Utilizing A Registry To Inform An External Control Arm

Randomized clinical trials are the gold standard for the evaluation of experimental interventions. In randomized clinical trials, patients are randomized to either an experimental intervention arm or a control arm that consists of placebo or standard of care alone.

Patient recruitment and retention are two key factors for the success of any trial, particularly so when there is a significant unmet clinical need and the study is potentially transformative. In this circumstance, the use of a control arm in which participants do not receive the experimental intervention can impose recruitment and retention challenges as well as potential ethical challenges. Patients have been shown to be less willing to participate in placebo-controlled randomized clinical trials in part due to the chance of receiving placebo.¹

 The ability to establish a simulated control arm based upon data present within an external, accessible historical database of patients, as an example, is an important step in clinical trial design, particularly for rare diseases.

Ultimately, for the patients involved in the new study, distinct benefits arise from use of an external control. All active trial participants receive the test product, which increases the likelihood of patient and physician buy-in. From a sponsor’s perspective, a trial may require fewer patients than a traditionally randomized trial because the external control is effectively acting as the arm randomized to placebo. When correctly planned, informed, and executed the utilization of external controls and the resulting analyses have the ability to provide compelling, potentially pivotal investigations with benefit to both sponsors and the patients alike.

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