Development of efficient approaches to utilizing individual patient data (e.g., improved study designs and sound statistical methods) is instrumental in bringing breakthrough therapies to the market early for treating rare diseases. Adjustment to the general standards applied to clinical trials for common diseases may be necessary for trials for rare diseases, although consensus is required to clarify the approach that ensures both the quality of good trials and the efficacy of approved drugs.

In my presentation, I will discuss the issues and challenges encountered in the rare disease clinical trials, in particular for the inborn error products. Moreover, I will show how innovative designs and statistical analyses are utilized to assist drug sponsors in conducting successful clinical trials.