Session (CTI)

ISSUE: Real-world evidence (RWE) is increasingly evaluated for regulatory and reimbursement decisions regarding medical devices. While numerous RWE guidance documents have been published, what constitutes “fit-for-purpose (FFP)” or “fitness for use” of real-world data (RWD) in generating RWE to inform decisions remains uncertain. Moreover, most guidance fails to differentiate between RWE for pharmaceuticals and devices. There are differences in what can be observed between pharmaceuticals and devices in RWD and in the decision making due to varying regulatory pathways and benefit\risk and value assessments. There are also differences in ability to demonstrate data is FFP by device type and data source. Often, devices are only identifiable at a class level in claims data; clinical information may be missing even within EHRs; privacy considerations may limit data validation. However, these data are valuable for RWE generation for numerous reasons like sample size and follow-up duration. This session will compare regulator, HTA, and US payers’ perspectives on demonstrating secondary RWD are FFP for regulatory and payer decision-making for devices. OVERVIEW: The moderator will summarize the issue and provide an overview of secondary RWD for devices. (8 mins) Each panelist will present their perspective on the use of secondary RWD and discuss how study designs and analytical methods offer solutions to challenges posed by secondary RWD, including machine learning and natural language processing; data linking that maintains deidentification; and identification strategies leveraging institutional context. They will also offer perspectives of regulators, HTAs, and payers to these proposed solutions and recommend analysis planning and design approaches and future directions for methods research and data curation that could help increase acceptance of medical device RWE. (42 mins) The remainder of the session will be reserved for audience questions. (10 mins)