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Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers

Nature Reviews Drug Discovery volume 9, pages 277–291 (2010)Cite this article

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Abstract

Drug regulatory agencies have traditionally assessed the quality, safety and efficacy of drugs, and the current paradigm dictates that a new drug should be licensed when the benefits outweigh the risks. By contrast, third-party payers base their reimbursement decisions predominantly on the health benefits of the drug relative to existing treatment options (termed relative efficacy; RE). Over the past decade, the role of payers has become more prominent, and time-to-market no longer means time-to-licensing but time-to-reimbursement. Companies now have to satisfy the sometimes divergent needs of both regulators and payers, and to address RE during the pre-marketing stages. This article describes the current political background to the RE debate and presents the scientific and methodological challenges as they relate to RE assessment. In addition, we explain the impact of RE on drug development, and speculate on future developments and actions that are likely to be required from key players.

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Figure 1: Decision-makers on the road to market access.
Figure 2: The concept and pitfalls of relative efficacy assessment.
Figure 3: Diagram showing the time trend of measures of disease activity for patients enrolled in the Swedish Rheumatology register at the start of first treatment with a biologic anti-rheumatic agent.
Figure 4: The anticipated evolution of relative efficacy assessment at the interface between drug regulatory agencies and payers.

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Author notes

  1. Hans-Georg Eichler and Brigitte Bloechl-Daum: Hans-Georg Eichler and Brigitte Bloechl-Daum contributed equally to this work.

  2. Disclaimer: The views expressed in this article are the personal views of the authors and may not be understood or quoted as being made on behalf of or reflecting the position of the regulatory agencies, health technology assessment bodies or other organizations that the authors work for.

Authors and Affiliations

  1. Hans-Georg Eichler and Franz König are at the European Medicines Agency, 7 Westferry Circus, London E14 4HB, UK.,

    Hans-Georg Eichler & Franz König

  2. Brigitte Bloechl-Daum is at the Department of Clinical Pharmacology, Medical University of Vienna, Waehringer Gürtel 18-20, A-1090 Vienna, Austria.,

    Brigitte Bloechl-Daum

  3. Eric Abadie is at the Agence française de sécurité sanitaire des produits de santé, 143/147, bd Anatole France 93285, Saint-Denis Cedex, France.,

    Eric Abadie

  4. Department of Cardiovascular Sciences, David Barnett is at the Pharmacology and Therapeutics Group, University of Leicester, Robert Kilpatrick Clinical Sciences Building, Leicester Royal Infirmary, Leicester LE2 7LX, UK.,

    David Barnett

  5. Steven Pearson is at the Institute for Clinical and Economic Review, 10th floor, 101 Merrimac Street, Boston, Massachusetts 20815, USA.,

    Steven Pearson

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Eichler, HG., Bloechl-Daum, B., Abadie, E. et al. Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers. Nat Rev Drug Discov 9, 277–291 (2010). https://doi.org/10.1038/nrd3079

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