Presentation (CTI)

OBJECTIVES: Patient-centered outcomes play a central role in the implementation of value-based healthcare (VBHC) framework. Within this framework, patient-centered outcomes serve multiple functions, including acting as quality indicators for comparing hospital performance. While these outcomes are commonly used to evaluate treatment effectiveness in hemophilia clinical trials, their application in comparing hospital performance is more challenging due to several factors, including the risk of confounding arising from the lack of randomization. Therefore, we evaluated the suitability of outcomes from two core sets developed using a VBHC approach, as indicators for hospital comparisons.
METHODS: We analyzed data from 807 adults with hemophilia enrolled in the HiN-6 cross-sectional study. Twelve outcomes were selected: five clinical (zero-treated bleeds, zero-treated joint bleeds, annual bleeding rate, annual joint bleeding rate, joint score) and seven patient-reported (physical functioning, hemophilia activity list, social functioning, pain, anxiety, depression, treatment satisfaction). Hospital scores were calculated as proportions for binary outcomes, and as medians for count and continuous outcomes. The suitability of these outcomes for hospital comparisons was evaluated using two criteria: (1) case-mix effects, evaluated by Spearman’s rank correlation between case-mix adjusted and unadjusted hospital-level scores; and (2) reliability of performance differences, determined by rankability defined as the proportion of between-hospital variation not due to chance.
RESULTS: Among the clinical outcomes, four demonstrated strong correlations with Spearman’s coefficients exceeding 0.80. For the patient-reported outcomes the correlations varied, with coefficients ranging from 0.03 to 1.00. In terms of rankability, three clinical outcomes demonstrated rankability percentages above 50%, whereas patient-reported outcomes ranged from 0% to 29%.
CONCLUSIONS: Clinical outcomes appeared to perform better than patient-reported outcomes as quality indicators for comparing hospital performance. Among the clinical outcomes, bleeding rate and joint score showed the most promise, with the annual (joint) bleeding rate demonstrating greater suitability than the zero-treated (joint) bleed rate.