RARE Daily

Rare Disease Organizations Sign Call for Strengthening the Accelerated Approval Pathway

April 8, 2022

The National Organization for Rare Disorders along with 90 other patient organizations sent letters to Health and Human Services Secretary Xavier Becerra and Congressional leaders urging them to protect patient access and strengthen the accelerated approval pathway.

Photo: Heidi Ross, acting vice president of policy and regulatory affairs

The accelerated approval pathway allows the U.S. Food and Drug Administration to approve drugs that treat serious conditions and that fill an unmet medical need based on a surrogate endpoint believed to predict a clinical benefit.

The pathway came under criticism and the target of reform efforts following the FDA’s controversial approval of Biogen’s Alzheimer’s therapy Aduhelm, which the agency approved despite an advisory panel’s near unanimous vote that studies failed to demonstrate adequate evidence of efficacy.

NORD sent a letter to Congressional leadership urging legislators to incorporate into legislation to reauthorize the Prescription Drug User Fee Act (PDUFA) several policy recommendations to strengthen the accelerated approval. PDUFA, a law created to charge industry fees to support the drug review process in exchange for timely action, is up for its seventh renewal.

The organization sent a separate letter to HHS Secretary Becerra, urging him to support the efforts underway within Congress and FDA to strengthen the accelerated approval pathway and reject policies that could limit patient access to rare disease and other therapies that utilize accelerated approval.

“Accelerated approval is vital to patients with rare diseases and other life-threatening medical conditions where no treatment currently exists,” said Heidi Ross, acting vice president of policy and regulatory affairs, NORD. “However, NORD and the other 90 co-signing organizations believe reforms are necessary to help ensure patients have timely access to rare disease treatments that have been FDA approved through the accelerated approval pathway.”

Author: Rare Daily Staff

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