Trends and landscape of clinical outcome assessment use to support oncology product approvals.

Abstract

e23186 Background: The regulatory landscape in oncology trials has evolved with growing emphasis on patient-focused drug development. Further to tumor and survival outcomes, measuring how patients feel and function via clinical outcome assessments (COAs) provides critical information on symptoms and treatment burden and may help to inform risk/benefit evaluation for new treatments. This study aimed to examine trends in the use of COAs to support efficacy or safety in oncology product labeling. Methods: Product labeling information was obtained from PROLABELS (Mapi Research Trust), a database containing COA-related data sourced from the efficacy or safety sections of FDA Product labels and EMA SmPC where at least one COA is mentioned. Oncology product labels were screened based on therapeutic indications and study information. The clinical study details published in the label/SPC between 1990 and 2022 were used for further analysis. Results: A total of 180 EMA and FDA approved drugs and devices for oncology indications where COAs were mentioned in the product label were identified. 288 studies with COAs supported these drug approvals. These studies were in 52 different indications, with most in multiple myeloma (31), followed by non-small-cell lung cancer (24), prostate cancer (22), and breast cancer (22). Of the COAs that were used, 47% were patient-reported outcomes, 18% were clinician-reported outcomes, 30% were composites, and 5% were unspecified. The number of studies conducted with COAs supporting a product label increased over time, with 3x more studies in the past 10 years (189 from 2013-2022) compared to the previous 10 years (60 from 2003-2012). For studies listing specific COA names, 63 unique COAs were used. Excluding response criteria assessments, the most common COAs were the EORTC-QLQ-C30 (57 studies), EORTC-QLQ-LC13 (18 studies), and EQ-5D-3L (17 studies). The two indications with the largest variety of COAs used were prostate cancer (10 unique COAs) and non-small-cell lung cancer (9 unique COAs). Conclusions: In line with regulators highlighting the utility of COAs in evaluating clinical benefit, results show an increase in studies using COAs to support efficacy and safety in oncology product approvals. With COAs providing critical information on symptoms, side effect impact, and quality of life, including this information on labels may help patients and providers make more informed healthcare decisions, and for market differentiation.