Methodological progress in the design of clinical trials for rare diseases

Abstract

Rare diseases have a low morbidity, include a wide variety of diseases, and involve a large number of people. However, because of the small sample size, high heterogeneity, and difficulty in recruiting, the research of rare diseases is difficult to carry out. This dilemma results in the lack of effective treatments for many rare diseases and the lack of high-quality efficacy and safety evidence of limited treatments. Therefore, it is of great significance to explore the design methods of clinical trials of rare disease. Recently, the breakthrough in methodology and the generation of new design methods provide a new clue for the clinical trials of rare diseases. The purpose of this paper is to introduce the innovative methods suitable for the clinical trials of rare diseases. We introduce four design methods according to their characteristics for researchers’ reference.