Abstract
Introduction. Hemophilia is a rare X-linked hereditary bleeding disorder requiring lifelong, resource-intensive, and multidisciplinary management. Despite substantial therapeutic advances, clinical outcomes are largely determined by the effectiveness of the organizational model of healthcare delivery. Standardized and psychometrically validated instruments for the quantitative assessment of organizational mechanisms in hemophilia care remain limited. Objective. To develop and psychometrically validate a structured questionnaire for assessing organizational mechanisms of medical care delivery for patients with hemophilia and to identify systemic, economic, and interlevel barriers affecting the functioning of the care model. Methods. A methodological study was conducted following established instrument development procedures, including conceptual framework development, item generation, expert assessment of content validity, pilot testing, and statistical evaluation of psychometric properties. The questionnaire was structured into four conceptual domains: clinical competence of healthcare professionals; referral pathways and interlevel coordination; organizational governance model; and accessibility of pharmaceutical provision and replacement therapy. The pilot study included 55 physicians involved in hemophilia care. Internal consistency was assessed using Cronbach’s alpha coefficient. Construct validity was examined through exploratory factor analysis with Varimax rotation. Sampling adequacy was evaluated using the Kaiser–Meyer–Olkin (KMO) measure and Bartlett’s test of sphericity. Results. The overall internal consistency of the questionnaire was high (Cronbach’s α = 0,870), indicating strong reliability. Domain-specific α coefficients ranged from 0,672 to 0,843. The KMO measure was 0,761, and Bartlett’s test was statistically significant (p < 0.001), confirming the suitability of the data for factor analysis. Exploratory factor analysis identified four factors with eigenvalues greater than 1, explaining 78,11% of the total variance. Factor loadings for the majority of items exceeded 0,70, supporting the multidimensional structure of the instrument. Conclusion. The developed questionnaire demonstrated satisfactory psychometric properties, including high overall internal consistency and an empirically supported multidimensional factor structure. The instrument may serve as a standardized tool for the quantitative evaluation of organizational mechanisms in hemophilia care and may contribute to strategic planning, resource optimization, and system-level improvement in the management of rare diseases. Keywords: hemophilia, questionnaire, organizational mechanisms, psychometric validation, healthcare organization.