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Gene Therapy: Understanding the Science, Assessing the Evidence, and Paying for Value


  • Marsden, G.
  • Towse, A.
  • Pearson, S.D
  • Dreitlein, B.
  • Henshall, C.


This report presents an analysis of the significant clinical potential of gene therapy and the unique challenges in developing and evaluating evidence on their effectiveness and value. Special attention is given to pricing and payment mechanisms, including new approaches to payment based on long-term amortization of initial costs. The 2016 ICER Policy Summit convened an influential group of evidence policy leaders from insurers, pharmacy benefit management firms, and life science companies to discuss gene therapies and to explore the opportunities and challenges of adopting this technology in the US health care system. Based on the discussions at the Policy Summit, this report is capped by a set of recommendations for future consideration by payers, manufacturers, and policymakers. Please note a [version of this paper]( has also been published in the Journal of Comparative Effectiveness Research.

Suggested Citation

  • Marsden, G. & Towse, A. & Pearson, S.D & Dreitlein, B. & Henshall, C., 2017. "Gene Therapy: Understanding the Science, Assessing the Evidence, and Paying for Value," Research Papers 001811, Office of Health Economics.
  • Handle: RePEc:ohe:respap:001811

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    Cited by:

    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. Hanna, E. & Toumi, M. & Dussart, C. & Borissov, B. & Dabbous, O. & Badora, K. & Auquier, P., 2018. "Funding breakthrough therapies: A systematic review and recommendation," Health Policy, Elsevier, vol. 122(3), pages 217-229.

    More about this item


    Judging value for money and improving decision making;

    JEL classification:

    • I1 - Health, Education, and Welfare - - Health


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