Patient-reported outcomes in clinical trials leading to breast cancer drug approvals: are we making them count?

Patient-reported outcomes (PROs) inform benefit-risk tradeoffs in breast cancer treatment, yet reporting quality across registration trials remains incompletely characterized.

We conducted a scoping review of PRO reporting in randomized trials supporting FDA breast cancer drug approvals (January 2011-December 2024), identified via the FDA Oncology Approval Notifications database. Associated manuscripts were retrieved through systematic PubMed and EBSCO searches using trial-specific NCT identifiers. We assessed PRO endpoint prespecification status, instruments used, PRO publication delay (defined as the interval in months between the primary trial manuscript and corresponding PRO-dedicated manuscript publication date), and missing data reporting across eligible primary trial, PRO-dedicated, and subpopulation-specific PRO manuscripts.

We identified 34 FDA breast cancer drug approvals supported by 36 unique randomized trials, yielding 45 eligible manuscripts. No trial designated PROs as a primary or co-primary endpoint; nine (25%) did not explicitly prespecify them. Median PRO publication delay was 18 months, with eight trials experiencing a delay of two or more years. Missing data handling methods were described in 33% of manuscripts and reasons for missingness in 11%.

In this scoping review, PROs were commonly collected but inconsistently prespecified, often delayed, and incompletely reported. Registration trials should strengthen PRO prespecification, reduce publication delay, and adhere to missing data reporting standards.

Not applicable.