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. 2021 Apr 21;5(1):36.
doi: 10.1186/s41687-021-00308-y.

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

Kyungwan Hong  1 , Kayleigh R Majercak  2 , Ester Villalonga-Olives  2 , Eleanor M Perfetto  2   3
Affiliations

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

Kyungwan Hong et al. J Patient Rep Outcomes. .

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.

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Conflict of interest statement

Kyungwan Hong reports this project was supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award U01FD005946 totaling $5,000 with 100% funded by FDA/HHS. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government.

Kayleigh Majercak and Ester Villalonga-Olives have no conflicts of interest to declare.

Eleanor Perfetto is an employee of the National health Council, a nonprofit membership organization that receives both dues and sponsorships from a variety of organizations. Please see the full list of members and sponsors at www.nationalhealthcouncil.org. Eleanor Perfetto has received funding from the Food and Drug Administration, the Patient-Centered Outcomes Research Institute, Excerpta Medical, Pfizer, Merck, and PhRMA.

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