Clinical Trial Design, Endpoints, and Regulatory Requirements.

ABSTRACT

A new therapeutic agent for heart failure can be approved if it improves survival and/or reduces hospitalisations or if it safely improves functional capacity. Therefore its clinical development program must demonstrate clinically relevant improvement in a robust clinical end point and adequate safety to justify regulatory approval and clinical use. Mortality and hospitalisations are now combined with new composite end points in order to improve trial efficiency and adequate assessment of efficacy of newer molecules, biologicals and cell therapies developed for the treatment of heart failure. Newer regulatory practices have been developed in the past few years and they will require design of innovative study designs able to demonstrate a sound clinical benefit alongside with adequate safety profile.