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White Paper

14 November 2018

A framework to guide the optimal development and use of real-world evidence for drug coverage and formulary decisions

Authors: Steven D Pearson, William B Dreitlein [email protected], Adrian Towse, Grace Hampson, and Chris HenshallAuthor Info & Affiliations

Publication: J. Comp. Eff. Res.

Volume 7, Number 12

https://doi.org/10.2217/cer-2018-0059

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Abstract

Aim: To provide a framework for optimizing the development and use of real-world evidence (RWE) in drug coverage decisions. Materials & methods: The Institute for Clinical and Economic Review convened a Policy Summit with representatives from 23 payer and life science companies that compose the Institute for Clinical and Economic Review membership. Results: Summit participants helped refine a new conceptual framework that emphasizes the central role of contextual considerations and the evidentiary argument that the RWE is intended to support in designing the process for the development and interpretation of RWE. Conclusion: This framework may provide a structured way for pharmaceutical manufacturers and payers to develop a shared understanding of the best way to develop RWE that will ultimately be useful in informing coverage and formulary decisions.

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References

Papers of special note have been highlighted as: • of interest; •• of considerable interest

1.

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2.

Malone DC, Brown M, Hurwitz JT, Peters L, Graff JS. Real-world evidence: useful in the real world of US payer decision making? How? When? And what studies? Value Health 21(3), 326–333 (2018).

• Illustrates how certain payers are using real-world evidence (RWE), and discusses the limitations of relevance, timeliness and transparency.

3.

Massachusetts Institute of Technology. MIT NEWDIGS Hosts Design Lab on Efficacy to Effectiveness (E2E) data and decision-making. https://newdigs.mit.edu/news/mit-newdigs-hosts-design-lab-efficacy-effectiveness-e2e-data-and-decision-making.

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• Provides a catalog of parameters and items that should be adequately described to ensure transparency in the reporting of RWE studies.

8.

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• Recommends best procedural practices to follow when developing RWE and complements the paper above by Wang and colleagues.

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10.

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•• Full description of the Institute for Clinical and Economic Review Policy Summit on RWE with deeper discussions of the challenges and opportunities of using RWE for coverage and formulary decisions.

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