OBJECTIVES: In Europe, an orphan disease is defined by a prevalence of less than 5 in 10 000 inhabitants which represent a maximum target population of 30 000 patients in France. Economic incentives are set up by authorities to encourage pharmaceutical development in rare disease treatments. This analysis aims at exploring French stakeholders’ policy toward orphan drugs on both Transparency Committee (TC) assessment and pricing decisions.

METHODS: All non-oncologic orphan drugs listed between 2006 and 2016 were identified. TC opinion (study design, comparator, target population) and price evolution were analyzed for each drug. First, results were compared to data for all drugs assessed in 2015, when available. Then, orphan drugs were clustered in two periods of time (2006-2010 and 2011-2016) in order to describe variations on assessment and price lifecycle.

RESULTS: CONCLUSIONS: In France, orphan drugs seem to benefit from a more favorable market access vis-a-vis drugs of all types regarding price evolution and HTA assessment. However, a strengthening of TC doctrine seems to emerge in the 5 past years despite similar level of demonstration.