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Rare Disease Drugmakers to Congress: Don’t Gut the Accelerated Approval Pathway

By: Zachary Brennan / August 3, 2021

The controversy over the FDA’s accelerated approval pathway is heating up.

Last week, the FDA’s top oncology official Rick Pazdur said the pathway is “under attack,” largely due to the agency’s recent accelerated approval of Biogen’s controversial Alzheimer’s drug and the surrogate endpoint used in that decision. In the meantime, three accelerated approval indications have been pulled since July 1 (two from Bristol Myers Squibb and one from Merck in recent weeks), even as Pazdur called on critics of the pathway to not miss the more positive, big picture, with some cancer drugs proving to be enormously helpful and approved years before their confirmatory trials were completed.

Now, a coalition of rare disease drugmakers is seeking to defend the use of the pathway, raising concerns with a recent call from the nonpartisan MACPAC to increase rebates around drugs approved under the FDA’s accelerated pathway. The rare disease firms claim such a move on rebates would disincentivize drug developers from pursuing therapies in otherwise intractable disease areas, the coalition said in a recent letter to top congressional health committees.

Source: Endpoints News