DiMe Journal Club: Regulatory Pathways for Qualification and Acceptance of Digital Health Technology-Derived Clinical Trial Endpoints: Considerations for Sponsors
Date/Time
Discussion Topic
Despite widespread interest and substantial investment in the adoption of sensor-based digital health technologies (sDHTs) for remote data capture in drug development trials, no drug has been approved based on an sDHT-derived primary endpoint in the United States (US). One reason for this lack of advancement is the complexity of obtaining regulatory endorsement for those endpoints within current US regulatory pathways. The goal of our review is to describe the two choices currently available to pharmaceutical study Sponsors: (i) they may navigate the traditional route of compiling the evidence to support the sDHT-derived endpoint in their investigational new drug (IND) application, requiring specific expertise and substantial resources; or (ii) they may navigate the drug development tool (DDT) pathway with the goal of qualifying their sDHT-derived endpoint as a biomarker or clinical outcome assessment applicable to a broader context of use (COU), either alone or as part of a partnership or consortium. We describe the nuances of each pathway; the evidentiary requirements for supporting an sDHT-derived endpoint and the technology used to capture it; and the impact that an sDHT’s regulatory status may have on a Sponsor’s decision to use it for data capture. By systematically comparing the IND and DDT pathways, our over-arching goals are to support the increasing deployment of sDHTs within the clinical research setting and help advance regulatory science in the field of digital medicine.
Takeaway messages:
Sponsors considering incorporating a digital endpoint into a drug development pipeline may pursue the following regulatory pathways in the United States: 1) measure qualification via the Drug Development Tool (DDT) program; or 2) measure acceptance via the Investigational New Drug (IND) pathway. Multiple considerations may influence this critical decision, including drug development timelines, the context of use, risk sharing, and the need for partnerships or consortia.
Regardless of the pathway, the evidentiary package supporting a digital endpoint includes key elements to ensure the data are accurate, reliable, and appropriate for the specified context of use. Additional evidence is required to support the classification of the measure as a biomarker versus a clinical outcome assessment.
Sponsors may select a digital health technology that is A) a regulated medical device; B) a data capture product developed specifically for research purposes; or C) a low-risk general wellness product. While there is no requirement to use a product with a medical device designation for data collection in clinical trials, the scope of evidence that a Sponsor must compile to support their choice may vary depending on the technology category in addition to the context of use.
Watch to learn about the pros and cons of these decisions within the context of United States regulations.
