External Control Arms as a Substitute for Placebo Groups

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While randomized controlled trials (RCTs) are the gold standard format of clinical trials, the need for a placebo group can extend development or even render their use impossible for rare diseases with small patient populations. The use of existing real-world data (RWD) and real-world evidence (RWE) in the form of electronic health records (EHR) and previous study data as an external control arm can help accelerate development, especially in rare diseases.

According to William Newton, “Going forward, experts say external arms could replace placebo arms in more and more RCTs. In a trial with an external control arm, patients receiving treatment are matched with patients from historical studies in the same indication and with similar baseline characteristics. As a result, trials can measure the relative safety and efficacy of a treatment to normal disease progression, without necessarily needing a placebo arm.”

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(Source: Clinical Trials Arena, September 29^th, 2022)