The Clinical Trials Transformation Initiative (CTTI), a public-private partnership established by Duke University and the US Food and Drug Administration (FDA), on Monday released new recommendations on developing novel clinical trial endpoints generated by mobile technology.
Such mobile technology can offer new ways to collect objective measurements of clinical trial participants as they go about their daily lives.
And though the use of such novel endpoints, which the partnership defines as: "new endpoints that have not previously been possible to assess," or "existing endpoints that can be measured in new and possibly better ways," has significant potential, substantial development "will be required to facilitate the use of mobile technology-derived novel endpoints to support regulatory approval and labeling claims, as the pathway for acceptance is not currently well-described."
"Technology-derived endpoints offer the benefit of capturing information about patients’ experience in ‘real-world’ settings," said Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research. "These tools have the potential to capture data that can be used to develop endpoints and evaluate therapies in the patient population."
CTTI offers three recommendations for optimizing novel endpoint selection and five recommendations on practical approaches to the novel endpoint development process.
In terms of optimization, CTTI recommends companies focus on measures that are meaningful to patients, select a suitable mobile device after selecting an outcome assessment and use a systematic approach to identify such novel endpoints as it can be a time-consuming and resource-intensive process.
CTTI also recommends "establishing industry-wide standards related to 1) terminology, 2) the collection and reporting of data captured by mobile devices, and 3) transparency requirements for the algorithms used to convert the data into physiologically and medically useful endpoints."
The partnership also encourages drug and medical device sponsors seeking to use a novel endpoint in a pivotal trial to interact with FDA during the Investigational New Drug (IND) application or Investigational Device Exemption (IDE) reviews.
"Notwithstanding the need for thoughtful selection and standards across measures, sponsors and academic investigators should add mobile technology-derived measures to existing studies and trials to determine which and how novel endpoints offer incremental utility over other assessments. CTTI recommends including novel endpoints as exploratory endpoints in existing studies in preference to solely conducting unique pilot studies," the recommendations say.
CTTI RECOMMENDATIONS: DEVELOPING NOVEL ENDPOINTS GENERATED BY MOBILE TECHNOLOGY FOR USE IN CLINICAL TRIALS