Overcoming obstacles in clinical-trial design: The MS experience

摘要

I'm going to start with just a general statement that I think we can all agree on which is that appropriate study design needs to include sufficient sample size and statistical power and control of bias to allow for a meaningful interpretation of results.I will go further and say that the ultimate goal of pivotal clinical trials, that is, trials conducted to obtain regulatory approval for use, is to provide definitive evidence regarding the benefit-to-risk profile of the experimental intervention respective to placebo or an existing standard of care intervention. Since it is the risk and benefit that must both be assessed, a clinically meaningful endpoint should be used.