Single-Arm Trials Using Real-World Evidence For Rare Disease Product Development

By Ruth Stevens, PhD, MBA, Strategic Advisor

As a sponsor designs clinical studies, the respective comparison control groups become a critical factor to consider. Often, to gain clinical trial design insights, a sponsor reviews the physician package inserts from approved New Drug Applications (NDAs) and Biologics License Applications (BLAs) with similar indications or in the same therapeutic area. If a single-arm trial was conducted as the pivotal trial for regulatory approval of those existing products, the sponsor will certainly want to do the same.

Similarly, in rare disease drug development, where a placebo control group may not be an option, a single-arm trial can be a successful approach.