OBJECTIVES: To compare the outcomes of HTA for oncology orphan drugs approved in Europe. Three countries were selected to represent the following cases: HTA driven by cost-effectiveness (England), based on clinical value (France), and a country with a special HTA procedure for orphan drugs (Germany).
METHODS: HTA reports for orphan drugs approved by the European Medicines Agency from 2003-2016 were retrieved from the websites of respective national HTA agencies
RESULTS: In France, almost all oncology orphan drugs available in Europe have been assessed by the agency and have obtained a positive recommendation for reimbursement.
In Germany, the additional benefit was non-quantifiable for all oncology orphan molecules except four drugs whose additional benefit was considered minor and two drugs whose additional benefit was estimated to be considerable.
In England, among 33 assessed drugs only 14 achieved a positive recommendation, and in another 8 assessments the indication was restricted to a smaller population. Fifteen recommendations involved a Market Access Agreement with the manufacturer (PAS), such as e.g. a price discount.
CONCLUSIONS: A HTA process based on clinical value of the orphan drug (France) has resulted in the highest rate of positive reimbursement recommendations. The special pathway in Germany has also led to a high rate of positive recommendations and enabled effective price negotiations. In contrast, a process based on cost-effectiveness (England) has led to the lowest rate of positive recommendations, even though in most cases a cost-reducing PAS was applied.