Alicia Ault

Physicians should be paid for the time they spend counseling Medicare patients on end-of-life matters, according to several dozen members of Congress.

Rep. Earl Blumenauer (D-Ore.) and 33 other House members have written to the Centers for Medicare & Medicaid Services, urging the agency to adopt new CPT codes created by the American Medical Association’s Relative Value Update Committee. The codes – 99497 and 99498 – would reimburse physicians for discussing advance care planning.

Courtesy of Rep. Blumenauer

Code 99497 would be used for advance care planning, including the explanation and discussion of advance directives, such as standard forms (with completion of such forms, when performed), by the physician or other qualified health care professional, and would cover the first 30 minutes, face to face with the patient, family member(s), and/or surrogate. Code 99498 would be employed for each additional 30 minutes of counseling.

The codes are included the 2015 CPT code set and can be used as of Jan. 1, if they are accepted and published in the Medicare physician fee schedule. The fee schedule is scheduled to be published by Nov. 1.

“Patients who wish to make clear their goals, values, and wishes through discussions with their trusted providers should have the opportunity to do so,” Rep. Blumenauer said in a statement. “In order to have these conversations, providers must have the adequate time, space, and reimbursement to conduct the complex and time-consuming discussions necessary to learn about the goals and values held by their patients and plan appropriately for their care.”

The Institute of Medicine also recently issued a report that called for reimbursement for physicians who talk to their patients about end-of-life issues.

[email protected]

On Twitter @aliciaault

Physicians should be paid for the time they spend counseling Medicare patients on end-of-life matters, according to several dozen members of Congress.

Rep. Earl Blumenauer (D-Ore.) and 33 other House members have written to the Centers for Medicare & Medicaid Services, urging the agency to adopt new CPT codes created by the American Medical Association’s Relative Value Update Committee. The codes – 99497 and 99498 – would reimburse physicians for discussing advance care planning.

Courtesy of Rep. Blumenauer

Code 99497 would be used for advance care planning, including the explanation and discussion of advance directives, such as standard forms (with completion of such forms, when performed), by the physician or other qualified health care professional, and would cover the first 30 minutes, face to face with the patient, family member(s), and/or surrogate. Code 99498 would be employed for each additional 30 minutes of counseling.

The codes are included the 2015 CPT code set and can be used as of Jan. 1, if they are accepted and published in the Medicare physician fee schedule. The fee schedule is scheduled to be published by Nov. 1.

“Patients who wish to make clear their goals, values, and wishes through discussions with their trusted providers should have the opportunity to do so,” Rep. Blumenauer said in a statement. “In order to have these conversations, providers must have the adequate time, space, and reimbursement to conduct the complex and time-consuming discussions necessary to learn about the goals and values held by their patients and plan appropriately for their care.”

The Institute of Medicine also recently issued a report that called for reimbursement for physicians who talk to their patients about end-of-life issues.

[email protected]

On Twitter @aliciaault

Physicians should be paid for the time they spend counseling Medicare patients on end-of-life matters, according to several dozen members of Congress.

Rep. Earl Blumenauer (D-Ore.) and 33 other House members have written to the Centers for Medicare & Medicaid Services, urging the agency to adopt new CPT codes created by the American Medical Association’s Relative Value Update Committee. The codes – 99497 and 99498 – would reimburse physicians for discussing advance care planning.

Courtesy of Rep. Blumenauer

Code 99497 would be used for advance care planning, including the explanation and discussion of advance directives, such as standard forms (with completion of such forms, when performed), by the physician or other qualified health care professional, and would cover the first 30 minutes, face to face with the patient, family member(s), and/or surrogate. Code 99498 would be employed for each additional 30 minutes of counseling.

The codes are included the 2015 CPT code set and can be used as of Jan. 1, if they are accepted and published in the Medicare physician fee schedule. The fee schedule is scheduled to be published by Nov. 1.

“Patients who wish to make clear their goals, values, and wishes through discussions with their trusted providers should have the opportunity to do so,” Rep. Blumenauer said in a statement. “In order to have these conversations, providers must have the adequate time, space, and reimbursement to conduct the complex and time-consuming discussions necessary to learn about the goals and values held by their patients and plan appropriately for their care.”

The Institute of Medicine also recently issued a report that called for reimbursement for physicians who talk to their patients about end-of-life issues.

[email protected]

On Twitter @aliciaault

Finding healthy stool donors for fecal transplant may be a tough prospect.

That’s what Australian researchers have discovered in the course of the FOCUS trial, which aims to determine whether fecal microbiota transplantation (FMT) is safe and efficacious in the treatment of chronic active ulcerative colitis and in the induction of remission.

Dr. Sudarshan Paramsothy and his colleagues at the University of New South Wales, Sydney, and the University of Melbourne, reported findings from donor recruitment for the FOCUS (Faecal Microbiota Transplantation in Ulcerative Colitis) trial at the American Gastroenterological Association’s 2014 James W. Freston Conference in Chicago.

The FOCUS study began enrolling patients in November, and is continuing to enroll, said Dr. Paramsothy. He and his colleagues also are continuing to recruit fecal donors. The data presented in Chicago were on an initial recruitment effort.

Overall, after screening, only 10% of recruits were considered eligible donors.

The researchers recruited donors through letters, newspaper ads, and online solicitations. They were told that they would be reimbursed for their time and for the transportation of their stool donations to the study site.

After responding, recruits were told that they would be expected to make stool donations five times a week for a minimum of 6 weeks.

The researchers had 116 potential donors over a 7-month recruitment period. Forty-seven declined immediately because of the 5-day-a-week donation requirement.

Twenty-seven had other issues, including medical comorbidities (13), risk factors for variant Cruetzfeldt-Jakob disease (6), and recent antibiotic use (1), that disqualified them from the study.

Thirty-eight potentially healthy donors underwent stool and blood testing. Fifteen of those donors were found to have a variety of parasites or indications of active infection that excluded them from donation: 5 had Dientamoeba fragilis, 5 had Blastocystis hominis, 1 had B. hominis and D. fragilis, 1 had Giardia intestinalis and D. fragilis, and 1 had norovirus and Clostridium difficile toxin, and 2 had leukocytes or erythrocytes on stool microscopy. One donor had indeterminate hepatitis C serology.

While it is not uncommon for people to have asymptomatic parasite carriage in the gastrointestinal tract, “we did not expect it in such a high proportion,” said Dr. Paramsothy. “Our screened donor population was not an at-risk group,” he said, adding that they were otherwise healthy and had no risk factors or gastrointestinal symptoms.

“Our detection rates may have been slightly higher as donor stool samples were sent to a pathology center with expert, specialized GI parasitologists for review,” Dr. Paramsothy said.

There’s also some question as to whether some parasites, such as Blastocystis and Dientamoeba, “are truly pathogenic or rather commensal organisms,” he said, adding that it was thought better to exclude patients with these parasites if there were any doubt.

That left 22 potential donors. Further questioning found that two had used antibiotics in between recruitment and stool testing, and one was living with a household member who was positive for D. fragilis.

Of the 19 remaining, 1 dropped out and 18 were screened again. Three were excluded because of a body mass index over 30 kg/m2, 1 because of illicit drug use, 1 because of irregular bowel movements after starting a new medication, and 1 because of uncontrolled anxiety and depression. Dr. Paramsothy said that high-BMI donors were excluded because some studies have shown that gut microbiota potentially influence insulin sensitivity and obesity. Illicit drug use is a red flag because it is potentially associated with blood-borne disease acquisition, he said.

At the end, there were only 12 healthy donors, 10% of the starting 116. Dr. Paramsothy said that it was not necessary to have a single donor for every single patient in the trial. He said he could not disclose currently the number needed for the study, however.

The donor results “suggest that while FMT is an exciting new therapy, it is difficult to identify appropriate and willing anonymous donors,” Dr. Paramsothy said. But that should not have an overall impact on FMT as a therapy, he said – rather, it might just make it harder for a small practice to establish an in-house FMT program.

Dr. Paramsothy reported no relevant financial conflicts.

[email protected]

Finding healthy stool donors for fecal transplant may be a tough prospect.

That’s what Australian researchers have discovered in the course of the FOCUS trial, which aims to determine whether fecal microbiota transplantation (FMT) is safe and efficacious in the treatment of chronic active ulcerative colitis and in the induction of remission.

Dr. Sudarshan Paramsothy and his colleagues at the University of New South Wales, Sydney, and the University of Melbourne, reported findings from donor recruitment for the FOCUS (Faecal Microbiota Transplantation in Ulcerative Colitis) trial at the American Gastroenterological Association’s 2014 James W. Freston Conference in Chicago.

The FOCUS study began enrolling patients in November, and is continuing to enroll, said Dr. Paramsothy. He and his colleagues also are continuing to recruit fecal donors. The data presented in Chicago were on an initial recruitment effort.

Overall, after screening, only 10% of recruits were considered eligible donors.

The researchers recruited donors through letters, newspaper ads, and online solicitations. They were told that they would be reimbursed for their time and for the transportation of their stool donations to the study site.

After responding, recruits were told that they would be expected to make stool donations five times a week for a minimum of 6 weeks.

The researchers had 116 potential donors over a 7-month recruitment period. Forty-seven declined immediately because of the 5-day-a-week donation requirement.

Twenty-seven had other issues, including medical comorbidities (13), risk factors for variant Cruetzfeldt-Jakob disease (6), and recent antibiotic use (1), that disqualified them from the study.

Thirty-eight potentially healthy donors underwent stool and blood testing. Fifteen of those donors were found to have a variety of parasites or indications of active infection that excluded them from donation: 5 had Dientamoeba fragilis, 5 had Blastocystis hominis, 1 had B. hominis and D. fragilis, 1 had Giardia intestinalis and D. fragilis, and 1 had norovirus and Clostridium difficile toxin, and 2 had leukocytes or erythrocytes on stool microscopy. One donor had indeterminate hepatitis C serology.

While it is not uncommon for people to have asymptomatic parasite carriage in the gastrointestinal tract, “we did not expect it in such a high proportion,” said Dr. Paramsothy. “Our screened donor population was not an at-risk group,” he said, adding that they were otherwise healthy and had no risk factors or gastrointestinal symptoms.

“Our detection rates may have been slightly higher as donor stool samples were sent to a pathology center with expert, specialized GI parasitologists for review,” Dr. Paramsothy said.

There’s also some question as to whether some parasites, such as Blastocystis and Dientamoeba, “are truly pathogenic or rather commensal organisms,” he said, adding that it was thought better to exclude patients with these parasites if there were any doubt.

That left 22 potential donors. Further questioning found that two had used antibiotics in between recruitment and stool testing, and one was living with a household member who was positive for D. fragilis.

Of the 19 remaining, 1 dropped out and 18 were screened again. Three were excluded because of a body mass index over 30 kg/m2, 1 because of illicit drug use, 1 because of irregular bowel movements after starting a new medication, and 1 because of uncontrolled anxiety and depression. Dr. Paramsothy said that high-BMI donors were excluded because some studies have shown that gut microbiota potentially influence insulin sensitivity and obesity. Illicit drug use is a red flag because it is potentially associated with blood-borne disease acquisition, he said.

At the end, there were only 12 healthy donors, 10% of the starting 116. Dr. Paramsothy said that it was not necessary to have a single donor for every single patient in the trial. He said he could not disclose currently the number needed for the study, however.

The donor results “suggest that while FMT is an exciting new therapy, it is difficult to identify appropriate and willing anonymous donors,” Dr. Paramsothy said. But that should not have an overall impact on FMT as a therapy, he said – rather, it might just make it harder for a small practice to establish an in-house FMT program.

Dr. Paramsothy reported no relevant financial conflicts.

[email protected]

Finding healthy stool donors for fecal transplant may be a tough prospect.

That’s what Australian researchers have discovered in the course of the FOCUS trial, which aims to determine whether fecal microbiota transplantation (FMT) is safe and efficacious in the treatment of chronic active ulcerative colitis and in the induction of remission.

Dr. Sudarshan Paramsothy and his colleagues at the University of New South Wales, Sydney, and the University of Melbourne, reported findings from donor recruitment for the FOCUS (Faecal Microbiota Transplantation in Ulcerative Colitis) trial at the American Gastroenterological Association’s 2014 James W. Freston Conference in Chicago.

The FOCUS study began enrolling patients in November, and is continuing to enroll, said Dr. Paramsothy. He and his colleagues also are continuing to recruit fecal donors. The data presented in Chicago were on an initial recruitment effort.

Overall, after screening, only 10% of recruits were considered eligible donors.

The researchers recruited donors through letters, newspaper ads, and online solicitations. They were told that they would be reimbursed for their time and for the transportation of their stool donations to the study site.

After responding, recruits were told that they would be expected to make stool donations five times a week for a minimum of 6 weeks.

The researchers had 116 potential donors over a 7-month recruitment period. Forty-seven declined immediately because of the 5-day-a-week donation requirement.

