OBJECTIVES : Since 2013, innovative products claiming an added clinical benefit (ASMR) level ≤III with a substantial impact on healthcare expenditures (≥€20 million the second year) are evaluated by the French economic and public health assessment committee (CEESP). The study objective was to review and analyse the CEESP opinions in oncology indications and to summarize the major concerns.

METHODS : All available CEESP opinions for oncology indications published until May 2019 were reviewed. Specific indications, nature of the request, model type used for cost-effectiveness analysis, and methodological concerns were extracted.

RESULTS : Overall, 38 CEESP opinions out of 88 opinions published on the HAS website were based on cancer treatment assessment (43%), including 14 in hematology (37%), with 71% of them being intended for first inscription (10/14). One third (8/24) of the reports evaluated in solid tumors (12 for extension and 9 for first inscription) were for lung cancers. The majority of the manufacturers used a partitioned survival model to assess the cost-effectiveness of their product (27/38). 64% (9/14) of the reports in hematology received ≥1 major concern, vs. 21% (5/24) of the opinions in solid tumors. Half of a total of 22 major concerns related to lack of robustness and uncertainty of clinical effectiveness. Lack or inconsistency in relevant strategies lead to a major concern in 3 reports. Other major concerns were associated to non applicability of health outcomes (3/22), non relevant simulated population (2/22), lack of sufficient sensitivity analyses (1/22), poor quality of utility data (1/22) or anonymity of the independent clinical experts solicited (1/22).

CONCLUSIONS : While cancer remains the main area for innovative products, oncology therapies face challenges around uncertainty in the clinical effectiveness evidence. The specificities of the evidence that is available for innovative cancer drugs, in particular immunotherapies, may require special consideration within HTA processes.