immunotherapy. They had an HLA-A2 phenotype, as determined by a blood draw to determine whether their immune system could respond to the vaccine.

In this trial, 219 patients were randomly assigned in a 2:1 ratio to receive the vaccine or standard-of-care chemotherapy (80% received docetaxel). The vaccine was administered subcutaneously on day 1 every 3 weeks for six cycles. After that point, the vaccine was administered every 8 weeks until 1 year of treatment and every 12 weeks thereafter. The primary endpoint was overall survival.
 

Secondary resistance

The plan was to enroll 363 patients in the protocol, but the study did not complete its recruitment phase because of the COVID-19 pandemic. As a result, the study was stopped after the enrollment of 219 patients.

“It didn’t have the power we would have liked, but it helped us understand that the people who benefited the most from the vaccine were patients who had responded to immunotherapy in the past. These patients have what is called ‘secondary resistance,’ ” explained Benjamin Besse, MD, PhD, during a press conference organized by OSE Immunotherapeutics. Dr. Besse, the study’s principal investigator, is the director of clinical research at Gustave Roussy, Villejuif, France.

Overall, the results weren’t significant. But the results were positive for patients who had previously responded well to immunotherapy for at least 3 months. Of the 219 patients, 118 (54%) had a positive response.

Among these patients with secondary resistance to immunotherapy, median OS was 11.1 months with Tedopi versus 7.5 months with docetaxel.

For these patients, the risk of death was reduced by 41% with the vaccine, compared with chemotherapy. Overall, 44% of patients lived for another year after receiving Tedopi, versus 27.5% with docetaxel.

“This study is a positive signal for overall survival in the selected population. In this study of 219 patients, we realized that just half of patients really benefited from the vaccine: those who had previously responded to immunotherapy,” said Dr. Besse. “The study needs confirmation from a further, larger phase 3 study in more than 300 patients with secondary resistance to immunotherapy to give us the statistical power we need to convince the regulatory authorities.”
 

Tolerability profile

Fewer serious adverse effects were reported with the vaccine than with chemotherapy (11.4% with Tedopi and 35.1% with docetaxel).

The vaccine also allowed patients to maintain a better quality of life. Scores from the Quality of Life Questionnaire Core 30, which explores several areas of daily life, were better with the vaccine. Change in patients’ overall well-being was delayed in the vaccine group: 3.3 months in the chemotherapy arm versus 9 months in the vaccine arm.

“The vaccine was well tolerated. It has benefits in terms of controlling disease symptoms and causes few side effects. Chemotherapy with docetaxel, meanwhile, is more toxic and may affect a patient’s overall well-being. It causes hair loss in practically 100% of patients, induces neuropathy, makes hands and feet swell, damages the nails, is associated with nausea and vomiting ...” noted Dr. Besse. He went on to say that after the trial, of the patients who stopped receiving the vaccine or chemotherapy (either for toxicity reasons or for disease progression), those who had been given the vaccine responded better to the subsequent chemotherapy “because their overall health was better.”
 

Clinical development

The clinical development of Tedopi is ongoing. Three trials are currently taking place. One study is comparing the Tedopi vaccine plus docetaxel with Tedopi plus nivolumab (immunotherapy not used as a first-line treatment) to determine whether the effects of these treatment combinations might might be enhanced for patients with previously treated lung cancer.

Another study relating to ovarian cancer is in the recruitment phase. The researchers seek to evaluate the vaccine alone or in combination with pembrolizumab for patients with platinum-sensitive ovarian cancer. Results from both trials are expected in 2025.

The third trial seeks to assess FOLFIRI as maintenance therapy or FOLFIRI as maintenance plus Tedopi for patients with pancreatic cancer to improve disease management. Efficacy data are expected next year.

OSE Immunotherapeutics is simultaneously working on a companion biomarker, the HLA-A2 test.

The study was funded by OSE Immunotherapeutics. Dr. Besse disclosed the following conflicts of interest (research funding, institution): AbbVie, Amgen, AstraZeneca, Chugai Pharmaceutical, Daiichi-Sankyo, Ellipse Pharma, EISAI, Genmab, Genzyme Corporation, Hedera Dx, Inivata, IPSEN, Janssen, MSD, Pharmamar, Roche-Genentech, Sanofi, Socar Research, Taiho Oncology, and Turning Point Therapeutics.

This article was translated from the Medscape French Edition and a version appeared on Medscape.com.

SINGAPORE – A few months after releasing its phase 1 and 2 data, OSE Immunotherapeutics, which is based in Nantes, France, has announced positive results for its therapeutic vaccine to treat cancer. Following its promising findings concerning early-stage melanoma, pancreatic cancer, ENT cancers, and HPV-associated anogenital cancer, the company-funded phase 3 Atalante-1 trial has shown the benefits of the Tedopi (OSE2101) vaccine in treating patients with advanced non–small cell lung cancer who are on their second or third line of treatment.

The results suggest that Tedopi is the most developmentally advanced therapeutic vaccine for cancer.

The data from Atalante-1 were presented at the World Conference on Lung Cancer and were simultaneously published in Annals of Oncology.

Tedopi is composed of synthetic tumoral neo-epitopes (peptide fragments) that target five tumoral antigens, permitting the activation of tumor-specific T-lymphocytes for patients who are HLA-A2 positive. In 95% of cases, tumors express at least one of these five antigens. The aim of integrating these five antigens is to prevent immune escape. The technology uses the human leukocyte antigen (HLA) system, one of the keys for presenting antigens to T-lymphocytes. The vaccine is effective for patients who express the HLA-A2 gene, which is present in around half of the population. The HLA-A2 biomarker, detected via a blood test, can identify appropriate patients.
 

Study protocol

In the Atalante-1 trial, participants had locally advanced (unresectable and not eligible for radiotherapy) or metastatic (without alteration of the EGFR and ALK genes) non–small cell lung cancer that was resistant to previous immunotherapy. They had an HLA-A2 phenotype, as determined by a blood draw to determine whether their immune system could respond to the vaccine.

In this trial, 219 patients were randomly assigned in a 2:1 ratio to receive the vaccine or standard-of-care chemotherapy (80% received docetaxel). The vaccine was administered subcutaneously on day 1 every 3 weeks for six cycles. After that point, the vaccine was administered every 8 weeks until 1 year of treatment and every 12 weeks thereafter. The primary endpoint was overall survival.
 

Secondary resistance

The plan was to enroll 363 patients in the protocol, but the study did not complete its recruitment phase because of the COVID-19 pandemic. As a result, the study was stopped after the enrollment of 219 patients.

“It didn’t have the power we would have liked, but it helped us understand that the people who benefited the most from the vaccine were patients who had responded to immunotherapy in the past. These patients have what is called ‘secondary resistance,’ ” explained Benjamin Besse, MD, PhD, during a press conference organized by OSE Immunotherapeutics. Dr. Besse, the study’s principal investigator, is the director of clinical research at Gustave Roussy, Villejuif, France.

Overall, the results weren’t significant. But the results were positive for patients who had previously responded well to immunotherapy for at least 3 months. Of the 219 patients, 118 (54%) had a positive response.

Among these patients with secondary resistance to immunotherapy, median OS was 11.1 months with Tedopi versus 7.5 months with docetaxel.

For these patients, the risk of death was reduced by 41% with the vaccine, compared with chemotherapy. Overall, 44% of patients lived for another year after receiving Tedopi, versus 27.5% with docetaxel.

“This study is a positive signal for overall survival in the selected population. In this study of 219 patients, we realized that just half of patients really benefited from the vaccine: those who had previously responded to immunotherapy,” said Dr. Besse. “The study needs confirmation from a further, larger phase 3 study in more than 300 patients with secondary resistance to immunotherapy to give us the statistical power we need to convince the regulatory authorities.”
 

Tolerability profile

Fewer serious adverse effects were reported with the vaccine than with chemotherapy (11.4% with Tedopi and 35.1% with docetaxel).

The vaccine also allowed patients to maintain a better quality of life. Scores from the Quality of Life Questionnaire Core 30, which explores several areas of daily life, were better with the vaccine. Change in patients’ overall well-being was delayed in the vaccine group: 3.3 months in the chemotherapy arm versus 9 months in the vaccine arm.

“The vaccine was well tolerated. It has benefits in terms of controlling disease symptoms and causes few side effects. Chemotherapy with docetaxel, meanwhile, is more toxic and may affect a patient’s overall well-being. It causes hair loss in practically 100% of patients, induces neuropathy, makes hands and feet swell, damages the nails, is associated with nausea and vomiting ...” noted Dr. Besse. He went on to say that after the trial, of the patients who stopped receiving the vaccine or chemotherapy (either for toxicity reasons or for disease progression), those who had been given the vaccine responded better to the subsequent chemotherapy “because their overall health was better.”
 

Clinical development

The clinical development of Tedopi is ongoing. Three trials are currently taking place. One study is comparing the Tedopi vaccine plus docetaxel with Tedopi plus nivolumab (immunotherapy not used as a first-line treatment) to determine whether the effects of these treatment combinations might might be enhanced for patients with previously treated lung cancer.

Another study relating to ovarian cancer is in the recruitment phase. The researchers seek to evaluate the vaccine alone or in combination with pembrolizumab for patients with platinum-sensitive ovarian cancer. Results from both trials are expected in 2025.

The third trial seeks to assess FOLFIRI as maintenance therapy or FOLFIRI as maintenance plus Tedopi for patients with pancreatic cancer to improve disease management. Efficacy data are expected next year.

OSE Immunotherapeutics is simultaneously working on a companion biomarker, the HLA-A2 test.

The study was funded by OSE Immunotherapeutics. Dr. Besse disclosed the following conflicts of interest (research funding, institution): AbbVie, Amgen, AstraZeneca, Chugai Pharmaceutical, Daiichi-Sankyo, Ellipse Pharma, EISAI, Genmab, Genzyme Corporation, Hedera Dx, Inivata, IPSEN, Janssen, MSD, Pharmamar, Roche-Genentech, Sanofi, Socar Research, Taiho Oncology, and Turning Point Therapeutics.

This article was translated from the Medscape French Edition and a version appeared on Medscape.com.

SINGAPORE – A few months after releasing its phase 1 and 2 data, OSE Immunotherapeutics, which is based in Nantes, France, has announced positive results for its therapeutic vaccine to treat cancer. Following its promising findings concerning early-stage melanoma, pancreatic cancer, ENT cancers, and HPV-associated anogenital cancer, the company-funded phase 3 Atalante-1 trial has shown the benefits of the Tedopi (OSE2101) vaccine in treating patients with advanced non–small cell lung cancer who are on their second or third line of treatment.

The results suggest that Tedopi is the most developmentally advanced therapeutic vaccine for cancer.

The data from Atalante-1 were presented at the World Conference on Lung Cancer and were simultaneously published in Annals of Oncology.

Tedopi is composed of synthetic tumoral neo-epitopes (peptide fragments) that target five tumoral antigens, permitting the activation of tumor-specific T-lymphocytes for patients who are HLA-A2 positive. In 95% of cases, tumors express at least one of these five antigens. The aim of integrating these five antigens is to prevent immune escape. The technology uses the human leukocyte antigen (HLA) system, one of the keys for presenting antigens to T-lymphocytes. The vaccine is effective for patients who express the HLA-A2 gene, which is present in around half of the population. The HLA-A2 biomarker, detected via a blood test, can identify appropriate patients.
 

Study protocol

In the Atalante-1 trial, participants had locally advanced (unresectable and not eligible for radiotherapy) or metastatic (without alteration of the EGFR and ALK genes) non–small cell lung cancer that was resistant to previous immunotherapy. They had an HLA-A2 phenotype, as determined by a blood draw to determine whether their immune system could respond to the vaccine.

In this trial, 219 patients were randomly assigned in a 2:1 ratio to receive the vaccine or standard-of-care chemotherapy (80% received docetaxel). The vaccine was administered subcutaneously on day 1 every 3 weeks for six cycles. After that point, the vaccine was administered every 8 weeks until 1 year of treatment and every 12 weeks thereafter. The primary endpoint was overall survival.
 

Secondary resistance

The plan was to enroll 363 patients in the protocol, but the study did not complete its recruitment phase because of the COVID-19 pandemic. As a result, the study was stopped after the enrollment of 219 patients.

“It didn’t have the power we would have liked, but it helped us understand that the people who benefited the most from the vaccine were patients who had responded to immunotherapy in the past. These patients have what is called ‘secondary resistance,’ ” explained Benjamin Besse, MD, PhD, during a press conference organized by OSE Immunotherapeutics. Dr. Besse, the study’s principal investigator, is the director of clinical research at Gustave Roussy, Villejuif, France.

Overall, the results weren’t significant. But the results were positive for patients who had previously responded well to immunotherapy for at least 3 months. Of the 219 patients, 118 (54%) had a positive response.

Among these patients with secondary resistance to immunotherapy, median OS was 11.1 months with Tedopi versus 7.5 months with docetaxel.

For these patients, the risk of death was reduced by 41% with the vaccine, compared with chemotherapy. Overall, 44% of patients lived for another year after receiving Tedopi, versus 27.5% with docetaxel.

“This study is a positive signal for overall survival in the selected population. In this study of 219 patients, we realized that just half of patients really benefited from the vaccine: those who had previously responded to immunotherapy,” said Dr. Besse. “The study needs confirmation from a further, larger phase 3 study in more than 300 patients with secondary resistance to immunotherapy to give us the statistical power we need to convince the regulatory authorities.”
 

Tolerability profile

Fewer serious adverse effects were reported with the vaccine than with chemotherapy (11.4% with Tedopi and 35.1% with docetaxel).

The vaccine also allowed patients to maintain a better quality of life. Scores from the Quality of Life Questionnaire Core 30, which explores several areas of daily life, were better with the vaccine. Change in patients’ overall well-being was delayed in the vaccine group: 3.3 months in the chemotherapy arm versus 9 months in the vaccine arm.

“The vaccine was well tolerated. It has benefits in terms of controlling disease symptoms and causes few side effects. Chemotherapy with docetaxel, meanwhile, is more toxic and may affect a patient’s overall well-being. It causes hair loss in practically 100% of patients, induces neuropathy, makes hands and feet swell, damages the nails, is associated with nausea and vomiting ...” noted Dr. Besse. He went on to say that after the trial, of the patients who stopped receiving the vaccine or chemotherapy (either for toxicity reasons or for disease progression), those who had been given the vaccine responded better to the subsequent chemotherapy “because their overall health was better.”
 

Clinical development

The clinical development of Tedopi is ongoing. Three trials are currently taking place. One study is comparing the Tedopi vaccine plus docetaxel with Tedopi plus nivolumab (immunotherapy not used as a first-line treatment) to determine whether the effects of these treatment combinations might might be enhanced for patients with previously treated lung cancer.

Another study relating to ovarian cancer is in the recruitment phase. The researchers seek to evaluate the vaccine alone or in combination with pembrolizumab for patients with platinum-sensitive ovarian cancer. Results from both trials are expected in 2025.

The third trial seeks to assess FOLFIRI as maintenance therapy or FOLFIRI as maintenance plus Tedopi for patients with pancreatic cancer to improve disease management. Efficacy data are expected next year.

OSE Immunotherapeutics is simultaneously working on a companion biomarker, the HLA-A2 test.

The study was funded by OSE Immunotherapeutics. Dr. Besse disclosed the following conflicts of interest (research funding, institution): AbbVie, Amgen, AstraZeneca, Chugai Pharmaceutical, Daiichi-Sankyo, Ellipse Pharma, EISAI, Genmab, Genzyme Corporation, Hedera Dx, Inivata, IPSEN, Janssen, MSD, Pharmamar, Roche-Genentech, Sanofi, Socar Research, Taiho Oncology, and Turning Point Therapeutics.

This article was translated from the Medscape French Edition and a version appeared on Medscape.com.

This transcript has been edited for clarity.

Hello. It’s Mark Kris from Memorial Sloan-Kettering, still musing on things I learned at ASCO 2023.

I learned that there is a new C word.

People used to be afraid to use the word “cancer,” so they would call it the C word. Hopefully we’ve gotten over that stigma, that cancer is an illness that can be fought like any other illness.

There’s a new C word now that people seem, again, afraid to use, and that word is “cure.” It’s almost a true rarity that – again, I’m talking about the lung cancer world in particular – folks use the word “cure.” I didn’t hear it at ASCO, but the truth of the matter is that’s a word we should be using and be using more.

