Will 21st Century Cures Act benefit hospitals?

captureFirst: The Globe is getting some attention for its five-part narrative about a family that helped  start a drug company to keep their son on an experimental cancer treatment.

Nicely written narrative with illustrations and audio. We won’t give away the ending. 

Also, here’s what people were saying a year ago about the “21st Century Cure Act.” It’s been amended since then, but much of what they have to say still applies. My take was: What’s in it for hospitals? 

The drug development and approval elements in the proposed legislation are the centerpiece of a gift basket that has something for everyone: engaged patients, drug makers, FitBit fanatics, NIH-funded scientists, those faced with rare diseases, and those fighting antibiotic resistance.

What do hospitals get out of it? Of course, it depends on whom you ask. One side says it will clear out bottlenecks that are delaying access to better drugs and new, effective antibiotics. That’s good news for hospitals struggling to improve patient outcomes and prevent readmissions. The other side says it will lead to unsafe drugs and a flawed approach to dealing with antibiotic resistance. That’s bad news for hospitals struggling to improve patient outcomes and prevent readmissions.

John Powers, MD, is a former head of the FDA’s Antimicrobial Drug Development and Resistance Initiatives. He is now a clinical professor at the George Washington University School of Medicine and one of the bill’s critics. If the bill’s provisions regarding FDA approval become law, hospitals should be worried that they will be held responsible for drugs that are ineffective or worse, he said.

“If you have a new drug and it doesn’t make the patient better, what you actually end up doing is spending more money in the long run because they stay in the hospital longer,” Powers said. “Or you may have to administer additional treatment to deal with the side effects of that medicine — which costs money.”

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Two Boston meetings look at the role of patient advocates. They offer two very different perspectives.

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Pharmaceutical manufacturers often  look to patient advocates for help winning approval for new drugs. Their most recent success in this area was the FDA’s approval of a new drug for Duchenne muscular dystrophy. That decision came despite recommendations against approval from FDA staff.

In an editorial, the Boston Globe questioned the FDA’s move while noting that eteplirsen’s “entry into the marketplace represents a major victory for the patient advocacy movement, and is bound to encourage more such engagement in the drug-approval process. Based on the infighting that went on over the Duchenne treatment, that’s going to be challenging for the FDA. It has to find a balance between public opinion and what’s truly in the public interest.” (The Globe also featured a story this week about one of those patient advocates.)

Today, drug makers in Boston are hosting a conference for patient advocates. The “Patient Advocacy Summit 2016 – Sharing Our Stories: Building a Patient-Centered Ecosystem” is underway at Novartis facility near MIT.

This event brings industry leaders together with patient advocates and other stakeholders to examine ways in which life sciences companies can more fully incorporate the patient voice into the work they do— not just approaching regulatory applications or at commercialization, but throughout the drug development cycle.

The day-long event will include panel discussions, case study presentations (spotlighting industry/patient partnerships), a keynote address, and awards ceremony, as well as a networking breakfast, lunch and cocktail reception. Expected attendance is 180 patient advocacy professionals, patient organizations and other stakeholders.

Worth noting that the same topic was the subject of  yesterday’s  panel at HUBweek, a  science/tech/arts series ongoing in Boston. The title: “The FDA and the Drug Approval Process: Is it Really Broken?”  Some made the point —  we should listen to the parents of sick children.  Others offered a different perspective: Patients might be better off in clinical trials with informed consent and free drugs,  rather paying  $300,000 per year for that same, unproven medication with unknown side effects.

Contacted after the panel, Zuckerman, president of the National Center for Health Research in DC,  offered these thoughts:

  1. Patient perspectives are crucial in helping us understand what scientific data mean, what the benefits and risks both mean to patients.  So patients should be part of the process – what should the outcome measures be and how can they be measured?
  2. The FDA is listening to patients who desperately want treatments but they are not listening well to patients who are harmed by ineffective or unsafe treatments.  That’s partly because the former are funded by Pharma to attend FDA meetings and to lobby Congress, but the latter are on their own, often don’t have the money to attend FDA public meetings, and wouldn’t even know about them if they don’t read the Federal Register, which is the only place they are announced in advance.

 A few more tweets worth noting.

 

April 26: Boston health news round up

Health writers on both ends of 135 Morrissey Boulevard have been busy — main Globe newsrofenway__1282224095_4483 (2)om to STAT down the hall:  From Stat;  from the Globe business desk. 

