Frontiers in Digital Technologies, When the New Approach Exceeds the “Gold Standard”

Regulators have signaled increasing openness to the use of digital tools in clinical trials to serve as surrogate endpoints. At the same time, clinical validity of these tools is typically measured against a current “gold standard.” In instances where novel, digital biomarkers generate more in depth genotypic or phenotypic characterization of disease, this comparison may result in discordance, stemming from poorer performance of the traditional clinically accepted measure. . For example, a patient wearing a continuous tracker to assess the effects of a medicine on osteoarthritis associated inactivity will give a far better characterization of drug effect than would a patient assessed every 2 months on a 6-minute walk test. Continuous monitoring is less likely to skew performance by daily variability and motivation issues associated with intermittent clinical visits. In our discussion we would like to explore how sponsors might approach regulators to gain their acceptance of novel, digital endpoints without imposing unnecessarily onerous obligations. In addition, we could discuss how the improved insights these tools could offer can benefit patients and lead to more detail and meaningful results being included in labeling. We would think to include a legal and policy angle to this discussion, as well as a project-based perspective leveraging internal expertise. We would seek to couple that with insights from technical development representatives and regulators.