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ArticleClinicoEconomics and outcomes research : CEOR2019

Cost-Effectiveness Of Midostaurin In The Treatment Of Acute Myeloid Leukemia With The FLT3 Mutation In Spain.

Ainhoa Arenaza, Raúl Diez, Jordi Esteve, Roberta Di Nicolantonio, Joana Gostkorzewicz, Carlos Martínez, Diana Martínez Llinàs, Joaquin Martinez-Lopez, Pau Montesinos, Aída Moure-Fernández and 2 more

Open access · goldAbstract read
In one paragraph

Article in ClinicoEconomics and outcomes research : CEOR, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed, 1 pooled it
0.8field-weighted citation impact, top 25% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed, 1 synthesis or guideline pooled it, 6 citations in OpenAlex.

  1. Pooled it
  2. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

12 authors at 10 institutions in 1 country.

Ainhoa ArenazaPharmacy Department, Clínico San Carlos Hospital, Madrid, Spain.
Raúl DiezPharmacy Department, University De Getafe Hospital, Madrid, Spain.ORCID 0000-0002-9627-5337
Jordi EsteveHematology Department, Clinic Hospital, Barcelona, Spain.
Roberta Di NicolantonioHealth Economics and Outcome Research, Novartis Farmacéutica S.A., Madrid, Spain.
Joana GostkorzewiczHealth Economics and Outcome Research, Novartis Farmacéutica S.A., Madrid, Spain.
Carlos MartínezPharmacy Department, University Hospital Araba, Vitoria, Spain.ORCID 0000-0002-0802-3181
Diana Martínez LlinàsOblikue Consulting, S.L., Barcelona, Spain.
Joaquin Martinez-LopezHematology Department, University Hospital 12 de Octubre, CNIO, Complutense University, Madrid, Spain.ORCID 0000-0001-7908-0063
Pau MontesinosHematology Department, University Hospital La Fe, Valencia, & CIBERONC, Instituto Carlos III, Madrid, Spain.
Aída Moure-FernándezOblikue Consulting, S.L., Barcelona, Spain.
Jorge SierraHematology Department, University Hospital Santa Creu i Sant Pau, Barcelona, Spain.
Joan Lluís VinentPharmacy Department, Sant Joan de Déu Hospital, Barcelona, Spain.
Novartis (Spain) · ESOblikue Consulting (Spain) · ESHospital Clínic de Barcelona · ESHospital Clínico San Carlos · ESHospital de Sant Pau · ESHospital Sant Joan de Déu Barcelona · ESHospital Universitario Araba · ESHospital Universitario de Getafe · ESInstituto de Salud Carlos III · ESUniversidad Complutense de Madrid · ES

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeThe addition of midostaurin to standard chemotherapy (cytarabine and daunorubicin) has shown significant improvements in the survival of patients with acute myeloid leukemia with the FLT3 mutation (FLT3-AML). The objective of this study was to determine whether this intervention would be cost-effective in Spain.

methodsA partitioned survival model with five health states was developed (diagnosis and induction, complete remission, no complete remission, transplantation and death). A lifetime time horizon and the Spanish National Health System perspective were adopted. During the first three years, permanence in the different health states was determined according to the results of the RATIFY study. In successive years, the death rates of the Spanish population adjusted by a factor to reflect long-term disease-related mortality were used. Utilities were obtained from the literature. Pharmacological costs (first and second line) and the costs of other health resources (hospitalizations, visits and tests) were included. The robustness of the model was evaluated by deterministic and probabilistic sensitivity analyses.

resultsThe addition of midostaurin resulted in 1.46 life years gained (LYG) and 1.23 quality-adjusted life years (QALY) gained and implied an additional cost of € 47,955, resulting in an incremental cost-effectiveness ratio (ICER) of € 32,854/LYG and an incremental cost-utility ratio of € 38,985/QALY. In the univariate sensitivity analysis, the threshold of € 50,000/QALY was not exceeded in any case; taking into consideration potential discounts of 20-40% in the PVL of midostaurin the ICER would be below € 30,000/QALY, a commonly accepted threshold in Spain. In the probabilistic analysis, when the threshold was € 50,000/QALY, midostaurin was cost-effective in 82.3% of simulations.

conclusionAccording to our modeling, midostaurin, in combination with standard chemotherapy, could be an efficient alternative for the treatment of FLT3-AML in Spain.

Indexed as

AMLeconomic evaluationefficiencyhealth economicsmodeling

Identifiers

PMID32009807
PMCPMC6859089
OpenAlexW2983082068

What OpenQuestion holds

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LicenceCC BY-NC
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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.