Department of Pharmacy, Levine Cancer Institute, Atrium Health, 1021 Morehead Medical Drive, Charlotte, NC, 28204, USA.
Department of Pharmacy, Levine Cancer Institute, Atrium Health, 100 Medical Park Drive, Concord, NC, 28025, USA.
Support Care Cancer. 2023 Sep 29;31(10):602. doi: 10.1007/s00520-023-08068-9.
Patient-reported outcomes (PROs) represent an important evaluation of health-related quality of life that has become more commonly incorporated into oncology drug clinical trials. The frequency of PRO inclusion as an endpoint in oncology drug clinical trials leading to the initial accelerated approval of a new therapy is not yet known. We conducted a cross-sectional study evaluating all new drug applications submitted to the FDA over the past 10 years (2013-2022) that led to the initial approval of an oncology drug through the accelerated approval process. The objective was to assess whether the trials leading to such an approval included PROs. Between 2013 and 2022, the FDA approved 59 unique drugs for an oncology indication via the accelerated approval pathway, and 35 (59%) included a PRO assessment in the clinical trial. A median of 1 PRO measurement was used in each trial, with 23 different types of PRO assessment tools were used across the 59 new drug applications. In summary, we found that PRO measurements are inconsistently utilized in trials leading to initial accelerated approval of oncology drugs, and there seems to be a lack of harmonization of different PRO measurement tools used across trials.
患者报告的结局（PROs）是对健康相关生活质量的重要评估，已越来越多地纳入肿瘤药物临床试验中。导致新疗法首次加速批准的肿瘤药物临床试验中纳入 PRO 作为终点的频率尚不清楚。我们进行了一项横断面研究，评估了过去 10 年（2013-2022 年）向 FDA 提交的所有导致肿瘤药物通过加速批准程序首次批准的新药申请。目的是评估导致此类批准的试验是否包括 PRO。2013 年至 2022 年间，FDA 通过加速批准途径批准了 59 种用于肿瘤适应证的独特药物，其中 35 种（59%）在临床试验中包含 PRO 评估。每项试验平均使用 1 项 PRO 测量，59 项新药申请中使用了 23 种不同类型的 PRO 评估工具。总之，我们发现 PRO 测量在导致肿瘤药物首次加速批准的试验中使用不一致，并且似乎缺乏不同试验中使用的不同 PRO 测量工具的协调。
