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Budget Impact Of Eltrombopag As First-Line Treatment For Severe Aplastic Anemia In The United States

BACKGROUND: Severe aplastic anemia (SAA) is a rare autoimmune condition resulting in low blood cell counts across lineages. Immunosuppressive therapy (IST) has demonstrated low response, toxicity, and risk of transformation. In a Phase I/II trial, the addition of eltrombopag to first-line IST increa...

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Autores principales: Tremblay, Gabriel, Said, Qayyim, Roy, Anuja Nidumolu, Cai, Beilei, Ashton Garib, Shan, Hearnden, Jaclyn, Forsythe, Anna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6858800/
https://www.ncbi.nlm.nih.gov/pubmed/31814747
http://dx.doi.org/10.2147/CEOR.S226323
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author Tremblay, Gabriel
Said, Qayyim
Roy, Anuja Nidumolu
Cai, Beilei
Ashton Garib, Shan
Hearnden, Jaclyn
Forsythe, Anna
author_facet Tremblay, Gabriel
Said, Qayyim
Roy, Anuja Nidumolu
Cai, Beilei
Ashton Garib, Shan
Hearnden, Jaclyn
Forsythe, Anna
author_sort Tremblay, Gabriel
collection PubMed
description BACKGROUND: Severe aplastic anemia (SAA) is a rare autoimmune condition resulting in low blood cell counts across lineages. Immunosuppressive therapy (IST) has demonstrated low response, toxicity, and risk of transformation. In a Phase I/II trial, the addition of eltrombopag to first-line IST increased response rates relative to an IST-only historical cohort. METHODS: A model was developed to estimate the budget impact of treating SAA with eltrombopag-based therapy from a US private healthcare system perspective. A simulated cohort of newly diagnosed SAA patients based on the total US population received 6 months of IST ± eltrombopag and were followed for 1 year, with mutually exclusive patient cohorts entering in years 1, 2, and 3. The model assessed the budget impact of first-year treatment for each cohort without considering subsequent years. At 6 months, responders in either arm received maintenance therapy (low-dose cyclosporine), and non-responders received 6 months of second-line eltrombopag monotherapy. Costs considered included first-line, maintenance, and second-line therapy, administration, routine care, mortality, and adverse events (AEs). All cost data were reported in 2018 US dollars. RESULTS: The annual incidence of aplastic anemia was 0.000234%, with 83.8% of cases assumed to be SAA. Based on trial data, 94% of patients receiving eltrombopag and IST responded versus 66% of patients receiving IST, with a 0.3% reduction in the annual risk of mortality for the eltrombopag + IST group. Use of first-line eltrombopag in a model SAA population based on the total US population increased overall costs by $50 million over 3 years. First-line drug costs accounted for an increase of $69 million, while improved response produced $19 million in secondary therapy cost savings. Sensitivity analyses confirmed the robustness of the analysis. CONCLUSION: High response rates combined with reduced rescue medication use and mortality in patients treated with eltrombopag and IST mediated higher medication costs.
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spelling pubmed-68588002019-12-06 Budget Impact Of Eltrombopag As First-Line Treatment For Severe Aplastic Anemia In The United States Tremblay, Gabriel Said, Qayyim Roy, Anuja Nidumolu Cai, Beilei Ashton Garib, Shan Hearnden, Jaclyn Forsythe, Anna Clinicoecon Outcomes Res Original Research BACKGROUND: Severe aplastic anemia (SAA) is a rare autoimmune condition resulting in low blood cell counts across lineages. Immunosuppressive therapy (IST) has demonstrated low response, toxicity, and risk of transformation. In a Phase I/II trial, the addition of eltrombopag to first-line IST increased response rates relative to an IST-only historical cohort. METHODS: A model was developed to estimate the budget impact of treating SAA with eltrombopag-based therapy from a US private healthcare system perspective. A simulated cohort of newly diagnosed SAA patients based on the total US population received 6 months of IST ± eltrombopag and were followed for 1 year, with mutually exclusive patient cohorts entering in years 1, 2, and 3. The model assessed the budget impact of first-year treatment for each cohort without considering subsequent years. At 6 months, responders in either arm received maintenance therapy (low-dose cyclosporine), and non-responders received 6 months of second-line eltrombopag monotherapy. Costs considered included first-line, maintenance, and second-line therapy, administration, routine care, mortality, and adverse events (AEs). All cost data were reported in 2018 US dollars. RESULTS: The annual incidence of aplastic anemia was 0.000234%, with 83.8% of cases assumed to be SAA. Based on trial data, 94% of patients receiving eltrombopag and IST responded versus 66% of patients receiving IST, with a 0.3% reduction in the annual risk of mortality for the eltrombopag + IST group. Use of first-line eltrombopag in a model SAA population based on the total US population increased overall costs by $50 million over 3 years. First-line drug costs accounted for an increase of $69 million, while improved response produced $19 million in secondary therapy cost savings. Sensitivity analyses confirmed the robustness of the analysis. CONCLUSION: High response rates combined with reduced rescue medication use and mortality in patients treated with eltrombopag and IST mediated higher medication costs. Dove 2019-11-12 /pmc/articles/PMC6858800/ /pubmed/31814747 http://dx.doi.org/10.2147/CEOR.S226323 Text en © 2019 Tremblay et al. http://creativecommons.org/licenses/by-nc/3.0/ This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution – Non Commercial (unported, v3.0) License (http://creativecommons.org/licenses/by-nc/3.0/). By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms (https://www.dovepress.com/terms.php).
spellingShingle Original Research
Tremblay, Gabriel
Said, Qayyim
Roy, Anuja Nidumolu
Cai, Beilei
Ashton Garib, Shan
Hearnden, Jaclyn
Forsythe, Anna
Budget Impact Of Eltrombopag As First-Line Treatment For Severe Aplastic Anemia In The United States
title Budget Impact Of Eltrombopag As First-Line Treatment For Severe Aplastic Anemia In The United States
title_full Budget Impact Of Eltrombopag As First-Line Treatment For Severe Aplastic Anemia In The United States
title_fullStr Budget Impact Of Eltrombopag As First-Line Treatment For Severe Aplastic Anemia In The United States
title_full_unstemmed Budget Impact Of Eltrombopag As First-Line Treatment For Severe Aplastic Anemia In The United States
title_short Budget Impact Of Eltrombopag As First-Line Treatment For Severe Aplastic Anemia In The United States
title_sort budget impact of eltrombopag as first-line treatment for severe aplastic anemia in the united states
topic Original Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6858800/
https://www.ncbi.nlm.nih.gov/pubmed/31814747
http://dx.doi.org/10.2147/CEOR.S226323
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