What do our patients want? I think if you truly ask a patient what their goal of care should be, it would be to cure the illness. What I mean by “cure” is to eradicate the cancer that is in their body, keep the cancer and its effects from interfering with their ability to continue their lives, and to do it for the length of their natural life. That’s what our patients want. Yes, overall survival is important, but not as much as a life free of cancer and the burden that it puts on people having cancer in the body.

When you start thinking about cure and how to make it a goal of care, a number of issues immediately crop up. The first one is defining what is meant by “cure.” We don’t have a strict definition of cure. Again, I would probably go to the patients and ask them what they mean by it. There may be some landmark part of the definition that needs to be discussed and addressed, but again, to me it’s having your life not disturbed by cancer, and that generally comes by eradicating cancer. Living with cancer is harder than the living after cancer has been cured. But we don’t have a good definition.

We also don’t have a good way of designing clinical trials to assess whether the regimen is curative. I don’t think I’ve ever seen a trial in lung cancer that looked at the ability of any given treatment to cure patients. We need to come up with ways to design trials to do that. Now, in addition to clinical trials, we don’t have a good body of evidence to design our preclinical experiments to look for those treatments that can lead to cures, or total eradication of cancer in whatever model system might be used. If we make cure the goal, then we need to find ways preclinically to identify those strategies that could lead to that.

Also in the realm of clinical trials, we need a very clear statistical underpinning to show that one or another treatment has a better chance of cure and to show with scientific rigor that one treatment is better than the other when it comes to cure. I think there needs to be more attention to this, and as we think about revamping the clinical trial process, we need to focus more on cure.

I’m saving the most important step for last. None of this can happen unless we try to make it happen and we say cure is possible. My mentor, George Boswell, always taught us that we would, in every single patient with cancer, try to develop a curative strategy. Is there a curative strategy for this patient? If so, pursue it with all the tools and vigor that we have. We really need to think that way.

Obviously, not every patient with cancer can be cured with our current armamentarium of anticancer treatments, but we need to make sure we put it on the table. We need to [confirm] that a strategy does not currently exist that could lead to cure. And of course, if we do find that strategy, we need to pursue it with all the energy and resources that we have.

Please don’t be afraid to use the word “cure.” Our patients want that. They deserve it. We should work hard to try to provide it and work toward developing strategies that we can propose and cure more patients.

Mark G. Kris, MD, is chief of the thoracic oncology service and the William and Joy Ruane Chair in Thoracic Oncology at Memorial Sloan Kettering Cancer Center in New York. His research interests include targeted therapies for lung cancer, multimodality therapy, the development of new anticancer drugs, and symptom management with a focus on preventing emesis.

A version of this article first appeared on Medscape.com.

This transcript has been edited for clarity.

Hello. It’s Mark Kris from Memorial Sloan-Kettering, still musing on things I learned at ASCO 2023.

I learned that there is a new C word.

People used to be afraid to use the word “cancer,” so they would call it the C word. Hopefully we’ve gotten over that stigma, that cancer is an illness that can be fought like any other illness.

There’s a new C word now that people seem, again, afraid to use, and that word is “cure.” It’s almost a true rarity that – again, I’m talking about the lung cancer world in particular – folks use the word “cure.” I didn’t hear it at ASCO, but the truth of the matter is that’s a word we should be using and be using more.

What do our patients want? I think if you truly ask a patient what their goal of care should be, it would be to cure the illness. What I mean by “cure” is to eradicate the cancer that is in their body, keep the cancer and its effects from interfering with their ability to continue their lives, and to do it for the length of their natural life. That’s what our patients want. Yes, overall survival is important, but not as much as a life free of cancer and the burden that it puts on people having cancer in the body.

When you start thinking about cure and how to make it a goal of care, a number of issues immediately crop up. The first one is defining what is meant by “cure.” We don’t have a strict definition of cure. Again, I would probably go to the patients and ask them what they mean by it. There may be some landmark part of the definition that needs to be discussed and addressed, but again, to me it’s having your life not disturbed by cancer, and that generally comes by eradicating cancer. Living with cancer is harder than the living after cancer has been cured. But we don’t have a good definition.

We also don’t have a good way of designing clinical trials to assess whether the regimen is curative. I don’t think I’ve ever seen a trial in lung cancer that looked at the ability of any given treatment to cure patients. We need to come up with ways to design trials to do that. Now, in addition to clinical trials, we don’t have a good body of evidence to design our preclinical experiments to look for those treatments that can lead to cures, or total eradication of cancer in whatever model system might be used. If we make cure the goal, then we need to find ways preclinically to identify those strategies that could lead to that.

Also in the realm of clinical trials, we need a very clear statistical underpinning to show that one or another treatment has a better chance of cure and to show with scientific rigor that one treatment is better than the other when it comes to cure. I think there needs to be more attention to this, and as we think about revamping the clinical trial process, we need to focus more on cure.

I’m saving the most important step for last. None of this can happen unless we try to make it happen and we say cure is possible. My mentor, George Boswell, always taught us that we would, in every single patient with cancer, try to develop a curative strategy. Is there a curative strategy for this patient? If so, pursue it with all the tools and vigor that we have. We really need to think that way.

Obviously, not every patient with cancer can be cured with our current armamentarium of anticancer treatments, but we need to make sure we put it on the table. We need to [confirm] that a strategy does not currently exist that could lead to cure. And of course, if we do find that strategy, we need to pursue it with all the energy and resources that we have.

Please don’t be afraid to use the word “cure.” Our patients want that. They deserve it. We should work hard to try to provide it and work toward developing strategies that we can propose and cure more patients.

Mark G. Kris, MD, is chief of the thoracic oncology service and the William and Joy Ruane Chair in Thoracic Oncology at Memorial Sloan Kettering Cancer Center in New York. His research interests include targeted therapies for lung cancer, multimodality therapy, the development of new anticancer drugs, and symptom management with a focus on preventing emesis.

A version of this article first appeared on Medscape.com.

This transcript has been edited for clarity.

Hello. It’s Mark Kris from Memorial Sloan-Kettering, still musing on things I learned at ASCO 2023.

I learned that there is a new C word.

People used to be afraid to use the word “cancer,” so they would call it the C word. Hopefully we’ve gotten over that stigma, that cancer is an illness that can be fought like any other illness.

There’s a new C word now that people seem, again, afraid to use, and that word is “cure.” It’s almost a true rarity that – again, I’m talking about the lung cancer world in particular – folks use the word “cure.” I didn’t hear it at ASCO, but the truth of the matter is that’s a word we should be using and be using more.

What do our patients want? I think if you truly ask a patient what their goal of care should be, it would be to cure the illness. What I mean by “cure” is to eradicate the cancer that is in their body, keep the cancer and its effects from interfering with their ability to continue their lives, and to do it for the length of their natural life. That’s what our patients want. Yes, overall survival is important, but not as much as a life free of cancer and the burden that it puts on people having cancer in the body.

When you start thinking about cure and how to make it a goal of care, a number of issues immediately crop up. The first one is defining what is meant by “cure.” We don’t have a strict definition of cure. Again, I would probably go to the patients and ask them what they mean by it. There may be some landmark part of the definition that needs to be discussed and addressed, but again, to me it’s having your life not disturbed by cancer, and that generally comes by eradicating cancer. Living with cancer is harder than the living after cancer has been cured. But we don’t have a good definition.

We also don’t have a good way of designing clinical trials to assess whether the regimen is curative. I don’t think I’ve ever seen a trial in lung cancer that looked at the ability of any given treatment to cure patients. We need to come up with ways to design trials to do that. Now, in addition to clinical trials, we don’t have a good body of evidence to design our preclinical experiments to look for those treatments that can lead to cures, or total eradication of cancer in whatever model system might be used. If we make cure the goal, then we need to find ways preclinically to identify those strategies that could lead to that.

Also in the realm of clinical trials, we need a very clear statistical underpinning to show that one or another treatment has a better chance of cure and to show with scientific rigor that one treatment is better than the other when it comes to cure. I think there needs to be more attention to this, and as we think about revamping the clinical trial process, we need to focus more on cure.

I’m saving the most important step for last. None of this can happen unless we try to make it happen and we say cure is possible. My mentor, George Boswell, always taught us that we would, in every single patient with cancer, try to develop a curative strategy. Is there a curative strategy for this patient? If so, pursue it with all the tools and vigor that we have. We really need to think that way.

Obviously, not every patient with cancer can be cured with our current armamentarium of anticancer treatments, but we need to make sure we put it on the table. We need to [confirm] that a strategy does not currently exist that could lead to cure. And of course, if we do find that strategy, we need to pursue it with all the energy and resources that we have.

Please don’t be afraid to use the word “cure.” Our patients want that. They deserve it. We should work hard to try to provide it and work toward developing strategies that we can propose and cure more patients.

Mark G. Kris, MD, is chief of the thoracic oncology service and the William and Joy Ruane Chair in Thoracic Oncology at Memorial Sloan Kettering Cancer Center in New York. His research interests include targeted therapies for lung cancer, multimodality therapy, the development of new anticancer drugs, and symptom management with a focus on preventing emesis.

A version of this article first appeared on Medscape.com.

HIV continues to be a significant public health concern in the United States, with an estimated 1.2 million people currently living with the virus and more than 30,000 new diagnoses in 2020 alone.

Primary care clinicians can help decrease rates of HIV infection by prescribing pre-exposure prophylaxis to people who are sexually active.

But many do not.

“In medical school, we don’t spend much time discussing sexuality, sexual behavior, sexually transmitted infections, and such, so providers may feel uncomfortable asking what kind of sex their patient is having and with whom, whether they use a condom, and other basics,” said Matthew M. Hamill, MBChB, PhD, MPH, a specialist in sexually transmitted diseases at Johns Hopkins Medicine, Baltimore.

PrEP (pre-exposure prophylaxis) is an antiviral medication that cuts the risk of contracting HIV through sex by around 99% when taken as prescribed, according to the Centers for Disease Control and Prevention.

“Many people who would benefit from PrEP are not receiving this highly effective medication,” said John B. Wong, MD, a primary care internist and professor of medicine at Tufts University, Boston. The gap is particularly acute among Black, Hispanic, and Latino people, who are significantly more likely to be diagnosed with HIV but are much less likely than Whites to receive PrEP, he said.

Dr. Wong, a member of the U.S. Preventive Services Task Force, helped write the group’s new PrEP recommendations. Published in August, the guidelines call for clinicians to prescribe the drugs to adolescents and adults who do not have HIV but are at an increased risk for infection.

“Primary care physicians are ideally positioned to prescribe PrEP for their patients because they have longitudinal relationships: They get to know their patients, and hopefully their patients feel comfortable talking with them about their sexual health,” said Brandon Pollak, MD, a primary care physician and HIV specialist at the Ohio State University College of Medicine, Columbus.

Dr. Pollak, who was not involved with the USPSTF recommendations, cares for patients who are heterosexual and living with HIV.

Clinicians should consider PrEP for all patients who have sex with someone who has HIV, do not use condoms, or have had a sexually transmitted infection within the previous 6 months. Men who have sex with men, transgender women who have sex with men, people who inject illicit drugs or engage in transactional sex, and Black, Hispanic, and Latino individuals also are at increased risk for the infection.

“The vast majority of patients on PrEP in any form sail through with no problems; they have regular lab work and can follow up in person or by telemedicine,” Dr. Hamill said. “They tend to be young, fit people without complicated medical histories, and the medications are very well-tolerated, particularly if people expect some short-term side effects.”
 

What you need to know when prescribing PrEP

Prescribing PrEP is similar in complexity to prescribing hypertension or diabetes medications, Dr. Hamill said.

Because taking the medications while already infected with the virus can lead to the emergence of drug-resistant HIV, patients must have a negative HIV test before starting PrEP. In addition, the USPSTF recommends testing for other sexually transmitted infections and for pregnancy, if appropriate. The task force also recommends conducting kidney function and hepatitis B tests, and a lipid profile before starting specific types of PrEP.

HIV screening is also recommended at 3-month intervals.

“Providers may order labs done at 3- to 4-month intervals but only see patients in clinic once or twice per year, depending on patient needs and risk behaviors,” said Jill S. Blumenthal, MD, associate professor of medicine at UC San Diego Health.

Clinicians should consider medication adherence and whether a patient is likely to take a pill once a day or could benefit from receiving an injection every 2 months. Patients may experience side effects such as diarrhea or headache with oral PrEP or soreness at the injection site. In rare cases, some of the drugs may cause kidney toxicity or bone mineral loss, according to Dr. Hamill.

Three similarly effective forms of PrEP approved by the U.S. Food and Drug Administration enable clinicians to tailor the medications to the specific needs and preferences of each patient. Truvada (emtricitabine and tenofovir disoproxil fumarate) and Descovy (emtricitabine and tenofovir alafenamide) are both daily tablets, although the latter is not advised for people assigned female sex at birth who have receptive vaginal sex. Apretude (cabotegravir), an injectable agent, is not recommended for people who inject illegal drugs.

Patients with renal or bone disease are not good candidates for Truvada.

“Truvada can decrease bone density, so for someone with osteoporosis, you might choose Descovy or Apretude,” Dr. Pollak said. “For someone with chronic kidney disease, consider Descovy or Apretude. “If a patient has hepatitis B, Truvada or Descovy are appropriate, because hepatitis B is treatable.”

Patients taking an injectable PrEP may need more attention, because the concentration of the medication in the body decreases slowly and may linger for many months at low levels that don’t prevent HIV, according to Dr. Hamill. Someone who acquires HIV during that “tail” period might develop resistance to PrEP.

New research also showed that Descovy users were at elevated risk of developing hypertension and statin initiation, especially among those over age 40 years.

Primary care physicians may want to consult with renal specialists about medication safety in patients with severe kidney disease or with rheumatologists or endocrinologists about metabolic bone disease concerns, Dr. Hamill said.

Meanwhile, if a person begins a monogamous relationship and their risk for HIV drops, “it’s fine to stop taking PrEP tablets,” Dr. Pollak said. “I would still recommend routine HIV screening every 6 or 12 months or however often, depending on other risk factors.”

Caring for these patients entails ensuring labs are completed, monitoring adherence, ordering refills, and scheduling regular follow-up visits.

“For the vast majority of patients, the primary care physician is perfectly equipped for their care through the entire PrEP journey, from discussion and initiation to provision of PrEP,” and most cases do not require specialist care, Dr. Hamill said.

However, “if PrEP fails, which is exceedingly rare, primary care physicians should refer patients immediately, preferably with a warm handoff, for linkage to HIV care,” Dr. Blumenthal said.

Talking about PrEP opens the door to conversations with patients about sexual health and broader health issues, Dr. Hamill said. Although these may not come naturally to primary care clinicians, training is available. The National Network of STD Clinical Prevention Training Centers, funded by the CDC, trains providers on how to overcome their anxiety and have open, inclusive conversations about sexuality and sexual behaviors with transgender and gender-diverse, nonbinary people.

“People worry about saying the wrong thing, about causing offense,” Dr. Hamill said. “But once you get comfortable discussing sexuality, you may open conversations around other health issues.”
 

Barriers for patients

The task force identified several barriers to PrEP access for patients because of lack of trusting relationships with health care, the effects of structural racism on health disparities, and persistent biases within the health care system.

Racial and ethnic disparities in HIV incidence persist, with 42% of new diagnoses occurring among Black people, 27% among Hispanic or Latino people, and 26% among White people in 2020.

Rates of PrEP usage for a year or longer are also low. Sometimes the patient no longer needs PrEP, but barriers often involve the costs of taking time off from work and arranging transportation to clinic visits.

Although nearly all insurance plans and state Medicaid programs cover PrEP, if a patient does not have coverage, the drugs and required tests and office visits can be expensive.

“One of the biggest barriers for all providers is navigating our complicated health system and drug assistance programs,” said Mehri S. McKellar, MD, associate professor of medicine at Duke University School of Medicine, Durham, N.C.

But lower-cost FDA-approved generic emtricitabine/tenofovir disoproxil fumarate is now available, and clinicians can direct patients to programs that help provide the medications at low or no cost.

“Providing PrEP care is straightforward, beneficial, and satisfying,” Dr. Hamill said. “You help people protect themselves from a life-changing diagnosis, and the health system doesn’t need to pay the cost of treating HIV. Everyone wins.”

