000282544 001__ 282544
000282544 005__ 20251201103322.0
000282544 0247_ $$2doi$$a10.1002/cpt.3684
000282544 0247_ $$2ISSN$$a0009-9236
000282544 0247_ $$2ISSN$$a1085-8733
000282544 0247_ $$2ISSN$$a1532-6535
000282544 037__ $$aDZNE-2025-01307
000282544 041__ $$aEnglish
000282544 082__ $$a610
000282544 1001_ $$0P:(DE-2719)9002837$$aRussek, Martin$$b0$$udzne
000282544 245__ $$aSupplementing Single‐Arm Trials with External Control Arms—Evaluation of German Real‐World Data
000282544 260__ $$aHoboken, NJ$$bWiley-Blackwell$$c2025
000282544 3367_ $$2DRIVER$$aarticle
000282544 3367_ $$2DataCite$$aOutput Types/Journal article
000282544 3367_ $$0PUB:(DE-HGF)16$$2PUB:(DE-HGF)$$aJournal Article$$bjournal$$mjournal$$s1764581485_18435
000282544 3367_ $$2BibTeX$$aARTICLE
000282544 3367_ $$2ORCID$$aJOURNAL_ARTICLE
000282544 3367_ $$00$$2EndNote$$aJournal Article
000282544 520__ $$aAs single-arm trials (SATs) are increasingly used in pharmaceutical research, the validity of such study designs needs to be critically assessed. We characterize the feasibility of supplementing SATs with real-world data (RWD)-derived external control arms by determining the proportion of SATs on breast cancer and amyotrophic lateral sclerosis (ALS) for which an external control arm based on RWD can be constructed. The main outcome measure is the number and percentage of trials for which all important eligibility criteria and at least one primary endpoint could be identified in one of five German RWD sources. We surveyed all SATs concerning breast cancer or ALS treatment registered in the European Union’s clinical trial registers between 2004 and 2023. Ten out of 379 breast cancer SATs and 2 of 11 ALS SATs could feasibly be supplemented with RWD-derived external control arms, if all important eligibility criteria and a primary endpoint have to be identifiable in the RWD source. Ninety-three breast cancer trials had at least one outcome ascertainable in a RWD source, and 35 trials had all important eligibility criteria recorded in a RWD source. Nine ALS trials had at least one primary endpoint ascertainable in RWD sources, and 2 had all important eligibility criteria recorded in a RWD source. Our study shows that SATs with RWD-derived external control arms will rarely be suitable to establish treatment effects of medicines in the current setting for the investigated phenotypes and that SATs should be designed with limitations of the source of external controls in mind.
000282544 536__ $$0G:(DE-HGF)POF4-354$$a354 - Disease Prevention and Healthy Aging (POF4-354)$$cPOF4-354$$fPOF IV$$x0
000282544 588__ $$aDataset connected to CrossRef, Journals: pub.dzne.de
000282544 7001_ $$0P:(DE-2719)9002192$$aPeltner, Jonas$$b1
000282544 7001_ $$0P:(DE-2719)2810511$$aHaenisch, Britta$$b2$$eLast author
000282544 773__ $$0PERI:(DE-600)2040184-X$$a10.1002/cpt.3684$$gVol. 118, no. 6, p. 1443 - 1450$$n6$$p1443 - 1450$$tClinical pharmacology & therapeutics$$v118$$x0009-9236$$y2025
000282544 8564_ $$uhttps://pub.dzne.de/record/282544/files/DZNE-2025-01307%20SUP1.docx
