Drug Industry Groups Ask FDA for Clearer Rules on Digital Health Tools in Trials

Pharmaceutical and digital health industry groups have asked the U.S. Food and Drug Administration to say more clearly how it will treat digital health technologies used in clinical trials, and to allow more flexibility in how sponsors validate them, according to comments described by Regulatory Focus, published by the Regulatory Affairs Professionals Society (RAPS), on June 8.

The comments respond to a request for information the FDA issued in March, asking what opportunities and challenges stakeholders see in using digital health technologies, or DHTs, to develop medical products. The agency said the feedback would be used to develop guidance and inform its decision-making, according to RAPS. DHTs cover a wide range of software and connected products, including sensors in wearable, implantable and ingestible devices.

What the drug and biotech groups asked for

The Pharmaceutical Research and Manufacturers of America (PhRMA) argued that there has been limited progress toward using DHTs to inform regulatory decisions, and that DHT-derived endpoints have not been used as primary or secondary trial endpoints to the extent anticipated, RAPS reported. The group asked for a clear, flexible and risk-based approach to fit-for-purpose DHTs, practical tools such as templates and decision trees, clarity on how to manage changes such as software updates, and harmonization across FDA product centers and with other drug regulators.

The Biotechnology Innovation Organization (BIO) said its members see unclear evidentiary expectations for DHT-derived endpoints, verification and validation requirements, and choice of regulatory pathway as the most significant barriers. It also cited a lack of cross-center consistency and of public information about FDA's internal DHT reviews.

"BIO members also highlight the need for practical tools, such as risk‑tiered templates, case studies, decision trees, and examples to support consistent implementation," the group said.

The Combination Products Coalition asked that the FDA's Digital Health Center of Excellence serve as a formal cross-center coordination body for DHT matters, and proposed a master file-type approach that would let third-party technology developers keep centralized documentation on DHT and AI tool performance that sponsors could reference across programs.

ATA Action letter

ATA Action filed a comment letter dated May 29, posted on the American Telemedicine Association website, to docket FDA-2026-N-2476, titled Advancing the Use of Digital Health Technologies in Clinical Investigations for Drugs and Biological Products.

The letter asks for consistent and predictable expectations across the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research and, where relevant, the Center for Devices and Radiological Health. It says variability in expectations can introduce uncertainty for sponsors, potentially limiting investment. ATA Action also asked for additional guidance on digitally derived endpoints, including how they are validated, where they sit among primary, secondary and exploratory endpoints, and how to handle missing data and real-world variability in decentralized or hybrid trials.

The FDA has not said when it will issue guidance based on the responses.