gms | German Medical Science

Background: In 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.

Materials and methods: To assess the benefit of new interventions as well as expanded indications, we extracted the median gain of the two study endpoints: progression-free survival (PFS) and overall survival (OS). Information is based on approval documents provided by the European Medicine Agency (EMA) and assessments from the Austrian Horizon Scanning programme (HSO). We included all cancer therapies approved in Europe between 2009 (January 1st) and 2016 (April 15th).

Results: Cancer drugs for 134 new indications approved since 2009 were identified. In the case of 37 indications (27%), no data was available for PFS or for OS. A positive difference in median overall survival was reached by 76 licensed indications (55.5%); 22 (16%) of them showed a difference of more than three months. Regarding the study endpoint progression-free survival, an improvement was shown in 90 indications (65.2%).

Discussion: Scarce knowledge regarding the clinical benefit of anti-cancer therapies is available at the time of approval. In addition, the survival benefit of the approved indications is less than three months in the majority of approved therapies.