{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,1]],"date-time":"2026-07-01T04:22:54Z","timestamp":1782879774654,"version":"3.54.5"},"reference-count":21,"publisher":"Wiley","issue":"22","license":[{"start":{"date-parts":[[2022,7,7]],"date-time":"2022-07-07T00:00:00Z","timestamp":1657152000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/501100000024","name":"Canadian Institutes of Health Research","doi-asserted-by":"publisher","award":["FRN 159834"],"award-info":[{"award-number":["FRN 159834"]}],"id":[{"id":"10.13039\/501100000024","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000038","name":"Natural Sciences and Engineering Research Council of Canada","doi-asserted-by":"publisher","award":["RGPIN 1280961"],"award-info":[{"award-number":["RGPIN 1280961"]}],"id":[{"id":"10.13039\/501100000038","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000038","name":"Natural Sciences and Engineering Research Council of Canada","doi-asserted-by":"publisher","award":["RGPIN 2017\u201000072"],"award-info":[{"award-number":["RGPIN 2017\u201000072"]}],"id":[{"id":"10.13039\/501100000038","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000038","name":"Natural Sciences and Engineering Research Council of Canada","doi-asserted-by":"publisher","award":["RGPIN 2017\u201004207"],"award-info":[{"award-number":["RGPIN 2017\u201004207"]}],"id":[{"id":"10.13039\/501100000038","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Statistics in Medicine"],"published-print":{"date-parts":[[2022,9,30]]},"abstract":"<jats:p>Large cohort studies now routinely involve biobanks in which biospecimens are stored for use in future biomarker studies. In such settings, two\u2010phase response\u2010dependent sampling designs involve subsampling individuals in the cohort, assaying their biospecimen to measure an expensive biomarker, and using this data to estimate key parameters of interest under budgetary constraints. When analyses are based on inverse probability weighted estimating functions, recent work has described adaptive two\u2010phase designs in which a preliminary phase of subsampling based on a standard design facilitates approximation of an optimal selection model for a second subsampling phase. In this article, we refine the definition of an optimal subsampling scheme within the framework of adaptive two\u2010phase designs, describe how adaptive two\u2010phase designs can be used when analyses are based on likelihood or conditional likelihood, and consider the setting of a continuous biomarker where the nuisance covariate distribution is estimated nonparametrically at the design stage and analysis stage as required; efficiency and robustness issues are investigated. We also explore these methods for the surrogate variable problem and describe a generalization to accommodate multiple stages of phase II subsampling. A study involving individuals with psoriatic arthritis is considered for illustration, where the aim is to assess the association between the biomarker MMP\u20103 and the development of joint damage.<\/jats:p>","DOI":"10.1002\/sim.9516","type":"journal-article","created":{"date-parts":[[2022,7,8]],"date-time":"2022-07-08T04:36:05Z","timestamp":1657254965000},"page":"4403-4425","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":13,"title":["Adaptive response\u2010dependent two\u2010phase designs: Some results on robustness and efficiency"],"prefix":"10.1002","volume":"41","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2830-6672","authenticated-orcid":false,"given":"Ce","family":"Yang","sequence":"first","affiliation":[{"name":"Department of Epidemiology Harvard T. H. Chan School of Public Health Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2965-8220","authenticated-orcid":false,"given":"Liqun","family":"Diao","sequence":"additional","affiliation":[{"name":"Department of Statistics and Actuarial Science University of Waterloo Waterloo Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1414-4908","authenticated-orcid":false,"given":"Richard J.","family":"Cook","sequence":"additional","affiliation":[{"name":"Department of Statistics and Actuarial Science University of Waterloo Waterloo Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,7,7]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/1467-9868.00185"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/1467-9876.00165"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1080\/01621459.1995.10476494"},{"key":"e_1_2_9_5_1","volume-title":"Semiparametric Theory and Missing Data","author":"Tsiatis AA","year":"2006"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1093\/biomet\/82.2.299"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/1467-9868.00084"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/(SICI)1097-0258(19970130)16:2<153::AID-SIM477>3.0.CO;2-7"},{"key":"e_1_2_9_9_1","first-page":"1895","article-title":"A mean score method for sensitivity analysis to departures from the missing at random assumption in randomized trials","volume":"28","author":"White IR","year":"2018","journal-title":"Stat Sin"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1198\/016214503388619184"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/cjs.10114"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1093\/biomet\/75.1.11"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.6523"},{"key":"e_1_2_9_14_1","first-page":"1","article-title":"Optimal multiwave sampling for regression modeling in two\u2010phase designs","volume":"1","author":"Chen T","year":"2020","journal-title":"Stat Med"},{"key":"e_1_2_9_15_1","volume-title":"Model Assisted Survey Sampling","author":"S\u00e4rndal CE","year":"2003"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/9781119013563"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1214\/aoms\/1177729952"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1080\/01621459.1995.10476493"},{"key":"e_1_2_9_19_1","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1111\/j.2517-6161.1970.tb00842.x","article-title":"Asymptotic properties of conditional maximum\u2010likelihood estimators","volume":"32","author":"Anderson EB","year":"1970","journal-title":"J Royal Stat Soc Ser B (Methodol)"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/cjs.11207"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1002\/cjs.5550340204"},{"key":"e_1_2_9_22_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.8321"}],"container-title":["Statistics in Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/sim.9516","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/sim.9516","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/sim.9516","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,9,28]],"date-time":"2024-09-28T19:21:49Z","timestamp":1727551309000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/sim.9516"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,7,7]]},"references-count":21,"journal-issue":{"issue":"22","published-print":{"date-parts":[[2022,9,30]]}},"alternative-id":["10.1002\/sim.9516"],"URL":"https:\/\/doi.org\/10.1002\/sim.9516","archive":["Portico"],"relation":{},"ISSN":["0277-6715","1097-0258"],"issn-type":[{"value":"0277-6715","type":"print"},{"value":"1097-0258","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,7,7]]},"assertion":[{"value":"2021-04-14","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-06-21","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-07-07","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}