Orphan drugs approved in Canada: availability and additional therapeutic value

Orphan drugs approved in Canada: availability and additional therapeutic valuein their recent article in expert opinion on rare drugs, which is no longer accepting submissions, on the accessibility of orphan drugs in Canada, rawson and Adams conclude that "Canadians with rare disorders continue to suffer from a lack of timely and equitable access to innovative treatments" [1].they based this conclusion on an analysis of the percent of orphan drugs approved by either the Food and drug Administration (FdA) or the european Medicines Agency (eMA) from 2015 to 2020 that were submitted to health Canada, the time taken for these drugs to be evaluated by both the Canadian Agency for drugs and technologies in health (CAdth) and the pan-Canadian Pharmaceutical Alliance, the recommendations from these two agencies and finally how many of the drugs were eventually listed on provincial formularies.this letter to the editor examines the biases in what data rawson and Adams left out and their interpretation of their data.

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Publication Details

Journal
Future Rare Diseases
Published
2025-02-18
DOI
https://doi.org/10.1080/23995270.2025.2466380
Primary Topic
Pharmaceutical Economics and Policy
Type
article
Field-Weighted Citation Impact
0.00
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article

Orphan drugs approved in Canada: availability and additional therapeutic value

Joel Lexchin
Future Rare Diseases
Pharmaceutical Economics and Policy
article

Orphan drugs approved in Canada: availability and additional therapeutic value

Joel Lexchin
article en

Abstract

Orphan drugs approved in Canada: availability and additional therapeutic valuein their recent article in expert opinion on rare drugs, which is no longer accepting submissions, on the accessibility of orphan drugs in Canada, rawson and Adams conclude that "Canadians with rare disorders continue to suffer from a lack of timely and equitable access to innovative treatments" [1].they based this conclusion on an analysis of the percent of orphan drugs approved by either the Food and drug Administration (FdA) or the european Medicines Agency (eMA) from 2015 to 2020 that were submitted to health Canada, the time taken for these drugs to be evaluated by both the Canadian Agency for drugs and technologies in health (CAdth) and the pan-Canadian Pharmaceutical Alliance, the recommendations from these two agencies and finally how many of the drugs were eventually listed on provincial formularies.this letter to the editor examines the biases in what data rawson and Adams left out and their interpretation of their data.

Future Rare DiseasesVol. 5(1)
University of Toronto (CA), York University (CA)
Openalex Percentile: Top 96%
Pharmaceutical Economics and Policy
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