FDA releases draft guidance on master protocols – efficient clinical trial design strategies to expedite development of cancer drugs and biologics

The Food and Drug Administration (FDA) has released a 21-page draft guidance on master protocols, which aims to provide recommendations to sponsors of drugs or biologics for the treatment of cancer, regarding the design and conduct of clinical trials intended to simultaneously evaluate more than one investigational drug and/or more than one cancer type within the same overall trial structure (master protocols) in adult and paediatric cancers.

The FDA Commissioner, Scott Gottlieb, has stated that “well-designed master protocols that look at multiple therapies in a single disease, a single therapy in multiple diseases, or multiple therapies across multiple diseases or disease subtypes, can provide answers more quickly and efficiently than traditional clinical trials.”

Sponsors are becoming increasing interested in expediting late-stage drug development with trial designs that test multiple drugs and/or multiple cancer subpopulations in parallel under a single protocol, without needing to develop new protocols for every trial.

The FDA states that “in general, the recommended phase 2 dose (RP2D) has been established for an investigational drug or drugs evaluated in a master protocol”. The draft guidance outlines aspects of master protocol designs, trial conduct and other considerations such as biomarker co-development and statistical analysis considerations. The document also includes advice on the information that sponsors should submit to the FDA and how sponsors can interact with the FDA to facilitate efficient review. Safety and regulatory considerations are also included as sections of the draft.

To read the draft guidance from the FDA regarding master protocols: efficient clinical trial design strategies to expedite development of oncology drugs and biologics, please click here.