Seoane-Vazquez Enrique, Rodriguez-Monguio Rosa, Szeinbach Sheryl L, Visaria Jay
Division of Pharmacy Practice & Administration, College of Pharmacy, Ohio State University, Columbus, Ohio, USA.
Orphanet J Rare Dis. 2008 Dec 16;3:33. doi: 10.1186/1750-1172-3-33.
The Orphan Drug Act (1983) established several incentives to encourage the development of orphan drugs (ODs) to treat rare diseases and conditions. This study analyzed the characteristics of OD designations, approvals, sponsors, and evaluated the effective patent and market exclusivity life of orphan new molecular entities (NMEs) approved in the US between 1983 and 2007.
Primary data sources were the FDA Orange Book, the FDA Office of Orphan Drugs Development, and the US Patent and Trademark Office. Data included all orphan designations and approvals listed by the FDA and all NMEs approved by the FDA during the study period.
The FDA listed 1,793 orphan designations and 322 approvals between 1983 and 2007. Cancer was the main group of diseases targeted for orphan approvals. Eighty-three companies concentrated 67.7% of the total orphan NMEs approvals. The average time from orphan designation to FDA approval was 4.0 +/- 3.3 years (mean +/- standard deviation). The average maximum effective patent and market exclusivity life was 11.7 +/- 5.0 years for orphan NME. OD market exclusivity increased the average maximum effective patent and market exclusivity life of ODs by 0.8 years.
Public programs, federal regulations, and policies support orphan drugs R&D. Grants, research design support, FDA fee waivers, tax incentives, and orphan drug market exclusivity are the main incentives for orphan drug R&D. Although the 7-year orphan drug market exclusivity provision had a positive yet relatively modest overall effect on effective patent and market exclusivity life, economic incentives and public support mechanisms provide a platform for continued orphan drug development for a highly specialized market.
《孤儿药法案》（1983年）设立了多项激励措施，以鼓励开发用于治疗罕见疾病和病症的孤儿药（ODs）。本研究分析了孤儿药指定、批准、申办方的特征，并评估了1983年至2007年间在美国获批的孤儿新分子实体（NMEs）的有效专利和市场独占期。
主要数据来源为美国食品药品监督管理局（FDA）橙皮书、FDA孤儿药开发办公室以及美国专利商标局。数据包括FDA列出的所有孤儿药指定和批准信息，以及研究期间FDA批准的所有NMEs。
1983年至2007年间，FDA列出了1793项孤儿药指定和322项批准。癌症是孤儿药获批的主要疾病类别。83家公司集中了全部孤儿NMEs批准的67.7%。从孤儿药指定到FDA批准的平均时间为4.0 +/- 3.3年（均值 +/- 标准差）。孤儿NME的平均最长有效专利和市场独占期为11.7 +/- 5.0年。孤儿药市场独占性使ODs的平均最长有效专利和市场独占期增加了0.8年。
公共项目、联邦法规和政策支持孤儿药研发。资助、研究设计支持、FDA费用豁免、税收激励以及孤儿药市场独占性是孤儿药研发的主要激励因素。尽管7年的孤儿药市场独占性条款对有效专利和市场独占期产生了积极但相对适度的总体影响，但经济激励和公共支持机制为高度专业化市场的孤儿药持续开发提供了一个平台。
