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.

 

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Would you take a pill to prevent AIDS? Those at risk should consider it, says Massachusetts.

The Globe reports today on the push to make Capturean AIDS prevention drug available. After
many years of struggling to come up with a vaccine, this was a true breakthrough. (We don’t use that word lightly on this page.) But instead of a shot that stays with you for year, here you have to take a pill.

Critics questioned the wisdom of giving powerful drugs to healthy people, and worried that access to a preventive drug would encourage promiscuity or lead to a spike in other sexually transmitted diseases by reducing condom use.

But as study after study — some conducted in Boston — found the drug safe and effective, public health officials came to embrace PrEP, concluding the benefits exceed the hazards. The US Centers for Disease Control and Prevention estimates that some 1.2 million people at risk of HIV infection should at least consider PrEP.

 

 

 

 

 

The New Yorker:#Boston researchers are hunting for a #Zika vaccine — trco201

The August 22 edition of The New Yorker includes a story by Siddhartha Mukhergee on Dan Barouch, director, Center for Virology and Vaccine Research at BIDMC. He’s on the hunt for a Zika vaccine and his work on HIV is informing the effort. From the article, which is not behind the NYer pay wall:

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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

@CommonHealth: Wikipedia and “energy” therapist clash over definition of “respectable scientific journals”

ss We link to a CommonHealth guest post by a Spaulding Rehab doc Eric Leskowitz. He promotes new age approach to pain known as “energy medicine “ and says he’s  been dismissed by Wikipedia as a “quack” despite his peer-reviewed work and Harvard cred

Hard to disagree with the first part of Wikipedia’s response to a petition supporting Leskowitz…

 If you can get your work published in respectable scientific journals – that is to say, if you can produce evidence through replicable scientific experiments, then Wikipedia will cover it appropriately..

.…except to say: What’s defines a “respectable”  journal.  True, not all peer review is equal, but who makes that call?

Wikipedia errs on the safe side and sets a high evidence bar. Still, Wikipedia’s ‘s citation of  Quackwatch — a virulently  anti-alt medicine web site — seems shaky. That site casts its net widely, ragging on everything from faith healers to acupuncture and massage therapy.

And the rest of Wikipedia’s response seemed kind of harsh…

What we won’t do is pretend that the work of lunatic charlatans is the equivalent of “true scientific discourse”. It isn’t.

At the same time, Leskowitz doesn’t do himself any favors by citing Dr. Mehmet Oz’s endorsement.  Dr. Oz has been known to promote less than substantiated therapies.

Energy Psychology has even gotten some fairly mainstream attention, from television’s Dr. Oz to The Huffington Post.

 

 

Supported by evidence? Mobile health screening gets scrutinized

My latest for HealthLeaders Media looks at Public Citizen charges that a health screening program overstates the promise of its services. Public Citizen takes issue with company claims that cardiac screening saves lives. After issuing am initial statement criticizing Public Citizen as proponent of “government-run health care”, HealthFair now says it wants to work with regulators to ensure the accuracy of its advertising.

The debate over screening for heart disease and other conditions is playing out in a consumer group’s campaign to get hospitals to cut ties with the mobile screening company HealthFair Health Screening.

Last week, Public Citizen expanded its campaign by asking the Federal Trade Commission to investigate whether HealthFair’s promotional materials amount to deceptive advertising.A handful of hospitals have discontinued their relationships with Florida-based HealthFair after Public Citizen’s Health Research Group (HRG) accused the company of “fear-mongering.” In June, Public Citizen contacted HealthFair’s hospital clients and The Joint Commission to complain that company overstates the health benefits of its screening programs.      

Science writers meet in Cambridge MA to discuss the shabby treatment of female sci scribes

On deadline today, but will be keeping an eye on this.  You can too.  

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The world of science writers has a dual problem that we have confronted recently, involving issues of sexual equality and of sexual harassment. As an initial step toward grappling with these problems, the National Association of Science Writers held a session at its 2013 meeting in Gainesville, the XX Science Question, featuring six panelists and a standing-room-only audience intent on airing these concerns. What was originally planned as a session on women and representation among science writers instead grew into a plenary in which the community brought forward a number of issues. But time was necessarily limited, precluding getting into any single concern in any depth.

Now, the members of that panel have expanded what began at the plenary in Gainesville and are coordinating a conference to address these issues, with generous funding from the National Association of Science Writers. The conference will take place at MIT on June 13-15, 2014, to bring together stakeholders in the community for training, discussion, and finding consensus on solutions. While the team listed below is serving to organize and coordinate, the important contributions at this conference will come from attendees, some invited and others registered through open registration, all with the goal of having as much representation as possible across community stakeholders.