On Rare Disease Day, we highlight the potential of Bayesian statistics to overcome recruitment challenges in rare disease trials. In an article for ACRP Clinical Researcher, Giles Partington, Principal Statistician at Phastar, explores how incorporating prior data and expert judgment can reduce sample size requirements, improve efficiency, and increase trial success rates.
”There are between 263 million and 446 million people worldwide living with rare diseases at any one time. It is vital that we optimize rare disease clinical trials by enabling effective and efficient data analysis. Bayesian statistics can and should be more widely used to support research innovations, increase trial success rates, and help those living with rare diseases.”