Patient-prioritized primary endpoints in clinical trials.

Abstract

In the 1940s the randomized controlled trial (RCT) ushered a new era of clinical research and has been a cornerstone in the improvement of treatments [1]. Since then we have to a large extend only applied single or un-weighted composite endpoints as outcome measures. Increasing demands for involvement of patients in design of research – not least from funding institutions and regulatory authorities – may markedly change future designs of clinical trials. Currently less than 1% of trials engage patients actively and meaningfully [2]. We may approach new challenges demanding a more holistic assessment with patient-prioritized endpoints, e.g. quality of life, side effects, costs, follow-up burden etc. to be integrated in primary endpoints.