ISCIII
Carlos III Health Institute.
IRDiRC
Consorcio Internacional de Investigación en Enfermedades Raras (International Rare Diseases Research Consortium).
FDA
US Food and Drug Administration.
EC
Clinical trial.
CIBERER
Centro de Investigación Biomédica en Red de Enfermedades Raras.
NICE
National Institute for Health and Care Excellence
NHS
National Health Service
MH
Orphan Drug
ERDF
Spanish Federation for Rare Diseases
ER
Rare disease