Presentation (CTI)

OBJECTIVES: To compare post-marketing authorisation (MA), pre-reimbursement access pathways in Europe that provide access to innovative medicines for patients with unmet medical need (UMN). To identify the barriers and facilitators of such pathways and consider their relevance to the Irish reimbursement landscape.
METHODS: A comparative review was undertaken of post-MA, pre-reimbursement access pathways in high-income European countries. These pathways allow the enrolment of new patients following MA and until a reimbursement decision is reached. Nine pathways were identified across six countries: France's Accès Précoce and Direct Access pathways; Germany's AMNOG pathway; Italy's Law 648/96 and Class C(nn) pathways; Portugal's Programa de Acesso Precoce; Belgium's Early and Equitable Fast Access framework and Special Solidarity Fund; and the Netherlands' Drug Access Protocol. Data were extracted on pathway characteristics, including access type, funding, decision-making authority, duration of access, and reported barriers and facilitators.
RESULTS: Across countries, these pathways seek to address delays between regulatory approval and reimbursement for patients with UMN. Key facilitators include formal governance, public or statutory funding, defined eligibility criteria, continuity of treatment provisions, and integration with HTA processes. Germany’s AMNOG model facilitates rapid access immediately after MA, while France, Portugal and the Netherlands provide structured temporary reimbursement. Italy's Law 648/96 is both an early and off-label access pathway. Common barriers include administrative burden and complexity, variable regional implementation, limited real-world evidence generation. Belgium’s Solidarity Fund appears to be less predictable and less suitable as routine access mechanisms. While the C(nn) pathway supports early access post-MA, reimbursement uncertainties remain key barriers.Overall, these pathways highlight tensions between rapid access, evidence uncertainty, and potential health opportunity costs.
CONCLUSIONS: The experiences of European countries may offer considerations for policy discussions on facilitating earlier access to innovative medicines for patients with UMN in Ireland during the period between MA and reimbursement decisions.