More thorough clinical studies needed to support PDT, study finds

FDA-authorized prescription digital therapies (PDTs) often lack rigorous evidence standards specific to the condition or diagnosis of the patient being treated, suggesting the need for more thorough and inclusive clinical research approaches to support the use of these therapies.

“Given the emerging policy environment for these technologies and the potential for expanding coverage of their use, we sought to identify emerging trends in clinical evidence generated by the FDA authorizing prescription digital therapeutics for use in the United States,” the report reads. Researchers of the study.

To the best of the researchers’ knowledge, this retrospective cross-sectional analysis was published in health affairs

is the first to evaluate the use of PDT in clinical studies that has received FDA authorization.

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

Because there is no central classification system for these technologies, the researchers used multiple data sources, including PDTs from the open formularies of Express Scripts and CVS Caremark, two of the largest pharmacy benefit managers in the United States. In addition, the researchers obtained data from product libraries created by the Digital Therapeutics Alliance and the FDA Product Code.

Researchers included characteristics of all clinical studies related to PDT as of November 29, 2022. Formulary characteristics include FDA authorization pathway, authorization year, FDA Breakthrough Device Program designation, and indication. Clinical characteristics data for each study included information such as premarketing versus postmarketing time, study type, comparator status, use of blinding, number and location of study sites, and funding sources. In addition, the researchers extracted 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. Among these studies, there were 179 primary endpoints, including 149 clinical outcomes and 21 nonclinical outcomes. Of the 119 study endpoints that were completed or terminated, 89 were met, 11 were not met, and the status of 19 is unclear due to a lack of published data.

Only 2 of these PDTs were evaluated in at least 1 randomized, blinded study and used other stringent evidence standards. Additionally, two-thirds of clinical studies are conducted post-marketing, using less stringent standards of evidence than pre-marketing studies.

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

Finally, the researchers found that more than one-third of the studies required participants to be proficient in English, while 46.9% of studies not involving children had an upper age limit, with a median (IQR) of 75 (65-85) years.

“The exclusion of older adults and those not proficient in English from clinical studies of prescribed digital therapies should be ended, and diverse populations should be recruited,” the researchers wrote. “These measures are needed to increase the rigor and generalizability of studies nature to improve reporting payer coverage and clinical adoption.”

refer to

Kumar A, Ross J, Patel N, et al. Research on prescribing digital therapeutics often lacks rigor and inclusivity. Department of Health Affairs (Millwood). Published on November 6, 2023. doi:10.1377/hlthaff.2023.00384