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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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    1. Gordon Tullock, 1964. "The Social Rate of Discount and the Optimal Rate of Investment: Comment," The Quarterly Journal of Economics, President and Fellows of Harvard College, vol. 78(2), pages 331-336.
    2. Donna Rowen & John Brazier & Clara Mukuria & Anju Keetharuth & Arne Risa Hole & Aki Tsuchiya & Sophie Whyte & Phil Shackley, 2016. "Eliciting Societal Preferences for Weighting QALYs for Burden of Illness and End of Life," Medical Decision Making, , vol. 36(2), pages 210-222, February.
    3. Dolan, Paul, 2000. "The measurement of health-related quality of life for use in resource allocation decisions in health care," Handbook of Health Economics, in: A. J. Culyer & J. P. Newhouse (ed.), Handbook of Health Economics, edition 1, volume 1, chapter 32, pages 1723-1760, Elsevier.
    4. Shah, Koonal K. & Tsuchiya, Aki & Wailoo, Allan J., 2018. "Valuing health at the end of life: A review of stated preference studies in the social sciences literature," Social Science & Medicine, Elsevier, vol. 204(C), pages 39-50.
    5. Eddie Gibson & Ian Koblbauer & Najida Begum & George Dranitsaris & Danny Liew & Phil McEwan & Amir Abbas Tahami Monfared & Yong Yuan & Ariadna Juarez-Garcia & David Tyas & Michael Lees, 2017. "Modelling the Survival Outcomes of Immuno-Oncology Drugs in Economic Evaluations: A Systematic Approach to Data Analysis and Extrapolation," PharmacoEconomics, Springer, vol. 35(12), pages 1257-1270, December.
    6. Aris Angelis & Ansgar Lange & Panos Kanavos, 2018. "Using health technology assessment to assess the value of new medicines: results of a systematic review and expert consultation across eight European countries," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 19(1), pages 123-152, January.
    7. Emmett B. Keeler & Shan Cretin, 1983. "Discounting of Life-Saving and Other Nonmonetary Effects," Management Science, INFORMS, vol. 29(3), pages 300-306, March.
    8. Peltzman, Sam, 1973. "An Evaluation of Consumer Protection Legislation: The 1962 Drug Amendments," Journal of Political Economy, University of Chicago Press, vol. 81(5), pages 1049-1091, Sept.-Oct.
    9. Julio López-Bastida & Juan Oliva & Fernando Antoñanzas & Anna García-Altés & Ramón Gisbert & Javier Mar & Jaume Puig-Junoy, 2010. "Spanish recommendations on economic evaluation of health technologies," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 11(5), pages 513-520, October.
    10. Hugh Gravelle & Dave Smith, 2001. "Discounting for health effects in cost–benefit and cost‐effectiveness analysis," Health Economics, John Wiley & Sons, Ltd., vol. 10(7), pages 587-599, October.
    11. Liz Morrell & Sarah Wordsworth & Sian Rees & Richard Barker, 2017. "Does the Public Prefer Health Gain for Cancer Patients? A Systematic Review of Public Views on Cancer and its Characteristics," PharmacoEconomics, Springer, vol. 35(8), pages 793-804, August.
    12. Christopher H. Jackson & Simon G. Thompson & Linda D. Sharples, 2009. "Accounting for uncertainty in health economic decision models by using model averaging," Journal of the Royal Statistical Society Series A, Royal Statistical Society, vol. 172(2), pages 383-404, April.
    13. Emma B. Rasiel & Kevin P. Weinfurt & Kevin A. Schulman, 2005. "Can Prospect Theory Explain Risk-Seeking Behavior by Terminally Ill Patients?," Medical Decision Making, , vol. 25(6), pages 609-613, November.
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    6. 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.
    7. 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.
    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. 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.
    10. 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.
    11. 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.
    12. 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.

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