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

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. RezumatTermenul "utilizare compasional" se refer la accesul individual la medicamente n faza de dezvoltare pentru pacienii care nu sunt implicai n studiile clinice, care trateaz afeciuni grave i care pun viaa n pericol.Definit de Agenia European a Medicamentelor (EMA), sintagma implic utilizarea de medicamente neaprobate n condiii strict controlate.Coordonate de statele membre ale UE, aceste programe ajut pacienii cu boli care pun viaa n pericol, funcionnd n mod independent, cu iniiere bazat pe regulile interne.Medicii trebuie s adere la procedurile UE i ale statelor membre, n timp ce ageniile de reglementare menin registrele pacienilor i gestioneaz monitorizarea efectelor adverse.Utilizarea compasional ofer acces la medicamente n rile n curs de dezvoltare i ncurajeaz colaborrile ntre productori, specialiti i instituii.Cu toate acestea, ridic ngrijorri cu privire la poteniala dominaie pe pia n timpul fazei de "program de acces extins", care i propune s colecteze experiene mai degrab dect date, valoroase mai ales n cazul bolilor rare.Informaiile clinice rezultate sunt recunoscute ca "informaii din lumea real", oferind perspective dincolo de criteriile rigide ale studiilor clinice.