Assessing the Real-World Value of Hemophilia Care: How Suitable Are Patient-Centered Outcomes for Hospital-Level Comparisons?
Diaz M Prameyllawati, Samantha C Gouw, Hester F Lingsma, Marjon H Cnossen, Floor C J I Heubel-Moenen, Frank W G Leebeek, Nick van Es, Johanna G van der Bom, Renske M T ten Ham, Martijn A H Oude VoshaarAbstract
Background
Hospital-level comparisons of patient-centered outcomes play a central role in value-based healthcare. In hemophilia care, several standardized sets of patient-centered outcomes have been developed to foster the implementation of value-based healthcare; however, their suitability for hospital-level comparisons has not yet been assessed. Therefore, we evaluated the suitability of patient-centered outcomes for hospital-level comparisons in hemophilia care.
Methods
We analyzed data from 807 adults with hemophilia enrolled in the 2019 HiN-6 study in the Netherlands. Twelve patient-centered outcomes were assessed. The suitability of these outcomes for hospital comparisons was evaluated using two criteria: (1) sensitivity to case mix, evaluated by Spearman’s rank correlation between case-mix adjusted and unadjusted hospital-level scores; and (2) reliability of between-hospital differences, determined by rankability defined as the proportion of between-hospital variation not attributable to chance. High correlations indicate a minimal case-mix influence, while a high rankability reflects a high degree of reliability.
Results
Most patient-centered outcomes were relatively insensitive to case mix (ρ > 0.70). Reliability of hospital differences was generally low (rankability <0.50). Only the annual bleeding rate and annual joint bleeding rate demonstrated moderate reliability with a rankability of 0.55 and 0.58, respectively.
Conclusion
Most patient-centered outcomes assessed were not suitable for comparing hemophilia care at the hospital level because of low reliability. Among the outcomes evaluated, the annual bleeding rate and annual joint bleeding rate appeared relatively more suitable for such comparisons.