AVAHO

A recent update to the U.S. recommendations for breast cancer screening is raising concerns about the costs associated with potential follow-up tests, while also renewing debates about the timing of these tests and the screening approaches used.
 

The U.S. Preventive Services Task Force is currently finalizing an update to its recommendations on breast cancer screening. In May, the task force released a proposed update that dropped the initial age for routine mammogram screening from 50 to 40.

The task force intends to give a “B” rating to this recommendation, which covers screening every other year up to age 74 for women deemed average risk for breast cancer.

The task force’s rating carries clout, A. Mark Fendrick, MD, director of the Value-Based Insurance Design at the University of Michigan, Ann Arbor, said in an interview.

For one, the Affordable Care Act requires that private insurers cover services that get top A or B marks from USPSTF without charging copays.

However, Dr. Fendrick noted, such coverage does not necessarily apply to follow-up testing when a routine mammogram comes back with a positive finding. The expense of follow-up testing may deter some women from seeking follow-up diagnostic imaging or biopsies after an abnormal result on a screening mammogram.

A recent analysis in JAMA Network Open found that women facing higher anticipated out-of-pocket costs for breast cancer diagnostic tests, based on their health insurance plan, were less likely to get that follow-up screening. For instance, the use of breast MRI decreased by nearly 24% between patients undergoing subsequent diagnostic testing in plans with the lowest out-of-pocket costs vs. those with the highest.

“The study’s central finding that some women who have an abnormal result on a mammogram may not get appropriate follow-up because of cost is worrisome,” said Dr. Fendrick and Ilana B. Richman, MD, MHS, in an accompanying commentary to the JAMA analysis. “On an individual level, high out-of-pocket costs may directly contribute to worse health outcomes or require individuals to use scarce financial resources that may otherwise be used for critical items such as food or rent.”

For patients to fully benefit from early detection, the USPSTF would also need to make clear that follow-up diagnostic mammograms are covered, Dr. Fendrick said.
 

The ongoing debates

Concerns over the costs of potential follow-up tests are not the only issues experts have highlighted since USPSTF released its updated draft guidance on screening mammography.

The task force’s proposed update has also reignited questions and uncertainties surrounding when to screen, how often, and what types are best.

When it comes to frequency, the major organizations that provide screening guidance don’t see eye to eye. The USPSTF recommends breast cancer screening every other year, while the American College of Radiology recommends screening every year because that approach leads to saves “the most lives.”

At this time, the American College of Obstetricians and Gynecologists guidance currently teeters in the middle, suggesting either annual or biennial screening and highlighting the pros and cons of either approach. According to ACOG, “annual screening intervals appear to result in the least number of breast cancer deaths, particularly in younger women, but at the cost of additional callbacks and biopsies.”

When to begin screening represents another point of contention. While some experts, such as ACOG, agree with the task force’s decision to lower the screening start age to 40, others point to the need for greater nuance on setting the appropriate screening age. The main issue: the task force’s draft sets a uniform age to begin screening, but the risk for breast cancer and breast cancer mortality is not uniform across different racial and ethnic groups.

A recent study published in JAMA Network Open found that, among women aged 40-49, breast cancer mortality was highest among Black women (27 deaths per 100,000 person-years) followed by White women (15 deaths per 100,000 person-years). Based on a recommended screening age of 50, the authors suggested that Black women should start screening at age 42, whereas White women could start at 51.

“These findings suggest that health policy makers and clinicians could consider an alternative, race and ethnicity–adapted approach in which Black female patients start screening earlier,” writes Tianhui Chen, PhD, of China’s Zhejiang Cancer Hospital and coauthor of the study.

Weighing in on the guidance, the nonprofit National Center for Health Research urged the task force to consider suggesting different screening schedules based on race and ethnicity data. That would mean the recommendation to start at age 40 should only apply to Black women and other groups with higher-than-average risk for breast cancer at a younger age.

“Women are capable of understanding why the age to start mammography screening may be different for women with different risk factors,” the National Center for Health Research wrote in a comment to USPSTF, provided to this news organization by request. “What is confusing is when some physician groups recommend annual mammograms for all women starting at age 40, even though the data do not support that recommendation.”

While the ACR agreed with the task force’s recommendation to lower the screening age, the organization suggested starting risk assessments based on racial variations in breast cancer incidence and death even earlier. Specifically, the ACR recommended that high-risk groups, such as Black women, get risk assessments by age 25 to determine whether mammography before age 40 is needed.

Screening options for women with dense breasts may be some of the most challenging to weigh. Having dense breasts increases an individual’s risk for breast cancer, and mammography alone is not as effective at identifying breast cancer among these women. However, the evidence on the benefits vs. harms of additional screening beyond mammography remains mixed.

As a result, the task force decided to maintain its “I” grade on additional screening beyond mammography for these women – a grade that indicates insufficient evidence to determine the benefits and harms for a service.

The task force largely based its decision on the findings of two key reports. One report from the Cancer Intervention and Surveillance Modeling Network, which modeled potential outcomes of different screening strategies, indicated that extra screening might reduce breast cancer mortality in those with dense breasts, but at a cost of more false-positive reports.

The second report, a review from the Kaiser Permanente Evidence-based Practice Center, reaffirmed the benefits of routine mammography for reducing deaths from breast cancer, but found no solid evidence that different strategies – including supplemental screening in women with denser breasts – lowered breast cancer mortality or the risk of progression to advanced cancer. Further studies may show which approaches work best to reduce breast cancer deaths, the report said.

In this instance, ACOG agreed with USPSTF: “Based on the lack of data, ACOG does not recommend routine use of alternative or adjunctive tests to screening mammography in women with dense breasts who are asymptomatic and have no additional risk factors.”

Women with dense breasts should still be encouraged to receive regular screening mammography, even if the results they get may not be as accurate as those for women with less dense breasts, said Diana L. Miglioretti, PhD, of the University of California, Davis, who worked on a report for the USPSTF guidelines.
 

What’s next?

Despite ongoing debate and uncertainties surrounding some breast screening guidance, support for ending copay requirements for follow-up tests after a positive mammogram finding is widespread.

According to Dr. Fendrick, the USPSTF should expand coverage of follow-up testing after a positive mammogram to ensure people receive routine screening and any necessary diagnostic tests, as it did with colon cancer.

Before 2021, patients could face high costs for a colonoscopy following a positive stool-based Cologuard test. But in 2021, the USPSTF said that positive results on stool-based tests would require follow-up with colonoscopy, defining this follow-up as part of the screening benefit. In 2022, Medicare followed by setting a policy that ended the copay for these follow-up colonoscopies.

For breast screening, there are efforts underway in Congress to end copays for breast screening. In May, Rep. Rosa DeLauro (D-Conn.) introduced a bill, the Find It Early Act, that would require both private and government insurers to cover the out-of-pocket costs for many women receiving screening with ultrasound and MRI.

When the USPSTF finalizes its breast screening guidelines, the recommendations will be woven into discussions between primary care physicians and patients about breast cancer screening.

As guidelines and evidence evolve, “we’re learning to adjust” and communicate these changes to patients, said Tochi Iroku-Malize, MD, president of the American Academy of Family Physicians.

However, gaps in the guidance will leave some open-ended questions about optimal screening practices and how much screening may cost.

Given that, Dr. Iroku-Malize takes many factors into account when discussing screening options with her patients. Based on the new information and the patient’s information, she said she will tell her patients, “We’re going to adjust our guidance as to what you need.”

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

A recent update to the U.S. recommendations for breast cancer screening is raising concerns about the costs associated with potential follow-up tests, while also renewing debates about the timing of these tests and the screening approaches used.
 

The U.S. Preventive Services Task Force is currently finalizing an update to its recommendations on breast cancer screening. In May, the task force released a proposed update that dropped the initial age for routine mammogram screening from 50 to 40.

The task force intends to give a “B” rating to this recommendation, which covers screening every other year up to age 74 for women deemed average risk for breast cancer.

The task force’s rating carries clout, A. Mark Fendrick, MD, director of the Value-Based Insurance Design at the University of Michigan, Ann Arbor, said in an interview.

For one, the Affordable Care Act requires that private insurers cover services that get top A or B marks from USPSTF without charging copays.

However, Dr. Fendrick noted, such coverage does not necessarily apply to follow-up testing when a routine mammogram comes back with a positive finding. The expense of follow-up testing may deter some women from seeking follow-up diagnostic imaging or biopsies after an abnormal result on a screening mammogram.

A recent analysis in JAMA Network Open found that women facing higher anticipated out-of-pocket costs for breast cancer diagnostic tests, based on their health insurance plan, were less likely to get that follow-up screening. For instance, the use of breast MRI decreased by nearly 24% between patients undergoing subsequent diagnostic testing in plans with the lowest out-of-pocket costs vs. those with the highest.

“The study’s central finding that some women who have an abnormal result on a mammogram may not get appropriate follow-up because of cost is worrisome,” said Dr. Fendrick and Ilana B. Richman, MD, MHS, in an accompanying commentary to the JAMA analysis. “On an individual level, high out-of-pocket costs may directly contribute to worse health outcomes or require individuals to use scarce financial resources that may otherwise be used for critical items such as food or rent.”

For patients to fully benefit from early detection, the USPSTF would also need to make clear that follow-up diagnostic mammograms are covered, Dr. Fendrick said.
 

The ongoing debates

Concerns over the costs of potential follow-up tests are not the only issues experts have highlighted since USPSTF released its updated draft guidance on screening mammography.

The task force’s proposed update has also reignited questions and uncertainties surrounding when to screen, how often, and what types are best.

When it comes to frequency, the major organizations that provide screening guidance don’t see eye to eye. The USPSTF recommends breast cancer screening every other year, while the American College of Radiology recommends screening every year because that approach leads to saves “the most lives.”

At this time, the American College of Obstetricians and Gynecologists guidance currently teeters in the middle, suggesting either annual or biennial screening and highlighting the pros and cons of either approach. According to ACOG, “annual screening intervals appear to result in the least number of breast cancer deaths, particularly in younger women, but at the cost of additional callbacks and biopsies.”

When to begin screening represents another point of contention. While some experts, such as ACOG, agree with the task force’s decision to lower the screening start age to 40, others point to the need for greater nuance on setting the appropriate screening age. The main issue: the task force’s draft sets a uniform age to begin screening, but the risk for breast cancer and breast cancer mortality is not uniform across different racial and ethnic groups.

A recent study published in JAMA Network Open found that, among women aged 40-49, breast cancer mortality was highest among Black women (27 deaths per 100,000 person-years) followed by White women (15 deaths per 100,000 person-years). Based on a recommended screening age of 50, the authors suggested that Black women should start screening at age 42, whereas White women could start at 51.

“These findings suggest that health policy makers and clinicians could consider an alternative, race and ethnicity–adapted approach in which Black female patients start screening earlier,” writes Tianhui Chen, PhD, of China’s Zhejiang Cancer Hospital and coauthor of the study.

Weighing in on the guidance, the nonprofit National Center for Health Research urged the task force to consider suggesting different screening schedules based on race and ethnicity data. That would mean the recommendation to start at age 40 should only apply to Black women and other groups with higher-than-average risk for breast cancer at a younger age.

“Women are capable of understanding why the age to start mammography screening may be different for women with different risk factors,” the National Center for Health Research wrote in a comment to USPSTF, provided to this news organization by request. “What is confusing is when some physician groups recommend annual mammograms for all women starting at age 40, even though the data do not support that recommendation.”

While the ACR agreed with the task force’s recommendation to lower the screening age, the organization suggested starting risk assessments based on racial variations in breast cancer incidence and death even earlier. Specifically, the ACR recommended that high-risk groups, such as Black women, get risk assessments by age 25 to determine whether mammography before age 40 is needed.

Screening options for women with dense breasts may be some of the most challenging to weigh. Having dense breasts increases an individual’s risk for breast cancer, and mammography alone is not as effective at identifying breast cancer among these women. However, the evidence on the benefits vs. harms of additional screening beyond mammography remains mixed.

As a result, the task force decided to maintain its “I” grade on additional screening beyond mammography for these women – a grade that indicates insufficient evidence to determine the benefits and harms for a service.

The task force largely based its decision on the findings of two key reports. One report from the Cancer Intervention and Surveillance Modeling Network, which modeled potential outcomes of different screening strategies, indicated that extra screening might reduce breast cancer mortality in those with dense breasts, but at a cost of more false-positive reports.

The second report, a review from the Kaiser Permanente Evidence-based Practice Center, reaffirmed the benefits of routine mammography for reducing deaths from breast cancer, but found no solid evidence that different strategies – including supplemental screening in women with denser breasts – lowered breast cancer mortality or the risk of progression to advanced cancer. Further studies may show which approaches work best to reduce breast cancer deaths, the report said.

In this instance, ACOG agreed with USPSTF: “Based on the lack of data, ACOG does not recommend routine use of alternative or adjunctive tests to screening mammography in women with dense breasts who are asymptomatic and have no additional risk factors.”

Women with dense breasts should still be encouraged to receive regular screening mammography, even if the results they get may not be as accurate as those for women with less dense breasts, said Diana L. Miglioretti, PhD, of the University of California, Davis, who worked on a report for the USPSTF guidelines.
 

What’s next?

Despite ongoing debate and uncertainties surrounding some breast screening guidance, support for ending copay requirements for follow-up tests after a positive mammogram finding is widespread.

According to Dr. Fendrick, the USPSTF should expand coverage of follow-up testing after a positive mammogram to ensure people receive routine screening and any necessary diagnostic tests, as it did with colon cancer.

Before 2021, patients could face high costs for a colonoscopy following a positive stool-based Cologuard test. But in 2021, the USPSTF said that positive results on stool-based tests would require follow-up with colonoscopy, defining this follow-up as part of the screening benefit. In 2022, Medicare followed by setting a policy that ended the copay for these follow-up colonoscopies.

For breast screening, there are efforts underway in Congress to end copays for breast screening. In May, Rep. Rosa DeLauro (D-Conn.) introduced a bill, the Find It Early Act, that would require both private and government insurers to cover the out-of-pocket costs for many women receiving screening with ultrasound and MRI.

