Clinically Meaningful, Patient-Centered Endpoints for Real-World Evidence Generation in Breast Cancer: Perspectives from the Multidisciplinary TRIUMPH Initiative.

Stakeholders have made major advances in generation of real-world evidence (RWE); however, core challenges remain, limiting its value and utility in guiding treatment decision-making. In particular, persisting lack of industry consensus on standardized, clinically meaningful, patient-centered endpoints was considered a critical area of interest for the multidisciplinary Think tank on RWE In the US from Multiple Perspectives in Healthcare (TRIUMPH). Collaborating in a group forum, TRIUMPH assembled and reviewed a first draft of potential endpoints that may be more consistently incorporated into RWE research. Among this list, time to next treatment, adherence/persistence, psychosocial well-being, and frailty were prioritized for subsequent discussion on ways to enable their broader adoption. Across the four endpoints, key opportunities for improving adoption included additional education for researchers, standardization of algorithms, and exploration of new approaches for derivation in real-world data. Over the course of discussion, TRIUMPH also identified frailty and area/neighborhood socioeconomic status as key baseline characteristics that may guide stratified analyses of the prioritized endpoints, to increase patient-centric value of the resulting RWE. In the future, other endpoints and characteristics (beyond those in focus for TRIUMPH) will also likely emerge, and should similarly be incorporated into routine care and research, to continue growing the utility of RWE in better informing treatment decision-making.