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Recalibrating Health Technology Assessment Methods for Cell and Gene Therapies


  • Aris Angelis

    (London School of Economics and Political Science, Cowdray House)

  • Huseyin Naci

    (London School of Economics and Political Science, Cowdray House)

  • Allan Hackshaw

    (University College London)


Recently licensed cell and gene therapies have promising but highly uncertain clinical benefits. They are entering the market at very high prices, with the latest entrants costing hundreds of thousands of dollars. The significant long-term uncertainty posed by these therapies has already complicated the use of conventional economic evaluation approaches such as cost-effectiveness and cost-utility analyses, which are widely used for assessing the value of new health interventions. Cell and gene therapies also risk jeopardising healthcare systems’ financial sustainability. As a result, there is a need to recalibrate the current health technology assessment methods used to measure and compensate their value. In this paper, we outline a set of technical adaptations and methodological refinements to address key challenges in the appraisal of cell and gene therapies’ value, including the assessment of efficiency and affordability. We also discuss the potential role of alternative financing mechanisms. Ultimately, uncertainties associated with cell and gene therapies can only be meaningfully addressed by improving the evidence base supporting their approval and adoption in healthcare systems.

Suggested Citation

  • Aris Angelis & Huseyin Naci & Allan Hackshaw, 2020. "Recalibrating Health Technology Assessment Methods for Cell and Gene Therapies," PharmacoEconomics, Springer, vol. 38(12), pages 1297-1308, December.
  • Handle: RePEc:spr:pharme:v:38:y:2020:i:12:d:10.1007_s40273-020-00956-w
    DOI: 10.1007/s40273-020-00956-w

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    References listed on IDEAS

    1. Bengt Jönsson & Grace Hampson & Jonathan Michaels & Adrian Towse & J.-Matthias Graf Schulenburg & Olivier Wong, 2019. "Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 20(3), pages 427-438, April.
    2. Mónica D. Oliveira & Inês Mataloto & Panos Kanavos, 2019. "Multi-criteria decision analysis for health technology assessment: addressing methodological challenges to improve the state of the art," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 20(6), pages 891-918, August.
    3. Briggs, Andrew & Sculpher, Mark & Claxton, Karl, 2006. "Decision Modelling for Health Economic Evaluation," OUP Catalogue, Oxford University Press, number 9780198526629.
    4. Swathi Iyengar & Kiu Tay-Teo & Sabine Vogler & Peter Beyer & Stefan Wiktor & Kees de Joncheere & Suzanne Hill, 2016. "Prices, Costs, and Affordability of New Medicines for Hepatitis C in 30 Countries: An Economic Analysis," PLOS Medicine, Public Library of Science, vol. 13(5), pages 1-22, May.
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    Blog mentions

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    1. Chris Sampson’s journal round-up for 30th November 2020
      by Chris Sampson in The Academic Health Economists' Blog on 2020-11-30 12:00:05


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    Cited by:

    1. Simone A. Huygens & Matthijs M. Versteegh & Stefan Vegter & L. Jan Schouten & Tim A. Kanters, 2021. "Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision," PharmacoEconomics, Springer, vol. 39(4), pages 383-397, April.
    2. Dabbous, Monique & Toumi, Mondher & Simoens, Steven & Wasem, Juergen & Saal, Gauri & Wang, Yitong & Osuna, José Luis Huerta & François, Clément & Annemans, Lieven & Graf von der Schulenburg, Johann-Ma, 2022. "Amortization of gene replacement therapies: A health policy analysis exploring a mechanism for mitigating budget impact of high-cost treatments," Health Policy, Elsevier, vol. 126(1), pages 49-59.

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