When the USPSTF finalizes its breast screening guidelines, the recommendations will be woven into discussions between primary care physicians and patients about breast cancer screening.

As guidelines and evidence evolve, “we’re learning to adjust” and communicate these changes to patients, said Tochi Iroku-Malize, MD, president of the American Academy of Family Physicians.

However, gaps in the guidance will leave some open-ended questions about optimal screening practices and how much screening may cost.

Given that, Dr. Iroku-Malize takes many factors into account when discussing screening options with her patients. Based on the new information and the patient’s information, she said she will tell her patients, “We’re going to adjust our guidance as to what you need.”

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

A recent update to the U.S. recommendations for breast cancer screening is raising concerns about the costs associated with potential follow-up tests, while also renewing debates about the timing of these tests and the screening approaches used.
 

The U.S. Preventive Services Task Force is currently finalizing an update to its recommendations on breast cancer screening. In May, the task force released a proposed update that dropped the initial age for routine mammogram screening from 50 to 40.

The task force intends to give a “B” rating to this recommendation, which covers screening every other year up to age 74 for women deemed average risk for breast cancer.

The task force’s rating carries clout, A. Mark Fendrick, MD, director of the Value-Based Insurance Design at the University of Michigan, Ann Arbor, said in an interview.

For one, the Affordable Care Act requires that private insurers cover services that get top A or B marks from USPSTF without charging copays.

However, Dr. Fendrick noted, such coverage does not necessarily apply to follow-up testing when a routine mammogram comes back with a positive finding. The expense of follow-up testing may deter some women from seeking follow-up diagnostic imaging or biopsies after an abnormal result on a screening mammogram.

A recent analysis in JAMA Network Open found that women facing higher anticipated out-of-pocket costs for breast cancer diagnostic tests, based on their health insurance plan, were less likely to get that follow-up screening. For instance, the use of breast MRI decreased by nearly 24% between patients undergoing subsequent diagnostic testing in plans with the lowest out-of-pocket costs vs. those with the highest.

“The study’s central finding that some women who have an abnormal result on a mammogram may not get appropriate follow-up because of cost is worrisome,” said Dr. Fendrick and Ilana B. Richman, MD, MHS, in an accompanying commentary to the JAMA analysis. “On an individual level, high out-of-pocket costs may directly contribute to worse health outcomes or require individuals to use scarce financial resources that may otherwise be used for critical items such as food or rent.”

For patients to fully benefit from early detection, the USPSTF would also need to make clear that follow-up diagnostic mammograms are covered, Dr. Fendrick said.
 

The ongoing debates

Concerns over the costs of potential follow-up tests are not the only issues experts have highlighted since USPSTF released its updated draft guidance on screening mammography.

The task force’s proposed update has also reignited questions and uncertainties surrounding when to screen, how often, and what types are best.

When it comes to frequency, the major organizations that provide screening guidance don’t see eye to eye. The USPSTF recommends breast cancer screening every other year, while the American College of Radiology recommends screening every year because that approach leads to saves “the most lives.”

At this time, the American College of Obstetricians and Gynecologists guidance currently teeters in the middle, suggesting either annual or biennial screening and highlighting the pros and cons of either approach. According to ACOG, “annual screening intervals appear to result in the least number of breast cancer deaths, particularly in younger women, but at the cost of additional callbacks and biopsies.”

When to begin screening represents another point of contention. While some experts, such as ACOG, agree with the task force’s decision to lower the screening start age to 40, others point to the need for greater nuance on setting the appropriate screening age. The main issue: the task force’s draft sets a uniform age to begin screening, but the risk for breast cancer and breast cancer mortality is not uniform across different racial and ethnic groups.

A recent study published in JAMA Network Open found that, among women aged 40-49, breast cancer mortality was highest among Black women (27 deaths per 100,000 person-years) followed by White women (15 deaths per 100,000 person-years). Based on a recommended screening age of 50, the authors suggested that Black women should start screening at age 42, whereas White women could start at 51.

“These findings suggest that health policy makers and clinicians could consider an alternative, race and ethnicity–adapted approach in which Black female patients start screening earlier,” writes Tianhui Chen, PhD, of China’s Zhejiang Cancer Hospital and coauthor of the study.

Weighing in on the guidance, the nonprofit National Center for Health Research urged the task force to consider suggesting different screening schedules based on race and ethnicity data. That would mean the recommendation to start at age 40 should only apply to Black women and other groups with higher-than-average risk for breast cancer at a younger age.

“Women are capable of understanding why the age to start mammography screening may be different for women with different risk factors,” the National Center for Health Research wrote in a comment to USPSTF, provided to this news organization by request. “What is confusing is when some physician groups recommend annual mammograms for all women starting at age 40, even though the data do not support that recommendation.”

While the ACR agreed with the task force’s recommendation to lower the screening age, the organization suggested starting risk assessments based on racial variations in breast cancer incidence and death even earlier. Specifically, the ACR recommended that high-risk groups, such as Black women, get risk assessments by age 25 to determine whether mammography before age 40 is needed.

Screening options for women with dense breasts may be some of the most challenging to weigh. Having dense breasts increases an individual’s risk for breast cancer, and mammography alone is not as effective at identifying breast cancer among these women. However, the evidence on the benefits vs. harms of additional screening beyond mammography remains mixed.

As a result, the task force decided to maintain its “I” grade on additional screening beyond mammography for these women – a grade that indicates insufficient evidence to determine the benefits and harms for a service.

The task force largely based its decision on the findings of two key reports. One report from the Cancer Intervention and Surveillance Modeling Network, which modeled potential outcomes of different screening strategies, indicated that extra screening might reduce breast cancer mortality in those with dense breasts, but at a cost of more false-positive reports.

The second report, a review from the Kaiser Permanente Evidence-based Practice Center, reaffirmed the benefits of routine mammography for reducing deaths from breast cancer, but found no solid evidence that different strategies – including supplemental screening in women with denser breasts – lowered breast cancer mortality or the risk of progression to advanced cancer. Further studies may show which approaches work best to reduce breast cancer deaths, the report said.

In this instance, ACOG agreed with USPSTF: “Based on the lack of data, ACOG does not recommend routine use of alternative or adjunctive tests to screening mammography in women with dense breasts who are asymptomatic and have no additional risk factors.”

Women with dense breasts should still be encouraged to receive regular screening mammography, even if the results they get may not be as accurate as those for women with less dense breasts, said Diana L. Miglioretti, PhD, of the University of California, Davis, who worked on a report for the USPSTF guidelines.
 

What’s next?

Despite ongoing debate and uncertainties surrounding some breast screening guidance, support for ending copay requirements for follow-up tests after a positive mammogram finding is widespread.

According to Dr. Fendrick, the USPSTF should expand coverage of follow-up testing after a positive mammogram to ensure people receive routine screening and any necessary diagnostic tests, as it did with colon cancer.

Before 2021, patients could face high costs for a colonoscopy following a positive stool-based Cologuard test. But in 2021, the USPSTF said that positive results on stool-based tests would require follow-up with colonoscopy, defining this follow-up as part of the screening benefit. In 2022, Medicare followed by setting a policy that ended the copay for these follow-up colonoscopies.

For breast screening, there are efforts underway in Congress to end copays for breast screening. In May, Rep. Rosa DeLauro (D-Conn.) introduced a bill, the Find It Early Act, that would require both private and government insurers to cover the out-of-pocket costs for many women receiving screening with ultrasound and MRI.

When the USPSTF finalizes its breast screening guidelines, the recommendations will be woven into discussions between primary care physicians and patients about breast cancer screening.

As guidelines and evidence evolve, “we’re learning to adjust” and communicate these changes to patients, said Tochi Iroku-Malize, MD, president of the American Academy of Family Physicians.

However, gaps in the guidance will leave some open-ended questions about optimal screening practices and how much screening may cost.

Given that, Dr. Iroku-Malize takes many factors into account when discussing screening options with her patients. Based on the new information and the patient’s information, she said she will tell her patients, “We’re going to adjust our guidance as to what you need.”

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

After 15 years of researching what works well in oncology – and where the field has gone awry – Christopher Booth, MD, had a career moment.

“As I approached mid-career, I realized publishing and describing problems wasn’t fulfilling. It wasn’t doing enough,” recalled Dr. Booth, an oncologist and professor at Queen’s University, Kingston, Ont. “I wanted to change mindsets and change systems so that things actually improved for the better for patients.”

His colleague, Bishal Gyawali, MD, PhD, described a similar epiphany. As a trainee, he noticed that the real-world effects of some so-called blockbuster cancer drugs too often failed to measure up to the hype.

“I realized we were lacking common sense in oncology,” said Dr. Gyawali, a medical oncologist and assistant professor at Queen’s University.

In 2019, Dr. Gyawali launched a Medscape column addressing what he considers to be that lack of common sense, and in 2022, he and Dr. Booth published a similarly titled opinion piece in Nature Medicine. The core idea: The cancer community needs to prioritize cancer treatments that benefit patients, treatments that meaningfully improve survival and quality of life.

Aaron Goodman, MD, a hematologist and associate professor at UC San Diego Health, was on the same page. He’d been interested in the evidence-based medicine movement since his time as a hematology fellow when that movement was “a bit of a counterculture,” he explained.

Dr. Goodman and Dr. Booth connected through their common interests and collaborated on a 2021 paper exploring the discomfort clinicians might feel when a patient’s needs fall on the “edge of oncology”: that is, when the guideline-recommended standard of care offers marginal benefit, at best, and could, at worst, cause patient harm.

“We said, ‘Now is the time to make change,’ ” he recalled. It was time to stop talking and do something.
 

Common sense and a common purpose

Dr. Booth, Dr. Gyawali, and Dr. Goodman joined forces and, with the backing of a philanthropist who had experience as a patient with cancer, convened an organizing committee of more than 30 like-minded oncologists and patient advocates from across the globe.

The group convened for a 3-day “meeting of the minds” in Kingston in April and laid out their intentions in a position paper published online in The Lancet Oncology.

The publication marks the official launch of an ambitious, multipronged, global initiative to enact change: Common Sense Oncology, a new patient-centered movement in cancer care.

In their paper, the committee outline the vision for Common Sense Oncology. The mission: prioritize patient-centered and equitable care by focusing on treatments that improve survival and quality of life, communication that promotes informed decision-making, and systems that ensure access to all patients.

However, increasingly, the cancer community faces a “troubling paradox,” the team wrote in The Lancet. In some instance, treatments that bring minimal benefit are overused while those that can make a meaningful difference in patients’ lives are not accessible to most worldwide.

One reason for this shift: Commercial interests, rather than patient interests, appear to be driving cancer research and care. The team explained, for instance, that over the past few decades, clinical trials have largely pivoted from publicly funded efforts to industry funded ones “designed to achieve regulatory approval or commercial advantage, [often] at the expense of investigating new approaches to surgery, radiotherapy, palliative care, and prevention.”

But “patients deserve better,” the group wrote.

The team outlined three pillars for the initiative: evidence generation, evidence interpretation, and evidence communication.

The evidence generation pillar will aim to improve trial design and reporting to prioritize outcomes that matter to patients.

“One concern is that over the last 10 years or so, most of our new treatments have had very, very small benefits, and we think the bar has dropped too low,” Dr. Booth said, explaining that many trials have moved away from focusing on improving survival and quality of life and toward detecting small differences between treatments on other endpoints – namely progression-free survival. “Those small benefits need to be balanced against the very real risks to our patients.”

The evidence interpretation pillar will aim to foster critical thinking so that clinicians can better identify poorly designed or reported trials and help patients make more informed decisions.

Lastly, the evidence communication pillar will focus on fostering better communication about treatment options among patients, the public, and policymakers. Without clear and thoughtful communication, patients may have unrealistic expectations about the effectiveness of treatments that offer only marginal clinical benefits.

The team also emphasized a need to focus on improving global equity and access to affordable treatments so all patients can benefit from care that extends survival or quality of life.

It’s an ambitious undertaking, especially for a group of full-time clinicians, researchers, and patient advocates “volunteering their time for societal good,” said Dr. Gyawali, but the project teams intend to hit the ground running.

The team has established short-term targets, such as identifying deficiencies in data interpretation within education programs within 6 months and developing educational materials that begin to correct those deficiencies within 12 months, Dr. Booth explained. In the longer term, the team will also aim to design clinical trials that focus on patient outcomes, such as overall survival and quality of life.

Breast cancer survivor and patient advocate Michelle Tregear, PhD, who was recruited to help with Common Sense Oncology, also hopes the initiative will lead to better regulatory control that requires trial sponsors to “focus on what matters to patients, not on surrogate endpoints.”

When it comes to clinical trials, “more, more, more is not always better,” said Dr. Tregear, director of Education and Training Programs for patient advocates at the National Breast Cancer Coalition, Washington, D.C. “Industry interests are not always aligned with patient interests,” and “the system, by and large, is not addressing questions that really matter to patients and their families.”

Although “it’s a tall order to change the direction that we’re going in,” Dr. Tregear is up to the challenge of helping raise awareness, which will hopefully spur patients to demand change.

When Dr. Goodman announced the Common Sense Oncology initiative on Twitter, the news brought excitement, with many oncologists asking to join.

With its sweeping, ambitious goals, the Common Sense Oncology initiative has a long road ahead. Figuring out how to implement some of its aims in practice will take time, Dr. Booth acknowledges, and the initial launch marks the first steps, which will continue to evolve over time.

“We’re not proposing we have all the answers or that we know what every patient would want – we’re saying we’ve not done a good job of communicating to patients the relative benefits and risks of different treatments,” Dr. Booth explained. “We want to celebrate and promote what helps and speak out about what’s not in the best interest of patients.”

Dr. Goodman reported consulting fees from Seattle Genetics and speaking honoraria from Curio. Dr. Booth, Dr. Gyawali, and Dr. Tregear reported having no financial conflicts of interest.

A version of this article appeared on Medscape.com.

