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Challenges and potential strategies utilizing external data for efficacy evaluation in small-sized clinical trials

ABSTRACT In clinical trials for diseases with very small patient populations, trial investigators may encounter recruitment difficulties. It can be challenging to conduct clinical trials with enough power to detect a treatment effect, and randomization may not be feasible due to timeline, budget, and ethical concerns. To bring breakthrough therapies to the market quickly, it is important to come up with efficient approaches to utilizing individual patient data through improved study design and sound statistical methods. Emerging topics in this area include the use of Bayesian approaches to flexibly incorporate prior information into the current clinical trials, the use of historical controls to efficiently conduct trials that will reduce the number of subjects recruited and ease ethical considerations, and the use of innovative study designs, such as a platform design, to improve the efficiency and speed of the medical therapy development progress. In this paper, we describe three scenarios which highlight some of the challenges encountered in small-sized clinical trial development and provide potential statistical approaches to overcome the aforementioned challenges.