ISCIII

Carlos III Health Institute.

IRDiRC

International Rare Diseases Research Consortium.

FDA

US Food and Drug Administration.

EC

Clinical trial.

CIBERER

Centre for Biomedical Research Network on Rare Diseases.

NICE

National Institute for Health and Care Excellence

NHS

National Health Service

MH

Orphan Drug

ERDF

Spanish Federation for Rare Diseases

ER

Rare disease