Opinion: Aduhelm backlash threatens to reverse progress in FDA’s reviews of rare and ultra-rare disease drugs

The FDA’s approval of Aduhelm to treat Alzheimer’s disease has unleashed criticism about the decision and calls for reform within the agency. As advocates for people living with Barth syndrome — an ultra-rare, life-threatening genetic condition affecting just 130 children and young adults in the U.S. — we fear the pushback will discourage the FDA from consistently exercising appropriate flexibility in its approval standards and stall progress being made in employing new regulatory strategies to improve access to safe and effective therapies.

Any moves that erect additional barriers to approvals will likely directly result in intensified reluctance to invest in future biomedical innovation, especially for ultra-rare diseases, and pose dire consequences for people living with the vast majority of the estimated 7,000 rare diseases for which there are currently no approved treatments. Taken together, these disorders affect as many as 30 million Americans, so this problem must not be overlooked.

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