Population screening for hereditary haemochromatosis in Australia: construction and validation of a state-transition cost-effectiveness model

Barbara de Graaff, Lei Si, Amanda L. Neil, Kwang Chien Yee, Kristy Sanderson, Lyle C. Gurrin, Andrew J. Palmer

Research output: Contribution to journalArticleResearchpeer-review

Abstract

Introduction: HFE-associated haemochromatosis, the most common monogenic disorder amongst populations of northern European ancestry, is characterised by iron overload. Excess iron is stored in parenchymal tissues, leading to morbidity and mortality. Population screening programmes are likely to improve early diagnosis, thereby decreasing associated disease. Our aim was to develop and validate a health economics model of screening using utilities and costs from a haemochromatosis cohort. Methods: A state-transition model was developed with Markov states based on disease severity. Australian males (aged 30 years) and females (aged 45 years) of northern European ancestry were the target populations. The screening strategy was the status quo approach in Australia; the model was run over a lifetime horizon. Costs were estimated from the government perspective and reported in 2015 Australian dollars ($A); costs and qualityadjusted life-years (QALYs) were discounted at 5% annually. Model validity was assessed using goodness-of- fit analyses. Second-order Monte-Carlo simulation was used to account for uncertainty in multiple parameters. Results: For validity, the model reproduced mortality, life expectancy (LE) and prevalence rates in line with published data. LE for C₂8₂Y homozygote males and females were 49.9 and 40.2 years, respectively, slightly lower than population rates. Mean (95% confidence interval) QALYS were 15.7 (7.7-23.7) for males and 14.4 (6.7-22.1) for females. Mean discounted lifetime costs for C282Y homozygotes were $A22,737 (3670-85,793) for males and $A13,840 (1335-67,377) for females. Sensitivity analyses revealed discount rates and prevalence had the greatest impacts on outcomes. Conclusion: We have developed a transparent, validated health economics model of C₂8₂Y homozygote haemochromatosis. The model will be useful to decision makers to identify cost-effective screening strategies.
LanguageEnglish
Pages37-51
Number of pages15
JournalPharmacoEconomics - Open
Volume1
Issue number1
DOIs
Publication statusPublished - Mar 2017
Externally publishedYes

Fingerprint

Hemochromatosis
Cost-Benefit Analysis
Homozygote
Costs and Cost Analysis
Economic Models
Population
Life Expectancy
Iron Overload
Population Control
Mortality
Health Services Needs and Demand
Health
Reproducibility of Results
Uncertainty
Early Diagnosis
Iron
Confidence Intervals
Morbidity

Bibliographical note

Copyright The Author(s) 2016. This article is published with open access at Springerlink.com. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher.

Keywords

  • Iron Overload
  • C₂8₂Y Homozygote
  • Northern European Ancestry
  • Irreversible Organ Damage
  • C₂8₂Y Homozygosity

Cite this

de Graaff, Barbara ; Si, Lei ; Neil, Amanda L. ; Yee, Kwang Chien ; Sanderson, Kristy ; Gurrin, Lyle C. ; Palmer, Andrew J. / Population screening for hereditary haemochromatosis in Australia : construction and validation of a state-transition cost-effectiveness model. In: PharmacoEconomics - Open. 2017 ; Vol. 1, No. 1. pp. 37-51.
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abstract = "Introduction: HFE-associated haemochromatosis, the most common monogenic disorder amongst populations of northern European ancestry, is characterised by iron overload. Excess iron is stored in parenchymal tissues, leading to morbidity and mortality. Population screening programmes are likely to improve early diagnosis, thereby decreasing associated disease. Our aim was to develop and validate a health economics model of screening using utilities and costs from a haemochromatosis cohort. Methods: A state-transition model was developed with Markov states based on disease severity. Australian males (aged 30 years) and females (aged 45 years) of northern European ancestry were the target populations. The screening strategy was the status quo approach in Australia; the model was run over a lifetime horizon. Costs were estimated from the government perspective and reported in 2015 Australian dollars ($A); costs and qualityadjusted life-years (QALYs) were discounted at 5{\%} annually. Model validity was assessed using goodness-of- fit analyses. Second-order Monte-Carlo simulation was used to account for uncertainty in multiple parameters. Results: For validity, the model reproduced mortality, life expectancy (LE) and prevalence rates in line with published data. LE for C₂8₂Y homozygote males and females were 49.9 and 40.2 years, respectively, slightly lower than population rates. Mean (95{\%} confidence interval) QALYS were 15.7 (7.7-23.7) for males and 14.4 (6.7-22.1) for females. Mean discounted lifetime costs for C282Y homozygotes were $A22,737 (3670-85,793) for males and $A13,840 (1335-67,377) for females. Sensitivity analyses revealed discount rates and prevalence had the greatest impacts on outcomes. Conclusion: We have developed a transparent, validated health economics model of C₂8₂Y homozygote haemochromatosis. The model will be useful to decision makers to identify cost-effective screening strategies.",
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author = "{de Graaff}, Barbara and Lei Si and Neil, {Amanda L.} and Yee, {Kwang Chien} and Kristy Sanderson and Gurrin, {Lyle C.} and Palmer, {Andrew J.}",
note = "Copyright The Author(s) 2016. This article is published with open access at Springerlink.com. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher.",
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Population screening for hereditary haemochromatosis in Australia : construction and validation of a state-transition cost-effectiveness model. / de Graaff, Barbara; Si, Lei; Neil, Amanda L.; Yee, Kwang Chien; Sanderson, Kristy; Gurrin, Lyle C.; Palmer, Andrew J.

