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FDA, NIH: Telehealth Key To Diversifying Trials, But Hurdles Remain

FDA, NIH: Telehealth Key To Diversifying Trials, But Hurdles Remain

FDA and NIH are recruiting clinical trial participants via telehealth in a bid to increase the number of enrollees of color and lower socioeconomic status, according to FDA and NIH. But an FDA official told Inside TeleHealth that the lack of interstate licensing infrastructure, poor access to broadband and gaps in regulatory guidance are slowing their efforts to use digital health technologies to increase diversity among trial enrollees.

NIH and FDA are scrambling to diversify the type of patients they recruit for clinical trials. Bioethics International applied its new Fair Inclusion score to rank 25 biopharma firms based on pivotal oncology drug trials conducted between 2012-2017 in a new study published in BMJ Medicine and concluded Black and Latinx are underrepresented in oncology trials. Only 10% of all 62 trials analyzed in the study met the benchmark for adequate racial representation, with especially poor inclusion of Black and Latinx patients.

Then National Institute of Allergy and Infectious Diseases Director Anthony Fauci told The Hill over two years ago that vaccine trials need to have a diverse representation of the population likely to use the treatment. Without greater diversity in clinical trials, it is difficult to ensure a treatment or vaccine is suitable for populations beyond white patients, he said.

Telehealth can be used whenever remote interviews are sufficient to acquire necessary information for recruiting and implementing clinical trials, the FDA official told Inside TeleHealth.

Having to travel to be screened in-person for a clinical trial puts an enormous burden on both the patient and caregiver — with some requiring patients to be in-person at a designated time and place on multiple occasions — for them to just be considered for a clinical trial.

The FDA official said telehealth may be more appealing to potential participants if they do not need to travel for initial assessments.

“It is possible telehealth could diversify recruitment by enabling access to patient populations who have difficulty visiting trial sites (e.g., patients with socioeconomic challenges who cannot take time off from work or have transportation challenges),” the FDA official said.

The Pharmaceutical Research and Manufacturers of America said using telehealth in clinical trial recruitment can also reduce geographical and logistical barriers by allowing patients to participate in studies locally, without the need to go to a major medical center.

“The opportunity to participate can be widely extended to patients who otherwise could not access certain physical enrollment locations or may have found participation inconvenient or burdensome,” PhRMA said. “This may allow further participation by populations that have traditionally been underrepresented in studies, such as people from certain racial and ethnic groups.”

Madhav Thambisetty, senior clinical investigator at the National Institute on Aging specializing in cognitive and behavioral neurology, said he is collaborating with a telehealth company Ro to recruit patients for NIH-sponsored trials after Ro tested whether erectile dysfunction medications can lower the risk of Alzheimer’s.

Thambisetty said job demands restrict access to clinical trials for people who may not be able to forgo job responsibilities or receive time off of work.

“For all of these reasons, we think that clinical trials typically have recruited from a very narrow segment of the population,” Thambisetty said.

Clinical trials that recruit and test patients via telehealth are classified as “decentralized clinical trials,” a representative from PhRMA explained. Decentralized clinical trials are where are some aspects traditionally completed in the clinic are performed outside of traditional clinical research centers like in a participant’s home or a local physician’s office using digital health technology to facilitate completion of the activities.

“America’s biopharmaceutical companies, working with the FDA, utilized telehealth and other digital technologies to enable continuity of clinical trials during the COVID pandemic,” PhRMA said. “We believe these learnings can be leveraged coming out of the pandemic to continue using digital technologies to conduct decentralized trials, removing certain barriers to clinical trial participation.”

Thambisetty said the use of telehealth and digital health technologies during the pandemic to continue clinical trials raised ethical questions on how studies were conducted before the expanded use of technology. Historically, trial participants have not represented the patient population that will use the treatment if approved.

“That raises questions about whether the safety data that were collected during the clinical trials are applicable to a generalized population in real world clinical situations,” Thambisetty said.

FDA’s controversial approval of Biogen’s Alzheimer’s drug aducanumab followed two large phase 3 clinical trials that recruited more than 3,200 patients in more than 120 counties worldwide, according to the NIH official. But there were only six Black patients who received the highest dose of the drug, which shows a huge disparity in the demographic characteristics of the patients recruited.

Thambisetty said the Alzheimer’s study is especially concerning because Black patients have a higher risk for Alzheimer’s disease as they have higher cardiovascular and cerebral vascular risk factors. Black patients could also be more prone to side effects of these medications, so to have a drug that was approved based on such few patients who might share the disproportionate burden of side effects is concerning, Thambisetty said.

Despite the potential for telehealth to diversify recruitment for clinical trials, persistent barriers remain to increased use of decentralized trials.

FDA said that current state licensing requirements present challenges for medical professionals conducting clinical trials using telehealth.

“They may limit the ability of health care professionals to conduct interstate telehealth visits,” the FDA official said. “State licensing requirements are outside the purview of the FDA.”

Additional barriers include patients not having proper technological systems or their inability to use such systems because of cognitive or physical limitations, according to FDA. A lack of broadband access for rural and underserved patients also has prevented patients from participating in recruiting programs that include the internet.

When asked whether any unique privacy issues arise when using telehealth to recruit participants for trials, FDA said that depends on how the telehealth visits are conducted and the technology used to support them. It is essential to implement appropriate electronic and procedural safeguards to protect participants’ privacy, according to FDA.

Privacy requirements currently differ depending on in which states the doctor and participant are located due to a lack of national data protection infrastructure. Despite the American Data Privacy and Protection Act (ADPPA) garnering bipartisan support on Capitol Hill, it has stalled and lawmakers have failed to enact federal data protections.

PhRMA said increased regulatory guidance also is needed to further use of telehealth and digital health technologies to diversify recruitment of participants. While FDA published draft guidance on Digital Health Technologies for Remote Data Acquisition in January 2022, PhRMA said there are still several areas where additional regulatory clarity would help facilitate the adoption of telehealth and digital health technologies in clinical trials.

“For example, additional guidance on the Agency’s expectations regarding the validation of digital health technologies used to collect data in clinical trials would help provide additional certainty to sponsors designing decentralized clinical trials,” PhRMA said.

FDA announced plans to publish draft guidance on decentralized clinical trials in 2022, according to PhRMA, which could provide additional recommendations on using health technologies to recruit for trials.

“Furthermore, outreach to communities through organizations with long-standing engagement with rural and underserved populations may help address technological access barriers, such as lack of broadband access, towards participation in clinical trials,” PhRMA said.

Thambisetty said there are several unanswered questions when it comes to identifying potential barriers for patients to participate in clinical trials. As he and his team start to screen and recruit patients, they will be able to identify specific challenges that the telehealth platform itself would face, but lack of broadband and digital literacy are likely to be barriers.

“I think there’s going to be an iterative learning process,” Thambisetty said. “We will be able to identify specific challenges and I think we’ll also be able to be addressing these challenges in real time as the study progresses.” — Cara Smith (