Clinical benefit of oncology drugs – Contrasting the evidence of clinically relevant endpoints three years after EMA approval
Duration: December 2017 – December 2019
Marketing authorisations on the basis of a scarcity of evidence are bound to increase due to new fast-track approval pathways [1-3]. Just recently, two additional accelerated licensing strategies were introduced by the European Medicines Agency (EMA), adaptive pathways and PRIME (PRIority MEdicines) that allow for faster access to medicines . This aggravates limited evidence and increasing uncertainties concerning the clinical benefit of anticancer therapies [1, 2]. Many recently published studies have shown that a large amount of EMA-approved cancer drugs have ambiguous benefit-risk profiles in combination with limited evidence available on patient relevant outcomes at the time of marketing authorisation [4, 5]. The regulatory changes from the EMA have profound impact on national medicine and cancer budgets, as well as the ability of health technology appraisal mechanisms to reach evidence based decisions. Therefore, stakeholders and decision makers need to frequently assess the benefit risk ratio of new cancer drugs, in particular for those where scarce knowledge was available on patient relevant outcomes at the time of approval.
Project aim and research question:
The project will contrast the evidence of endpoints (overall survival and progression-free survival) of oncological therapies at least three years after approval. The focus will be on those therapies where no information on overall survival or progression-free survival was available at the time of marketing authorisation. Therefore the main research question will be: How much evidence on the clinical benefit of oncology drugs, with ambiguous benefit-risk profiles, is available three years after approval?
- systematic literature search in the database clinicaltrials.gov
- manual hand search to elucidate any study updates missed in the systematic search
- contrasting the evidence of clinically relevant endpoints
- December 2017–March 2018 literature search and data extraction by two researcher
- April 2018–January 2019: data analysis and presentation
February 2019–December 2019: assembly of evidence and publication
 Banzi R, Gerardi C, Bertele V, Garattini S. Approvals of drugs with uncertain benefit-risk profiles in Europe. European journal of internal medicine. 2015;26(8):572-84.
 Rupp T, Zuckerman D. Quality of life, overall survival, and costs of cancer drugs approved based on surrogate endpoints. JAMA Internal Medicine. 2017;177(2):276-7.
 European Medicines Agency (EMA). Annual Report 2015. London: 2016.
 Grössmann N, Wild C. Between January 2009 and April 2016, 134 novel anticancer therapies were approved: what is the level of knowledge concerning the clinical benefit at the time of approval? ESMO Open. 2017;1(6).
 Grössmann N, Del Paggio JC, Wolf S, Sullivan R, Booth CM, Rosian K, et al. Five years of EMA-approved systemic cancer therapies for solid tumours - a comparison of two thresholds for meaningful clinical benefit. European Journal of Cancer.82:66-71.