Presentation (CTI)

OBJECTIVES: Health technologies often launch amid uncertainty, making post‑launch evidence generation critical for payer and regulatory decisions. Lifecycle Health Technology Assessment (HTA) is an iterative approach that reassesses technology’s value over time, using real-world evidence (RWE) and stakeholder input. However, integration of RWE and patient‑reported outcomes (PROs) remain inconsistent. This study evaluated how major HTA agencies integrate RWE and PROs within lifecycle HTA processes to address post-launch uncertainty.
METHODS: Targeted review of publicly available appraisal and reassessment documents from NICE, HAS, CADTH, and ICER (2021-2025) was conducted. Evaluations were included if they demonstrated at least one lifecycle feature (managed-access or scheduled re‑evaluation, evidence‑update requirements), and documented RWE/PRO use. A standardized analytic matrix compared access frameworks, RWE source categories, PRO instrument presence, stopping rules, and cost‑effectiveness reassessment. Analyses were descriptive given heterogeneity; counts were reported.
RESULTS: Forty evaluations met inclusion criteria (NICE: 15, HAS: 12, CADTH: 6, ICER: 7), with 53% (n=21) targeting rare diseases. All evaluations incorporated PROs and utilized RWE sources to inform lifecycle assessments. Dominant RWE sources were clinical trial/extension data (n=17), registries (n=15), observational studies (n=14), and National Health Service datasets (n=11). Formal managed‑access frameworks appeared in 14/40 evaluations (primarily NICE: 12; HAS: 2). Cost‑effectiveness reassessments occurred in 18/40 (NICE: 8, ICER: 7, HAS: 3). Stopping/continuation rules were reported in 29/40. Patient/equity input was explicit in 33/40 evaluations. Cross‑agency comparisons showed NICE and ICER applied the structured lifecycle mechanisms (managed access and scheduled reassessment), while HAS emphasized stopping rules and registry follow‑up; CADTH combined conditional reimbursement with registry planning, and universal patient/clinician input.
CONCLUSIONS: Lifecycle integration of RWE and PROs is heterogeneous across HTA agencies. PROs were consistently incorporated, whereas RWE adoption varied markedly. NICE and ICER lead with formal managed-access and reassessment pathways; HAS and CADTH increasingly embed registry-based approaches for high-cost innovations. Harmonized post-launch evidence frameworks are essential for reducing uncertainty and supporting value-based decisions.