FDA In Brief: FDA Provides Guidance on Master Protocols for Evaluating Prevention, Treatment Options for COVID-19

FDA in Brief

• For Immediate Release: May 17, 2021

The following quote is attributed to Janet Woodcock, M.D., Acting FDA Commissioner

“During this global pandemic, the FDA worked to protect the American public by using every tool at its disposal to quickly review and act on new therapies for COVID-19.

Conducting clinical trials to see if a drug is safe and effective can be a very time-consuming process. To meet the urgent demand for effective therapies, FDA has worked with clinical trial experts to rapidly advance best practices in the design and execution of clinical trials. Today, we’re providing industry guidance for creating master protocols (an overarching protocol designed to answer multiple questions) when evaluating drugs for the treatment or prevention of COVID-19.

In COVID-19, master protocols have been used to evaluate multiple drugs at the same time. The Accelerating COVID-19 Therapeutics Interventions and Vaccines (ACTIV) public-private initiative coordinated by the Foundation for the National Institutes of Health is one excellent example of master protocols being used to simultaneously study a number of promising drugs.

Master protocols that are well designed and executed can accelerate drug development by maximizing the amount of information obtained from the research effort. These trials can be updated to incorporate new scientific information, as medical science advances. Master protocols also reduce administrative costs and time associated with starting up new trial sites for each investigational drug. They can also increase data quality and efficiency through shared and reusable infrastructure. These advantages are of particular importance during a public health emergency such as the current SARS-CoV-2 pandemic, where there is a critical need for efficient drug development.

The FDA expects master protocols to continue to play an important role in addressing the public health needs created by the pandemic and in generating clinical evidence in general.”

Additional Information

• Today, the U.S. Food and Drug Administration issued a final guidance entitled, “COVID-19: Master Protocols Evaluating Drugs and Biological Products for Treatment or Prevention Guidance for Industry.” This guidance describes FDA’s current recommendations to sponsors of master protocols evaluating drugs for the treatment or prevention of COVID-19. A master protocol is defined as a protocol designed with multiple substudies, which involve coordinated efforts to evaluate one or more investigational drugs, in one or more disease subtypes, with one or more objectives, all within the same overall trial structure.
• This guidance focuses on the design, conduct, and statistical considerations of master protocols intended to generate or contribute to substantial evidence of effectiveness and adequate characterization of safety of drugs for the treatment or prevention of COVID-19. Additionally, this guidance provides administrative and procedural recommendations to sponsors of master protocols for COVID-19 drugs.