Incentives for orphan drug research and development in the United States
Incentives for orphan drug research and development in the United States
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DOI：
10.1186/1750-1172-3-33
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发表时间：
2008-12-16
影响因子：
3.7
通讯作者：
Visaria, Jay
中科院分区：
文献类型：
--
作者：
Seoane-Vazquez, Enrique;Rodriguez-Monguio, Rosa;Visaria, Jay
背景资料：《孤儿药法案》（1983年）制定了几项激励措施，以鼓励开发孤儿药（OD）来治疗罕见疾病和病症。本研究分析了1983 - 2007年美国孤儿药新分子实体（NME）的OD名称、批准、申办者的特征，并评估了其有效专利和市场独占期。方法：主要数据来源于FDA橙子手册、FDA孤儿药开发办公室和美国专利商标局。数据包括FDA列出的所有孤儿药指定和批准以及FDA在研究期间批准的所有NME。结果：FDA在1983年至2007年期间列出了1，793项孤儿药指定和322项批准。癌症是孤儿药批准的主要目标疾病。83家公司集中了孤儿NME批准总数的67.7%。从孤儿药认定到FDA批准的平均时间为4.0 +/- 3.3年（平均值+/-标准差）。孤儿NME的平均最大有效专利和市场独占期为11.7 +/- 5.0年。OD市场排他性增加了平均最大有效的专利和市场排他性寿命的ODs.Conclusion：公共计划，联邦法规和政策支持孤儿药的R&D。赠款、研究设计支持、FDA费用减免、税收优惠和孤儿药市场独占权是孤儿药研发的主要激励因素。尽管7年孤儿药市场独占条款对有效专利和市场独占期的总体影响相对较小，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平台。
Background: 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.Methods: 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.Results: 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.Conclusion: 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.