Stat shared this story with the print edition of Globe today: Lam, a 22-year-old aspiring doctor, is part of the fast-growing industry of medical scribes working in hospitals and clinics across the country. These scribes, often premed college students, help doctors with a dreaded task — checking boxes and typing words into electronic health records.

And both outlet covered yesterday’s disturbing FDA meeting. This is an ongoing story — desperate patients insist on FDA approval treatments despite lack of solid evidence of efficacy.

Video up soon: What are the public health implications of terrorism? This (Harvard School of Public Health) Forum — which took place a week after the 3rd anniversary of the Boston Marathon bombings — asked what makes a society resilient in the face of attacks or perceived threats. Experts in homeland security, psychological resiliency, crisis leadership, and disaster preparedness and response participated. 

 Dumb question award: Boston Magazine: Should the Media Report on Health Research?  A good topic, but a bit too much to bite off in a blog post. For a more thorough analysis, see Health News Review, which this week looks at what happens when the media doesn’t report on Research

Health Wonk Review: Lots of blog commentary and an invitation you to this afternoon’s “blab” on health policy.

Is female Viagra a scam? Boston women’s health group questions drug, supporters

indexThe non-profits pressuring the FDA to approve a drug billed as female Viagra do not quite make up a top ten list or women’s health advocacy organizations. This health writer has never heard of most of the groups cited in Sunday’s New York Times story.

But familiar women’s health groups, like Boston’s Our Bodies, Ourselves, are siding with the FDA on this one. From their response to an earlier review of the drug.

imagesWomen taking the drug had less than one additional “sexually satisfying event” (orgasm not required) than women taking a placebo. And in the meantime, the drug caused dizziness, nausea and fatigue, particularly with long-term daily use, in some women — hardly the recipe for sexual excitement.

The FDA also considered whether the drug had increased women’s desire — a crucial element of the HSDD diagnosis, which involves low or no sexual interest to the point of distress in people who are physically healthy and not depressed — and found that the drug failed in this area.

The FDA takes another look — and offers  a live webcast of the deliberations —  on Thursday.

In it’s report on the 2010 FDA rejection of the drug, the OBOS  website notes another Bay State-based critic of the drug:

According to Julia Johnson, the panel’s chairwoman and head of the department of obstetrics and gynecology at the University of Massachusetts Medical School, the impact of the drug flibanserin … was “not robust enough to justify the risks.”

More here from another independent, feminist women’s health group, The National Women’s Health Network:

Members of the campaign called “Even the Score” are challenging the FDA on what they claim is a perpetuation of a gender bias by virtue of the claim that the FDA is holding drugs that treat women’s sexual problem to a higher standard than those for erectile dysfunction.  Even the Score has engaged prominent women’s rights organizations, health care providers, the media and members of Congress in a public relations misinformation campaign to criticize the FDA.  There are Female Sexual Dysfunction drugs currently under FDA review, and Even the Score is attempting to move the discussion away from the safety and effectiveness of these drugs and towards controversy about gender bias. 

The reality is that no amount of public relations or slick marketing can get around the fact that the drugs currently being proposed for Female Sexual Dysfunction simply don’t work and may be quite dangerous. Poor efficacy, a strong placebo effect, and valid safety concerns have plagued all of the drugs that have been tested so far. There are many reasons why the proposed drugs may not have been effective in increasing women’s sexual enjoyment; chief among them is the heterogeneity of female sexuality and, of course, research demonstrating that sexual problems are mostly shaped by interpersonal, psychological, and social factors. Nevertheless, pharmaceutical executives will continue to drum up hype over the possibility of a “pink Viagra” because the profit market for this type of drug is estimated to be over $2 billion a year.

Note that neither of these groups accept funding from the pharmaceutical industry. Even the score supporter include Sprout, the company seeking approval for the drug and Trimel Pharmaceuticals, a company testing a nasal testosterone gel for “female orgasmic disorder.”

Should the FDA approve Vertex’s new cystic fibrosis drug?

The scene at the FDA hearing was familiar. All the  advisory committees have seen it play out again and again. Yesterday, it was the Pulmonary-Allergy Drug committee: The pharma doc with the convincing statement. The weeping patients. The drug likely to cost a couple thousand a month offering a slim  benefit over an existing drugs. But it works, and it’s safe.

From the Globe:VertexLogoSOP

“I think this is a much-needed advance for patients with cystic fibrosis,” said committee member Dr. Michelle S. Harkins, associate professor of medicine at the University of New Mexico Albuquerque, one of the majority voting to recommend approval.

