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

Caridad Pontes (), 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 and Brian Godman
Additional contact information
Caridad Pontes: Catalan Health Service
Corinne Zara: Catalan Health Service
Josep Torrent-Farnell: Catalan Health Service
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
Antony P. Martin: University of Liverpool Management School
Tanja Mueller: University of Strathclyde
Anna Nachtnebel: Hauptverband der Österreichischen Sozialversicherungsträger
Stephen Campbell: 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

Applied Health Economics and Health Policy, 2020, vol. 18, issue 1, No 2, 5-16

Abstract: 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.

Date: 2020
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DOI: 10.1007/s40258-019-00527-x

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