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How to Deal with the Inevitable: Generating Real-World Data and Using Real-World Evidence for HTA Purposes – From Theory to Action

  • Wija Oortwijn (a1), Laura Sampietro-Colom (a2) and Rebecca Trowman (a3)

Abstract

Objectives

Real-world evidence (RWE), derived from real-world data (RWD), is already used, to some extent, for health technology assessment (HTA) purposes. With the increased availability of RWD, there is potential for more widespread use but also challenges ensuring reliable RWE for HTA. Opportunities to overcome key challenges, identified at a scoping meeting, were discussed during the 2019 HTA international (HTAi) Global Policy Forum (GPF).

Methods

Reflection of discussions using Design Thinking (an interactive process aimed to solve complex problems) between seventy-three representatives from not-for-profit, for-profit organizations, and HTAi leadership. The discussions were informed by a background paper, and presentations from three invited keynote speakers and eleven GPF members.

Results

Several options were listed for addressing the identified key challenges: quality and acceptability, governance and accountability, transferability, and informing decision making. The GPF emphasized that the HTA community should first understand what questions could be answered with RWE. Additionally, more clarity on methods, standards, streamlining RWD collection, data sharing across jurisdictions, replication of RWD, and expert analysis were mentioned as important priorities.

Conclusions

The HTA community is currently standing at a cross-road as it is not yet fully equipped to address these key challenges. It is, therefore, time for action. The community should start aligning on what is the best source of evidence according to purpose and how the data should be collected to create reliable evidence. It should also initiate the development of actions and guidance to properly develop and manage RWD/RWE to inform decision making across the technology lifecycle.

Copyright

Corresponding author

Author for correspondence: Wija Oortwijn, Email: w.oortwijn@radboudumc.nl

Footnotes

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We thank all Global Policy Forum Organizing Committee members (in alphabetical order) for steering our work: Barbara Calvert, Abbott; Joseph Cook, Pfizer Inc.; Tammy Clifford, Canadian Agency for Drugs and Technologies in Health (CADTH), Canada; Wim Goettsch, National Health Care Institute (ZIN), The Netherlands; Elizabeth Cobbs, Merck; Andrew Mitchell, Australian Government Department of Health, Australia; and Sean Tunis, Centre for Medical Technology Policy (CMTP), US. We also thank the HTAi Global Policy Forum attendees for sharing their thoughts and experiences during the HTAi Global Policy Forum 2019 (see Supplementary Table 1) and those GPF members who provided valuable comments and suggestions on an earlier version of this paper.

Footnotes

References

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Supplementary materials

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