title: Controlling type I error rate for fast track drug development programmes.
abstract: 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.
authors.affiliation: University of Medicine & Dentistry of New Jersey, 335 George Street, Liberty Plaza Room 3456, New Brunswick, NJ 08903, USA. shihwj@umdnj.edu
authors.forename: Weichung J
authors.initials: WJ
authors.lastname: Shih
authors.forename: Peter
authors.initials: P
authors.lastname: Ouyang
authors.forename: Hui
authors.initials: H
authors.lastname: Quan
authors.forename: Yong
authors.initials: Y
authors.lastname: Lin
authors.forename: Bart
authors.initials: B
authors.lastname: Michiels
authors.forename: Luc
authors.initials: L
authors.lastname: Bijnens
mesh_terms: D017326:Clinical Trials, Phase III as Topic / Q000379:methods; D018450:Disease Progression; D017277:Drug Approval / Q000331:legislation & jurisprudence / Q000379:methods*; D006801:Humans; D004364:Pharmaceutical Preparations / Q000592:standards; D011358:Product Surveillance, Postmarketing; D012107:Research Design; D013223:Statistics as Topic / Q000379:methods*; D016019:Survival Analysis; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D004364:Pharmaceutical Preparations