Presentation (CTI)

OBJECTIVES: To evaluate the value of France’s Early Access pathway—Autorisation d’Accès Compassionnel (AAC) from a Biotech industry perspective, highlighting its role in accelerating patient access and informing clinical strategies for novel therapies in areas of high unmet need.
METHODS: Real-world data (RWD) are collected during the AAC process for the investigational antifungal therapeutic fosmanogepix (Basilea Pharmaceutica Int. Ltd) targeting difficult to treat invasive fungal infections. Anonymized data include patient demographics and relevant clinical characteristics enabling descriptive analyses and comparison to data from interventional and observational clinical studies to assess early signals of effectiveness and disease burden. Operational and strategic insights are gathered through cross-functional stakeholder engagement and regulatory interactions.
RESULTS: The AAC pathway in France enables early, funded access to fosmanogepix for patients with invasive fungal disease and limited treatment options. RWD collection provides potentially valuable insights into real-world treatment patterns and outcomes. Early engagement with the French regulatory agency (ANSM) facilitated alignment on data expectations and supporting future evidence-based strategies and plans. Enablers of success included proactive regulatory planning and readiness of required logistics to coordinate timely provision of fosmanogepix to patients, enable data capture across various sites requesting fosmanogepix and to address administrative challenges.
CONCLUSIONS: Beyond patient access, France’s AAC pathway offers additional value, enabling sponsors to generate early evidence, build regulatory relationships, and prepare for market entry. Early engagement with national access frameworks can be a valuable component in drug development of therapies addressing high unmet clinical needs. Integrating early access strategies into broader Health Economics and Outcomes Research (HEOR) and market access planning is a key aspect in this Health Policy and Regulatory Research.