Companies looking to execute decentralized clinical trials should watch out for data variability and precision as information comes in from patients’ homes or local healthcare facilities, according to new FDA guidance. But the agency is otherwise keen to see more of these studies to help boost diversity in clinical research.

The FDA issued new DCT guidance (PDF) Tuesday to provide clarity on the agency’s support of these innovative trial measures. DCTs can include obtaining lab tests at local facilities instead of a research medical center or conducting follow-up visits at a trial participant’s home using telemedicine. These measures allow patients to participate in trials with less burden, opening up studies to a more diverse population.

“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, M.D., said in a statement. “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.” 

Greater adoption of DCTs will facilitate drug development in areas of medical need, which means more treatment options and better outcomes for patients, the FDA added.

The guidance covers DCT design, conduct of remote clinical trial visits and related activities, use of digital health technologies, roles of the sponsor and investigators, informed consent considerations and more.

In terms of DCT design, the FDA urges investigators to have a physical location where all clinical trial-related records for the participants are held, even if trial activities will occur somewhere other than traditional trial sites. This should all be monitored by the investigator. The variability and precision of data collected using DCT methods may be different than a traditional trial, but should not affect the validity of a finding of superiority, according to the FDA. There could, however, be impacts on a finding of non-inferiority for a drug, because the active control drug would likely have been tested in a more traditional environment. The FDA recommends that investigators reach out when planning a non-inferiority study in a DCT setting.

Investigators should consider telehealth visits instead of in-person in instances where such interactions are not needed. Trial personnel or local healthcare personnel can also go to a participant’s home for trial-related activities. However, any activities unique to the research or that need detailed knowledge of the trial protocol should be conducted by qualified trial personnel.

Trials should also follow the agency’s digital health technology guidance when transmitting data remotely. The guidance also defines the roles and responsibilities of the investigator, sponsor and more. Informed consent can be confirmed remotely but an institutional review board should oversee the process.