Presentation (CTI)

OBJECTIVES: Patients with ulcerative colitis (UC) experience disabling symptoms and patient-reported outcomes (PRO)/health-related quality-of-life (HRQoL) measures aim to capture patients’ experiences. To understand Canadian HTA bodies’ critiques of HRQoL/PRO measures, we reviewed appraisals of outcome measures in HTA submissions in UC.
METHODS: HTAs for UC indications from 2004-2024 were retrieved from CDA. Corresponding INESSS reports for these products were retrieved. Information on indication, therapy, pivotal trial, HRQoL/PRO measures, and appraisal of the measures were extracted from the HTA reports.
RESULTS: Among 1241 HTAs by CDA, 15 were for UC therapies. PRO/HRQoL measures were submitted in all HTAs evaluating UC therapies. Most frequently reported generic PRO/HRQoL instruments include SF-36 (n=9) and EQ-5D (n=8). IBDQ and WPAI-UC were frequently reported disease-specific instruments, in 10 and 7 submissions, respectively. Eleven submissions presented both generic and disease-specific instruments. CDA appraised outcome measures in terms of validity, reliability, and responsiveness for 11 HTAs. CDA noted in their appraisal the SF-36, EQ-5D, IBDQ, and WPAI-UC instruments to be valid, reliable, and responsive in five, four, six, and one HTA, respectively. Overall, most submissions (n=11) used well-recognized instruments that have been validated for UC. However, CDA mentioned QoL data in their rationale for recommendation in only eight submissions; in five of these, CDA noted uncertainty in QoL data (e.g., missing patient data, not adjusted for type I error). Among these HTAs with insufficient QoL evidence, one received a negative recommendation. INESSS commented on QoL measures in 7/15 reports, focusing on disease-specific measures and their validity.
CONCLUSIONS: Impact on QoL is a significant aspect of patient experience in chronic disease. Findings show that CDA and INESSS routinely consider QoL data in assessments, with data from clinically-validated instruments and methodologically sound approaches included in appraisals. However, impact of QoL data on decisionmaking is sometimes unclear due to variability in reporting.