Twenty-seven had other issues, including medical comorbidities (13), risk factors for variant Cruetzfeldt-Jakob disease (6), and recent antibiotic use (1), that disqualified them from the study.

Thirty-eight potentially healthy donors underwent stool and blood testing. Fifteen of those donors were found to have a variety of parasites or indications of active infection that excluded them from donation: 5 had Dientamoeba fragilis, 5 had Blastocystis hominis, 1 had B. hominis and D. fragilis, 1 had Giardia intestinalis and D. fragilis, and 1 had norovirus and Clostridium difficile toxin, and 2 had leukocytes or erythrocytes on stool microscopy. One donor had indeterminate hepatitis C serology.

While it is not uncommon for people to have asymptomatic parasite carriage in the gastrointestinal tract, “we did not expect it in such a high proportion,” said Dr. Paramsothy. “Our screened donor population was not an at-risk group,” he said, adding that they were otherwise healthy and had no risk factors or gastrointestinal symptoms.

“Our detection rates may have been slightly higher as donor stool samples were sent to a pathology center with expert, specialized GI parasitologists for review,” Dr. Paramsothy said.

There’s also some question as to whether some parasites, such as Blastocystis and Dientamoeba, “are truly pathogenic or rather commensal organisms,” he said, adding that it was thought better to exclude patients with these parasites if there were any doubt.

That left 22 potential donors. Further questioning found that two had used antibiotics in between recruitment and stool testing, and one was living with a household member who was positive for D. fragilis.

Of the 19 remaining, 1 dropped out and 18 were screened again. Three were excluded because of a body mass index over 30 kg/m2, 1 because of illicit drug use, 1 because of irregular bowel movements after starting a new medication, and 1 because of uncontrolled anxiety and depression. Dr. Paramsothy said that high-BMI donors were excluded because some studies have shown that gut microbiota potentially influence insulin sensitivity and obesity. Illicit drug use is a red flag because it is potentially associated with blood-borne disease acquisition, he said.

At the end, there were only 12 healthy donors, 10% of the starting 116. Dr. Paramsothy said that it was not necessary to have a single donor for every single patient in the trial. He said he could not disclose currently the number needed for the study, however.

The donor results “suggest that while FMT is an exciting new therapy, it is difficult to identify appropriate and willing anonymous donors,” Dr. Paramsothy said. But that should not have an overall impact on FMT as a therapy, he said – rather, it might just make it harder for a small practice to establish an in-house FMT program.

Dr. Paramsothy reported no relevant financial conflicts.

[email protected]

WASHINGTON – While the U.S. waits for its first potential case of Ebola from a traveler, earlier this spring, officials at one hospital in Canada thought that they had a case and found themselves woefully unprepared.

In late March, a man who had returned from Liberia and had a fever of unknown origin, was admitted through the emergency department to the intensive care unit at St. Paul’s Hospital in Saskatoon, Sask. The clinicians suspected Ebola, but weren’t sure and late that night called the Saskatoon regional health department for a consultation.

Dr. Joseph Blondeau, interim head of pathology and laboratory medicine for the Saskatoon Health Region and the Royal University Hospital, took the call and set a process in motion that had been established for just such a moment, but later proved to have a variety of shortcomings, said Dr. Blondeau during a presentation at the Interscience Conference on Antimicrobial Agents and Chemotherapy.

Initially, the ICU clinicians treated the man empirically with a third-generation cephalosporin and vancomycin. There were still not enough details to make a definitive diagnosis, but by early the next morning there was a high level of suspicion. The patient was hemorrhaging from his eye, there was blood in his nasogastric tube, some rectal bleeding, and a diffuse and nonspecific rash.

Dr. Blondeau ordered all specimens from the man to be quarantined, and he activated the emergency response system for Canada. At that time, there were 27 cases in Liberia, with a 40% mortality rate.

Alarmingly, one of the patient’s cerebrospinal fluid specimens had leaked in transit from St. Paul’s Hospital to a biocontainment lab at the Royal University hospital. A technician at the lab attempted to clean the container and was potentially exposed.

Both St. Paul and Royal University hospitals decided they needed a communication strategy to help allay fear and anxiety among the staff. Many were questioning why the patient was not elevated to a high infection threat when admitted to the ICU, and why higher-level precautions had not been put into place earlier.

On his way to an emergency meeting at St. Paul’s to discuss these issues, Dr. Blondeau received a call from the director of one of Canada’s biosafety level 4 labs, the National Microbiology Lab in Winnipeg, Man., who said that it was likely the patient had Ebola.

“At that point in time all hell broke loose,” said Dr. Blondeau.

The patient’s specimens needed to be immediately transported for confirmation to that national lab, a 9-hour drive from Saskatoon. The regular couriers weren’t interested. Dr. Blondeau volunteered, initially thinking he would drive them.

The specimens were prepared and packaged for containment, but the government made the decision to transport them by jet instead.

Even so, Dr. Blondeau had to drive the specimens to the air ambulance that was waiting at the Saskatoon airport. He wondered whether that was the right decision. There were questions as to whether law enforcement should be informed of the transport – what if he had an accident? He put a sign in the windshield stating that he was transporting a potential Ebola specimen as a means of making it look official, and so that no one would mistake him for a terrorist. No one had worked out whether he should accompany the specimens to Winnipeg to maintain a chain of control. He did not go.

Simultaneously, the health authorities began trying to track down all of the patient’s contacts, from arrival in the country, through an urgent clinic visit, a busy emergency room, and staff and family visits after ICU admission.

Meanwhile, the patient was deteriorating and was already ventilated and required cardiovascular support. Dr. Blondeau began discussions to bring a more sophisticated mobile lab to Saskatoon so that the patient could be repeatedly tested on-site.

“I can say with certainty that there were not many people in the province of Saskatchewan who fully understood what was about to happen should this patient have tested positive for Ebola,” said Dr. Blondeau. “We were learning as we went.”

The plans were changing by the moment, he said.

And, he said, he still had many concerns about how the situation would be perceived by those inside and outside the hospital. There was a potential for public panic and for a breach of the patient’s and family’s privacy.

Among the staff, “there was tremendous fear and panic,” Dr. Blondeau said. The spouse of the lab technician who had a potential exposure wanted her to quit her job. Another technician was spreading incorrect information, he said.

While staff worried about their own exposure and whether they had exposed their families, the patient was still critically ill and needed care and acute testing.

Then, just 24 hours after the patient had been admitted, it was determined that he did not have Ebola or any other viral hemorrhagic fever.

But “we still didn’t have a diagnosis,” said Dr. Blondeau.

He ordered routine microbiology testing on all the specimens. A day later, it looked like the culprit was Staphylococcus aureus. Further testing confirmed that it was indeed S. aureus and that it was a methicillin-susceptible strain.

Officials and staff went back to routine care processes.

In retrospect, there was much to be concerned about, said Dr. Blondeau. Use of personal protective equipment was inconsistent, which could have led to exposures. There was uncertainty about how to keep the environment clean, including linens and uniforms. For instance, he noted, many health care staff wear uniforms to work or wear them home. “Is this a practice we should be endorsing?” he asked.

There were potential problems with the physical space; for instance, some patient room doors did not close tightly.

On the plus side, no staff refused to care for the patient or to do what was asked, said Dr. Blondeau.

The entire 96-hour experience “was exciting but it was terrifying,” he said.

The lack of preparedness and the lack of a more tightly-knit lab system in the U.S. and Canada are warning signs, he said.

“The reality is we’re only the next landing flight away from a potential infectious disease threat,” said Dr. Blondeau.

On Aug. 1, Saskatoon health authorities received an alert that a passenger on an inbound flight from Senegal had many of the symptoms of a viral hemorrhagic fever: vomiting, diarrhea, and headache. They put their response system in place, and “were much better prepared the second time around,” he said, adding, “but we aren’t where we need to be.”

Dr. Blondeau reported having no conflicts of interest.

[email protected]

On Twitter @aliciaault

WASHINGTON – While the U.S. waits for its first potential case of Ebola from a traveler, earlier this spring, officials at one hospital in Canada thought that they had a case and found themselves woefully unprepared.

In late March, a man who had returned from Liberia and had a fever of unknown origin, was admitted through the emergency department to the intensive care unit at St. Paul’s Hospital in Saskatoon, Sask. The clinicians suspected Ebola, but weren’t sure and late that night called the Saskatoon regional health department for a consultation.

Dr. Joseph Blondeau, interim head of pathology and laboratory medicine for the Saskatoon Health Region and the Royal University Hospital, took the call and set a process in motion that had been established for just such a moment, but later proved to have a variety of shortcomings, said Dr. Blondeau during a presentation at the Interscience Conference on Antimicrobial Agents and Chemotherapy.

Initially, the ICU clinicians treated the man empirically with a third-generation cephalosporin and vancomycin. There were still not enough details to make a definitive diagnosis, but by early the next morning there was a high level of suspicion. The patient was hemorrhaging from his eye, there was blood in his nasogastric tube, some rectal bleeding, and a diffuse and nonspecific rash.

Dr. Blondeau ordered all specimens from the man to be quarantined, and he activated the emergency response system for Canada. At that time, there were 27 cases in Liberia, with a 40% mortality rate.

Alarmingly, one of the patient’s cerebrospinal fluid specimens had leaked in transit from St. Paul’s Hospital to a biocontainment lab at the Royal University hospital. A technician at the lab attempted to clean the container and was potentially exposed.

Both St. Paul and Royal University hospitals decided they needed a communication strategy to help allay fear and anxiety among the staff. Many were questioning why the patient was not elevated to a high infection threat when admitted to the ICU, and why higher-level precautions had not been put into place earlier.

On his way to an emergency meeting at St. Paul’s to discuss these issues, Dr. Blondeau received a call from the director of one of Canada’s biosafety level 4 labs, the National Microbiology Lab in Winnipeg, Man., who said that it was likely the patient had Ebola.

“At that point in time all hell broke loose,” said Dr. Blondeau.

The patient’s specimens needed to be immediately transported for confirmation to that national lab, a 9-hour drive from Saskatoon. The regular couriers weren’t interested. Dr. Blondeau volunteered, initially thinking he would drive them.

The specimens were prepared and packaged for containment, but the government made the decision to transport them by jet instead.

Even so, Dr. Blondeau had to drive the specimens to the air ambulance that was waiting at the Saskatoon airport. He wondered whether that was the right decision. There were questions as to whether law enforcement should be informed of the transport – what if he had an accident? He put a sign in the windshield stating that he was transporting a potential Ebola specimen as a means of making it look official, and so that no one would mistake him for a terrorist. No one had worked out whether he should accompany the specimens to Winnipeg to maintain a chain of control. He did not go.

Simultaneously, the health authorities began trying to track down all of the patient’s contacts, from arrival in the country, through an urgent clinic visit, a busy emergency room, and staff and family visits after ICU admission.

Meanwhile, the patient was deteriorating and was already ventilated and required cardiovascular support. Dr. Blondeau began discussions to bring a more sophisticated mobile lab to Saskatoon so that the patient could be repeatedly tested on-site.

“I can say with certainty that there were not many people in the province of Saskatchewan who fully understood what was about to happen should this patient have tested positive for Ebola,” said Dr. Blondeau. “We were learning as we went.”

The plans were changing by the moment, he said.

And, he said, he still had many concerns about how the situation would be perceived by those inside and outside the hospital. There was a potential for public panic and for a breach of the patient’s and family’s privacy.

Among the staff, “there was tremendous fear and panic,” Dr. Blondeau said. The spouse of the lab technician who had a potential exposure wanted her to quit her job. Another technician was spreading incorrect information, he said.

While staff worried about their own exposure and whether they had exposed their families, the patient was still critically ill and needed care and acute testing.

Then, just 24 hours after the patient had been admitted, it was determined that he did not have Ebola or any other viral hemorrhagic fever.

But “we still didn’t have a diagnosis,” said Dr. Blondeau.

He ordered routine microbiology testing on all the specimens. A day later, it looked like the culprit was Staphylococcus aureus. Further testing confirmed that it was indeed S. aureus and that it was a methicillin-susceptible strain.

Officials and staff went back to routine care processes.

In retrospect, there was much to be concerned about, said Dr. Blondeau. Use of personal protective equipment was inconsistent, which could have led to exposures. There was uncertainty about how to keep the environment clean, including linens and uniforms. For instance, he noted, many health care staff wear uniforms to work or wear them home. “Is this a practice we should be endorsing?” he asked.

There were potential problems with the physical space; for instance, some patient room doors did not close tightly.

On the plus side, no staff refused to care for the patient or to do what was asked, said Dr. Blondeau.

The entire 96-hour experience “was exciting but it was terrifying,” he said.

The lack of preparedness and the lack of a more tightly-knit lab system in the U.S. and Canada are warning signs, he said.

“The reality is we’re only the next landing flight away from a potential infectious disease threat,” said Dr. Blondeau.

