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Time to Review Authorisation and Funding for New Cancer Medicines in Europe? Inferences from the Case of Olaratumab

Author

Listed:
  • Caridad Pontes

    (Catalan Health Service
    Universitat Autònoma de Barcelona)

  • Corinne Zara

    (Catalan Health Service)

  • Josep Torrent-Farnell

    (Catalan Health Service
    Universitat Autònoma de Barcelona)

  • Merce Obach

    (Catalan Health Service)

  • Cristina Nadal

    (Catalan Health Service)

  • Patricia Vella-Bonanno

    (University of Strathclyde)

  • Michael Ermisch

    (National Association of Statutory Health Insurance Funds)

  • Steven Simoens

    (KU Leuven)

  • Renata Curi Hauegen

    (National Institute of Science and Technology for Innovation on Diseases of Neglected Populations (INCT-IDPN), Center for Technological Development in Health (CDTS), Osvaldo Cruz Foundation (Fiocruz))

  • Jolanta Gulbinovic

    (Vilnius University)

  • Angela Timoney

    (University of Strathclyde
    NHS Lothian)

  • Antony P. Martin

    (University of Liverpool Management School)

  • Tanja Mueller

    (University of Strathclyde)

  • Anna Nachtnebel

    (Hauptverband der Österreichischen Sozialversicherungsträger)

  • Stephen Campbell

    (University of Manchester
    University of Manchester)

  • Gisbert Selke

    (Wissenschaftliches Institut der AOK (WidO))

  • Tomasz Bochenek

    (Jagiellonian University Medical College)

  • Celia C. Rothe

    (Jagiellonian University Medical College)

  • Ileana Mardare

    (“Carol Davila” University of Medicine and Pharmacy)

  • Marion Bennie

    (University of Strathclyde)

  • Jurij Fürst

    (Health Insurance Institute)

  • Rickard E. Malmstrom

    (Karolinska University Hospital Solna)

  • Brian Godman

    (University of Strathclyde
    University of Liverpool Management School
    Karolinska University Hospital Huddinge
    Sefako Makgatho Health Sciences University)

Abstract

The potential benefits of early patient access to new medicines in areas of high unmet medical need are recognised, but uncertainties concerning effectiveness, safety and added value when new medicines are authorised, and subsequently funded based on initial preliminary data only, have important implications. In 2016 olaratumab received accelerated conditional approval from both the European Medicines Agency and the US Food and Drug Administration for the treatment of soft-tissue sarcoma, based on the claims of a substantial reduction in the risk of death with an 11.8-month improvement in median overall survival in a phase II trial in combination with doxorubicin vs. doxorubicin alone. The failure to confirm these benefits in the post-authorisation pivotal trial has highlighted key concerns regarding early access and conditional approvals for new medicines. Concerns include potentially considerable clinical and economic costs, so that patients may have received suboptimal treatment and any money spent has foregone the opportunity to improve access to effective treatments. As a result, it seems reasonable to reconsider current marketing authorisation models and approaches. Potential pathways forward include closer collaboration between regulators, pharmaceutical companies and payers to enhance the generation of rapid and comparative confirmatory trials in a safe and fair manner, with minimal patient exposure as required to achieve robust evidence. Additionally, it may be time to review early access systems, and to explore new avenues regarding who should pay or part pay for new treatments whilst information is being collected as part of any obligations for conditional marketing authorisation. Greater co-operation between countries regarding the collection of data in routine clinical care, and further research on post-marketing data analysis and interpretation, may also contribute to improved appraisal and continued access to new innovative cancer treatments.

Suggested Citation

  • Caridad Pontes & Corinne Zara & Josep Torrent-Farnell & Merce Obach & Cristina Nadal & Patricia Vella-Bonanno & Michael Ermisch & Steven Simoens & Renata Curi Hauegen & Jolanta Gulbinovic & Angela Tim, 2020. "Time to Review Authorisation and Funding for New Cancer Medicines in Europe? Inferences from the Case of Olaratumab," Applied Health Economics and Health Policy, Springer, vol. 18(1), pages 5-16, February.
  • Handle: RePEc:spr:aphecp:v:18:y:2020:i:1:d:10.1007_s40258-019-00527-x
    DOI: 10.1007/s40258-019-00527-x
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    1. Makady, A. & van Veelen, A. & de Boer, A. & Hillege, H. & Klungel, O.H. & Goettsch, W., 2019. "Implementing managed entry agreements in practice: The Dutch reality check," Health Policy, Elsevier, vol. 123(3), pages 267-274.
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    2. Carolina Zampirolli Dias & Brian Godman & Ludmila Peres Gargano & Pâmela Santos Azevedo & Marina Morgado Garcia & Maurílio Souza Cazarim & Laís Lessa Neiva Pantuzza & Nelio Gomes Ribeiro-Junior & Andr, 2020. "Integrative Review of Managed Entry Agreements: Chances and Limitations," PharmacoEconomics, Springer, vol. 38(11), pages 1165-1185, November.
    3. Nasuh C. Buyukkaramikli & Peter Wigfield & Men Thi Hoang, 2021. "A MEA is a MEA is a MEA? Sequential decision making and the impact of different managed entry agreements at the manufacturer and payer level, using a case study for an oncology drug in England," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 22(1), pages 51-73, February.

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