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dc.contributor.authorHong, Kyungwan
dc.contributor.authorMajercak, Kayleigh R
dc.contributor.authorVillalonga-Olives, Ester
dc.contributor.authorPerfetto, Eleanor M
dc.date.accessioned2021-05-03T16:51:44Z
dc.date.available2021-05-03T16:51:44Z
dc.date.issued2021-04-21
dc.identifier.urihttp://hdl.handle.net/10713/15554
dc.description.abstractBackground: Patient-reported outcomes (PROs) can provide valuable information about drug benefit-risk tradeoffs from the patient perspective and are particularly important to patients with breast cancer due to its symptoms and adverse events from breast cancer treatments. The United States Food and Drug Administration (U.S. FDA) has acknowledged PROs as important approval endpoints used in clinical trials of cancer drugs. However, previous studies found that PROs are rarely mentioned in cancer drug labels, a widely used and trusted source of information about drugs. Our objectives were to compare PRO data reported in FDA labeling versus FDA medical review documents for breast cancer drugs approved in the U.S. between 2000 and 2019 to identify possible causes for PRO-data labeling exclusions. Methods: We included new molecular entities (NMEs) and biologic license applications (BLAs) initially approved for breast cancer treatment by the FDA between 1/1/2000 and 12/31/2019. Product labeling and FDA medical review documents were collected from the FDA-Approved Drugs database (Drugs@FDA). From these resources, details on PRO measures used in trials, design of trials using PRO measures, PRO-endpoint status, analytical methods, and FDA reviewer comments regarding PRO measurement were extracted. Results: Of 633 FDA-approved drugs, 13 were indicated for breast cancer treatment; none of their prescribing information contained information about PROs. However, 11 of 13 (85%) included PRO measures and endpoint information in FDA medical review documents. PRO measures were used in 14 different clinical trials, and FDA reviewers’ comments regarding PRO measurement were related to lack of meaningfulness and clinical significance, lack of content validity, and inadequate analytical methods. Conclusions: Despite the importance of PROs to patients with breast cancer, PRO measures were only described in FDA medical review documents of breast cancer drugs, but not in drug product labeling. Therefore, it appears that PRO data are often collected in breast cancer trials, but have not been methodologically acceptable to FDA reviewers. Collaborative efforts between the FDA and industry are warranted to increase the number of breast cancer drug applications with appropriate use of PRO measures and endpoints. © 2021, The Author(s).en_US
dc.description.urihttps://doi.org/10.1186/s41687-021-00308-yen_US
dc.language.isoenen_US
dc.publisherSpringer Natureen_US
dc.relation.ispartofJournal of Patient-Reported Outcomesen_US
dc.subjectFDA drug labelsen_US
dc.subject.lcshBreast--Canceren_US
dc.subject.meshBreast Neoplasms--therapyen_US
dc.subject.meshPatient Reported Outcome Measuresen_US
dc.titlePatient-reported outcomes in breast cancer FDA drug labels and review documents.en_US
dc.typeArticleen_US
dc.identifier.doi10.1186/s41687-021-00308-y
dc.identifier.pmid33881661
dc.source.journaltitleJournal of patient-reported outcomes
dc.source.volume5
dc.source.issue1
dc.source.beginpage36
dc.source.endpage
dc.source.countryGermany


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