On Aug. 1, Saskatoon health authorities received an alert that a passenger on an inbound flight from Senegal had many of the symptoms of a viral hemorrhagic fever: vomiting, diarrhea, and headache. They put their response system in place, and “were much better prepared the second time around,” he said, adding, “but we aren’t where we need to be.”

Dr. Blondeau reported having no conflicts of interest.

[email protected]

On Twitter @aliciaault

WASHINGTON – While the U.S. waits for its first potential case of Ebola from a traveler, earlier this spring, officials at one hospital in Canada thought that they had a case and found themselves woefully unprepared.

In late March, a man who had returned from Liberia and had a fever of unknown origin, was admitted through the emergency department to the intensive care unit at St. Paul’s Hospital in Saskatoon, Sask. The clinicians suspected Ebola, but weren’t sure and late that night called the Saskatoon regional health department for a consultation.

Dr. Joseph Blondeau, interim head of pathology and laboratory medicine for the Saskatoon Health Region and the Royal University Hospital, took the call and set a process in motion that had been established for just such a moment, but later proved to have a variety of shortcomings, said Dr. Blondeau during a presentation at the Interscience Conference on Antimicrobial Agents and Chemotherapy.

Initially, the ICU clinicians treated the man empirically with a third-generation cephalosporin and vancomycin. There were still not enough details to make a definitive diagnosis, but by early the next morning there was a high level of suspicion. The patient was hemorrhaging from his eye, there was blood in his nasogastric tube, some rectal bleeding, and a diffuse and nonspecific rash.

Dr. Blondeau ordered all specimens from the man to be quarantined, and he activated the emergency response system for Canada. At that time, there were 27 cases in Liberia, with a 40% mortality rate.

Alarmingly, one of the patient’s cerebrospinal fluid specimens had leaked in transit from St. Paul’s Hospital to a biocontainment lab at the Royal University hospital. A technician at the lab attempted to clean the container and was potentially exposed.

Both St. Paul and Royal University hospitals decided they needed a communication strategy to help allay fear and anxiety among the staff. Many were questioning why the patient was not elevated to a high infection threat when admitted to the ICU, and why higher-level precautions had not been put into place earlier.

On his way to an emergency meeting at St. Paul’s to discuss these issues, Dr. Blondeau received a call from the director of one of Canada’s biosafety level 4 labs, the National Microbiology Lab in Winnipeg, Man., who said that it was likely the patient had Ebola.

“At that point in time all hell broke loose,” said Dr. Blondeau.

The patient’s specimens needed to be immediately transported for confirmation to that national lab, a 9-hour drive from Saskatoon. The regular couriers weren’t interested. Dr. Blondeau volunteered, initially thinking he would drive them.

The specimens were prepared and packaged for containment, but the government made the decision to transport them by jet instead.

Even so, Dr. Blondeau had to drive the specimens to the air ambulance that was waiting at the Saskatoon airport. He wondered whether that was the right decision. There were questions as to whether law enforcement should be informed of the transport – what if he had an accident? He put a sign in the windshield stating that he was transporting a potential Ebola specimen as a means of making it look official, and so that no one would mistake him for a terrorist. No one had worked out whether he should accompany the specimens to Winnipeg to maintain a chain of control. He did not go.

Simultaneously, the health authorities began trying to track down all of the patient’s contacts, from arrival in the country, through an urgent clinic visit, a busy emergency room, and staff and family visits after ICU admission.

Meanwhile, the patient was deteriorating and was already ventilated and required cardiovascular support. Dr. Blondeau began discussions to bring a more sophisticated mobile lab to Saskatoon so that the patient could be repeatedly tested on-site.

“I can say with certainty that there were not many people in the province of Saskatchewan who fully understood what was about to happen should this patient have tested positive for Ebola,” said Dr. Blondeau. “We were learning as we went.”

The plans were changing by the moment, he said.

And, he said, he still had many concerns about how the situation would be perceived by those inside and outside the hospital. There was a potential for public panic and for a breach of the patient’s and family’s privacy.

Among the staff, “there was tremendous fear and panic,” Dr. Blondeau said. The spouse of the lab technician who had a potential exposure wanted her to quit her job. Another technician was spreading incorrect information, he said.

While staff worried about their own exposure and whether they had exposed their families, the patient was still critically ill and needed care and acute testing.

Then, just 24 hours after the patient had been admitted, it was determined that he did not have Ebola or any other viral hemorrhagic fever.

But “we still didn’t have a diagnosis,” said Dr. Blondeau.

He ordered routine microbiology testing on all the specimens. A day later, it looked like the culprit was Staphylococcus aureus. Further testing confirmed that it was indeed S. aureus and that it was a methicillin-susceptible strain.

Officials and staff went back to routine care processes.

In retrospect, there was much to be concerned about, said Dr. Blondeau. Use of personal protective equipment was inconsistent, which could have led to exposures. There was uncertainty about how to keep the environment clean, including linens and uniforms. For instance, he noted, many health care staff wear uniforms to work or wear them home. “Is this a practice we should be endorsing?” he asked.

There were potential problems with the physical space; for instance, some patient room doors did not close tightly.

On the plus side, no staff refused to care for the patient or to do what was asked, said Dr. Blondeau.

The entire 96-hour experience “was exciting but it was terrifying,” he said.

The lack of preparedness and the lack of a more tightly-knit lab system in the U.S. and Canada are warning signs, he said.

“The reality is we’re only the next landing flight away from a potential infectious disease threat,” said Dr. Blondeau.

On Aug. 1, Saskatoon health authorities received an alert that a passenger on an inbound flight from Senegal had many of the symptoms of a viral hemorrhagic fever: vomiting, diarrhea, and headache. They put their response system in place, and “were much better prepared the second time around,” he said, adding, “but we aren’t where we need to be.”

Dr. Blondeau reported having no conflicts of interest.

[email protected]

On Twitter @aliciaault

First-time meaningful use participants who are using older technology, but who did not get a hardship exemption by July 1, will see their Medicare pay reduced 1% in 2015.

Because of a confluence of factors, a certain subset of physicians who engaged in meaningful use for the first time this year will not be able to attest to their participation by the Oct. 1 deadline set by the Centers for Medicare & Medicaid Services – and thus, will be penalized.

“Physicians are not only frustrated, but perhaps reaching despondency on the topic,” Dr. Steven J. Stack, president-elect of the American Medical Association, said in an interview.

Overall, about half of physicians had not participated in meaningful use as of the beginning of this year. Theoretically, all of them could face the 1% reduction in Medicare pay, though it’s likely that a smaller subset will, Dr. Stack said.

Those who did not meet meaningful use criteria in 2013 and who anticipated that they would not do so again in 2014 could have applied for a hardship exemption by July 1 to avoid a penalty in 2015. Physicians who were starting on the meaningful use process for the first time in 2014 also had until July 1 to apply for that exemption.

After determining that many vendors did not have 2014 software ready for physicians, CMS proposed in late May to give all meaningful users more flexibility. Physicians were told they could use either a 2011 version, a 2014 version, or some combination, and not be penalized in 2015. That proposal was made final in late August.

Applying for that flexibility will be done via the CMS website, which is slated to be ready a few weeks after Oct. 1.

But first-time meaningful use participants must make their attestation to the CMS by Oct. 1.

The bottom line is that first-time participants who are using older technology and did not get the exemption will be penalized.

A CMS spokesman said that they could still receive an incentive payment for 2014 – if they go online in mid-October and apply for the flexibility option.

But Dr. Stack called that little consolation. It is as if the CMS took away $100 of a $1,000 pot and said that there was still $900 left, he said.

“It doesn’t seem reasonable and certainly undermines the premise that they offered any kind of relief,” said Dr. Stack.

Thom Kuhn, a staff member at the American College of Physicians, also said that the CMS explanation was not good enough.

“Failure to have a system ready by the time a final rule is issued is a management failure,” said Mr. Kuhn, in an interview.

Meanwhile, two members of Congress - Rep. Renee Ellmers (R-N.C.) and Rep. Jim Matheson (D-Utah) - have called on the CMS to extend that Oct. 1 deadline. In a letter to the agency, they requested "an administrative delay" for those attempting to attest to meaningful use for the first time in 2014.*

[email protected]

On Twitter @aliciaault

*Correction, 9/24/2014: An earlier version of this article implied that the administrative delay was requested in legislation. It was requested in a letter.

First-time meaningful use participants who are using older technology, but who did not get a hardship exemption by July 1, will see their Medicare pay reduced 1% in 2015.

Because of a confluence of factors, a certain subset of physicians who engaged in meaningful use for the first time this year will not be able to attest to their participation by the Oct. 1 deadline set by the Centers for Medicare & Medicaid Services – and thus, will be penalized.

“Physicians are not only frustrated, but perhaps reaching despondency on the topic,” Dr. Steven J. Stack, president-elect of the American Medical Association, said in an interview.

Overall, about half of physicians had not participated in meaningful use as of the beginning of this year. Theoretically, all of them could face the 1% reduction in Medicare pay, though it’s likely that a smaller subset will, Dr. Stack said.

Those who did not meet meaningful use criteria in 2013 and who anticipated that they would not do so again in 2014 could have applied for a hardship exemption by July 1 to avoid a penalty in 2015. Physicians who were starting on the meaningful use process for the first time in 2014 also had until July 1 to apply for that exemption.

After determining that many vendors did not have 2014 software ready for physicians, CMS proposed in late May to give all meaningful users more flexibility. Physicians were told they could use either a 2011 version, a 2014 version, or some combination, and not be penalized in 2015. That proposal was made final in late August.

Applying for that flexibility will be done via the CMS website, which is slated to be ready a few weeks after Oct. 1.

But first-time meaningful use participants must make their attestation to the CMS by Oct. 1.

The bottom line is that first-time participants who are using older technology and did not get the exemption will be penalized.

A CMS spokesman said that they could still receive an incentive payment for 2014 – if they go online in mid-October and apply for the flexibility option.

But Dr. Stack called that little consolation. It is as if the CMS took away $100 of a $1,000 pot and said that there was still $900 left, he said.

“It doesn’t seem reasonable and certainly undermines the premise that they offered any kind of relief,” said Dr. Stack.

Thom Kuhn, a staff member at the American College of Physicians, also said that the CMS explanation was not good enough.

“Failure to have a system ready by the time a final rule is issued is a management failure,” said Mr. Kuhn, in an interview.

Meanwhile, two members of Congress - Rep. Renee Ellmers (R-N.C.) and Rep. Jim Matheson (D-Utah) - have called on the CMS to extend that Oct. 1 deadline. In a letter to the agency, they requested "an administrative delay" for those attempting to attest to meaningful use for the first time in 2014.*

[email protected]

On Twitter @aliciaault

*Correction, 9/24/2014: An earlier version of this article implied that the administrative delay was requested in legislation. It was requested in a letter.

First-time meaningful use participants who are using older technology, but who did not get a hardship exemption by July 1, will see their Medicare pay reduced 1% in 2015.

Because of a confluence of factors, a certain subset of physicians who engaged in meaningful use for the first time this year will not be able to attest to their participation by the Oct. 1 deadline set by the Centers for Medicare & Medicaid Services – and thus, will be penalized.

“Physicians are not only frustrated, but perhaps reaching despondency on the topic,” Dr. Steven J. Stack, president-elect of the American Medical Association, said in an interview.

Overall, about half of physicians had not participated in meaningful use as of the beginning of this year. Theoretically, all of them could face the 1% reduction in Medicare pay, though it’s likely that a smaller subset will, Dr. Stack said.

Those who did not meet meaningful use criteria in 2013 and who anticipated that they would not do so again in 2014 could have applied for a hardship exemption by July 1 to avoid a penalty in 2015. Physicians who were starting on the meaningful use process for the first time in 2014 also had until July 1 to apply for that exemption.

After determining that many vendors did not have 2014 software ready for physicians, CMS proposed in late May to give all meaningful users more flexibility. Physicians were told they could use either a 2011 version, a 2014 version, or some combination, and not be penalized in 2015. That proposal was made final in late August.

Applying for that flexibility will be done via the CMS website, which is slated to be ready a few weeks after Oct. 1.

But first-time meaningful use participants must make their attestation to the CMS by Oct. 1.

The bottom line is that first-time participants who are using older technology and did not get the exemption will be penalized.

A CMS spokesman said that they could still receive an incentive payment for 2014 – if they go online in mid-October and apply for the flexibility option.

But Dr. Stack called that little consolation. It is as if the CMS took away $100 of a $1,000 pot and said that there was still $900 left, he said.

“It doesn’t seem reasonable and certainly undermines the premise that they offered any kind of relief,” said Dr. Stack.

Thom Kuhn, a staff member at the American College of Physicians, also said that the CMS explanation was not good enough.

“Failure to have a system ready by the time a final rule is issued is a management failure,” said Mr. Kuhn, in an interview.

