Pamela P Pei, Milton C Weinstein, X Cynthia Li, Michael D Hughes, A David Paltiel, Taige Hou, Robert A Parker, Melanie R Gaynes, Paul E Sax, Kenneth A Freedberg, Bruce R Schackman, Rochelle P Walensky
{"title":"基于信息价值优先考虑HIV比较有效性试验:在美国仿制药与品牌抗逆转录病毒药物。","authors":"Pamela P Pei, Milton C Weinstein, X Cynthia Li, Michael D Hughes, A David Paltiel, Taige Hou, Robert A Parker, Melanie R Gaynes, Paul E Sax, Kenneth A Freedberg, Bruce R Schackman, Rochelle P Walensky","doi":"10.1080/15284336.2015.1123942","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Value of Information (VOI) analysis examines whether to acquire information before making a decision. We introduced VOI to the HIV audience, using the example of generic antiretroviral therapy (ART) in the US.</p><p><strong>Methods and findings: </strong>We used a mathematical model and probabilistic sensitivity analysis (PSA) to generate probability distributions of survival (in quality-adjusted life years, QALYs) and cost for three potential first-line ART regimens: three-pill generic, two-pill generic, and single-pill branded. These served as input for a comparison of two hypothetical two-arm trials: three-pill generic versus single-pill branded; and two-pill generic versus single-pill branded. We modeled pre-trial uncertainty by defining probability distributions around key inputs, including 24-week HIV-RNA suppression and subsequent ART failure. We assumed that, without a trial, patients received the single-pill branded strategy. Post-trial, we assumed that patients received the most cost-effective strategy. For both trials, we quantified the probability of changing to a generic-based regimen upon trial completion and the expected VOI in terms of improved health outcomes and costs. Assuming a willingness to pay (WTP) threshold of $100 000/QALY, the three-pill trial led to more treatment changes (84%) than the two-pill trial (78%). Estimated VOI was $48 000 (three-pill trial) and $35 700 (two-pill trial) per future patient initiating ART.</p><p><strong>Conclusions: </strong>A three-pill trial of generic ART is more likely to lead to post-trial treatment changes and to provide more value than a two-pill trial if policy decisions are based on cost-effectiveness. Value of Information analysis can identify trials likely to confer the greatest impact and value for HIV care.</p>","PeriodicalId":13216,"journal":{"name":"HIV Clinical Trials","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2015-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/15284336.2015.1123942","citationCount":"4","resultStr":"{\"title\":\"Prioritizing HIV comparative effectiveness trials based on value of information: generic versus brand-name ART in the US.\",\"authors\":\"Pamela P Pei, Milton C Weinstein, X Cynthia Li, Michael D Hughes, A David Paltiel, Taige Hou, Robert A Parker, Melanie R Gaynes, Paul E Sax, Kenneth A Freedberg, Bruce R Schackman, Rochelle P Walensky\",\"doi\":\"10.1080/15284336.2015.1123942\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Value of Information (VOI) analysis examines whether to acquire information before making a decision. We introduced VOI to the HIV audience, using the example of generic antiretroviral therapy (ART) in the US.</p><p><strong>Methods and findings: </strong>We used a mathematical model and probabilistic sensitivity analysis (PSA) to generate probability distributions of survival (in quality-adjusted life years, QALYs) and cost for three potential first-line ART regimens: three-pill generic, two-pill generic, and single-pill branded. These served as input for a comparison of two hypothetical two-arm trials: three-pill generic versus single-pill branded; and two-pill generic versus single-pill branded. We modeled pre-trial uncertainty by defining probability distributions around key inputs, including 24-week HIV-RNA suppression and subsequent ART failure. We assumed that, without a trial, patients received the single-pill branded strategy. Post-trial, we assumed that patients received the most cost-effective strategy. For both trials, we quantified the probability of changing to a generic-based regimen upon trial completion and the expected VOI in terms of improved health outcomes and costs. Assuming a willingness to pay (WTP) threshold of $100 000/QALY, the three-pill trial led to more treatment changes (84%) than the two-pill trial (78%). Estimated VOI was $48 000 (three-pill trial) and $35 700 (two-pill trial) per future patient initiating ART.