Dangerous Proposal Would Exempt Rare Disease Drugs from FDA Randomized Trial Requirement

November 7, 2024

Dangerous Proposal Would Exempt Rare Disease Drugs from FDA Randomized Trial Requirement

WASHINGTON, D.C. - A forthcoming legislative proposalwould allow the U.S. Food and Drug Administration (FDA) to approve drugs for rare diseases based on substantial evidence of effectiveness without randomized controlled trials, the "gold standard" process underlying drug approvals for decades.

If the proposal, expected in 2025, were to move forward, it would be one of the most dramatic changes to the drug approval process in FDA's history. Dr. Janet Woodcock, who served as FDA Principal Deputy Commissioner and Acting Commissioner before retiring in 2024, is collaborating on the initiativewith the Haystack Project, a rare disease non-profit organization that partnerswith Big Pharma.

Dr. Robert Steinbrook, director of Public Citizen's Health Research Group, called the proposal a major step backward for the FDA.

"This proposal threatens to undermine the drug review and approval process and by extension, could endanger patients," said Steinbrook. "Although the FDA has erred on specific decisions, such as the recent approvals of new drugs for Alzheimer's disease, the agency already has ample authorities and flexibilities to consider drugs, including those for rare diseases, within its existing regulatory structure. The focus of researchers and industry should be on developing better drugs for rare diseases, not changing or weakening the FDA's approval standards.