Considerations of Practical Study Designs for Rare Disease Drug Development
Mingxiu Hu, Takeda Pharmaceuticals  Wei Huang, RedBudTek Inc  Serap Sankoh, Takeda Pharmaceuticals  *Huyuan Yang, Takeda Pharmaceuticals International Co 

Keywords: Rare Disease drug development, Frequentist method, Bayesian design

With the rapidly growing development in rare disease drug, the adaptation of sensible clinical trial designs becomes the most challenging task to both investigator/sponsors and regulatory. Given the sample size limit of rare disease patient population, the conventional statistical designs and rules of clinical trials are not suitable anymore. Frequentist method based on historical control or crossover patient self-control have been considered to perform hypothesis testing in controlling type I/II error. However, the underline assumptions for these designs can be easily violated. Bayesian adaptive design, as been used more and more in early phases in drug development, has now been considered in confirmatory trials for rare disease clinical trials. In this talk we will go over these statistical considerations and illustrate with real and simulated examples.