After 15 years of researching what works well in oncology – and where the field has gone awry – Christopher Booth, MD, had a career moment.

“As I approached mid-career, I realized publishing and describing problems wasn’t fulfilling. It wasn’t doing enough,” recalled Dr. Booth, an oncologist and professor at Queen’s University, Kingston, Ont. “I wanted to change mindsets and change systems so that things actually improved for the better for patients.”

His colleague, Bishal Gyawali, MD, PhD, described a similar epiphany. As a trainee, he noticed that the real-world effects of some so-called blockbuster cancer drugs too often failed to measure up to the hype.

“I realized we were lacking common sense in oncology,” said Dr. Gyawali, a medical oncologist and assistant professor at Queen’s University.

In 2019, Dr. Gyawali launched a Medscape column addressing what he considers to be that lack of common sense, and in 2022, he and Dr. Booth published a similarly titled opinion piece in Nature Medicine. The core idea: The cancer community needs to prioritize cancer treatments that benefit patients, treatments that meaningfully improve survival and quality of life.

Aaron Goodman, MD, a hematologist and associate professor at UC San Diego Health, was on the same page. He’d been interested in the evidence-based medicine movement since his time as a hematology fellow when that movement was “a bit of a counterculture,” he explained.

Dr. Goodman and Dr. Booth connected through their common interests and collaborated on a 2021 paper exploring the discomfort clinicians might feel when a patient’s needs fall on the “edge of oncology”: that is, when the guideline-recommended standard of care offers marginal benefit, at best, and could, at worst, cause patient harm.

“We said, ‘Now is the time to make change,’ ” he recalled. It was time to stop talking and do something.
 

Common sense and a common purpose

Dr. Booth, Dr. Gyawali, and Dr. Goodman joined forces and, with the backing of a philanthropist who had experience as a patient with cancer, convened an organizing committee of more than 30 like-minded oncologists and patient advocates from across the globe.

The group convened for a 3-day “meeting of the minds” in Kingston in April and laid out their intentions in a position paper published online in The Lancet Oncology.

The publication marks the official launch of an ambitious, multipronged, global initiative to enact change: Common Sense Oncology, a new patient-centered movement in cancer care.

In their paper, the committee outline the vision for Common Sense Oncology. The mission: prioritize patient-centered and equitable care by focusing on treatments that improve survival and quality of life, communication that promotes informed decision-making, and systems that ensure access to all patients.

However, increasingly, the cancer community faces a “troubling paradox,” the team wrote in The Lancet. In some instance, treatments that bring minimal benefit are overused while those that can make a meaningful difference in patients’ lives are not accessible to most worldwide.

One reason for this shift: Commercial interests, rather than patient interests, appear to be driving cancer research and care. The team explained, for instance, that over the past few decades, clinical trials have largely pivoted from publicly funded efforts to industry funded ones “designed to achieve regulatory approval or commercial advantage, [often] at the expense of investigating new approaches to surgery, radiotherapy, palliative care, and prevention.”

But “patients deserve better,” the group wrote.

The team outlined three pillars for the initiative: evidence generation, evidence interpretation, and evidence communication.

The evidence generation pillar will aim to improve trial design and reporting to prioritize outcomes that matter to patients.

“One concern is that over the last 10 years or so, most of our new treatments have had very, very small benefits, and we think the bar has dropped too low,” Dr. Booth said, explaining that many trials have moved away from focusing on improving survival and quality of life and toward detecting small differences between treatments on other endpoints – namely progression-free survival. “Those small benefits need to be balanced against the very real risks to our patients.”

The evidence interpretation pillar will aim to foster critical thinking so that clinicians can better identify poorly designed or reported trials and help patients make more informed decisions.

Lastly, the evidence communication pillar will focus on fostering better communication about treatment options among patients, the public, and policymakers. Without clear and thoughtful communication, patients may have unrealistic expectations about the effectiveness of treatments that offer only marginal clinical benefits.

The team also emphasized a need to focus on improving global equity and access to affordable treatments so all patients can benefit from care that extends survival or quality of life.

It’s an ambitious undertaking, especially for a group of full-time clinicians, researchers, and patient advocates “volunteering their time for societal good,” said Dr. Gyawali, but the project teams intend to hit the ground running.

The team has established short-term targets, such as identifying deficiencies in data interpretation within education programs within 6 months and developing educational materials that begin to correct those deficiencies within 12 months, Dr. Booth explained. In the longer term, the team will also aim to design clinical trials that focus on patient outcomes, such as overall survival and quality of life.

Breast cancer survivor and patient advocate Michelle Tregear, PhD, who was recruited to help with Common Sense Oncology, also hopes the initiative will lead to better regulatory control that requires trial sponsors to “focus on what matters to patients, not on surrogate endpoints.”

When it comes to clinical trials, “more, more, more is not always better,” said Dr. Tregear, director of Education and Training Programs for patient advocates at the National Breast Cancer Coalition, Washington, D.C. “Industry interests are not always aligned with patient interests,” and “the system, by and large, is not addressing questions that really matter to patients and their families.”

Although “it’s a tall order to change the direction that we’re going in,” Dr. Tregear is up to the challenge of helping raise awareness, which will hopefully spur patients to demand change.

When Dr. Goodman announced the Common Sense Oncology initiative on Twitter, the news brought excitement, with many oncologists asking to join.

With its sweeping, ambitious goals, the Common Sense Oncology initiative has a long road ahead. Figuring out how to implement some of its aims in practice will take time, Dr. Booth acknowledges, and the initial launch marks the first steps, which will continue to evolve over time.

“We’re not proposing we have all the answers or that we know what every patient would want – we’re saying we’ve not done a good job of communicating to patients the relative benefits and risks of different treatments,” Dr. Booth explained. “We want to celebrate and promote what helps and speak out about what’s not in the best interest of patients.”

Dr. Goodman reported consulting fees from Seattle Genetics and speaking honoraria from Curio. Dr. Booth, Dr. Gyawali, and Dr. Tregear reported having no financial conflicts of interest.

A version of this article appeared on Medscape.com.

After 15 years of researching what works well in oncology – and where the field has gone awry – Christopher Booth, MD, had a career moment.

“As I approached mid-career, I realized publishing and describing problems wasn’t fulfilling. It wasn’t doing enough,” recalled Dr. Booth, an oncologist and professor at Queen’s University, Kingston, Ont. “I wanted to change mindsets and change systems so that things actually improved for the better for patients.”

His colleague, Bishal Gyawali, MD, PhD, described a similar epiphany. As a trainee, he noticed that the real-world effects of some so-called blockbuster cancer drugs too often failed to measure up to the hype.

“I realized we were lacking common sense in oncology,” said Dr. Gyawali, a medical oncologist and assistant professor at Queen’s University.

In 2019, Dr. Gyawali launched a Medscape column addressing what he considers to be that lack of common sense, and in 2022, he and Dr. Booth published a similarly titled opinion piece in Nature Medicine. The core idea: The cancer community needs to prioritize cancer treatments that benefit patients, treatments that meaningfully improve survival and quality of life.

Aaron Goodman, MD, a hematologist and associate professor at UC San Diego Health, was on the same page. He’d been interested in the evidence-based medicine movement since his time as a hematology fellow when that movement was “a bit of a counterculture,” he explained.

Dr. Goodman and Dr. Booth connected through their common interests and collaborated on a 2021 paper exploring the discomfort clinicians might feel when a patient’s needs fall on the “edge of oncology”: that is, when the guideline-recommended standard of care offers marginal benefit, at best, and could, at worst, cause patient harm.

“We said, ‘Now is the time to make change,’ ” he recalled. It was time to stop talking and do something.
 

Common sense and a common purpose

Dr. Booth, Dr. Gyawali, and Dr. Goodman joined forces and, with the backing of a philanthropist who had experience as a patient with cancer, convened an organizing committee of more than 30 like-minded oncologists and patient advocates from across the globe.

The group convened for a 3-day “meeting of the minds” in Kingston in April and laid out their intentions in a position paper published online in The Lancet Oncology.

The publication marks the official launch of an ambitious, multipronged, global initiative to enact change: Common Sense Oncology, a new patient-centered movement in cancer care.

In their paper, the committee outline the vision for Common Sense Oncology. The mission: prioritize patient-centered and equitable care by focusing on treatments that improve survival and quality of life, communication that promotes informed decision-making, and systems that ensure access to all patients.

However, increasingly, the cancer community faces a “troubling paradox,” the team wrote in The Lancet. In some instance, treatments that bring minimal benefit are overused while those that can make a meaningful difference in patients’ lives are not accessible to most worldwide.

One reason for this shift: Commercial interests, rather than patient interests, appear to be driving cancer research and care. The team explained, for instance, that over the past few decades, clinical trials have largely pivoted from publicly funded efforts to industry funded ones “designed to achieve regulatory approval or commercial advantage, [often] at the expense of investigating new approaches to surgery, radiotherapy, palliative care, and prevention.”

But “patients deserve better,” the group wrote.

The team outlined three pillars for the initiative: evidence generation, evidence interpretation, and evidence communication.

The evidence generation pillar will aim to improve trial design and reporting to prioritize outcomes that matter to patients.

“One concern is that over the last 10 years or so, most of our new treatments have had very, very small benefits, and we think the bar has dropped too low,” Dr. Booth said, explaining that many trials have moved away from focusing on improving survival and quality of life and toward detecting small differences between treatments on other endpoints – namely progression-free survival. “Those small benefits need to be balanced against the very real risks to our patients.”

The evidence interpretation pillar will aim to foster critical thinking so that clinicians can better identify poorly designed or reported trials and help patients make more informed decisions.

Lastly, the evidence communication pillar will focus on fostering better communication about treatment options among patients, the public, and policymakers. Without clear and thoughtful communication, patients may have unrealistic expectations about the effectiveness of treatments that offer only marginal clinical benefits.

The team also emphasized a need to focus on improving global equity and access to affordable treatments so all patients can benefit from care that extends survival or quality of life.

It’s an ambitious undertaking, especially for a group of full-time clinicians, researchers, and patient advocates “volunteering their time for societal good,” said Dr. Gyawali, but the project teams intend to hit the ground running.

The team has established short-term targets, such as identifying deficiencies in data interpretation within education programs within 6 months and developing educational materials that begin to correct those deficiencies within 12 months, Dr. Booth explained. In the longer term, the team will also aim to design clinical trials that focus on patient outcomes, such as overall survival and quality of life.

Breast cancer survivor and patient advocate Michelle Tregear, PhD, who was recruited to help with Common Sense Oncology, also hopes the initiative will lead to better regulatory control that requires trial sponsors to “focus on what matters to patients, not on surrogate endpoints.”

When it comes to clinical trials, “more, more, more is not always better,” said Dr. Tregear, director of Education and Training Programs for patient advocates at the National Breast Cancer Coalition, Washington, D.C. “Industry interests are not always aligned with patient interests,” and “the system, by and large, is not addressing questions that really matter to patients and their families.”

Although “it’s a tall order to change the direction that we’re going in,” Dr. Tregear is up to the challenge of helping raise awareness, which will hopefully spur patients to demand change.

When Dr. Goodman announced the Common Sense Oncology initiative on Twitter, the news brought excitement, with many oncologists asking to join.

With its sweeping, ambitious goals, the Common Sense Oncology initiative has a long road ahead. Figuring out how to implement some of its aims in practice will take time, Dr. Booth acknowledges, and the initial launch marks the first steps, which will continue to evolve over time.

“We’re not proposing we have all the answers or that we know what every patient would want – we’re saying we’ve not done a good job of communicating to patients the relative benefits and risks of different treatments,” Dr. Booth explained. “We want to celebrate and promote what helps and speak out about what’s not in the best interest of patients.”

Dr. Goodman reported consulting fees from Seattle Genetics and speaking honoraria from Curio. Dr. Booth, Dr. Gyawali, and Dr. Tregear reported having no financial conflicts of interest.

A version of this article appeared on Medscape.com.

Implementing stewardship strategies for immune checkpoint inhibitor (ICI) therapy, including personalized weight-based dosing, dose rounding, and pharmacy-level vial sharing, could generate savings of as much as $74 million each year for the Veterans Health Administration (VHA), a new analysis suggests.

That $74 million in savings would translate to nearly 14% less spent on ICI therapy annually.

“Our work suggests that implementing these strategies across the VHA could lead to tens of millions of dollars in annual savings – and that’s just for immunotherapy – without sacrificing outcomes,” first author Alex Bryant, MD, University of Michigan, Ann Arbor, said in an interview.

The study was published in Health Affairs.

ICI therapy is used in about 40 unique cancer indications and, in 2020, accounted for more than $6 billion in Medicare Part B spending.

Two of the most prescribed ICIs – pembrolizumab and nivolumab – initially received their U.S. approval at personalized weight-based doses. But at the request of the manufacturers, the Food and Drug Administration approved “one-size-fits-all” flat doses, despite a lack of data to support this strategy compared with weight-based dosing.

With a fixed dose strategy, “patients with cancer not only tend to get too high a dose of the drug, but costs go up significantly,” Daniel Goldstein, MD, a medical oncologist at the Rabin Medical Center, Petah Tikva, Israel,  told this news organization last year. “Why should we give a higher dose with the same efficacy when that dose will cost significantly more and has the potential to increase adverse events?”

To compare the cost of a weight-based vs. fixed-dose strategy, Dr. Bryant and colleagues conducted a simulation analysis under four stewardship scenarios, using data from the VHA and Medicare drug prices. Strategy one looked at weight-based dosing; strategy two combined weight-based dosing and dose rounding but not single-use vial sharing; strategy three used weight-based dosing and single-use vial sharing but not dose rounding; and strategy four, the most aggressive, combined all three.

ICIs in the VHA national formulary included pembrolizumab, nivolumab, atezolizumab, durvalumab, and cemiplimab-rwlc.

Using an algorithm to extract data, the team identified 49,851 administration events in 8,276 unique patients in 2021 – just over half were pembrolizumab, nearly 23% were nivolumab, and the remaining 26% largely included atezolizumab (12.1%) and durvalumab (11.9%).

