No abstract

In most of the cancer drugs, the MCB threshold is not met at the time of approval when measured using both ESMO-MCBS scales. Since approval status does not translate into a MCB, stakeholders and decision makers should focus on the benefit/risk ratio of anticancer drugs to assure an appropriate allocation of resources in health care systems.

ObjectiveIn the last decade an increasing number of high-priced, new cancer treatments received marketing authorisation in Europe. What is actually known about the clinical benefit of those therapies at the time of approval needs to be elucidated in order to inform decisions about the use and reimbursement of these novel treatment options. Thus, the aim of the current analysis was to systematically investigate oncological therapies approved between January 2009 and April 2016 and extract as well as quantify the level of knowledge of the clinical benefit at the time of marketing authorisation.MethodsTo assess the benefit of new interventions as well as expanded indications, we extracted the median gain of the two study end points: progression-free survival (PFS) and overall survival (OS). Information is based on approval documents provided by the European Medicines Agency and assessments from the Austrian Horizon Scanning programme. We included all cancer therapies approved in Europe between 2009 (January 1) and 2016 (April 15).ResultsCancer drugs for 134 new indications approved since 2009 were identified. In the case of 37 indications (27%), no data were available for PFS or for OS. A positive difference in median OS was reached by 76 licensed indications (55.5%); 22 (16%) of them showed a difference of more than 3 months. Regarding the study end point PFS, an improvement was shown in 90 indications (65.2%).ConclusionScarce knowledge regarding the clinical benefit of anticancer therapies is available at the time of approval. In addition, the survival benefit of the approved indications is less than 3 months in the majority of approved therapies.

The introduction provisional approval strategies increases the approval of anticancer drugs with ambiguous benefit-risk profiles. Thus, in many instances, there is lacking evidence about overall survival (OS) at the time of marketing authorisation. Our objective was to monitor and characterise therapies with ambiguous benefit-risk profiles and identify any postapproval updates on median OS after at least 3 years of approval by the European Medicines Agency (EMA). Methods: We included all originator anticancer drugs with initially ambiguous benefit-risk profiles that received marketing authorisation by the EMA between January 1, 2009 and May 31, 2015. Our monitoring timeframe was at least 3 years after EMA approval. To identify study updates, the following three sources were included: clinicaltrials.gov, European Public Assessments Reports and PubMed. Results: In total, we identified 102 eligible approval studies. Out of these, a negative difference in median OS or no information was available in 43 (42.2%) instances. During monitoring, 14 updates with accessible positive information on OS could be identified. Including monitoring results, there are still 29 remaining therapies (28.4%) where no or negative information (n Z 24 [23.5%] and n Z 5 [4.9%], respectively) regarding OS is present at least 3 years after EMA approval. Conclusion: One-third of oncology drugs with ambiguous benefit-risk profiles at the time of approval fail to demonstrate a survival benefit even after several years of marketing