Dr. Hamill, Dr. McKellar, Dr. Pollak, and Dr. Wong have reported no relevant financial relationships. Dr. Blumenthal has reported a financial relationship with Gilead Sciences.

A version of this article appeared on Medscape.com.

HIV continues to be a significant public health concern in the United States, with an estimated 1.2 million people currently living with the virus and more than 30,000 new diagnoses in 2020 alone.

Primary care clinicians can help decrease rates of HIV infection by prescribing pre-exposure prophylaxis to people who are sexually active.

But many do not.

“In medical school, we don’t spend much time discussing sexuality, sexual behavior, sexually transmitted infections, and such, so providers may feel uncomfortable asking what kind of sex their patient is having and with whom, whether they use a condom, and other basics,” said Matthew M. Hamill, MBChB, PhD, MPH, a specialist in sexually transmitted diseases at Johns Hopkins Medicine, Baltimore.

PrEP (pre-exposure prophylaxis) is an antiviral medication that cuts the risk of contracting HIV through sex by around 99% when taken as prescribed, according to the Centers for Disease Control and Prevention.

“Many people who would benefit from PrEP are not receiving this highly effective medication,” said John B. Wong, MD, a primary care internist and professor of medicine at Tufts University, Boston. The gap is particularly acute among Black, Hispanic, and Latino people, who are significantly more likely to be diagnosed with HIV but are much less likely than Whites to receive PrEP, he said.

Dr. Wong, a member of the U.S. Preventive Services Task Force, helped write the group’s new PrEP recommendations. Published in August, the guidelines call for clinicians to prescribe the drugs to adolescents and adults who do not have HIV but are at an increased risk for infection.

“Primary care physicians are ideally positioned to prescribe PrEP for their patients because they have longitudinal relationships: They get to know their patients, and hopefully their patients feel comfortable talking with them about their sexual health,” said Brandon Pollak, MD, a primary care physician and HIV specialist at the Ohio State University College of Medicine, Columbus.

Dr. Pollak, who was not involved with the USPSTF recommendations, cares for patients who are heterosexual and living with HIV.

Clinicians should consider PrEP for all patients who have sex with someone who has HIV, do not use condoms, or have had a sexually transmitted infection within the previous 6 months. Men who have sex with men, transgender women who have sex with men, people who inject illicit drugs or engage in transactional sex, and Black, Hispanic, and Latino individuals also are at increased risk for the infection.

“The vast majority of patients on PrEP in any form sail through with no problems; they have regular lab work and can follow up in person or by telemedicine,” Dr. Hamill said. “They tend to be young, fit people without complicated medical histories, and the medications are very well-tolerated, particularly if people expect some short-term side effects.”
 

What you need to know when prescribing PrEP

Prescribing PrEP is similar in complexity to prescribing hypertension or diabetes medications, Dr. Hamill said.

Because taking the medications while already infected with the virus can lead to the emergence of drug-resistant HIV, patients must have a negative HIV test before starting PrEP. In addition, the USPSTF recommends testing for other sexually transmitted infections and for pregnancy, if appropriate. The task force also recommends conducting kidney function and hepatitis B tests, and a lipid profile before starting specific types of PrEP.

HIV screening is also recommended at 3-month intervals.

“Providers may order labs done at 3- to 4-month intervals but only see patients in clinic once or twice per year, depending on patient needs and risk behaviors,” said Jill S. Blumenthal, MD, associate professor of medicine at UC San Diego Health.

Clinicians should consider medication adherence and whether a patient is likely to take a pill once a day or could benefit from receiving an injection every 2 months. Patients may experience side effects such as diarrhea or headache with oral PrEP or soreness at the injection site. In rare cases, some of the drugs may cause kidney toxicity or bone mineral loss, according to Dr. Hamill.

Three similarly effective forms of PrEP approved by the U.S. Food and Drug Administration enable clinicians to tailor the medications to the specific needs and preferences of each patient. Truvada (emtricitabine and tenofovir disoproxil fumarate) and Descovy (emtricitabine and tenofovir alafenamide) are both daily tablets, although the latter is not advised for people assigned female sex at birth who have receptive vaginal sex. Apretude (cabotegravir), an injectable agent, is not recommended for people who inject illegal drugs.

Patients with renal or bone disease are not good candidates for Truvada.

“Truvada can decrease bone density, so for someone with osteoporosis, you might choose Descovy or Apretude,” Dr. Pollak said. “For someone with chronic kidney disease, consider Descovy or Apretude. “If a patient has hepatitis B, Truvada or Descovy are appropriate, because hepatitis B is treatable.”

Patients taking an injectable PrEP may need more attention, because the concentration of the medication in the body decreases slowly and may linger for many months at low levels that don’t prevent HIV, according to Dr. Hamill. Someone who acquires HIV during that “tail” period might develop resistance to PrEP.

New research also showed that Descovy users were at elevated risk of developing hypertension and statin initiation, especially among those over age 40 years.

Primary care physicians may want to consult with renal specialists about medication safety in patients with severe kidney disease or with rheumatologists or endocrinologists about metabolic bone disease concerns, Dr. Hamill said.

Meanwhile, if a person begins a monogamous relationship and their risk for HIV drops, “it’s fine to stop taking PrEP tablets,” Dr. Pollak said. “I would still recommend routine HIV screening every 6 or 12 months or however often, depending on other risk factors.”

Caring for these patients entails ensuring labs are completed, monitoring adherence, ordering refills, and scheduling regular follow-up visits.

“For the vast majority of patients, the primary care physician is perfectly equipped for their care through the entire PrEP journey, from discussion and initiation to provision of PrEP,” and most cases do not require specialist care, Dr. Hamill said.

However, “if PrEP fails, which is exceedingly rare, primary care physicians should refer patients immediately, preferably with a warm handoff, for linkage to HIV care,” Dr. Blumenthal said.

Talking about PrEP opens the door to conversations with patients about sexual health and broader health issues, Dr. Hamill said. Although these may not come naturally to primary care clinicians, training is available. The National Network of STD Clinical Prevention Training Centers, funded by the CDC, trains providers on how to overcome their anxiety and have open, inclusive conversations about sexuality and sexual behaviors with transgender and gender-diverse, nonbinary people.

“People worry about saying the wrong thing, about causing offense,” Dr. Hamill said. “But once you get comfortable discussing sexuality, you may open conversations around other health issues.”
 

Barriers for patients

The task force identified several barriers to PrEP access for patients because of lack of trusting relationships with health care, the effects of structural racism on health disparities, and persistent biases within the health care system.

Racial and ethnic disparities in HIV incidence persist, with 42% of new diagnoses occurring among Black people, 27% among Hispanic or Latino people, and 26% among White people in 2020.

Rates of PrEP usage for a year or longer are also low. Sometimes the patient no longer needs PrEP, but barriers often involve the costs of taking time off from work and arranging transportation to clinic visits.

Although nearly all insurance plans and state Medicaid programs cover PrEP, if a patient does not have coverage, the drugs and required tests and office visits can be expensive.

“One of the biggest barriers for all providers is navigating our complicated health system and drug assistance programs,” said Mehri S. McKellar, MD, associate professor of medicine at Duke University School of Medicine, Durham, N.C.

But lower-cost FDA-approved generic emtricitabine/tenofovir disoproxil fumarate is now available, and clinicians can direct patients to programs that help provide the medications at low or no cost.

“Providing PrEP care is straightforward, beneficial, and satisfying,” Dr. Hamill said. “You help people protect themselves from a life-changing diagnosis, and the health system doesn’t need to pay the cost of treating HIV. Everyone wins.”

Dr. Hamill, Dr. McKellar, Dr. Pollak, and Dr. Wong have reported no relevant financial relationships. Dr. Blumenthal has reported a financial relationship with Gilead Sciences.

A version of this article appeared on Medscape.com.

HIV continues to be a significant public health concern in the United States, with an estimated 1.2 million people currently living with the virus and more than 30,000 new diagnoses in 2020 alone.

Primary care clinicians can help decrease rates of HIV infection by prescribing pre-exposure prophylaxis to people who are sexually active.

But many do not.

“In medical school, we don’t spend much time discussing sexuality, sexual behavior, sexually transmitted infections, and such, so providers may feel uncomfortable asking what kind of sex their patient is having and with whom, whether they use a condom, and other basics,” said Matthew M. Hamill, MBChB, PhD, MPH, a specialist in sexually transmitted diseases at Johns Hopkins Medicine, Baltimore.

PrEP (pre-exposure prophylaxis) is an antiviral medication that cuts the risk of contracting HIV through sex by around 99% when taken as prescribed, according to the Centers for Disease Control and Prevention.

“Many people who would benefit from PrEP are not receiving this highly effective medication,” said John B. Wong, MD, a primary care internist and professor of medicine at Tufts University, Boston. The gap is particularly acute among Black, Hispanic, and Latino people, who are significantly more likely to be diagnosed with HIV but are much less likely than Whites to receive PrEP, he said.

Dr. Wong, a member of the U.S. Preventive Services Task Force, helped write the group’s new PrEP recommendations. Published in August, the guidelines call for clinicians to prescribe the drugs to adolescents and adults who do not have HIV but are at an increased risk for infection.

“Primary care physicians are ideally positioned to prescribe PrEP for their patients because they have longitudinal relationships: They get to know their patients, and hopefully their patients feel comfortable talking with them about their sexual health,” said Brandon Pollak, MD, a primary care physician and HIV specialist at the Ohio State University College of Medicine, Columbus.

Dr. Pollak, who was not involved with the USPSTF recommendations, cares for patients who are heterosexual and living with HIV.

Clinicians should consider PrEP for all patients who have sex with someone who has HIV, do not use condoms, or have had a sexually transmitted infection within the previous 6 months. Men who have sex with men, transgender women who have sex with men, people who inject illicit drugs or engage in transactional sex, and Black, Hispanic, and Latino individuals also are at increased risk for the infection.

“The vast majority of patients on PrEP in any form sail through with no problems; they have regular lab work and can follow up in person or by telemedicine,” Dr. Hamill said. “They tend to be young, fit people without complicated medical histories, and the medications are very well-tolerated, particularly if people expect some short-term side effects.”
 

What you need to know when prescribing PrEP

Prescribing PrEP is similar in complexity to prescribing hypertension or diabetes medications, Dr. Hamill said.

Because taking the medications while already infected with the virus can lead to the emergence of drug-resistant HIV, patients must have a negative HIV test before starting PrEP. In addition, the USPSTF recommends testing for other sexually transmitted infections and for pregnancy, if appropriate. The task force also recommends conducting kidney function and hepatitis B tests, and a lipid profile before starting specific types of PrEP.

HIV screening is also recommended at 3-month intervals.

“Providers may order labs done at 3- to 4-month intervals but only see patients in clinic once or twice per year, depending on patient needs and risk behaviors,” said Jill S. Blumenthal, MD, associate professor of medicine at UC San Diego Health.

Clinicians should consider medication adherence and whether a patient is likely to take a pill once a day or could benefit from receiving an injection every 2 months. Patients may experience side effects such as diarrhea or headache with oral PrEP or soreness at the injection site. In rare cases, some of the drugs may cause kidney toxicity or bone mineral loss, according to Dr. Hamill.

Three similarly effective forms of PrEP approved by the U.S. Food and Drug Administration enable clinicians to tailor the medications to the specific needs and preferences of each patient. Truvada (emtricitabine and tenofovir disoproxil fumarate) and Descovy (emtricitabine and tenofovir alafenamide) are both daily tablets, although the latter is not advised for people assigned female sex at birth who have receptive vaginal sex. Apretude (cabotegravir), an injectable agent, is not recommended for people who inject illegal drugs.

Patients with renal or bone disease are not good candidates for Truvada.

“Truvada can decrease bone density, so for someone with osteoporosis, you might choose Descovy or Apretude,” Dr. Pollak said. “For someone with chronic kidney disease, consider Descovy or Apretude. “If a patient has hepatitis B, Truvada or Descovy are appropriate, because hepatitis B is treatable.”

Patients taking an injectable PrEP may need more attention, because the concentration of the medication in the body decreases slowly and may linger for many months at low levels that don’t prevent HIV, according to Dr. Hamill. Someone who acquires HIV during that “tail” period might develop resistance to PrEP.

New research also showed that Descovy users were at elevated risk of developing hypertension and statin initiation, especially among those over age 40 years.

Primary care physicians may want to consult with renal specialists about medication safety in patients with severe kidney disease or with rheumatologists or endocrinologists about metabolic bone disease concerns, Dr. Hamill said.

Meanwhile, if a person begins a monogamous relationship and their risk for HIV drops, “it’s fine to stop taking PrEP tablets,” Dr. Pollak said. “I would still recommend routine HIV screening every 6 or 12 months or however often, depending on other risk factors.”

Caring for these patients entails ensuring labs are completed, monitoring adherence, ordering refills, and scheduling regular follow-up visits.

“For the vast majority of patients, the primary care physician is perfectly equipped for their care through the entire PrEP journey, from discussion and initiation to provision of PrEP,” and most cases do not require specialist care, Dr. Hamill said.

However, “if PrEP fails, which is exceedingly rare, primary care physicians should refer patients immediately, preferably with a warm handoff, for linkage to HIV care,” Dr. Blumenthal said.

Talking about PrEP opens the door to conversations with patients about sexual health and broader health issues, Dr. Hamill said. Although these may not come naturally to primary care clinicians, training is available. The National Network of STD Clinical Prevention Training Centers, funded by the CDC, trains providers on how to overcome their anxiety and have open, inclusive conversations about sexuality and sexual behaviors with transgender and gender-diverse, nonbinary people.

“People worry about saying the wrong thing, about causing offense,” Dr. Hamill said. “But once you get comfortable discussing sexuality, you may open conversations around other health issues.”
 

Barriers for patients

The task force identified several barriers to PrEP access for patients because of lack of trusting relationships with health care, the effects of structural racism on health disparities, and persistent biases within the health care system.

Racial and ethnic disparities in HIV incidence persist, with 42% of new diagnoses occurring among Black people, 27% among Hispanic or Latino people, and 26% among White people in 2020.

Rates of PrEP usage for a year or longer are also low. Sometimes the patient no longer needs PrEP, but barriers often involve the costs of taking time off from work and arranging transportation to clinic visits.

Although nearly all insurance plans and state Medicaid programs cover PrEP, if a patient does not have coverage, the drugs and required tests and office visits can be expensive.

“One of the biggest barriers for all providers is navigating our complicated health system and drug assistance programs,” said Mehri S. McKellar, MD, associate professor of medicine at Duke University School of Medicine, Durham, N.C.

But lower-cost FDA-approved generic emtricitabine/tenofovir disoproxil fumarate is now available, and clinicians can direct patients to programs that help provide the medications at low or no cost.

“Providing PrEP care is straightforward, beneficial, and satisfying,” Dr. Hamill said. “You help people protect themselves from a life-changing diagnosis, and the health system doesn’t need to pay the cost of treating HIV. Everyone wins.”

Dr. Hamill, Dr. McKellar, Dr. Pollak, and Dr. Wong have reported no relevant financial relationships. Dr. Blumenthal has reported a financial relationship with Gilead Sciences.

A version of this article appeared on Medscape.com.

Editor’s note: As of this writing, the following proposed health insurance policy from Blue Cross and Blue Shield of North Carolina is still active. The Coalition of State Rheumatology Organizations and other rheumatology advocacy groups are in ongoing discussions with the health insurer and hope to have major changes to this policy implemented.

While AI has been in our world for years, it is expanding by the minute, perhaps by the nanosecond, within the health care sector. The $6.7 billion dollar health care AI market in 2020 is expected to climb to more than $120 billion by 2028. There are many questions regarding the application of AI in our world. Is it a mere instructional algorithm that computes things in a much faster way, or does it create a new story based on the information it has access to? Does it engender excitement or fear ... or both? Remember HAL? As we have seen throughout history with new inventions and technologies, there are risks and rewards. Even the best can have harmful unintended consequences. AI is no different, particularly when it comes to health care. In this case, AI can get a bad name if it is utilized along with biased data input and bad policy.

