Early Phase Trial Designs in Rare Diseases

The development of novel therapies for rare diseases involves many challenges due to small and heterogeneous patient populations, limited knowledge of natural history data, ethical constraints, etc. This tutorial will focus on statistical methods for early phase clinical trials. It will start with some background on the drug development issues for rare diseases. After that, we will consider adaptive phase 1 trial designs that facilitate learning of the underlying dose–toxicity relationship while protecting study participants from exposure to overly toxic doses. We will discuss data analysis issues following these designs and the approaches for making decisions on the maximum tolerated dose (MTD). We will also cover adaptive phase 1/2 trial designs that incorporate toxicity and early efficacy (response) in dose-finding objectives and discuss the added value of such designs. Some additional important topics on early development clinical trials will be highlighted.

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