{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T04:16:43Z","timestamp":1767241003228,"version":"3.48.0"},"reference-count":17,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"vor","delay-in-days":731,"URL":"http:\/\/www.elsevier.com\/open-access\/userlicense\/1.0\/"}],"funder":[{"DOI":"10.13039\/100000897","name":"Cystic Fibrosis Foundation","doi-asserted-by":"publisher","award":["SIMON23A0"],"award-info":[{"award-number":["SIMON23A0"]}],"id":[{"id":"10.13039\/100000897","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000897","name":"Cystic Fibrosis Foundation","doi-asserted-by":"publisher","award":["MORGAN23A0"],"award-info":[{"award-number":["MORGAN23A0"]}],"id":[{"id":"10.13039\/100000897","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","cysticfibrosisjournal.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Journal of Cystic Fibrosis"],"published-print":{"date-parts":[[2024,1]]},"DOI":"10.1016\/j.jcf.2023.12.010","type":"journal-article","created":{"date-parts":[[2023,12,27]],"date-time":"2023-12-27T01:42:02Z","timestamp":1703641322000},"page":"65-67","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["Omitting placebos from early-stage clinical trials of cystic fibrosis therapies"],"prefix":"10.1016","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8411-0607","authenticated-orcid":false,"given":"Richard H.","family":"Simon","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-9175-154X","authenticated-orcid":false,"given":"Lynne M.","family":"Quittell","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6346-8140","authenticated-orcid":false,"given":"Wayne J.","family":"Morgan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.jcf.2023.12.010_bib0001","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1111\/bcpt.12352","article-title":"Inclusion of placebos and blinding for ascending dose first-in-human studies and other underpowered phase 1 studies has not been justified and on balance is not useful","volume":"117","author":"Parasrampuria","year":"2015","journal-title":"Basic Clin Pharmacol Toxicol"},{"key":"10.1016\/j.jcf.2023.12.010_bib0002","doi-asserted-by":"crossref","first-page":"677","DOI":"10.1016\/j.jcf.2020.05.011","article-title":"Building global development strategies for CF therapeutics during a transitional CFTR modulator era","volume":"19","author":"Mayer-Hamblett","year":"2020","journal-title":"J Cyst Fibros"},{"key":"10.1016\/j.jcf.2023.12.010_bib0003","unstructured":"U.S. Department of Health and Human Services Food and Drug Administration, Guidance for Industry. E 10 Choice of Control Group and Related Issues in Clinical Trials. 2001. https:\/\/www.fda.gov\/media\/71349\/download. Accessed on October 18, 2023."},{"key":"10.1016\/j.jcf.2023.12.010_bib0004","unstructured":"U.S. Department of Health and Human Services Food and Drug Administration, Rare Diseases: Common Issues in Drug Development. Guidance for Industry. 2019. https:\/\/www.fda.gov\/media\/119757\/download. Accessed on October 18, 2023."},{"key":"10.1016\/j.jcf.2023.12.010_bib0005","unstructured":"U.S. Department of Health and Human Services Food and Drug Administration, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products. 2019. https:\/\/www.fda.gov\/media\/133660\/download. Accessed on October 18, 2023."},{"key":"10.1016\/j.jcf.2023.12.010_bib0006","unstructured":"U.S. Department of Health and Human Services Food and Drug Administration, Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products. 2023. https:\/\/www.fda.gov\/media\/164960\/download. Accessed on October 18, 2023."},{"key":"10.1016\/j.jcf.2023.12.010_bib0007","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1016\/j.jcf.2021.11.007","article-title":"A new path for CF clinical trials through the use of historical controls","volume":"21","author":"Magaret","year":"2022","journal-title":"J Cyst Fibros"},{"key":"10.1016\/j.jcf.2023.12.010_bib0008","doi-asserted-by":"crossref","first-page":"1019","DOI":"10.1007\/s43441-021-00302-y","article-title":"The use of external controls in FDA regulatory decision making","volume":"55","author":"Jahanshahi","year":"2021","journal-title":"Ther Innov Regul Sci"},{"key":"10.1016\/j.jcf.2023.12.010_bib0009","doi-asserted-by":"crossref","first-page":"3002","DOI":"10.1200\/JCO.2009.27.4282","article-title":"Evaluation