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
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.