Fig. 1

Flowchart identifying historical and pipeline drugs for rare diseases in Canada. As Canada does not have an official framework for drugs for rare diseases (DRDs), candidate DRDs were identified using the European Medicines Agency (EMA) Community Register of Orphan Medicinal Products, the US Food and Drug Administration (FDA) Orphan Drug Product designation database, and the Health Canada (HC) Notice of Compliance database. Various inclusion and exclusion criteria were applied to determine whether a DRD would reach the Canadian market by 2025. Additional assumptions used to project whether a candidate DRD would be marketed in Canada by 2025 are described in the “Budget forecasting” section. DRDs drugs for rare diseases, EMA European Medicines Agency, FDA US Food and Drug Administration