Shared savings

Here is where “shared savings” comes into play. A shared savings program starts with a baseline cost analysis of a particular care plan and then tracks costs (performance) going forward after certain changes to the original care plan are instituted. If savings are accrued when compared with baseline spending, those savings are shared with the providers of the care. Depending on how the shared savings program is implemented, the optics can be very bad if it appears as though physicians are being paid to reduce care.

‘The volunteer opportunity’

Recently, Blue Cross and Blue Shield of North Carolina, in partnership with Outcomes Matter Innovations, a data analysis company that uses AI/machine-learning technology, offered rheumatologists a new voluntary shared savings, value-based care (VBC) “opportunity.” Rheumatologists would be able to “utilize a web-based machine-learning technology platform that suggests evidence-based care pathways” in the treatment of rheumatoid arthritis and psoriatic arthritis (PsA). The VBC/shared savings model uses the AI platform to propose two different pathways. One model would delay the start of biologics or Janus kinase inhibitors (JAKi), and the second model would taper and/or stop biologics or JAKi altogether.

Delaying the start of biologics/JAKi would be achieved through “methotrexate optimization” and/or the use of triple therapy with methotrexate, sulfasalazine, and hydroxychloroquine. The other model would recommend tapering biologic/JAKi dosing in patients in remission or low disease activity and might even suggest a “medication holiday.”

The intention of this 3-year VBC/shared savings program is to reduce costs and create savings by reducing the use of biologics or JAKi. A tangential question might be, “Reduce costs and create savings for whom?” Apparently, the patients will not reap any of the cost savings, as this is proposed to be a shared savings program with the savings going to the physicians and the insurance company. Perhaps the idea is that patients will benefit by reducing unneeded expensive medications.
 

How will it work?

A cost baseline will be established on biologic and JAKi use prior to the start of the program. Once started, there will be a calculation of savings based on biologic/JAKi use going forward. It was stated that physicians would receive 22% of the total costs saved. In one flyer, it was estimated that, with methotrexate optimization, rheumatologists could be paid an average of $1,527 a month per patient per month of delay before starting a biologic or JAKi.

The American College of Rheumatology has guidelines for the treatment of RA and PsA, and while optimizing methotrexate and triple therapy is mentioned, tapering or stopping treatment with biologics or JAKi is not. Additionally, after lack of response at 3 months, the standard of care is to change to a more effective treatment, which for most patients is a biologic disease-modifying antirheumatic drug (DMARD). It could be construed that rheumatologists are being monetarily incentivized to reduce the use of expensive medications through ways that are not included in ACR guidelines and are not standard of care.

What if after the medication holiday the patient cannot recapture control of their disease? Is there a liability concern? Remember, there is no institutional review board or informed patient consent for this VBC data gathering model.

How will a patient feel knowing that their physician was paid to withhold care, or even worse, if a patient is not told of this and then finds out later? Not only are the optics for this suboptimal (at best), where does the liability fall if the patient does not do well and it comes out that their rheumatologist was paid to reduce the care, particularly in a way that is not supported in the guideline. Clearly, this appears to be a clinical study without an institutional review board and without patient consent.

There are also the data that are collected from this voluntary “opportunity.” A valid question would be, “What kind of data will this produce if rheumatologists are paid to delay, reduce, or stop the use of biologics/JAKi?” Is it possible that physicians may subconsciously delay putting patients on a biologic and taper more rapidly because of the reimbursement? This could lead to faulty, biased, AI-generated data that erroneously show this type of care is working. It would not be unheard of to wonder whether this once-voluntary opportunity might evolve into mandatory policy because now, they have “data to prove it.” … only this time there is no shared savings.
 

Low disease activity results in long-term savings

This is not meant to be an indictment of AI in health care, value-based care, or shared savings programs. In reality, AI had very little to do with how poorly this program was presented. Hopefully, it will bring about further discussions on how to achieve savings without sacrificing care. In fact, optimal care in RA and PsA is probably one of the best ways to save money in the long run. Nowhere in this program is there any mention of the high cost associated with uncontrolled disease activity in patients with RA or PsA. The downstream costs can be enormous when long- and short-term sequelae are taken into consideration: joint replacements, cardiovascular disease, certain kinds of malignancies, and all the side effects of increased steroid usage are just a few of the consequences we see with uncontrolled disease activity. It is only recently that we have been able to achieve low disease activity and remission in our patients. The rush to get patients off these medications is not the answer to achieving long-term savings. In addition to the very bad optics of paying rheumatologists to delay, taper, or stop using expensive mediations in their patients, the ultimate data achieved will be biased, and the only real winner will be the health insurance company.

Again, AI machine-learning and shared saving programs are not the guilty parties here. In fact, AI may be helpful in coming up with solutions to long-term health care costs, whether in the realm of economics or scientific research. CSRO and our state member organizations continue to educate the health insurance company on the significant drawbacks to this “volunteer opportunity.” Let’s hope a more reasonable program is put forward with AI-generated data that can be trusted. Hopefully not with a platform named “HAL,” for those of you old enough to remember “2001: A Space Odyssey.”

Dr. Feldman is a rheumatologist in private practice with The Rheumatology Group in New Orleans. She is the CSRO’s vice president of advocacy and government affairs and its immediate past president, as well as past chair of the Alliance for Safe Biologic Medicines and a past member of the American College of Rheumatology insurance subcommittee. You can reach her at [email protected].

Editor’s note: As of this writing, the following proposed health insurance policy from Blue Cross and Blue Shield of North Carolina is still active. The Coalition of State Rheumatology Organizations and other rheumatology advocacy groups are in ongoing discussions with the health insurer and hope to have major changes to this policy implemented.

While AI has been in our world for years, it is expanding by the minute, perhaps by the nanosecond, within the health care sector. The $6.7 billion dollar health care AI market in 2020 is expected to climb to more than $120 billion by 2028. There are many questions regarding the application of AI in our world. Is it a mere instructional algorithm that computes things in a much faster way, or does it create a new story based on the information it has access to? Does it engender excitement or fear ... or both? Remember HAL? As we have seen throughout history with new inventions and technologies, there are risks and rewards. Even the best can have harmful unintended consequences. AI is no different, particularly when it comes to health care. In this case, AI can get a bad name if it is utilized along with biased data input and bad policy.

Shared savings

Here is where “shared savings” comes into play. A shared savings program starts with a baseline cost analysis of a particular care plan and then tracks costs (performance) going forward after certain changes to the original care plan are instituted. If savings are accrued when compared with baseline spending, those savings are shared with the providers of the care. Depending on how the shared savings program is implemented, the optics can be very bad if it appears as though physicians are being paid to reduce care.

‘The volunteer opportunity’

Recently, Blue Cross and Blue Shield of North Carolina, in partnership with Outcomes Matter Innovations, a data analysis company that uses AI/machine-learning technology, offered rheumatologists a new voluntary shared savings, value-based care (VBC) “opportunity.” Rheumatologists would be able to “utilize a web-based machine-learning technology platform that suggests evidence-based care pathways” in the treatment of rheumatoid arthritis and psoriatic arthritis (PsA). The VBC/shared savings model uses the AI platform to propose two different pathways. One model would delay the start of biologics or Janus kinase inhibitors (JAKi), and the second model would taper and/or stop biologics or JAKi altogether.

Delaying the start of biologics/JAKi would be achieved through “methotrexate optimization” and/or the use of triple therapy with methotrexate, sulfasalazine, and hydroxychloroquine. The other model would recommend tapering biologic/JAKi dosing in patients in remission or low disease activity and might even suggest a “medication holiday.”

The intention of this 3-year VBC/shared savings program is to reduce costs and create savings by reducing the use of biologics or JAKi. A tangential question might be, “Reduce costs and create savings for whom?” Apparently, the patients will not reap any of the cost savings, as this is proposed to be a shared savings program with the savings going to the physicians and the insurance company. Perhaps the idea is that patients will benefit by reducing unneeded expensive medications.
 

How will it work?

A cost baseline will be established on biologic and JAKi use prior to the start of the program. Once started, there will be a calculation of savings based on biologic/JAKi use going forward. It was stated that physicians would receive 22% of the total costs saved. In one flyer, it was estimated that, with methotrexate optimization, rheumatologists could be paid an average of $1,527 a month per patient per month of delay before starting a biologic or JAKi.

The American College of Rheumatology has guidelines for the treatment of RA and PsA, and while optimizing methotrexate and triple therapy is mentioned, tapering or stopping treatment with biologics or JAKi is not. Additionally, after lack of response at 3 months, the standard of care is to change to a more effective treatment, which for most patients is a biologic disease-modifying antirheumatic drug (DMARD). It could be construed that rheumatologists are being monetarily incentivized to reduce the use of expensive medications through ways that are not included in ACR guidelines and are not standard of care.

What if after the medication holiday the patient cannot recapture control of their disease? Is there a liability concern? Remember, there is no institutional review board or informed patient consent for this VBC data gathering model.

How will a patient feel knowing that their physician was paid to withhold care, or even worse, if a patient is not told of this and then finds out later? Not only are the optics for this suboptimal (at best), where does the liability fall if the patient does not do well and it comes out that their rheumatologist was paid to reduce the care, particularly in a way that is not supported in the guideline. Clearly, this appears to be a clinical study without an institutional review board and without patient consent.

There are also the data that are collected from this voluntary “opportunity.” A valid question would be, “What kind of data will this produce if rheumatologists are paid to delay, reduce, or stop the use of biologics/JAKi?” Is it possible that physicians may subconsciously delay putting patients on a biologic and taper more rapidly because of the reimbursement? This could lead to faulty, biased, AI-generated data that erroneously show this type of care is working. It would not be unheard of to wonder whether this once-voluntary opportunity might evolve into mandatory policy because now, they have “data to prove it.” … only this time there is no shared savings.
 

Low disease activity results in long-term savings

This is not meant to be an indictment of AI in health care, value-based care, or shared savings programs. In reality, AI had very little to do with how poorly this program was presented. Hopefully, it will bring about further discussions on how to achieve savings without sacrificing care. In fact, optimal care in RA and PsA is probably one of the best ways to save money in the long run. Nowhere in this program is there any mention of the high cost associated with uncontrolled disease activity in patients with RA or PsA. The downstream costs can be enormous when long- and short-term sequelae are taken into consideration: joint replacements, cardiovascular disease, certain kinds of malignancies, and all the side effects of increased steroid usage are just a few of the consequences we see with uncontrolled disease activity. It is only recently that we have been able to achieve low disease activity and remission in our patients. The rush to get patients off these medications is not the answer to achieving long-term savings. In addition to the very bad optics of paying rheumatologists to delay, taper, or stop using expensive mediations in their patients, the ultimate data achieved will be biased, and the only real winner will be the health insurance company.

Again, AI machine-learning and shared saving programs are not the guilty parties here. In fact, AI may be helpful in coming up with solutions to long-term health care costs, whether in the realm of economics or scientific research. CSRO and our state member organizations continue to educate the health insurance company on the significant drawbacks to this “volunteer opportunity.” Let’s hope a more reasonable program is put forward with AI-generated data that can be trusted. Hopefully not with a platform named “HAL,” for those of you old enough to remember “2001: A Space Odyssey.”

Dr. Feldman is a rheumatologist in private practice with The Rheumatology Group in New Orleans. She is the CSRO’s vice president of advocacy and government affairs and its immediate past president, as well as past chair of the Alliance for Safe Biologic Medicines and a past member of the American College of Rheumatology insurance subcommittee. You can reach her at [email protected].

Editor’s note: As of this writing, the following proposed health insurance policy from Blue Cross and Blue Shield of North Carolina is still active. The Coalition of State Rheumatology Organizations and other rheumatology advocacy groups are in ongoing discussions with the health insurer and hope to have major changes to this policy implemented.

While AI has been in our world for years, it is expanding by the minute, perhaps by the nanosecond, within the health care sector. The $6.7 billion dollar health care AI market in 2020 is expected to climb to more than $120 billion by 2028. There are many questions regarding the application of AI in our world. Is it a mere instructional algorithm that computes things in a much faster way, or does it create a new story based on the information it has access to? Does it engender excitement or fear ... or both? Remember HAL? As we have seen throughout history with new inventions and technologies, there are risks and rewards. Even the best can have harmful unintended consequences. AI is no different, particularly when it comes to health care. In this case, AI can get a bad name if it is utilized along with biased data input and bad policy.

Shared savings

Here is where “shared savings” comes into play. A shared savings program starts with a baseline cost analysis of a particular care plan and then tracks costs (performance) going forward after certain changes to the original care plan are instituted. If savings are accrued when compared with baseline spending, those savings are shared with the providers of the care. Depending on how the shared savings program is implemented, the optics can be very bad if it appears as though physicians are being paid to reduce care.

‘The volunteer opportunity’

Recently, Blue Cross and Blue Shield of North Carolina, in partnership with Outcomes Matter Innovations, a data analysis company that uses AI/machine-learning technology, offered rheumatologists a new voluntary shared savings, value-based care (VBC) “opportunity.” Rheumatologists would be able to “utilize a web-based machine-learning technology platform that suggests evidence-based care pathways” in the treatment of rheumatoid arthritis and psoriatic arthritis (PsA). The VBC/shared savings model uses the AI platform to propose two different pathways. One model would delay the start of biologics or Janus kinase inhibitors (JAKi), and the second model would taper and/or stop biologics or JAKi altogether.

Delaying the start of biologics/JAKi would be achieved through “methotrexate optimization” and/or the use of triple therapy with methotrexate, sulfasalazine, and hydroxychloroquine. The other model would recommend tapering biologic/JAKi dosing in patients in remission or low disease activity and might even suggest a “medication holiday.”

The intention of this 3-year VBC/shared savings program is to reduce costs and create savings by reducing the use of biologics or JAKi. A tangential question might be, “Reduce costs and create savings for whom?” Apparently, the patients will not reap any of the cost savings, as this is proposed to be a shared savings program with the savings going to the physicians and the insurance company. Perhaps the idea is that patients will benefit by reducing unneeded expensive medications.
 

How will it work?

A cost baseline will be established on biologic and JAKi use prior to the start of the program. Once started, there will be a calculation of savings based on biologic/JAKi use going forward. It was stated that physicians would receive 22% of the total costs saved. In one flyer, it was estimated that, with methotrexate optimization, rheumatologists could be paid an average of $1,527 a month per patient per month of delay before starting a biologic or JAKi.

The American College of Rheumatology has guidelines for the treatment of RA and PsA, and while optimizing methotrexate and triple therapy is mentioned, tapering or stopping treatment with biologics or JAKi is not. Additionally, after lack of response at 3 months, the standard of care is to change to a more effective treatment, which for most patients is a biologic disease-modifying antirheumatic drug (DMARD). It could be construed that rheumatologists are being monetarily incentivized to reduce the use of expensive medications through ways that are not included in ACR guidelines and are not standard of care.

What if after the medication holiday the patient cannot recapture control of their disease? Is there a liability concern? Remember, there is no institutional review board or informed patient consent for this VBC data gathering model.

How will a patient feel knowing that their physician was paid to withhold care, or even worse, if a patient is not told of this and then finds out later? Not only are the optics for this suboptimal (at best), where does the liability fall if the patient does not do well and it comes out that their rheumatologist was paid to reduce the care, particularly in a way that is not supported in the guideline. Clearly, this appears to be a clinical study without an institutional review board and without patient consent.

There are also the data that are collected from this voluntary “opportunity.” A valid question would be, “What kind of data will this produce if rheumatologists are paid to delay, reduce, or stop the use of biologics/JAKi?” Is it possible that physicians may subconsciously delay putting patients on a biologic and taper more rapidly because of the reimbursement? This could lead to faulty, biased, AI-generated data that erroneously show this type of care is working. It would not be unheard of to wonder whether this once-voluntary opportunity might evolve into mandatory policy because now, they have “data to prove it.” … only this time there is no shared savings.
 

Low disease activity results in long-term savings

This is not meant to be an indictment of AI in health care, value-based care, or shared savings programs. In reality, AI had very little to do with how poorly this program was presented. Hopefully, it will bring about further discussions on how to achieve savings without sacrificing care. In fact, optimal care in RA and PsA is probably one of the best ways to save money in the long run. Nowhere in this program is there any mention of the high cost associated with uncontrolled disease activity in patients with RA or PsA. The downstream costs can be enormous when long- and short-term sequelae are taken into consideration: joint replacements, cardiovascular disease, certain kinds of malignancies, and all the side effects of increased steroid usage are just a few of the consequences we see with uncontrolled disease activity. It is only recently that we have been able to achieve low disease activity and remission in our patients. The rush to get patients off these medications is not the answer to achieving long-term savings. In addition to the very bad optics of paying rheumatologists to delay, taper, or stop using expensive mediations in their patients, the ultimate data achieved will be biased, and the only real winner will be the health insurance company.

