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Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare

Author

Listed:
  • Bengt Jönsson

    (Stockholm School of Economics)

  • Grace Hampson

    (Office of Health Economics)

  • Jonathan Michaels

    (University of Sheffield)

  • Adrian Towse

    (Office of Health Economics)

  • J.-Matthias Graf Schulenburg

    (Leibniz University Hannover)

  • Olivier Wong

    (Medi-Qualite Omega)

Abstract

Background Advanced therapy medicinal products (ATMPs) are beginning to reach European markets, and questions are being asked about their value for patients and how healthcare systems should pay for them. Objectives To identify and discuss potential challenges of ATMPs in view of current health technology assessment (HTA) methodology—specifically economic evaluation methods—in Europe as it relates to ATMPs, and to suggest potential solutions to these challenges. Methods An Expert Panel reviewed current HTA principles and practices in relation to the specific characteristics of ATMPs. Results Three key topics were identified and prioritised for discussion—uncertainty, discounting, and health outcomes and value. The panel discussed that evidence challenges linked to increased uncertainty may be mitigated by collection of follow-on data, use of value of information analysis, and/or outcomes-based contracts. For discount rates, an international, multi-disciplinary forum should be established to consider the economic, social and ethical implications of the choice of rate. Finally, consideration of the feasibility of assessing the value of ATMPs beyond health gain may also be key for decision-making. Conclusions ATMPs face a challenge in demonstrating their value within current HTA frameworks. Consideration of current HTA principles and practices with regards to the specific characteristics of ATMPs and continued dialogue will be key to ensuring appropriate market access. Classification code I.

Suggested Citation

  • 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.
    • Handle: RePEc:spr:eujhec:v:20:y:2019:i:3:d:10.1007_s10198-018-1007-x
    DOI: 10.1007/s10198-018-1007-x
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    6. 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.
    7. 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.
    8. 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.
    9. 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.
    10. 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.
    11. 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.
    12. 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.

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