Navigating The Risks: Why Compassionate Use Should Not Replace Rigorous Clinical Trials

By Gabriel Kremmidiotis, Chief Scientific Officer and Alex Kavros, Executive Vice President, Scientific & Regulatory Affairs

Compassionate Use and Expanded Access programs offer critical access to investigational therapies for patients with severe, life-threatening conditions. However, as alternatives to traditional clinical trials, these programs raise concerns regarding scientific rigor, regulatory compliance, and ethical standards. While they provide lifesaving treatments outside clinical trials, the lack of structured protocols can lead to inconsistent data, compromising efficacy and safety evaluations.

Regulatory agencies, such as the FDA and TGA, oversee these programs, but the added complexity can delay treatment initiation. Moreover, compassionate use may disrupt clinical trial recruitment, skew trial outcomes, and burden sponsors operationally. Ethical challenges also arise, as patients may not fully understand the experimental nature of treatments. Despite these challenges, compassionate use programs remain essential for addressing urgent medical needs but should complement, not replace, rigorous clinical trials. High-quality evidence from clinical trials is crucial for regulatory approval, broader access, and integration into clinical practice. A balanced approach ensures that compassionate use pathways serve their intended purpose without undermining the integrity of the drug development process.