FDA Loosens Approval Process for Drugs Targeting Rare Diseases

The drugs eligible for the looser standards are for diseases that affect fewer than 1,000 people in the United States, regulators said.

The U.S. Food and Drug Administration building in White Oak, Md., on June 5, 2023. Madalina Vasiliu/The Epoch Times

Food and Drug Administration officials on Sept. 3 said they will approve some drugs that target rare diseases with no traditional randomized controlled trial data, citing challenges with testing investigational products for diseases that affect low numbers of Americans.

Manufacturers can apply to the FDA under the new process for drugs against rare diseases driven by genetic defects. The new requirements are that the drug targets a rare disease that impacts fewer than 1,000 people in the United States, the drug is aimed at treating a genetic defect that is a major driver of the disease, people with the disease deteriorate rapidly, and there are no cleared products for the disease.