Characterizing Patient-Reported Outcome Data Availability for Oral Anticancer Therapies

Abstract

Purpose: Since the first oncology-specific US Food and Drug Administration (FDA) guidance on patient-reported outcomes (PROs) was released in 2016, substantial progress has been made in integrating PROs into clinical trials. Nonetheless, PRO data for many oral anticancer therapies remain unreported at the time of approval, underscoring the need to further streamline data collection and enhance outcome availability.

Methods: We conducted a retrospective, cross-sectional review of oral anticancer therapies approved by the US FDA from January 2018 through August 2023. The primary objective was to quantify the rate of PRO data availability for oral anticancer therapies approved by the FDA during the study period. Secondary objectives aimed at identifying factors that influence PRO reporting. Descriptive data analysis and tests for significance of differences were performed using SPSS version 29.

Results: Seventy-five percent of FDA approvals during the study period collected PROs, with a median of three PROs collected per approval. Of those that collected PROs, 69.7% (175/251) were reported. PROs that were collected as secondary objectives were more likely to be reported than exploratory objective PROs (80.1% [125/156] v 52.6% [50/95], respectively). The PRO survey used had a significant influence on reporting rates, with functional assessment of cancer therapy (FACT) surveys being the most likely to be reported (20/21; 95.2%) v euroQol 5-dimension Questionnaire (EQ-5D) (30/59; 50.8%) and European Organisation for Research and Treatment of Cancer 30 (44/53; 83.0%). When not reported in the initial publication, the median impact factor of the subsequent PRO publication was lower (7.1), compared with that of the initial publication (158.6).

Conclusion: The majority of oral anticancer therapy clinical trials collect PROs; however, there remains an opportunity for increased data availability. Further study is warranted to characterize factors influencing PRO reporting and to provide direction for optimizing timely PRO data availability.