Research Centre for Pharmaceutical Care and Pharmaco-economics, KU Leuven, Herestraat 49 P.O. Box 521, 3000 Leuven, Belgium.
Health Policy. 2012 Nov;108(1):1-9. doi: 10.1016/j.healthpol.2012.08.023. Epub 2012 Sep 16.
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.
为鼓励孤儿药的开发，欧盟于 2000 年实施了特定政策。然而，自这些政策实施以来，政治、社会、科学和经济环境已经发生了变化。因此，本文旨在评估欧洲的孤儿药政策。首先，通过案头研究确定了孤儿药政策的关键问题。其次，对来自不同背景的 47 名欧洲孤儿药专家进行了德尔菲政策研究，以探讨这些问题。在第一轮德尔菲中，收到了 18 名专家（38.3%）的回复，在第二轮中收到了 10 名（55.5%）的回复。专家们一致认为，欧洲的孤儿药政策仍有其作用。此外，强调了减少患者获得孤儿药的国家间不平等的重要性。尽管如此，孤儿药政策仍有进一步完善的空间。在这种情况下，我们制定了一些政策建议（例如，执行旨在减少盈利性孤儿药市场独占期的政策，规定授权孤儿药所需的临床证据水平等），其总体目标是优化患者获得孤儿药的机会。
