Overview
- The new default, outlined by Commissioner Marty Makary and deputy Vinay Prasad in a Feb. 18 New England Journal of Medicine article, replaces the FDA’s longtime expectation of two “adequate and well-controlled” trials.
- Regulators cite advances in trial design and supplementary evidence to justify relying on one adequate study for efficacy and safety determinations.
- Roughly 60% of first-in-class drugs in the past five years were already cleared on a single pivotal study, a trend that began expanding in the 1990s.
- Makary and Prasad predict the shift will spur a surge in development and chiefly affect treatments for common conditions, not cancers or rare diseases that often used single trials already.
- Consistency questions sharpened as the vaccines office initially declined, then agreed to review Moderna’s mRNA flu shot after the company committed to an added study in older adults.