{"title":"用于卫生技术评估的混合与非混合治愈模型:您需要了解的知识。","authors":"Nicholas R Latimer, Mark J Rutherford","doi":"10.1007/s40273-024-01406-7","DOIUrl":null,"url":null,"abstract":"<p><p>There is increasing interest in the use of cure modelling to inform health technology assessment (HTA) due to the development of new treatments that appear to offer the potential for cure in some patients. However, cure models are often not included in evidence dossiers submitted to HTA agencies, and they are relatively rarely relied upon to inform decision-making. This is likely due to a lack of understanding of how cure models work, what they assume, and how reliable they are. In this tutorial we explain why and when cure models may be useful for HTA, describe the key characteristics of mixture and non-mixture cure models, and demonstrate their use in a range of scenarios, providing Stata code. We highlight key issues that must be taken into account by analysts when fitting these models and by reviewers and decision-makers when interpreting their predictions. In particular, we note that flexible parametric non-mixture cure models have not been used in HTA, but they offer advantages that make them well suited to an HTA context when a cure assumption is valid but follow-up is limited.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1073-1090"},"PeriodicalIF":4.4000,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11405446/pdf/","citationCount":"0","resultStr":"{\"title\":\"Mixture and Non-mixture Cure Models for Health Technology Assessment: What You Need to Know.\",\"authors\":\"Nicholas R Latimer, Mark J Rutherford\",\"doi\":\"10.1007/s40273-024-01406-7\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>There is increasing interest in the use of cure modelling to inform health technology assessment (HTA) due to the development of new treatments that appear to offer the potential for cure in some patients. However, cure models are often not included in evidence dossiers submitted to HTA agencies, and they are relatively rarely relied upon to inform decision-making. This is likely due to a lack of understanding of how cure models work, what they assume, and how reliable they are. In this tutorial we explain why and when cure models may be useful for HTA, describe the key characteristics of mixture and non-mixture cure models, and demonstrate their use in a range of scenarios, providing Stata code. We highlight key issues that must be taken into account by analysts when fitting these models and by reviewers and decision-makers when interpreting their predictions. In particular, we note that flexible parametric non-mixture cure models have not been used in HTA, but they offer advantages that make them well suited to an HTA context when a cure assumption is valid but follow-up is limited.</p>\",\"PeriodicalId\":19807,\"journal\":{\"name\":\"PharmacoEconomics\",\"volume\":\" \",\"pages\":\"1073-1090\"},\"PeriodicalIF\":4.4000,\"publicationDate\":\"2024-10-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11405446/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"PharmacoEconomics\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s40273-024-01406-7\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/7/5 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q1\",\"JCRName\":\"ECONOMICS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"PharmacoEconomics","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s40273-024-01406-7","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/7/5 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"ECONOMICS","Score":null,"Total":0}
引用次数: 0
摘要
由于新疗法的开发似乎为某些患者提供了治愈的可能,人们对使用治愈模型为健康技术评估(HTA)提供信息的兴趣与日俱增。然而,治愈模型通常不包括在提交给 HTA 机构的证据档案中,也很少被用来作为决策依据。这可能是由于人们对治愈模型的工作原理、假设条件以及可靠性缺乏了解。在本教程中,我们将解释为何以及何时固化模型可能对 HTA 有用,描述混合和非混合固化模型的主要特征,并提供 Stata 代码演示其在各种情况下的使用。我们强调了分析人员在拟合这些模型时以及评审人员和决策者在解释其预测时必须考虑的关键问题。我们特别指出,灵活的参数非混杂治愈模型尚未用于 HTA,但它们的优势使其非常适合于治愈假设有效但随访有限的 HTA 情况。
Mixture and Non-mixture Cure Models for Health Technology Assessment: What You Need to Know.
There is increasing interest in the use of cure modelling to inform health technology assessment (HTA) due to the development of new treatments that appear to offer the potential for cure in some patients. However, cure models are often not included in evidence dossiers submitted to HTA agencies, and they are relatively rarely relied upon to inform decision-making. This is likely due to a lack of understanding of how cure models work, what they assume, and how reliable they are. In this tutorial we explain why and when cure models may be useful for HTA, describe the key characteristics of mixture and non-mixture cure models, and demonstrate their use in a range of scenarios, providing Stata code. We highlight key issues that must be taken into account by analysts when fitting these models and by reviewers and decision-makers when interpreting their predictions. In particular, we note that flexible parametric non-mixture cure models have not been used in HTA, but they offer advantages that make them well suited to an HTA context when a cure assumption is valid but follow-up is limited.
期刊介绍:
PharmacoEconomics is the benchmark journal for peer-reviewed, authoritative and practical articles on the application of pharmacoeconomics and quality-of-life assessment to optimum drug therapy and health outcomes. An invaluable source of applied pharmacoeconomic original research and educational material for the healthcare decision maker.
PharmacoEconomics is dedicated to the clear communication of complex pharmacoeconomic issues related to patient care and drug utilization.
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