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

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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.

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Authors and Affiliations

  1. Drug Area, Catalan Health Service, Travessera de les Corts 131, Edifici Olimpia, 08028, Barcelona, Spain

    Caridad Pontes, Corinne Zara, Josep Torrent-Farnell & Merce Obach

  2. Department of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, Barcelona, Spain

    Caridad Pontes & Josep Torrent-Farnell

  3. Catalan Health Service, Barcelona, Spain

    Cristina Nadal

  4. Strathclyde Institute of Pharmacy and Biomedical Sciences, University of Strathclyde, Glasgow, UK

    Patricia Vella-Bonanno, Angela Timoney, Tanja Mueller, Marion Bennie & Brian Godman

  5. Pharmaceutical Department, National Association of Statutory Health Insurance Funds, Berlin, Germany

    Michael Ermisch

  6. Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium

    Steven Simoens

  7. 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), Rio de Janeiro, Brazil

    Renata Curi Hauegen

  8. Department of Pathology, Forensic Medicine and Pharmacology, Institute of Biomedical Sciences, Faculty of Medicine, Vilnius University, Vilnius, Lithuania

    Jolanta Gulbinovic

  9. NHS Lothian, Edinburgh, UK

    Angela Timoney

  10. Health Economics Centre, University of Liverpool Management School, Liverpool, UK

    Antony P. Martin & Brian Godman

  11. Hauptverband der Österreichischen Sozialversicherungsträger, Vienna, Austria

    Anna Nachtnebel

  12. Centre for Primary Care, Division of Population Health, Health Services Research and Primary Care, University of Manchester, Manchester, UK

    Stephen Campbell

  13. NIHR Greater Manchester Patient Safety Translational Research Centre, School of Health Sciences, University of Manchester, Manchester, UK

    Stephen Campbell

  14. Wissenschaftliches Institut der AOK (WidO), Berlin, Germany

    Gisbert Selke

  15. Department of Drug Management, Faculty of Health Sciences, Jagiellonian University Medical College, Kraków, Poland

    Tomasz Bochenek & Celia C. Rothe

  16. Department of Public Health and Management, Faculty of Medicine, “Carol Davila” University of Medicine and Pharmacy, Bucharest, Romania

    Ileana Mardare

  17. Health Insurance Institute, Ljubljana, Slovenia

    Jurij Fürst

  18. Department of Medicine Solna, Karolinska Institute, Karolinska University Hospital Solna, Stockholm, Sweden

    Rickard E. Malmstrom

  19. Division of Clinical Pharmacology, Karolinska Institute, Karolinska University Hospital Huddinge, Stockholm, Sweden

    Brian Godman

  20. School of Pharmacy, Sefako Makgatho Health Sciences University, Ga-Rankuwa, South Africa

    Brian Godman

Authors
  1. Caridad Pontes
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  2. Corinne Zara
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  3. Josep Torrent-Farnell
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  4. Merce Obach
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  6. Patricia Vella-Bonanno
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  9. Renata Curi Hauegen
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  10. Jolanta Gulbinovic
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  11. Angela Timoney
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  12. Antony P. Martin
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  13. Tanja Mueller
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  15. Stephen Campbell
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  16. Gisbert Selke
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  18. Celia C. Rothe
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  20. Marion Bennie
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  21. Jurij Fürst
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  22. Rickard E. Malmstrom
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  23. Brian Godman
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Contributions

Caridad Pontes, Corinne Zara, Josep Torrent-Farnell and Merce Obach developed the initial concept and undertook the initial draft. All authors contributed to subsequent drafts based on their experience. All authors approved the submitted paper.

Corresponding author

Correspondence to Caridad Pontes.

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Funding

No funding was received for the conduct of this study or the preparation of this article.

Conflict of interest

Anumber of the co-authors are employed by health authorities or are advisers to them. Caridad Pontes, Corinne Zara, Josep Torrent-Farnell, Merce Obach, Cristina Nadal, Patricia Vella-Bonanno, Michael Ermisch, Renata Curi Hauegen, Jolanta Gulbinovic, Angela Timoney, Antony P. Martin, Tanja Mueller, Anna Nachtnebel, Stephen Campbell, Gisbert Selke, Tomasz Bochenek, Celia C. Rothe, Ileana Mardare, Marion Bennie, Jurij Fürst, Rickard E. Malmstrom and Brian Godman have no conflicts of interest that are directly relevant to the content of this article. Steven Simoens was a speaker at the European Digestive Oncology Research Forum funded by Lilly and participated in an advisory board on the sustainability of cancer care funded by Hexal.

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Pontes, C., Zara, C., Torrent-Farnell, J. et al. Time to Review Authorisation and Funding for New Cancer Medicines in Europe? Inferences from the Case of Olaratumab. Appl Health Econ Health Policy 18, 5–16 (2020). https://doi.org/10.1007/s40258-019-00527-x

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