Shih Weichung J, Ouyang Peter, Quan Hui, Lin Yong, Michiels Bart, Bijnens Luc
University of Medicine & Dentistry of New Jersey, 335 George Street, Liberty Plaza Room 3456, New Brunswick, NJ 08903, USA.
Stat Med. 2003 Mar 15;22(5):665-75. doi: 10.1002/sim.1396.
The U.S. Food and Drug Administration (FDA) Modernization Act of 1997 has a Section (No. 112) entitled 'Expediting Study and Approval of Fast Track Drugs' (the Act). In 1998, the FDA issued a 'Guidance for Industry: the Fast Track Drug Development Programs' (the FTDD programmes) to meet the requirement of the Act. The purpose of FTDD programmes is to 'facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs'. Since then many health products have reached patients who suffered from AIDS, cancer, osteoporosis, and many other diseases, sooner by utilizing the Fast Track Act and the FTDD programmes. In the meantime several scientific issues have also surfaced when following the FTDD programmes. In this paper we will discuss the concept of two kinds of type I errors, namely, the 'conditional approval' and the 'final approval' type I errors, and propose statistical methods for controlling them in a new drug submission process.
美国1997年《食品药品监督管理局（FDA）现代化法案》中有一个章节（第112条）名为“加快快速通道药物的研究与审批”（该法案）。1998年，FDA发布了一份“行业指南：快速通道药物开发计划”（快速通道药物开发计划）以满足该法案的要求。快速通道药物开发计划的目的是“促进旨在治疗严重或危及生命疾病且显示出有潜力满足未满足医疗需求的新药的开发并加快其审评”。从那时起，许多健康产品通过利用《快速通道法案》和快速通道药物开发计划，更快地惠及了患有艾滋病、癌症、骨质疏松症及许多其他疾病的患者。与此同时，在遵循快速通道药物开发计划的过程中也出现了一些科学问题。在本文中，我们将讨论两种I类错误的概念，即“有条件批准”和“最终批准”I类错误，并提出在新药申报过程中控制这些错误的统计方法。
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