Abstract | BACKGROUND: METHODS: We use the limited QOL data available in the Fabry- Anderson disease literature on ERT to derive standard economic metrics. These were derived by bootstrap estimates of the incremental net benefit (INB) statistics together with a cost-effectiveness acceptability curve relating the willingness to pay to the probability that the INB was >0. The estimates were further developed by adoption of a supplementary Bayesian approach utilising a sceptical and enthusiastic prior of the INB of ERT in Fabry- Anderson disease. RESULTS: ERT for Fabry- Anderson disease is not economically viable by standard health programme evaluation metrics. Based on current ERT costs (year 2005 values), derivation of the INB distribution, and a Bayesian analysis using an enthusiastic and sceptical prior of the INB, an upper (350,000 dollars over 1 year) and lower (175,000 dollars over 1 year) economic cost, respectively, of ERT was derived. CONCLUSION: The cost of ERT will always result in a net deficit to society under current costing and ERT efficacy as determined by the QALY metric. The rules of fair cooperation should govern decision making both for ERT in Fabry- Anderson disease and for funding therapeutic advances in other rare diseases belonging to the orphan and ultra-orphan categories.
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Authors | David F Moore, Markus Ries, Evelyn L Forget, Raphael Schiffmann |
Journal | PharmacoEconomics
(Pharmacoeconomics)
Vol. 25
Issue 3
Pg. 201-8
( 2007)
ISSN: 1170-7690 [Print] New Zealand |
PMID | 17335306
(Publication Type: Journal Article)
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Chemical References |
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Topics |
- Cost of Illness
- Cost-Benefit Analysis
- Fabry Disease
(drug therapy, economics)
- Female
- Humans
- Injections, Intravenous
- Male
- Meta-Analysis as Topic
- Quality of Life
- Rare Diseases
(drug therapy, economics)
- alpha-Galactosidase
(administration & dosage, economics, therapeutic use)
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