Meanwhile, two members of Congress - Rep. Renee Ellmers (R-N.C.) and Rep. Jim Matheson (D-Utah) - have called on the CMS to extend that Oct. 1 deadline. In a letter to the agency, they requested "an administrative delay" for those attempting to attest to meaningful use for the first time in 2014.*

[email protected]

On Twitter @aliciaault

*Correction, 9/24/2014: An earlier version of this article implied that the administrative delay was requested in legislation. It was requested in a letter.

Physicians should be compensated for counseling patients on end-of-life planning, according to recommendations from the Institute of Medicine, and the health care delivery and payment system should be realigned to encourage and financially reward higher-quality, more comprehensive, more efficient, and more humane care for those with serious illnesses.

“Individuals should have time with their doctors to talk about end-of-life issues, and clinicians should receive the training and financial incentives for such discussions,” said David Walker, the former comptroller general of the United States, who was the cochair of the IOM committee that created the report published Sept. 17.

Dying in America calls on health care providers, payers, policy makers, and the American public to have a more open discourse about death and dying.

End-of-life care was caught up in a political firestorm in 2009 when the Obama administration proposed to pay for advanced care conversations under the Affordable Care Act, Dr. Harvey Fineberg, former IOM president, noted in a video message at a briefing to release the report. Such efforts were construed as “death panels,” and the administration withdrew its proposal within days of it going into effect in 2011, said Dr. Fineberg, who is now at the University of California, San Francisco.

“The controversy on this topic and the political desire to avoid it do not alter the fact that every person will face the end of life one day, and many have had hard experience with the final days of a parent, a spouse, a child, a sibling, another relative, or a dear friend,” Dr. Fineberg said.

The report focuses on three areas: what individuals and their families can do to take more control over their own life and their health care throughout their life; what clinicians and other professionals can do; and what policy makers and payers need to do to try to effectuate change. The 21-member IOM committee spent 2 years developing the consensus report, Mr. Walker said.

“For most people, death does not come suddenly,” said Dr. Philip Pizzo, committee cochair. “Instead, dying is a result of one or more diseases that must be managed carefully and compassionately over weeks, months, or even years, through many ups and downs,” said Dr. Pizzo of Stanford (Calif.) University.

The committee made five broad recommendations:

• Comprehensive care for patients with advanced, serious illness who are nearing the end of life should be covered by public and private payers.

• Evidence-based standards for clinician-patient communication and advanced care planning should be developed by professional societies; such standards should be used as measures for payment, licensing, and credentialing.

• Standardized training and requirements should be developed and implemented.

• Care standards should seek to avoid unnecessary emergency department or acute care services; care should be coordinated across settings and providers by using tools such as interoperable electronic health records and physician orders for life-sustaining treatment programs.

• Fact-based information on end-of-life care planning should be developed and disseminated broadly through public health and other governmental agencies, community-based organizations, and faith-based organizations, as well as through health care providers and payers.

More needs to be done to educate physicians, other health care providers, and patients about the differences between hospice care and palliative care, and the potential they have for improving quality of life and reducing potentially unnecessary – and costly – medical services, according to the committee report.

Dr. Pizzo noted that many physicians, when asked in surveys, have said that they would personally prefer less-aggressive care at the end of life, and if possible, having the patient receive care at home. Yet, when it comes to their patients, they tend to pull out the stops.

He said that was being driven by a lack of understanding of patients’ individual needs and preferences, and by what he and the committee called the “perverse incentives” of the health care system.

The system rewards more care, not less, and acute care more so than palliative or supportive care, Mr. Walker noted. “Our current system is broken. It does not result in the honoring of individual preferences as much as it should.”

The IOM report was financed by an anonymous donor, Dr. Fineberg said.

The donor made money available for the IOM to continue for at least a year, disseminating the findings and encouraging adoption of its recommendations, Mr. Walker said.

[email protected]

On Twitter @aliciaault

Physicians should be compensated for counseling patients on end-of-life planning, according to recommendations from the Institute of Medicine, and the health care delivery and payment system should be realigned to encourage and financially reward higher-quality, more comprehensive, more efficient, and more humane care for those with serious illnesses.

“Individuals should have time with their doctors to talk about end-of-life issues, and clinicians should receive the training and financial incentives for such discussions,” said David Walker, the former comptroller general of the United States, who was the cochair of the IOM committee that created the report published Sept. 17.

Dying in America calls on health care providers, payers, policy makers, and the American public to have a more open discourse about death and dying.

End-of-life care was caught up in a political firestorm in 2009 when the Obama administration proposed to pay for advanced care conversations under the Affordable Care Act, Dr. Harvey Fineberg, former IOM president, noted in a video message at a briefing to release the report. Such efforts were construed as “death panels,” and the administration withdrew its proposal within days of it going into effect in 2011, said Dr. Fineberg, who is now at the University of California, San Francisco.

“The controversy on this topic and the political desire to avoid it do not alter the fact that every person will face the end of life one day, and many have had hard experience with the final days of a parent, a spouse, a child, a sibling, another relative, or a dear friend,” Dr. Fineberg said.

The report focuses on three areas: what individuals and their families can do to take more control over their own life and their health care throughout their life; what clinicians and other professionals can do; and what policy makers and payers need to do to try to effectuate change. The 21-member IOM committee spent 2 years developing the consensus report, Mr. Walker said.

“For most people, death does not come suddenly,” said Dr. Philip Pizzo, committee cochair. “Instead, dying is a result of one or more diseases that must be managed carefully and compassionately over weeks, months, or even years, through many ups and downs,” said Dr. Pizzo of Stanford (Calif.) University.

The committee made five broad recommendations:

• Comprehensive care for patients with advanced, serious illness who are nearing the end of life should be covered by public and private payers.

• Evidence-based standards for clinician-patient communication and advanced care planning should be developed by professional societies; such standards should be used as measures for payment, licensing, and credentialing.

• Standardized training and requirements should be developed and implemented.

• Care standards should seek to avoid unnecessary emergency department or acute care services; care should be coordinated across settings and providers by using tools such as interoperable electronic health records and physician orders for life-sustaining treatment programs.

• Fact-based information on end-of-life care planning should be developed and disseminated broadly through public health and other governmental agencies, community-based organizations, and faith-based organizations, as well as through health care providers and payers.

More needs to be done to educate physicians, other health care providers, and patients about the differences between hospice care and palliative care, and the potential they have for improving quality of life and reducing potentially unnecessary – and costly – medical services, according to the committee report.

Dr. Pizzo noted that many physicians, when asked in surveys, have said that they would personally prefer less-aggressive care at the end of life, and if possible, having the patient receive care at home. Yet, when it comes to their patients, they tend to pull out the stops.

He said that was being driven by a lack of understanding of patients’ individual needs and preferences, and by what he and the committee called the “perverse incentives” of the health care system.

The system rewards more care, not less, and acute care more so than palliative or supportive care, Mr. Walker noted. “Our current system is broken. It does not result in the honoring of individual preferences as much as it should.”

The IOM report was financed by an anonymous donor, Dr. Fineberg said.

The donor made money available for the IOM to continue for at least a year, disseminating the findings and encouraging adoption of its recommendations, Mr. Walker said.

[email protected]

On Twitter @aliciaault

Physicians should be compensated for counseling patients on end-of-life planning, according to recommendations from the Institute of Medicine, and the health care delivery and payment system should be realigned to encourage and financially reward higher-quality, more comprehensive, more efficient, and more humane care for those with serious illnesses.

“Individuals should have time with their doctors to talk about end-of-life issues, and clinicians should receive the training and financial incentives for such discussions,” said David Walker, the former comptroller general of the United States, who was the cochair of the IOM committee that created the report published Sept. 17.

Dying in America calls on health care providers, payers, policy makers, and the American public to have a more open discourse about death and dying.

End-of-life care was caught up in a political firestorm in 2009 when the Obama administration proposed to pay for advanced care conversations under the Affordable Care Act, Dr. Harvey Fineberg, former IOM president, noted in a video message at a briefing to release the report. Such efforts were construed as “death panels,” and the administration withdrew its proposal within days of it going into effect in 2011, said Dr. Fineberg, who is now at the University of California, San Francisco.

“The controversy on this topic and the political desire to avoid it do not alter the fact that every person will face the end of life one day, and many have had hard experience with the final days of a parent, a spouse, a child, a sibling, another relative, or a dear friend,” Dr. Fineberg said.

The report focuses on three areas: what individuals and their families can do to take more control over their own life and their health care throughout their life; what clinicians and other professionals can do; and what policy makers and payers need to do to try to effectuate change. The 21-member IOM committee spent 2 years developing the consensus report, Mr. Walker said.

“For most people, death does not come suddenly,” said Dr. Philip Pizzo, committee cochair. “Instead, dying is a result of one or more diseases that must be managed carefully and compassionately over weeks, months, or even years, through many ups and downs,” said Dr. Pizzo of Stanford (Calif.) University.

The committee made five broad recommendations:

• Comprehensive care for patients with advanced, serious illness who are nearing the end of life should be covered by public and private payers.

• Evidence-based standards for clinician-patient communication and advanced care planning should be developed by professional societies; such standards should be used as measures for payment, licensing, and credentialing.

• Standardized training and requirements should be developed and implemented.

• Care standards should seek to avoid unnecessary emergency department or acute care services; care should be coordinated across settings and providers by using tools such as interoperable electronic health records and physician orders for life-sustaining treatment programs.

• Fact-based information on end-of-life care planning should be developed and disseminated broadly through public health and other governmental agencies, community-based organizations, and faith-based organizations, as well as through health care providers and payers.

More needs to be done to educate physicians, other health care providers, and patients about the differences between hospice care and palliative care, and the potential they have for improving quality of life and reducing potentially unnecessary – and costly – medical services, according to the committee report.

Dr. Pizzo noted that many physicians, when asked in surveys, have said that they would personally prefer less-aggressive care at the end of life, and if possible, having the patient receive care at home. Yet, when it comes to their patients, they tend to pull out the stops.

He said that was being driven by a lack of understanding of patients’ individual needs and preferences, and by what he and the committee called the “perverse incentives” of the health care system.

The system rewards more care, not less, and acute care more so than palliative or supportive care, Mr. Walker noted. “Our current system is broken. It does not result in the honoring of individual preferences as much as it should.”

The IOM report was financed by an anonymous donor, Dr. Fineberg said.

The donor made money available for the IOM to continue for at least a year, disseminating the findings and encouraging adoption of its recommendations, Mr. Walker said.

[email protected]

On Twitter @aliciaault

The Senate unanimously approved a bill designed to speed up Food and Drug Administration approval of sunscreen ingredients, adding to the likelihood that the legislation will become law.

The full Senate took quick action on Sept. 17, the same day that the Health, Education, Labor & Pensions (HELP) Committee unanimously voted 12-0 in favor of the Sunscreen Innovation Act (S.2141).

©mark wragg/iStockphoto.com

“We have a number of ingredients for sunscreens that have languished at the FDA for years – as long as a decade,” Sen. Johnny Isakson (R-Ga.), a cosponsor of the bill, said at the HELP hearing. “This doesn’t mean Congress makes the decision,” he said, adding that, instead, the law does establish time frames for review.

The legislation “is also about holding the FDA accountable to timelines and reforming the process,” said Sen. Lamar Alexander (R-Tenn.).

The bill would require the FDA to make final decisions within a year on the backlog of ingredients under review, and within a year and a half on new applications. It also sets up more congressional oversight.

At a recent FDA advisory committee hearing on sunscreen ingredient safety, Dr. Theresa Michele, an FDA official, said that eight ingredients were awaiting approval through what was supposed to be an expedited process. These are the same ingredients that have been under review for 10 years or more. The agency has responded to manufacturers of five of the eight, telling them that so far, there’s not enough data to determine whether they can be marketed, said Dr. Michele.

The Senate joins the House in calling on the FDA to move those approvals along. The full House approved companion legislation (H.R.4250) in late July.

The legislation is supported by the American Academy of Dermatology and by consumer advocates and manufacturers, including the Public Access to SunScreens (PASS) Coalition.

“Congress’s commitment to addressing the skin cancer epidemic in the United States was clearly demonstrated in tonight’s Senate passage of the Sunscreen Innovation Act,” said Michael Werner, PASS Coalition Policy Adviser, in a statement after the vote. “We now call on the House and Senate to swiftly reconcile the differences in their bills and enact final legislation,” Werner said.

Chris Hansen, president of the American Cancer Society Cancer Action Network, said in a statement that if the bill became law, it would add more predictability to FDA reviews.

“The Senate took a critical step yesterday to fix a broken process at FDA for the review of new sunscreen ingredients that could potentially help more Americans prevent skin cancer,” he said.

Tim Turnham, executive director of the Melanoma Research Foundation, said in a statement that some of the ingredients under FDA review have been widely available in Europe, Asia, and Central and South America, in some cases for more than 15 years.

“Americans are limited in their choices for compounds that block UV radiation because of the long-standing bureaucratic gridlock at the FDA that prevents new agents from being approved,” said Mr. Turnham.

