Thumbs Up/Thumbs Down: Major insurer delivers a bad blow to Duchenne families
Endpoints assesses the big biopharma R&D stories of the week, with a little added commentary on what they mean for the industry.
Anthem’s ruling on Duchenne drug is a bad blow to families
The big US insurer Anthem has determined that Sarepta’s controversial Duchenne muscular dystrophy drug Exondys 51 is “investigational and not medically necessary,” a blow for families looking to gain coverage for this drug. The determination cites a long and troubled history for this drug, with failed studies and a host of questionable moves Sarepta $SRPT made in advancing this drug on the back of a tiny study. “Exondys 51 failed to show it improves health outcomes, and therefore it is not a covered benefit for our members,” Anthem spokeswoman Leslie Porras told Reuters, confirming the obvious. The discussion over this drug turned into a battle inside the FDA, with CDER director Janet Woodcock winning the argument in favor of an accelerated approval over the vehement objections of senior-level officials. As insurers refuse to cover this drug, which will be sold for $300,000, Woodcock has set up a scenario where families will be expected to fork over huge sums to treat children with a drug they have vowed works just fine. That would be a tragic outcome. The drug is experimental, of course. But families shouldn’t be left on the hook for the cost of a rare disease treatment like this, which may have no affect on the disease at all. And therein lies the tragedy behind this painful situation. Insurers can afford it and should step up and do the right thing, but we doubt they will. After all, insurers have investors as well. Sarepta’s shares were down 8% today on the news.
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