An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries
Abstract Background The Canadian government has committed to developing a national strategy for drugs for rare diseases starting in 2022. Considering this announcement, we conducted a comparative analysis to examine patient access to therapies for rare disease in Canada relative to Europe and the U....
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ftdatacite:10.6084/m9.figshare.c.5877425 2023-05-15T17:23:01+02:00 An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries Ward, Leanne Marie Chambers, Alexandra Mechichi, Emine Wong-Rieger, Durhane Campbell, Craig 2022 https://dx.doi.org/10.6084/m9.figshare.c.5877425 https://springernature.figshare.com/collections/An_international_comparative_analysis_of_public_reimbursement_of_orphan_drugs_in_Canadian_provinces_compared_to_European_countries/5877425 unknown figshare https://dx.doi.org/10.1186/s13023-022-02260-6 Creative Commons Attribution 4.0 International https://creativecommons.org/licenses/by/4.0/legalcode cc-by-4.0 CC-BY Medicine Genetics FOS Biological sciences Pharmacology Biotechnology Ecology Sociology FOS Sociology Marine Biology 111714 Mental Health FOS Health sciences Computational Biology article Collection 2022 ftdatacite https://doi.org/10.6084/m9.figshare.c.5877425 https://doi.org/10.1186/s13023-022-02260-6 2022-04-01T10:56:53Z Abstract Background The Canadian government has committed to developing a national strategy for drugs for rare diseases starting in 2022. Considering this announcement, we conducted a comparative analysis to examine patient access to therapies for rare disease in Canada relative to Europe and the U.S. Methods Given its similarity to the Canadian health care system, we used Europe as the reference point to analyze all of the therapies with an orphan drug designation approved by the European Medicine Agency (EMA) from 1 January 2015 to 31 March 2020. We then contrasted access to these drugs in Canada (Health Canada) and the U.S. (Food and Drug Administration, FDA). We focused on: (1) the number of therapies for rare diseases entering the Canadian market; (2) the percentage of these therapies that are publicly available to Canadians; and (3) the timelines for patients to access these therapies in Canada. Results Sixty-three approved therapies with an orphan drug designation from the EMA were identified. Fifty-three (84%) of these drugs had also been submitted to the FDA for approval, and 41 (65%) were submitted to Health Canada for approval. In Europe, Germany, Denmark, and the U.K. had the highest percentage of publicly reimbursed orphan drugs (84%, 70%, 68%, respectively). In comparison, Ontario (32%), Quebec (25%), and Alberta (25%) had the highest percentage of drugs reimbursed among the Canadian provinces. The shortest median duration (in months) from EMA approval to jurisdictional decision on reimbursement was in Austria (3.2), followed by Germany (4.1), and Finland (6.0). In Canada, the shortest median duration (in months) from regulatory approval to reimbursement was in British Columbia (17.3), Quebec (19.6) and Manitoba (19.6), while the longest duration was in P.E.I (38.5), followed by Nova Scotia (25.9), and Newfoundland (25.1). Conclusions Our comparative analysis found that relative to the EU Canadians had less frequent and timely access to therapies for rare diseases. This highlights the need for a rare disease strategy in Canada that allows for clear identification and transparent tracking of the pathway for rare disease drugs, and ultimately optimizes the number of patients with access to these therapies. Article in Journal/Newspaper Newfoundland DataCite Metadata Store (German National Library of Science and Technology) British Columbia ENVELOPE(-125.003,-125.003,54.000,54.000) Canada |
institution |
Open Polar |
collection |
DataCite Metadata Store (German National Library of Science and Technology) |
op_collection_id |
ftdatacite |
language |
unknown |
topic |
Medicine Genetics FOS Biological sciences Pharmacology Biotechnology Ecology Sociology FOS Sociology Marine Biology 111714 Mental Health FOS Health sciences Computational Biology |
spellingShingle |