The House and Senate have to reconcile the two versions of the bill, and then the legislation will have to be approved again by both bodies before being sent to the White House for final approval.

[email protected]

On Twitter @aliciaault

The Senate unanimously approved a bill designed to speed up Food and Drug Administration approval of sunscreen ingredients, adding to the likelihood that the legislation will become law.

The full Senate took quick action on Sept. 17, the same day that the Health, Education, Labor & Pensions (HELP) Committee unanimously voted 12-0 in favor of the Sunscreen Innovation Act (S.2141).

©mark wragg/iStockphoto.com

“We have a number of ingredients for sunscreens that have languished at the FDA for years – as long as a decade,” Sen. Johnny Isakson (R-Ga.), a cosponsor of the bill, said at the HELP hearing. “This doesn’t mean Congress makes the decision,” he said, adding that, instead, the law does establish time frames for review.

The legislation “is also about holding the FDA accountable to timelines and reforming the process,” said Sen. Lamar Alexander (R-Tenn.).

The bill would require the FDA to make final decisions within a year on the backlog of ingredients under review, and within a year and a half on new applications. It also sets up more congressional oversight.

At a recent FDA advisory committee hearing on sunscreen ingredient safety, Dr. Theresa Michele, an FDA official, said that eight ingredients were awaiting approval through what was supposed to be an expedited process. These are the same ingredients that have been under review for 10 years or more. The agency has responded to manufacturers of five of the eight, telling them that so far, there’s not enough data to determine whether they can be marketed, said Dr. Michele.

The Senate joins the House in calling on the FDA to move those approvals along. The full House approved companion legislation (H.R.4250) in late July.

The legislation is supported by the American Academy of Dermatology and by consumer advocates and manufacturers, including the Public Access to SunScreens (PASS) Coalition.

“Congress’s commitment to addressing the skin cancer epidemic in the United States was clearly demonstrated in tonight’s Senate passage of the Sunscreen Innovation Act,” said Michael Werner, PASS Coalition Policy Adviser, in a statement after the vote. “We now call on the House and Senate to swiftly reconcile the differences in their bills and enact final legislation,” Werner said.

Chris Hansen, president of the American Cancer Society Cancer Action Network, said in a statement that if the bill became law, it would add more predictability to FDA reviews.

“The Senate took a critical step yesterday to fix a broken process at FDA for the review of new sunscreen ingredients that could potentially help more Americans prevent skin cancer,” he said.

Tim Turnham, executive director of the Melanoma Research Foundation, said in a statement that some of the ingredients under FDA review have been widely available in Europe, Asia, and Central and South America, in some cases for more than 15 years.

“Americans are limited in their choices for compounds that block UV radiation because of the long-standing bureaucratic gridlock at the FDA that prevents new agents from being approved,” said Mr. Turnham.

The House and Senate have to reconcile the two versions of the bill, and then the legislation will have to be approved again by both bodies before being sent to the White House for final approval.

[email protected]

On Twitter @aliciaault

The Senate unanimously approved a bill designed to speed up Food and Drug Administration approval of sunscreen ingredients, adding to the likelihood that the legislation will become law.

The full Senate took quick action on Sept. 17, the same day that the Health, Education, Labor & Pensions (HELP) Committee unanimously voted 12-0 in favor of the Sunscreen Innovation Act (S.2141).

©mark wragg/iStockphoto.com

“We have a number of ingredients for sunscreens that have languished at the FDA for years – as long as a decade,” Sen. Johnny Isakson (R-Ga.), a cosponsor of the bill, said at the HELP hearing. “This doesn’t mean Congress makes the decision,” he said, adding that, instead, the law does establish time frames for review.

The legislation “is also about holding the FDA accountable to timelines and reforming the process,” said Sen. Lamar Alexander (R-Tenn.).

The bill would require the FDA to make final decisions within a year on the backlog of ingredients under review, and within a year and a half on new applications. It also sets up more congressional oversight.

At a recent FDA advisory committee hearing on sunscreen ingredient safety, Dr. Theresa Michele, an FDA official, said that eight ingredients were awaiting approval through what was supposed to be an expedited process. These are the same ingredients that have been under review for 10 years or more. The agency has responded to manufacturers of five of the eight, telling them that so far, there’s not enough data to determine whether they can be marketed, said Dr. Michele.

The Senate joins the House in calling on the FDA to move those approvals along. The full House approved companion legislation (H.R.4250) in late July.

The legislation is supported by the American Academy of Dermatology and by consumer advocates and manufacturers, including the Public Access to SunScreens (PASS) Coalition.

“Congress’s commitment to addressing the skin cancer epidemic in the United States was clearly demonstrated in tonight’s Senate passage of the Sunscreen Innovation Act,” said Michael Werner, PASS Coalition Policy Adviser, in a statement after the vote. “We now call on the House and Senate to swiftly reconcile the differences in their bills and enact final legislation,” Werner said.

Chris Hansen, president of the American Cancer Society Cancer Action Network, said in a statement that if the bill became law, it would add more predictability to FDA reviews.

“The Senate took a critical step yesterday to fix a broken process at FDA for the review of new sunscreen ingredients that could potentially help more Americans prevent skin cancer,” he said.

Tim Turnham, executive director of the Melanoma Research Foundation, said in a statement that some of the ingredients under FDA review have been widely available in Europe, Asia, and Central and South America, in some cases for more than 15 years.

“Americans are limited in their choices for compounds that block UV radiation because of the long-standing bureaucratic gridlock at the FDA that prevents new agents from being approved,” said Mr. Turnham.

The House and Senate have to reconcile the two versions of the bill, and then the legislation will have to be approved again by both bodies before being sent to the White House for final approval.

[email protected]

On Twitter @aliciaault

WASHINGTON – When two Ebola-infected patients were airlifted to Emory University Hospital in Atlanta in early August, dedicated staffers were prepared to care for the patients. But those staffers had to act quickly to deal with unanticipated challenges that came from regulators and some of the hospital’s contractors, who initially refused to dispose of the mountains of medical waste generated by the patients and the clinicians who cared for them, according to Dr. Aneesh Mehta.

Dr. Mehta, associate chief of Emory’s infectious disease service, was one of the primary attending physicians for Nancy Writebol and Dr. Kent Brantly, who recovered from their Ebola infections and were discharged from Emory’s isolation unit on Aug. 19 and Aug. 21.

The lessons learned from caring for these initial patients are now being applied to Emory’s latest Ebola-infected patient, who arrived at the facility from West Africa on Sept. 9, said Dr. Mehta, who spoke on the logistics, successes, and challenges of caring for Ebola-infected patients at Emory at the annual Interscience Conference on Antimicrobial Agents and Chemotherapy.

The local civil authorities asked that the Emory unit not introduce any untreated patient waste into the municipal waste stream. The staff disinfected with bleach or detergents all of the patients’ liquid wastes for more than 5 minutes before flushing them down the toilet.

And there was plenty of other potentially hazardous and infectious material that had to be addressed. At the peak of the patients’ illness, up to 40 bags a day of medical waste were being produced, said Dr. Mehta.

Initially, the hospital’s waste management contractor refused to pick up the medical waste for several days. To manage the situation temporarily, the Emory staff "went to Home Depot and bought up every large trash can and sealed canister that we could get," said Dr. Mehta. The filled cans were kept in a containment room until the hospital completed negotiations with the contractor to pick up the waste.

Even the commercial courier company that the hospital had always used to transport infectious and potentially lethal samples to the labs at the Centers for Disease Control and Prevention "suddenly said ‘no,’ " Dr. Mehta said. "If the label said ‘CDC’ on it, they refused to touch it."

Even some Emory staffers who had previously handled and packed dangerous samples for shipping refused to come to the isolation unit’s lab, said Dr. Mehta. The hospital’s safety officers had to train some of the dedicated Ebola staff to pack samples appropriately for shipping.

But even as the hospital dealt with these issues and the growing media circus, the Ebola staff stayed focused, Dr. Mehta said. "It was very chaotic outside, but very calm in the hospital and with our team."

The hospital spent a lot of time on communications – with the public, with staff, and with other patients in the hospital and their families. Emory held twice-daily town halls with staff to give information and answer questions, and also sent regular e-mail updates about the Ebola patients. Every inpatient and new admission was given a letter explaining why Emory had taken the patients, and physician and administrative leaders rounded throughout the hospital to answer questions.

Emory already had a "serious communicable diseases" unit, which it created in 2002 as a place to receive any CDC workers who might be exposed to pathogens on the job. The six-room unit has a patient room on each end, with a private bathroom in each, a staff dressing area, and an anteroom that was used as a staging area for nurses.

After the decision to accept the Ebola-infected patients, a new point-of-care lab facility was built in less than 72 hours in an adjoining office space, said Dr. Mehta. Having this kind of dedicated unit was not absolutely necessary, but it allowed the staff to more conveniently perform chemistry, hematology, blood gas, urinalysis, coagulation, and malaria tests and get immediate results. Having a dedicated lab space also limited the exposure fears of staff elsewhere in the hospital.

To prevent transmission of the virus, the unit followed the CDC’s recommendations, which included keeping a detailed log of anyone who entered and exited a patient room, using disposable equipment whenever possible, and using personal protective equipment that included gloves, fluid-resistant or impermeable gowns, and goggles or face shields. Initially, the Emory caregivers had leg and foot coverings because the patients had vomited blood before arriving, and one of the patients had fulminant diarrhea, up to four liters per day, said Dr. Mehta.

Because the disease can be transmitted from protective gear, all staff had a refresher course on appropriate use of the gear, everyone was observed by another team member when putting on or taking off the gear, and reminders about appropriate use of the gear were placed on the walls of the unit and in the changing area.

The clinical care team met every day to review plans and protocols and to answer staff questions. Twice daily, all personnel had to enter into an online registry their body temperatures and any symptoms. Dr. Mehta told the ICAAC audience that he had just completed his 21-day observation period.

The patient care itself was far from clear cut, as there is no proven treatment and Emory officials initially were not clear on the availability of any experimental therapy, said Dr. Mehta. The CDC helped to monitor the patients’ viral load, and both patients had marked electrolyte imbalances, including hypokalemia, hypocalcemia, and hyponatremia, as well as severe nutritional deficiencies. Both required significant potassium replacement.

Both patients received a three-dose course of Zmapp, which consists of three monoclonal antibodies and is under development by Mapp Biopharmaceutical. While in Africa, Dr. Brantly also had received a transfusion of plasma from a patient who was recovering from the Ebola virus.

High-level, one-on-one nursing care also was noted as a significant factor in patient recovery, said Dr. Mehta.

"It’s hard to derive a lot of meaningful data from the care of those two patients," Dr. Mehta said. It’s not clear yet which of these factors – Zmapp, the transfusion, the supportive care, or the combination – was responsible for the patients’ recovery.

Emory will publish its experiences in Ebola care, but "the real front line is in West Africa," Dr. Mehta added.

[email protected]

On Twitter @aliciaault

WASHINGTON – When two Ebola-infected patients were airlifted to Emory University Hospital in Atlanta in early August, dedicated staffers were prepared to care for the patients. But those staffers had to act quickly to deal with unanticipated challenges that came from regulators and some of the hospital’s contractors, who initially refused to dispose of the mountains of medical waste generated by the patients and the clinicians who cared for them, according to Dr. Aneesh Mehta.

Dr. Mehta, associate chief of Emory’s infectious disease service, was one of the primary attending physicians for Nancy Writebol and Dr. Kent Brantly, who recovered from their Ebola infections and were discharged from Emory’s isolation unit on Aug. 19 and Aug. 21.

The lessons learned from caring for these initial patients are now being applied to Emory’s latest Ebola-infected patient, who arrived at the facility from West Africa on Sept. 9, said Dr. Mehta, who spoke on the logistics, successes, and challenges of caring for Ebola-infected patients at Emory at the annual Interscience Conference on Antimicrobial Agents and Chemotherapy.

The local civil authorities asked that the Emory unit not introduce any untreated patient waste into the municipal waste stream. The staff disinfected with bleach or detergents all of the patients’ liquid wastes for more than 5 minutes before flushing them down the toilet.

And there was plenty of other potentially hazardous and infectious material that had to be addressed. At the peak of the patients’ illness, up to 40 bags a day of medical waste were being produced, said Dr. Mehta.

Initially, the hospital’s waste management contractor refused to pick up the medical waste for several days. To manage the situation temporarily, the Emory staff "went to Home Depot and bought up every large trash can and sealed canister that we could get," said Dr. Mehta. The filled cans were kept in a containment room until the hospital completed negotiations with the contractor to pick up the waste.

Even the commercial courier company that the hospital had always used to transport infectious and potentially lethal samples to the labs at the Centers for Disease Control and Prevention "suddenly said ‘no,’ " Dr. Mehta said. "If the label said ‘CDC’ on it, they refused to touch it."

