Antiviral Medicines Assessment: What Is Driving High Clinical Added Value In France?

OBJECTIVES: to analyse the basis of clinical added value (CAV) appraisal regarding the high cost of new medicines including hepatitis C treatment In France. The CAV appraisal is done by the French National Authority for Health (HAS) on a 5-point scale from I (major) to V (no CAV). The CAV level is partly used by the economic committee for healthcare products to negotiate drug prices. METHODS: a retrospective and descriptive study analysing HAS appraisals of all new antiviral indications assessed between 2010 and 2015 were conducted. For each CAV appraisal, information regarding the level of evidence and clinical effectiveness was collected. RESULTS: Twenty antiviral drugs (47 indications) were assessed. All obtained a favourable opinion for reimbursement with 11 a major to moderate CAV, 14 a minor and 22 no CAV. No one obtained a major CAV The majority of studies [46% (22/47)] were non-active comparator studies based on virological response and supported an important to moderate CAV in 32% (7/22), as sofosbuvir that granted the highest CAV level (important CAV). Among the 19 comparative trials, 68% (13/19) were of superiority design supporting a major to moderate CAV in 31% (4/13) of the cases. A higher proportion of important to moderate CAV were observed in trials using a relevant primary endpoint (26%) versus no one for a study using an exploratory endpoint such as pharmacokinetic endpoint. CONCLUSIONS: A high proportion (53%) of CAV is recognized in virology compared to the other therapeutic areas (15%) while non-active comparator studies are made. This report shows that HAS appraisals remain multi-factorial. HAS' expectations in terms of level of evidence take into account the medical need, therapeutic area specificity and a rapid evolution of the therapeutic strategy. The emergence of anti-viral drug resistance can also influence the CAV appraisal.