CTTI Holds Meeting to Advance Novel, Digitally Derived Endpoints in Clinical Trials

Despite the increased use of digital health technologies (DHTs) in trials over the past several years, few examples of a digitally-derived primary endpoint exist today. Working to fill this gap, CTTI held an “Obtaining Novel Endpoint Reliability & Acceptance” Expert Meeting on July 27-28 to inform its new recommendations and resources for driving novel endpoint acceptance.

Attendees discussed challenges and potential solutions to advancing the practical use of novel, digitally derived endpoints in clinical trials. They also uncovered and agreed on some important common themes:

• The Time is Now. Digitally derived endpoints have the ability to capture information that is more reflective of how patients feel and function in their day-to-day lives – but many factors hinder their acceptance. Collaborative partnerships and solutions that increase the understanding and use of DHT-derived endpoints are needed to make this happen.
• Fit-for-Purpose is a Must. DHT-derived endpoints need to be based on the specific context of use and validly measure a concept of interest in a way that is accurate, interpretable, and not misleading.
• “Meaningful” in More than One Way. To develop a DHT-derived endpoint, have the community – including patients and clinicians – discuss what constitutes “meaningful” change. A meaningful measure should show treatment benefit as well as clinical benefit.
• Engage Early and Often. Engage patients, investigative site personnel, and the FDA early and often when planning; involve biostatisticians and data scientists, as appropriate, in decisions regarding protocol design, data collection, analysis, and interpretation
• Validate, Validate, Validate. It’s important to remember that validating a device is a separate, yet parallel, process from validating the clinical measure. It is also especially important to demonstrate analytical validation of DHT algorithms within the specific patient populations.

This meeting and its findings, part of CTTI’s Novel Endpoint Acceptance project, will help expand CTTI’s 2017 Novel Endpoint work, which produced a set of recommendations, flowchart, detailed steps, and many other resources for developing novel endpoints.

For a preview of CTTI’s expanded set of novel endpoints recommendations and resources, register to attend its Oct. 5 “Next Steps for Obtaining Novel Endpoint Reliability & Acceptance” webinar.