Even some Emory staffers who had previously handled and packed dangerous samples for shipping refused to come to the isolation unit’s lab, said Dr. Mehta. The hospital’s safety officers had to train some of the dedicated Ebola staff to pack samples appropriately for shipping.

But even as the hospital dealt with these issues and the growing media circus, the Ebola staff stayed focused, Dr. Mehta said. "It was very chaotic outside, but very calm in the hospital and with our team."

The hospital spent a lot of time on communications – with the public, with staff, and with other patients in the hospital and their families. Emory held twice-daily town halls with staff to give information and answer questions, and also sent regular e-mail updates about the Ebola patients. Every inpatient and new admission was given a letter explaining why Emory had taken the patients, and physician and administrative leaders rounded throughout the hospital to answer questions.

Emory already had a "serious communicable diseases" unit, which it created in 2002 as a place to receive any CDC workers who might be exposed to pathogens on the job. The six-room unit has a patient room on each end, with a private bathroom in each, a staff dressing area, and an anteroom that was used as a staging area for nurses.

After the decision to accept the Ebola-infected patients, a new point-of-care lab facility was built in less than 72 hours in an adjoining office space, said Dr. Mehta. Having this kind of dedicated unit was not absolutely necessary, but it allowed the staff to more conveniently perform chemistry, hematology, blood gas, urinalysis, coagulation, and malaria tests and get immediate results. Having a dedicated lab space also limited the exposure fears of staff elsewhere in the hospital.

To prevent transmission of the virus, the unit followed the CDC’s recommendations, which included keeping a detailed log of anyone who entered and exited a patient room, using disposable equipment whenever possible, and using personal protective equipment that included gloves, fluid-resistant or impermeable gowns, and goggles or face shields. Initially, the Emory caregivers had leg and foot coverings because the patients had vomited blood before arriving, and one of the patients had fulminant diarrhea, up to four liters per day, said Dr. Mehta.

Because the disease can be transmitted from protective gear, all staff had a refresher course on appropriate use of the gear, everyone was observed by another team member when putting on or taking off the gear, and reminders about appropriate use of the gear were placed on the walls of the unit and in the changing area.

The clinical care team met every day to review plans and protocols and to answer staff questions. Twice daily, all personnel had to enter into an online registry their body temperatures and any symptoms. Dr. Mehta told the ICAAC audience that he had just completed his 21-day observation period.

The patient care itself was far from clear cut, as there is no proven treatment and Emory officials initially were not clear on the availability of any experimental therapy, said Dr. Mehta. The CDC helped to monitor the patients’ viral load, and both patients had marked electrolyte imbalances, including hypokalemia, hypocalcemia, and hyponatremia, as well as severe nutritional deficiencies. Both required significant potassium replacement.

Both patients received a three-dose course of Zmapp, which consists of three monoclonal antibodies and is under development by Mapp Biopharmaceutical. While in Africa, Dr. Brantly also had received a transfusion of plasma from a patient who was recovering from the Ebola virus.

High-level, one-on-one nursing care also was noted as a significant factor in patient recovery, said Dr. Mehta.

"It’s hard to derive a lot of meaningful data from the care of those two patients," Dr. Mehta said. It’s not clear yet which of these factors – Zmapp, the transfusion, the supportive care, or the combination – was responsible for the patients’ recovery.

Emory will publish its experiences in Ebola care, but "the real front line is in West Africa," Dr. Mehta added.

[email protected]

On Twitter @aliciaault

WASHINGTON – When two Ebola-infected patients were airlifted to Emory University Hospital in Atlanta in early August, dedicated staffers were prepared to care for the patients. But those staffers had to act quickly to deal with unanticipated challenges that came from regulators and some of the hospital’s contractors, who initially refused to dispose of the mountains of medical waste generated by the patients and the clinicians who cared for them, according to Dr. Aneesh Mehta.

Dr. Mehta, associate chief of Emory’s infectious disease service, was one of the primary attending physicians for Nancy Writebol and Dr. Kent Brantly, who recovered from their Ebola infections and were discharged from Emory’s isolation unit on Aug. 19 and Aug. 21.

The lessons learned from caring for these initial patients are now being applied to Emory’s latest Ebola-infected patient, who arrived at the facility from West Africa on Sept. 9, said Dr. Mehta, who spoke on the logistics, successes, and challenges of caring for Ebola-infected patients at Emory at the annual Interscience Conference on Antimicrobial Agents and Chemotherapy.

The local civil authorities asked that the Emory unit not introduce any untreated patient waste into the municipal waste stream. The staff disinfected with bleach or detergents all of the patients’ liquid wastes for more than 5 minutes before flushing them down the toilet.

And there was plenty of other potentially hazardous and infectious material that had to be addressed. At the peak of the patients’ illness, up to 40 bags a day of medical waste were being produced, said Dr. Mehta.

Initially, the hospital’s waste management contractor refused to pick up the medical waste for several days. To manage the situation temporarily, the Emory staff "went to Home Depot and bought up every large trash can and sealed canister that we could get," said Dr. Mehta. The filled cans were kept in a containment room until the hospital completed negotiations with the contractor to pick up the waste.

Even the commercial courier company that the hospital had always used to transport infectious and potentially lethal samples to the labs at the Centers for Disease Control and Prevention "suddenly said ‘no,’ " Dr. Mehta said. "If the label said ‘CDC’ on it, they refused to touch it."

Even some Emory staffers who had previously handled and packed dangerous samples for shipping refused to come to the isolation unit’s lab, said Dr. Mehta. The hospital’s safety officers had to train some of the dedicated Ebola staff to pack samples appropriately for shipping.

But even as the hospital dealt with these issues and the growing media circus, the Ebola staff stayed focused, Dr. Mehta said. "It was very chaotic outside, but very calm in the hospital and with our team."

The hospital spent a lot of time on communications – with the public, with staff, and with other patients in the hospital and their families. Emory held twice-daily town halls with staff to give information and answer questions, and also sent regular e-mail updates about the Ebola patients. Every inpatient and new admission was given a letter explaining why Emory had taken the patients, and physician and administrative leaders rounded throughout the hospital to answer questions.

Emory already had a "serious communicable diseases" unit, which it created in 2002 as a place to receive any CDC workers who might be exposed to pathogens on the job. The six-room unit has a patient room on each end, with a private bathroom in each, a staff dressing area, and an anteroom that was used as a staging area for nurses.

After the decision to accept the Ebola-infected patients, a new point-of-care lab facility was built in less than 72 hours in an adjoining office space, said Dr. Mehta. Having this kind of dedicated unit was not absolutely necessary, but it allowed the staff to more conveniently perform chemistry, hematology, blood gas, urinalysis, coagulation, and malaria tests and get immediate results. Having a dedicated lab space also limited the exposure fears of staff elsewhere in the hospital.

To prevent transmission of the virus, the unit followed the CDC’s recommendations, which included keeping a detailed log of anyone who entered and exited a patient room, using disposable equipment whenever possible, and using personal protective equipment that included gloves, fluid-resistant or impermeable gowns, and goggles or face shields. Initially, the Emory caregivers had leg and foot coverings because the patients had vomited blood before arriving, and one of the patients had fulminant diarrhea, up to four liters per day, said Dr. Mehta.

Because the disease can be transmitted from protective gear, all staff had a refresher course on appropriate use of the gear, everyone was observed by another team member when putting on or taking off the gear, and reminders about appropriate use of the gear were placed on the walls of the unit and in the changing area.

The clinical care team met every day to review plans and protocols and to answer staff questions. Twice daily, all personnel had to enter into an online registry their body temperatures and any symptoms. Dr. Mehta told the ICAAC audience that he had just completed his 21-day observation period.

The patient care itself was far from clear cut, as there is no proven treatment and Emory officials initially were not clear on the availability of any experimental therapy, said Dr. Mehta. The CDC helped to monitor the patients’ viral load, and both patients had marked electrolyte imbalances, including hypokalemia, hypocalcemia, and hyponatremia, as well as severe nutritional deficiencies. Both required significant potassium replacement.

Both patients received a three-dose course of Zmapp, which consists of three monoclonal antibodies and is under development by Mapp Biopharmaceutical. While in Africa, Dr. Brantly also had received a transfusion of plasma from a patient who was recovering from the Ebola virus.

High-level, one-on-one nursing care also was noted as a significant factor in patient recovery, said Dr. Mehta.

"It’s hard to derive a lot of meaningful data from the care of those two patients," Dr. Mehta said. It’s not clear yet which of these factors – Zmapp, the transfusion, the supportive care, or the combination – was responsible for the patients’ recovery.

Emory will publish its experiences in Ebola care, but "the real front line is in West Africa," Dr. Mehta added.

[email protected]

On Twitter @aliciaault

A diabetes drug is now poised to become a weight loss therapeutic, even in patients without the condition, after a panel of Food and Drug Administration advisers recommended approval of liraglutide for weight management.

The glucagon-like peptide-1 receptor agonist, to be marketed as Saxenda by Novo Nordisk, has been on the market since 2010 for treatment of type 2 diabetes under the brand name Victoza. The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 14-1 that Saxenda is safe and effective and should be approved as an adjunct to a reduced-calorie diet and increased physical activity for chronic weight management in adults with an initial body mass index of at least 30 kg/m², or 27 kg/m² with at least one weight-related comorbidity.

Liraglutide is marketed at a lower dose for diabetes – a 1.8-mg, once-daily dose that’s delivered subcutaneously through a pen-like device – than that proposed for weight management, which would be a 3-mg dose delivered the same way.

The vote to approve came despite the fact that even the lower dose of liraglutide currently carries a boxed warning about the potential for medullary thyroid (C-cell) tumors, and includes warnings and precautions about the potential for pancreatitis, serious hypoglycemia, renal impairment, and hypersensitivity. There also have been significant adverse events in clinical trials and reported to the FDA since the drug has been on the market, including nausea and vomiting and increases in heart rate.

In the phase III studies of Saxenda – which included more than 3,000 patients who took the drug – there were 48 cancers detected, including 7 C-cell thyroid tumors and 14 breast neoplasms.

The panel discussed at length whether the 3-mg dose may trigger more tumors or spur a greater incidence of other side effects, especially since the anticipated wider use may expose even more patients to the drug’s downsides.

Fourteen of the committee members put aside their concerns and voted for approval, but it was not cut-and-dried for all of them.

“For me it was more of a difficult decision rendering an opinion on the risk-benefit ratio,” said Dr. Kenneth Burman, acting chairperson of the committee.

He said he was not completely convinced that the risk for medullary thyroid tumors was minimal. Dr. Burman, director of the endocrine section at the Washington Hospital Center, also said that he was concerned that there was no real guidance on how long a patient should take liraglutide for weight management, especially if the effect plateaued as it did in trials, or was reversed.

Medullary tumors are rare in the general population, and though rare in the phase III clinical trials in 3-mg liraglutide, the data still suggested the possibility of an increased risk, said FDA reviewers. There was also a small excess risk of breast cancers in those trials, which suggested a potential signal, said Christian Hampp, Ph.D., a reviewer from the epidemiology division at the FDA’s Center for Drug Evaluation and Research.

However, Dr. Julie Golden, a medical officer in the FDA’s division of metabolism and endocrinology products, noted that there’s little evidence to suggest that liraglutide has a role in promoting breast cancer.

Panelist Barbara Hansen, Ph.D., agreed that there seemed to be little biological plausibility. “I’m happy with the amount of risk that has been identified,” said Dr. Hansen, professor of internal medicine, University of South Florida College of Medicine, Tampa. “I don’t see a signal there,” she said.

The majority of the committee agreed that liraglutide was effective for weight loss and met the FDA criteria, which was a 5% difference in mean weight loss between the drug and placebo; or at least 35% of patients on therapy losing at least 5% of their baseline weight and double the number on placebo who lost that much; and, improvement in cardiometabolic parameters.

More than 5,000 patients were enrolled in the four phase III trials; over 3,000 took the 3-mg dose of liraglutide. All patients were required to have a stable body weight and to have failed previous dietary intervention. Once in the study, they were told to reduce caloric intake by 500 kcal a day and to maintain or add at least 150 minutes week of physical activity.

The mean age was 47 years, and most participants were women. The mean BMI was 38 kg/m².

In the largest trial, 3,731 patients were studied for 56 weeks, and were stratified by the presence or absence of prediabetes. Of patients taking liraglutide, 64% lost at least 5% of their body weight and 33% lost at least 10%, compared with 27% and 10% of the placebo patients, respectively. There were no significant differences in these endpoints between patients with prediabetes and those without.

Results were similar across the three other phase III studies, though not as pronounced. In addition, Novo Nordisk said that weight loss began to plateau for many patients at 34 weeks. Although liraglutide also helped reduce hypertension, blood lipids, blood glucose, and waist circumference, there were still signs that it might have some adverse cardiovascular effects.

Many panel members said they’d like to see more specific study of cardiovascular outcomes in patients taking a 3-mg dose.

