ABSTRACT
One of the most challenges for rare disease clinical trials is probably the availability of a small patient population. It is then a great concern how to conduct clinical trials with a small number of subjects available for obtaining substantial evidence regarding safety and effectiveness for approval of the rare disease drug product under investigation. The FDA, however, harbors no intention to create a statutory standard for approval of orphan drugs that is different from the standard for approval of more typical drugs. Thus, it is suggested that innovative trial designs, such as a complete n-of-1 trial design or an adaptive design, should be used for an accurate and reliable assessment of rare disease drug products. In this chapter, basic considerations, innovative trial designs, and statistical methods for data analysis are discussed. In addition, some innovative methods for evaluation of rare disease drug products are proposed.
