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A Big Push for Decentralized Trials

More remote monitoring, fewer visits to a research center would make it easier for participants to take part in clinical trials, say patient advocates and federal regulators.

A woman with gray hair talks to a doctor on her iPad at home

Many agree that clinical trials  – strictly controlled research that tests medical treatments – should be easier and more convenient for participants. When trials are tied to a geographic location, it often means burdensome travel and life disruptions are needed to meet the requirements for visits and check-ins.

But measures taken to keep trials going during the COVID-19 pandemic could lead to more “decentralized trials” (DCTs), which could ease the burden for people who want to take part. Decentralized clinical trials, which can include remote monitoring, video visits and mobile nursing care, reduce the need to go to a research site for all the appointments and lab tests. Instead of going to a trial site to pick up medicine, the company doing the trial can ship it to a participant’s home.

“I dropped out of one trial I was in since the commute was too much on a monthly basis, and some months more than one visit per month,” said Jennifer Arters, eClinical manager for CSL Behring who has celiac disease. “Had I had the choice to participate in a decentralized trial, I could have continued.”

This new direction in clinical trials will be especially welcome in studies involving rare disease patients, a U.S. Food and Drug Administration official said during a panel about DCTs at this year’s Breakthrough Summit hosted by the National Organization for Rare Disorders (NORD).

“Enrolling patients with rare diseases has always been a significant hurdle. There are many patients, for example, in rural areas, that don’t have access to some of the bigger health care centers and we really are excited that DCTs can be a tool for many of these patients to get access to health care they might not otherwise have,” FDA Health Science Policy Analyst Elizabeth Kunkoski said.

A decade ago, when Arters participated in a clinical trial, no one was talking about decentralizing trials, she said. But then in March of 2020, the pandemic forced ongoing trials to become more decentralized. It worked well thanks to new technologies and the FDA’s emergency guidelines and accommodations, said Ed Neilan, Chief Scientific and Medical Officer at NORD. Now, it’s important to take those gains and build upon them, he said, instead of reverting to old-style trial design. He’d like to see a show of support for DCTs, telemedicine and equitable trial participation.

“We encourage patients and families to work with NORD and other patient advocacy groups to help make their voices heard,” he said.

Next steps include listening sessions with the FDA and conversations with interested biopharmaceutical companies about how to lessen the burden on participants and families, he said. They have new expectations after seeing how trial sponsors responded during the pandemic with more accessible, decentralized trials.

“To the sponsors, we recognize that it’s a significant lift to go down this path but it’s worth it to help these patients and advance drug development for rare diseases,” Kunkoski said.

To learn more about clinical trial participation, watch Arters in conversation with CSL Head of R&D and Chief Medical Officer Bill Mezzanotte.