Earlier this week, the FDA approved a combination drug, Contrave (naltrexone and bupropion) for weight management. If approved, Saxenda would be the fourth weight management drug on the U.S. market, after Contrave, the phentermine/topiramate combo Qsymia, and lorcaserin (Belviq).

The FDA is not bound by its advisory panels’ advice, but usually follows their recommendations.

[email protected]

On Twitter @aliciaault

A diabetes drug is now poised to become a weight loss therapeutic, even in patients without the condition, after a panel of Food and Drug Administration advisers recommended approval of liraglutide for weight management.

The glucagon-like peptide-1 receptor agonist, to be marketed as Saxenda by Novo Nordisk, has been on the market since 2010 for treatment of type 2 diabetes under the brand name Victoza. The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 14-1 that Saxenda is safe and effective and should be approved as an adjunct to a reduced-calorie diet and increased physical activity for chronic weight management in adults with an initial body mass index of at least 30 kg/m², or 27 kg/m² with at least one weight-related comorbidity.

Liraglutide is marketed at a lower dose for diabetes – a 1.8-mg, once-daily dose that’s delivered subcutaneously through a pen-like device – than that proposed for weight management, which would be a 3-mg dose delivered the same way.

The vote to approve came despite the fact that even the lower dose of liraglutide currently carries a boxed warning about the potential for medullary thyroid (C-cell) tumors, and includes warnings and precautions about the potential for pancreatitis, serious hypoglycemia, renal impairment, and hypersensitivity. There also have been significant adverse events in clinical trials and reported to the FDA since the drug has been on the market, including nausea and vomiting and increases in heart rate.

In the phase III studies of Saxenda – which included more than 3,000 patients who took the drug – there were 48 cancers detected, including 7 C-cell thyroid tumors and 14 breast neoplasms.

The panel discussed at length whether the 3-mg dose may trigger more tumors or spur a greater incidence of other side effects, especially since the anticipated wider use may expose even more patients to the drug’s downsides.

Fourteen of the committee members put aside their concerns and voted for approval, but it was not cut-and-dried for all of them.

“For me it was more of a difficult decision rendering an opinion on the risk-benefit ratio,” said Dr. Kenneth Burman, acting chairperson of the committee.

He said he was not completely convinced that the risk for medullary thyroid tumors was minimal. Dr. Burman, director of the endocrine section at the Washington Hospital Center, also said that he was concerned that there was no real guidance on how long a patient should take liraglutide for weight management, especially if the effect plateaued as it did in trials, or was reversed.

Medullary tumors are rare in the general population, and though rare in the phase III clinical trials in 3-mg liraglutide, the data still suggested the possibility of an increased risk, said FDA reviewers. There was also a small excess risk of breast cancers in those trials, which suggested a potential signal, said Christian Hampp, Ph.D., a reviewer from the epidemiology division at the FDA’s Center for Drug Evaluation and Research.

However, Dr. Julie Golden, a medical officer in the FDA’s division of metabolism and endocrinology products, noted that there’s little evidence to suggest that liraglutide has a role in promoting breast cancer.

Panelist Barbara Hansen, Ph.D., agreed that there seemed to be little biological plausibility. “I’m happy with the amount of risk that has been identified,” said Dr. Hansen, professor of internal medicine, University of South Florida College of Medicine, Tampa. “I don’t see a signal there,” she said.

The majority of the committee agreed that liraglutide was effective for weight loss and met the FDA criteria, which was a 5% difference in mean weight loss between the drug and placebo; or at least 35% of patients on therapy losing at least 5% of their baseline weight and double the number on placebo who lost that much; and, improvement in cardiometabolic parameters.

More than 5,000 patients were enrolled in the four phase III trials; over 3,000 took the 3-mg dose of liraglutide. All patients were required to have a stable body weight and to have failed previous dietary intervention. Once in the study, they were told to reduce caloric intake by 500 kcal a day and to maintain or add at least 150 minutes week of physical activity.

The mean age was 47 years, and most participants were women. The mean BMI was 38 kg/m².

In the largest trial, 3,731 patients were studied for 56 weeks, and were stratified by the presence or absence of prediabetes. Of patients taking liraglutide, 64% lost at least 5% of their body weight and 33% lost at least 10%, compared with 27% and 10% of the placebo patients, respectively. There were no significant differences in these endpoints between patients with prediabetes and those without.

Results were similar across the three other phase III studies, though not as pronounced. In addition, Novo Nordisk said that weight loss began to plateau for many patients at 34 weeks. Although liraglutide also helped reduce hypertension, blood lipids, blood glucose, and waist circumference, there were still signs that it might have some adverse cardiovascular effects.

Many panel members said they’d like to see more specific study of cardiovascular outcomes in patients taking a 3-mg dose.

Earlier this week, the FDA approved a combination drug, Contrave (naltrexone and bupropion) for weight management. If approved, Saxenda would be the fourth weight management drug on the U.S. market, after Contrave, the phentermine/topiramate combo Qsymia, and lorcaserin (Belviq).

The FDA is not bound by its advisory panels’ advice, but usually follows their recommendations.

[email protected]

On Twitter @aliciaault

A diabetes drug is now poised to become a weight loss therapeutic, even in patients without the condition, after a panel of Food and Drug Administration advisers recommended approval of liraglutide for weight management.

The glucagon-like peptide-1 receptor agonist, to be marketed as Saxenda by Novo Nordisk, has been on the market since 2010 for treatment of type 2 diabetes under the brand name Victoza. The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 14-1 that Saxenda is safe and effective and should be approved as an adjunct to a reduced-calorie diet and increased physical activity for chronic weight management in adults with an initial body mass index of at least 30 kg/m², or 27 kg/m² with at least one weight-related comorbidity.

Liraglutide is marketed at a lower dose for diabetes – a 1.8-mg, once-daily dose that’s delivered subcutaneously through a pen-like device – than that proposed for weight management, which would be a 3-mg dose delivered the same way.

The vote to approve came despite the fact that even the lower dose of liraglutide currently carries a boxed warning about the potential for medullary thyroid (C-cell) tumors, and includes warnings and precautions about the potential for pancreatitis, serious hypoglycemia, renal impairment, and hypersensitivity. There also have been significant adverse events in clinical trials and reported to the FDA since the drug has been on the market, including nausea and vomiting and increases in heart rate.

In the phase III studies of Saxenda – which included more than 3,000 patients who took the drug – there were 48 cancers detected, including 7 C-cell thyroid tumors and 14 breast neoplasms.

The panel discussed at length whether the 3-mg dose may trigger more tumors or spur a greater incidence of other side effects, especially since the anticipated wider use may expose even more patients to the drug’s downsides.

Fourteen of the committee members put aside their concerns and voted for approval, but it was not cut-and-dried for all of them.

“For me it was more of a difficult decision rendering an opinion on the risk-benefit ratio,” said Dr. Kenneth Burman, acting chairperson of the committee.

He said he was not completely convinced that the risk for medullary thyroid tumors was minimal. Dr. Burman, director of the endocrine section at the Washington Hospital Center, also said that he was concerned that there was no real guidance on how long a patient should take liraglutide for weight management, especially if the effect plateaued as it did in trials, or was reversed.

Medullary tumors are rare in the general population, and though rare in the phase III clinical trials in 3-mg liraglutide, the data still suggested the possibility of an increased risk, said FDA reviewers. There was also a small excess risk of breast cancers in those trials, which suggested a potential signal, said Christian Hampp, Ph.D., a reviewer from the epidemiology division at the FDA’s Center for Drug Evaluation and Research.

However, Dr. Julie Golden, a medical officer in the FDA’s division of metabolism and endocrinology products, noted that there’s little evidence to suggest that liraglutide has a role in promoting breast cancer.

Panelist Barbara Hansen, Ph.D., agreed that there seemed to be little biological plausibility. “I’m happy with the amount of risk that has been identified,” said Dr. Hansen, professor of internal medicine, University of South Florida College of Medicine, Tampa. “I don’t see a signal there,” she said.

The majority of the committee agreed that liraglutide was effective for weight loss and met the FDA criteria, which was a 5% difference in mean weight loss between the drug and placebo; or at least 35% of patients on therapy losing at least 5% of their baseline weight and double the number on placebo who lost that much; and, improvement in cardiometabolic parameters.

More than 5,000 patients were enrolled in the four phase III trials; over 3,000 took the 3-mg dose of liraglutide. All patients were required to have a stable body weight and to have failed previous dietary intervention. Once in the study, they were told to reduce caloric intake by 500 kcal a day and to maintain or add at least 150 minutes week of physical activity.

The mean age was 47 years, and most participants were women. The mean BMI was 38 kg/m².

In the largest trial, 3,731 patients were studied for 56 weeks, and were stratified by the presence or absence of prediabetes. Of patients taking liraglutide, 64% lost at least 5% of their body weight and 33% lost at least 10%, compared with 27% and 10% of the placebo patients, respectively. There were no significant differences in these endpoints between patients with prediabetes and those without.

Results were similar across the three other phase III studies, though not as pronounced. In addition, Novo Nordisk said that weight loss began to plateau for many patients at 34 weeks. Although liraglutide also helped reduce hypertension, blood lipids, blood glucose, and waist circumference, there were still signs that it might have some adverse cardiovascular effects.

Many panel members said they’d like to see more specific study of cardiovascular outcomes in patients taking a 3-mg dose.

Earlier this week, the FDA approved a combination drug, Contrave (naltrexone and bupropion) for weight management. If approved, Saxenda would be the fourth weight management drug on the U.S. market, after Contrave, the phentermine/topiramate combo Qsymia, and lorcaserin (Belviq).

The FDA is not bound by its advisory panels’ advice, but usually follows their recommendations.

[email protected]

On Twitter @aliciaault

WASHINGTON – A rationally designed microbiome-based pill successfully eradicated recurrent Clostridium difficile infection in 29 of 30 patients in an early study presented at the annual Interscience Conference on Antimicrobial Agents and Chemotherapy.

Preliminary data were presented at the 2014 James W. Freston Conference sponsored by the American Gastroenterological Association in August.

The patients in the phase I/II study were divided into  two separate 15-patient cohorts and were given two different dose levels of the therapy, SER-109. The drug contains spores from gram-positive bacteria that are taken from stool and then purified to kill the vegetative bacteria, said David Cook, Ph.D., executive vice president and chief scientific officer of Cambridge, Mass.–based Seres Health.

The therapy appears to work by restoring the gut flora to a normal balance after having been disrupted by antibiotic treatment.

It is not the first attempt to encapsulate a fecal transplant; Dr. Thomas Louie of the University of Calgary (Alberta), presented data on his very effective formulation against C. difficile at the ID Week annual meeting in 2013.

In the current study, the first cohort received a mean dose of 1.5 × 10⁹ spores and the second cohort received a dose of 1 × 10⁸ spores. In the early studies, the number of pills given to achieve that dose varied. The aim is to contain the dose in a few tablets for a commercial product, Dr. Cook said.

All doses were given on 1 or 2 days at the study’s start; there was no need for additional doses.

Patients were as young as 22 years old and as old as 88 years, and all had three or more laboratory-confirmed C. difficile infections over the previous year. They had to have a life expectancy of 3 months or longer and be able to give informed consent. They were excluded if they had any immunosuppression, a history of irritable bowel disease, total colectomy, cirrhosis, a need for antibiotics within 6 weeks of baseline, prior fecal transplant, or if they were in intensive care. There were 10 women and five men in each cohort.

After patients stopped taking antibiotics, there was a washout period after which they took SER-109. Stool was collected on day four and at 1, 2, 4, 8, and 24 weeks. Efficacy was assessed at the 8-week time point.

In the first group, 13 of the 15 patients achieved the protocol-defined endpoint: absence of C. difficile over the 8 weeks.

The two patients who failed had self-limited, transient diarrhea with a positive C. difficile test, but neither required antibiotics and both recovered within 24 hours, so they were considered to not have recurrent C. difficile, said Dr. Cook. In the second cohort, 14 of the 15 patients were free of C. difficile at 8 weeks. The patient who failed had diarrhea and a positive C. difficile test, and the diarrhea did not resolve on its own. She required antibiotics to achieve remission.

There were no serious adverse events in the study.

The company hopes to eventually conduct a phase III study and seek Food and Drug Administration approval, but it is too early to say when that might occur, said Dr. Cook.

[email protected]

WASHINGTON – A rationally designed microbiome-based pill successfully eradicated recurrent Clostridium difficile infection in 29 of 30 patients in an early study presented at the annual Interscience Conference on Antimicrobial Agents and Chemotherapy.

Preliminary data were presented at the 2014 James W. Freston Conference sponsored by the American Gastroenterological Association in August.

The patients in the phase I/II study were divided into  two separate 15-patient cohorts and were given two different dose levels of the therapy, SER-109. The drug contains spores from gram-positive bacteria that are taken from stool and then purified to kill the vegetative bacteria, said David Cook, Ph.D., executive vice president and chief scientific officer of Cambridge, Mass.–based Seres Health.

