OBJECTIVES: Since the release of October 2^nd, 2012 French decree requiring economic evaluation for innovant drugs, around 130 efficiency opinions were published by the French Health Authority “HAS/CEESP”. Among them, 18 were in hemato-oncology. Objectives were to identify the choices made by pharmaceutical manufacturers and the objections raised by the HAS, to raise improvement areas in health economic evaluation in hemato-oncology in France.

METHODS: We analyzed the 18 opinions which evaluated 9 drugs: axicabtagene ciloleucel(1), carfilzomib(1), vénétoclax(2), daratumumab(1), ibrutinib(3), idelalisib(3), obinutuzumab(3), midostaurine(2), tisangenlecleucel(2) and venetroclax(2) in different indications: leukemias, lymphomas, multiple myeloma and systemic mastocytosis. Evaluation was conducted by the HAS/CEESP between April 2015 and April 2019. In each of the 18 opinions, the acceptability level was analysed in terms of major and important methodological caveats.

RESULTS: Opinions covering leukemias raised the highest number of major caveats(15) with 12/15 in chronic lymphoblastic leukemia. Lymphomas’ opinions raised 5 major caveats while systemic mastocytosis’ one raised only one. Multiple myeloma opinions raised no major caveats. Regardless of indication, data sources and probabilities estimation were the methodological settings leading to the highest number of both major(12) and important caveats(14). The main major ones were related to available data not allowing long-term extrapolation(5) and to indirect-comparison related uncertainty(5).The other methodological settings raising either major or important caveats dealt with lack of population representativity(6), omission of relevant comparators(5), uncertainty related to utility data(4), results transposability(1) and anonymity of clinical experts(1).

CONCLUSIONS: The HAS/CEESP concluded that only three dossiers demonstrated the efficiency; the ICER was partially validated in one dossier and 14 opinions were not validated due to major caveats. The analysis of the objections allowed to highlight the parameters on which pharmaceutical manufacturers need to be more robust and rigorous, for cost effectiveness analyses in hemato-oncology.