Innovative Methods for Rare Disease Drug Development
这本书介绍了针对罕见病的临床试验设计、假设检验、终点选择等统计方法，适合学术界和工业界的研究人员以及相关专业学生参考。
Although it is rare for any individual to have a particular rare disease, there are over 7,000 rare diseases, and collectively rare disease affects countless individuals around the world, including more than 25-30 million individuals in the United States.Therefore, rare diseases are important to investigate.The book covers standard topics for a book on clinical trials (but tailored for rare disease applications), including study design, hypothesis testing, selection of endpoints, margin selection, probability of inconclusiveness of a study, probability monitoring procedure for sample size, real world data, n-of-1 trials, two-stage seamless adaptive designs, master protocol/platform studies, gene therapy, and finally provides a case study for a clinical development programme in a liver disease (non-alcoholic steatohepatitis).Dr Chow's writing style is very clear and concise.Sufficient mathematical detail is provided for readers to implement the methods, but the level of mathematics is appropriate for most applied researchers.In particular, I found the explanation of master protocols and platform trials very clear, and I would recommend this book as a reference for researchers wanting a succinct explanation of this topic.I highly recommend this book for investigators in both academia and industry planning to design clinical studies for rare diseases.It would also be useful for upper level undergraduate students, clinical investigators, graduate students from a variety of disciplines (including biostatistics, clinical investigation, epidemiology, and Master of Public Health).Overall, this book provides an excellent introduction to statistical methods for rare diseases.