IDEAS home Printed from https://ideas.repec.org/a/spr/pharme/v39y2021i4d10.1007_s40273-021-01003-y.html
   My bibliography  Save this article

Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision

Author

Listed:
  • Simone A. Huygens

    (Erasmus University Rotterdam)

  • Matthijs M. Versteegh

    (Erasmus University Rotterdam)

  • Stefan Vegter

    (Novartis Pharma B.V.)

  • L. Jan Schouten

    (Canisius Wilhelmina Hospital)

  • Tim A. Kanters

    (Erasmus University Rotterdam)

Abstract

The emergence of gene therapies challenge health economists to evaluate interventions that are often provided to a small patient population with a specific gene mutation in a single dose with high upfront costs and uncertain long-term benefits. The objective of this study was to illustrate the methodological challenges of evaluating gene therapies and their implications by discussing four economic evaluations of voretigene neparvovec (VN) for the treatment of RPE65-mediated inherited retinal disease. The checklist for economic evaluations of gene therapies of Drummond et al. was applied to the economic evaluations of VN performed by US Institute for Clinical and Economic Review, two country adaptations of the company model in the UK and the Netherlands, and another US publication. The main differences in methodological choices and their impact on cost-effectiveness results were assessed and further explored with sensitivity analyses using the Dutch model. To enable comparison between the economic evaluations, costs were converted to US dollars. Different methodological choices were made in the economic evaluations of VN resulting in large differences in the incremental cost-effectiveness ratio varying from US$79,618 to US$643,813 per QALY. The chosen duration of treatment effect, source of utility values, discount rate and model structure had the largest impact on the cost-effectiveness. This study underlines the findings from Drummond et al. that standard methods can be used to evaluate gene therapies. However, given uncertainty about (particularly long-term) outcomes of gene therapies, guidance is required on the acceptable extrapolation of treatment effect of gene therapies and on how to handle the uncertainty around this extrapolation in scenario and sensitivity analyses to aid health technology assessment research and align submissions of future gene therapies.

Suggested Citation

  • 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.
  • Handle: RePEc:spr:pharme:v:39:y:2021:i:4:d:10.1007_s40273-021-01003-y
    DOI: 10.1007/s40273-021-01003-y
    as

    Download full text from publisher

    File URL: http://link.springer.com/10.1007/s40273-021-01003-y
    File Function: Abstract
    Download Restriction: Access to the full text of the articles in this series is restricted.

    File URL: https://libkey.io/10.1007/s40273-021-01003-y?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    As the access to this document is restricted, you may want to search for a different version of it.

    References listed on IDEAS

    as
    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. 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.
    Full references (including those not matched with items on IDEAS)

    Citations

    Blog mentions

    As found by EconAcademics.org, the blog aggregator for Economics research:
    1. Journal round-up: PharmacoEconomics 39(4)
      by Chris Sampson in The Academic Health Economists' Blog on 2021-06-15 06:00:05

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. Justyna Berniak-Woźny & Małgorzata Rataj, 2023. "Towards Green and Sustainable Healthcare: A Literature Review and Research Agenda for Green Leadership in the Healthcare Sector," IJERPH, MDPI, vol. 20(2), pages 1-18, January.
    2. 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.
    3. Tunis, Sean & Hanna, Eve & Neumann, Peter J. & Toumi, Mondher & Dabbous, Omar & Drummond, Michael & Fricke, Frank-Ulrich & Sullivan, Sean D. & Malone, Daniel C. & Persson, Ulf & Chambers, James D., 2021. "Variation in market access decisions for cell and gene therapies across the United States, Canada, and Europe," Health Policy, Elsevier, vol. 125(12), pages 1550-1556.
    4. 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.
    5. Elisabete Gonçalves, 2022. "Value-based pricing for advanced therapy medicinal products: emerging affordability solutions," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 23(2), pages 155-163, March.
    6. Sarri, Grammati & Freitag, Andreas & Szegvari, Boglarka & Mountian, Irina & Brixner, Diana & Bertelsen, Neil & Kaló, Zoltán & Upadhyaya, Sheela, 2021. "The Role of Patient Experience in the Value Assessment of Complex Technologies – Do HTA Bodies Need to Reconsider How Value is Assessed?," Health Policy, Elsevier, vol. 125(5), pages 593-601.
    7. Anderson, Michael & Drummond, Michael & Taylor, David & McGuire, Alistair & Carter, Paul & Mossialos, Elias, 2022. "Promoting innovation while controlling cost: The UK's approach to health technology assessment," Health Policy, Elsevier, vol. 126(3), pages 224-233.
    8. Paul Catchpole & Victoria Barrett, 2020. "Keeping Pace with Pharmaceutical Innovation: The Importance of the NICE Methods Review," PharmacoEconomics, Springer, vol. 38(9), pages 901-903, September.
    9. Elisabete Gonçalves, 2020. "Advanced therapy medicinal products: value judgement and ethical evaluation in health technology assessment," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 21(3), pages 311-320, April.
    10. Aguilera-Cobos, Lorena & Rosario-Lozano, María Piedad & Ponce-Polo, Angela & Blasco-Amaro, Juan Antonio & Epstein, David, 2022. "Barriers for the evaluation of advanced therapy medicines and their translation to clinical practice: Umbrella review," Health Policy, Elsevier, vol. 126(12), pages 1248-1255.
    11. Heleen Vellekoop & Simone Huygens & Matthijs Versteegh & László Szilberhorn & Tamás Zelei & Balázs Nagy & Rositsa Koleva-Kolarova & Apostolos Tsiachristas & Sarah Wordsworth & Maureen Rutten-van Mölke, 2021. "Guidance for the Harmonisation and Improvement of Economic Evaluations of Personalised Medicine," PharmacoEconomics, Springer, vol. 39(7), pages 771-788, July.
    12. 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.

    More about this item

    Statistics

    Access and download statistics

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:spr:pharme:v:39:y:2021:i:4:d:10.1007_s40273-021-01003-y. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Sonal Shukla or Springer Nature Abstracting and Indexing (email available below). General contact details of provider: http://www.springer.com .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.