What is the expected benefit of patient-centric clinical development in oncology?

The identification and quantification of predictive biomarkers characterize personalized medicine approaches and patient-centric clinical development. In practice, the sponsor needs evaluating whether biomarker-informed clinical development strategies are more likely to benefit current and future patients. To this end, a simple metric is proposed and assessed here quantifying the expected clinical benefit (ECB) of clinical development programmes. Using simulation scenarios and endpoints relevant to oncology, the ECB of a simple biomarker-informed strategy is shown to be specific and sensitive. Also, the ECB difference is shown to increase in the biomarker-driven incremental efficacy and with the population prevalence of biomarker-positive study participants.

Automated summary

The identification and quantification of predictive biomarkers play a crucial role in personalized medicine and patient-centric clinical development. This study proposes a simple metric, called expected clinical benefit (ECB), to evaluate the clinical benefit of biomarker-informed clinical development programs. The results show that the ECB of biomarker-driven strategies is specific and sensitive, and the difference in ECB increases with the incremental efficacy driven by biomarkers and the population prevalence of biomarker-positive study participants.