Medicine Genetics FOS Biological sciences Pharmacology Biotechnology Ecology Sociology FOS Sociology Marine Biology 111714 Mental Health FOS Health sciences Computational Biology Ward, Leanne Marie Chambers, Alexandra Mechichi, Emine Wong-Rieger, Durhane Campbell, Craig An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries |
topic_facet |
Medicine Genetics FOS Biological sciences Pharmacology Biotechnology Ecology Sociology FOS Sociology Marine Biology 111714 Mental Health FOS Health sciences Computational Biology |
description |
Abstract Background The Canadian government has committed to developing a national strategy for drugs for rare diseases starting in 2022. Considering this announcement, we conducted a comparative analysis to examine patient access to therapies for rare disease in Canada relative to Europe and the U.S. Methods Given its similarity to the Canadian health care system, we used Europe as the reference point to analyze all of the therapies with an orphan drug designation approved by the European Medicine Agency (EMA) from 1 January 2015 to 31 March 2020. We then contrasted access to these drugs in Canada (Health Canada) and the U.S. (Food and Drug Administration, FDA). We focused on: (1) the number of therapies for rare diseases entering the Canadian market; (2) the percentage of these therapies that are publicly available to Canadians; and (3) the timelines for patients to access these therapies in Canada. Results Sixty-three approved therapies with an orphan drug designation from the EMA were identified. Fifty-three (84%) of these drugs had also been submitted to the FDA for approval, and 41 (65%) were submitted to Health Canada for approval. In Europe, Germany, Denmark, and the U.K. had the highest percentage of publicly reimbursed orphan drugs (84%, 70%, 68%, respectively). In comparison, Ontario (32%), Quebec (25%), and Alberta (25%) had the highest percentage of drugs reimbursed among the Canadian provinces. The shortest median duration (in months) from EMA approval to jurisdictional decision on reimbursement was in Austria (3.2), followed by Germany (4.1), and Finland (6.0). In Canada, the shortest median duration (in months) from regulatory approval to reimbursement was in British Columbia (17.3), Quebec (19.6) and Manitoba (19.6), while the longest duration was in P.E.I (38.5), followed by Nova Scotia (25.9), and Newfoundland (25.1). Conclusions Our comparative analysis found that relative to the EU Canadians had less frequent and timely access to therapies for rare diseases. This highlights the need for a rare disease strategy in Canada that allows for clear identification and transparent tracking of the pathway for rare disease drugs, and ultimately optimizes the number of patients with access to these therapies. |
format |
Article in Journal/Newspaper |
author |
Ward, Leanne Marie Chambers, Alexandra Mechichi, Emine Wong-Rieger, Durhane Campbell, Craig |
author_facet |
Ward, Leanne Marie Chambers, Alexandra Mechichi, Emine Wong-Rieger, Durhane Campbell, Craig |
author_sort |
Ward, Leanne Marie |
title |
An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries |
title_short |
An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries |
title_full |
An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries |
title_fullStr |
An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries |
title_full_unstemmed |
An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries |
title_sort |
international comparative analysis of public reimbursement of orphan drugs in canadian provinces compared to european countries |
publisher |
figshare |
publishDate |
2022 |
url |
https://dx.doi.org/10.6084/m9.figshare.c.5877425 https://springernature.figshare.com/collections/An_international_comparative_analysis_of_public_reimbursement_of_orphan_drugs_in_Canadian_provinces_compared_to_European_countries/5877425 |
long_lat |
ENVELOPE(-125.003,-125.003,54.000,54.000) |
geographic |
British Columbia Canada |
geographic_facet |
British Columbia Canada |
genre |
Newfoundland |
genre_facet |
Newfoundland |
op_relation |
https://dx.doi.org/10.1186/s13023-022-02260-6 |
op_rights |
Creative Commons Attribution 4.0 International https://creativecommons.org/licenses/by/4.0/legalcode cc-by-4.0 |
op_rightsnorm |
CC-BY |
op_doi |
https://doi.org/10.6084/m9.figshare.c.5877425 https://doi.org/10.1186/s13023-022-02260-6 |
_version_ |
1766110024200880128 |