OBJECTIVES: Overactive bladder (OAB) significantly affects health-related quality of life (HRQoL). We compared the effects of OAB treatments on PRO measures, in particular HRQoL, between randomized controlled trials (RCTs) and observational studies.

METHODS: We conducted systematic literature reviews on RCTs and observational studies in OAB. Searches were carried out in MEDLINE, EMBASE, clinical trial registries and conference websites from 2000 to 2017 to identify studies reporting PROs in adult OAB patients without restrictions on intervention/comparator.

RESULTS: In 376 identified articles, 66 PRO instruments were reported in 211 RCTs, and 86 in 165 observational studies. In RCTs, the 5 most frequently used instruments were King’s Health Questionnaire (KHQ, 55 studies), Patient’s Perception of Bladder Condition (PPBC, 52), OAB-q quality of life questionnaire (49), Overactive Bladder Symptom Score (OABSS, 21) and Urgency Perception Scale (UPS, 18). In observational studies, the most commonly used were Urogenital Distress Inventory (UDI-6, 22), Incontinence Impact Questionnaire (IIQ, 20), International Consultation on Incontinence Questionnaire - Short Form (ICIQ-SF, 14), KHQ (12) and OAB-q quality of life questionnaire (10). Tolterodine (32), solifenacin (26) and fesoterodine (20) were the most commonly investigated treatments in RCTs, while observational studies focused on Botox (14), solifenacin (6) and electrical stimulation (6). When a treatment was assessed with the same questionnaire both in RCTs and observational studies, the direction the treatment effect was in general consistent; KHQ scales and OAB-q symptom bother sub-scale showed on average 25% to 35% higher decrease from baseline in observational studies compared to RCT.

CONCLUSIONS: We observed a greater variety of PRO instruments used in observational studies compared to RCT. Observational studies suggest that improvements in HRQOL may be be higher than the changes estimated in RCTS. It remains to be determined whether this difference is due to selection bias or other factors.