PBI18 The Global EARLY Access Programme Landscape and Its Application to Cell and GENE Therapies

To identify and analyse the global early access program (EAP) landscape, assessing different national framework’s applicability for cell gene therapies. A targeted systematic literature review was conducted to EAPs across markets: Africa; Asia; Europe; North America; Australia South America. National healthcare agency websites were used compile information relating guidelines, laws criteria of specific EAPs. EAP therapies assessed, considering potential reimbursement individual patient through these schemes. The heterogeneity landscape is significant rapidly evolving. Various paradigms currently exist, utilising a variety regulatory frameworks criteria, but all share goal ensuring patient’s timely potentially lifesaving Of 57 countries included in this analysis, 34 have well defined, established place. European particular carefully defined EAPs, (present 16 out 25 countries, including EU5 markets). In contrast, medicines Asian American markets still an evolutionary phase; discerning details degree scheme challenging due paucity uncertainty information. 11 with offered treatment. 12 had schemes ambiguous or highly conditional reimbursement. Manufacturers rare diseases may find be unfeasible, as facilitating their without reimbursing can greatly diminish cohort patients, especially one-time treatments. Taking holistic view Europe presents most attractive option manufactures seeking diseases, formalised pathways reimbursed prior market authorisation.