Clinical Trials Transformation Initiative releases guidelines on mobile-derived novel endpoints

By Jonah Comstock
Share

The Clinical Trials Transformation Initiative (CTTI), a public-private partnership of pharma companies, academics, and regulators, including FDA, looking to create a new “gold standard” of clinical trial design using digital technology, released new endpoint recommendations today focused on the use of mobile technology in clinical trials. 

“By engaging with experts who have been early champions of mobile technology in trials and combining that with patient insights, CTTI has created practical recommendations and action-oriented tools that have the potential to really accelerate the use of mobile technology in clinical trials,” Craig Lipset, Pfizer’s head of clinical innovation, said in a statement. “In particular, the use cases provide a realistic pathway for incorporating novel endpoints through technology into clinical development programs. CTTI’s recommendations show we may be closer than previously believed to realizing the benefits of these novel endpoints, creating a sense of urgency to act.”

Mobile devices have a lot of potential to enhance clinical trials, by capturing more complete data about individuals outside the clinic. But those devices open up a wide range of possible endpoints to measure in a trial. CTTI has set out to help organizations choose valuable outcomes measures when designing trials.

“Technology-derived endpoints offer the benefit of capturing information about patients’ experience in ‘real-world’ settings,” Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, said in a statement. “These tools have the potential to capture data that can be used to develop endpoints and evaluate therapies in the patient population.”

The guidelines, meant to be the first in a series of documents supporting the use of mobile devices in clinical trials, include two sections: recommendations for selecting novel endpoints and practical approaches to developing endpoints. 

To select novel endpoints, CTTI recommends first that organizations focus on measures that are meaningful to patients and involve patients in the process. Importantly, they shouldn’t use novel endpoints merely because they’re novel.

“Subsequent selection of a novel endpoint in preference to an existing outcome assessment should only occur if the novel endpoint offers incremental utility,” the guidelines state. “Specifically, the mobile technology-derived assessment should be better than the alternative measure(s) in one or more ways: for example, the measure should be more informative or meaningful to patients. CTTI strongly cautions against developing novel endpoints simply because new mobile technology makes it technically feasible.”

Similarly, CTTI also suggests that clinical trial organizers select the endpoint before selecting the mobile device to use, and that they use a systematic approach when developing new endpoints.

The advice for developing novel endpoints includes some fairly straightforward suggestions: Work with a wide range of stakeholders, engage early on with regulators, and create technical standards for endpoints derived from mobile technology. Also, CTTI suggests including novel endpoints as exploratory endpoints in existing trials with an established endpoint before testing them as the primary endpoint.

Finally, CTTI suggests that organizers think carefully about the role mobile technology-enabled endpoints can play in clinical trials and future drug and therapy development.

“Novel endpoints are unique in their ability to objectively capture data about patients outside of the clinic in the context of their activities of daily living,” CTTI writes. “As such, novel endpoints may offer valuable data in support of labeling claims for new therapies. Similarly, novel endpoint data may provide evidence to support the roles of other stakeholders in the healthcare ecosystem, including providers and payers who make coverage decisions related to the use of medical products.”