To Placebo Or Not To Placebo? The Great Debate In Rare Disease Trials

By Jaclyn Brown and Abie Ekangaki

Sponsors of rare disease studies often struggle with small patient populations, geographic dispersion, competing studies, and more when working to recruit patients. As a result, clinical researchers are further pressured to design and implement studies that limit the burden of participation while keeping patients engaged and generating high-quality data that support regulatory approval.

While randomized, double-blind, placebo-controlled studies are considered the gold standard of clinical research, it can still be difficult to recruit patients with rare diseases into such trials. Some argue that it is necessary to prove the efficacy and safety of investigational treatments, while others believe that it is unethical to withhold treatment from patients.

Consider the severity of the disease, the availability of existing therapies, and more when deciding whether or not to use placebo control in rare disease trials.