Again, AI machine-learning and shared saving programs are not the guilty parties here. In fact, AI may be helpful in coming up with solutions to long-term health care costs, whether in the realm of economics or scientific research. CSRO and our state member organizations continue to educate the health insurance company on the significant drawbacks to this “volunteer opportunity.” Let’s hope a more reasonable program is put forward with AI-generated data that can be trusted. Hopefully not with a platform named “HAL,” for those of you old enough to remember “2001: A Space Odyssey.”

Dr. Feldman is a rheumatologist in private practice with The Rheumatology Group in New Orleans. She is the CSRO’s vice president of advocacy and government affairs and its immediate past president, as well as past chair of the Alliance for Safe Biologic Medicines and a past member of the American College of Rheumatology insurance subcommittee. You can reach her at [email protected].

TOPLINE:

Young adults who simultaneously use alcohol and marijuana (SAM) consume more drinks, are high for more hours in the day, and report more negative alcohol-related consequences.

METHODOLOGY:

• The 2-year study included 409 people aged 18-25 years with a history of simultaneous alcohol and marijuana use (50.9% were women; 48.2% were non-Hispanic White; 48.9% were college students).
• Participants completed daily online surveys about substance use and negative substance-related consequences for 14 continuous days every 4 months.

TAKEAWAY:

• Alcohol use was reported on 36.1% of survey days, marijuana use on 28.0%, and alcohol and marijuana use on 15.0%.
• Negative substance-related consequences were reported on 28.0% of drinking days and 56.4% of marijuana days.
• SAM use was reported in 81.7% of alcohol users and 86.6% of marijuana users.
• On SAM use days, participants consumed an average of 37% more drinks, with 43% more negative alcohol consequences; were high for 10% more hours; and were more likely to feel clumsy or dizzy, compared with non-SAM use days.

IN PRACTICE:

“This finding should be integrated into psychoeducational programs highlighting the risk of combining alcohol and marijuana,” the authors write. “A more nuanced harm-reduction [approach] could also encourage young adults to closely monitor and limit the amount of each substance being used if they choose to combine substances.”

SOURCE:

The study was conducted by Anne M. Fairlie, PhD, University of Washington, Seattle, and colleagues, and funded by the National Institute on Alcohol Abuse and Alcoholism. The study was published online in Alcohol Clinical and Experimental Research.

LIMITATIONS:

Study participants were recruited based on their substance use and lived in a region where recreational marijuana is legal, so the findings may not be generalizable to other populations. Substance use and consequences were self-reported and subject to bias.

DISCLOSURES:

The authors have reported no relevant financial relationships.

A version of this article first appeared on Medscape.com.

TOPLINE:

Young adults who simultaneously use alcohol and marijuana (SAM) consume more drinks, are high for more hours in the day, and report more negative alcohol-related consequences.

METHODOLOGY:

• The 2-year study included 409 people aged 18-25 years with a history of simultaneous alcohol and marijuana use (50.9% were women; 48.2% were non-Hispanic White; 48.9% were college students).
• Participants completed daily online surveys about substance use and negative substance-related consequences for 14 continuous days every 4 months.

TAKEAWAY:

• Alcohol use was reported on 36.1% of survey days, marijuana use on 28.0%, and alcohol and marijuana use on 15.0%.
• Negative substance-related consequences were reported on 28.0% of drinking days and 56.4% of marijuana days.
• SAM use was reported in 81.7% of alcohol users and 86.6% of marijuana users.
• On SAM use days, participants consumed an average of 37% more drinks, with 43% more negative alcohol consequences; were high for 10% more hours; and were more likely to feel clumsy or dizzy, compared with non-SAM use days.

IN PRACTICE:

“This finding should be integrated into psychoeducational programs highlighting the risk of combining alcohol and marijuana,” the authors write. “A more nuanced harm-reduction [approach] could also encourage young adults to closely monitor and limit the amount of each substance being used if they choose to combine substances.”

SOURCE:

The study was conducted by Anne M. Fairlie, PhD, University of Washington, Seattle, and colleagues, and funded by the National Institute on Alcohol Abuse and Alcoholism. The study was published online in Alcohol Clinical and Experimental Research.

LIMITATIONS:

Study participants were recruited based on their substance use and lived in a region where recreational marijuana is legal, so the findings may not be generalizable to other populations. Substance use and consequences were self-reported and subject to bias.

DISCLOSURES:

The authors have reported no relevant financial relationships.

A version of this article first appeared on Medscape.com.

TOPLINE:

Young adults who simultaneously use alcohol and marijuana (SAM) consume more drinks, are high for more hours in the day, and report more negative alcohol-related consequences.

METHODOLOGY:

• The 2-year study included 409 people aged 18-25 years with a history of simultaneous alcohol and marijuana use (50.9% were women; 48.2% were non-Hispanic White; 48.9% were college students).
• Participants completed daily online surveys about substance use and negative substance-related consequences for 14 continuous days every 4 months.

TAKEAWAY:

• Alcohol use was reported on 36.1% of survey days, marijuana use on 28.0%, and alcohol and marijuana use on 15.0%.
• Negative substance-related consequences were reported on 28.0% of drinking days and 56.4% of marijuana days.
• SAM use was reported in 81.7% of alcohol users and 86.6% of marijuana users.
• On SAM use days, participants consumed an average of 37% more drinks, with 43% more negative alcohol consequences; were high for 10% more hours; and were more likely to feel clumsy or dizzy, compared with non-SAM use days.

IN PRACTICE:

“This finding should be integrated into psychoeducational programs highlighting the risk of combining alcohol and marijuana,” the authors write. “A more nuanced harm-reduction [approach] could also encourage young adults to closely monitor and limit the amount of each substance being used if they choose to combine substances.”

SOURCE:

The study was conducted by Anne M. Fairlie, PhD, University of Washington, Seattle, and colleagues, and funded by the National Institute on Alcohol Abuse and Alcoholism. The study was published online in Alcohol Clinical and Experimental Research.

LIMITATIONS:

Study participants were recruited based on their substance use and lived in a region where recreational marijuana is legal, so the findings may not be generalizable to other populations. Substance use and consequences were self-reported and subject to bias.

DISCLOSURES:

The authors have reported no relevant financial relationships.

A version of this article first appeared on Medscape.com.

A patient inquires about whether he or she can use an EpiPen after the expiration date. What should you advise?

A. The EpiPen is unlikely to be effective after the expiration date.

B. The EpiPen may be dangerous to use after the expiration date.

C. The EpiPen is likely to be okay up to 2 years past the expiration date.

I think that choice C is the most accurate and will get to all the evidence shortly. The expiration date is not the date that the drug stops being effective or potentially becomes toxic. It is a date, required by law, that the manufacturer can guarantee greater than 90% original potency of the medication.

Epinephrine is a costly drug and is usually replaced when the Epipen expires. Weir and colleagues studied six epinephrine syringes 30 months past their expiration date.¹ Three of the syringes and one control, nonexpired syringe were analyzed using liquid chromatography-mass spectrometry and nuclear magnetic resonance to determine epinephrine content. The contents of the other three syringes of epinephrine were cultured for bacteria and fungus, which yielded no microbial growth. The study showed that the content of epinephrine present in the original sample remained unchanged, compared with the control.

Rachid et al. looked at 35 EpiPens 3-36 months past their expiration dates.² The percentage of epinephrine found remained 84%-101%, with all EpiPens less than 24 months past expiration having > 90% of the labeled epinephrine dose. Cantrell and colleagues evaluated a combination of 40 EpiPens and Epipen Jrs that were 1-50 months past expiration.³ These pens had not been kept in ideal conditions, as some had been in cars, outdoor cabins, and other environments without temperature control. Sixty-one percent of the Epipens and 56% of the EpiPen Juniors had > 90% of the labeled epinephrine content. I think expired Epipens can be used as a back-up option – that is, they are safe to use if there is not an Epipen available that is not expired.
 

Shelf life extension program

Lyon and colleagues reported data from the Shelf Life Extension Program (SLEP).⁴ A total of 122 drugs were studied representing 3,005 lots. Based on testing and stability assessment, 88% of the lots were extended at least 1 year beyond their original expiration date for an average extension of 66 months, but the additional stability period was highly variable. Several antibiotics were studied, including ciprofloxacin (mean extension, 55 months), amoxicillin (mean extension, 23 months), and doxycycline (mean extension, 50 months).

What about other drugs not in pill form?

I am frequently asked about the longevity of medication formulations that are not in pill form. For example, I have been asked about using expired eye drops. There are few data on this. Reis at al. studied whether travoprost that was past the expiration date still lowered intraocular pressures.⁵ Intraocular pressures in glaucoma patients treated with travoprost 6 weeks after the seal was broken were compared with pressures when drops were used immediately after the container seal was broken. There was no significant difference in intraocular pressure between the two treatment groups during the study.

I found one case report of harm from using expired eye medications. Use of expired eye drops was associated with a case of bilateral toxic epithelial keratopathy.⁶ Eye drops can be contaminated and cause irritation from the breakdown products of preservatives.

Many people use inhalers for many years. This is especially true for albuterol, which is often used for very intermittent symptoms. I found one recent study on the stability of albuterol. Kutty et al. studied expired albuterol inhalers and solutions up to 20 years past expiration.⁷ Almost all lots of albuterol maintained > 90% of product (73%-103%), many years past their expiration date. Even at 73% retained activity, the dose would likely be effective.
 

Pearl: Expired epinephrine and albuterol appear to retain activity several years past expiration.

Dr. Paauw is professor of medicine in the division of general internal medicine at the University of Washington, Seattle, and he serves as third-year medical student clerkship director at the University of Washington. He has no conflicts of interest. Contact Dr. Paauw at [email protected].

References

1. Weir WB et al. Prehosp Emerg Care. 2018 Jul-Aug;22(4):414-8.

2. Rachid O et al. Ann Allergy Asthma Immunol. 2015 Apr;114(4):354-6.

3. Cantrell FL et al. Ann Intern Med. 2017 Jun 20;166(12):918-9.

4. Lyon RC et al. J Pharmaceut Sci. 2006;95(7):1549-60.

5. Reis R et al. Clin Ther. 2004 Dec;26(12):2121-7.

6. AlGhadeer H, AlHumaiden A. J Clin Pharm Ther. 2022 Dec;47(12):2379-82.

7. Kutty RG et al. Heliyon. 2022 Aug 5;8(8):e10104.

A patient inquires about whether he or she can use an EpiPen after the expiration date. What should you advise?

A. The EpiPen is unlikely to be effective after the expiration date.

B. The EpiPen may be dangerous to use after the expiration date.

C. The EpiPen is likely to be okay up to 2 years past the expiration date.

I think that choice C is the most accurate and will get to all the evidence shortly. The expiration date is not the date that the drug stops being effective or potentially becomes toxic. It is a date, required by law, that the manufacturer can guarantee greater than 90% original potency of the medication.

Epinephrine is a costly drug and is usually replaced when the Epipen expires. Weir and colleagues studied six epinephrine syringes 30 months past their expiration date.¹ Three of the syringes and one control, nonexpired syringe were analyzed using liquid chromatography-mass spectrometry and nuclear magnetic resonance to determine epinephrine content. The contents of the other three syringes of epinephrine were cultured for bacteria and fungus, which yielded no microbial growth. The study showed that the content of epinephrine present in the original sample remained unchanged, compared with the control.

Rachid et al. looked at 35 EpiPens 3-36 months past their expiration dates.² The percentage of epinephrine found remained 84%-101%, with all EpiPens less than 24 months past expiration having > 90% of the labeled epinephrine dose. Cantrell and colleagues evaluated a combination of 40 EpiPens and Epipen Jrs that were 1-50 months past expiration.³ These pens had not been kept in ideal conditions, as some had been in cars, outdoor cabins, and other environments without temperature control. Sixty-one percent of the Epipens and 56% of the EpiPen Juniors had > 90% of the labeled epinephrine content. I think expired Epipens can be used as a back-up option – that is, they are safe to use if there is not an Epipen available that is not expired.
 

Shelf life extension program

Lyon and colleagues reported data from the Shelf Life Extension Program (SLEP).⁴ A total of 122 drugs were studied representing 3,005 lots. Based on testing and stability assessment, 88% of the lots were extended at least 1 year beyond their original expiration date for an average extension of 66 months, but the additional stability period was highly variable. Several antibiotics were studied, including ciprofloxacin (mean extension, 55 months), amoxicillin (mean extension, 23 months), and doxycycline (mean extension, 50 months).

What about other drugs not in pill form?

I am frequently asked about the longevity of medication formulations that are not in pill form. For example, I have been asked about using expired eye drops. There are few data on this. Reis at al. studied whether travoprost that was past the expiration date still lowered intraocular pressures.⁵ Intraocular pressures in glaucoma patients treated with travoprost 6 weeks after the seal was broken were compared with pressures when drops were used immediately after the container seal was broken. There was no significant difference in intraocular pressure between the two treatment groups during the study.

I found one case report of harm from using expired eye medications. Use of expired eye drops was associated with a case of bilateral toxic epithelial keratopathy.⁶ Eye drops can be contaminated and cause irritation from the breakdown products of preservatives.

Many people use inhalers for many years. This is especially true for albuterol, which is often used for very intermittent symptoms. I found one recent study on the stability of albuterol. Kutty et al. studied expired albuterol inhalers and solutions up to 20 years past expiration.⁷ Almost all lots of albuterol maintained > 90% of product (73%-103%), many years past their expiration date. Even at 73% retained activity, the dose would likely be effective.
 

Pearl: Expired epinephrine and albuterol appear to retain activity several years past expiration.

Dr. Paauw is professor of medicine in the division of general internal medicine at the University of Washington, Seattle, and he serves as third-year medical student clerkship director at the University of Washington. He has no conflicts of interest. Contact Dr. Paauw at [email protected].

References

1. Weir WB et al. Prehosp Emerg Care. 2018 Jul-Aug;22(4):414-8.

2. Rachid O et al. Ann Allergy Asthma Immunol. 2015 Apr;114(4):354-6.

3. Cantrell FL et al. Ann Intern Med. 2017 Jun 20;166(12):918-9.

4. Lyon RC et al. J Pharmaceut Sci. 2006;95(7):1549-60.

5. Reis R et al. Clin Ther. 2004 Dec;26(12):2121-7.

6. AlGhadeer H, AlHumaiden A. J Clin Pharm Ther. 2022 Dec;47(12):2379-82.

7. Kutty RG et al. Heliyon. 2022 Aug 5;8(8):e10104.

A patient inquires about whether he or she can use an EpiPen after the expiration date. What should you advise?

A. The EpiPen is unlikely to be effective after the expiration date.

B. The EpiPen may be dangerous to use after the expiration date.

C. The EpiPen is likely to be okay up to 2 years past the expiration date.

I think that choice C is the most accurate and will get to all the evidence shortly. The expiration date is not the date that the drug stops being effective or potentially becomes toxic. It is a date, required by law, that the manufacturer can guarantee greater than 90% original potency of the medication.

Epinephrine is a costly drug and is usually replaced when the Epipen expires. Weir and colleagues studied six epinephrine syringes 30 months past their expiration date.¹ Three of the syringes and one control, nonexpired syringe were analyzed using liquid chromatography-mass spectrometry and nuclear magnetic resonance to determine epinephrine content. The contents of the other three syringes of epinephrine were cultured for bacteria and fungus, which yielded no microbial growth. The study showed that the content of epinephrine present in the original sample remained unchanged, compared with the control.

Rachid et al. looked at 35 EpiPens 3-36 months past their expiration dates.² The percentage of epinephrine found remained 84%-101%, with all EpiPens less than 24 months past expiration having > 90% of the labeled epinephrine dose. Cantrell and colleagues evaluated a combination of 40 EpiPens and Epipen Jrs that were 1-50 months past expiration.³ These pens had not been kept in ideal conditions, as some had been in cars, outdoor cabins, and other environments without temperature control. Sixty-one percent of the Epipens and 56% of the EpiPen Juniors had > 90% of the labeled epinephrine content. I think expired Epipens can be used as a back-up option – that is, they are safe to use if there is not an Epipen available that is not expired.
 

