FDA issues draft guidance on decentralized clinical trials

In a newly released draft guidance, the US Food and Drug Administration (FDA) is proposing recommendations on conducting decentralized clinical trials (DCTs) of drugs, biologics and medical devices.

While the agency initially released draft guidance on trial decentralization during the early months of the COVID-19 pandemic, the new draft guidance was created to fulfill a requirement established in the Food and Drug Omnibus Reform Act (FDORA) to “issue or revise draft guidance that includes recommendations to clarify and advance the use of decentralized clinical studies to support the development of drugs and devices,” by late December 2023.

FDA defines a decentralized clinical trial as one where “some or all trial-related activities will occur at locations other than traditional clinical trial sites” such as performing a follow-up visit in a trial participant’s home over a telehealth appointment or conducting laboratory tests at a local health care facility. The goal of the draft guidance is to “advance medical product development and research” through the use of decentralized clinical trials with the aim of reducing participation barriers, increasing the “breadth and diversity” of trial participants and further drug development, the agency said.

“The FDA has long considered the benefits of decentralized clinical trials. Advancements in digital health technologies and the COVID-19 pandemic—when in-person visits were limited or unavailable for many trial participants—have accelerated the broader adoption of these activities,” FDA Commissioner Robert Califf stated in a press release. “As we seek to improve our evidence generation system, decentralized clinical trials may enhance convenience for trial participants, reduce the burden on caregivers, expand access to more diverse populations, improve trial efficiencies, and facilitate research on rare diseases and diseases affecting populations with limited mobility.”

Noting that the agency has identical requirements for decentralized clinical trials and traditional site-based trials, FDA provided recommendations for decentralized trials that included design considerations, how to conduct a remote trial visit and other related activities, using digital health technology to obtain data, sponsor and investigator roles and responsibilities, how to obtain informed consent and institutional review board oversight of informed consent, how to identify appropriate use of investigational products, how to package and ship investigational products and how to safely monitor participants.

FDA also acknowledged trials can be fully decentralized or contain components of decentralization. For instance, fully decentralized trials have all their activities in non-traditional trial site locations, while hybrid decentralized trials might have some in-person visits at traditional sites and other visits at non-traditional trial sites. Sponsors with simple investigational products may benefit from a fully decentralized trial, the agency noted, while a hybrid solution may be better for an investigational product with a complex medical assessment at a particular study site with remote follow-up visits.

The agency said a decentralized clinical trial’s investigator is still responsible for the overall conduct of the trial as well as the persons who conduct activities in the trial. The investigator of the decentralized trial is also still responsible for obtaining informed consent, ensuring the appropriate administration of the investigational product, and that the appropriate efficacy and safety assessments are performed and documented. FDA noted coordinating all the trial activities in a decentralized clinical trials may be a potential challenge, but decentralized clinical trials should have a plan in place to “facilitate the decentralization of the trial.”

“These plans should include, as appropriate, the use of local health care facilities, local [health care providers], and local clinical laboratory facilities; visits to trial participants’ homes; and direct distribution of the IP to trial participants at their locations,” they said. “Specific issues related to the feasibility, design, implementation, or analysis of a DCT should be discussed early with the relevant FDA review divisions. Appropriate training, oversight, and up-front risk assessment and management will be key to implementing a DCT successfully.”

In a press release, the agency promised to work with sponsors to implement decentralized components into clinical trials. FDA is accepting public comments on the draft guidance until August 1, 2023.

Draft guidance

Federal Register notice

FDA issues draft guidance on decentralized clinical trials

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