Definition of biomarkers and efficacy end-points

Nearly a third of the medicines in development today have some form of a genomic or proteomic marker. There has been a rapid increase over recent years.(1)

This varies across disease areas, however cancer (oncology) research was one of the first areas where the use of such biomarkers was adopted.

• Biomarkers are being used to make exploratory trials of medicines more efficient. Exploratory trials are early trials/Phase I trials.
• Only a limited number of biomarkers can be used as a substitute for a clinical endpoint in a confirmatory trial. Confirmatory trials include late stage trials/Phase III. Biomarkers may however be used in late stage trials in combination with clinical endpoints.
• For medicines that are being designed for specific molecular targets, only a sub-group of patients might respond. It is important to identify these patients for clinical trials, using biomarker measures.

(1) Evers M, Kulkarni S, Ma P, Moller M, Ostojic I. Managing for success in biomarker R&D: Challenges and opportunities. In Personalized Medicine The Path Forward. McKinsey and Company (2013).