Session (CTI)

ISSUE: While Early Access Programs (EAPs) remain focused on providing compassionate access for patients with serious conditions lacking therapeutic alternatives, price and reimbursement stakeholders increasingly debate whether EAPs should generate real-world evidence for HTA evaluations. This dual-purpose expectation creates fundamental tensions: programs designed for urgent medical access lack the rigorous data collection infrastructure of clinical trials, potentially yielding evidence that HTA bodies may consider insufficient for reimbursement decisions. As EU-HTA standardizes evidence requirements, the question becomes whether EAPs can legitimately support pricing and reimbursement decisions without compromising their primary humanitarian mission. OVERVIEW: Kieron Lewis will provide a 10-minute overview examining how different EAP implementation models impact pricing and reimbursement strategies. While EAP data may inform EU-HTA PICO determinations and contextualize patient populations, manufacturers risk significant financial exposure when funding programs that regulators may later restrict. Varying reimbursement models across European jurisdictions create further complexity and may lead to inequitable access patterns. Mandatory safety monitoring adds complexity to programs already struggling to balance compassionate access with data quality. The panel will explore contrasting perspectives: whether EAPs should maintain their primary focus on compassionate access with minimal data requirements beyond safety monitoring, or evolve to incorporate structured data collection that could support HTA evaluations. Discussion will address the practical challenges manufacturers face in balancing patient access objectives with increasing pressure to generate reimbursement-grade evidence, while managing associated financial risks. Following 35 minutes of panel debate, 15 minutes of audience discussion will explore implementation challenges and ethical considerations. Key stakeholders include regulatory agencies, HTA bodies, pharmaceutical companies, ethics committees, and policymakers navigating patient access versus evidence requirements.