</p><p><strong>Conclusions: </strong>A three-pill trial of generic ART is more likely to lead to post-trial treatment changes and to provide more value than a two-pill trial if policy decisions are based on cost-effectiveness. Value of Information analysis can identify trials likely to confer the greatest impact and value for HIV care.</p>\",\"PeriodicalId\":13216,\"journal\":{\"name\":\"HIV Clinical Trials\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2015-11-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://sci-hub-pdf.com/10.1080/15284336.2015.1123942\",\"citationCount\":\"4\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"HIV Clinical Trials\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1080/15284336.2015.1123942\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2015/12/11 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q2\",\"JCRName\":\"Medicine\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"HIV Clinical Trials","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1080/15284336.2015.1123942","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2015/12/11 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"Medicine","Score":null,"Total":0}
引用次数: 4
摘要
背景:信息价值(Value of Information, VOI)分析考察的是在做出决策之前是否需要获取信息。我们以美国的非专利抗逆转录病毒治疗(ART)为例,向艾滋病毒受众介绍了VOI。方法和发现:我们使用数学模型和概率敏感性分析(PSA)来生成三种潜在一线ART方案(三粒仿制药、两粒仿制药和单粒品牌药)的生存(质量调整生命年,QALYs)和成本的概率分布。这些作为两项假设的两组试验的比较输入:三粒仿制药与单粒品牌药;两粒非专利药和单粒品牌药。我们通过定义关键输入的概率分布来模拟试验前的不确定性,包括24周HIV-RNA抑制和随后的ART失败。我们假设,在没有试验的情况下,患者接受的是单药品牌策略。试验后,我们假设患者接受了最具成本效益的策略。对于这两项试验,我们量化了试验完成后改为基于仿制药方案的可能性,以及在改善健康结果和成本方面的预期VOI。假设支付意愿(WTP)阈值为10万美元/QALY,三丸试验导致的治疗变化(84%)多于两丸试验(78%)。估计VOI为48000美元(三粒试验)和35700美元(两粒试验)每个未来开始抗逆转录病毒治疗的患者。结论:如果政策决定是基于成本效益的,那么三粒非专利抗逆转录病毒药物试验更有可能导致试验后治疗的改变,并且比两粒试验提供更多的价值。信息价值分析可以确定可能对艾滋病毒护理产生最大影响和价值的试验。
Prioritizing HIV comparative effectiveness trials based on value of information: generic versus brand-name ART in the US.
Background: Value of Information (VOI) analysis examines whether to acquire information before making a decision. We introduced VOI to the HIV audience, using the example of generic antiretroviral therapy (ART) in the US.
Methods and findings: We used a mathematical model and probabilistic sensitivity analysis (PSA) to generate probability distributions of survival (in quality-adjusted life years, QALYs) and cost for three potential first-line ART regimens: three-pill generic, two-pill generic, and single-pill branded. These served as input for a comparison of two hypothetical two-arm trials: three-pill generic versus single-pill branded; and two-pill generic versus single-pill branded. We modeled pre-trial uncertainty by defining probability distributions around key inputs, including 24-week HIV-RNA suppression and subsequent ART failure. We assumed that, without a trial, patients received the single-pill branded strategy. Post-trial, we assumed that patients received the most cost-effective strategy. For both trials, we quantified the probability of changing to a generic-based regimen upon trial completion and the expected VOI in terms of improved health outcomes and costs. Assuming a willingness to pay (WTP) threshold of $100 000/QALY, the three-pill trial led to more treatment changes (84%) than the two-pill trial (78%). Estimated VOI was $48 000 (three-pill trial) and $35 700 (two-pill trial) per future patient initiating ART.
Conclusions: A three-pill trial of generic ART is more likely to lead to post-trial treatment changes and to provide more value than a two-pill trial if policy decisions are based on cost-effectiveness. Value of Information analysis can identify trials likely to confer the greatest impact and value for HIV care.
期刊介绍:
HIV Clinical Trials is devoted exclusively to presenting information on the latest developments in HIV/AIDS clinical research. This journal enables readers to obtain the most up-to-date, innovative research from around the world.