000282544 8564_ $$uhttps://pub.dzne.de/record/282544/files/DZNE-2025-01307%20SUP2.xlsx
000282544 8564_ $$uhttps://pub.dzne.de/record/282544/files/DZNE-2025-01307.pdf$$yOpenAccess
000282544 8564_ $$uhttps://pub.dzne.de/record/282544/files/DZNE-2025-01307%20SUP1.doc
000282544 8564_ $$uhttps://pub.dzne.de/record/282544/files/DZNE-2025-01307%20SUP1.odt
000282544 8564_ $$uhttps://pub.dzne.de/record/282544/files/DZNE-2025-01307%20SUP1.pdf
000282544 8564_ $$uhttps://pub.dzne.de/record/282544/files/DZNE-2025-01307%20SUP2.csv
000282544 8564_ $$uhttps://pub.dzne.de/record/282544/files/DZNE-2025-01307%20SUP2.ods
000282544 8564_ $$uhttps://pub.dzne.de/record/282544/files/DZNE-2025-01307%20SUP2.xls
000282544 8564_ $$uhttps://pub.dzne.de/record/282544/files/DZNE-2025-01307.pdf?subformat=pdfa$$xpdfa$$yOpenAccess
000282544 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)9002837$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b0$$kDZNE
000282544 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)9002192$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b1$$kDZNE
000282544 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)2810511$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b2$$kDZNE
000282544 9131_ $$0G:(DE-HGF)POF4-354$$1G:(DE-HGF)POF4-350$$2G:(DE-HGF)POF4-300$$3G:(DE-HGF)POF4$$4G:(DE-HGF)POF$$aDE-HGF$$bGesundheit$$lNeurodegenerative Diseases$$vDisease Prevention and Healthy Aging$$x0
000282544 915__ $$0StatID:(DE-HGF)0200$$2StatID$$aDBCoverage$$bSCOPUS$$d2024-12-27
000282544 915__ $$0StatID:(DE-HGF)0300$$2StatID$$aDBCoverage$$bMedline$$d2024-12-27
000282544 915__ $$0StatID:(DE-HGF)1050$$2StatID$$aDBCoverage$$bBIOSIS Previews$$d2024-12-27
000282544 915__ $$0StatID:(DE-HGF)1190$$2StatID$$aDBCoverage$$bBiological Abstracts$$d2024-12-27
000282544 915__ $$0LIC:(DE-HGF)CCBY4$$2HGFVOC$$aCreative Commons Attribution CC BY 4.0
000282544 915__ $$0StatID:(DE-HGF)0100$$2StatID$$aJCR$$bCLIN PHARMACOL THER : 2022$$d2024-12-27
000282544 915__ $$0StatID:(DE-HGF)3001$$2StatID$$aDEAL Wiley$$d2024-12-27$$wger
000282544 915__ $$0StatID:(DE-HGF)1030$$2StatID$$aDBCoverage$$bCurrent Contents - Life Sciences$$d2024-12-27
000282544 915__ $$0StatID:(DE-HGF)0113$$2StatID$$aWoS$$bScience Citation Index Expanded$$d2024-12-27
000282544 915__ $$0StatID:(DE-HGF)0150$$2StatID$$aDBCoverage$$bWeb of Science Core Collection$$d2024-12-27
000282544 915__ $$0StatID:(DE-HGF)0510$$2StatID$$aOpenAccess
000282544 915__ $$0StatID:(DE-HGF)9905$$2StatID$$aIF >= 5$$bCLIN PHARMACOL THER : 2022$$d2024-12-27
000282544 915__ $$0StatID:(DE-HGF)0160$$2StatID$$aDBCoverage$$bEssential Science Indicators$$d2024-12-27
000282544 915__ $$0StatID:(DE-HGF)1110$$2StatID$$aDBCoverage$$bCurrent Contents - Clinical Medicine$$d2024-12-27
000282544 915__ $$0StatID:(DE-HGF)0199$$2StatID$$aDBCoverage$$bClarivate Analytics Master Journal List$$d2024-12-27
000282544 9201_ $$0I:(DE-2719)1013010$$kAG Hänisch$$lPharmacoepidemiology$$x0
000282544 980__ $$ajournal
000282544 980__ $$aVDB
000282544 980__ $$aUNRESTRICTED
000282544 980__ $$aI:(DE-2719)1013010
000282544 9801_ $$aFullTexts