The therapy appears to work by restoring the gut flora to a normal balance after having been disrupted by antibiotic treatment.

It is not the first attempt to encapsulate a fecal transplant; Dr. Thomas Louie of the University of Calgary (Alberta), presented data on his very effective formulation against C. difficile at the ID Week annual meeting in 2013.

In the current study, the first cohort received a mean dose of 1.5 × 10⁹ spores and the second cohort received a dose of 1 × 10⁸ spores. In the early studies, the number of pills given to achieve that dose varied. The aim is to contain the dose in a few tablets for a commercial product, Dr. Cook said.

All doses were given on 1 or 2 days at the study’s start; there was no need for additional doses.

Patients were as young as 22 years old and as old as 88 years, and all had three or more laboratory-confirmed C. difficile infections over the previous year. They had to have a life expectancy of 3 months or longer and be able to give informed consent. They were excluded if they had any immunosuppression, a history of irritable bowel disease, total colectomy, cirrhosis, a need for antibiotics within 6 weeks of baseline, prior fecal transplant, or if they were in intensive care. There were 10 women and five men in each cohort.

After patients stopped taking antibiotics, there was a washout period after which they took SER-109. Stool was collected on day four and at 1, 2, 4, 8, and 24 weeks. Efficacy was assessed at the 8-week time point.

In the first group, 13 of the 15 patients achieved the protocol-defined endpoint: absence of C. difficile over the 8 weeks.

The two patients who failed had self-limited, transient diarrhea with a positive C. difficile test, but neither required antibiotics and both recovered within 24 hours, so they were considered to not have recurrent C. difficile, said Dr. Cook. In the second cohort, 14 of the 15 patients were free of C. difficile at 8 weeks. The patient who failed had diarrhea and a positive C. difficile test, and the diarrhea did not resolve on its own. She required antibiotics to achieve remission.

There were no serious adverse events in the study.

The company hopes to eventually conduct a phase III study and seek Food and Drug Administration approval, but it is too early to say when that might occur, said Dr. Cook.

[email protected]

WASHINGTON – A rationally designed microbiome-based pill successfully eradicated recurrent Clostridium difficile infection in 29 of 30 patients in an early study presented at the annual Interscience Conference on Antimicrobial Agents and Chemotherapy.

Preliminary data were presented at the 2014 James W. Freston Conference sponsored by the American Gastroenterological Association in August.

The patients in the phase I/II study were divided into  two separate 15-patient cohorts and were given two different dose levels of the therapy, SER-109. The drug contains spores from gram-positive bacteria that are taken from stool and then purified to kill the vegetative bacteria, said David Cook, Ph.D., executive vice president and chief scientific officer of Cambridge, Mass.–based Seres Health.

The therapy appears to work by restoring the gut flora to a normal balance after having been disrupted by antibiotic treatment.

It is not the first attempt to encapsulate a fecal transplant; Dr. Thomas Louie of the University of Calgary (Alberta), presented data on his very effective formulation against C. difficile at the ID Week annual meeting in 2013.

In the current study, the first cohort received a mean dose of 1.5 × 10⁹ spores and the second cohort received a dose of 1 × 10⁸ spores. In the early studies, the number of pills given to achieve that dose varied. The aim is to contain the dose in a few tablets for a commercial product, Dr. Cook said.

All doses were given on 1 or 2 days at the study’s start; there was no need for additional doses.

Patients were as young as 22 years old and as old as 88 years, and all had three or more laboratory-confirmed C. difficile infections over the previous year. They had to have a life expectancy of 3 months or longer and be able to give informed consent. They were excluded if they had any immunosuppression, a history of irritable bowel disease, total colectomy, cirrhosis, a need for antibiotics within 6 weeks of baseline, prior fecal transplant, or if they were in intensive care. There were 10 women and five men in each cohort.

After patients stopped taking antibiotics, there was a washout period after which they took SER-109. Stool was collected on day four and at 1, 2, 4, 8, and 24 weeks. Efficacy was assessed at the 8-week time point.

In the first group, 13 of the 15 patients achieved the protocol-defined endpoint: absence of C. difficile over the 8 weeks.

The two patients who failed had self-limited, transient diarrhea with a positive C. difficile test, but neither required antibiotics and both recovered within 24 hours, so they were considered to not have recurrent C. difficile, said Dr. Cook. In the second cohort, 14 of the 15 patients were free of C. difficile at 8 weeks. The patient who failed had diarrhea and a positive C. difficile test, and the diarrhea did not resolve on its own. She required antibiotics to achieve remission.

There were no serious adverse events in the study.

The company hopes to eventually conduct a phase III study and seek Food and Drug Administration approval, but it is too early to say when that might occur, said Dr. Cook.

[email protected]

WASHINGTON – A rationally designed microbiome-based pill successfully eradicated recurrent Clostridium difficile infection in 29 of 30 patients in an early study presented at the annual Interscience Conference on Antimicrobial Agents and Chemotherapy.

Preliminary data were presented at the 2014 James W. Freston Conference sponsored by the American Gastroenterological Association in August.

CDC/D. Holdeman

The patients in the phase I/II study were divided into two separate 15-patient cohorts and were given two different dose levels of the therapy, SER-109. The drug contains spores from gram-positive bacteria that are taken from stool and then purified to kill the vegetative bacteria, said David Cook, Ph.D., executive vice president and chief scientific officer of Cambridge, Mass.–based Seres Health.

The therapy appears to work by restoring the gut flora to a normal balance after having been disrupted by antibiotic treatment.

It is not the first attempt to encapsulate a fecal transplant; Dr. Thomas Louie of the University of Calgary (Alberta), presented data on his very effective formulation against C. difficile at the ID Week annual meeting in 2013.

In the current study, the first cohort received a mean dose of 1.5 × 10⁹ spores and the second cohort received a dose of 1 × 10⁸ spores. In the early studies, the number of pills given to achieve that dose varied. The aim is to contain the dose in a few tablets for a commercial product, Dr. Cook said.

All doses were given on 1 or 2 days at the study’s start; there was no need for additional doses.

Patients were as young as 22 years old and as old as 88 years, and all had three or more laboratory-confirmed C. difficile infections over the previous year. They had to have a life expectancy of 3 months or longer and be able to give informed consent. They were excluded if they had any immunosuppression, a history of irritable bowel disease, total colectomy, cirrhosis, a need for antibiotics within 6 weeks of baseline, prior fecal transplant, or if they were in intensive care. There were 10 women and five men in each cohort.

After patients stopped taking antibiotics, there was a washout period after which they took SER-109. Stool was collected on day four and at 1, 2, 4, 8, and 24 weeks. Efficacy was assessed at the 8-week time point.

In the first group, 13 of the 15 patients achieved the protocol-defined endpoint: absence of C. difficile over the 8 weeks.

The two patients who failed had self-limited, transient diarrhea with a positive C. difficile test, but neither required antibiotics and both recovered within 24 hours, so they were considered to not have recurrent C. difficile, said Dr. Cook. In the second cohort, 14 of the 15 patients were free of C. difficile at 8 weeks. The patient who failed had diarrhea and a positive C. difficile test, and the diarrhea did not resolve on its own. She required antibiotics to achieve remission.

There were no serious adverse events in the study.

The company hopes to eventually conduct a phase III study and seek Food and Drug Administration approval, but it is too early to say when that might occur, said Dr. Cook.

[email protected]

On Twitter @aliciaault

This story was updated on October 14.

WASHINGTON – A rationally designed microbiome-based pill successfully eradicated recurrent Clostridium difficile infection in 29 of 30 patients in an early study presented at the annual Interscience Conference on Antimicrobial Agents and Chemotherapy.

Preliminary data were presented at the 2014 James W. Freston Conference sponsored by the American Gastroenterological Association in August.

CDC/D. Holdeman

The patients in the phase I/II study were divided into two separate 15-patient cohorts and were given two different dose levels of the therapy, SER-109. The drug contains spores from gram-positive bacteria that are taken from stool and then purified to kill the vegetative bacteria, said David Cook, Ph.D., executive vice president and chief scientific officer of Cambridge, Mass.–based Seres Health.

The therapy appears to work by restoring the gut flora to a normal balance after having been disrupted by antibiotic treatment.

It is not the first attempt to encapsulate a fecal transplant; Dr. Thomas Louie of the University of Calgary (Alberta), presented data on his very effective formulation against C. difficile at the ID Week annual meeting in 2013.

In the current study, the first cohort received a mean dose of 1.5 × 10⁹ spores and the second cohort received a dose of 1 × 10⁸ spores. In the early studies, the number of pills given to achieve that dose varied. The aim is to contain the dose in a few tablets for a commercial product, Dr. Cook said.

All doses were given on 1 or 2 days at the study’s start; there was no need for additional doses.

Patients were as young as 22 years old and as old as 88 years, and all had three or more laboratory-confirmed C. difficile infections over the previous year. They had to have a life expectancy of 3 months or longer and be able to give informed consent. They were excluded if they had any immunosuppression, a history of irritable bowel disease, total colectomy, cirrhosis, a need for antibiotics within 6 weeks of baseline, prior fecal transplant, or if they were in intensive care. There were 10 women and five men in each cohort.

After patients stopped taking antibiotics, there was a washout period after which they took SER-109. Stool was collected on day four and at 1, 2, 4, 8, and 24 weeks. Efficacy was assessed at the 8-week time point.

In the first group, 13 of the 15 patients achieved the protocol-defined endpoint: absence of C. difficile over the 8 weeks.

The two patients who failed had self-limited, transient diarrhea with a positive C. difficile test, but neither required antibiotics and both recovered within 24 hours, so they were considered to not have recurrent C. difficile, said Dr. Cook. In the second cohort, 14 of the 15 patients were free of C. difficile at 8 weeks. The patient who failed had diarrhea and a positive C. difficile test, and the diarrhea did not resolve on its own. She required antibiotics to achieve remission.

There were no serious adverse events in the study.

The company hopes to eventually conduct a phase III study and seek Food and Drug Administration approval, but it is too early to say when that might occur, said Dr. Cook.

[email protected]

On Twitter @aliciaault

This story was updated on October 14.

WASHINGTON – A rationally designed microbiome-based pill successfully eradicated recurrent Clostridium difficile infection in 29 of 30 patients in an early study presented at the annual Interscience Conference on Antimicrobial Agents and Chemotherapy.

Preliminary data were presented at the 2014 James W. Freston Conference sponsored by the American Gastroenterological Association in August.

CDC/D. Holdeman

The patients in the phase I/II study were divided into two separate 15-patient cohorts and were given two different dose levels of the therapy, SER-109. The drug contains spores from gram-positive bacteria that are taken from stool and then purified to kill the vegetative bacteria, said David Cook, Ph.D., executive vice president and chief scientific officer of Cambridge, Mass.–based Seres Health.

The therapy appears to work by restoring the gut flora to a normal balance after having been disrupted by antibiotic treatment.

It is not the first attempt to encapsulate a fecal transplant; Dr. Thomas Louie of the University of Calgary (Alberta), presented data on his very effective formulation against C. difficile at the ID Week annual meeting in 2013.

In the current study, the first cohort received a mean dose of 1.5 × 10⁹ spores and the second cohort received a dose of 1 × 10⁸ spores. In the early studies, the number of pills given to achieve that dose varied. The aim is to contain the dose in a few tablets for a commercial product, Dr. Cook said.

All doses were given on 1 or 2 days at the study’s start; there was no need for additional doses.

Patients were as young as 22 years old and as old as 88 years, and all had three or more laboratory-confirmed C. difficile infections over the previous year. They had to have a life expectancy of 3 months or longer and be able to give informed consent. They were excluded if they had any immunosuppression, a history of irritable bowel disease, total colectomy, cirrhosis, a need for antibiotics within 6 weeks of baseline, prior fecal transplant, or if they were in intensive care. There were 10 women and five men in each cohort.

After patients stopped taking antibiotics, there was a washout period after which they took SER-109. Stool was collected on day four and at 1, 2, 4, 8, and 24 weeks. Efficacy was assessed at the 8-week time point.

In the first group, 13 of the 15 patients achieved the protocol-defined endpoint: absence of C. difficile over the 8 weeks.

The two patients who failed had self-limited, transient diarrhea with a positive C. difficile test, but neither required antibiotics and both recovered within 24 hours, so they were considered to not have recurrent C. difficile, said Dr. Cook. In the second cohort, 14 of the 15 patients were free of C. difficile at 8 weeks. The patient who failed had diarrhea and a positive C. difficile test, and the diarrhea did not resolve on its own. She required antibiotics to achieve remission.

There were no serious adverse events in the study.

The company hopes to eventually conduct a phase III study and seek Food and Drug Administration approval, but it is too early to say when that might occur, said Dr. Cook.

[email protected]

On Twitter @aliciaault

This story was updated on October 14.