USA – FDA: Master protocols have value in COVID-19, and beyond

An official from the US Food and Drug Administration (FDA) recommended that developers of COVID-19 drugs consider using master protocols to assess the benefits and the risks of potential COVID-19 treatments and outlined ways to ensure the integrity of the data generated from these protocols.

Gregory Levin, the deputy director of the Division of Biometrics III in the FDA’s Office of Biostatistics, described how master protocols can be leveraged to evaluate the safety and effectiveness of COVID-19 drugs at the 14 April DIA/FDA Biostatistics Industry and Regulator Forum.

Levin’s remarks drew from recent submissions of master protocols to the agency for COVID-19 treatments.

FDA’s 2018 guidance defines master protocols as “a protocol designed with multiple sub-studies, which may have different objectives and involves coordinated efforts to evaluate one or more investigational drugs in one or more disease subtypes within the overall trial structure.” (RAPS Focus 21 December 2018) These protocols have often been used to boost statistical power in trials with small sample sizes…