The team found that the VHA spends roughly $537 million annually on ICIs. But implementing the stewardship measures that combined weight-based dosing, dose rounding, and vial sharing could save the VHA $74 million, or about 14%, annually on ICIs.

Most of the savings came from dosing changes to pembrolizumab and nivolumab, with greater savings achieved by combining more stewardship strategies. For instance, using strategy one (weight-based dosing alone) could lead to annual pembrolizumab savings of $14 million. Adding dose rounding (strategy two) could reduce pembrolizumab spending by $24 million. And using strategy four, with an unlimited window for vial sharing, could mean annual savings of nearly $60 million.

“Our results should prompt cost-conscious systems and payers to ask whether the amounts of drugs they’re providing to patients and how they go about making those doses are the most cost-effective approaches,” said corresponding author Garth W. Strohbehn, MD, of the University of Michigan and the VA Ann Arbor Healthcare System.

Dr. Strohbehn said the prospect of adopting these strategies hinges on several factors, with financial incentives at the prescriber and medical center level likely being the most influential.

“In fee-for-service systems, reimbursement scales with the amount of drug administered, so there can be a financial disincentive to decreasing overall drug usage,” Dr. Strohbehn explained.

“Conversely, integrated systems such as Kaiser Permanente or the VHA and large self-insured employers are incentivized to contain costs and take great care of patients, so they may be more inclined to promote these strategies,” he added.

However, Adam C. Powell, PhD, president, Payer+Provider Syndicate, who wasn’t involved in the analysis, cautioned that such a shift may come with unintended consequences.

The Infrastructure, Investment, and Jobs Act of 2021 let the Centers for Medicare and Medicaid Services seek reimbursement for discarded drugs – in effect, changing the reimbursement model for medications. That led pharmaceutical manufacturers to respond in kind by changing the dosing model, Dr. Powell said. 

“Drugs that previously had personalized weight-based dosing were moved to uniform flat dosing, eliminating the potential for the manufacturer to have to issue a reimbursement if the patient’s personalized dose fell short of the amount in the single-use vial,” Dr. Powell added.  

If there is a substantial migration to weight-based dosing, “it is possible that pharmaceutical manufacturers will rethink their dosing and pricing models, just as happened previously,” he cautioned.

However, these strategies could also provide relief for another escalating issue: drug shortages. Especially in the current moment, having a stewardship mindset, “might be helpful in navigating drug shortages,” Dr. Strohbehn said.

This research had no commercial funding. Dr. Bryant, Dr. Strohbehn, and Dr. Powell report no relevant financial relationships.
 

A version of this article appeared on Medscape.com.

Implementing stewardship strategies for immune checkpoint inhibitor (ICI) therapy, including personalized weight-based dosing, dose rounding, and pharmacy-level vial sharing, could generate savings of as much as $74 million each year for the Veterans Health Administration (VHA), a new analysis suggests.

That $74 million in savings would translate to nearly 14% less spent on ICI therapy annually.

“Our work suggests that implementing these strategies across the VHA could lead to tens of millions of dollars in annual savings – and that’s just for immunotherapy – without sacrificing outcomes,” first author Alex Bryant, MD, University of Michigan, Ann Arbor, said in an interview.

The study was published in Health Affairs.

ICI therapy is used in about 40 unique cancer indications and, in 2020, accounted for more than $6 billion in Medicare Part B spending.

Two of the most prescribed ICIs – pembrolizumab and nivolumab – initially received their U.S. approval at personalized weight-based doses. But at the request of the manufacturers, the Food and Drug Administration approved “one-size-fits-all” flat doses, despite a lack of data to support this strategy compared with weight-based dosing.

With a fixed dose strategy, “patients with cancer not only tend to get too high a dose of the drug, but costs go up significantly,” Daniel Goldstein, MD, a medical oncologist at the Rabin Medical Center, Petah Tikva, Israel,  told this news organization last year. “Why should we give a higher dose with the same efficacy when that dose will cost significantly more and has the potential to increase adverse events?”

To compare the cost of a weight-based vs. fixed-dose strategy, Dr. Bryant and colleagues conducted a simulation analysis under four stewardship scenarios, using data from the VHA and Medicare drug prices. Strategy one looked at weight-based dosing; strategy two combined weight-based dosing and dose rounding but not single-use vial sharing; strategy three used weight-based dosing and single-use vial sharing but not dose rounding; and strategy four, the most aggressive, combined all three.

ICIs in the VHA national formulary included pembrolizumab, nivolumab, atezolizumab, durvalumab, and cemiplimab-rwlc.

Using an algorithm to extract data, the team identified 49,851 administration events in 8,276 unique patients in 2021 – just over half were pembrolizumab, nearly 23% were nivolumab, and the remaining 26% largely included atezolizumab (12.1%) and durvalumab (11.9%).

The team found that the VHA spends roughly $537 million annually on ICIs. But implementing the stewardship measures that combined weight-based dosing, dose rounding, and vial sharing could save the VHA $74 million, or about 14%, annually on ICIs.

Most of the savings came from dosing changes to pembrolizumab and nivolumab, with greater savings achieved by combining more stewardship strategies. For instance, using strategy one (weight-based dosing alone) could lead to annual pembrolizumab savings of $14 million. Adding dose rounding (strategy two) could reduce pembrolizumab spending by $24 million. And using strategy four, with an unlimited window for vial sharing, could mean annual savings of nearly $60 million.

“Our results should prompt cost-conscious systems and payers to ask whether the amounts of drugs they’re providing to patients and how they go about making those doses are the most cost-effective approaches,” said corresponding author Garth W. Strohbehn, MD, of the University of Michigan and the VA Ann Arbor Healthcare System.

Dr. Strohbehn said the prospect of adopting these strategies hinges on several factors, with financial incentives at the prescriber and medical center level likely being the most influential.

“In fee-for-service systems, reimbursement scales with the amount of drug administered, so there can be a financial disincentive to decreasing overall drug usage,” Dr. Strohbehn explained.

“Conversely, integrated systems such as Kaiser Permanente or the VHA and large self-insured employers are incentivized to contain costs and take great care of patients, so they may be more inclined to promote these strategies,” he added.

However, Adam C. Powell, PhD, president, Payer+Provider Syndicate, who wasn’t involved in the analysis, cautioned that such a shift may come with unintended consequences.

The Infrastructure, Investment, and Jobs Act of 2021 let the Centers for Medicare and Medicaid Services seek reimbursement for discarded drugs – in effect, changing the reimbursement model for medications. That led pharmaceutical manufacturers to respond in kind by changing the dosing model, Dr. Powell said. 

“Drugs that previously had personalized weight-based dosing were moved to uniform flat dosing, eliminating the potential for the manufacturer to have to issue a reimbursement if the patient’s personalized dose fell short of the amount in the single-use vial,” Dr. Powell added.  

If there is a substantial migration to weight-based dosing, “it is possible that pharmaceutical manufacturers will rethink their dosing and pricing models, just as happened previously,” he cautioned.

However, these strategies could also provide relief for another escalating issue: drug shortages. Especially in the current moment, having a stewardship mindset, “might be helpful in navigating drug shortages,” Dr. Strohbehn said.

This research had no commercial funding. Dr. Bryant, Dr. Strohbehn, and Dr. Powell report no relevant financial relationships.
 

A version of this article appeared on Medscape.com.

Implementing stewardship strategies for immune checkpoint inhibitor (ICI) therapy, including personalized weight-based dosing, dose rounding, and pharmacy-level vial sharing, could generate savings of as much as $74 million each year for the Veterans Health Administration (VHA), a new analysis suggests.

That $74 million in savings would translate to nearly 14% less spent on ICI therapy annually.

“Our work suggests that implementing these strategies across the VHA could lead to tens of millions of dollars in annual savings – and that’s just for immunotherapy – without sacrificing outcomes,” first author Alex Bryant, MD, University of Michigan, Ann Arbor, said in an interview.

The study was published in Health Affairs.

ICI therapy is used in about 40 unique cancer indications and, in 2020, accounted for more than $6 billion in Medicare Part B spending.

Two of the most prescribed ICIs – pembrolizumab and nivolumab – initially received their U.S. approval at personalized weight-based doses. But at the request of the manufacturers, the Food and Drug Administration approved “one-size-fits-all” flat doses, despite a lack of data to support this strategy compared with weight-based dosing.

With a fixed dose strategy, “patients with cancer not only tend to get too high a dose of the drug, but costs go up significantly,” Daniel Goldstein, MD, a medical oncologist at the Rabin Medical Center, Petah Tikva, Israel,  told this news organization last year. “Why should we give a higher dose with the same efficacy when that dose will cost significantly more and has the potential to increase adverse events?”

To compare the cost of a weight-based vs. fixed-dose strategy, Dr. Bryant and colleagues conducted a simulation analysis under four stewardship scenarios, using data from the VHA and Medicare drug prices. Strategy one looked at weight-based dosing; strategy two combined weight-based dosing and dose rounding but not single-use vial sharing; strategy three used weight-based dosing and single-use vial sharing but not dose rounding; and strategy four, the most aggressive, combined all three.

ICIs in the VHA national formulary included pembrolizumab, nivolumab, atezolizumab, durvalumab, and cemiplimab-rwlc.

Using an algorithm to extract data, the team identified 49,851 administration events in 8,276 unique patients in 2021 – just over half were pembrolizumab, nearly 23% were nivolumab, and the remaining 26% largely included atezolizumab (12.1%) and durvalumab (11.9%).

The team found that the VHA spends roughly $537 million annually on ICIs. But implementing the stewardship measures that combined weight-based dosing, dose rounding, and vial sharing could save the VHA $74 million, or about 14%, annually on ICIs.

Most of the savings came from dosing changes to pembrolizumab and nivolumab, with greater savings achieved by combining more stewardship strategies. For instance, using strategy one (weight-based dosing alone) could lead to annual pembrolizumab savings of $14 million. Adding dose rounding (strategy two) could reduce pembrolizumab spending by $24 million. And using strategy four, with an unlimited window for vial sharing, could mean annual savings of nearly $60 million.

“Our results should prompt cost-conscious systems and payers to ask whether the amounts of drugs they’re providing to patients and how they go about making those doses are the most cost-effective approaches,” said corresponding author Garth W. Strohbehn, MD, of the University of Michigan and the VA Ann Arbor Healthcare System.

Dr. Strohbehn said the prospect of adopting these strategies hinges on several factors, with financial incentives at the prescriber and medical center level likely being the most influential.

“In fee-for-service systems, reimbursement scales with the amount of drug administered, so there can be a financial disincentive to decreasing overall drug usage,” Dr. Strohbehn explained.

“Conversely, integrated systems such as Kaiser Permanente or the VHA and large self-insured employers are incentivized to contain costs and take great care of patients, so they may be more inclined to promote these strategies,” he added.

However, Adam C. Powell, PhD, president, Payer+Provider Syndicate, who wasn’t involved in the analysis, cautioned that such a shift may come with unintended consequences.

The Infrastructure, Investment, and Jobs Act of 2021 let the Centers for Medicare and Medicaid Services seek reimbursement for discarded drugs – in effect, changing the reimbursement model for medications. That led pharmaceutical manufacturers to respond in kind by changing the dosing model, Dr. Powell said. 

“Drugs that previously had personalized weight-based dosing were moved to uniform flat dosing, eliminating the potential for the manufacturer to have to issue a reimbursement if the patient’s personalized dose fell short of the amount in the single-use vial,” Dr. Powell added.  

If there is a substantial migration to weight-based dosing, “it is possible that pharmaceutical manufacturers will rethink their dosing and pricing models, just as happened previously,” he cautioned.

However, these strategies could also provide relief for another escalating issue: drug shortages. Especially in the current moment, having a stewardship mindset, “might be helpful in navigating drug shortages,” Dr. Strohbehn said.

This research had no commercial funding. Dr. Bryant, Dr. Strohbehn, and Dr. Powell report no relevant financial relationships.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Adults with high-risk serrated polyps (with or without high-risk adenomas) have a high risk for metachronous colorectal cancer (CRC) within a median of 3 years after the baseline colonoscopy for a positive fecal immunochemical test (FIT) screen, a study suggests.

,

METHODOLOGY:

• Investigators conducted a retrospective analysis of 253,833 colonoscopies performed after FIT-positive screens in a Dutch CRC screening program.
• A Cox regression analysis assessed the association between the findings at baseline colonoscopy and metachronous CRC risk.
• Investigators categorized patients into subgroups based on removed polyp subtypes and used groups without polyps as a reference.
• High-risk subgroups included those with high-risk serrated polyps, which were defined as a serrated polyp of at least 10 mm, sessile serrated lesions with dysplasia, or traditional serrated adenomas, as well as high-risk adenomas, which were defined as an adenoma of at least 10 mm or containing high-grade dysplasia.

TAKEAWAY:

• Over a median follow-up of 36 months, 504 metachronous CRCs were identified.
• Individuals with high-risk serrated polyps without co-occurring high-risk adenomas had an increased risk for metachronous CRC (hazard ratio, 1.70).
• The highest risk was seen in individuals with both high-risk serrated polyps and high-risk adenomas (HR, 2.0), as well as those with villous adenomas (HR, 2.07).
• Individuals with only high-risk adenomas did not show a significantly increased risk for metachronous CRC (HR, 1.22).

IN PRACTICE:

“Our results suggest that individuals with high-risk serrated polyps might comprise the higher CRC risk in the first years after colonoscopy. Results of this study could contribute to establish more restrictive polyp surveillance guidelines in a quality-assured setting,” the authors wrote.

SOURCE:

The study was led by David E. F. W. M. van Toledo, MD, department of gastroenterology and hepatology, Amsterdam University Medical Centers. It was published online July 5, 2023, in eClinicalMedicine. The study received no funding.

LIMITATIONS:

The relatively short median follow-up time of 3 years may limit the assessment of long-term metachronous CRC risk. The study population consisted of FIT-positive individuals, which may introduce selection bias. The incidence of metachronous CRC in the study was lower compared with other studies, potentially affecting the risk estimates. The limited number of cases in some subgroups may result in unreliable risk estimations.

