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STAT Plus: Debate erupts over whether Sarepta’s rare disease drug has enough clinical data to determine cost-effectiveness

A new controversy is emerging over a costly treatment for Duchenne muscular dystrophy, a rare disease that can send boys to an early death, thanks to a new analysis suggesting that clinical data is so limited that it is impossible to determine whether the medication is worth a price tag of $300,000 or more.

The medicine, which is called Exondys 51 and marketed by Sarepta Therapeutics (SRPT), caused a heated public debate over regulatory approval standards and satisfying unmet medical needs during the run-up to its endorsement nearly three years ago by the Food and Drug Administration.

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