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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