Diseases are singularly defined but often represent varied groupings of contributing factors and pathways that ultimately produce a shared pathology. Heterogeneous molecular pathogenesis creates heterogeneity in treatment responsiveness, which in turn drives up clinical trial size, timing, cost, and failure rates. For agents that ultimately achieve FDA approval, generally 40% of patients in a trial are found to be “responders”, with over half or more of all enrolled participants having minimal or no therapy response. Without strong understanding of underlying disease biology, a wide net must be cast to ensure inclusion of enough individuals likely to benefit from treatment.
In this session, we will discuss how circulating biomarkers can be used to enrich clinical trials by stratifying patient populations according to disease pathways and pathogenesis. We will provide examples in which these biomarkers have been applied to drive smaller, faster, and more successful clinical trials, as well as to prevent potential non-responder patients from being exposed to unnecessary drugs. Ultimately these approaches enable trials to align the right patient with the right disease pathway and the most effective therapy.
Learning Objectives:
- Learn the driving causes of large, costly, and time-consuming clinical trials, and why drugs approved through these studies may be ineffective for as much as two-thirds of patients.
- Explore how biomarker-driven clinical trial enrichment strategies can reduce trial size by as much as ten-fold by helping to align patients, disease biology, and specific therapies.
- See provided examples of how circulating biomarkers are being discovered and applied to enrich clinical trials for sponsor and patient benefits.