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Cost-Effectiveness Of Midostaurin In The Treatment Of Acute Myeloid Leukemia With The FLT3 Mutation In Spain
Ainhoa Arenaza1, Raúl Diez2, Jordi Esteve3
1Pharmacy Department, Clínico San Carlos Hospital, Madrid, Spain.
Clinicoeconomics and Outcomes Research : CEOR
|February 4, 2020
Summary
Midostaurin plus standard chemotherapy offers improved survival for FLT3-mutated acute myeloid leukemia (FLT3-AML). This study indicates it is a cost-effective treatment option in Spain, with an ICER below €30,000/QALY under potential discounts.
Area of Science:
- Hematology
- Oncology
- Health Economics
Background:
- Acute myeloid leukemia (AML) with FLT3 mutation (FLT3-AML) presents a significant therapeutic challenge.
- Standard chemotherapy combined with midostaurin has demonstrated survival benefits in FLT3-AML patients.
- Evaluating the economic viability of novel treatments is crucial for healthcare systems.
Purpose of the Study:
- To assess the cost-effectiveness of adding midostaurin to standard chemotherapy for FLT3-AML in Spain.
- To determine if this combination therapy represents an efficient use of National Health System resources.
Main Methods:
- A partitioned survival model with five health states and a lifetime horizon was employed.
- Data from the RATIFY study informed the initial three years, followed by Spanish population mortality rates.
- Costs included pharmacological treatments and healthcare resource utilization; sensitivity analyses were performed.
Main Results:
- The midostaurin combination yielded 1.46 life years gained (LYG) and 1.23 quality-adjusted life years (QALY) gained.
- The incremental cost-effectiveness ratio (ICER) was €38,985/QALY.
- Sensitivity analyses indicated cost-effectiveness below €30,000/QALY with potential midostaurin discounts, and 82.3% cost-effectiveness at a €50,000/QALY threshold.
Conclusions:
- Midostaurin combined with standard chemotherapy is projected to be a cost-effective strategy for FLT3-AML in Spain.
- The modeling suggests this intervention aligns with commonly accepted cost-effectiveness thresholds in the Spanish healthcare system.

