Compassionate Use

A regulatory pathway that allows patients with serious or life-threatening conditions to access investigational drugs outside of clinical trials when no comparable alternatives exist. Also known as Expanded Access in the US. Requires physician application, sponsor agreement, and FDA/IRB authorization.

Ví dụ

• Remdesivir expanded access during COVID-19 pandemic
• Experimental CAR-T therapy for refractory leukemia
• EU compassionate use for gene therapies