Diffusion and Launch Sequencing of Cell and Gene Therapies across the USA, Europe, Canada and Australia
Author(s)
Matthew Bergsteedt, BSc, PGDip, MSc Candidate1, Fiona Tolkmitt, BSc1, Mackenzie Mills, PhD1, Panos Kanavos, BSc, MSc, PhD2.
1HTA-Hive, London, United Kingdom, 2Department of Health Policy, Medical Technology Research Group (MTRG), London School of Economics and Political Science, London, United Kingdom.
1HTA-Hive, London, United Kingdom, 2Department of Health Policy, Medical Technology Research Group (MTRG), London School of Economics and Political Science, London, United Kingdom.
OBJECTIVES: Cell and gene therapies (CGTs) represent a significant advancement in healthcare, providing potential cures for previously untreatable conditions. However, they often carry high prices and their availability across settings remains mixed. This study examines the diffusion of FDA approved CGTs in Canada, Australia, France, Germany, England and Scotland.
METHODS: The FDA website was screened to identify all approved CGTs. Marketing authorisation agency websites in Australia (TGA), Canada (Health Canada), the United Kingdom (MHRA), and Europe (EMA) and HTA agencies in Canada (CDA), Australia (PBAC and MSAC), France (HAS), Germany (GBA), England (NICE) and Scotland (SMC), to identify matching regulatory approvals and HTA reports. For each product, data was extracted on disease area, firm characteristics, and clinical trial evidence to assess whether these factors influence likelihood of uptake across markets. Total market penetration, approval timelines, and launch sequence was assessed across products and settings.
RESULTS: Out of 30 FDA approved CGTs, 16 treatments have authorisation in at least one other setting. Marketing authorisation of FDA approved cell and gene therapies was highest in Europe (53%, n=16) followed by Canada (33%, n=10), the UK (23%, n=7) and TGA (23%, n=7). Of those assessed, HTA approval was highest for HAS (100%, n=12) followed by CADTH (91%, n=10), GBA (69%, n=9) and NICE (63%, n=7). Only four products had positive HTA recommendations in all screened markets: Zolgensma, Luxturna, and Yescarta and Tecartus. Large companies (>$10 billion annual revenue) were more likely to launch products in more settings, with 89% of products (n=8) launching in 3 or more markets.
CONCLUSIONS: Low rates of marketing authorisation and HTA approval have contributed to a fragmented access landscape for CGTs. While large firms may be better equipped to launch these technologies globally, high prices, uncertainties in clinical evidence, and technical complexities in administering therapies present continued barriers to uptake.
METHODS: The FDA website was screened to identify all approved CGTs. Marketing authorisation agency websites in Australia (TGA), Canada (Health Canada), the United Kingdom (MHRA), and Europe (EMA) and HTA agencies in Canada (CDA), Australia (PBAC and MSAC), France (HAS), Germany (GBA), England (NICE) and Scotland (SMC), to identify matching regulatory approvals and HTA reports. For each product, data was extracted on disease area, firm characteristics, and clinical trial evidence to assess whether these factors influence likelihood of uptake across markets. Total market penetration, approval timelines, and launch sequence was assessed across products and settings.
RESULTS: Out of 30 FDA approved CGTs, 16 treatments have authorisation in at least one other setting. Marketing authorisation of FDA approved cell and gene therapies was highest in Europe (53%, n=16) followed by Canada (33%, n=10), the UK (23%, n=7) and TGA (23%, n=7). Of those assessed, HTA approval was highest for HAS (100%, n=12) followed by CADTH (91%, n=10), GBA (69%, n=9) and NICE (63%, n=7). Only four products had positive HTA recommendations in all screened markets: Zolgensma, Luxturna, and Yescarta and Tecartus. Large companies (>$10 billion annual revenue) were more likely to launch products in more settings, with 89% of products (n=8) launching in 3 or more markets.
CONCLUSIONS: Low rates of marketing authorisation and HTA approval have contributed to a fragmented access landscape for CGTs. While large firms may be better equipped to launch these technologies globally, high prices, uncertainties in clinical evidence, and technical complexities in administering therapies present continued barriers to uptake.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HTA92
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
SDC: Oncology, SDC: Rare & Orphan Diseases, STA: Genetic, Regenerative & Curative Therapies