Orphan Drug Approval in Canada, 1999-2022: A Cross-sectional Study

Lexchin, Joel

doi:10.34172/ijhpm.8916

Orphan Drug Approval in Canada, 1999-2022: A Cross-sectional Study

Document Type : Short Communication

Author

Joel Lexchin ¹^{, 2}

¹ School of Health Policy and Management, York University, Toronto, ON, Canada

² Department of Family and Community Medicine, University of Toronto, Toronto, ON, Canada

10.34172/ijhpm.8916

Abstract

The number of drugs for orphan indications has been increasing significantly in Canada and the federal government recently announced an investment of $1.5 billion dollars over 3 years primarily directed at helping to fund the cost of these drugs. There are claims and counterclaims about what percent of Food and Drug Administration (FDA) orphan drugs are available in Canada and how delayed these drugs are in being approved by Health Canada. This study uses FDA and Health Canada databases and data from three health technology assessment agencies and one drug bulletin to provide objective data about the percent of FDA approved drugs that were also approved by Health Canada, any delays in Canadian approval and the additional therapeutic value of new orphan drugs. Decisions about what drugs should be publicly covered and how long it took to make those decisions were not investigated. From 1999 to 2022, the FDA approved 326 new drugs for an orphan indication and Health Canada approved 231 (70.9%) for the same indication. The median time between FDA and Health Canada approval was 346 days (interquartile range [IQR] 181, 785). The percent rated as major improvements declined from 50% of the total in 2004-2008 to 13.6% in 2019-2022. These findings need to be taken into account as Canada develops an orphan drug policy and decides on criteria for funding this group of drugs. Specifically, when high quality evidence about the additional therapeutic value of orphan drugs is not available at the time of approval, risk sharing funding agreements with manufacturers should be put in place. Manufacturers should understand that if the results of post-market trials do not provide convincing evidence of value, funding will be withdrawn. Finally, the quality of any research plan should be used to prioritize candidates for federal funding.

Highlights

Joel Lexchin (Google Scholar) (PubMed)

Keywords

References

Lexchin J. Quality and quantity of data used by Health Canada in approving new drugs. Front Med (Lausanne). 2023;10:1299239. doi:3389/fmed.2023.1299239
Government of Canada. Investments to Support Access to Drugs for Rare Diseases. 2023. https://www.canada.ca/en/health-canada/news/2023/03/investments-to-support-access-to-drugs-for-rarediseases.html. Accessed November 4, 2024.
Canadian Organization for Rare Disorders. Strategy & Access. 2024. https://www.raredisorders.ca/work/strategy-access. Accessed November 4, 2024.
Rawson NS, Adams J. Orphan drugs approved in Canada: health technology assessment, price negotiation, and government formulary listing. Expert Opin Orphan Drugs. 2024;12(1):1-11. doi:1080/21678707.2024.2313766
Lexchin J. Orphan drugs approved in Canada: availability and additional therapeutic value. Future Rare Dis. 2025;5(1):2466380. doi:1080/23995270.2025.2466380
US Department of Health and Human Services. New Molecular Entity (NME) Drug and New Biologic Approvals. 2017. https://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/DrugandBiologicApprovalReports/NDAandBLAApprovalReports/ucm373420.htm. Accessed November 4, 2024.
US Food & Drug Administration. New Molecular Entity (NME) Drug and New Biologic Approvals. 2024. https://www.fda.gov/drugs/nda-and-bla-approvals/new-molecular-entity-nme-drug-and-new-biologic-approvals. Accessed November 4, 2024.
Government of Canada. Drug and Health Product Portal: Review Decisions. 2024. https://dhpp.hpfb-dgpsa.ca/review-documents?search=&f%5B0%5D=content_type%3Asbd. Accessed November 4, 2024.
Government of Canada. Drug Product Database Online Query. 2024. https://health-products.canada.ca/dpd-bdpp/. Accessed November 4, 2024.
Government of Canada. Notice of Compliance Search. 2024. https://health-products.canada.ca/dpd-bdpp/. Accessed November 4, 2024.
Government of Canada. Drug and Health Product Submissions Under Review (SUR). 2024. https://www.canada.ca/en/health-canada/services/drug-health-product-review-approval/submissions-under-review.html?_ga=2.233746589.2014837886.1506726303-1326787507.1398655537#_Submissions_currently_under. Accessed November 4, 2024.
Norwegian Institute of Public Health, WHO Collaborating Centre for Drug Statistics Methodology. ATC/DDD Index 2024. 2024. https://atcddd.fhi.no/atc_ddd_index/. Accessed November 4, 2024.
Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen. 2024. https://www.iqwig.de/en/. Accessed August 29, 2024.
Haute Autorité de Santé. 2024. https://www.has-sante.fr. Accessed August 29, 2024.
Prescrire International. 2024. https://english.prescrire.org/en/Summary.aspx. Accessed August 29, 2024.
Patented Medicine Prices Review Board. Annual Reports. 2024. https://www.canada.ca/en/patented-medicine-prices-review/services/annual-reports.html. Accessed August 29, 2024.
Hwang TJ, Ross JS, Vokinger KN, Kesselheim AS. Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study. BMJ. 2020;371:m3434. doi:1136/bmj.m3434
Kesselheim AS, Wang B, Franklin JM, Darrow JJ. Trends in utilization of FDA expedited drug development and approval programs, 1987-2014: cohort study. BMJ. 2015;351:h4633. doi:1136/bmj.h4633
Lexchin J, Sirrs S. Federal funding for expensive drugs for rare diseases: how do we pick and choose? Healthc Policy. 2024;19(4):19-26. doi:12927/hcpol.2024.27334
Patented Medicine Prices Review Board. Annual Report 2021. Ottawa; 2022.