Studies for labeling versus reimbursement

Abstract Studies designed for regulatory approval are necessary but not sufficient for many payer stakeholders. Beyond safety and efficacy data generated within regulatory-purposed randomized controlled trials, payers are concerned with whether the new technology works among more broadly defined ‘real world’ patients treated under more usual care settings and with comparators reflecting those utilized by their populations (i.e. effectiveness data). In addition, endpoints most important to payers go beyond those in regulatory-purposed trials and include healthcare resource utilization, direct medical care costs, health-related quality of life and increasingly a variety of quality metrics. The pragmatic randomized controlled trial (pRCT) offers a blend of the study rigor required for regulatory approval (e.g., randomization) with the concerns of payers (e.g., broader inclusion criteria, routine practice settings, cost-related endpoints). This chapter delves deeper into these issues and reviews why two recent pRCTs are significant to payers.