A new CTTI publication, published in Digital Biomarkers, investigates the challenges sponsors encounter when using digitally derived endpoints and offers potential solutions. Digital health technologies – including mobile apps, smart devices, sensors, and wearables – can capture robust data from participants’ daily lives. These digital tools allow researchers to collect data for novel endpoints that can provide a broader view of health and treatment effects using measures that matter to patients. Despite these benefits to clinical trial quality and efficiency, digitally derived endpoints are still underutilized as primary evidence in regulatory submissions.

To evaluate the barriers and challenges associated with digitally derived endpoints to support labeling claims, CTTI interviewed 20 sponsor representatives from 10 different organizations representing 11 individual clinical trials. During these interviews, conducted between November 2020 and March 2021, sponsor representatives were asked about their experiences related to digitally derived endpoints, their interactions with regulators, and the challenges they encountered. Based on these interviews, CTTI identified five key challenges to incorporating digitally derived endpoints in clinical trials:

1. a lack of regulatory guidance specific to digitally derived endpoints;
2. a qualification process for new clinical outcomes that is impractical for most industry sponsors;
3. a lack of comparable clinical endpoints to validate novel digitally derived endpoints;
4. a lack of validated DHTs and algorithms for a concept of interest; and
5. a lack of operational support from technology vendors.

CTTI shared these interview findings with the U.S. Food & Drug Administration (FDA) and the European Medicines Agency (EMA) and during a multi-stakeholder expert meeting. Through these discussions, CTTI drafted and refined a set of recommendations and tools for developing novel digitally derived endpoints as part of the Digital Health Trials Hub. These resources provide a robust toolkit for sponsors, investigators, and operational partners navigating the regulatory landscape around digitally derived endpoints.