Cost-Effectiveness and Budget Impact of Gene Therapy for Eligible Patients With Sickle Cell Disease in the Netherlands
Author(s)
Melanie Bruinooge, MD1, Anita Rijnveld, MD, PhD2, Charlotte Van Tuijn, MD, PhD3, E. Nur, MD, PhD3, Ward Van Beers, MD, PhD4, A. Mohseny, MD, PhD5, Marjon Cnossen, MD, PhD2, Carin A. Uyl-De Groot, Sr., PhD6.
1Erasmus University Medical Center/Erasmus University Rotterdam, Rotterdam, Netherlands, 2Erasmus Medical Center, Rotterdam, Netherlands, 3Amsterdam University Medical Center, Amsterdam, Netherlands, 4Utrecht University Medical Center, Utrecht, Netherlands, 5Leiden University Medical Center, Leiden, Netherlands, 6Professor, ESHPM/iMTA Erasmus University Rotterdam, Rotterdam, Netherlands.
1Erasmus University Medical Center/Erasmus University Rotterdam, Rotterdam, Netherlands, 2Erasmus Medical Center, Rotterdam, Netherlands, 3Amsterdam University Medical Center, Amsterdam, Netherlands, 4Utrecht University Medical Center, Utrecht, Netherlands, 5Leiden University Medical Center, Leiden, Netherlands, 6Professor, ESHPM/iMTA Erasmus University Rotterdam, Rotterdam, Netherlands.
OBJECTIVES: Gene therapy offers promising benefits for patients with sickle cell disease (SCD) but also raises questions regarding affordability. This study aimed to evaluate the cost-effectiveness and budget impact of gene therapy compared to comprehensive care in patients with severe SCD genotypes (HbSS or HbSβ⁰/β⁺) aged ≥12 years with severe disease-related symptoms.
METHODS: We conducted a cost-effectiveness analysis (CEA) using a Markov state-transition model from a Dutch societal perspective over a lifetime horizon. Efficacy and safety data for gene therapy were sourced from the HGB-206 (NCT02140554) and HGB-210 (NCT03499818) clinical trials. Additional data on clinical outcomes, quality of life (QoL), and costs were obtained from the HGB-206 and HGB-210 trial, and scientific literature. Budget impact analysis (BIA) was performed from a healthcare perspective, covering a five-year time horizon. Uncertainty in the CEA and BIA were assessed through probabilistic sensitivity and scenario analysis.
RESULTS: After applying discounting and half-cycle correction, gene therapy yielded 11.70 additional life years, and 13.29 additional quality-adjusted life years (QALYs) compared to comprehensive care. The deterministic incremental cost-effectiveness ratio (ICER) was €123,384 per QALY gained; the probabilistic ICER was €124,972 per QALY gained. Results were sensitive to drug acquisition costs, survival rates, and utility of patients with no or mild vaso-occlusive events in the gene therapy group, and the method used to calculate productivity losses (human capital versus friction period). The budget impact of treating eligible patients with gene therapy over 5 years was projected to be approximately €71 million.
CONCLUSIONS: Gene therapy substantially improves survival and QoL for patients with severe SCD compared to comprehensive care but at a high cost. At the current cost-effectiveness threshold of €80,000 per QALY in the Netherlands, it is not considered cost-effective. Reducing the market price of gene therapy is essential to make it more economically viable and accessible.
METHODS: We conducted a cost-effectiveness analysis (CEA) using a Markov state-transition model from a Dutch societal perspective over a lifetime horizon. Efficacy and safety data for gene therapy were sourced from the HGB-206 (NCT02140554) and HGB-210 (NCT03499818) clinical trials. Additional data on clinical outcomes, quality of life (QoL), and costs were obtained from the HGB-206 and HGB-210 trial, and scientific literature. Budget impact analysis (BIA) was performed from a healthcare perspective, covering a five-year time horizon. Uncertainty in the CEA and BIA were assessed through probabilistic sensitivity and scenario analysis.
RESULTS: After applying discounting and half-cycle correction, gene therapy yielded 11.70 additional life years, and 13.29 additional quality-adjusted life years (QALYs) compared to comprehensive care. The deterministic incremental cost-effectiveness ratio (ICER) was €123,384 per QALY gained; the probabilistic ICER was €124,972 per QALY gained. Results were sensitive to drug acquisition costs, survival rates, and utility of patients with no or mild vaso-occlusive events in the gene therapy group, and the method used to calculate productivity losses (human capital versus friction period). The budget impact of treating eligible patients with gene therapy over 5 years was projected to be approximately €71 million.
CONCLUSIONS: Gene therapy substantially improves survival and QoL for patients with severe SCD compared to comprehensive care but at a high cost. At the current cost-effectiveness threshold of €80,000 per QALY in the Netherlands, it is not considered cost-effective. Reducing the market price of gene therapy is essential to make it more economically viable and accessible.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
P43
Topic
Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Budget Impact Analysis
Disease
Genetic, Regenerative & Curative Therapies, No Additional Disease & Conditions/Specialized Treatment Areas, Pediatrics, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)