{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,4,9]],"date-time":"2025-04-09T05:55:06Z","timestamp":1744178106534,"version":"3.37.3"},"reference-count":38,"publisher":"Oxford University Press (OUP)","issue":"12","license":[{"start":{"date-parts":[[2024,6,24]],"date-time":"2024-06-24T00:00:00Z","timestamp":1719187200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/open_access\/funder_policies\/chorus\/standard_publication_model"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["P30 DK 089507","U01HL114589","U01HL114623","UL1 TR002319"],"award-info":[{"award-number":["P30 DK 089507","U01HL114589","U01HL114623","UL1 TR002319"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2024,12,2]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Development of new therapeutics for a rare disease such as cystic fibrosis is hindered by challenges in accruing enough patients for clinical trials. Use of external controls from well-matched historical trials can reduce prospective trial sizes, and this approach has supported regulatory approval of new interventions for other rare diseases. Here we consider 3 statistical methods that incorporate external controls into a hypothetical clinical trial of a new treatment to reduce pulmonary exacerbations in cystic fibrosis patients: (1) inverse probability weighting, (2) bayesian modeling with propensity-score\u2013based power priors, and (3) hierarchical bayesian modeling with commensurate priors. We compare the methods via simulation study and in a real clinical-trial data setting. Simulations showed that bias in the treatment effect was less than\u20094% using any of the methods, with type I error (or in the bayesian cases, posterior probability of the null hypothesis) usually less than\u20095%. Inverse probability weighting was sensitive to similarity in prevalence of the covariates between historical and prospective trial populations. The commensurate prior method performed best with real clinical trial data. Using external controls to reduce trial size in future clinical trials holds promise and can advance the therapeutic pipeline for rare diseases.<\/jats:p>\n               <jats:p>This article is part of a Special Collection on Pharmacoepidemiology.<\/jats:p>","DOI":"10.1093\/aje\/kwae148","type":"journal-article","created":{"date-parts":[[2024,6,26]],"date-time":"2024-06-26T00:29:16Z","timestamp":1719361756000},"page":"1796-1804","source":"Crossref","is-referenced-by-count":1,"title":["Statistical approaches for the integration of external controls in a cystic fibrosis clinical trial: a simulation and an application"],"prefix":"10.1093","volume":"193","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1630-6018","authenticated-orcid":false,"given":"Mark N","family":"Warden","sequence":"first","affiliation":[{"name":"Seattle Children\u2019s Hospital Cystic Fibrosis Therapeutics Development Network Coordinating Center, , Seattle, WA 98145,","place":["United States"]},{"name":"University of Washington Department of Epidemiology, School of Public Health, , Seattle, WA 98195,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2696-2382","authenticated-orcid":false,"given":"Sonya L","family":"Heltshe","sequence":"additional","affiliation":[{"name":"Seattle Children\u2019s Hospital Cystic Fibrosis Therapeutics Development Network Coordinating Center, , Seattle, WA 98145,","place":["United States"]},{"name":"University of Washington Department of Pediatrics, School of Medicine, , Seattle, WA 98195,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8985-2474","authenticated-orcid":false,"given":"Noah","family":"Simon","sequence":"additional","affiliation":[{"name":"Seattle Children\u2019s Hospital Cystic Fibrosis Therapeutics Development Network Coordinating Center, , Seattle, WA 98145,","place":["United States"]},{"name":"University of Washington Department of Biostatistics, School of Public Health, , Seattle, WA 98195,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stephen J","family":"Mooney","sequence":"additional","affiliation":[{"name":"University of Washington Department of Epidemiology, School of Public Health, , Seattle, WA 98195,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5538-9197","authenticated-orcid":false,"given":"Nicole","family":"Mayer-Hamblett","sequence":"additional","affiliation":[{"name":"Seattle Children\u2019s Hospital Cystic Fibrosis Therapeutics Development Network Coordinating Center, , Seattle, WA 98145,","place":["United States"]},{"name":"University of Washington Department of Pediatrics, School of Medicine, , Seattle, WA 98195,","place":["United States"]},{"name":"University of Washington Department of Biostatistics, School of Public Health, , Seattle, WA 98195,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1221-3746","authenticated-orcid":false,"given":"Amalia S","family":"Magaret","sequence":"additional","affiliation":[{"name":"Seattle Children\u2019s Hospital Cystic Fibrosis Therapeutics Development Network Coordinating Center, , Seattle, WA 98145,","place":["United States"]},{"name":"University of Washington Department of Epidemiology, School of Public Health, , Seattle, WA 98195,","place":["United States"]},{"name":"University of Washington Department of Pediatrics, School of Medicine, , Seattle, WA 98195,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"286","published-online":{"date-parts":[[2024,6,24]]},"reference":[{"issue":"1","key":"2024121223375524000_ref1","doi-asserted-by":"publisher","first-page":"21","DOI":"10.1111\/cts.12500","article-title":"Future of rare diseases research 2017-2027: an IRDiRC 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