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

• Published in: ClinicoEconomics and outcomes research Vol. 11; pp. 673 - 681
• Main Authors: Tremblay, Gabriel, Said, Qayyim, Roy, Anuja Nidumolu, Cai, Beilei, Ashton Garib, Shan, Hearnden, Jaclyn, Forsythe, Anna
• Format: Journal Article
• Language: English
• Published: New Zealand Dove Medical Press Limited 01-11-2019; Taylor & Francis Ltd; Dove; Dove Medical Press
• Subjects: Analysis; Anemia; Aplastic anemia; Blood; Bone marrow; budget impact analysis; Budgets; Care and treatment; Consumer Price Index; Costs; Cyclosporins; Drug dosages; Eltrombopag; Epidemiology; Health aspects; Health care policy; Immunotherapy; Impact analysis; Mortality; Original Research; Pharmacoeconomics; Quality of life; Response rates; severe aplastic anemia; Stem cells; thrombopoietin receptor agonist; Toxicity; Transplants & implants; united states; New York; United States > US; Europe; severe aplastic anemia; costs; thrombopoietin receptor agonist; budget impact analysis; United States; eltrombopag
• ISSN: 1178-6981; 1178-6981
• DOI: 10.2147/CEOR.S226323

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.
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.
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.
High response rates combined with reduced rescue medication use and mortality in patients treated with eltrombopag and IST mediated higher medication costs.