Abstract
Background: The cost-effectiveness of enzyme replacement therapy (ERT) compared to standard medical care was evaluated in the Dutch cohort of patients with Fabry disease. Methods: Cost-effectiveness analysis was performed using a life-time state-transition model. Transition probabilities, effectiveness data and costs were derived from retrospective data and prospective follow-up of the Dutch study cohort consisting of males and females aged 5–78 years. Intervention with ERT (either agalsidase alfa or agalsidase beta) was compared to the standard medical care. The main outcome measures were years without end organ damage (renal, cardiac en cerebrovascular complications), quality adjusted life years (QALYs), and costs. Results: Over a 70 year lifetime, an untreated Fabry patient will generate 55.0 years free of end-organ damage (53.5 years in males, 56.9 years in females) and 48.6 QALYs (47.8 in males, 49.7 in females). Starting ERT in a symptomatic patient increases the number of years free of end-organ damage by 1.5 year (1.6 in males, 1.3 in females), while the number of QALYs gained increases by a similar amount (1.7 in males, 1.4 in females). The costs of ERT starting in the symptomatic stage are between € 9- €10 million (£ 7.9 - £ 8.8 million, $13.0- $14.5 million) during a patient’s lifetime. Consequently, the extra costs per additional year free of end-organ damage and the extra costs per additional QALY range from €5.5 - €7.5 million (£ 4.8 – £ 6.6 million, $ 8.0 – $ 10.8 million), undiscounted. Conclusions: In symptomatic patients with Fabry disease, ERT has limited effect on quality of life and progression to end organ damage. The pharmaco-economic evaluation shows that this modest effectiveness drives the costs per QALY and the costs per year free of end-organ damage to millions of euros. Differentiation of patients who may benefit from ERT should be improved to enhance cost-effectiveness.
Highlights
The cost-effectiveness of enzyme replacement therapy (ERT) compared to standard medical care was evaluated in the Dutch cohort of patients with Fabry disease
Monetary benefits of ERT versus standard medical care were calculated for willingness-to-pay levels ranging from € 20,000 to € 10,000,000 per quality adjusted life years (QALYs) with the results summarized in a cost-effectiveness acceptability curve for ERT
Starting ERT in a symptomatic patient increases the number of years free of end-organ damage by 1.5 years (1.6 for males, 1.3 for females), while the number of QALYs gained increases by 1.6 QALY (1.7 for males, 1.4 for females)
Summary
Model structure A life-time Markov state-transition model of the course of Fabry disease was used to evaluate the costs and effects of ERT against standard medical care. – state-transition probabilities for the natural (untreated) course of Fabry disease are based on the period prior to the introduction of ERT therapy;. The costs of AMC hospital care were averaged per patient per disease state per year, and subsequently, the mean yearly AMC costs per disease state overall (Additional file 1: Table S2). All other used health care costs and production loss were derived from the patient by quarterly disseminated questionnaires and linked to the appropriate unit costs in Euros from the most recent Dutch costing manual (Additional file 1: Table S3 and S4). (6) adding indirect non-medical costs of production loss to the direct and indirect medical costs, assuming that these costs originate not before the 18th year of a patient’s lifetime
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