Global Regulatory Science and Policy - North America, Sanofi, 4520 East West Highway, Suite 210, Bethesda, MD, 20814, USA.
Ther Innov Regul Sci. 2020 May;54(3):709-716. doi: 10.1007/s43441-019-00106-1. Epub 2020 Jan 6.
"Patient experience data" (PED) refers to the systematic collection of meaningful data relating to the experiences, perspectives, needs, and priorities of patients. PED can augment traditional clinical trial data in the FDA's review of product applications. Section 3001 of the 2016 21st Century Cures Act requires the FDA to make a public statement about the PED considered in the approval of a drug application. Here, we present one of the first assessments of PED consideration during drug application approval, as reported by the FDA under Sec. 3001 of the Cures Act.
FDA reported use of PED in the Review Documentation of the 59 new molecular entities (NMEs) approved in 2018 were collected, indexed, and cross-referenced against information regarding FDA review and product regulatory designation. The data reported in the PED tables were quantitatively described and visualized.
Of the 59 approved NMEs in 2018, 48 include a table that summarized whether PED was or was not used during the FDA drug review. Thirty-four of those 48 approvals (70.8%) reported using PED in the drug review. Patient-reported outcomes(PROs) represented the most significant source of PED and were used in 60.4% of approved drug reviews. Additional findings, including PED use by FDA review division and by FDA regulatory designation, are described.
This assessment is a first step to better understanding how FDA considers PED in regulatory decision making. This analysis should help develop a baseline regarding FDA use of PED and may inform decisions to ensure patients' experiences are adequately heard in future drug development.
“患者体验数据”（PED）是指与患者的体验、观点、需求和优先事项相关的有意义数据的系统收集。PED 可以补充 FDA 审查产品申请时的传统临床试验数据。2016 年 21 世纪治愈法案第 3001 节要求 FDA 就药物申请批准中考虑的 PED 发表公开声明。在这里，我们根据 Cures 法案第 3001 节，展示了 FDA 在药物申请批准过程中首次评估 PED 考虑因素之一。
收集了 2018 年批准的 59 种新分子实体（NME）的 FDA 审查文件中报告的 PED 使用情况，对其进行索引，并与 FDA 审查和产品监管指定的信息进行交叉引用。报告的 PED 表中的数据进行了定量描述和可视化。
在 2018 年批准的 59 种 NME 中，有 48 种包含一个总结表，说明在 FDA 药物审查期间是否使用了 PED。在这 48 个批准中有 34 个（70.8%）报告在药物审查中使用了 PED。患者报告的结果（PROs）是 PED 的最重要来源，在 60.4%的批准药物审查中使用。还描述了其他发现，包括 FDA 审查部门和 FDA 监管指定使用 PED 的情况。
这项评估是更好地了解 FDA 在监管决策中如何考虑 PED 的第一步。该分析应有助于了解 FDA 使用 PED 的基线情况，并可能为确保未来药物开发中充分听取患者意见的决策提供信息。
