77 – Complex Designs and Composite Endpoints of Medical Device Clinical Studies and Benefit-Risk Analysis of Diagnostic Tests
Survey of Composite Endpoints in Therapeutic Premarket Approval (PMA) Submissions
Ja-An Lin
U.S. Food and Drug Administration
Rajesh Nair
U.S. Food and Drug Administration
Natasha Sahr
St. Jude Children's Research Hospital
A composite clinical endpoint combines the most relevant clinical endpoints for assessing the effectiveness and safety of a device and combines them into a single endpoint. However, composite endpoints often lead to difficulties in the interpretation of results. In general, effects on the composite could be driven by the effects on the components as well as any differences in the correlation of the component endpoints between the treatment groups. Difficulties in interpretation arise when the results on single components of the composite endpoint go in opposite directions, and when hard clinical outcomes are combined with soft endpoints, particularly if the latter occur much more frequently but are of inferior relevance. In this paper, we present the results of a survey of the clinical studies with composite endpoints used to support medical device PMA approval from 2007–2016 at CDRH.A goal of the survey was to develop good practice guidelines for reporting results of studies with composite endpoints.