ANALYSIS OF GENE THERAPIES AUTHORIZED BY HEALTH CANADA, AND COMPARISON WITH THE U.S. FOOD AND DRUG ADMINISTRATION, AND THE EUROPEAN MEDICINES AGENCY
Author(s)
Anna Pelc, MS, MBA, MPH1, Mana Rahimian, PharmD, MS1, Lawrence M. Brown, PharmD, PhD, FAPhA1, Marc L. Fleming, BSPharm, MPH, MS, PhD1, Vaishali Shukla, MSc, MSRA, MS, PhD1, Rosa Rodriguez-monguio, PhD, MS2, Enrique Seoane-Vazquez, PhD1;
1Chapman University School of Pharmacy, Department of Pharmaceutical Economic and Policy, Irvine, CA, USA, 2University of California San Francisco, Department of Clinical Pharmacy, School of Pharmacy, San Francisco, CA, USA
1Chapman University School of Pharmacy, Department of Pharmaceutical Economic and Policy, Irvine, CA, USA, 2University of California San Francisco, Department of Clinical Pharmacy, School of Pharmacy, San Francisco, CA, USA
OBJECTIVES: Gene therapies represent a shift in disease treatment and prevention. We compared the characteristics of gene therapies authorized by Health Canada (HC), U.S. Food and Drug Administration (FDA), and European Medicines Agency (EMA).
METHODS: Data from HC, FDA, and EMA websites as of December 31, 2025 were analyzed descriptively.
RESULTS: Of the 30 gene therapies authorized by at least one agency, FDA authorized 27 (90.0%), EMA 22 (73.3%), and HC 11 (36.7%). The 11 therapies authorized by HC were also authorized by the other two agencies, with the FDA authorizing 10 (90.9%) of those therapies first, and HC 1 (9.1%). The median approval gap was 346.0 days (interquartile range (IQR): 164.5 days) between FDA and HC, and 176.0 days (IQR: 213.5 days) between EMA and HC. The therapeutic classes with the largest number of HC authorizations were antineoplastic and immunomodulating (n=6, 54.5%) and blood and blood forming organs (3, 27.3%). All 11 products received orphan designation by FDA and EMA. Two therapies had an HC notice of compliance with conditions, seven conditional marketing authorizations by EMA, and one accelerated approval by FDA.
All therapies had at least one difference in the indications authorized by the three agencies. The most frequent differences in indication between FDA and HC were related to line of therapy (5, 45.5%) and disease characteristics (3, 27.3%). Between EMA and HC, the most frequent differences were related to line of therapy (4, 36.4%).
CONCLUSIONS: One third of the gene therapies authorized by the FDA and/or EMA have also been authorized by Health Canada. Most therapies were authorized last by HC with a gap exceeding eleven months for FDA and five months for EMA. There were notable differences in the indications of gene therapies authorized by HC, FDA, and EMA.
METHODS: Data from HC, FDA, and EMA websites as of December 31, 2025 were analyzed descriptively.
RESULTS: Of the 30 gene therapies authorized by at least one agency, FDA authorized 27 (90.0%), EMA 22 (73.3%), and HC 11 (36.7%). The 11 therapies authorized by HC were also authorized by the other two agencies, with the FDA authorizing 10 (90.9%) of those therapies first, and HC 1 (9.1%). The median approval gap was 346.0 days (interquartile range (IQR): 164.5 days) between FDA and HC, and 176.0 days (IQR: 213.5 days) between EMA and HC. The therapeutic classes with the largest number of HC authorizations were antineoplastic and immunomodulating (n=6, 54.5%) and blood and blood forming organs (3, 27.3%). All 11 products received orphan designation by FDA and EMA. Two therapies had an HC notice of compliance with conditions, seven conditional marketing authorizations by EMA, and one accelerated approval by FDA.
All therapies had at least one difference in the indications authorized by the three agencies. The most frequent differences in indication between FDA and HC were related to line of therapy (5, 45.5%) and disease characteristics (3, 27.3%). Between EMA and HC, the most frequent differences were related to line of therapy (4, 36.4%).
CONCLUSIONS: One third of the gene therapies authorized by the FDA and/or EMA have also been authorized by Health Canada. Most therapies were authorized last by HC with a gap exceeding eleven months for FDA and five months for EMA. There were notable differences in the indications of gene therapies authorized by HC, FDA, and EMA.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR84
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Rare & Orphan Diseases