IDEAS home Printed from https://ideas.repec.org/a/spr/pharme/v39y2021i7d10.1007_s40273-021-01010-z.html
   My bibliography  Save this article

Guidance for the Harmonisation and Improvement of Economic Evaluations of Personalised Medicine

Author

Listed:
  • Heleen Vellekoop

    (Erasmus University Rotterdam)

  • Simone Huygens

    (Erasmus University Rotterdam)

  • Matthijs Versteegh

    (Erasmus University Rotterdam)

  • László Szilberhorn

    (Syreon Research Institute)

  • Tamás Zelei

    (Syreon Research Institute)

  • Balázs Nagy

    (Syreon Research Institute)

  • Rositsa Koleva-Kolarova

    (University of Oxford)

  • Apostolos Tsiachristas

    (University of Oxford)

  • Sarah Wordsworth

    (University of Oxford)

  • Maureen Rutten-van Mölken

    (Erasmus University Rotterdam
    Erasmus University Rotterdam)

Abstract

Objective The objective of this study was to develop guidance contributing to improved consistency and quality in economic evaluations of personalised medicine (PM), given current ambiguity about how to measure the value of PM as well as considerable variation in the methodology and reporting in economic evaluations of PM. Methods A targeted literature review of methodological papers was performed for an overview of modelling challenges in PM. Expert interviews were held to discuss best modelling practice. A systematic literature review of economic evaluations of PM was conducted to gain insight into current modelling practice. The findings were synthesised and used to develop a set of draft recommendations. The draft recommendations were discussed at a stakeholder workshop and subsequently finalised. Results Twenty-two methodological papers were identified. Some argued that the challenges in modelling PM can be addressed within existing methodological frameworks, others disagreed. Eighteen experts were interviewed. They believed large uncertainty to be a key concern. Out of 195 economic evaluations of PM identified, 56% addressed none of the identified modelling challenges. A set of 23 recommendations was developed. Eight recommendations focus on the modelling of test-treatment pathways. The use of non-randomised controlled trial data is discouraged but several recommendations are provided in case randomised controlled trial data are unavailable. The parameterisation of structural uncertainty is recommended. Other recommendations consider perspective and discounting; premature survival data; additional value elements; patient and clinician compliance; and managed entry agreements. Conclusions This study provides a comprehensive list of recommendations to modellers of PM and to evaluators and reviewers of PM models.

Suggested Citation

  • 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.
    • Handle: RePEc:spr:pharme:v:39:y:2021:i:7:d:10.1007_s40273-021-01010-z
    DOI: 10.1007/s40273-021-01010-z
    as

    Download full text from publisher

    File URL: http://link.springer.com/10.1007/s40273-021-01010-z
    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/s40273-021-01010-z?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.
    Full references (including those not matched with items on IDEAS)

    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. 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.
    5. 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.
    6. 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.
    7. 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.
    8. 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.
    9. 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.
    10. 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.
    11. 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:7:d:10.1007_s40273-021-01010-z. 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.