Orphan drug considerations in Health Technology Assessment in eight European countries

David Tordrup, Victoria Tzouma, Panos Kanavos


Health Technology Assessment (HTA) is used to assess the value of new technologies and by producing coverage recommendations it indirectly controls the uptake of new technologies in many European countries. While HTA generally relies on a robust estimation of the clinical cost-effectiveness of a new technology, the clinical and economic evidence required for this purpose is often not available for Orphan Drugs (ODs), partly because of challenges related to the recruitment of patients to participate in clinical trials. A number of European HTA agencies have started to implement specific policies to address the challenges related to evidence requirements for the case of ODs. In this study, we map out the policies currently in place in eight European countries regarding HTA and its application to the case of ODs and explore the implications these policies have for coverage decisions.


health technology assessment, rare diseases, orphan drugs, health systems, comparative health policy, risk sharing


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