Reporting of patient-reported outcomes in randomized phase 3 clinical trials of FDA-approved medications in breast cancer.

Abstract

e12693 Background: Patient-reported outcomes (PROs) capture symptoms, functional limitations, and quality of life directly from patients and complement traditional efficacy and safety endpoints in the FDA registration drug trials. PRO data is relevant in breast cancer treatment selection as therapies may require prolonged treatment and offer modest survival gains. Despite the increasing recognition of the importance of PROs, it remains unclear whether PRO data in registration clinical trials of FDA-approved breast cancer medications are prioritized, consistent and reported in a timely manner. Methods: The FDA’s drug database was queried to identify breast cancer drug approvals between 2011 and 2024, and the corresponding clinical trial publications were identified through systematic searches of EBSCO and PubMed. Manuscripts were included if they reported PRO data from FDA registration clinical trials in breast cancer that were associated with drug approval. Extracted variables included PRO endpoint classification, trial characteristics, sample size, time to PRO publication, and reporting of missing data. Descriptive statistics were used to summarize PRO reporting outcomes. Results: Thirty-six randomized breast cancer trials supporting 34 FDA approvals were identified, corresponding to 45 manuscripts reporting PRO data. PROs were included as secondary or exploratory endpoints in most trials; however, one-quarter of trials (9/36, 25%) did not explicitly list any PROs as endpoints. Trials included a median of 3 PRO measures most commonly the EORTC QLQ-C30, FACT-B/FACIT scales. Among 26 PRO-specific manuscripts, the median time from publication of the primary trial results to PRO publication was 18 months, with 6 trials reporting PRO after two years or longer. Missing PRO data were reported in 16 manuscripts (36%), while only 5 (31%) provided reasons for the missing data. Conclusions: In FDA-approved breast cancer trials, patient-reported outcomes are commonly collected; however, they are frequently developed with inconsistent trial standardization, published with substantial delay, and reported without adequate transparency. Future work should focus on standardization to enable meaningful comparison and regulatory relevance. Reporting of patient-reported outcomes in FDA-approved breast cancer trials. Variable Value FDA approvals included (n) 34 Clinical trials included (n) 36 Trials with PROs as secondary endpoint (n, %) 23 (64%) Trials with PROs as exploratory endpoint (n, %) 3 (8%) Trials with PROs not prespecified (n, %) 9 (25%) Time (months) to PRO publication (median, range) 18 (0–44) Manuscripts reporting missing PRO data (n, %) 16 (36%)