of the value of attribution in the interpretation of adverse event data: a north central cancer treatment group and American college of surgeons oncology group investigation","volume":"28","author":"Hillman","year":"2010","journal-title":"J Clin Oncol"},{"key":"10.1016\/j.jcf.2023.12.010_bib0010","doi-asserted-by":"crossref","first-page":"1183","DOI":"10.1093\/annonc\/mdx043","article-title":"Statistical controversies in clinical research: Value of adverse events relatedness to study treatment: analyses of data from randomized double-blind placebo-controlled clinical trials","volume":"28","author":"Le-Rademacher","year":"2017","journal-title":"Ann Oncol"},{"key":"10.1016\/j.jcf.2023.12.010_bib0011","doi-asserted-by":"crossref","first-page":"669","DOI":"10.1016\/j.jcf.2023.05.014","article-title":"Potential implicit bias in attribution of adverse events in randomized controlled trials in cystic fibrosis","volume":"22","author":"Somayaji","year":"2023","journal-title":"J Cyst Fibros"},{"key":"10.1016\/j.jcf.2023.12.010_bib0012","doi-asserted-by":"crossref","first-page":"1068","DOI":"10.1016\/j.molmed.2020.08.002","article-title":"Accelerated approval or risk reduction? How response biomarkers advance therapeutics through clinical trials in cystic fibrosis","volume":"26","author":"Mayer-Hamblett","year":"2020","journal-title":"Trends Mol Med"},{"key":"10.1016\/j.jcf.2023.12.010_bib0013","doi-asserted-by":"crossref","first-page":"1612","DOI":"10.1056\/NEJMoa1807120","article-title":"Vx-445-tezacaftor-ivacaftor in patients with cystic fibrosis and one or two phe508del alleles","volume":"379","author":"Keating","year":"2018","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jcf.2023.12.010_bib0014","doi-asserted-by":"crossref","first-page":"932","DOI":"10.1016\/S2213-2600(23)00297-7","article-title":"Advancing the pipeline of cystic fibrosis clinical trials: a new roadmap with a global trial network perspective","volume":"11","author":"Mayer-Hamblett","year":"2023","journal-title":"Lancet Respir Med"},{"key":"10.1016\/j.jcf.2023.12.010_bib0015","unstructured":"U.S. Department of Health and Human Services Food and Drug Administration, Long term follow-up after administration of human gene therapy products. https:\/\/www.fda.gov\/media\/113768\/download. Accessed on October 18, 2023."},{"key":"10.1016\/j.jcf.2023.12.010_bib0016","doi-asserted-by":"crossref","first-page":"985","DOI":"10.1016\/S0002-8703(03)00507-6","article-title":"Hypertensive patients' willingness to participate in placebo-controlled trials: implications for recruitment efficiency","volume":"146","author":"Halpern","year":"2003","journal-title":"Am Heart J"},{"key":"10.1016\/j.jcf.2023.12.010_bib0017","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1177\/1740774510392257","article-title":"Personal benefit, or benefiting others? Deciding whether to take part in clinical trials","volume":"8","author":"Locock","year":"2011","journal-title":"Clin Trials"}],"container-title":["Journal of Cystic Fibrosis"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1569199323017344?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1569199323017344?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T04:12:30Z","timestamp":1767240750000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1569199323017344"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,1]]},"references-count":17,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2024,1]]}},"alternative-id":["S1569199323017344"],"URL":"https:\/\/doi.org\/10.1016\/j.jcf.2023.12.010","relation":{},"ISSN":["1569-1993"],"issn-type":[{"type":"print","value":"1569-1993"}],"subject":[],"published":{"date-parts":[[2024,1]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Omitting placebos from early-stage clinical trials of cystic fibrosis therapies","name":"articletitle","label":"Article Title"},{"value":"Journal of Cystic Fibrosis","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jcf.2023.12.010","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"simple-article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 European Cystic Fibrosis Society. Published by Elsevier B.V.","name":"copyright","label":"Copyright"}]}}