Decoding FDA Guidance on Decentralized Trials

By MedTech Intelligence Staff
June 21, 2023


• Categories: 01-Digital Health, 05-Quality/Regulatory, Clinical Trials, Market Access, Remote Patient Monitoring

Last month the FDA released its draft guidance on the use of decentralized clinical trials (DCTs) for drug and device development. We spoke with Chris Fourment, MD, Senior Vice President, Clinical Strategy at Iterative Health, who specializes in structuring clinical trial programs, about the new guidance. He shares the opportunities for full and hybrid DCTs and the challenges that remain in designing reproducible DCTs.

Did the recent guidance from the FDA help answer the questions the industry was facing in terms of using DCTs for drugs or device approval, or are there still some big questions out there that need to be answered? 

Fourment: It’s absolutely a step in the right direction. DCTs have been a hot button topic in research for at least the last five years, but there really has been no guidance to date. Sites, investigators and sponsors have been trying to figure it out on their own. So, this is a major step.

There’s a lot of interpretation that needs to be done. The FDA is really good at setting the guidelines, but not the exact details. This is good because it gives sponsors the ability to look at their protocols and decide what might be appropriate to decentralize, and then decide how to decentralize that piece or pieces of the trial.

For clinical trial sites, the draft guidance allows us to help sponsors guide their new protocols to find populations that are underserved. And instead of trying to bring the patients to the trial, we’re going to be able to bring the trial to the patients. Using that approach, I hope that we’re able to shorten the length of the clinical trials and find more patients that reflect the diversity that we have in the U.S.

Are there examples out there where you’ve seen DCT designs that really worked well?

Fourment: In the gastroenterology space, the work that we do on blood draw studies for colorectal cancer protection is an example of a trial that can pretty easily be decentralized. Sponsors that have made greater strides toward DCT are those that have worked with phlebotomy partners who offer mobile services, so they go to a patient’s home or to the patient’s work for the blood draw. Those sorts of DCT approaches certainly are working well and a step in the right direction. But many of the trials in gastroenterology are difficult to fully decentralize.

For example, when we talk about inflammatory bowel disease which includes Crohn’s and ulcerative colitis in which we have a major procedure—the colonoscopy—that must be done as one of the clinical trial endpoints, we have found no good way to bring that procedure to the patient. However, the guidance that the FDA has given us includes the potential for a hybrid approach, which could be incredibly valuable in the IBD space. We could take some portions of that clinical trial to the patient and still require them to come in for the colonoscopy.

The other thing that is valuable for this population is that the FDA has allowed use of healthcare providers (HCPs), even outside of the clinical trial per se. You don’t have to be a direct investigator in that clinical study. The HCP could do some of the more straightforward procedures, things like physical exams or vitals, and this could be done off-site, which makes it a lot easier for the patient.

Do you foresee any challenges in enlisting HCPs to take part in DCTs?

Fourment: One potential challenge is compensation. You are taking up the physician’s time, so how do you compensate them effectively? The FDA offers some guidance in their document that will allow us to pay a reasonable rate to the HCPs that are performing those activities. So we can take that challenge off the table.

Another challenge the FDA has taken away is how to list that provider’s involvement in the clinical trial in terms of documentation and regulatory requirements. The draft guidance suggests that if a person is performing a physical exam, they wouldn’t necessarily be part of the 1572; they would be on a task log where the investigator would delegate that ticket or that task to that person. From a regulatory perspective, that makes things much easier.

Normally, the HCPs taking part in clinical trials have to be trained in the protocol and often trained on the investigational product or treatment that’s going to be delivered. Allowing a smoother route for these local HCPs to be involved is a huge advantage.

Clinical trials are very controlled in terms of subject enrollment, how will this guidance and the use of local HCPs affect subject screening and enrollment?

Fourment: The principal investigator is still going to be in charge of determining whether or not a patient qualifies for the trial. So in the sense of working with local HCPs, that person is not going to have any role in determining the patient’s eligibility for the clinical trial.

I do think this draft guidance will fuel sponsors and sites to do more DCTs, which will lead to better access to more disparate patient populations that we have not had access to before—that’s the reason I’m so excited about the guidance.

If you look at clinical trials in gastroenterology, we are starting to recognize that specifically within the IBD population we need more share of voice. The populations of patients that we have traditionally had in IBD studies have been Caucasian, and what we’re coming to recognize is that we are likely underrepresenting certain populations, which is a multifactorial challenge. Some of these populations that have been traditionally marginalized don’t have trust with clinical trials—that’s a barrier we still need to overcome. But for some, especially in the lower socioeconomic groups and sectors, there are a lot of challenges, such as lack of transportation or inability to take time off from work, that we can overcome with DCTs.

When we look at the technology and communication needed to perform a DCT, when working with more rural healthcare centers, for example, is this typically in place or do we need more Infrastructure to fuel widespread adoption?

Fourment: There is going to be some technology needed to fuel widespread adoption. The use of local HCPs could be a critical piece in overcoming this challenge. We also can think about digital health technology in terms of a sponsor sending the right technology to a patient’s home, and the patient uses that technology on their own. But we need to make sure the technology is standardized and that it is being used in the same way time over time.

When we think about clinical trials, the main thing the FDA is looking for is accuracy and consistency, and then at the end of the day it needs to be reproducible. We need to be able to put the right technology in the hands of the patient that’s going to make it easy for them to either self-administer or have a family member administer, and that has to be reproducible.

If we look at putting the leads on for an EKG, a family member might not necessarily put those leads on correctly and the EKG could be read incorrectly in the home. On the flip side, if we allow a local HCP to conduct some of these basic procedures, you don’t have to send that equipment to the patient’s home, but they can access someone right down the street.

There’s a lot that goes into finding clinical trial subjects. Most of the time the most effective method is to query your EMR, but that doesn’t provide access to folks who are either socioeconomically disadvantaged or aren’t yet on your schedule. I think the DCT design will force us to figure out new ways to get into underserved communities and make an impact in these communities.