Three — well two — cheers for Dr. Janet Woodcock. This past September, as director of the FDA drug evaluation and research center, Woodcock pushed through an accelerated approval of eteplirsen (Exondys 51) for Duchenne muscular dystrophy (DMD). For the past 20 years, families have lobbied vigorously for at least a “chance to try” this promising drug, which is designed to alleviate the mutation causing DMD.
During this time, FDA reviewers have balked, calling the drug a “sophisticated placebo,” demanding additional clinical trials, and insisting on more rigorous endpoints.
The issue is complicated: studies so far have not shown much of a clinical benefit in terms of slowing disease progression. On the other hand, there’s plenty of anecdotal testimony from patient families and, in any case, they have no approved alternative.
Yet while Woodcock deserves kudos for standing up for the patients, it’s unclear if she was entirely motivated by heartwarming tales of improving situations. According to a review of FDA documents, Woodcock was mainly concerned that if eteplirsen were not approved, the stock price would collapse for Sarepta, the drug’s manufacturer. As a result, the company would run out of money and be forced to abandon the drug and others in its pipeline.
Because many scientists believe the approach pioneered by Sarepta may eventually lead to major advances in DMD treatment, perhaps her logic makes sense. And, importantly, eteplirsen is safe. Unlike thalidomide, the FDA’s poster child, it doesn’t cause birth defects, it seeks to correct one. So why not give the drug accelerated — and conditional — approval and let the company continue its research? At least that seemed to be the argument.
But then it gets even stranger. It turns out — again according to FDA documents — the FDA reviewers’ main objection to accelerated approval came down not to their belief that the drug doesn’t work, but that it costs too much!
If you’re shaking your head, so am I. It appears as though Woodcock would have had no problem with rejecting eteplirsen if only Sarepta’s stock price could have remained high enough to continue to fund their research.
On the flip side, the reviewers would have been fine approving eteplirsen if the projected price tag of $300,000 had been more “reasonable.”
Sure enough, when eteplirsen received accelerated approval from the FDA, Sarepta’s stock soared. And equally inevitable, when reviewers began crafting the approval letter, they stuffed it with language suggesting the drug is not medically necessary.
As a result, some payers have already said they won’t pay for the drug. Sound familiar? The FDA approving a drug with one hand, while stabbing it in the back with the other.
None of us are naïve about the role of money in drug development. Medical care costs are exploding, and so is the cost of creating cures. But in our struggle with the bean counters and the stockbrokers, can’t we make patient needs a priority, too?
In a decade of remarkable progress, it’s unfair to ask those with “incurable” diseases to remain patient. There must be some middle ground for the FDA to stake out, between a simple thumbs up and a guillotine thumbs down, that will give appropriate children with DMD a chance, while not opening the floodgates for indiscriminate and fruitless use.
Sander Flaum is a principal at Flaum Navigators