Methods for planning and analyzing clinical trials with composite endpoints

• Applicant: Professor Dr. Heiko Becher, since 7/2017
• Subject Area: Epidemiology and Medical Biometry/Statistics
• Term: from 2013 to 2019
• Project identifier: Deutsche Forschungsgemeinschaft (DFG) - Project number 234625736

Composite endpoints combine several events of interest within a single variable. They are commonly used if the efficacy of the intervention under investigation cannot directly be assessed by a single specific outcome. Moreover, using a composite measure instead of a single event variable increases the number of expected events and is thus meant to increase the power. In many clinical applications, the events of interest are relatively rare which is problematical from a statistical point of view, as in such cases a high number of patients is needed to conduct the study with adequate power. Here, a composite endpoint might be a solution.However, the combination of several event variables, which often are of different clinical relevance, can also result in serious problems concerning the calculation of an adequate sample size and a valid clinical interpretation of the results. The aim of this project is thus to develop statistical methods which maintain the advantages of composite endpoints but allow a valid planning of sample size and a meaningful result interpretation. As composite endpoints are routinely used in many clinical indications, for example in the field of cardiology and oncology, the topic of this project is very relevant and highly important for application in clinical trials.

• DFG Programme: Research Grants
• Ehemalige Antragstellerin: Professorin Dr. Geraldine Rauch, Ph.D., until 6/2017