Use of Real-World Evidence for Regulatory Approval and Coverage of Medical Devices: A Landscape Assessment

Value Health. 2021 Dec;24(12):1792-1798. doi: 10.1016/j.jval.2021.07.003. Epub 2021 Aug 18.


Objectives: To enhance the generalizability of the evidence it reviews, the US Food and Drug Administration (FDA) has encouraged manufacturers to expand the submission of real-world evidence (RWE). The extent to which this evidence, which is generated outside of research settings, can support decision making remains unclear. We described the current use of RWE for medical devices, assessed manufacturers' challenges in generating and using it for regulatory and coverage decisions, and identified opportunities to expand its use.

Methods: We conducted 27 solo and group interviews with FDA officials and representatives of device manufacturers, payers, and health technology assessment organizations. All interviews used a semistructured protocol and were transcribed to allow thematic analysis.

Results: Accessing and linking real-world data sources, identifying unique devices, capturing longitudinal data, limited staff expertise, and uncertain return on investment have hampered efforts to use real-world data. Many companies in our sample were conducting research using real-world data, but none had submitted RWE as the primary evidence supporting a premarket approval. FDA guidance was helpful, but regulatory requirements remained ambiguous and examples of successful regulatory decisions based on RWE were limited. Payers mainly used RWE to supplement experimental evidence in coverage decisions, evaluated both types of evidence in similar ways, and had concerns about the rigor of RWE.

Conclusions: Technical challenges may slow efforts to generate and use RWE in the near term. Additional regulatory guidance and examples, greater use of rigorous study designs and analytic methods, and continued stakeholder engagement could accelerate the use of RWE.

Keywords: claims data; clinical trials; coverage policy; electronic health records; medical devices; patient registry; payer; real-world data; real-world evidence; regulatory approval.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Device Approval*
  • Interviews as Topic
  • Technology Assessment, Biomedical*
  • United States
  • United States Food and Drug Administration*