Review
Open access
Jul 2026
Evidence mapping of concepts, methods, and outcomes in the evaluation of diagnostic and therapeutic technologies for ultra-rare diseases: a systematic scoping review.
Despite heterogeneity, there is growing convergence on prevalence-based criteria and adaptive strategies for URDs, highlighting the need for robust patient registries, international data sharing, and HTA frameworks that incorporate severity, equity, and social value.
Raul Edison Luna Lazo, D. Gorski, F. Vilela et al.
· Value in Health · 0 citations