Master Protocol (Basket / Umbrella / Platform)
A single protocol that evaluates multiple interventions, populations, or sub-studies under a unified statistical and operational framework.
Definition
A master protocol is a single overarching trial protocol that simultaneously evaluates multiple interventions or populations: basket trials test one therapy across multiple diseases sharing a common biomarker; umbrella trials test multiple therapies in a single disease stratified by biomarker; platform trials evaluate multiple therapies against a common control with sub-studies that can drop or add over time. The architecture reduces administrative overhead, shares control arms, and accelerates evidence generation.What this means in practice
Master protocols originated in oncology and have spread to neurology, infectious disease, and increasingly device evidence generation (companion diagnostic studies, multi-arm imaging AI evaluations).- •Underestimating the operational complexity, master protocols simplify design and complicate execution.
- •Failing to define add/drop rules and DSMB authority precisely up front.
Related terms
Grouped by themeEditor's picks
· Hand-selected related conceptsTrial design that pre-specifies opportunities to modify aspects (sample size, arms, endpoints) based on interim data.
Trial design that uses Bayesian statistics, often incorporating prior data from related studies or registries.
Independent committee that periodically reviews accumulating safety and (sometimes) efficacy data during a trial.
Definitive clinical study designed to establish safety and effectiveness for marketing authorization.
More in Clinical & Trials
· Same categoryAny untoward medical occurrence in a subject, whether or not related to the device.
Data integrity principles: Attributable, Legible, Contemporaneous, Original, Accurate (plus Complete, Consistent, Enduring, Available).
An IVD's ability to detect small quantities of an analyte.
Procedures that hide treatment assignment from patients, investigators, or assessors to reduce bias.
Primary references
3 sources- 1
FDA, Master Protocols Guidance (2022)VerifiedFDAfda.gov
- 2
NIH NCATS, Master ProtocolsVerifiedNIHncbi.nlm.nih.gov
- 3
FDA - Clinical Trials and Human Subject ProtectionVerifiedFDAfda.gov
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