Presentation (CTI)

OBJECTIVES: This research aims to evaluate PRO acceptance in EU5 HTA frameworks, identify misalignments between HTA and patient-prioritised outcomes across therapeutic areas, and assess how recent policy advancements may enhance patient-aligned health technology development.
METHODS: HTA guidelines and reports from HAS, GBA/IQWiG, NICE, AIFA, and AEMPS were reviewed to assess the role of PROs in value assessments. Patient-prioritised outcomes were identified through targeted literature review, including burden of disease studies and advocacy group surveys, and a comparative analysis was performed across oncology, autoimmune disease, neurology, and rare diseases. Recent patient-centric policy developments and initiatives were also reviewed.
RESULTS: GBA/IQWiG demonstrate the most structured PRO integration, requiring validated instruments, ≥15% responder thresholds, pre-specified analyses, and high completion rates. PROs can meaningfully influence added benefit ratings only when these criteria are met. NICE incorporates PROs through EQ-5D in QALY calculations, non-utility PROs have limited standalone impact. HAS inconsistently accepts PROs, while AIFA and AEMPS lack standardised methodological pathways. Across diseases, patients frequently prioritise independence, treatment burden, ability to work, and mental well-being. In conditions like MS and RA where fatigue, depression, and cognitive impairment shape daily life, alignment between HTA and patient value remains especially poor. The EU HTA Regulation mandates patient and clinician input in JCA, while the EHDS establishes infrastructure for cross-border real-world data access. The EU-funded EUPATI HTA4Patients initiative trains patients and caregivers to participate effectively in JCA/JSC processes. The 2025 methodological updates from NICE and IQWiG reflect greater emphasis on PROs and stakeholder engagement.
CONCLUSIONS: EU5 HTA frameworks have historically not fully captured patient value. While PROs offer a tool for bridging this gap, their acceptance and impact remain inconsistent. Recent policies and methodological reforms, however, create an opportunity to realign assessment processes with patient needs, broadening HTA definitions of value and moving towards access to health technologies that reflect patient-prioritised outcomes.