Murakami Manabu, Narukawa Mamoru
Department of Clinical Medicine (Pharmaceutical Medicine), Kitasato University Graduate School of Pharmaceutical Sciences, Shirokane 5-9-1, Minato-ku, Tokyo 108-8641, Japan.
Department of Clinical Medicine (Pharmaceutical Medicine), Kitasato University Graduate School of Pharmaceutical Sciences, Shirokane 5-9-1, Minato-ku, Tokyo 108-8641, Japan.
Drug Discov Today. 2016 Apr;21(4):544-9. doi: 10.1016/j.drudis.2016.02.016. Epub 2016 Mar 2.
Orphan drugs have become a key area of focus in drug development for resolving unmet medical needs. The Orphan Drug Act in the USA and similar legislation in Japan, the European Union (EU), and several other countries has been enacted since 1983. This study provides a quantitative review of all orphan drug designations and approvals since the implementation of orphan drug legislation in key three regions. This study also identified and reviewed 'commonly designated' drugs across regions. Out of approximately 5000 designations, approximately 800 designations were common among the USA, EU, and/or Japan. Regional similarities, differences, and trends were identified. It is important to understand these aspects and the crucial role of orphan drug designation in global drug development.
孤儿药已成为药物研发中解决未满足医疗需求的关键重点领域。自1983年以来，美国的《孤儿药法案》以及日本、欧盟（EU）和其他几个国家的类似立法相继颁布。本研究对关键三个地区实施孤儿药立法以来的所有孤儿药指定和批准情况进行了定量综述。本研究还识别并审查了各地区的“常见指定”药物。在大约5000个指定中，约800个指定在美国、欧盟和/或日本之间是共有的。研究确定了地区间的异同及趋势。了解这些方面以及孤儿药指定在全球药物研发中的关键作用非常重要。
