AAN 2021 | Should placebo-controlled trials be used in neuromuscular disease?

Nizar Souayah, MD, FAAN, Rutgers New Jersey Medical School, Newark, NJ, talks on the value of randomized placebo-controlled trials in neuromuscular disease. There is some debate on whether the benefits outweigh the negatives of randomized controlled trials (RCTs) in the setting of rare diseases since recruitment can be difficult and time-consuming. The use of placebos would delay treatment onset, which would have detrimental effects on the patient. Dr Souayah discusses the importance of RCTs and how trial designs could be altered to minimize the negative effects of placebo treatment. This interview took place during the American Academy of Neurology (AAN) 2021 Annual Meeting.