{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T05:24:55Z","timestamp":1789017895525,"version":"build-2803163510"},"reference-count":56,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2022,11,4]],"date-time":"2022-11-04T00:00:00Z","timestamp":1667520000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100010767","name":"Innovative Medicines Initiative","doi-asserted-by":"publisher","award":["115974"],"award-info":[{"award-number":["115974"]}],"id":[{"id":"10.13039\/501100010767","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["ascpt.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Clin Pharma and Therapeutics"],"published-print":{"date-parts":[[2023,1]]},"abstract":"<jats:p>Real\u2010world data\/evidence (RWD\/RWE) may provide insightful information on medicines' clinical effects to guide regulatory decisions. While its contribution has been recognized for safety monitoring and disease epidemiology across medicines' life cycles, using RWD\/RWE to demonstrate efficacy requires further evaluation. This study aimed to (i) characterize RWD\/RWE presented by applicants to support claims on medicines' efficacy within initial marketing authorization applications (MAAs) and extension of indication applications (EoIs), and (ii) analyze the contribution of RWD\/RWE to regulatory decisions on medicines' benefit\u2013risk profile. RWD\/RWE was included to support efficacy in 32 MAAs and 14 EoIs submitted 2018\u20132019. Of these, RWD\/RWE was part of the preauthorization package of 16 MAAs and 10 EoIs, and was (i) considered supporting the regulatory decision in 10 applications (five MAAs, five EoIs), (ii) considered not supporting the regulatory decision in 11 (seven MAAs, four EoIs), and (iii) not addressed at all in the evaluation of 5 applications (four MAAs, one EoI). Common limitations of submitted RWD\/RWE included missing data, lack of representativeness of populations, small sample size, absence of an adequate or prespecified analysis plan, and risk of several types of bias. The suitability of RWD\/RWE in a given application still requires a case\u2010by\u2010case analysis considering its purpose of use, implying reflection on the data source, together with its assets and limitations, study objectives and designs, and the overall data package issued. Early interactions and continuous dialogues with regulators and relevant stakeholders is key to optimize fit\u2010for\u2010purpose RWE generation, enabling its broader use in medicines development.<\/jats:p>","DOI":"10.1002\/cpt.2766","type":"journal-article","created":{"date-parts":[[2022,10,18]],"date-time":"2022-10-18T06:44:36Z","timestamp":1666075476000},"page":"135-151","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":84,"title":["Contribution of Real\u2010World Evidence in European Medicines Agency's Regulatory Decision Making"],"prefix":"10.1111","volume":"113","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3359-9588","authenticated-orcid":false,"given":"Elisabeth","family":"Bakker","sequence":"first","affiliation":[{"name":"Department of Clinical Pharmacy and Pharmacology, University Medical Centre Groningen University of Groningen  Groningen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kelly","family":"Plueschke","sequence":"additional","affiliation":[{"name":"Data Analytics and Methods Task Force European Medicines Agency  Amsterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carla J.","family":"Jonker","sequence":"additional","affiliation":[{"name":"Data Analytics and Methods Task Force European Medicines Agency  Amsterdam The Netherlands"},{"name":"Dutch Medicines Evaluation Board (MEB)  Utrecht The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9838-7754","authenticated-orcid":false,"given":"Xavier","family":"Kurz","sequence":"additional","affiliation":[{"name":"Data Analytics and Methods Task Force European Medicines Agency  Amsterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Viktoriia","family":"Starokozhko","sequence":"additional","affiliation":[{"name":"Department of Clinical Pharmacy and Pharmacology, University Medical Centre Groningen University of Groningen  Groningen The Netherlands"},{"name":"Dutch Medicines Evaluation Board (MEB)  Utrecht The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4262-3180","authenticated-orcid":false,"given":"Peter G. 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