The FDA is certainly aking a lonnnnng time to set things straight on DMD and etep. The illogical if not immoral option of a placebo arm is probably off the table, thanks to the advocacy of the supermoms et al. But the conundrum remains: how does the FDA save face and reverse course? JW is obviously riding herd over an internal group that includes old-schoolers who prefer long-term studies. So...what if....the FDA takes this opportunity to introduce a new way of responding to drugs aimed at deadly orphan diseases? When a drug show dramatic P2 results (as etep certainly has), the FDA can open the door (via AA) to make the drug available to everyone. Caveat here is that the FDA will then monitor the population of drug recipients (essentially, every DMD boy, because there is not a family that will not beg for this drug) as a de facto P3 cohort -- complete with reporting requirements involving physicians and medical facilities. The hindrance is "who will pay for the drug," with insurers balking at covering an expensive drug that is not yet P3 vetted. But dollars be damned: the upside is that DMD boys will gain immediate access to a drug that shows every indication of turning Duschenne into Becker. And JW & Co. can claim full credit for an innovative approach to saving lives. OK.... why can't this work?
I don't get why it would be such a "conundrum" for the FDA to reverse course - the obvious answer to how they do that is to simply state the truth - "Based on our rudimentary understanding of the similarities between Drisa and Etep, we made an erroneous assessment of the likelihood of Etep's clinical benefit after the failure of the Drisa Phase III trial in November, 2013. After receiving additional data on the pharmacological differences between Etep and Drisa, as well as receiving detailed data from the Drisa trial, we have concluded that based on the clinical and surrogate data generated by SRPT to date, there may well be a reasonable likelihood that Etep is generating a clinical benefit. Due to the horrendous impact that DMD inflicts on its victims and their families and the lack of any treatments for DMD, we are advising SRPT to submit an application for accelerated approval".
I'd go one step further. The FDA can just cut out the first sentence of that statement and absolve themselves of making a mistake. Frankly, if that gets the drug to market faster, it's something I can forgive.
yup, that's what should happen. But you and I both know, the FDA is many things, erratic being one of them. I still can't fathom how the November debacle was allowed to happen. The FDA ended up making themselves look like idiots basically, while they continued to delay ANY progress. It boggles my mind, but I have to agree, a simple statement along the lines you've suggested would get the FDA out of the hole they put themselves in, while doing what is clearly in these kids best interests.