The lone dissenter in the 12-1 vote recommending approval of the drug, Dr. Yanling Yu, the president and cofounder of Washington Advocates for Patient Safety in Seattle, said she was not convinced the data generated by the Vertex testing supported the approval of Orkambi.

“I really understand the patients critically need a new drug, but sometimes a new drug does not provide [the needed effectiveness],” she said.

From The New York Times story:

An issue for the advisory committee was that Orkambi had what the F.D.A. said was only modest effectiveness, improving lung function by only about 3 percentage points relative to placebo.

Some family members or advocates, some of them crying, pleaded with the committee to endorse the drug.

Some patients who took the drug in clinical trials said it had made a huge difference in their lives, reducing their coughing, allowing them to exercise better, helping them gain weight or reducing how often they ended up in the hospital…

Michael Yee of RBC Capital Markets, for instance, expects the price will be $225,000 to $250,000 a year.

The vote is advisory. The FDA staff will make the final call.

More here:

The financial stakes for Vertex.

FDA briefing for meeting.

Vertex briefing for the meeting

Avandia, the Duke Clinical Research Institute and conflict of interest

The  debate over potential bias at the Duke Clinical Research Institute emerging fro  this morning’s Avandia hearing inspired a trip to the archives. This story looked at many of the same issues, which came up when  the Duke clinical trials operation was young.

By Tinker Ready

from The Raleigh News & Observer

Genentech and university researchers wanted to ensure that a study of the comparative effectiveness of  tPA was definitive. Critics say they failed.

By TINKER READY and TRISH WILSON

STAFF WRITERS

THE NEWS & OBSERVER

 3/31/1994  

All corporate research spending on campus isn’t aimed at inventions. Sometimes the goal is to torpedo a competitor.

That was the hope of Genentech, a San Francisco biotechnology company. It paid $55 million for a study comparing its heart attack drug to a far cheaper drug cornering the market for thrombolytics. The drugs dissolve blood clots and reopen arteries after a heart attack.

The study — completed last year — was coordinated at Duke University, where researchers said their goal was to determine which method of using the two drugs would save more lives.

Genentech’s sponsorship of the massive study raised eyebrows, but the company tried to reassure the medical community that the study would be unbiased. A previous tPA study had been dogged by charges of conflict of interest, and Genentech wanted to avoid a replay.

The company chose Duke University to coordinate the research because it had a relationship with Duke scientists and their collaborators, who had worked with tPA in the past.

“If Genentech was forking over the money, they wanted someone to run the study who they trusted, ” said Robert Califf, a Duke cardiologist and the study’s clinical director.

The company also had reason to trust Ralph Snyderman, the top administrator at Duke’s medical center and former Genentech executive. Snyderman left Duke to join Genentech in 1987 as director of research and development. While there, he successfully ushered tPA through a long and contentious Food and Drug Administration approval process before returning to Duke in 1989.

In the end, Genentech’s study paid off. After examining medical records of 41,000 heart attack patients in 15 countries, researchers announced that Genentech’s tPA (about $2,200 a dose) was superior to streptokinase (about $300 a dose). They concluded that the prompt use of tPA could reduce by 14 percent the number of people who died from heart attacks.

Eric J. Topol — a cardiologist at the Cleveland Clinic Foundation and chairman of the study — claimed victory for tPA.

“We have to put to rest this battle of the thrombolytics, ” Topol said at an April 1993 news conference in Washington. “Accelerated tPA was significantly better.”

That day, the price of Genentech stock jumped $4.75 a share to $37.50.

Not everyone shared the stock market’s optimism. Some doctors thought the results were ambiguous and failed to reflect tPA’s risks, particularly a higher incidence of stroke among users.

One member of the steering committee — streptokinase advocate Victor Marder — said Genentech had generated what it wanted.

“On the face of it, Genentech was not part of the decision-making apparatus, ” said Marder, the chief of hematology at the University of Rochester medical school.

On the other hand, he said, the test was not a fair comparison. It compared a hyped-up dose of tPA to a standard dose of streptokinase, Marder said. In addition, he said, he did not think the study properly accounted for the disproportionate number of tPA patients who might have done better because they had undergone heart bypass operations.

When he tried to raise these issues to the steering committee, “everybody got really angry, ” Marder said.

Marder thinks the study fails to prove that tPA is better.

“I don’t think it’s dishonest, he said. “I think it was an exaggerated conclusion made to influence people.”

Califf said that Marder is off the mark. The comparison between the two drugs was valid, he said, and the study accounted for the bypass operations.