In: PharmacoEconomics - Open, Vol. 1, No. 1, 03.2017, p. 37-51.

Research output: Contribution to journalArticleResearchpeer-review

TY - JOUR

T1 - Population screening for hereditary haemochromatosis in Australia

T2 - PharmacoEconomics - Open

AU - de Graaff, Barbara

AU - Si, Lei

AU - Neil, Amanda L.

AU - Yee, Kwang Chien

AU - Sanderson, Kristy

AU - Gurrin, Lyle C.

AU - Palmer, Andrew J.

N1 - Copyright The Author(s) 2016. This article is published with open access at Springerlink.com. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher.

PY - 2017/3

Y1 - 2017/3

N2 - Introduction: HFE-associated haemochromatosis, the most common monogenic disorder amongst populations of northern European ancestry, is characterised by iron overload. Excess iron is stored in parenchymal tissues, leading to morbidity and mortality. Population screening programmes are likely to improve early diagnosis, thereby decreasing associated disease. Our aim was to develop and validate a health economics model of screening using utilities and costs from a haemochromatosis cohort. Methods: A state-transition model was developed with Markov states based on disease severity. Australian males (aged 30 years) and females (aged 45 years) of northern European ancestry were the target populations. The screening strategy was the status quo approach in Australia; the model was run over a lifetime horizon. Costs were estimated from the government perspective and reported in 2015 Australian dollars ($A); costs and qualityadjusted life-years (QALYs) were discounted at 5% annually. Model validity was assessed using goodness-of- fit analyses. Second-order Monte-Carlo simulation was used to account for uncertainty in multiple parameters. Results: For validity, the model reproduced mortality, life expectancy (LE) and prevalence rates in line with published data. LE for C₂8₂Y homozygote males and females were 49.9 and 40.2 years, respectively, slightly lower than population rates. Mean (95% confidence interval) QALYS were 15.7 (7.7-23.7) for males and 14.4 (6.7-22.1) for females. Mean discounted lifetime costs for C282Y homozygotes were $A22,737 (3670-85,793) for males and $A13,840 (1335-67,377) for females. Sensitivity analyses revealed discount rates and prevalence had the greatest impacts on outcomes. Conclusion: We have developed a transparent, validated health economics model of C₂8₂Y homozygote haemochromatosis. The model will be useful to decision makers to identify cost-effective screening strategies.

AB - Introduction: HFE-associated haemochromatosis, the most common monogenic disorder amongst populations of northern European ancestry, is characterised by iron overload. Excess iron is stored in parenchymal tissues, leading to morbidity and mortality. Population screening programmes are likely to improve early diagnosis, thereby decreasing associated disease. Our aim was to develop and validate a health economics model of screening using utilities and costs from a haemochromatosis cohort. Methods: A state-transition model was developed with Markov states based on disease severity. Australian males (aged 30 years) and females (aged 45 years) of northern European ancestry were the target populations. The screening strategy was the status quo approach in Australia; the model was run over a lifetime horizon. Costs were estimated from the government perspective and reported in 2015 Australian dollars ($A); costs and qualityadjusted life-years (QALYs) were discounted at 5% annually. Model validity was assessed using goodness-of- fit analyses. Second-order Monte-Carlo simulation was used to account for uncertainty in multiple parameters. Results: For validity, the model reproduced mortality, life expectancy (LE) and prevalence rates in line with published data. LE for C₂8₂Y homozygote males and females were 49.9 and 40.2 years, respectively, slightly lower than population rates. Mean (95% confidence interval) QALYS were 15.7 (7.7-23.7) for males and 14.4 (6.7-22.1) for females. Mean discounted lifetime costs for C282Y homozygotes were $A22,737 (3670-85,793) for males and $A13,840 (1335-67,377) for females. Sensitivity analyses revealed discount rates and prevalence had the greatest impacts on outcomes. Conclusion: We have developed a transparent, validated health economics model of C₂8₂Y homozygote haemochromatosis. The model will be useful to decision makers to identify cost-effective screening strategies.

KW - Iron Overload

KW - C₂8₂Y Homozygote

KW - Northern European Ancestry

KW - Irreversible Organ Damage

KW - C₂8₂Y Homozygosity

U2 - 10.1007/s41669-016-0005-0

DO - 10.1007/s41669-016-0005-0

M3 - Article

VL - 1

SP - 37

EP - 51

JO - PharmacoEconomics - Open

JF - PharmacoEconomics - Open

SN - 2509-4254

IS - 1

ER -