This article at the Wall Street Journal should not come as a particular surprise to anyone who followed the Sarepta/eteplirsen story over the last few years. But it’s a good overview of it, and it brings up several questions. (My own take on the drug approval is here – I didn’t think it should have passed).
A brief summary, in case you haven’t followed this one: the company had been developing the exon-skipping therapy for Duchenne muscular dystrophy (DMD) for years, but had very thin clinical data when they went to the FDA. In addition, a broadly similar therapy backed by GSK had failed to show any benefit. But after delays and arguments about accelerated approval (or approval in any form), the drug was approved, to the surprise of many, when Janet Woodcock of the FDA overruled other recommendations.
This is the part that surprises me, I think:
The company, Sarepta Therapeutics Inc., leveraged the emotional appeal of the parents’ stories. Without that, the drug—eteplirsen—likely wouldn’t have been approved, said stock analysts, investors and people close to the matter.
An FDA advisory committee voted 7-6 last year to oppose the drug’s approval, a position later overruled. The Wall Street Journal found that a majority didn’t know how Sarepta’s consultant had guided the patients’ families.
“More might have voted no if they were aware,” said Dr. Bruce Ovbiagele, a committee member, when he learned the role the company consultant played in helping parents prepare for FDA hearings.
I am not surprised at the company using the parents’ stories to make an emotional appeal – this happens all the time in drug approval decisions for rare and serious diseases. I’m not surprised at the company carefully grooming the families to help sway the FDA committee vote, because this happens all the time, too. What I find odd is that the committee members say that they didn’t know that this was happening. Where have you been? What species do you belong to?
Note that in the paragraph above I am not making value judgments about the wisdom or appropriateness of these sorts of maneuvers. That’s another topic. All I’m saying is that patients and drugmakers, since they often find themselves on the same side of a drug approval issue, cooperate with each other to try to achieve their goals. This is exactly how it works with every other key decision taken by a government body, I should also note. Now, I think that this sort of thing should be disclosed, let me make that clear. When some political issue comes up for a vote, and the airwaves are full of ads taken out by the Concerned People Who Want Only Good Stuff For All of Us Committee, I think that the sources of the CPWWOGSFAUC’s funding should be on record for anyone who wants to know where the money is really coming from. And the same goes for lobbying for (or against) drug approvals.
The relationship between companies and patient advocacy groups is complex, and runs along a scale. At one end, you have patient groups that have existed long before any possible therapy has been in the works, and who are likely willing to help out any drug company that might seriously be able to deliver something. At the other end, you have groups that have been created ex nihilo simply to push an agenda. (This same spectrum is found in every other public lobbying effort, and it’s possible to have every shade in between).
These seem, at least to me, to be well known features of public policy, so I’m really baffled by the FDA committee members saying “Had I but known”. The second thing I find odd about that is, what difference should that have made? The decision was supposed to have been based on the data from the clinical trials. I know that the FDA has been getting patients (and patient advocacy groups) more involved in these hearings, but that doesn’t mean that approvals should be tilted to those whose consultants have coached the most wrenching testimony and the most coordinated talking points. We’ve known about these forms of argument for a long, long time now, and the idea is not to elevate pathos at the expense of ethos or (especially) logos. What a mess.