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

  • Caridad PontesEmail author
  • Corinne Zara
  • Josep Torrent-Farnell
  • Merce Obach
  • Cristina Nadal
  • Patricia Vella-Bonanno
  • Michael Ermisch
  • Steven Simoens
  • 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
  • Brian Godman
Current Opinion

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.

Notes

Author 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.

Compliance with Ethical Standards

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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Copyright information

© Springer Nature Switzerland AG 2019

Authors and Affiliations

  • Caridad Pontes
    • 1
    • 2
    Email author
  • Corinne Zara
    • 1
  • Josep Torrent-Farnell
    • 1
    • 2
  • Merce Obach
    • 1
  • Cristina Nadal
    • 3
  • Patricia Vella-Bonanno
    • 4
  • Michael Ermisch
    • 5
  • Steven Simoens
    • 6
  • Renata Curi Hauegen
    • 7
  • Jolanta Gulbinovic
    • 8
  • Angela Timoney
    • 4
    • 9
  • Antony P. Martin
    • 10
  • Tanja Mueller
    • 4
  • Anna Nachtnebel
    • 11
  • Stephen Campbell
    • 12
    • 13
  • Gisbert Selke
    • 14
  • Tomasz Bochenek
    • 15
  • Celia C. Rothe
    • 15
  • Ileana Mardare
    • 16
  • Marion Bennie
    • 4
  • Jurij Fürst
    • 17
  • Rickard E. Malmstrom
    • 18
  • Brian Godman
    • 4
    • 10
    • 19
    • 20
  1. 1.Drug AreaCatalan Health ServiceBarcelonaSpain
  2. 2.Department of Pharmacology, Therapeutics and ToxicologyUniversitat Autònoma de BarcelonaBarcelonaSpain
  3. 3.Catalan Health ServiceBarcelonaSpain
  4. 4.Strathclyde Institute of Pharmacy and Biomedical SciencesUniversity of StrathclydeGlasgowUK
  5. 5.Pharmaceutical DepartmentNational Association of Statutory Health Insurance FundsBerlinGermany
  6. 6.Department of Pharmaceutical and Pharmacological SciencesKU LeuvenLeuvenBelgium
  7. 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 JaneiroBrazil
  8. 8.Department of Pathology, Forensic Medicine and Pharmacology, Institute of Biomedical Sciences, Faculty of MedicineVilnius UniversityVilniusLithuania
  9. 9.NHS LothianEdinburghUK
  10. 10.Health Economics CentreUniversity of Liverpool Management SchoolLiverpoolUK
  11. 11.Hauptverband der Österreichischen SozialversicherungsträgerViennaAustria
  12. 12.Centre for Primary Care, Division of Population Health, Health Services Research and Primary CareUniversity of ManchesterManchesterUK
  13. 13.NIHR Greater Manchester Patient Safety Translational Research Centre, School of Health SciencesUniversity of ManchesterManchesterUK
  14. 14.Wissenschaftliches Institut der AOK (WidO)BerlinGermany
  15. 15.Department of Drug Management, Faculty of Health SciencesJagiellonian University Medical CollegeKrakówPoland
  16. 16.Department of Public Health and Management, Faculty of Medicine“Carol Davila” University of Medicine and PharmacyBucharestRomania
  17. 17.Health Insurance InstituteLjubljanaSlovenia
  18. 18.Department of Medicine Solna, Karolinska InstituteKarolinska University Hospital SolnaStockholmSweden
  19. 19.Division of Clinical Pharmacology, Karolinska InstituteKarolinska University Hospital HuddingeStockholmSweden
  20. 20.School of PharmacySefako Makgatho Health Sciences UniversityGa-RankuwaSouth Africa

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