RARE Daily

Lawmakers Raise Concerns about Differing Use of Accelerated Approval by FDA’s CDER and CBER

July 11, 2024

Rare Daily Staff

A report from the Republican-lead House Appropriations committee accompanying a proposed 1 percent cut in the budget for the U.S. Food and Drug Administrations raises concerns about differing approaches to the use of accelerated approval by the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research.

Senior Editor Zachary Brennan writes in Endpoints that the House Appropriates Committee is not only marking up a bill that provides a total of $6.7 billion to the FDA, a 1 percent cut to the FDA budget over the previous fiscal year’s budget but nearly 10 percent less that President Joe Biden had requested. Of that amount, $3.2 billion would come from user fees.

More troubling is what the Republican-led committee cites as misalignment between the FDA’s CDER and CBER divisions on their use of accelerated approval. Accelerated Approval has been a critical tool the FDA has used to provide access to drugs for serious disease with unmet needs where evidence may be encouraging based on a biomarker, but not conclusive of a drug’s efficacy. Drug developers are allowed to market the drug while they conduct a confirmatory trial. CBER Director Peter Marks has been advocate of the use accelerated approvals for rare disease drugs to speed access to patients in need, but it has been controversial as companies can reap big revenues from drugs that are ultimately found not to be effective.

“The Committee is concerned that the application of the Accelerated Approval Pathway is not uniform between CDER and CBER,” lawmakers said in the report. It wants the FDA to brief the committee on how accelerated approvals can be administered equally by both divisions.

Read the full article in Endpoints

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