Presentation (CTI)

OBJECTIVES: As patient-centered care gains focus, health systems increasingly include patient-reported outcomes (PROs) in health technology assessments (HTAs) to reflect patient perspectives. This review analyzed how PROs influenced value dossiers submitted to HTA agencies across the EU4 + UK (2020-2025), with emphasis on pricing and reimbursement decisions.
METHODS: A targeted literature review identified peer-reviewed studies and HTA reports with PROs in HTA submissions to EU4 + UK . Eligible studies/submissions explicitly cited PROs in the assessments. Results were synthesized by country and therapeutic area.
RESULTS: Among 45 HTA submissions reviewed, 58% incorporated PROs, particularly in oncology, respiratory, and rheumatology therapeutic areas where quality of life is central. However, their impact on reimbursement decisions varied based on therapeutic context, instrument validity, and data quality. Some cases are highlighted below. In Germany, crizotinib’s positive benefit rating was supported by robust PRO data (EORTC QLQ-C30, EQ-5D), while regorafenib’s absence of robust PRO evidence downgraded its rating from “minor added benefit” to “no proven added benefit”. UK’s NICE cited PROs as pivotal in positive appraisals of pemetrexed and roflumilast, referencing significant improvements in symptom scales and quality-of-life. Conversely, NICE issued restricted recommendations when PROs were missing or poorly designed, increasing uncertainty in cost-effectiveness. France’s HAS rejected olaratumab and avelumab due to lack of robust PROs, but supported dupilumab and onasemnogene abeparvovec, where validated PROs and caregiver-reported outcomes strengthened value claims. Limited or inconclusive endpoints, missing data, unvalidated tools, and unclear responder thresholds hinder PRO reliability, creating uncertainty in clinical and economic models. France and Germany have launched pilot programs to enhance PRO submission quality and address these persistent methodological issues.
CONCLUSIONS: PROs are increasingly integrated into HTA submissions across EU4 + UK, yet their impact on reimbursement varies. Methodological rigor, validated instruments, and disease context remain critical for influencing positive decisions.