With that in mind, the FDA has released draft guidance for decentralized clinical trials to advance medical product development and research.
Decentralized clinical trials use locations other than traditional clinical trial sites for some or all of the trial-related activities. That means patients who might have previously needed to travel to research sites — their doctor’s office, for example, or a university hospital — can instead participate in trials at home or closer to home.
This allows a broader, more diverse representation of the population to take part in experimental medicine, helping to reduce gender, racial or socioeconomic inequality in medical care.
“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 Dr. Robert Califf said in a news 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.”
What are decentralized clinical trials for?
FDA requirements are the same for decentralized clinical trials and traditional, site-based clinical trials. A trial sponsor’s responsibilities are also the same for both. The FDA still wants a physical location where all clinical trial-related records are stored and available for inspection, and where trial personnel can be interviewed.
Investigational products that are simple to administer or use, have well-characterized safety profiles, and do not require complex medical assessments might be good fits for fully decentralized trials.
But there’s also middle ground. Hybrid decentralized trials describe trials where patients participate at traditional sites for some activities, and can stay home or closer to home for others. For example, a patient might need to visit a clinical trial site for a complex medical assessment, but could submit follow-up assessments remotely using telehealth technology or in-home visits from local health care providers.
The FDA cautions that data collected remotely could vary from collections by dedicated trial personnel. “This would not affect the validity of a finding of superiority in a trial using such data (although it could reduce the effect size), but it could affect the validity of a finding of non-inferiority,” the guidance says. “… FDA review divisions should be consulted when planning a non-inferiority trial in a DCT setting.”
Tips for remote clinical trial activities
The trial protocol should specify when a participant should be seen in person versus via telehealth. “In general, investigators can consider telehealth visits instead of in-person visits with trial participants if no in-person interaction is needed,” the FDA says.
Trial personnel or local health care providers can be used for trial services at or near a participant’s home. Local providers shouldn’t need to have a detailed knowledge of the protocol or the investigational product, and shouldn’t perform services beyond their qualifications.
Remote trial visits should include confirmation of a participant’s identity. FDA doesn’t endorse a particular method, but suggested the National Institute of Standards and Technology’s Digital Identity Guidelines.
The trial protocol should also say how to evaluate and manage adverse events that are identified remotely, including “how care will be provided for adverse events that require urgent or in-person attention.”
Digital health tools can enable remote data collection, and the FDA’s draft guidance on that topic offers recommendations. Trial sponsors should ensure that those tools are available and suitable for all trial participants. For example, an app used on a participant’s personal smartphone might exclude participants who can’t afford to buy such a device.
Trial population diversity and inclusiveness should be a goal for trial sponsors, the FDA said. The agency recommended outreach through local healthcare institutions such as pharmacies and clinics. Remote trial activities can reduce the need for travel, reducing the financial and time burden for participants and increasing engagement, recruitment and retention. Using local health care providers can do the same, while also reducing cultural and linguistic barriers to trial participation.
Safety monitoring in decentralized clinical trials
Decentralized trials for medical devices require additional considerations for safety. A safety monitoring plan should ensure adverse events are captured and addressed. Additionally, the plan should determine ahead of time if and when telehealth and in-person visits will be scheduled to collet safety data.
The safety monitoring plan should describe how trial participants are expected to respond to adverse events and how to report them. They need ways to contact trial personnel about adverse events, have questions answered, and arrange for an unscheduled visit if needed.
“If significant safety risks emerge because of the remote administration or use of an IP, sponsors must discontinue remote administration or use; notify FDA, the IRB, and all investigators who have participated in the trial; and determine if the trial should continue,” the FDA says.
More from the FDA
The FDA’s draft guidance for decentralized clinical trials also includes recommendations on data management plans, trial monitoring plans, delegation of trial-related activities, and software considerations.