Cost-Utility of Acromegaly Pharmacological Treatments in a French Context

ObjectiveEfficacy of pharmacological treatments for acromegaly has been assessed in many clinical or real-world studies but no study was interested in economics evaluation of these treatments in France. Therefore, the objective of this study was to estimate the cost-utility of second-line pharmacolo...

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Main Authors: Thierry Brue, Philippe Chanson, Patrice Rodien, Brigitte Delemer, Delphine Drui, Lucile Marié, Laurène Juban, Lara Salvi, Robin Henocque, Gérald Raverot
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-10-01
Series:Frontiers in Endocrinology
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fendo.2021.745843/full
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spelling doaj-5c4b68987749411bbc7ec693382e94e62021-10-08T07:40:28ZengFrontiers Media S.A.Frontiers in Endocrinology1664-23922021-10-011210.3389/fendo.2021.745843745843Cost-Utility of Acromegaly Pharmacological Treatments in a French ContextThierry Brue0Thierry Brue1Philippe Chanson2Patrice Rodien3Brigitte Delemer4Delphine Drui5Lucile Marié6Laurène Juban7Lara Salvi8Robin Henocque9Gérald Raverot10Assistance Publique-Hôpitaux de Marseille (AP-HM), Department of Endocrinology, Hôpital de la Conception, Centre de Référence des Maladies Rares de l’hypophyse HYPO, Marseille, FranceAix-Marseille Université, Institut National de la Santé et de la Recherche Médicale (INSERM), U1251, Marseille Medical Genetics (MMG), Institut Marseille Maladies Rares (MarMaRa), Marseille, FranceUniversité Paris-Saclay, Inserm, Physiologie et Physiopathologie Endocriniennes, Assistance Publique-Hôpitaux de Paris, Hôpital Bicêtre, Service d’Endocrinologie et des Maladies de la Reproduction, Centre de Référence des Maladies Rares de l’Hypophyse, Le Kremlin-Bicêtre, FranceUniversité d’Angers, CHU d’Angers, service d’Endocrinologie-Diabétologie-Nutrition, Centre de Référence des Maladies Rares de l’Hypophyse, Angers, FranceCHU de Reims - Hôpital Robert Debré, Service d’Endocrinologie – Diabète – Nutrition, Reims Cedex, FranceEndocrinology Department, L’institut du thorax, University Hospital of Nantes, Nantes Cedex, FranceStève Consultants, Paris, FranceStève Consultants, Paris, FranceRare Disease, Pfizer France, Paris Cedex, FranceStève Consultants, Paris, FranceEndocrinology Department, “Groupement Hospitalier Est” Hospices Civils de Lyon, Bron, FranceObjectiveEfficacy of pharmacological treatments for acromegaly has been assessed in many clinical or real-world studies but no study was interested in economics evaluation of these treatments in France. Therefore, the objective of this study was to estimate the cost-utility of second-line pharmacological treatments in acromegaly patients.MethodsA Markov model was developed to follow a cohort of 1,000 patients for a lifetime horizon. First-generation somatostatin analogues (FGSA), pegvisomant, pasireotide and pegvisomant combined with FGSA (off label) were compared. Efficacy was defined as the normalization of insulin-like growth factor-1 (IGF-1) concentration and was obtained from pivotal trials and adjusted by a network meta-analysis. Costs data were obtained from French databases and literature. Utilities from the literature were used to estimate quality-adjusted life year (QALY).ResultsThe incremental cost-utility ratios (ICUR) of treatments compared to FGSA were estimated to be 562,463 € per QALY gained for pasireotide, 171,332 € per QALY gained for pegvisomant, and 186,242 € per QALY gained for pegvisomant + FGSA. Pasireotide seems to be the least cost-efficient treatment. Sensitivity analyses showed the robustness of the results.ConclusionFGSA, pegvisomant and pegvisomant + FGSA were on the cost-effective frontier, therefore, depending on the willingness-to-pay for an additional QALY, they are the most cost-effective treatments. This medico-economic analysis highlighted the consistency of the efficiency results with the efficacy results assessed in the pivotal trials. However, most recent treatment guidelines recommend an individualized treatment strategy based on the patient and disease profile.https://www.frontiersin.org/articles/10.3389/fendo.2021.745843/fullacromegalycost-utilitygrowth hormoneinsulin-like growth factor-1QALYpasireotide
collection DOAJ
language English
format Article
sources DOAJ
author Thierry Brue
Thierry Brue
Philippe Chanson
Patrice Rodien
Brigitte Delemer
Delphine Drui
Lucile Marié
Laurène Juban
Lara Salvi
Robin Henocque
Gérald Raverot
spellingShingle Thierry Brue
Thierry Brue
Philippe Chanson
Patrice Rodien
Brigitte Delemer
Delphine Drui
Lucile Marié
Laurène Juban
Lara Salvi
Robin Henocque
Gérald Raverot
Cost-Utility of Acromegaly Pharmacological Treatments in a French Context
Frontiers in Endocrinology
acromegaly
cost-utility
growth hormone
insulin-like growth factor-1
QALY
pasireotide
author_facet Thierry Brue
Thierry Brue
Philippe Chanson
Patrice Rodien
Brigitte Delemer
Delphine Drui
Lucile Marié
Laurène Juban
Lara Salvi
Robin Henocque
Gérald Raverot
author_sort Thierry Brue
title Cost-Utility of Acromegaly Pharmacological Treatments in a French Context
title_short Cost-Utility of Acromegaly Pharmacological Treatments in a French Context
title_full Cost-Utility of Acromegaly Pharmacological Treatments in a French Context
title_fullStr Cost-Utility of Acromegaly Pharmacological Treatments in a French Context
title_full_unstemmed Cost-Utility of Acromegaly Pharmacological Treatments in a French Context
title_sort cost-utility of acromegaly pharmacological treatments in a french context
publisher Frontiers Media S.A.
series Frontiers in Endocrinology
issn 1664-2392
publishDate 2021-10-01
description ObjectiveEfficacy of pharmacological treatments for acromegaly has been assessed in many clinical or real-world studies but no study was interested in economics evaluation of these treatments in France. Therefore, the objective of this study was to estimate the cost-utility of second-line pharmacological treatments in acromegaly patients.MethodsA Markov model was developed to follow a cohort of 1,000 patients for a lifetime horizon. First-generation somatostatin analogues (FGSA), pegvisomant, pasireotide and pegvisomant combined with FGSA (off label) were compared. Efficacy was defined as the normalization of insulin-like growth factor-1 (IGF-1) concentration and was obtained from pivotal trials and adjusted by a network meta-analysis. Costs data were obtained from French databases and literature. Utilities from the literature were used to estimate quality-adjusted life year (QALY).ResultsThe incremental cost-utility ratios (ICUR) of treatments compared to FGSA were estimated to be 562,463 € per QALY gained for pasireotide, 171,332 € per QALY gained for pegvisomant, and 186,242 € per QALY gained for pegvisomant + FGSA. Pasireotide seems to be the least cost-efficient treatment. Sensitivity analyses showed the robustness of the results.ConclusionFGSA, pegvisomant and pegvisomant + FGSA were on the cost-effective frontier, therefore, depending on the willingness-to-pay for an additional QALY, they are the most cost-effective treatments. This medico-economic analysis highlighted the consistency of the efficiency results with the efficacy results assessed in the pivotal trials. However, most recent treatment guidelines recommend an individualized treatment strategy based on the patient and disease profile.
topic acromegaly
cost-utility
growth hormone
insulin-like growth factor-1
QALY
pasireotide
url https://www.frontiersin.org/articles/10.3389/fendo.2021.745843/full
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