Evaluating and improving orphan drug regulations in Europe: a Delphi policy study.

To encourage the development of orphan drugs, the European Union has implemented specific policies in 2000. However, the political, social, scientific and economic context has changed since the implementation of these policies. For that reason, the aim of this article is to evaluate orphan drug policies in Europe. Firstly, key issues on the orphan drug policy were identified based on desk research. Secondly, a Delphi policy study with 47 European orphan drug experts from different backgrounds was carried out to explore these issues. In the round one of the Delphi, responses were received from 18 experts (38.3%) and from ten (55.5%) in the round two. Experts agree that the orphan drug policies in Europe have not outlived their usefulness. Additionally, the importance of reducing country-dependent inequalities in patient access to orphan drugs has been emphasized. Still, there is room for further refinement of the orphan drug policies. Within that context, we formulated several policy recommendations (e.g. enforcing the policy that is in place to reduce the period of market exclusivity for profitable orphan drugs, stating the level of clinical evidence needed to authorize orphan drugs, etc.) with the overall goal to optimize patient access to orphan drugs.