Abstract
In France, the cost-effectiveness analysis (CEA) submitted by manufacturers, to the French National Authority for Health (HAS), is reviewed by the Committee of Economic and Public Health (CEESP) which then leads to an “efficiency opinion”. ICERs from these economic evaluations allow the price fixing. However, the interpretation of ICERs is difficult in particular in the case of orphan diseases due to no willingness to pay threshold value and no characterized level of ICERs. The aim of this study is to assess ICERs of orphan diseases and critiques issued by the CEESP. This study was based on a developed database analyzing available “efficiency opinion” issued by the CEESP. This IQVIA board allows to analyze ICERs of orphan drugs and critiques issued by the CEESP. Orphan drugs were identified through transparency committee opinions. In France, 58 efficiency reports were published by the HAS between 2014 and 2018 including 12 orphan drugs in different therapeutic area: haematology treatments (N=4), cardiology treatments (N=2), oncology treatment (N=1), pneumology treatment (N=1), endocrinology treatment (N=1) and a musculoskeletal treatment (N=1). ICERs of orphan drugs ranged from 33,127€/QALY to 2,661,514€/QALY. Among the 4 heamatology drugs, only defibrotide was cost-effective with an ICER of 33,273€/QALY. For cardiology treatments, macitentan and riociguat indicated in pulmonary arterial hypertension had ICERs of 85,359€/QALY and 108,876€/QALY, respectively. However, only two ICERs of orphan drugs from French cost-effectiveness analyses have been criticized. These drugs were riociguat with an ICER of 108 876€/QALY and nusinersen sodium with an ICER of 2,661,514€/QALY. Both ICERs were considered extremely high by the CEESP despite no characterized level of ICERs. Although, no willingness to pay threshold value is clearly specified in France in 2018, efficiency opinions may be used by the Economic Committee for Medicinal Products (CEPS) for the price fixing.
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