{"title":"Parametric analysis and model selection for economic evaluation of survival data","authors":"Szilárd Nemes","doi":"10.3233/mas-241506","DOIUrl":null,"url":null,"abstract":"Health technology assessments of interventions impacting survival often require extrapolating current data to gain a better understanding of the interventions’ long-term benefits. Both a comprehensive examination of the trial data up to the maximum follow-up period and the fitting of parametric models are required for extrapolation. It is standard practice to visually compare the parametric curves to the Kaplan-Meier survival estimate (or comparison of hazard estimates) and to assess the parametric models using likelihood-based information criteria. In place of these two steps, this work demonstrates how to minimize the squared distance of parametric estimators to the Kaplan-Meier estimate. This is in line with the selection of the model using Mean Squared Error, with the modification that the unknown true survival is replaced by the Kaplan-Meier estimate. We would assure the internal validity of the extrapolated model and its appropriate representation of the data by adhering to this procedure. We use both simulation and real-world data with a scenario where no model that properly fits the data could be found to illustrate how this process can aid in model selection.","PeriodicalId":35000,"journal":{"name":"Model Assisted Statistics and Applications","volume":"85 2","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Model Assisted Statistics and Applications","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.3233/mas-241506","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"Mathematics","Score":null,"Total":0}
引用次数: 0
Abstract
Health technology assessments of interventions impacting survival often require extrapolating current data to gain a better understanding of the interventions’ long-term benefits. Both a comprehensive examination of the trial data up to the maximum follow-up period and the fitting of parametric models are required for extrapolation. It is standard practice to visually compare the parametric curves to the Kaplan-Meier survival estimate (or comparison of hazard estimates) and to assess the parametric models using likelihood-based information criteria. In place of these two steps, this work demonstrates how to minimize the squared distance of parametric estimators to the Kaplan-Meier estimate. This is in line with the selection of the model using Mean Squared Error, with the modification that the unknown true survival is replaced by the Kaplan-Meier estimate. We would assure the internal validity of the extrapolated model and its appropriate representation of the data by adhering to this procedure. We use both simulation and real-world data with a scenario where no model that properly fits the data could be found to illustrate how this process can aid in model selection.
期刊介绍:
Model Assisted Statistics and Applications is a peer reviewed international journal. Model Assisted Statistics means an improvement of inference and analysis by use of correlated information, or an underlying theoretical or design model. This might be the design, adjustment, estimation, or analytical phase of statistical project. This information may be survey generated or coming from an independent source. Original papers in the field of sampling theory, econometrics, time-series, design of experiments, and multivariate analysis will be preferred. Papers of both applied and theoretical topics are acceptable.