Expanding Patient Access to Advanced Therapies.
Advanced therapy medicinal products (ATMPs) promise transformative clinical potential, but their use remains limited by complex manufacturing, high prices, and fragmented market access. To explore the existing and potential regulatory pathways to increase access to ATMPs, we conducted a product-level analysis of ATMPs and biologic medicines submitted to the European Medicines Agency up to May 2026, and a review of regulatory pathways and policy frameworks. While certain scientific principles from biologic and biosimilar regulation, such as totality of evidence, fit-for-purpose data, and tailored approaches are transferable, the biosimilar pathway may not be suitable for certain ATMPs. Other approaches, including bio-hybrid applications or prior knowledge-based dossiers, may be better suited to support competitive entry and second-wave innovation. Tailored follow-on pathways, open and well governed platform technologies, and supportive industrial and data sharing policies, are key to facilitate the translation of the scientific promise of ATMPs into sustainable and equitable patient access.