• Login
    View Item 
    •   UMB Digital Archive
    • UMB Open Access Articles
    • UMB Open Access Articles
    • View Item
    •   UMB Digital Archive
    • UMB Open Access Articles
    • UMB Open Access Articles
    • View Item
    JavaScript is disabled for your browser. Some features of this site may not work without it.

    Browse

    All of UMB Digital ArchiveCommunitiesPublication DateAuthorsTitlesSubjectsThis CollectionPublication DateAuthorsTitlesSubjects

    My Account

    LoginRegister

    Statistics

    Display statistics

    Patient-reported outcomes in breast cancer FDA drug labels and review documents.

    • CSV
    • RefMan
    • EndNote
    • BibTex
    • RefWorks
    Author
    Hong, Kyungwan
    Majercak, Kayleigh R
    Villalonga-Olives, Ester
    Perfetto, Eleanor M
    Date
    2021-04-21
    Journal
    Journal of Patient-Reported Outcomes
    Publisher
    Springer Nature
    Type
    Article
    
    Metadata
    Show full item record
    See at
    https://doi.org/10.1186/s41687-021-00308-y
    Abstract
    Background: 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).
    Keyword
    FDA drug labels
    Breast--Cancer
    Breast Neoplasms--therapy
    Patient Reported Outcome Measures
    Identifier to cite or link to this item
    http://hdl.handle.net/10713/15554
    ae974a485f413a2113503eed53cd6c53
    10.1186/s41687-021-00308-y
    Scopus Count
    Collections
    UMB Open Access Articles

    entitlement

    Related articles

    • Potential of patient-reported outcomes as nonprimary endpoints in clinical trials.
    • Authors: Gnanasakthy A, Lewis S, Clark M, Mordin M, DeMuro C
    • Issue date: 2013 May 15
    • Patient-Reported Outcomes in Orphan Drug Labels Approved by the US Food and Drug Administration.
    • Authors: Hong YD, Villalonga-Olives E, Perfetto EM
    • Issue date: 2019 Aug
    • Patient-Reported Outcomes Labeling for Products Approved by the Office of Hematology and Oncology Products of the US Food and Drug Administration (2010-2014).
    • Authors: Gnanasakthy A, DeMuro C, Clark M, Haydysch E, Ma E, Bonthapally V
    • Issue date: 2016 Jun 1
    • Measuring treatment impact: a review of patient-reported outcomes and other efficacy endpoints in approved product labels.
    • Authors: Willke RJ, Burke LB, Erickson P
    • Issue date: 2004 Dec
    • A Review of Patient-Reported Outcomes Labeling for Oncology Drugs Approved by the FDA and the EMA (2012-2016).
    • Authors: Gnanasakthy A, Barrett A, Evans E, D'Alessio D, Romano CD
    • Issue date: 2019 Feb
    DSpace software (copyright © 2002 - 2022)  DuraSpace
    Quick Guide | Policies | Contact Us | UMB Health Sciences & Human Services Library
    Open Repository is a service operated by 
    Atmire NV
     

    Export search results

    The export option will allow you to export the current search results of the entered query to a file. Different formats are available for download. To export the items, click on the button corresponding with the preferred download format.

    By default, clicking on the export buttons will result in a download of the allowed maximum amount of items.

    To select a subset of the search results, click "Selective Export" button and make a selection of the items you want to export. The amount of items that can be exported at once is similarly restricted as the full export.

    After making a selection, click one of the export format buttons. The amount of items that will be exported is indicated in the bubble next to export format.