Romanian Society of Pharmaceutical Sciences
 

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COMPASSIONATE USE ACCESS FOR PATIENTS TO EXPENSIVE DRUGS IN THE EARLY STAGES OF THEIR DEVELOPMENT

JOVANA DIMISHKOVSKA 1*, KRUME BOGEVSKI 2, ZORICA NAUMOVSKA 1

1 Institute of Pharmaceutical Chemistry, Faculty of Pharmacy, Ss. Cyril & Methodius University, Majka Tereza 47, 1000 Skopje, North Macedonia
2 Faculty of Medical Sciences, Goce Delcev University of Stip, Krste Misirkov 10A, 2000 Stip, Republic of North Macedonia

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The term “compassionate use” refers to individual access to drugs in the developmental phase for patients not involved in clinical trials, treating serious and life-threatening conditions. Defined by the European Medicines Agency (EMA), it involves the use of unapproved drugs in strictly controlled conditions. Coordinated by EU member states, these programs assist patients with life-threatening illnesses, operating independently with initiation based on domestic rules. Doctors must adhere to EU and member state procedures, while regulatory agencies maintain patient registers and manage adverse effect tracking. Compassionate use provides access to drugs in developing countries and fosters collaborations between manufacturers, specialists, and institutions. However, it raises concerns about potential market dominance during the “expanded access programs” phase, which aims to gather experiences rather than data, especially valuable for rare diseases. The resulting clinical information is recognized as “real-world information” offering insights beyond clinical trials' rigid criteria.