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Using Real-World Data in the Health Technology Assessment of Pharmaceuticals: Strengths, Difficulties and a Pragmatic Way Forward

Published:January 24, 2023DOI:https://doi.org/10.1016/j.jval.2023.01.010
Using Real-World Data in the Health Technology Assessment of Pharmaceuticals: Strengths, Difficulties and a Pragmatic Way Forward
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      Abstract

      In the past decade there have been increasing calls for greater use of real-world evidence and data (RWE/D), with the explicit goal of enabling faster provision of effective medicines to patients in need. The push for decision makers to accept RWE is especially noticeable in the pursuit of regulatory approval, but RWE, particularly when used to estimate the relative effectiveness of interventions, is not always readily accepted by agencies responsible for reimbursement and pricing of new pharmaceuticals and, to a varying degree, is not accepted across jurisdictions. This lack of trust hampers the use of RWE despite a very large and growing literature base on the principles of how RWE should be used. In this paper, we suggest an important part of the explanation of why this situation has arisen and make suggestions for its alleviation. Since problems commonly arise that are particular to the question being asked and the data source(s) being used, general guidance on the principles of how to use RWD cannot cover all eventualities. We are therefore suggesting the creation of an archive, or repository, to record uses of RWD in support of decisions by funding bodies or their advisors. This paper introduces a proposed, structured classification of decision types using RWE, around which evidence can be assembled in a curated source (RWD/E taxonomy) and thus facilitate judgements on when evidence is ‘good enough’. This is the first paper in a series in a special issue of this journal that looks at the barriers to optimal use of RWE in health technology assessment and how to overcome them. We begin significantly to populate our ‘taxonomy’ with examples in an accompanying paper (Paper 3). We also propose recommendations for international standards of evaluating the acceptability of RWD governance practices (Paper 5).
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      Article info

      Publication history

      Accepted: January 12, 2023
      Received in revised form: December 19, 2022
      Received: August 7, 2022

      Publication stage

      In Press Accepted Manuscript

      Footnotes

      Précis: Real-world evidence for new pharmaceuticals is not always accepted by decision makers. This article explores why, offering a potential solution to address some issues.

      Author contributions

      Concept and design: Akehurst, Murphy, Solà-Morales, Cunningham, Mestre-Ferrandiz, de Pouvourville

      Acquisition of data: Murphy

      Analysis and interpretation of data: Akehurst, Murphy, Solà-Morales, Cunningham, Mestre-Ferrandiz, de Pouvourville

      Drafting of the manuscript: Akehurst, Murphy, Solà-Morales, Cunningham, Mestre-Ferrandiz, de Pouvourville

      Critical revision of the paper for important intellectual content: Akehurst, Murphy, Solà-Morales, Cunningham, Mestre-Ferrandiz, de Pouvourville

      Obtaining funding: Akehurst, Murphy, Solà-Morales, Cunningham, Mestre-Ferrandiz, de Pouvourville

      Conflict of Interest Disclosures

      Dr Akehurst and Dr Murphy are employees of Lumanity, which received funding from Novartis AG for the work submitted. Drs Solà-Morales and Cunningham reported receiving institutional fees from Novartis AG for the work submitted, and also reported receiving institutional fees from Novartis AG outside of the work submitted. Dr Mestre-Ferrandiz and Dr de Pouvourville reported receiving personal fees from Novartis AG for the work submitted. Dr de Pouvourville has also reported receiving personal fees from Novartis AG outside the submitted work. None of the authors declare any patents or copyrights, or any other personal relationships of relevance. No other disclosures were reported.

      Funding/Support: Novartis

      Description of the article: Real-world evidence (RWE) is not always readily accepted by agencies responsible for reimbursement and pricing for new pharmaceuticals, despite a large and growing literature base on the principles of how such evidence should be used. This is the first paper in a series that looks at the barriers to optimal use of RWE in health technology assessment and how to overcome them. It provides background on why this situation has arisen and, more importantly, proposes a way forward to help future researchers and decision makers improve their acceptance and use of RWE.

      Identification

      DOI: https://doi.org/10.1016/j.jval.2023.01.010

      Copyright

      © 2023 International Society for Pharmacoeconomics and Outcomes Research, Inc. Published by Elsevier Inc.

      ScienceDirect

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