FDA Commissioner Says Agency Wants to Develop Master Protocol Trials to Test Multiple COVID-19 Drug and Vaccine Candidates at Once
To get COVID-19 vaccine and therapeutics candidates through the pipeline faster, FDA Commissioner Stephen Hahn said yesterday that the agency is interested in developing master protocols, possibly in conjunction with regulatory agencies in other countries.
In contrast to traditional trial designs, where a single drug is tested in a single disease population in one clinical trial, master protocols use a single infrastructure, trial design and protocol to simultaneously evaluate multiple drugs and/or disease populations in multiple substudies, allowing for efficient and accelerated drug development. The drug candidates are each compared to the control group but not to one another.
“It’s a very efficient way of looking at multiple different therapeutics, vaccines,” said Hahn in a public presentation.
If FDA gets behind a master protocol trial to focus on COVID-19 candidates, it won’t be the first time the agency used that trial design to address a pandemic. During the 2015 Ebola outbreak in Africa, the FDA and the National Institutes for Allergy and Infectious Disease employed a master protocol to test multiple therapeutic candidates. The Ebola epidemic faded before the trial showed robust results, but the design held up. The FDA said then that the master protocol concept “successfully illustrated the feasibility and desirability of innovative trial designs to meet the demands of a public health crises.”
In late March, the World Health Organization proposed a master protocol for companies and institutions that aim to test therapeutics against COVID-19.
FDA announced the availability of a draft guidance for master protocols — also called basket trials, umbrella trials and platform trials — in 2018 for sponsors working on cancer drugs.
Examples of trials now using master protocols include: the Lung-MAP trial, which is testing several different treatments that target genetic changes found in non-small cell lung cancer; the NCI-MATCH trial, a phase 2 study endeavoring to determine whether matching certain drugs or drug combinations in adults whose tumors have specific gene abnormalities will effectively treat their cancer regardless of their cancer type; and the Pediatric MATCH trial, a phase 2 trial that aims to investigate more than eight study drugs, each targeting a defined set of gene mutations, in order to match patients with therapies aimed at the molecular abnormalities in his or her tumor.