Methodological progress in the design of clinical trials for rare diseases
2018
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.
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