Shelf life extension program

Lyon and colleagues reported data from the Shelf Life Extension Program (SLEP).⁴ A total of 122 drugs were studied representing 3,005 lots. Based on testing and stability assessment, 88% of the lots were extended at least 1 year beyond their original expiration date for an average extension of 66 months, but the additional stability period was highly variable. Several antibiotics were studied, including ciprofloxacin (mean extension, 55 months), amoxicillin (mean extension, 23 months), and doxycycline (mean extension, 50 months).

What about other drugs not in pill form?

I am frequently asked about the longevity of medication formulations that are not in pill form. For example, I have been asked about using expired eye drops. There are few data on this. Reis at al. studied whether travoprost that was past the expiration date still lowered intraocular pressures.⁵ Intraocular pressures in glaucoma patients treated with travoprost 6 weeks after the seal was broken were compared with pressures when drops were used immediately after the container seal was broken. There was no significant difference in intraocular pressure between the two treatment groups during the study.

I found one case report of harm from using expired eye medications. Use of expired eye drops was associated with a case of bilateral toxic epithelial keratopathy.⁶ Eye drops can be contaminated and cause irritation from the breakdown products of preservatives.

Many people use inhalers for many years. This is especially true for albuterol, which is often used for very intermittent symptoms. I found one recent study on the stability of albuterol. Kutty et al. studied expired albuterol inhalers and solutions up to 20 years past expiration.⁷ Almost all lots of albuterol maintained > 90% of product (73%-103%), many years past their expiration date. Even at 73% retained activity, the dose would likely be effective.
 

Pearl: Expired epinephrine and albuterol appear to retain activity several years past expiration.

Dr. Paauw is professor of medicine in the division of general internal medicine at the University of Washington, Seattle, and he serves as third-year medical student clerkship director at the University of Washington. He has no conflicts of interest. Contact Dr. Paauw at [email protected].

References

1. Weir WB et al. Prehosp Emerg Care. 2018 Jul-Aug;22(4):414-8.

2. Rachid O et al. Ann Allergy Asthma Immunol. 2015 Apr;114(4):354-6.

3. Cantrell FL et al. Ann Intern Med. 2017 Jun 20;166(12):918-9.

4. Lyon RC et al. J Pharmaceut Sci. 2006;95(7):1549-60.

5. Reis R et al. Clin Ther. 2004 Dec;26(12):2121-7.

6. AlGhadeer H, AlHumaiden A. J Clin Pharm Ther. 2022 Dec;47(12):2379-82.

7. Kutty RG et al. Heliyon. 2022 Aug 5;8(8):e10104.

I wanted to very briefly highlight a truly extraordinary event in my professional experience as a clinical investigator for almost 40 years in the area of the gynecologic malignancies: the simultaneous publication in The New England Journal of Medicine of two landmark, paradigm-changing studies involving the management of advanced endometrial cancer.

City of Hope

In my career, of course, I’ve treated endometrial cancer, but the paradigm, the algorithms, and the strategies we’ve used have, for the most part, simply followed what we’ve done for ovarian cancer. If platinums worked in ovarian cancer, they probably worked in endometrial cancer, and that was true. If paclitaxel worked and had activity in ovarian cancer, it probably would in endometrial cancer, and that was true. It took some time, but basically, we use the same frontline chemotherapy in advanced or recurrent endometrial cancer as we’ve used in ovarian cancer, and on and on.

That world has changed, very much for the positive. Not only have pharmaceutical companies, academic investigators, and individual investigators in the community setting seen endometrial cancer as a major priority, but we have exciting new developments, and very specifically, of course, the immunotherapeutic agents known as checkpoint inhibitors.

One of these two papers was titled “Pembrolizumab Plus Chemotherapy in Advanced Endometrial Cancer” and the second one was titled “Dostarlimab for Primary Advanced or Recurrent Endometrial Cancer.” Obviously, these were separate studies, but both used checkpoint inhibitor plus the chemotherapeutic agents carboplatin-paclitaxel, compared with chemotherapy alone as frontline therapy for advanced or recurrent ovarian cancer and demonstrated a statistically significant, and in my opinion, highly clinically meaningful improvement, in progression-free survival in favor of the regimen that included the checkpoint inhibitors.

Clearly, we will need longer follow-up to see both the overall magnitude of the effect of these therapies on overall survival and the duration of the effect – the shape of the curve. Do we cure many more people? Do we delay time to progression and death? That remains to be seen.

But the outcomes we have now are remarkably positive for patients and have absolutely changed the standard of care in the management of recurrent or advanced endometrial cancer.

I should note that this includes both patients who have evidence of mismatch repair deficiency and those patients who do not have evidence of deficiency, which is a large patient population. These studies demonstrated the benefit to the entire population of patients.

However, on the basis of the data that we have – not only in endometrial cancer, but in other tumor types – the greatest impact was seen in patients with evidence of mismatch repair deficiency, where the immunotherapy agent has been shown to be most relevant; not exclusively, but most relevant.

These are very important papers. If you have an interest in endometrial cancer or immunotherapy, I would encourage you to read these papers. They change the paradigm of management for advanced endometrial cancer, and they clearly point out directions for future research in the management of this class of gynecologic cancers.

Dr. Markman is a professor in the department of medical oncology and therapeutics research at City of Hope in Duarte, Calif., and the president of Medicine & Science at City of Hope Atlanta, Chicago, and Phoenix. He reported conflicts of interest with AstraZeneca and GlaxoSmithKline.

This transcript has been edited for clarity. A version of this article first appeared on Medscape.com.

I wanted to very briefly highlight a truly extraordinary event in my professional experience as a clinical investigator for almost 40 years in the area of the gynecologic malignancies: the simultaneous publication in The New England Journal of Medicine of two landmark, paradigm-changing studies involving the management of advanced endometrial cancer.

City of Hope

In my career, of course, I’ve treated endometrial cancer, but the paradigm, the algorithms, and the strategies we’ve used have, for the most part, simply followed what we’ve done for ovarian cancer. If platinums worked in ovarian cancer, they probably worked in endometrial cancer, and that was true. If paclitaxel worked and had activity in ovarian cancer, it probably would in endometrial cancer, and that was true. It took some time, but basically, we use the same frontline chemotherapy in advanced or recurrent endometrial cancer as we’ve used in ovarian cancer, and on and on.

That world has changed, very much for the positive. Not only have pharmaceutical companies, academic investigators, and individual investigators in the community setting seen endometrial cancer as a major priority, but we have exciting new developments, and very specifically, of course, the immunotherapeutic agents known as checkpoint inhibitors.

One of these two papers was titled “Pembrolizumab Plus Chemotherapy in Advanced Endometrial Cancer” and the second one was titled “Dostarlimab for Primary Advanced or Recurrent Endometrial Cancer.” Obviously, these were separate studies, but both used checkpoint inhibitor plus the chemotherapeutic agents carboplatin-paclitaxel, compared with chemotherapy alone as frontline therapy for advanced or recurrent ovarian cancer and demonstrated a statistically significant, and in my opinion, highly clinically meaningful improvement, in progression-free survival in favor of the regimen that included the checkpoint inhibitors.

Clearly, we will need longer follow-up to see both the overall magnitude of the effect of these therapies on overall survival and the duration of the effect – the shape of the curve. Do we cure many more people? Do we delay time to progression and death? That remains to be seen.

But the outcomes we have now are remarkably positive for patients and have absolutely changed the standard of care in the management of recurrent or advanced endometrial cancer.

I should note that this includes both patients who have evidence of mismatch repair deficiency and those patients who do not have evidence of deficiency, which is a large patient population. These studies demonstrated the benefit to the entire population of patients.

However, on the basis of the data that we have – not only in endometrial cancer, but in other tumor types – the greatest impact was seen in patients with evidence of mismatch repair deficiency, where the immunotherapy agent has been shown to be most relevant; not exclusively, but most relevant.

These are very important papers. If you have an interest in endometrial cancer or immunotherapy, I would encourage you to read these papers. They change the paradigm of management for advanced endometrial cancer, and they clearly point out directions for future research in the management of this class of gynecologic cancers.

Dr. Markman is a professor in the department of medical oncology and therapeutics research at City of Hope in Duarte, Calif., and the president of Medicine & Science at City of Hope Atlanta, Chicago, and Phoenix. He reported conflicts of interest with AstraZeneca and GlaxoSmithKline.

This transcript has been edited for clarity. A version of this article first appeared on Medscape.com.

I wanted to very briefly highlight a truly extraordinary event in my professional experience as a clinical investigator for almost 40 years in the area of the gynecologic malignancies: the simultaneous publication in The New England Journal of Medicine of two landmark, paradigm-changing studies involving the management of advanced endometrial cancer.

City of Hope

In my career, of course, I’ve treated endometrial cancer, but the paradigm, the algorithms, and the strategies we’ve used have, for the most part, simply followed what we’ve done for ovarian cancer. If platinums worked in ovarian cancer, they probably worked in endometrial cancer, and that was true. If paclitaxel worked and had activity in ovarian cancer, it probably would in endometrial cancer, and that was true. It took some time, but basically, we use the same frontline chemotherapy in advanced or recurrent endometrial cancer as we’ve used in ovarian cancer, and on and on.

That world has changed, very much for the positive. Not only have pharmaceutical companies, academic investigators, and individual investigators in the community setting seen endometrial cancer as a major priority, but we have exciting new developments, and very specifically, of course, the immunotherapeutic agents known as checkpoint inhibitors.

One of these two papers was titled “Pembrolizumab Plus Chemotherapy in Advanced Endometrial Cancer” and the second one was titled “Dostarlimab for Primary Advanced or Recurrent Endometrial Cancer.” Obviously, these were separate studies, but both used checkpoint inhibitor plus the chemotherapeutic agents carboplatin-paclitaxel, compared with chemotherapy alone as frontline therapy for advanced or recurrent ovarian cancer and demonstrated a statistically significant, and in my opinion, highly clinically meaningful improvement, in progression-free survival in favor of the regimen that included the checkpoint inhibitors.

Clearly, we will need longer follow-up to see both the overall magnitude of the effect of these therapies on overall survival and the duration of the effect – the shape of the curve. Do we cure many more people? Do we delay time to progression and death? That remains to be seen.

But the outcomes we have now are remarkably positive for patients and have absolutely changed the standard of care in the management of recurrent or advanced endometrial cancer.

I should note that this includes both patients who have evidence of mismatch repair deficiency and those patients who do not have evidence of deficiency, which is a large patient population. These studies demonstrated the benefit to the entire population of patients.

However, on the basis of the data that we have – not only in endometrial cancer, but in other tumor types – the greatest impact was seen in patients with evidence of mismatch repair deficiency, where the immunotherapy agent has been shown to be most relevant; not exclusively, but most relevant.

These are very important papers. If you have an interest in endometrial cancer or immunotherapy, I would encourage you to read these papers. They change the paradigm of management for advanced endometrial cancer, and they clearly point out directions for future research in the management of this class of gynecologic cancers.

Dr. Markman is a professor in the department of medical oncology and therapeutics research at City of Hope in Duarte, Calif., and the president of Medicine & Science at City of Hope Atlanta, Chicago, and Phoenix. He reported conflicts of interest with AstraZeneca and GlaxoSmithKline.

This transcript has been edited for clarity. A version of this article first appeared on Medscape.com.

A phase 2 trial of the investigational factor XI inhibitor abelacimab, used in patients with atrial fibrillation (AFib) who are at moderate to high risk for stroke, has been stopped early due to “an overwhelming reduction” in the primary endpoint – major and clinically relevant nonmajor bleeding – in patients taking abelacimab versus those on rivaroxaban.

The announcement of topline results of the AZALEA-TIMI 71 trial was made by Anthos Therapeutics, the company developing abelacimab.

“The AZALEA-TIMI 71 study is the largest and longest head-to-head study of a Factor XI inhibitor to provide definitive evidence of a highly significant reduction in bleeding as compared to the standard-of-care anticoagulant,” Marc Sabatine, MD, chair of cardiovascular medicine at Brigham and Women’s Hospital and chair of the TIMI study group, both in Boston, stated in the Anthos press release.

“With a median of 21 months of follow-up, spanning more than 2,000 patient years, AZALEA-TIMI 71 represents a landmark study confirming the promise of Factor XI inhibition as causing substantially less bleeding than a current standard-of-care,” Dr. Sabatine added.

Abelacimab is a novel, highly selective, fully human monoclonal antibody with dual inhibitory activity against factor XI and its active form, factor XIa. At the 150-mg dose given subcutaneously once monthly, the drug maintains around 98% inhibition of factor XI, in line with the benign bleeding profile of patients with genetic factor XI deficiency, the company notes.

The AZALEA-TIMI 71 study is an event-driven, randomized study comparing two blinded doses of abelacimab (90 mg or 150 mg given by subcutaneous injection once-monthly) with rivaroxaban 20 mg daily in 1,287 patients with AFib who are at moderate to high risk for stroke. Full results of the study will be presented at an upcoming scientific congress.

Patients in the rivaroxaban arm can transition to abelacimab in an extension study.

In a previous proof-of-concept study published in The New England Journal of Medicine, a single IV dose of abelacimab achieved a large reduction in venous thromboembolism versus enoxaparin in patients undergoing knee surgery.

A phase 3 trial in AFib patients is now planned. The LILAC-TIMI 76 study is an event-driven, randomized trial to evaluate the efficacy and safety of abelacimab relative to placebo on the rate of ischemic stroke or systemic embolism in AFib patients who have been deemed to be unsuitable for currently available anticoagulation therapy. Patients will be randomized to receive abelacimab 150 mg subcutaneously or matching placebo once monthly. The researchers aim to enroll approximately 1,900 patients from North America, Europe, Latin America, the Middle East, and Asia.

Dan Bloomfield, MD, chief medical officer of Anthos Therapeutics, said that, “Abelacimab embodies its promise as a hemostasis-sparing anticoagulant and represents a paradigm shift in the prevention of stroke and other thrombotic conditions.”

It is estimated that 12.1 million people in the United States will have AFib by 2030, but 40%-60% of patients with AFib are not prescribed anticoagulants today, one of the main reasons being concerns about bleeding, the company notes.

“Abelacimab has the potential to provide a game-changing treatment option for all those patients who live with the daily fear of bleeding while taking current anticoagulants,” said Leslie Lake, president of the National Blood Clot Alliance.

Abelacimab has been granted a fast-track designation by the U.S. Food and Drug Administration for the prevention of stroke and systemic embolism in patients with atrial fibrillation.

Several other Factor XI inhibitors are in development and have also shown promising results in terms of a more benign bleeding profile than current standard-of-care anticoagulants.

A version of this article first appeared on Medscape.com.

A phase 2 trial of the investigational factor XI inhibitor abelacimab, used in patients with atrial fibrillation (AFib) who are at moderate to high risk for stroke, has been stopped early due to “an overwhelming reduction” in the primary endpoint – major and clinically relevant nonmajor bleeding – in patients taking abelacimab versus those on rivaroxaban.

The announcement of topline results of the AZALEA-TIMI 71 trial was made by Anthos Therapeutics, the company developing abelacimab.

“The AZALEA-TIMI 71 study is the largest and longest head-to-head study of a Factor XI inhibitor to provide definitive evidence of a highly significant reduction in bleeding as compared to the standard-of-care anticoagulant,” Marc Sabatine, MD, chair of cardiovascular medicine at Brigham and Women’s Hospital and chair of the TIMI study group, both in Boston, stated in the Anthos press release.

“With a median of 21 months of follow-up, spanning more than 2,000 patient years, AZALEA-TIMI 71 represents a landmark study confirming the promise of Factor XI inhibition as causing substantially less bleeding than a current standard-of-care,” Dr. Sabatine added.

Abelacimab is a novel, highly selective, fully human monoclonal antibody with dual inhibitory activity against factor XI and its active form, factor XIa. At the 150-mg dose given subcutaneously once monthly, the drug maintains around 98% inhibition of factor XI, in line with the benign bleeding profile of patients with genetic factor XI deficiency, the company notes.

The AZALEA-TIMI 71 study is an event-driven, randomized study comparing two blinded doses of abelacimab (90 mg or 150 mg given by subcutaneous injection once-monthly) with rivaroxaban 20 mg daily in 1,287 patients with AFib who are at moderate to high risk for stroke. Full results of the study will be presented at an upcoming scientific congress.

