Thamer M, Brennan N, Semansky R
Urban Institute, USA.
J Health Polit Policy Law. 1998 Apr;23(2):265-90. doi: 10.1215/03616878-23-2-265.
Six countries--Canada, France, Japan, Sweden, the United Kingdom, and the United States--were studied to compare public policies affecting the development and marketing of pharmaceuticals for rare diseases (i.e., orphan drugs). Information was obtained from a variety of published and unpublished sources, including interviews with public policy and pharmaceutical experts in each country. This article presents different approaches to encouraging the development of orphan drugs while ensuring access by regulating their prices. Additionally, the article describes access to orphan drugs as promoted by special coverage for population subgroups, disease categories, and/or specific drugs. Not all efforts to increase access to orphan pharmaceuticals have been the result of government action, as illustrated by the proliferation of for-profit organizations that specialize in orphan drugs. The many policy options from other countries identified in this study are especially relevant, given increasing calls for reform of the U.S. Orphan Drug Act.
对加拿大、法国、日本、瑞典、英国和美国这六个国家进行了研究，以比较影响罕见病药品（即孤儿药）研发和销售的公共政策。信息来自各种已发表和未发表的来源，包括对每个国家的公共政策和制药专家的访谈。本文介绍了在通过调控价格确保可及性的同时鼓励孤儿药研发的不同方法。此外，本文还描述了针对人群亚组、疾病类别和/或特定药物的特殊医保覆盖所推动的孤儿药可及性。并非所有增加孤儿药可及性的努力都是政府行动的结果，专门从事孤儿药业务的营利性组织的激增就说明了这一点。鉴于对美国《孤儿药法案》改革的呼声越来越高，本研究中确定的其他国家的众多政策选择尤其具有参考价值。
