When Should Unmet Need Trump Inadequate Data?

The Esbriet review exemplifies FDA’s eternal struggle between requiring adequate evidence of efficacy and giving patients with deadly diseases a chance at treatment.

FDA has proven time and again that it will extend considerable regulatory flexibility to drugs for serious unmet medical needs and orphan diseases, but it decided to issue a “complete response” letter for InterMune Inc.’s Esbriet (pirfenidone) for idiopathic pulmonary fibrosis in 2010 – and IPF patients had to wait four more years for approval of the first drugs to treat the rare and fatal lung condition.

Review documents show that FDA struggled with the decision, and that it had an unexpected ally in choosing to wait for better evidence of efficacy: patient advocates.

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