TARGET POPULATION SIZE AND LAUNCH PRICE DYNAMICS OF CELL AND GENE THERAPIES (CGTS)
Author(s)
Ahmad Hecham Alani, PharmD1, Mackenzie Mills, PhD1, Panos Kanavos, BSc, MSc, PhD2.
1HTA-Hive, London, United Kingdom, 2London School of Economics and Political Science, London, United Kingdom.
1HTA-Hive, London, United Kingdom, 2London School of Economics and Political Science, London, United Kingdom.
OBJECTIVES: CGTs offer transformative benefits but present pricing challenges due to high upfront costs and long-term uncertainty. This study evaluates the relationship between CGT launch prices and target population size in HTA-driven European markets.
METHODS: A cross-sectional analysis was conducted using HTA-Hive database to identify relevant HTA reports published between 2015 and 2025. Estimated annual target populations were extracted from HTA reports issued by G-BA (Germany), HAS (France), and NICE (England). First publicly available launch prices were obtained from public sources (standardised to 2025 EUR). Prices and target populations were summarised using medians and interquartile ranges (IQR), and associations were assessed using Spearman rank correlations.
RESULTS: A total of 73 CGT entries covering 17 products were identified with both HTA-derived target population estimates and publicly available launch price data and were included in the core analysis. Median launch prices were EUR 397,680 in France (IQR: 366,450-467,334), EUR 361,860 in Germany (IQR: 340,554-437,958), and EUR 356,428 in England (IQR: 322,435-1,978,510). Median estimated annual target populations were 480 in Germany (IQR: 125-1,071), 234 in France (IQR: 103-705), and 233 in England (IQR: 90-1,193). An inverse association between price and target population size was observed across markets, strongest in England (ρ=-0.51), moderate in Germany (ρ=-0.39), and weaker in France (ρ=-0.22).
CONCLUSIONS: In HTA-driven markets, launch prices were higher for therapies targeting smaller populations, consistent with principles of rarity-weighted value-based pricing. The availability of HTA-derived population estimates enables structured assessments of affordability and value. In contrast, the lack of centralised HTA infrastructure in the US limits cross-market comparability. As MFN pricing proposals gain traction, linking US prices to HTA-based benchmarks may influence global launch strategies and pricing decisions, particularly for therapies targeting rare conditions.
METHODS: A cross-sectional analysis was conducted using HTA-Hive database to identify relevant HTA reports published between 2015 and 2025. Estimated annual target populations were extracted from HTA reports issued by G-BA (Germany), HAS (France), and NICE (England). First publicly available launch prices were obtained from public sources (standardised to 2025 EUR). Prices and target populations were summarised using medians and interquartile ranges (IQR), and associations were assessed using Spearman rank correlations.
RESULTS: A total of 73 CGT entries covering 17 products were identified with both HTA-derived target population estimates and publicly available launch price data and were included in the core analysis. Median launch prices were EUR 397,680 in France (IQR: 366,450-467,334), EUR 361,860 in Germany (IQR: 340,554-437,958), and EUR 356,428 in England (IQR: 322,435-1,978,510). Median estimated annual target populations were 480 in Germany (IQR: 125-1,071), 234 in France (IQR: 103-705), and 233 in England (IQR: 90-1,193). An inverse association between price and target population size was observed across markets, strongest in England (ρ=-0.51), moderate in Germany (ρ=-0.39), and weaker in France (ρ=-0.22).
CONCLUSIONS: In HTA-driven markets, launch prices were higher for therapies targeting smaller populations, consistent with principles of rarity-weighted value-based pricing. The availability of HTA-derived population estimates enables structured assessments of affordability and value. In contrast, the lack of centralised HTA infrastructure in the US limits cross-market comparability. As MFN pricing proposals gain traction, linking US prices to HTA-based benchmarks may influence global launch strategies and pricing decisions, particularly for therapies targeting rare conditions.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE208
Topic
Economic Evaluation
Disease
SDC: Oncology, SDC: Rare & Orphan Diseases, STA: Genetic, Regenerative & Curative Therapies, STA: Multiple/Other Specialized Treatments