Single-Arm Trials in Oncology: Key Insights from the Latest EMA and FDA Guidance

In the fast-paced realm of oncology drug development, the quest for expeditious approval pathways has reached unprecedented heights. Over the past years, there has been a seismic shift towards streamlined processes that enables the swift authorization of oncology medicines. Notably, the United States has embraced accelerated approval, while the European Union has pioneered the concept of conditional approval. These regulatory pathways, often reliant on single pivotal trials, including the innovative single-arm trial (SAT) design, have emerged as prominent avenues for rapidly advancing cancer therapies.

Recently released draft guidance documents from the EMA and FDA in 2023 provide valuable regulatory insights on SATs. This white paper, written by Adriana Andrić, Senior Consultant at NDA Group, explores the regulatory guidance considerations in relation to the design, planning, conduct, analysis, and interpretation of results derived from SATs.