New IPECAD open-source model framework for the health technology assessment of early Alzheimer's disease treatment: development and use cases.

Alzheimer’s disease cross-validation decision-analytic modeling health-economic evaluation open-source modeling

Journal

Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
ISSN: 1524-4733
Titre abrégé: Value Health
Pays: United States
ID NLM: 100883818

Informations de publication

Date de publication:
31 Jul 2024
Historique:
received: 02 04 2024
revised: 02 07 2024
accepted: 21 07 2024
medline: 3 8 2024
pubmed: 3 8 2024
entrez: 2 8 2024
Statut: aheadofprint

Résumé

Reimbursement decisions for new Alzheimer's disease (AD) treatments are informed by economic evaluations. An open-source model with intuitive structure for model cross-validation can support the transparency and credibility of such evaluations. We describe the new IPECAD open-source model framework (version 2) for the health-economic evaluation of early AD treatment and use it for cross-validation and addressing uncertainty. A cohort state transition model using a categorized composite domain (cognition and function) was developed by replicating an existing reference model and testing it for internal validity. Then, features of existing "ICER" and "AD-ACE" models assessing lecanemab treatment were implemented for model cross-validation. Additional uncertainty scenarios were performed on choice of efficacy outcome from trial, natural disease progression, treatment effect waning and stopping rules, and other methodological choices. The model is available open-source as R code, spreadsheet and web-based version via https://github.com/ronhandels/IPECAD. In the IPECAD model incremental life years, QALY gains and cost savings were 21-31% smaller compared to the ICER model and 36-56% smaller compared to the AD-ACE model. IPECAD model results were particularly sensitive to assumptions on treatment effect waning and stopping rules and choice of efficacy outcome from trial. We demonstrated the ability of a new IPECAD opens-source model framework for researchers and decision-makers to cross-validate other (HTA submission) models and perform additional uncertainty analyses, setting an example for open science in AD decision modeling and supporting important reimbursement decisions.

Identifiants

pubmed: 39094686
pii: S1098-3015(24)02790-6
doi: 10.1016/j.jval.2024.07.009
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

Copyright © 2024. Published by Elsevier Inc.

Auteurs

Ron Handels (R)

Alzheimer Centre Limburg, Faculty of Health Medicine and Life Sciences, Mental Health and Neuroscience Research Institute, Department of Psychiatry and Neuropsychology; Maastricht University; The Netherlands; 6200 MD, Maastricht; Division of Neurogeriatrics, Department of Neurobiology Care Sciences and Society; Karolinska Institutet; Sweden; BioClinicum J9:20, Akademiska stråket 171 64 Solna. Electronic address: ron.handels@maastrichtuniversity.nl.

William L Herring (WL)

Division of Neurogeriatrics, Department of Neurobiology Care Sciences and Society; Karolinska Institutet; Sweden; BioClinicum J9:20, Akademiska stråket 171 64 Solna; Health Economics; RTI Health Solutions; USA; 3040 East Cornwallis Road, Research Triangle Park, NC 27709.

Sabine Grimm (S)

Department of Clinical Epidemiology and Medical Technology Assessment, School for Public Health and Primary Care (CAPHRI); Maastricht University Medical Centre; The Netherlands; P. Debyelaan 25, PO Box 5800, 6202 AZ Maastricht.

Anders Sköldunger (A)

Division of Neurogeriatrics, Department of Neurobiology Care Sciences and Society; Karolinska Institutet; Sweden; BioClinicum J9:20, Akademiska stråket 171 64 Solna.

Bengt Winblad (B)

Division of Neurogeriatrics, Department of Neurobiology Care Sciences and Society; Karolinska Institutet; Sweden; BioClinicum J9:20, Akademiska stråket 171 64 Solna; Theme Inflammation and Aging; Karolinska University Hospital; Sweden; Huddinge.

Anders Wimo (A)

Division of Neurogeriatrics, Department of Neurobiology Care Sciences and Society; Karolinska Institutet; Sweden; BioClinicum J9:20, Akademiska stråket 171 64 Solna.

Linus Jönsson (L)

Division of Neurogeriatrics, Department of Neurobiology Care Sciences and Society; Karolinska Institutet; Sweden; BioClinicum J9:20, Akademiska stråket 171 64 Solna.

Classifications MeSH