Controlling type I error rate for fast track drug development programmes

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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. Copyright © 2003 John Wiley & Sons, Ltd.