Compassionate Use Programs: An Overview of the Global Landscape
Compassionate use serves as a useful pathway to provide access to unauthorized medicines to patients that are otherwise unable to participate in clinical trials, fulfilling an unmet medical need. These programs vary widely across the globe, from regulatory definition/terminology to submission and safety reporting requirements. For example, some countries may restrict this use to an individual (also referred to as ‘named patient’), in which an application needs to be lodged for every patient the healthcare professional wishes to prescribe the unauthorized medicine for; in other cases, some countries do not have any regulatory frameworks for such programs, leading to gaps in access to critical care. This white paper explores the global landscape of compassionate use, highlighting similarities and differences between programs across different countries.