Patients in the rivaroxaban arm can transition to abelacimab in an extension study.

In a previous proof-of-concept study published in The New England Journal of Medicine, a single IV dose of abelacimab achieved a large reduction in venous thromboembolism versus enoxaparin in patients undergoing knee surgery.

A phase 3 trial in AFib patients is now planned. The LILAC-TIMI 76 study is an event-driven, randomized trial to evaluate the efficacy and safety of abelacimab relative to placebo on the rate of ischemic stroke or systemic embolism in AFib patients who have been deemed to be unsuitable for currently available anticoagulation therapy. Patients will be randomized to receive abelacimab 150 mg subcutaneously or matching placebo once monthly. The researchers aim to enroll approximately 1,900 patients from North America, Europe, Latin America, the Middle East, and Asia.

Dan Bloomfield, MD, chief medical officer of Anthos Therapeutics, said that, “Abelacimab embodies its promise as a hemostasis-sparing anticoagulant and represents a paradigm shift in the prevention of stroke and other thrombotic conditions.”

It is estimated that 12.1 million people in the United States will have AFib by 2030, but 40%-60% of patients with AFib are not prescribed anticoagulants today, one of the main reasons being concerns about bleeding, the company notes.

“Abelacimab has the potential to provide a game-changing treatment option for all those patients who live with the daily fear of bleeding while taking current anticoagulants,” said Leslie Lake, president of the National Blood Clot Alliance.

Abelacimab has been granted a fast-track designation by the U.S. Food and Drug Administration for the prevention of stroke and systemic embolism in patients with atrial fibrillation.

Several other Factor XI inhibitors are in development and have also shown promising results in terms of a more benign bleeding profile than current standard-of-care anticoagulants.

A version of this article first appeared on Medscape.com.

A phase 2 trial of the investigational factor XI inhibitor abelacimab, used in patients with atrial fibrillation (AFib) who are at moderate to high risk for stroke, has been stopped early due to “an overwhelming reduction” in the primary endpoint – major and clinically relevant nonmajor bleeding – in patients taking abelacimab versus those on rivaroxaban.

The announcement of topline results of the AZALEA-TIMI 71 trial was made by Anthos Therapeutics, the company developing abelacimab.

“The AZALEA-TIMI 71 study is the largest and longest head-to-head study of a Factor XI inhibitor to provide definitive evidence of a highly significant reduction in bleeding as compared to the standard-of-care anticoagulant,” Marc Sabatine, MD, chair of cardiovascular medicine at Brigham and Women’s Hospital and chair of the TIMI study group, both in Boston, stated in the Anthos press release.

“With a median of 21 months of follow-up, spanning more than 2,000 patient years, AZALEA-TIMI 71 represents a landmark study confirming the promise of Factor XI inhibition as causing substantially less bleeding than a current standard-of-care,” Dr. Sabatine added.

Abelacimab is a novel, highly selective, fully human monoclonal antibody with dual inhibitory activity against factor XI and its active form, factor XIa. At the 150-mg dose given subcutaneously once monthly, the drug maintains around 98% inhibition of factor XI, in line with the benign bleeding profile of patients with genetic factor XI deficiency, the company notes.

The AZALEA-TIMI 71 study is an event-driven, randomized study comparing two blinded doses of abelacimab (90 mg or 150 mg given by subcutaneous injection once-monthly) with rivaroxaban 20 mg daily in 1,287 patients with AFib who are at moderate to high risk for stroke. Full results of the study will be presented at an upcoming scientific congress.

Patients in the rivaroxaban arm can transition to abelacimab in an extension study.

In a previous proof-of-concept study published in The New England Journal of Medicine, a single IV dose of abelacimab achieved a large reduction in venous thromboembolism versus enoxaparin in patients undergoing knee surgery.

A phase 3 trial in AFib patients is now planned. The LILAC-TIMI 76 study is an event-driven, randomized trial to evaluate the efficacy and safety of abelacimab relative to placebo on the rate of ischemic stroke or systemic embolism in AFib patients who have been deemed to be unsuitable for currently available anticoagulation therapy. Patients will be randomized to receive abelacimab 150 mg subcutaneously or matching placebo once monthly. The researchers aim to enroll approximately 1,900 patients from North America, Europe, Latin America, the Middle East, and Asia.

Dan Bloomfield, MD, chief medical officer of Anthos Therapeutics, said that, “Abelacimab embodies its promise as a hemostasis-sparing anticoagulant and represents a paradigm shift in the prevention of stroke and other thrombotic conditions.”

It is estimated that 12.1 million people in the United States will have AFib by 2030, but 40%-60% of patients with AFib are not prescribed anticoagulants today, one of the main reasons being concerns about bleeding, the company notes.

“Abelacimab has the potential to provide a game-changing treatment option for all those patients who live with the daily fear of bleeding while taking current anticoagulants,” said Leslie Lake, president of the National Blood Clot Alliance.

Abelacimab has been granted a fast-track designation by the U.S. Food and Drug Administration for the prevention of stroke and systemic embolism in patients with atrial fibrillation.

Several other Factor XI inhibitors are in development and have also shown promising results in terms of a more benign bleeding profile than current standard-of-care anticoagulants.

A version of this article first appeared on Medscape.com.

With ever-expanding treatment options for the ablation of benign thyroid nodules, the American Thyroid Association has issued an expert consensus statement that addresses the safe implementation and utilization of the techniques.

“There are no documents to date in the United States focusing primarily on the safe adoption and implementation of ablation techniques, including learning curve considerations and necessary pre-procedural skillsets,” reports the ATA task force in the consensus statement, which was published in Thyroid.

“Although these emerging technologies hold great promise, they are not without risk and require development of a unique skill set and environment for optimal, safe performance and consistent outcomes,” task force co-author Catherine F. Sinclair, MD, an associate professor at the Icahn School of Medicine at Mount Sinai, New York, said in an interview.

Chemical ablation has long been utilized as a nonsurgical option for benign thyroid nodule ablation. However, the current array of treatment options has expanded with thermal ablation. Techniques such as radiofrequency ablation (RFA), laser ablation, microwave ablation, and high-intensity focused ultrasound have gained favor as minimally invasive alternatives to surgery.

Much has been published on indications and outcomes with the use of the techniques. The multidisciplinary global task force was convened to address key issues regarding safety and utilization. The report is directed toward specialists, including surgeons, endocrinologists, and interventional radiologists.

The recommendations cover three broad categories: safety considerations spanning preprocedural to postprocedural periods; necessary skill sets for optimal, safe performance with the approaches; and the expectations for success in the context of risks and benefits.
 

Ablation methods can depend on nodule type

Among key issues addressed are which ablation methods are most appropriate for which types of nodules. Recommendations include chemical ablation, typically involving the injection of dehydrated ethanol in a target nodule. In solid nodules, diffusion with chemical ablation can be unpredictable, which makes it more appropriate for cystic nodules.

Thermal ablation is considered best suited for patients with compressive and/or cosmetic complaints that clearly involve a single or dominant nodule, as well as for autonomously functioning thyroid nodules that cause subclinical or overt hyperthyroidism.

While ethanol ablation is recommended as a first-line treatment for benign cystic thyroid nodules, its efficacy decreases when there is an increase of more than 20% of the solid component. In such cases, RFA or a combination of ethanol ablation and RFA may be considered, the task force recommends.
 

Patient counseling – managing expectations

Another key consideration in treatment with thyroid nodule ablation is managing patients’ expectations.

Patients should be advised of benefits, such as the avoidance of surgery and general anesthesia and less recovery time. Risks can include thermal or chemical injury to the recurrent laryngeal nerve and other vital structures. The task force underscores discussion of alternative options with patients.

Alternative management options to ablation, including observation, radioactive iodine for functioning nodules, and surgery should also be discussed, and “their relative advantages and disadvantages should be presented without bias such that the patient can make an informed, individual treatment decision,” the task force recommends.

Patients should be informed that, in contrast to surgical management, the benefits of ablation are not immediate; rather, they accrue over the course of months. Reduction in nodule size within the first month is often limited.

Pain, soreness, and some swelling of the nodule and surrounding tissues are common in the first week. These symptoms usually peak in the first 3-5 days after the procedure. Importantly, patients rarely require opioid medications, and their use should be avoided, the task force recommends.

Patients should also be informed about the possibilities of nodule regrowth following ablation and the possible need for more than one ablation procedure.

“Although regrowth definitions in the literature vary, risk of regrowth after thermal ablation is 5%-40% and increases the larger the baseline nodule volume,” the task force notes.

Of note, most studies on ablation to date have shown that thermal ablation complication rates are low. Twelve months post procedure, volume reductions are typically greater than 50%.
 

Follow-up

For long-term monitoring following ablation, follow-up neck ultrasound is typically recommended at 1-3 months and at 6 and 12 months post ablation to assess volume reduction, nodule appearance, nodule vascularity, and areas at risk for regrowth, the authors note.

Prolonged serial biochemical evaluation of thyroid function is only recommended in cases of hyperfunctioning thyroid nodules.

Key considerations for additional ablative sessions for nodules greater than 20-30 mL in volume should include a failure to achieve adequate reduction in volume, nodule regrowth in previously untreated peripheral areas, and/or persistent or new compressive symptoms.
 

Learning curve

Dr. Sinclair underscored that successful thyroid nodule ablation requires skill – and experience.

“Probably the greatest concern shared by the writing group on this statement was the potential for clinicians to start ablation practices without having an appropriate prior skill set,” she said.

“Ablation is an advanced, ultrasound-guided procedure, and clinicians need to be experienced in performing neck ultrasounds and biopsies,” she added. “To consider performing ablations without this skill set is both unrealistic and dangerous.”

RFA, currently the most commonly used thermal ablation method for benign thyroid nodule ablation in the U.S., “has a good safety profile but can have a steep learning curve initially,” she said.

Among the most important recommendations is that for their first 20-60 ablation procedures, clinicians should consider limiting treatment to small- to medium-sized benign nodules rather than large-volume disease, Dr. Sinclair added.

“In addition, prior to starting thyroid ablation practices, clinicians should be proficient in ultrasound imaging and fine-needle biopsies and can gain valuable experience by practicing on phantoms and having expert proctoring for the first few cases,” she said.

For initial ablative cases, the task force recommends that clinicians select moderate-size (< 20-30 mL), nonvascular nodules with favorable characteristics and location. The final volume reduction should be based not only on baseline nodule characteristics, such as volume and vascularity, but also on the practitioner’s skill.

Clinicians furthermore should be board certified or eligible in an appropriate medical specialty, have extensive background knowledge, and “should have clinical experience in the clinical diagnosis and treatment of thyroid nodules; neck imaging anatomy; thyroid ultrasound imaging and fine needle aspiration biopsy procedures; and ultrasound risk stratification for benign and malignant thyroid tumors,” the group recommends.

Importantly, the statement is designed to reflect a consensus opinion of the panel of experts but is not meant to serve as a formal guideline or a standard of care for the clinical practice of thermal ablation, Dr. Sinclair added.

“It is not the intent of the statement to replace individual decision-making, the wishes of the patient or family, or clinical judgment.”

The authors’ disclosures are detailed in the published report.

A version of this article first appeared on Medscape.com.

With ever-expanding treatment options for the ablation of benign thyroid nodules, the American Thyroid Association has issued an expert consensus statement that addresses the safe implementation and utilization of the techniques.

“There are no documents to date in the United States focusing primarily on the safe adoption and implementation of ablation techniques, including learning curve considerations and necessary pre-procedural skillsets,” reports the ATA task force in the consensus statement, which was published in Thyroid.

“Although these emerging technologies hold great promise, they are not without risk and require development of a unique skill set and environment for optimal, safe performance and consistent outcomes,” task force co-author Catherine F. Sinclair, MD, an associate professor at the Icahn School of Medicine at Mount Sinai, New York, said in an interview.

Chemical ablation has long been utilized as a nonsurgical option for benign thyroid nodule ablation. However, the current array of treatment options has expanded with thermal ablation. Techniques such as radiofrequency ablation (RFA), laser ablation, microwave ablation, and high-intensity focused ultrasound have gained favor as minimally invasive alternatives to surgery.

Much has been published on indications and outcomes with the use of the techniques. The multidisciplinary global task force was convened to address key issues regarding safety and utilization. The report is directed toward specialists, including surgeons, endocrinologists, and interventional radiologists.

The recommendations cover three broad categories: safety considerations spanning preprocedural to postprocedural periods; necessary skill sets for optimal, safe performance with the approaches; and the expectations for success in the context of risks and benefits.
 

Ablation methods can depend on nodule type

Among key issues addressed are which ablation methods are most appropriate for which types of nodules. Recommendations include chemical ablation, typically involving the injection of dehydrated ethanol in a target nodule. In solid nodules, diffusion with chemical ablation can be unpredictable, which makes it more appropriate for cystic nodules.

Thermal ablation is considered best suited for patients with compressive and/or cosmetic complaints that clearly involve a single or dominant nodule, as well as for autonomously functioning thyroid nodules that cause subclinical or overt hyperthyroidism.

While ethanol ablation is recommended as a first-line treatment for benign cystic thyroid nodules, its efficacy decreases when there is an increase of more than 20% of the solid component. In such cases, RFA or a combination of ethanol ablation and RFA may be considered, the task force recommends.
 

Patient counseling – managing expectations

Another key consideration in treatment with thyroid nodule ablation is managing patients’ expectations.

Patients should be advised of benefits, such as the avoidance of surgery and general anesthesia and less recovery time. Risks can include thermal or chemical injury to the recurrent laryngeal nerve and other vital structures. The task force underscores discussion of alternative options with patients.

Alternative management options to ablation, including observation, radioactive iodine for functioning nodules, and surgery should also be discussed, and “their relative advantages and disadvantages should be presented without bias such that the patient can make an informed, individual treatment decision,” the task force recommends.

Patients should be informed that, in contrast to surgical management, the benefits of ablation are not immediate; rather, they accrue over the course of months. Reduction in nodule size within the first month is often limited.

Pain, soreness, and some swelling of the nodule and surrounding tissues are common in the first week. These symptoms usually peak in the first 3-5 days after the procedure. Importantly, patients rarely require opioid medications, and their use should be avoided, the task force recommends.

Patients should also be informed about the possibilities of nodule regrowth following ablation and the possible need for more than one ablation procedure.

“Although regrowth definitions in the literature vary, risk of regrowth after thermal ablation is 5%-40% and increases the larger the baseline nodule volume,” the task force notes.

Of note, most studies on ablation to date have shown that thermal ablation complication rates are low. Twelve months post procedure, volume reductions are typically greater than 50%.
 

Follow-up

For long-term monitoring following ablation, follow-up neck ultrasound is typically recommended at 1-3 months and at 6 and 12 months post ablation to assess volume reduction, nodule appearance, nodule vascularity, and areas at risk for regrowth, the authors note.

Prolonged serial biochemical evaluation of thyroid function is only recommended in cases of hyperfunctioning thyroid nodules.

Key considerations for additional ablative sessions for nodules greater than 20-30 mL in volume should include a failure to achieve adequate reduction in volume, nodule regrowth in previously untreated peripheral areas, and/or persistent or new compressive symptoms.
 

Learning curve

Dr. Sinclair underscored that successful thyroid nodule ablation requires skill – and experience.

“Probably the greatest concern shared by the writing group on this statement was the potential for clinicians to start ablation practices without having an appropriate prior skill set,” she said.

“Ablation is an advanced, ultrasound-guided procedure, and clinicians need to be experienced in performing neck ultrasounds and biopsies,” she added. “To consider performing ablations without this skill set is both unrealistic and dangerous.”

RFA, currently the most commonly used thermal ablation method for benign thyroid nodule ablation in the U.S., “has a good safety profile but can have a steep learning curve initially,” she said.

Among the most important recommendations is that for their first 20-60 ablation procedures, clinicians should consider limiting treatment to small- to medium-sized benign nodules rather than large-volume disease, Dr. Sinclair added.

“In addition, prior to starting thyroid ablation practices, clinicians should be proficient in ultrasound imaging and fine-needle biopsies and can gain valuable experience by practicing on phantoms and having expert proctoring for the first few cases,” she said.