DISCLOSURES:

Dr. van Toledo declared no relevant financial relationships.

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

TOPLINE:

Adults with high-risk serrated polyps (with or without high-risk adenomas) have a high risk for metachronous colorectal cancer (CRC) within a median of 3 years after the baseline colonoscopy for a positive fecal immunochemical test (FIT) screen, a study suggests.

,

METHODOLOGY:

• Investigators conducted a retrospective analysis of 253,833 colonoscopies performed after FIT-positive screens in a Dutch CRC screening program.
• A Cox regression analysis assessed the association between the findings at baseline colonoscopy and metachronous CRC risk.
• Investigators categorized patients into subgroups based on removed polyp subtypes and used groups without polyps as a reference.
• High-risk subgroups included those with high-risk serrated polyps, which were defined as a serrated polyp of at least 10 mm, sessile serrated lesions with dysplasia, or traditional serrated adenomas, as well as high-risk adenomas, which were defined as an adenoma of at least 10 mm or containing high-grade dysplasia.

TAKEAWAY:

• Over a median follow-up of 36 months, 504 metachronous CRCs were identified.
• Individuals with high-risk serrated polyps without co-occurring high-risk adenomas had an increased risk for metachronous CRC (hazard ratio, 1.70).
• The highest risk was seen in individuals with both high-risk serrated polyps and high-risk adenomas (HR, 2.0), as well as those with villous adenomas (HR, 2.07).
• Individuals with only high-risk adenomas did not show a significantly increased risk for metachronous CRC (HR, 1.22).

IN PRACTICE:

“Our results suggest that individuals with high-risk serrated polyps might comprise the higher CRC risk in the first years after colonoscopy. Results of this study could contribute to establish more restrictive polyp surveillance guidelines in a quality-assured setting,” the authors wrote.

SOURCE:

The study was led by David E. F. W. M. van Toledo, MD, department of gastroenterology and hepatology, Amsterdam University Medical Centers. It was published online July 5, 2023, in eClinicalMedicine. The study received no funding.

LIMITATIONS:

The relatively short median follow-up time of 3 years may limit the assessment of long-term metachronous CRC risk. The study population consisted of FIT-positive individuals, which may introduce selection bias. The incidence of metachronous CRC in the study was lower compared with other studies, potentially affecting the risk estimates. The limited number of cases in some subgroups may result in unreliable risk estimations.

DISCLOSURES:

Dr. van Toledo declared no relevant financial relationships.

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

TOPLINE:

Adults with high-risk serrated polyps (with or without high-risk adenomas) have a high risk for metachronous colorectal cancer (CRC) within a median of 3 years after the baseline colonoscopy for a positive fecal immunochemical test (FIT) screen, a study suggests.

,

METHODOLOGY:

• Investigators conducted a retrospective analysis of 253,833 colonoscopies performed after FIT-positive screens in a Dutch CRC screening program.
• A Cox regression analysis assessed the association between the findings at baseline colonoscopy and metachronous CRC risk.
• Investigators categorized patients into subgroups based on removed polyp subtypes and used groups without polyps as a reference.
• High-risk subgroups included those with high-risk serrated polyps, which were defined as a serrated polyp of at least 10 mm, sessile serrated lesions with dysplasia, or traditional serrated adenomas, as well as high-risk adenomas, which were defined as an adenoma of at least 10 mm or containing high-grade dysplasia.

TAKEAWAY:

• Over a median follow-up of 36 months, 504 metachronous CRCs were identified.
• Individuals with high-risk serrated polyps without co-occurring high-risk adenomas had an increased risk for metachronous CRC (hazard ratio, 1.70).
• The highest risk was seen in individuals with both high-risk serrated polyps and high-risk adenomas (HR, 2.0), as well as those with villous adenomas (HR, 2.07).
• Individuals with only high-risk adenomas did not show a significantly increased risk for metachronous CRC (HR, 1.22).

IN PRACTICE:

“Our results suggest that individuals with high-risk serrated polyps might comprise the higher CRC risk in the first years after colonoscopy. Results of this study could contribute to establish more restrictive polyp surveillance guidelines in a quality-assured setting,” the authors wrote.

SOURCE:

The study was led by David E. F. W. M. van Toledo, MD, department of gastroenterology and hepatology, Amsterdam University Medical Centers. It was published online July 5, 2023, in eClinicalMedicine. The study received no funding.

LIMITATIONS:

The relatively short median follow-up time of 3 years may limit the assessment of long-term metachronous CRC risk. The study population consisted of FIT-positive individuals, which may introduce selection bias. The incidence of metachronous CRC in the study was lower compared with other studies, potentially affecting the risk estimates. The limited number of cases in some subgroups may result in unreliable risk estimations.

DISCLOSURES:

Dr. van Toledo declared no relevant financial relationships.

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

New research supports the use of liquid biopsy as an adjunct biomarker for the diagnosis and surveillance of human papillomavirus (HPV)–associated oropharyngeal cancer.

In a retrospective observational cohort study, a commercially available blood test used to evaluate tumor tissue–modified viral-HPV DNA demonstrated 100% specificity for both diagnosis of oropharyngeal cancer and surveillance for recurrence. Sensitivity was 91.5% for correctly identifying patients who have the disease and 88.4% for surveillance.

“A positive result appeared to confirm the presence of disease, [but] approximately 1 in 10 negative results in patients with pathologically confirmed HPV-associated oropharyngeal squamous cell carcinoma were falsely negative,” lead investigator Rocco Ferrandino, MD, with Mount Sinai, New York, said in an interview.

“Therefore, further workup should still be pursued when clinical suspicion for HPV-associated oropharynx cancer is high,” Dr. Ferrandino said.

The study was published online, in JAMA Otolaryngology–Head and Neck Surgery, to coincide with presentation at the annual meeting of the American Head and Neck Society in Montreal.
 

‘Remarkable promise’

The diagnosis of HPV-associated oropharyngeal cancer currently relies on a tissue-based biopsy of the primary site or a regional lymph node; however, there has been growing interest in the potential of liquid biopsy for diagnosis and surveillance.

The commercially available assay that was evaluated in the study uses a distinct method to identify and quantify a tumor-associated or tumor-modified pattern of DNA fragments that significantly increases the specificity for identifying an HPV-associated malignant tumor. However, evaluation of the assay has been limited to small cohort studies and clinical trials.

In the current study, Dr. Ferrandino and colleagues evaluated the performance of the assay used during routine clinical practice at their high-volume institution over a period of nearly 3 years.

The study included 163 patients in the diagnostic cohort and 290 in the surveillance cohort. In the diagnostic cohort, 152 had HPV-associated oropharyngeal cancer, and 11 had HPV-negative oropharyngeal cancer. The sensitivity of the assay in pretreatment diagnosis was 91.5% (139 of 152 tests), and the specificity was 100% (11 of 11 tests).

In the surveillance cohort of 290 patients, 591 tests were evaluated. A total of 23 patients developed pathologically confirmed recurrences over a median follow-up of 40.5 months. The assay demonstrated sensitivity of 88.4% (38 of 43 tests) and specificity of 100% (548 of 548 tests) in detecting recurrences.

The median lead time from positive test to pathologic confirmation was 47 days.

“The lead time provided by positive assay results may allow a window of opportunity for salvage treatment or for the application of adjuvant systemic therapy,” Dr. Ferrandino and colleagues explain.

“While these results are exciting and may support adjunctive use of circulating tumor DNA testing for diagnosis and surveillance, we really need more prospective and multicenter studies to validate these findings,” Dr. Ferrandino said in an interview.

In an accompanying commentary, Miriam Lango, MD, department of head and neck surgery, the University of Texas MD Anderson Cancer Center, Houston, said she agrees that a prospective clinical validation study is needed.

“Nevertheless, the use of this technology shows remarkable promise to transform the ability to identify and follow patients with HPV-related disease. Testing is likely to be increasingly used in routine clinical care, as it is commercially available,” Dr. Lango writes.

Still, she noted, “It is incumbent on us to establish evidence for strong and detailed surveillance guidelines to share among the cancer community.”

The study had no specific funding. Dr. Ferrandino and Dr. Lango have disclosed no relevant financial relationships.

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

New research supports the use of liquid biopsy as an adjunct biomarker for the diagnosis and surveillance of human papillomavirus (HPV)–associated oropharyngeal cancer.

In a retrospective observational cohort study, a commercially available blood test used to evaluate tumor tissue–modified viral-HPV DNA demonstrated 100% specificity for both diagnosis of oropharyngeal cancer and surveillance for recurrence. Sensitivity was 91.5% for correctly identifying patients who have the disease and 88.4% for surveillance.

“A positive result appeared to confirm the presence of disease, [but] approximately 1 in 10 negative results in patients with pathologically confirmed HPV-associated oropharyngeal squamous cell carcinoma were falsely negative,” lead investigator Rocco Ferrandino, MD, with Mount Sinai, New York, said in an interview.

“Therefore, further workup should still be pursued when clinical suspicion for HPV-associated oropharynx cancer is high,” Dr. Ferrandino said.

The study was published online, in JAMA Otolaryngology–Head and Neck Surgery, to coincide with presentation at the annual meeting of the American Head and Neck Society in Montreal.
 

‘Remarkable promise’

The diagnosis of HPV-associated oropharyngeal cancer currently relies on a tissue-based biopsy of the primary site or a regional lymph node; however, there has been growing interest in the potential of liquid biopsy for diagnosis and surveillance.

The commercially available assay that was evaluated in the study uses a distinct method to identify and quantify a tumor-associated or tumor-modified pattern of DNA fragments that significantly increases the specificity for identifying an HPV-associated malignant tumor. However, evaluation of the assay has been limited to small cohort studies and clinical trials.

In the current study, Dr. Ferrandino and colleagues evaluated the performance of the assay used during routine clinical practice at their high-volume institution over a period of nearly 3 years.

The study included 163 patients in the diagnostic cohort and 290 in the surveillance cohort. In the diagnostic cohort, 152 had HPV-associated oropharyngeal cancer, and 11 had HPV-negative oropharyngeal cancer. The sensitivity of the assay in pretreatment diagnosis was 91.5% (139 of 152 tests), and the specificity was 100% (11 of 11 tests).

In the surveillance cohort of 290 patients, 591 tests were evaluated. A total of 23 patients developed pathologically confirmed recurrences over a median follow-up of 40.5 months. The assay demonstrated sensitivity of 88.4% (38 of 43 tests) and specificity of 100% (548 of 548 tests) in detecting recurrences.

The median lead time from positive test to pathologic confirmation was 47 days.

“The lead time provided by positive assay results may allow a window of opportunity for salvage treatment or for the application of adjuvant systemic therapy,” Dr. Ferrandino and colleagues explain.

“While these results are exciting and may support adjunctive use of circulating tumor DNA testing for diagnosis and surveillance, we really need more prospective and multicenter studies to validate these findings,” Dr. Ferrandino said in an interview.

In an accompanying commentary, Miriam Lango, MD, department of head and neck surgery, the University of Texas MD Anderson Cancer Center, Houston, said she agrees that a prospective clinical validation study is needed.

“Nevertheless, the use of this technology shows remarkable promise to transform the ability to identify and follow patients with HPV-related disease. Testing is likely to be increasingly used in routine clinical care, as it is commercially available,” Dr. Lango writes.

Still, she noted, “It is incumbent on us to establish evidence for strong and detailed surveillance guidelines to share among the cancer community.”

The study had no specific funding. Dr. Ferrandino and Dr. Lango have disclosed no relevant financial relationships.

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

New research supports the use of liquid biopsy as an adjunct biomarker for the diagnosis and surveillance of human papillomavirus (HPV)–associated oropharyngeal cancer.

In a retrospective observational cohort study, a commercially available blood test used to evaluate tumor tissue–modified viral-HPV DNA demonstrated 100% specificity for both diagnosis of oropharyngeal cancer and surveillance for recurrence. Sensitivity was 91.5% for correctly identifying patients who have the disease and 88.4% for surveillance.

“A positive result appeared to confirm the presence of disease, [but] approximately 1 in 10 negative results in patients with pathologically confirmed HPV-associated oropharyngeal squamous cell carcinoma were falsely negative,” lead investigator Rocco Ferrandino, MD, with Mount Sinai, New York, said in an interview.

“Therefore, further workup should still be pursued when clinical suspicion for HPV-associated oropharynx cancer is high,” Dr. Ferrandino said.

The study was published online, in JAMA Otolaryngology–Head and Neck Surgery, to coincide with presentation at the annual meeting of the American Head and Neck Society in Montreal.
 

‘Remarkable promise’

The diagnosis of HPV-associated oropharyngeal cancer currently relies on a tissue-based biopsy of the primary site or a regional lymph node; however, there has been growing interest in the potential of liquid biopsy for diagnosis and surveillance.

The commercially available assay that was evaluated in the study uses a distinct method to identify and quantify a tumor-associated or tumor-modified pattern of DNA fragments that significantly increases the specificity for identifying an HPV-associated malignant tumor. However, evaluation of the assay has been limited to small cohort studies and clinical trials.

In the current study, Dr. Ferrandino and colleagues evaluated the performance of the assay used during routine clinical practice at their high-volume institution over a period of nearly 3 years.

The study included 163 patients in the diagnostic cohort and 290 in the surveillance cohort. In the diagnostic cohort, 152 had HPV-associated oropharyngeal cancer, and 11 had HPV-negative oropharyngeal cancer. The sensitivity of the assay in pretreatment diagnosis was 91.5% (139 of 152 tests), and the specificity was 100% (11 of 11 tests).

In the surveillance cohort of 290 patients, 591 tests were evaluated. A total of 23 patients developed pathologically confirmed recurrences over a median follow-up of 40.5 months. The assay demonstrated sensitivity of 88.4% (38 of 43 tests) and specificity of 100% (548 of 548 tests) in detecting recurrences.

The median lead time from positive test to pathologic confirmation was 47 days.

