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HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies


  • Doug Coyle

    (University of Ottawa)

  • Isabelle Durand-Zaleski

    (University of Paris Est)

  • Jasmine Farrington

    (PHMR Ltd)

  • Louis Garrison

    (University of Washington)

  • Johann-Matthias Graf von der Schulenburg

    (Center for Health Economics Research, Hannover)

  • Wolfgang Greiner

    (Bielefeld University)

  • Louise Longworth

    (Bielefeld University)

  • Aurélie Meunier

    (PHMR Ltd)

  • Anne-Sophie Moutié

    (PHMR Ltd)

  • Stephen Palmer

    (Center for Health Economics, University of York)

  • Zack Pemberton-Whiteley

    (Acute Leukemia Advocates Network (ALAN)-Chair)

  • Mark Ratcliffe

    (PHMR Ltd)

  • Jie Shen


  • Doug Sproule


  • Kun Zhao

    (China National Health Development Research Center)

  • Koonal Shah

    (PHMR Ltd)


This last decade has been marked by significant advances in the development of cell and gene (C&G) therapies, such as gene targeting or stem cell-based therapies. C&G therapies offer transformative benefits to patients but present a challenge to current health technology decision-making systems because they are typically reviewed when clinical efficacy data are very limited and when there is uncertainty about the long-term durability of outcomes. These challenges are not unique to C&G therapies, but they face more of these barriers, reflecting the need for adapting existing value assessment frameworks. Still, C&G therapies have the potential to be cost-effective even at very high price points. The impact on healthcare budgets will depend on the success rate of pipeline assets and on the extent to which C&G therapies will expand to wider pathologies beyond rare or ultra-rare diseases. Getting pricing and reimbursement models right is important for incentivising research and development investment while not jeopardising the sustainability of healthcare systems. Payers and manufacturers therefore need to acknowledge each other’s constraints—limitations in the evidence generation on the manufacturer side, budget considerations on the payer side—and embrace innovative thinking and approaches to ensure timely delivery of therapies to patients. Several experts in health technology assessment and clinical experts have worked together to produce this publication and identify methodological and policy options to improve the assessment of C&G therapies, and make it happen better, faster and sustainably in the coming years.

Suggested Citation

  • Doug Coyle & Isabelle Durand-Zaleski & Jasmine Farrington & Louis Garrison & Johann-Matthias Graf von der Schulenburg & Wolfgang Greiner & Louise Longworth & Aurélie Meunier & Anne-Sophie Moutié & Ste, 2020. "HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 21(9), pages 1421-1437, December.
  • Handle: RePEc:spr:eujhec:v:21:y:2020:i:9:d:10.1007_s10198-020-01212-w
    DOI: 10.1007/s10198-020-01212-w

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    References listed on IDEAS

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    Blog mentions

    As found by, the blog aggregator for Economics research:
    1. Chris Sampson’s journal round-up for 9th November 2020
      by Chris Sampson in The Academic Health Economists' Blog on 2020-11-09 12:00:00


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    2. Daniel Gallacher & Nigel Stallard & Peter Kimani & Elvan Gökalp & Juergen Branke, 2022. "Development of a model to demonstrate the impact of National Institute of Health and Care Excellence cost‐effectiveness assessment on health utility for targeted medicines," Health Economics, John Wiley & Sons, Ltd., vol. 31(2), pages 417-430, February.

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