Soon, Marder and other critics will be able to see that for themselves, because all the data is about to be published in 40 manuscripts.

“Anyone can look at it and draw their own conclusions, ” Califf said.

Califf doesn’t deny that Genentech had a huge stake in the outcome. But he vehemently denies that the study was designed with a foregone conclusion.

The project began when Califf and Topol — who had done research with tPA — began talking to Genentech about a larger study. At the time, the company was trying to cope with 1990 Italian study that found no significant difference in tPA and streptokinase.

By the time a second damaging European study came out in 1992, Genentech was ready. The company promised that a new, bigger study would definitively prove whether tPA was safer and more effective than its low-cost rival.

That study — know as GUSTO for Global Utilization of Streptokinase and tPA for Occluded Coronary Arteries — was headed by Topol. Duke, with its massive cardiac computer data bases, served as a data clearinghouse.

“They needed to do this study, ” Califf said. “They were losing market share. We were very concerned that the European studies had missed the point.”

Both the company and the researchers wanted to make sure the findings were above question, he said.

The company had good reason to expect skepticism. In 1989, a congressional investigation found that 11 of the researchers involved in the original, federally sponsored studies of tPA owned Genentech stock. That stock became much more valuable when the FDA approved the drug.

So, when GUSTO trials came along, Genentech set up an independent steering committee to run them. None of the researchers could own Genentech stock, nor could they consult for the company until at least a year after release of the final results.

Genentech staff say that Snyderman played no role in bringing the study to Duke. Snyderman said in a recent interview that he still consults for Genentech and will continue to do so for the next couple of months.

“There was absolutely no conflict of interest, ” Snyderman said.

Califf called GUSTO’s ethical precautions “draconian.” Genentech staff saw none of the data and had no role in running the study, he said.

“The risk that Genentech had to take is that they had no control over this trial, ” he said.

Califf said his goal was not to compare the clot busters — it was to find out if a doctor can save more lives if he moves faster to open arteries after a heart attack.

Still, if the study also proved that Genentech makes a better drug, and its corporate fortunes turned around, more power to the company.

“I will not make apologies for industry being involved, ” Califf said. “That’s the American way.”

Is Avandia safe? FDA to consider restrictions on the diabetes drug, local docs in the fray

Dr. Clifford J. Rosen, director of clinical and translational medicine at the Maine Medical Center in Portland,  led the advisory committee that on 2010 set strict limits on the use of the  diabetes drug Avandia after it was linked to  heart problems.

avandiaThe New York Times reported on Sunday on the FDA’s  meeting this week, where the restriction will be reconsidered.


More here from the FDA.
Rosen tells the NY Times that would be a bad idea :“It’s turning back the clock. …I  have no idea why they would revisit this issue. It’s a done deal.”

More from the Times here:

That is just one of several options before the advisory committee, but lifting the limits would amount to a major policy reversal and could be a huge victory for the drug’s maker, GlaxoSmithKline. Avandia was once a top-selling drug, reaching more than $3 billion in sales in 2006 before controversy flared. It could also help rewrite one of the most embarrassing chapters in the F.D.A.’s recent history.

imagesBut critics, like Dr. Steven Nissen, the well-known Cleveland Clinic cardiologist who was the first to sound a public alarm about the drug, say it is far too dangerous to use in diabetes treatment. He said an analysis of more than 50 studies linked Avandia to an elevated risk of heart attack; one study linked the drug to more than 47,000 cases of heart attack, stroke or heart failure from 1999 to 2009.

Dr. Nissen and others contend that the F.D.A.’s decision to revisit the drug is more about saving face than protecting patients.

The issue is being takes at a joint meeting of the FDA’s endocrine and drug safety panels.

Dr. Ellen Seely of BWH and Robert J. Smith of Brown are members of the FDA’s Endocrinilogic and Metabolic Drugs Advisory Committee.

Sonia Hernandez-Diaz is a member of the Harvard School of Public Health is a member of the Drug Safety and Risk Management Advisory Committee.

Bloomberg has a different take on the story:

A second look at GlaxoSmithKline Plc (GSK)’s once-best-selling diabetes pill found it doesn’t increase overall cardiac death risk, U.S. regulators said.

Findings by London-based Glaxo that Avandia doesn’t raise the overall risk of death, heart attack or stroke appear to be supported in a reanalysis by independent researchers, some Food and Drug Administration staff said in a report today. FDA advisers will reconsider restrictions imposed on Avandia in 2010 at a meeting June 5-6, according to the report.