More Thorough, Inclusive Clinical Research Is Needed to Support PDTs, Study Finds

FDA-authorized prescription digital therapeutics (PDTs) often lack rigorous standards of evidence for the patients with conditions or diagnoses being treated, suggesting a need for a more thorough and inclusive approach to clinical research supporting the use of these therapies.

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“Given the nascent policy landscape for these technologies, as well as the potential for increased coverage of their use, we sought to identify emerging trends in the clinical evidence generated on prescription digital therapeutics authorized by the FDA for use in the US,” wrote the researchers of the study.

To the researchers’ knowledge, this retrospective cross-sectional analysis, published in Health Affairs, is the first to evaluate the use of clinical studies of PDTs that have been authorized by the FDA.

PDTs are digital health tools that primarily rely on software to diagnose and treat patients with a diverse set of medical conditions. However, uncertainty on the clinical and cost effectiveness of these tools has resulted in limited clinical evidence generalizability and coverage.

Because there is no central classification system for these technologies, the researchers used multiple data sources, including PDTs from publicly available formularies of the 2 largest pharmacy benefit managers in the United States, Express Scripts and CVS Caremark. Additionally, the researchers obtained data from a product library created by the Digital Therapeutics Alliance and FDA product codes.

The researchers included characteristics of all clinical studies associated with PDTs as of November 29, 2022. Prescription characteristics included FDA authorization pathway, authorization year, FDA Breakthrough Devices Program designation, and indications. Data on clinical characteristics of each study included information on premarket vs postmarket timing, study type, comparator status, use of blinding, number and location of study sites, and funding source. Additionally, the researchers abstracted data on language proficiency requirements and age selection criteria for study participants.

A total of 20 PDTs and 117 clinical studies were included in the analysis, with a median (IQR) of 5.5 (3.8-6.0) studies per PDT. In these studies, there were 179 primary end points, including 149 clinical outcomes and 21 nonclinical outcomes. Of the 119 end points from studies that were completed or terminated, 89 were met, 11 were not met, and the status of 19 was unclear due to lack of publicly available data.

Only 2 of these PDTs had been evaluated in at least 1 study that was randomized and blinded and used other rigorous standards of evidence. Additionally, two-thirds of clinical studies were conducted on a postmarket basis, with less rigorous standards of evidence used compared with premarket studies.

Furthermore, more than half of these studies did not include report data on the participants’ race, and more than 80% did not report their ethnicity.

Lastly, the researchers found that more than one-third of studies required the participant be proficient in English, and 46.9% of the studies that did not involve children had an upper age limit with a median (IQR) of 75 (65-85) years.

“Exclusion of older adults and people not proficient in English from clinical studies of prescription digital therapeutics should be ended, and diverse populations should be enrolled,” wrote the researchers. “These steps to improve study rigor and generalizability are needed to better inform payer coverage and clinical adoption.”

Reference

Kumar A, Ross J, Patel N, et al. Studies of prescription digital therapeutics often lack rigor and inclusivity. Health Aff (Millwood). Published November 6, 2023. doi:10.1377/hlthaff.2023.00384