Ethical and psychosocial aspects

This project focuses on two main objectives: first, the integration of age- and disease-specific Patient-Reported Outcome Measures (PROMs) into clinical trials on rare diseases, ensuring that patient-centered and relevant outcomes are captured. Second, the exploration of the psychosocial impact of childhood rare diseases on families. The study employs a longitudinal design incorporating a phase of frequent momentary assessments, enabling a nuanced understanding of how rare diseases impact parents and affected children. This design captures both retrospective reports and real-time experiences. By combining standardized assessments, clinical interviews, and advanced analysis methods, the project aims to inform family-centered care approaches and contribute to evidence-based strategies for improving support systems in the field of rare diseases.