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Original Research

Orphan drugs approved in Canada: health technology assessment, price negotiation, and government formulary listing

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Pages 1-11 | Received 01 Nov 2023, Accepted 30 Jan 2024, Published online: 13 Feb 2024

Figures & data

Table 1. Processes and responsibilities for the listing of medicines in government drug plans.

Table 2. 41 oncology orphan drugs by Health Canada approval date.

Table 3. 41 non-oncology orphan drugs by Health Canada approval date.

Table 4. Health Canada, FDA and EMA regulatory review times.

Table 5. Flow of the 82 orphan drugs through CADTH and the pCPA.

Table 6. Time for CADTH and pCPA processes & between approval and pCPA completion.

Figure 1. Listing rates for orphan drugs with successful price negotiations in Canada, approved in Canada, and approved by the FDA and/or the EMA.

EMA: European Medicines Agency; FDA: Food and Drug Administration; BC: British Columbia; AB: Alberta; SK: Saskatchewan; MB: Manitoba; ON: Ontario; QC: Quebec; NB: New Brunswick; NS: Nova Scotia; PE: Prince Edward Island; NL: Newfoundland and Labrador.
Figure 1. Listing rates for orphan drugs with successful price negotiations in Canada, approved in Canada, and approved by the FDA and/or the EMA.

Data availability statement

The data used in this analysis are available from publicly accessible resources [Citation8–23, Citation25–31,Citation53–56].