For initial ablative cases, the task force recommends that clinicians select moderate-size (< 20-30 mL), nonvascular nodules with favorable characteristics and location. The final volume reduction should be based not only on baseline nodule characteristics, such as volume and vascularity, but also on the practitioner’s skill.

Clinicians furthermore should be board certified or eligible in an appropriate medical specialty, have extensive background knowledge, and “should have clinical experience in the clinical diagnosis and treatment of thyroid nodules; neck imaging anatomy; thyroid ultrasound imaging and fine needle aspiration biopsy procedures; and ultrasound risk stratification for benign and malignant thyroid tumors,” the group recommends.

Importantly, the statement is designed to reflect a consensus opinion of the panel of experts but is not meant to serve as a formal guideline or a standard of care for the clinical practice of thermal ablation, Dr. Sinclair added.

“It is not the intent of the statement to replace individual decision-making, the wishes of the patient or family, or clinical judgment.”

The authors’ disclosures are detailed in the published report.

A version of this article first appeared on Medscape.com.

With ever-expanding treatment options for the ablation of benign thyroid nodules, the American Thyroid Association has issued an expert consensus statement that addresses the safe implementation and utilization of the techniques.

“There are no documents to date in the United States focusing primarily on the safe adoption and implementation of ablation techniques, including learning curve considerations and necessary pre-procedural skillsets,” reports the ATA task force in the consensus statement, which was published in Thyroid.

“Although these emerging technologies hold great promise, they are not without risk and require development of a unique skill set and environment for optimal, safe performance and consistent outcomes,” task force co-author Catherine F. Sinclair, MD, an associate professor at the Icahn School of Medicine at Mount Sinai, New York, said in an interview.

Chemical ablation has long been utilized as a nonsurgical option for benign thyroid nodule ablation. However, the current array of treatment options has expanded with thermal ablation. Techniques such as radiofrequency ablation (RFA), laser ablation, microwave ablation, and high-intensity focused ultrasound have gained favor as minimally invasive alternatives to surgery.

Much has been published on indications and outcomes with the use of the techniques. The multidisciplinary global task force was convened to address key issues regarding safety and utilization. The report is directed toward specialists, including surgeons, endocrinologists, and interventional radiologists.

The recommendations cover three broad categories: safety considerations spanning preprocedural to postprocedural periods; necessary skill sets for optimal, safe performance with the approaches; and the expectations for success in the context of risks and benefits.
 

Ablation methods can depend on nodule type

Among key issues addressed are which ablation methods are most appropriate for which types of nodules. Recommendations include chemical ablation, typically involving the injection of dehydrated ethanol in a target nodule. In solid nodules, diffusion with chemical ablation can be unpredictable, which makes it more appropriate for cystic nodules.

Thermal ablation is considered best suited for patients with compressive and/or cosmetic complaints that clearly involve a single or dominant nodule, as well as for autonomously functioning thyroid nodules that cause subclinical or overt hyperthyroidism.

While ethanol ablation is recommended as a first-line treatment for benign cystic thyroid nodules, its efficacy decreases when there is an increase of more than 20% of the solid component. In such cases, RFA or a combination of ethanol ablation and RFA may be considered, the task force recommends.
 

Patient counseling – managing expectations

Another key consideration in treatment with thyroid nodule ablation is managing patients’ expectations.

Patients should be advised of benefits, such as the avoidance of surgery and general anesthesia and less recovery time. Risks can include thermal or chemical injury to the recurrent laryngeal nerve and other vital structures. The task force underscores discussion of alternative options with patients.

Alternative management options to ablation, including observation, radioactive iodine for functioning nodules, and surgery should also be discussed, and “their relative advantages and disadvantages should be presented without bias such that the patient can make an informed, individual treatment decision,” the task force recommends.

Patients should be informed that, in contrast to surgical management, the benefits of ablation are not immediate; rather, they accrue over the course of months. Reduction in nodule size within the first month is often limited.

Pain, soreness, and some swelling of the nodule and surrounding tissues are common in the first week. These symptoms usually peak in the first 3-5 days after the procedure. Importantly, patients rarely require opioid medications, and their use should be avoided, the task force recommends.

Patients should also be informed about the possibilities of nodule regrowth following ablation and the possible need for more than one ablation procedure.

“Although regrowth definitions in the literature vary, risk of regrowth after thermal ablation is 5%-40% and increases the larger the baseline nodule volume,” the task force notes.

Of note, most studies on ablation to date have shown that thermal ablation complication rates are low. Twelve months post procedure, volume reductions are typically greater than 50%.
 

Follow-up

For long-term monitoring following ablation, follow-up neck ultrasound is typically recommended at 1-3 months and at 6 and 12 months post ablation to assess volume reduction, nodule appearance, nodule vascularity, and areas at risk for regrowth, the authors note.

Prolonged serial biochemical evaluation of thyroid function is only recommended in cases of hyperfunctioning thyroid nodules.

Key considerations for additional ablative sessions for nodules greater than 20-30 mL in volume should include a failure to achieve adequate reduction in volume, nodule regrowth in previously untreated peripheral areas, and/or persistent or new compressive symptoms.
 

Learning curve

Dr. Sinclair underscored that successful thyroid nodule ablation requires skill – and experience.

“Probably the greatest concern shared by the writing group on this statement was the potential for clinicians to start ablation practices without having an appropriate prior skill set,” she said.

“Ablation is an advanced, ultrasound-guided procedure, and clinicians need to be experienced in performing neck ultrasounds and biopsies,” she added. “To consider performing ablations without this skill set is both unrealistic and dangerous.”

RFA, currently the most commonly used thermal ablation method for benign thyroid nodule ablation in the U.S., “has a good safety profile but can have a steep learning curve initially,” she said.

Among the most important recommendations is that for their first 20-60 ablation procedures, clinicians should consider limiting treatment to small- to medium-sized benign nodules rather than large-volume disease, Dr. Sinclair added.

“In addition, prior to starting thyroid ablation practices, clinicians should be proficient in ultrasound imaging and fine-needle biopsies and can gain valuable experience by practicing on phantoms and having expert proctoring for the first few cases,” she said.

For initial ablative cases, the task force recommends that clinicians select moderate-size (< 20-30 mL), nonvascular nodules with favorable characteristics and location. The final volume reduction should be based not only on baseline nodule characteristics, such as volume and vascularity, but also on the practitioner’s skill.

Clinicians furthermore should be board certified or eligible in an appropriate medical specialty, have extensive background knowledge, and “should have clinical experience in the clinical diagnosis and treatment of thyroid nodules; neck imaging anatomy; thyroid ultrasound imaging and fine needle aspiration biopsy procedures; and ultrasound risk stratification for benign and malignant thyroid tumors,” the group recommends.

Importantly, the statement is designed to reflect a consensus opinion of the panel of experts but is not meant to serve as a formal guideline or a standard of care for the clinical practice of thermal ablation, Dr. Sinclair added.

“It is not the intent of the statement to replace individual decision-making, the wishes of the patient or family, or clinical judgment.”

The authors’ disclosures are detailed in the published report.

A version of this article first appeared on Medscape.com.

This transcript has been edited for clarity.

Let’s assume, for the sake of argument, that I am a healthy 43-year old man. Nevertheless, I am interested in getting my vitamin D level checked. My primary care doc says it’s unnecessary, but that doesn’t matter because a variety of direct-to-consumer testing companies will do it without a doctor’s prescription – for a fee of course.

Is that okay? Should I be able to get the test?

What if instead of my vitamin D level, I want to test my testosterone level, or my PSA, or my cadmium level, or my Lyme disease antibodies, or even have a full-body MRI scan? All of these tests are available from a variety of direct-to-consumer testing companies. If I am willing to pay, should I be able to get those too?

These questions are becoming more and more common, because the direct-to-consumer testing market is exploding.

We’re talking about direct-to-consumer testing, thanks to this paper: Policies of US Companies Offering Direct-to-Consumer Laboratory Tests, appearing in JAMA Internal Medicine, which characterizes the testing practices of direct-to-consumer testing companies.

But before we get to the study, a word on this market. Direct-to-consumer lab testing is projected to be a $2 billion industry by 2025, and lab testing megacorporations Quest Diagnostics and Labcorp are both jumping headlong into this space.

Why is this happening? A couple of reasons, I think. First, the increasing cost of health care has led payers to place significant restrictions on what tests can be ordered and under what circumstances. Physicians are all too familiar with the “prior authorization” system that seeks to limit even the tests we think would benefit our patients.

Frustrated with such a system, it’s no wonder that patients are increasingly deciding to go it on their own. Sure, insurance won’t cover these tests, but the prices are transparent and competition actually keeps them somewhat reasonable. So, is this a win-win? Shouldn’t we allow people to get the tests they want, at least if they are willing to pay for it?

Of course, it’s not quite that simple. If the tests are normal, or negative, then sure – no harm, no foul. But when they are positive, everything changes. What happens when the PSA test I got myself via a direct-to-consumer testing company comes back elevated? Well, at that point, I am right back into the traditional mode of medicine – seeing my doctor, probably getting repeat testing, biopsies, etc., – and some payer will be on the hook for that, which is to say that all of us will be on the hook for that.

One other reason direct-to-consumer testing is getting more popular is a more difficult-to-characterize phenomenon which I might call postpandemic individualism. I’ve seen this across several domains, but I think in some ways the pandemic led people to focus more attention on themselves, perhaps because we were so isolated from each other. Optimizing health through data – whether using a fitness tracking watch, meticulously counting macronutrient intake, or ordering your own lab tests – may be a form of exerting control over a universe that feels increasingly chaotic. But what do I know? I’m not a psychologist.

The study characterizes a total of 21 direct-to-consumer testing companies. They offer a variety of services, as you can see here, with the majority in the endocrine space: thyroid, diabetes, men’s and women’s health. A smattering of companies offer more esoteric testing, such as heavy metals and Lyme disease.

Dr. F. Perry Wilson

Dr. F. Perry Wilson

F. Perry Wilson, MD, MSCE, is an associate professor of medicine and public health and director of Yale’s Clinical and Translational Research Accelerator in New Haven, Conn. He reported no relevant conflicts of interest.

A version of this article first appeared on Medscape.com.

This transcript has been edited for clarity.

Let’s assume, for the sake of argument, that I am a healthy 43-year old man. Nevertheless, I am interested in getting my vitamin D level checked. My primary care doc says it’s unnecessary, but that doesn’t matter because a variety of direct-to-consumer testing companies will do it without a doctor’s prescription – for a fee of course.

Is that okay? Should I be able to get the test?

What if instead of my vitamin D level, I want to test my testosterone level, or my PSA, or my cadmium level, or my Lyme disease antibodies, or even have a full-body MRI scan? All of these tests are available from a variety of direct-to-consumer testing companies. If I am willing to pay, should I be able to get those too?

These questions are becoming more and more common, because the direct-to-consumer testing market is exploding.

We’re talking about direct-to-consumer testing, thanks to this paper: Policies of US Companies Offering Direct-to-Consumer Laboratory Tests, appearing in JAMA Internal Medicine, which characterizes the testing practices of direct-to-consumer testing companies.

But before we get to the study, a word on this market. Direct-to-consumer lab testing is projected to be a $2 billion industry by 2025, and lab testing megacorporations Quest Diagnostics and Labcorp are both jumping headlong into this space.

Why is this happening? A couple of reasons, I think. First, the increasing cost of health care has led payers to place significant restrictions on what tests can be ordered and under what circumstances. Physicians are all too familiar with the “prior authorization” system that seeks to limit even the tests we think would benefit our patients.

Frustrated with such a system, it’s no wonder that patients are increasingly deciding to go it on their own. Sure, insurance won’t cover these tests, but the prices are transparent and competition actually keeps them somewhat reasonable. So, is this a win-win? Shouldn’t we allow people to get the tests they want, at least if they are willing to pay for it?

Of course, it’s not quite that simple. If the tests are normal, or negative, then sure – no harm, no foul. But when they are positive, everything changes. What happens when the PSA test I got myself via a direct-to-consumer testing company comes back elevated? Well, at that point, I am right back into the traditional mode of medicine – seeing my doctor, probably getting repeat testing, biopsies, etc., – and some payer will be on the hook for that, which is to say that all of us will be on the hook for that.

One other reason direct-to-consumer testing is getting more popular is a more difficult-to-characterize phenomenon which I might call postpandemic individualism. I’ve seen this across several domains, but I think in some ways the pandemic led people to focus more attention on themselves, perhaps because we were so isolated from each other. Optimizing health through data – whether using a fitness tracking watch, meticulously counting macronutrient intake, or ordering your own lab tests – may be a form of exerting control over a universe that feels increasingly chaotic. But what do I know? I’m not a psychologist.

The study characterizes a total of 21 direct-to-consumer testing companies. They offer a variety of services, as you can see here, with the majority in the endocrine space: thyroid, diabetes, men’s and women’s health. A smattering of companies offer more esoteric testing, such as heavy metals and Lyme disease.

Dr. F. Perry Wilson

Dr. F. Perry Wilson

F. Perry Wilson, MD, MSCE, is an associate professor of medicine and public health and director of Yale’s Clinical and Translational Research Accelerator in New Haven, Conn. He reported no relevant conflicts of interest.

A version of this article first appeared on Medscape.com.

This transcript has been edited for clarity.

Let’s assume, for the sake of argument, that I am a healthy 43-year old man. Nevertheless, I am interested in getting my vitamin D level checked. My primary care doc says it’s unnecessary, but that doesn’t matter because a variety of direct-to-consumer testing companies will do it without a doctor’s prescription – for a fee of course.

Is that okay? Should I be able to get the test?

What if instead of my vitamin D level, I want to test my testosterone level, or my PSA, or my cadmium level, or my Lyme disease antibodies, or even have a full-body MRI scan? All of these tests are available from a variety of direct-to-consumer testing companies. If I am willing to pay, should I be able to get those too?

These questions are becoming more and more common, because the direct-to-consumer testing market is exploding.

We’re talking about direct-to-consumer testing, thanks to this paper: Policies of US Companies Offering Direct-to-Consumer Laboratory Tests, appearing in JAMA Internal Medicine, which characterizes the testing practices of direct-to-consumer testing companies.

But before we get to the study, a word on this market. Direct-to-consumer lab testing is projected to be a $2 billion industry by 2025, and lab testing megacorporations Quest Diagnostics and Labcorp are both jumping headlong into this space.

Why is this happening? A couple of reasons, I think. First, the increasing cost of health care has led payers to place significant restrictions on what tests can be ordered and under what circumstances. Physicians are all too familiar with the “prior authorization” system that seeks to limit even the tests we think would benefit our patients.

Frustrated with such a system, it’s no wonder that patients are increasingly deciding to go it on their own. Sure, insurance won’t cover these tests, but the prices are transparent and competition actually keeps them somewhat reasonable. So, is this a win-win? Shouldn’t we allow people to get the tests they want, at least if they are willing to pay for it?

Of course, it’s not quite that simple. If the tests are normal, or negative, then sure – no harm, no foul. But when they are positive, everything changes. What happens when the PSA test I got myself via a direct-to-consumer testing company comes back elevated? Well, at that point, I am right back into the traditional mode of medicine – seeing my doctor, probably getting repeat testing, biopsies, etc., – and some payer will be on the hook for that, which is to say that all of us will be on the hook for that.

One other reason direct-to-consumer testing is getting more popular is a more difficult-to-characterize phenomenon which I might call postpandemic individualism. I’ve seen this across several domains, but I think in some ways the pandemic led people to focus more attention on themselves, perhaps because we were so isolated from each other. Optimizing health through data – whether using a fitness tracking watch, meticulously counting macronutrient intake, or ordering your own lab tests – may be a form of exerting control over a universe that feels increasingly chaotic. But what do I know? I’m not a psychologist.

The study characterizes a total of 21 direct-to-consumer testing companies. They offer a variety of services, as you can see here, with the majority in the endocrine space: thyroid, diabetes, men’s and women’s health. A smattering of companies offer more esoteric testing, such as heavy metals and Lyme disease.

Dr. F. Perry Wilson

Dr. F. Perry Wilson

F. Perry Wilson, MD, MSCE, is an associate professor of medicine and public health and director of Yale’s Clinical and Translational Research Accelerator in New Haven, Conn. He reported no relevant conflicts of interest.

A version of this article first appeared on Medscape.com.