Presentation (CTI)

OBJECTIVES: Early-access schemes enable patients to access promising therapies—particularly for cell and gene therapies— before full market authorisation. Real-world evidence (RWE) generated during these schemes can inform pricing and reimbursement outcomes. This review aims to examine how RWE from early-access settings is captured, appraised and used to support reimbursement decisions across UK, France, Germany, Italy, Spain.
METHODS: A targeted literature review was conducted. Eight peer-reviewed studies and two policy articles were included: two methodological overviews on early-access data capture, two empirical HTA reviews focused on rare-disease ATMPs, a value-framework primer anchored to the EUnetHTA Core Model, two policy analyses of conditional reimbursement mechanisms and a quantitative study of EU reimbursement timelines. Findings were consolidated thematically and mapped to areas of payer uncertainty: clinical effectiveness, durability, safety, budget impact, and societal value.
RESULTS: Clinical effectiveness and durability emerged as the most frequently cited drivers of access-related risk, with RWE most often positioned as a tool to address these gaps. This has been shown in EUnetHTA assessments (e.g., Zolgensma). Budget impact was also a recurring concern in policy-focused sources, while societal value appeared less consistently across the literature. Trends observed were: (1) Impact on coverage: In 37 EU/UK ATMP appraisals, early robust registry or observational data consistently helped de-risk payer decisions, speed up listings, and support outcomes-based payments. 2) Regulators vs. payers: EMA conditional approvals emphasised surrogate endpoints; national HTA bodies still demanded post-launch comparative RWE, particularly for long-term durability. 3) Data-capture infrastructure: early-access schemes with predefined RWE templates fed evidence directly into subsequent NICE evaluations, while ad-hoc data efforts often stalled.
CONCLUSIONS: Early-access programmes only bridge evidence gaps when RWE is prospectively planned, aligned with payer expectations, and tied to transparent managed-entry contracts. Harmonised data, validated controls, and clear HTA feedback are key to making early-access RWE a standard driver reimbursement.