“The lead time provided by positive assay results may allow a window of opportunity for salvage treatment or for the application of adjuvant systemic therapy,” Dr. Ferrandino and colleagues explain.

“While these results are exciting and may support adjunctive use of circulating tumor DNA testing for diagnosis and surveillance, we really need more prospective and multicenter studies to validate these findings,” Dr. Ferrandino said in an interview.

In an accompanying commentary, Miriam Lango, MD, department of head and neck surgery, the University of Texas MD Anderson Cancer Center, Houston, said she agrees that a prospective clinical validation study is needed.

“Nevertheless, the use of this technology shows remarkable promise to transform the ability to identify and follow patients with HPV-related disease. Testing is likely to be increasingly used in routine clinical care, as it is commercially available,” Dr. Lango writes.

Still, she noted, “It is incumbent on us to establish evidence for strong and detailed surveillance guidelines to share among the cancer community.”

The study had no specific funding. Dr. Ferrandino and Dr. Lango have disclosed no relevant financial relationships.

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

TOPLINE:

Delaying surgery after neoadjuvant therapy may improve tumor regression and decrease recurrence in advanced rectal cancer but does not translate to better overall survival.

METHODOLOGY:

• A total of 1,506 patients with locally advanced rectal cancer who underwent neoadjuvant therapy followed by total mesorectal excision were divided into three groups based on the time interval between therapy and surgery: short (8 weeks), intermediate (> 8 to 12 weeks), and long (> 12 weeks).
• The primary outcome was pathologic complete response, and secondary outcomes included other histopathologic results, perioperative events, and survival outcomes.
• Median follow-up was 33 months.

TAKEAWAY:

• Overall, a pathologic complete response was observed in 255 patients (17.2%).
• Compared with the intermediate interval (reference) group, investigators found no association between time interval and pathologic complete response in the short-interval (odds ratio, 0.74; 95% CI, 0.55-1.01) or long-interval groups (OR, 1.07; P = .70).
• A long interval was significantly associated with a lower risk of a bad response as measured by tumor regression grade 2-3, compared with the reference category (OR, 0.47), but a higher risk of minor postoperative complications (OR, 1.43), conversion to open surgery (OR, 3.14), and longer operative time.
• The long-interval group was associated with a significantly reduced risk of systemic recurrence, compared with the reference group (hazard ratio, 0.59; P = .04), but not improved overall survival (HR, 1.38; P = .11) or locoregional recurrence (HR, 0.53; P = .18); no significant findings occurred for the short versus intermediate group.

IN PRACTICE:

“Findings suggest that delaying surgery may improve tumor regression and decrease risk of distant metastasis but increase surgical complexity,” the authors conclude. “Nonetheless, the reported improvements in tumor regression and systemic recurrence in the long-interval group were unexpectedly not followed by improved [overall survival].”

SOURCE:

F. Borja de Lacy, MD, PhD, Hospital Clinic of Barcelona, University of Barcelona, led the study, published online in JAMA Surgery, with an accompanying editorial.

LIMITATIONS:

• The study’s main limitation was its retrospective design, which could have resulted in missing or inconsistent data, as well as the short follow-up time.
• Decisions about time interval were based more on professional preference rather than specific tumor characteristics.

DISCLOSURES:

Dr. de Lacy has reported no relevant financial relationships. No outside funding source was disclosed.

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

TOPLINE:

Delaying surgery after neoadjuvant therapy may improve tumor regression and decrease recurrence in advanced rectal cancer but does not translate to better overall survival.

METHODOLOGY:

• A total of 1,506 patients with locally advanced rectal cancer who underwent neoadjuvant therapy followed by total mesorectal excision were divided into three groups based on the time interval between therapy and surgery: short (8 weeks), intermediate (> 8 to 12 weeks), and long (> 12 weeks).
• The primary outcome was pathologic complete response, and secondary outcomes included other histopathologic results, perioperative events, and survival outcomes.
• Median follow-up was 33 months.

TAKEAWAY:

• Overall, a pathologic complete response was observed in 255 patients (17.2%).
• Compared with the intermediate interval (reference) group, investigators found no association between time interval and pathologic complete response in the short-interval (odds ratio, 0.74; 95% CI, 0.55-1.01) or long-interval groups (OR, 1.07; P = .70).
• A long interval was significantly associated with a lower risk of a bad response as measured by tumor regression grade 2-3, compared with the reference category (OR, 0.47), but a higher risk of minor postoperative complications (OR, 1.43), conversion to open surgery (OR, 3.14), and longer operative time.
• The long-interval group was associated with a significantly reduced risk of systemic recurrence, compared with the reference group (hazard ratio, 0.59; P = .04), but not improved overall survival (HR, 1.38; P = .11) or locoregional recurrence (HR, 0.53; P = .18); no significant findings occurred for the short versus intermediate group.

IN PRACTICE:

“Findings suggest that delaying surgery may improve tumor regression and decrease risk of distant metastasis but increase surgical complexity,” the authors conclude. “Nonetheless, the reported improvements in tumor regression and systemic recurrence in the long-interval group were unexpectedly not followed by improved [overall survival].”

SOURCE:

F. Borja de Lacy, MD, PhD, Hospital Clinic of Barcelona, University of Barcelona, led the study, published online in JAMA Surgery, with an accompanying editorial.

LIMITATIONS:

• The study’s main limitation was its retrospective design, which could have resulted in missing or inconsistent data, as well as the short follow-up time.
• Decisions about time interval were based more on professional preference rather than specific tumor characteristics.

DISCLOSURES:

Dr. de Lacy has reported no relevant financial relationships. No outside funding source was disclosed.

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

TOPLINE:

Delaying surgery after neoadjuvant therapy may improve tumor regression and decrease recurrence in advanced rectal cancer but does not translate to better overall survival.

METHODOLOGY:

• A total of 1,506 patients with locally advanced rectal cancer who underwent neoadjuvant therapy followed by total mesorectal excision were divided into three groups based on the time interval between therapy and surgery: short (8 weeks), intermediate (> 8 to 12 weeks), and long (> 12 weeks).
• The primary outcome was pathologic complete response, and secondary outcomes included other histopathologic results, perioperative events, and survival outcomes.
• Median follow-up was 33 months.

TAKEAWAY:

• Overall, a pathologic complete response was observed in 255 patients (17.2%).
• Compared with the intermediate interval (reference) group, investigators found no association between time interval and pathologic complete response in the short-interval (odds ratio, 0.74; 95% CI, 0.55-1.01) or long-interval groups (OR, 1.07; P = .70).
• A long interval was significantly associated with a lower risk of a bad response as measured by tumor regression grade 2-3, compared with the reference category (OR, 0.47), but a higher risk of minor postoperative complications (OR, 1.43), conversion to open surgery (OR, 3.14), and longer operative time.
• The long-interval group was associated with a significantly reduced risk of systemic recurrence, compared with the reference group (hazard ratio, 0.59; P = .04), but not improved overall survival (HR, 1.38; P = .11) or locoregional recurrence (HR, 0.53; P = .18); no significant findings occurred for the short versus intermediate group.

IN PRACTICE:

“Findings suggest that delaying surgery may improve tumor regression and decrease risk of distant metastasis but increase surgical complexity,” the authors conclude. “Nonetheless, the reported improvements in tumor regression and systemic recurrence in the long-interval group were unexpectedly not followed by improved [overall survival].”

SOURCE:

F. Borja de Lacy, MD, PhD, Hospital Clinic of Barcelona, University of Barcelona, led the study, published online in JAMA Surgery, with an accompanying editorial.

LIMITATIONS:

• The study’s main limitation was its retrospective design, which could have resulted in missing or inconsistent data, as well as the short follow-up time.
• Decisions about time interval were based more on professional preference rather than specific tumor characteristics.

DISCLOSURES:

Dr. de Lacy has reported no relevant financial relationships. No outside funding source was disclosed.

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

This transcript has been edited for clarity.

I’m Maurie Markman, MD, from Cancer Treatment Centers of America in Philadelphia. I wanted to discuss a highly provocative paper that I think deserves attention. It was published in the Journal of Clinical Oncology, titled Physical Activity in Stage III Colon Cancer: CALGB/SWOG 80702 (Alliance).

This is an incredibly important paper that highlights something that has not been emphasized enough in oncology practice. What are the things that we can recommend to our patients that are not expensive, but which they can do for themselves to impact a potential for adding to a positive outcome? In this case, we’re talking about physical activity.

This was an extremely well-conducted study. It was a prospective cohort study that was built into an ongoing phase 3 randomized, multicenter study looking at adjuvant therapy of stage III colon cancer. The median follow-up in this population was almost 6 years. We’re talking about 1,696 patients.

The investigators did a survey, asking patients when they started treatment and then a short time after that, and measured the level of recreational physical activity. They didn’t do a design. They asked the individuals how much activity they had.

There were a number of analyses done in terms of looking at this that were reported in the paper. I want to highlight one because it’s so simple. The investigators looked at brisk walking. For brisk walking, the 3-year disease-free survival was 81.7% for individuals who had less than 1 hour per week of brisk walking versus 88.4% for individuals who walked briskly more than 3 hours per week.

Walking an additional 2 hours or more per week, prospectively viewed in individuals who had the same baseline characteristics otherwise, impacted disease-free survival in stage III colon cancer. There is no additional expense. It’s walking. There were other activities that were looked at here, including aerobic activities.

The bottom line is that physical activity is positive, is not expensive, and focuses on what the individual patient can do for themselves. It’s something I believe that, in the oncology community, we need to emphasize more.

I encourage you to review this paper and use your own opinion as to what you want to do with this information, but I strongly urge you to look at this – and other types of activities – that we can recommend that individuals do themselves to impact their outcomes related to cancer.

Dr. Markman is a clinical professor of medicine at Drexel University, Philadelphia. He reported conflicts of interest with Genentech, AstraZeneca, Celgene, Clovis, and Amgen.

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

This transcript has been edited for clarity.

I’m Maurie Markman, MD, from Cancer Treatment Centers of America in Philadelphia. I wanted to discuss a highly provocative paper that I think deserves attention. It was published in the Journal of Clinical Oncology, titled Physical Activity in Stage III Colon Cancer: CALGB/SWOG 80702 (Alliance).

This is an incredibly important paper that highlights something that has not been emphasized enough in oncology practice. What are the things that we can recommend to our patients that are not expensive, but which they can do for themselves to impact a potential for adding to a positive outcome? In this case, we’re talking about physical activity.

This was an extremely well-conducted study. It was a prospective cohort study that was built into an ongoing phase 3 randomized, multicenter study looking at adjuvant therapy of stage III colon cancer. The median follow-up in this population was almost 6 years. We’re talking about 1,696 patients.

The investigators did a survey, asking patients when they started treatment and then a short time after that, and measured the level of recreational physical activity. They didn’t do a design. They asked the individuals how much activity they had.

There were a number of analyses done in terms of looking at this that were reported in the paper. I want to highlight one because it’s so simple. The investigators looked at brisk walking. For brisk walking, the 3-year disease-free survival was 81.7% for individuals who had less than 1 hour per week of brisk walking versus 88.4% for individuals who walked briskly more than 3 hours per week.

Walking an additional 2 hours or more per week, prospectively viewed in individuals who had the same baseline characteristics otherwise, impacted disease-free survival in stage III colon cancer. There is no additional expense. It’s walking. There were other activities that were looked at here, including aerobic activities.

The bottom line is that physical activity is positive, is not expensive, and focuses on what the individual patient can do for themselves. It’s something I believe that, in the oncology community, we need to emphasize more.

I encourage you to review this paper and use your own opinion as to what you want to do with this information, but I strongly urge you to look at this – and other types of activities – that we can recommend that individuals do themselves to impact their outcomes related to cancer.

Dr. Markman is a clinical professor of medicine at Drexel University, Philadelphia. He reported conflicts of interest with Genentech, AstraZeneca, Celgene, Clovis, and Amgen.

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

This transcript has been edited for clarity.

I’m Maurie Markman, MD, from Cancer Treatment Centers of America in Philadelphia. I wanted to discuss a highly provocative paper that I think deserves attention. It was published in the Journal of Clinical Oncology, titled Physical Activity in Stage III Colon Cancer: CALGB/SWOG 80702 (Alliance).

This is an incredibly important paper that highlights something that has not been emphasized enough in oncology practice. What are the things that we can recommend to our patients that are not expensive, but which they can do for themselves to impact a potential for adding to a positive outcome? In this case, we’re talking about physical activity.

This was an extremely well-conducted study. It was a prospective cohort study that was built into an ongoing phase 3 randomized, multicenter study looking at adjuvant therapy of stage III colon cancer. The median follow-up in this population was almost 6 years. We’re talking about 1,696 patients.

The investigators did a survey, asking patients when they started treatment and then a short time after that, and measured the level of recreational physical activity. They didn’t do a design. They asked the individuals how much activity they had.

There were a number of analyses done in terms of looking at this that were reported in the paper. I want to highlight one because it’s so simple. The investigators looked at brisk walking. For brisk walking, the 3-year disease-free survival was 81.7% for individuals who had less than 1 hour per week of brisk walking versus 88.4% for individuals who walked briskly more than 3 hours per week.

Walking an additional 2 hours or more per week, prospectively viewed in individuals who had the same baseline characteristics otherwise, impacted disease-free survival in stage III colon cancer. There is no additional expense. It’s walking. There were other activities that were looked at here, including aerobic activities.

The bottom line is that physical activity is positive, is not expensive, and focuses on what the individual patient can do for themselves. It’s something I believe that, in the oncology community, we need to emphasize more.

I encourage you to review this paper and use your own opinion as to what you want to do with this information, but I strongly urge you to look at this – and other types of activities – that we can recommend that individuals do themselves to impact their outcomes related to cancer.

Dr. Markman is a clinical professor of medicine at Drexel University, Philadelphia. He reported conflicts of interest with Genentech, AstraZeneca, Celgene, Clovis, and Amgen.

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

TOPLINE:

Although practice patterns vary widely, modified, reduced-dose FOLFIRINOX is as effective as standard, full-dose regimens for patients with metastatic pancreatic cancer in the first-line setting, and it is less likely to cause febrile neutropenia.

