Confirmatory trials and the new realities of FDA expedited approval

The US Food and Drug Administration (FDA) established the accelerated approval pathway through regulation in 1992 in direct response to the HIV and AIDS crisis. The program was later written into law in 2012 to provide earlier access to promising novel therapies for severe conditions.

Under this framework, the regulator grants authorizations based on an effect on a surrogate endpoint or an intermediate clinical endpoint. These measures must be reasonably likely to predict genuine clinical benefit, allowing companies to market therapies before completing protracted trials evaluating final overall survival data.