EMA has opened a public consultation on a 
reflection paper that discusses key concepts for single-arm clinical trials that are submitted as pivotal evidence in support of marketing authorisation applications for medicines in the European Union (EU). This is the first guidance document by an international medicine regulator articulating the considerations and challenges associated with this type of clinical trials. Stakeholders are invited to send their comments via an online form by midnight (CET) on 30 September 2023.
Randomised clinical trials (RCTs) in which a new treatment is compared against a placebo or an existing standard of care are widely considered as the gold standard for generating evidence needed by regulatory authorities to assess the efficacy and safety of a new medicine. In RCTs, patients are randomly assigned to either the active treatment or the control arm. Usually, large numbers of patients are included for these trials to generate robust data on the efficacy of a treatment…