METHODOLOGY:

• No randomized controlled trials have directly compared modified FOLFIRINOX to standard FOLFIRINOX; this meta-analysis aims to fill the evidence gap.
• The investigators winnowed hundreds of first-line FOLFIRINOX studies down to 37 – 11 prospective and 26 retrospective analyses – to assess practice patterns and clinical outcomes.
• Dose information was grouped into four categories: planned dose in the standard FOLFIRINOX group; actual administered dose in the standard group; planned dose in the modified group; actual administered dose in the modified group.

TAKEAWAY:

• There were 12 types of “planned” dose reductions in FOLFIRINOX: 75%-100% oxaliplatin, 75%-100% irinotecan, 0%-100% 5-fluorouracil (5-FU) bolus, and 75%-133% 5-FU continuous injection.
• Doses actually delivered fell further to 54%-96% for oxaliplatin, 61%-88% for irinotecan, 0%-92% for 5-FU bolus, and 63%-98% 5-FU continuous injection.
• Despite the variations in dosing, reduced doses of FOLFIRINOX were associated with a slightly but not significantly higher objective response rate: 33.8% versus 28.2% for standard dosing (P = .1).
• The incidence of febrile neutropenia was significantly lower in the reduced-dose groups: 5.5% with modified FOLFIRINOX versus 11.6% with standard (P = .03).

IN PRACTICE:

Although the study supports reduced-dose regimens, it also shows that there is “still no consensus” on appropriate dose modification, the authors said. “The best dose modification protocol” remains to be determined and standardized for metastatic pancreatic cancer.

SOURCE:

The study was led by Kwangrok Jung at Seoul (South Korea) National University, and was published June 29 in Therapeutic Advances in Medical Oncology.

LIMITATIONS:

• Only 11 of the 37 studies were prospective.
• The studies often lacked key information, including the reason for dose reductions or detailed dose reduction protocols.
• Studies were also inconsistent in how they reported FOLFIRINOX dose modifications.

DISCLOSURES:

There was no funding for the study, and the investigators had no disclosures.

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

TOPLINE:

Although practice patterns vary widely, modified, reduced-dose FOLFIRINOX is as effective as standard, full-dose regimens for patients with metastatic pancreatic cancer in the first-line setting, and it is less likely to cause febrile neutropenia.

METHODOLOGY:

• No randomized controlled trials have directly compared modified FOLFIRINOX to standard FOLFIRINOX; this meta-analysis aims to fill the evidence gap.
• The investigators winnowed hundreds of first-line FOLFIRINOX studies down to 37 – 11 prospective and 26 retrospective analyses – to assess practice patterns and clinical outcomes.
• Dose information was grouped into four categories: planned dose in the standard FOLFIRINOX group; actual administered dose in the standard group; planned dose in the modified group; actual administered dose in the modified group.

TAKEAWAY:

• There were 12 types of “planned” dose reductions in FOLFIRINOX: 75%-100% oxaliplatin, 75%-100% irinotecan, 0%-100% 5-fluorouracil (5-FU) bolus, and 75%-133% 5-FU continuous injection.
• Doses actually delivered fell further to 54%-96% for oxaliplatin, 61%-88% for irinotecan, 0%-92% for 5-FU bolus, and 63%-98% 5-FU continuous injection.
• Despite the variations in dosing, reduced doses of FOLFIRINOX were associated with a slightly but not significantly higher objective response rate: 33.8% versus 28.2% for standard dosing (P = .1).
• The incidence of febrile neutropenia was significantly lower in the reduced-dose groups: 5.5% with modified FOLFIRINOX versus 11.6% with standard (P = .03).

IN PRACTICE:

Although the study supports reduced-dose regimens, it also shows that there is “still no consensus” on appropriate dose modification, the authors said. “The best dose modification protocol” remains to be determined and standardized for metastatic pancreatic cancer.

SOURCE:

The study was led by Kwangrok Jung at Seoul (South Korea) National University, and was published June 29 in Therapeutic Advances in Medical Oncology.

LIMITATIONS:

• Only 11 of the 37 studies were prospective.
• The studies often lacked key information, including the reason for dose reductions or detailed dose reduction protocols.
• Studies were also inconsistent in how they reported FOLFIRINOX dose modifications.

DISCLOSURES:

There was no funding for the study, and the investigators had no disclosures.

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

TOPLINE:

Although practice patterns vary widely, modified, reduced-dose FOLFIRINOX is as effective as standard, full-dose regimens for patients with metastatic pancreatic cancer in the first-line setting, and it is less likely to cause febrile neutropenia.

METHODOLOGY:

• No randomized controlled trials have directly compared modified FOLFIRINOX to standard FOLFIRINOX; this meta-analysis aims to fill the evidence gap.
• The investigators winnowed hundreds of first-line FOLFIRINOX studies down to 37 – 11 prospective and 26 retrospective analyses – to assess practice patterns and clinical outcomes.
• Dose information was grouped into four categories: planned dose in the standard FOLFIRINOX group; actual administered dose in the standard group; planned dose in the modified group; actual administered dose in the modified group.

TAKEAWAY:

• There were 12 types of “planned” dose reductions in FOLFIRINOX: 75%-100% oxaliplatin, 75%-100% irinotecan, 0%-100% 5-fluorouracil (5-FU) bolus, and 75%-133% 5-FU continuous injection.
• Doses actually delivered fell further to 54%-96% for oxaliplatin, 61%-88% for irinotecan, 0%-92% for 5-FU bolus, and 63%-98% 5-FU continuous injection.
• Despite the variations in dosing, reduced doses of FOLFIRINOX were associated with a slightly but not significantly higher objective response rate: 33.8% versus 28.2% for standard dosing (P = .1).
• The incidence of febrile neutropenia was significantly lower in the reduced-dose groups: 5.5% with modified FOLFIRINOX versus 11.6% with standard (P = .03).

IN PRACTICE:

Although the study supports reduced-dose regimens, it also shows that there is “still no consensus” on appropriate dose modification, the authors said. “The best dose modification protocol” remains to be determined and standardized for metastatic pancreatic cancer.

SOURCE:

The study was led by Kwangrok Jung at Seoul (South Korea) National University, and was published June 29 in Therapeutic Advances in Medical Oncology.

LIMITATIONS:

• Only 11 of the 37 studies were prospective.
• The studies often lacked key information, including the reason for dose reductions or detailed dose reduction protocols.
• Studies were also inconsistent in how they reported FOLFIRINOX dose modifications.

DISCLOSURES:

There was no funding for the study, and the investigators had no disclosures.

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

TOPLINE:

Irradiating a small number of metastatic lesions does not appear to improve progression-free or overall survival in patients receiving immune checkpoint inhibitor monotherapy for advanced cancer.

METHODOLOGY:

• In the phase 2 CHEERS trial, 52 patients with advanced solid tumors were randomized to anti-PD-1/PD-L1 monotherapy and 47 patients to the same treatment plus stereotactic body radiotherapy (3 x 8 Gy) to a maximum of three lesions before the second or third cycle of an immune checkpoint inhibitor.
• Patients had locally advanced or metastatic melanoma, renal cell carcinoma, urothelial carcinoma, non-small cell lung carcinoma, or head and neck squamous cell carcinoma and were treated at five Belgian hospitals.
• Most patients had more than three lesions.
• Seven patients in the experimental group did not complete radiotherapy because of early progression or intercurrent illness.

TAKEAWAY:

• Over a median follow-up of 12.5 months, median progression-free survival was 4.4 months in the radiotherapy group versus 2.8 months in the control group (hazard ratio, 0.95; P = .82).
• Median overall survival was not significantly better with radiotherapy, compared with the control group (14.3 vs. 11 months; HR, 0.82; P = .47), nor was the objective response rate (27% vs. 22%; P = .56).
• However, a post hoc analysis demonstrated a significant association between the number of irradiated lesions and overall survival among patients receiving radiotherapy (HR, 0.31; P = .002).
• The incidence of grade 3 or worse treatment-related adverse events was 18% in both groups.

IN PRACTICE:

Although the study was negative overall, the post hoc analysis coupled with “recent evidence suggests that treating all active disease sites with higher radiation doses ... may be a more promising strategy to optimize systemic disease control,” the authors concluded.
 

SOURCE:

The study was led by Mathieu Spaas, MD, department of radiation oncology, Ghent (Bellgium) University, and published online in JAMA Oncology.

LIMITATIONS:

• There was insufficient power to detect if certain cancers benefited more from add-on radiation because of the small sample size.
• More than half of patients in the control group had already received some form of radiotherapy before study inclusion, which may mean the study underestimated the benefit of radiotherapy.

DISCLOSURES:

The work was funded by Kom Op Tegen Kanker and Varian Medical Systems.

Investigators disclosed numerous industry ties, including Merck, Novartis, and Bristol Myers Squibb.

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

TOPLINE:

Irradiating a small number of metastatic lesions does not appear to improve progression-free or overall survival in patients receiving immune checkpoint inhibitor monotherapy for advanced cancer.

METHODOLOGY:

• In the phase 2 CHEERS trial, 52 patients with advanced solid tumors were randomized to anti-PD-1/PD-L1 monotherapy and 47 patients to the same treatment plus stereotactic body radiotherapy (3 x 8 Gy) to a maximum of three lesions before the second or third cycle of an immune checkpoint inhibitor.
• Patients had locally advanced or metastatic melanoma, renal cell carcinoma, urothelial carcinoma, non-small cell lung carcinoma, or head and neck squamous cell carcinoma and were treated at five Belgian hospitals.
• Most patients had more than three lesions.
• Seven patients in the experimental group did not complete radiotherapy because of early progression or intercurrent illness.

TAKEAWAY:

• Over a median follow-up of 12.5 months, median progression-free survival was 4.4 months in the radiotherapy group versus 2.8 months in the control group (hazard ratio, 0.95; P = .82).
• Median overall survival was not significantly better with radiotherapy, compared with the control group (14.3 vs. 11 months; HR, 0.82; P = .47), nor was the objective response rate (27% vs. 22%; P = .56).
• However, a post hoc analysis demonstrated a significant association between the number of irradiated lesions and overall survival among patients receiving radiotherapy (HR, 0.31; P = .002).
• The incidence of grade 3 or worse treatment-related adverse events was 18% in both groups.

IN PRACTICE:

Although the study was negative overall, the post hoc analysis coupled with “recent evidence suggests that treating all active disease sites with higher radiation doses ... may be a more promising strategy to optimize systemic disease control,” the authors concluded.
 

SOURCE:

The study was led by Mathieu Spaas, MD, department of radiation oncology, Ghent (Bellgium) University, and published online in JAMA Oncology.

LIMITATIONS:

• There was insufficient power to detect if certain cancers benefited more from add-on radiation because of the small sample size.
• More than half of patients in the control group had already received some form of radiotherapy before study inclusion, which may mean the study underestimated the benefit of radiotherapy.

DISCLOSURES:

The work was funded by Kom Op Tegen Kanker and Varian Medical Systems.

Investigators disclosed numerous industry ties, including Merck, Novartis, and Bristol Myers Squibb.

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

TOPLINE:

Irradiating a small number of metastatic lesions does not appear to improve progression-free or overall survival in patients receiving immune checkpoint inhibitor monotherapy for advanced cancer.

METHODOLOGY:

• In the phase 2 CHEERS trial, 52 patients with advanced solid tumors were randomized to anti-PD-1/PD-L1 monotherapy and 47 patients to the same treatment plus stereotactic body radiotherapy (3 x 8 Gy) to a maximum of three lesions before the second or third cycle of an immune checkpoint inhibitor.
• Patients had locally advanced or metastatic melanoma, renal cell carcinoma, urothelial carcinoma, non-small cell lung carcinoma, or head and neck squamous cell carcinoma and were treated at five Belgian hospitals.
• Most patients had more than three lesions.
• Seven patients in the experimental group did not complete radiotherapy because of early progression or intercurrent illness.

TAKEAWAY:

• Over a median follow-up of 12.5 months, median progression-free survival was 4.4 months in the radiotherapy group versus 2.8 months in the control group (hazard ratio, 0.95; P = .82).
• Median overall survival was not significantly better with radiotherapy, compared with the control group (14.3 vs. 11 months; HR, 0.82; P = .47), nor was the objective response rate (27% vs. 22%; P = .56).
• However, a post hoc analysis demonstrated a significant association between the number of irradiated lesions and overall survival among patients receiving radiotherapy (HR, 0.31; P = .002).
• The incidence of grade 3 or worse treatment-related adverse events was 18% in both groups.

IN PRACTICE:

Although the study was negative overall, the post hoc analysis coupled with “recent evidence suggests that treating all active disease sites with higher radiation doses ... may be a more promising strategy to optimize systemic disease control,” the authors concluded.
 

SOURCE:

The study was led by Mathieu Spaas, MD, department of radiation oncology, Ghent (Bellgium) University, and published online in JAMA Oncology.

LIMITATIONS:

• There was insufficient power to detect if certain cancers benefited more from add-on radiation because of the small sample size.
• More than half of patients in the control group had already received some form of radiotherapy before study inclusion, which may mean the study underestimated the benefit of radiotherapy.

DISCLOSURES:

The work was funded by Kom Op Tegen Kanker and Varian Medical Systems.

Investigators disclosed numerous industry ties, including Merck, Novartis, and Bristol Myers Squibb.

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

This transcript has been edited for clarity.

Hi. I’m Art Caplan, PhD. I’m director of the division of medical ethics at the New York University Grossman School of Medicine.

Every once in a while at my school, I get referrals about interesting or difficult clinical cases where doctors would like some input or advice that they can consider in managing a patient. Sometimes those requests come from other hospitals to me. I’ve been doing that kind of ethics consulting, both as a member of various ethics committees and sometimes individually, when, for various reasons, doctors don’t want to go to the Ethics Committee as a first stop.

