A debate over a rare disease drug’s cost

Sarepta Therapeutics drug treatment for Duchenne muscular dystrophy is generating controversy. Again.

This time, the controversy is not over whether Exondys 51 is efficacious. Rather the question is whether it’s cost-effective with a $300,000 price tag.

In a new report, the Institute for Clinical and Economic Review concludes that it’s impossible to know, arguing that there is simply insufficient evidence to assess the matter. At the same time, ICER concluded a Duchenne medicine sold by PTC Therapeutics offers only a modest clinical benefit and would have to carry a much lower price in order to be considered cost-effective.

Sarepta called the analysis “fatally flawed.”

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