FDA Guidance May Bolster Confidence in Decentralized Clinical Trials

Pictured: A patient takes her own vitals during a remote doctor's visit / iStock, Maria Symchych-Navrotska

On May 2, the U.S. Food and Drug Administration (FDA) released draft guidance with recommendations regarding decentralized clinical trial (DCT) implementation. Aiming to support DCT adoption, the guidance provided updated advice for sponsors, investigators and other stakeholders on everything from trial design to data collection.

Jeffrey Zucker, senior vice president of decentralized clinical trial solutions and optimization at the contract research organization Worldwide Clinical Trials, said the draft guidance had been “long anticipated,” adding that investigators and industry sponsors can “breathe a sigh of relief.” He said he expects decentralized clinical trials will now grow in number.

“It’s a real game-changer,” he told BioSpace.

Unlike traditional trials, DCTs conduct all or part of trial-related activity outside a clinical trial site, performing a follow-up visit in a trial participant’s home or via telehealth, for example. DCTs are not new but have grown in popularity since the COVID-19 pandemic began, Zucker explained, when investigators were forced to use telehealth and other digital health technologies to limit viral spread.

While the guidance provides recommendations on trial design and implementation, regulatory requirements remain the same between decentralized and traditional clinical trials.

However, in a press release, the agency said that the guidance is meant to support DCTs, with the ultimate goal of diversifying clinical trial participation and improving trial recruitment and retention.

“As we seek to improve our evidence generation system, DCTs 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,” FDA Commissioner Robert Califf said in the release.

DCTs allow sponsors such as pharmaceutical companies to reach a larger and more diverse pool of potential participants, Zucker agreed. Such a model can also help clinical trials recruit participants with rare diseases worldwide.

The agency released earlier guidelines on DCTs in the early months of the COVID-19 pandemic, but since then, decentralized trials have become much more common. The FDA is legally obligated to finalize the new guidance by December of this year under the Food and Drug Omnibus Reform Act (FDORA), which was signed into law in 2022.

Zucker noted significant improvements to the guidance, especially concerning the role of local healthcare providers in DCTs. As per the new guidelines, healthcare providers can participate in patient monitoring, particularly in long-term studies, without being listed on Form FDA 1572, a contractual agreement that, among other things, lists all of the people that will contribute to the study.

“The clinical trial investigator or sponsor they work with can communicate with that health care provider,” Zucker said. “I think that’s a huge step for us.”

The new guidance also follows previous guidance released in December 2021 on the use of digital health technologies, clarifying that sponsors and investigators can use digital devices and services to collect patient data in DCTs, and expounding on how they can do so.

Zucker added that investigators and sponsors are concerned that the guidance specifies that all trial activities, such as shipping medicine to patients, need to be listed in the protocol, which is uncommon. Because the protocol is immutable, “this takes some decisions out of our hands,” he said. “Being able to make the decision for the patient would be better.”

However, the guidance is still in draft form and the FDA will receive comments before it is finalized.

“In general, I think it is a fantastic start,” Zucker said. “And something we’ve been waiting for a long time, because we’ve been conducting clinical trials for a long time.”

Natalia Mesa is a freelance science writer based in Seattle. Reach her at natalia.mesa.v@gmail.com.