{"title":"随机临床试验开始后,终点何时以及如何改变?","authors":"Scott Evans","doi":"10.1371/journal.pctr.0020018","DOIUrl":null,"url":null,"abstract":"Endpoints are outcome measures used to address the objectives of a clinical trial. The primary endpoint is the most important outcome and is used to assess the primary objective of a trial (e.g., the variable used to compare the effect difference of two treatment groups). A fundamental principle in the design of randomized trials involves setting out in advance the endpoints that will be assessed in the trial [1], as failure to prespecify endpoints can introduce bias into a trial and creates opportunities for manipulation. However, sometimes new information may come to light that could merit changes to endpoints during the course of a trial. This new information might include, for example, results from other trials or identification of better biomarkers or surrogate outcome measures. Such changes can allow incorporation of up-to-date knowledge into the trial design. However, changes to endpoints can also compromise the scientific integrity of a trial. Here I discuss some of the issues and decision-making processes that should be considered when evaluating whether to make changes to endpoints, and discuss the documentation and reporting of clinical trials that have revised endpoints.","PeriodicalId":87416,"journal":{"name":"PLoS clinical trials","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2007-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1371/journal.pctr.0020018","citationCount":"66","resultStr":"{\"title\":\"When and how can endpoints be changed after initiation of a randomized clinical trial?\",\"authors\":\"Scott Evans\",\"doi\":\"10.1371/journal.pctr.0020018\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Endpoints are outcome measures used to address the objectives of a clinical trial. The primary endpoint is the most important outcome and is used to assess the primary objective of a trial (e.g., the variable used to compare the effect difference of two treatment groups). A fundamental principle in the design of randomized trials involves setting out in advance the endpoints that will be assessed in the trial [1], as failure to prespecify endpoints can introduce bias into a trial and creates opportunities for manipulation. However, sometimes new information may come to light that could merit changes to endpoints during the course of a trial. This new information might include, for example, results from other trials or identification of better biomarkers or surrogate outcome measures. Such changes can allow incorporation of up-to-date knowledge into the trial design. However, changes to endpoints can also compromise the scientific integrity of a trial. Here I discuss some of the issues and decision-making processes that should be considered when evaluating whether to make changes to endpoints, and discuss the documentation and reporting of clinical trials that have revised endpoints.\",\"PeriodicalId\":87416,\"journal\":{\"name\":\"PLoS clinical trials\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2007-04-13\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://sci-hub-pdf.com/10.1371/journal.pctr.0020018\",\"citationCount\":\"66\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"PLoS clinical trials\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1371/journal.pctr.0020018\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"PLoS clinical trials","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1371/journal.pctr.0020018","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
When and how can endpoints be changed after initiation of a randomized clinical trial?
Endpoints are outcome measures used to address the objectives of a clinical trial. The primary endpoint is the most important outcome and is used to assess the primary objective of a trial (e.g., the variable used to compare the effect difference of two treatment groups). A fundamental principle in the design of randomized trials involves setting out in advance the endpoints that will be assessed in the trial [1], as failure to prespecify endpoints can introduce bias into a trial and creates opportunities for manipulation. However, sometimes new information may come to light that could merit changes to endpoints during the course of a trial. This new information might include, for example, results from other trials or identification of better biomarkers or surrogate outcome measures. Such changes can allow incorporation of up-to-date knowledge into the trial design. However, changes to endpoints can also compromise the scientific integrity of a trial. Here I discuss some of the issues and decision-making processes that should be considered when evaluating whether to make changes to endpoints, and discuss the documentation and reporting of clinical trials that have revised endpoints.