OBJECTIVES: In 2017 an article from Cohen et al published in the British Medical Journal criticized an almost nonexistent regulation regarding the market entry of anticancer drugs assessed by the European Medicine Agency (EMA), claiming that oncology molecules enter the European market without providing sufficient evidence of their clinical benefit specifically in terms of prolongation of overall survival (OS) or improvement of quality of life (QoL).

However, EMA Authorization is only the first step to enter a local market and drugs must be evaluated at a national level to be granted a price and a reimbursement. In France, drugs are assessed by the Health Technology Assessment (HAS) to obtain an SMR/ASMR. The objective of this study was to evaluate if recent anticancer drugs which could enter the French market provided evidence of clinical benefit.

METHODS: A study was conducted to determine whether oncology molecules favorably assessed by the HAS (sufficient SMR) between 2014 and 2018 had proven a significative efficacy in their clinical trials. 91 indications from 77 Transparency Commission advices were analyzed detailing clinical data provided (designs, OS/QoL results…) in regards of the HAS assessment (SMR, ASMR,…). In particular, clinical data submitted were confronted to HAS clinical studies requirements, to identify major trends concerning the evaluation of OS, QoL, and to identify exceptions to the rules.

RESULTS: 58 indications out of the 91 (64%) demonstrated an improvement in OS and/or PFS and/or QoL. Out of the 33 indications remaining the majority (54%) was granted an ASMR 5 often because they only had single arm trials and could not bring evidence of a gain of OS.

CONCLUSIONS: Pharmaceutical companies and the HAS have a different perception of value. In fact, the HAS does not recognize non comparative data nor a gain in OS if the median is not reached.