Testimony: 21st Century Cures: Modernizing Clinical Trials

Kesselheim argues that the FDA’s requirement for rigorous premarket testing of drugs and high-risk devices emerged as a rational response to public health tragedies, and that the agency already has substantial flexibility to expedite important new products. He cautions that approving drugs based on unvalidated trial designs or surrogate endpoints puts patients at risk, citing bedaquiline as a cautionary example, and notes that post-approval confirmatory studies and safety surveillance systems remain underdeveloped. Watch his testimony.