Review
Open access
Aug 2026
Should rare and ultrarare diseases be treated differently in health coverage decisions?
Health technology assessment (HTA) is widely used to inform coverage and reimbursement decisions in health systems by integrating evidence on clinical effectiveness, safety, cost-effectiveness, and broader social considerations. However, the application of conventional HTA frameworks to rare diseases remains chal...
L. Probst, Renata Cristina Ferreira Rola, Viviane Karoline da Silva Carvalho et al.
· Orphanet Journal of Rare Dis... · 0 citations