The Value of Detailed Clinical Trial Simulations for Rare Diseases

In this latest blog, Cytel statistician Ursula Garczarek discusses the contentious topic of measuring treatment effect during rare disease trials that must stimulate investigations using small samples. In contrast to statistically significant results, meaningful measures of treatment effect are still being developed for such small trials. Furthermore, in these types of trials, the summary statistic relies on many assumptions that might not correlate with reality Read more here.