ISSUE: New opportunities for patient engagement in value assessment and regulatory activities have revealed insights on which outcomes are important to diverse patients. Decision-makers are increasingly aware of the incongruity between representativeness of those enrolled, what matters to patients, and data collected in clinical trials. In parallel, real-world evidence (RWE) has attained a larger role in drug development and value assessment, with examples of RWE being used to support regulatory approval and measure treatment outcomes most important to patients.

Despite the volume of data and broad recognition of the critical role that real world data (RWD) can play in supplementing evidence from clinical trials, the extent to which the US data infrastructure supports analyses of diverse populations on patient-centered outcomes is unclear. To answer this question, a closer look at the current state of RWD generation and the infrastructure that supports its collection and dissemination may be appropriate.

OVERVIEW: The panel will open with introductions and discussion of the extent to which the current data infrastructure may or may not facilitate a crucial next step in patient-focused drug development (panelist 1), learning how diseases and drugs affect diverse patient populations (panelist 2), and measuring outcomes that matter to patients (panelist 3) (10min each; 30min total). The panel will then debate what investments and improvements are needed to ensure a data infrastructure in the US that can generate evidence that is both germane and of sufficient quality to support drug development and reimbursement for diverse patient populations (20 min). The panel will close with audience polling on several key questions and audience questions for the panel (10 min). Stakeholders interested in use of RWD to support drug development, including industry, RWD vendors, regulators, and public and private payers should attend.