There was a very interesting case recently involving a young woman I’m going to call Tinslee. She was 17 years old and she suffered, sadly, from recurrent metastatic osteogenic sarcoma. She had bone cancer. It had first been diagnosed at the age of 9. She had received chemotherapy and been under that treatment for a while.

If osteosarcoma is treated before it spreads outside the area where it began, the 5-year survival rate for people like her is about 75%. If the cancer spreads outside of the bones and gets into surrounding tissues, organs, or – worse – into the lymph nodes and starts traveling around, the 5-year survival rate drops to about 60%. The two approaches are chemotherapy and amputation. That’s what we have to offer patients like Tinslee.

Initially, her chemotherapy worked. She went to school and enjoyed sports. She was a real fan of softball and tried to manage the team and be involved. At the time I learned about her, she was planning to go to college. Her love of softball remained, but given the recurrence of the cancer, she had no chance to pursue her athletic interests, not only as a player, but also as a manager or even as a coach for younger players. That was all off the table.

She’d been very compliant up until this time with her chemotherapy. When the recommendation came in that she undergo nonstandard chemotherapy because of the reoccurrence, with experimental drugs using an experimental protocol, she said to her family and the doctors that she didn’t want to do it. She would rather die. She couldn’t take any more chemotherapy and she certainly didn’t want to do it if it was experimental, with the outcomes of this intervention being uncertain.

Her dad said, “She can’t decide. She’s a minor. She’s only 17. I want you to do it – administer this novel form of chemotherapy and try to save her.” Her mother said, “Her input matters. I want to listen to her.” Her mom wasn’t as adamant about doing it or not, but she really felt that Tinslee should be heard loudly because she felt she was mature enough or old enough, even though a minor, to really have a position about what it is to undergo chemotherapy.

Time matters in trying to control the spread, and the doctors were pushing for experimental intervention. I should add, by the way, that although it didn’t really drive the decision about whether to do it or not do it, experimental care like this is not covered by most insurance, and it wasn’t covered by their insurance, so they were facing a big bill if the experimental intervention was administered.

There was some money in a grant to cover some of it, but they were going to face some big financial costs. It never came up in my discussions with the doctors about what to do. I’m not sure whether it ever came up with the family’s discussion with the doctors about what to do, or even whether Tinslee was worrying and didn’t want her family to face a financial burden.

I suggested that we bring the family in. We did some counseling. We had a social worker and we brought in a pastor because these people were fairly religious. We talked about all scenarios, including accepting death, knowing that this disease was not likely to go into remission with the experimental effort; maybe it would, but the doctors were not optimistic.

We tried to talk about how much we should listen to what this young woman wanted. We knew there was the possibility of going to court and having a judge decide this, but in my experience, I do not like going to judges and courts because I know what they’re going to say. They almost always say “administer the intervention.” They don’t want to be in a position of saying don’t do something. They’re a little less willing to do that if something is experimental, but generally speaking, if you’re headed to court, it’s because you’ve decided that you want this to happen.

I felt, in all honesty, that this young woman should have some real respect of her position because the treatment was experimental. She is approaching the age of competency and consent, and she’s been through many interventions. She knows what’s involved. I think you really have to listen hard to what she’s saying.

By the way, after this case, I looked and there have been some surveys of residents in pediatrics. A large number of them said that they hadn’t received any training about what to do when mature minors refuse experimental treatments. The study I saw said that 30% had not undergone any training about this, so we certainly want to introduce that into the appropriate areas of medicine and talk about this with residents and fellows.

Long story short, we had the family meeting, we had another meeting with dad and mom and Tinslee, and the dad began to come around and he began to listen hard. Tinslee said what she wanted was to go to her prom. She wanted to get to her sister’s junior high school softball championship game. If you will, setting some smaller goals that seemed to make her very, very happy began to satisfy mom and dad and they could accept her refusal.

Ultimately, an agreement was reached that she would not undergo the experimental intervention. We agreed on a course of palliative care, recommended that as what the doctors follow, and they decided to do so. Sadly, Tinslee died. She died at home. She did make it to her prom.

I think the outcome, while difficult, sad, tragic, and a close call, was correct. Mature minors who have been through a rough life of interventions and know the price to pay – and for those who have recurrent disease and now face only experimental options – if they say no, that’s something we really have to listen to very hard.



Dr. Kaplan is director, division of medical ethics, New York University Langone Medical Center, New York. He reported a conflict of interest with Johnson & Johnson’s Panel for Compassionate Drug Use.
 

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

This transcript has been edited for clarity.

Hi. I’m Art Caplan, PhD. I’m director of the division of medical ethics at the New York University Grossman School of Medicine.

Every once in a while at my school, I get referrals about interesting or difficult clinical cases where doctors would like some input or advice that they can consider in managing a patient. Sometimes those requests come from other hospitals to me. I’ve been doing that kind of ethics consulting, both as a member of various ethics committees and sometimes individually, when, for various reasons, doctors don’t want to go to the Ethics Committee as a first stop.

There was a very interesting case recently involving a young woman I’m going to call Tinslee. She was 17 years old and she suffered, sadly, from recurrent metastatic osteogenic sarcoma. She had bone cancer. It had first been diagnosed at the age of 9. She had received chemotherapy and been under that treatment for a while.

If osteosarcoma is treated before it spreads outside the area where it began, the 5-year survival rate for people like her is about 75%. If the cancer spreads outside of the bones and gets into surrounding tissues, organs, or – worse – into the lymph nodes and starts traveling around, the 5-year survival rate drops to about 60%. The two approaches are chemotherapy and amputation. That’s what we have to offer patients like Tinslee.

Initially, her chemotherapy worked. She went to school and enjoyed sports. She was a real fan of softball and tried to manage the team and be involved. At the time I learned about her, she was planning to go to college. Her love of softball remained, but given the recurrence of the cancer, she had no chance to pursue her athletic interests, not only as a player, but also as a manager or even as a coach for younger players. That was all off the table.

She’d been very compliant up until this time with her chemotherapy. When the recommendation came in that she undergo nonstandard chemotherapy because of the reoccurrence, with experimental drugs using an experimental protocol, she said to her family and the doctors that she didn’t want to do it. She would rather die. She couldn’t take any more chemotherapy and she certainly didn’t want to do it if it was experimental, with the outcomes of this intervention being uncertain.

Her dad said, “She can’t decide. She’s a minor. She’s only 17. I want you to do it – administer this novel form of chemotherapy and try to save her.” Her mother said, “Her input matters. I want to listen to her.” Her mom wasn’t as adamant about doing it or not, but she really felt that Tinslee should be heard loudly because she felt she was mature enough or old enough, even though a minor, to really have a position about what it is to undergo chemotherapy.

Time matters in trying to control the spread, and the doctors were pushing for experimental intervention. I should add, by the way, that although it didn’t really drive the decision about whether to do it or not do it, experimental care like this is not covered by most insurance, and it wasn’t covered by their insurance, so they were facing a big bill if the experimental intervention was administered.

There was some money in a grant to cover some of it, but they were going to face some big financial costs. It never came up in my discussions with the doctors about what to do. I’m not sure whether it ever came up with the family’s discussion with the doctors about what to do, or even whether Tinslee was worrying and didn’t want her family to face a financial burden.

I suggested that we bring the family in. We did some counseling. We had a social worker and we brought in a pastor because these people were fairly religious. We talked about all scenarios, including accepting death, knowing that this disease was not likely to go into remission with the experimental effort; maybe it would, but the doctors were not optimistic.

We tried to talk about how much we should listen to what this young woman wanted. We knew there was the possibility of going to court and having a judge decide this, but in my experience, I do not like going to judges and courts because I know what they’re going to say. They almost always say “administer the intervention.” They don’t want to be in a position of saying don’t do something. They’re a little less willing to do that if something is experimental, but generally speaking, if you’re headed to court, it’s because you’ve decided that you want this to happen.

I felt, in all honesty, that this young woman should have some real respect of her position because the treatment was experimental. She is approaching the age of competency and consent, and she’s been through many interventions. She knows what’s involved. I think you really have to listen hard to what she’s saying.

By the way, after this case, I looked and there have been some surveys of residents in pediatrics. A large number of them said that they hadn’t received any training about what to do when mature minors refuse experimental treatments. The study I saw said that 30% had not undergone any training about this, so we certainly want to introduce that into the appropriate areas of medicine and talk about this with residents and fellows.

Long story short, we had the family meeting, we had another meeting with dad and mom and Tinslee, and the dad began to come around and he began to listen hard. Tinslee said what she wanted was to go to her prom. She wanted to get to her sister’s junior high school softball championship game. If you will, setting some smaller goals that seemed to make her very, very happy began to satisfy mom and dad and they could accept her refusal.

Ultimately, an agreement was reached that she would not undergo the experimental intervention. We agreed on a course of palliative care, recommended that as what the doctors follow, and they decided to do so. Sadly, Tinslee died. She died at home. She did make it to her prom.

I think the outcome, while difficult, sad, tragic, and a close call, was correct. Mature minors who have been through a rough life of interventions and know the price to pay – and for those who have recurrent disease and now face only experimental options – if they say no, that’s something we really have to listen to very hard.



Dr. Kaplan is director, division of medical ethics, New York University Langone Medical Center, New York. He reported a conflict of interest with Johnson & Johnson’s Panel for Compassionate Drug Use.
 

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

This transcript has been edited for clarity.

Hi. I’m Art Caplan, PhD. I’m director of the division of medical ethics at the New York University Grossman School of Medicine.

Every once in a while at my school, I get referrals about interesting or difficult clinical cases where doctors would like some input or advice that they can consider in managing a patient. Sometimes those requests come from other hospitals to me. I’ve been doing that kind of ethics consulting, both as a member of various ethics committees and sometimes individually, when, for various reasons, doctors don’t want to go to the Ethics Committee as a first stop.

There was a very interesting case recently involving a young woman I’m going to call Tinslee. She was 17 years old and she suffered, sadly, from recurrent metastatic osteogenic sarcoma. She had bone cancer. It had first been diagnosed at the age of 9. She had received chemotherapy and been under that treatment for a while.

If osteosarcoma is treated before it spreads outside the area where it began, the 5-year survival rate for people like her is about 75%. If the cancer spreads outside of the bones and gets into surrounding tissues, organs, or – worse – into the lymph nodes and starts traveling around, the 5-year survival rate drops to about 60%. The two approaches are chemotherapy and amputation. That’s what we have to offer patients like Tinslee.

Initially, her chemotherapy worked. She went to school and enjoyed sports. She was a real fan of softball and tried to manage the team and be involved. At the time I learned about her, she was planning to go to college. Her love of softball remained, but given the recurrence of the cancer, she had no chance to pursue her athletic interests, not only as a player, but also as a manager or even as a coach for younger players. That was all off the table.

She’d been very compliant up until this time with her chemotherapy. When the recommendation came in that she undergo nonstandard chemotherapy because of the reoccurrence, with experimental drugs using an experimental protocol, she said to her family and the doctors that she didn’t want to do it. She would rather die. She couldn’t take any more chemotherapy and she certainly didn’t want to do it if it was experimental, with the outcomes of this intervention being uncertain.

Her dad said, “She can’t decide. She’s a minor. She’s only 17. I want you to do it – administer this novel form of chemotherapy and try to save her.” Her mother said, “Her input matters. I want to listen to her.” Her mom wasn’t as adamant about doing it or not, but she really felt that Tinslee should be heard loudly because she felt she was mature enough or old enough, even though a minor, to really have a position about what it is to undergo chemotherapy.

Time matters in trying to control the spread, and the doctors were pushing for experimental intervention. I should add, by the way, that although it didn’t really drive the decision about whether to do it or not do it, experimental care like this is not covered by most insurance, and it wasn’t covered by their insurance, so they were facing a big bill if the experimental intervention was administered.

There was some money in a grant to cover some of it, but they were going to face some big financial costs. It never came up in my discussions with the doctors about what to do. I’m not sure whether it ever came up with the family’s discussion with the doctors about what to do, or even whether Tinslee was worrying and didn’t want her family to face a financial burden.

I suggested that we bring the family in. We did some counseling. We had a social worker and we brought in a pastor because these people were fairly religious. We talked about all scenarios, including accepting death, knowing that this disease was not likely to go into remission with the experimental effort; maybe it would, but the doctors were not optimistic.

We tried to talk about how much we should listen to what this young woman wanted. We knew there was the possibility of going to court and having a judge decide this, but in my experience, I do not like going to judges and courts because I know what they’re going to say. They almost always say “administer the intervention.” They don’t want to be in a position of saying don’t do something. They’re a little less willing to do that if something is experimental, but generally speaking, if you’re headed to court, it’s because you’ve decided that you want this to happen.

I felt, in all honesty, that this young woman should have some real respect of her position because the treatment was experimental. She is approaching the age of competency and consent, and she’s been through many interventions. She knows what’s involved. I think you really have to listen hard to what she’s saying.

By the way, after this case, I looked and there have been some surveys of residents in pediatrics. A large number of them said that they hadn’t received any training about what to do when mature minors refuse experimental treatments. The study I saw said that 30% had not undergone any training about this, so we certainly want to introduce that into the appropriate areas of medicine and talk about this with residents and fellows.

Long story short, we had the family meeting, we had another meeting with dad and mom and Tinslee, and the dad began to come around and he began to listen hard. Tinslee said what she wanted was to go to her prom. She wanted to get to her sister’s junior high school softball championship game. If you will, setting some smaller goals that seemed to make her very, very happy began to satisfy mom and dad and they could accept her refusal.

Ultimately, an agreement was reached that she would not undergo the experimental intervention. We agreed on a course of palliative care, recommended that as what the doctors follow, and they decided to do so. Sadly, Tinslee died. She died at home. She did make it to her prom.

I think the outcome, while difficult, sad, tragic, and a close call, was correct. Mature minors who have been through a rough life of interventions and know the price to pay – and for those who have recurrent disease and now face only experimental options – if they say no, that’s something we really have to listen to very hard.



Dr. Kaplan is director, division of medical ethics, New York University Langone Medical Center, New York. He reported a conflict of interest with Johnson & Johnson’s Panel for Compassionate Drug Use.
 

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