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Table 1 Definitions of terminology used in the current study

From: Characteristics of drugs for ultra-rare diseases versus drugs for other rare diseases in HTA submissions made to the CADTH CDR

Term

Definition

Drugs for rare diseases (DRDs)

Drugs used to treat diseases that affect ≤ 50 per 100,000 people.

Drugs for ultra-rare diseases (DURDs)

Drugs used to treat diseases that affect ≤ 1 per 100,000 people. -

Drugs for other rare diseases (DORDs)

Drugs used to treat rare diseases that affect > 1 to 50 per 100,000 people.

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172