RAND, Arlington, VA 22202, USA.
RAND, Santa Monica, CA 90401, USA.
J Comp Eff Res. 2024 Sep;13(9):e240078. doi: 10.57264/cer-2024-0078. Epub 2024 Aug 16.
Although the US FDA encourages manufacturers of medical devices to submit real-world evidence (RWE) to support regulatory decisions, the ability of real-world data (RWD) to generate evidence suitable for decision making remains unclear. The 2017 Medical Device User Fee Amendments (MDUFA IV), authorized the National Evaluation System for health Technology Coordinating Center (NESTcc) to conduct pilot projects, or 'Test-Cases', to assess whether current RWD captures the information needed to answer research questions proposed by industry stakeholders. We synthesized key lessons about the challenges conducting research with RWD and the strategies used by research teams to enhance their ability to generate evidence from RWD based on 18 Test-Cases conducted between 2020 and 2022. We reviewed study protocols and reports from each Test-Case team and conducted 49 semi-structured interviews with representatives of participating organizations. Interview transcripts were coded and thematically analyzed. Challenges that stakeholders encountered in working with RWD included the lack of unique device identifiers, capturing key data elements and their appropriate meaning in structured data, limited reliability of diagnosis and procedure codes in structured data, extracting information from unstructured electronic health record (EHR) data, limited capture of long-term study end points, missing data and data sharing. Successful strategies included using manufacturer and supply chain data, leveraging clinical registries and registry reporting processes to collect and aggregate data, querying standardized EHR data, implementing natural language processing algorithms and using multidisciplinary research teams. The Test-Cases identified numerous challenges working with RWD but also opportunities to address these challenges and improve researchers' ability to use RWD to generate evidence on medical devices.
尽管美国食品和药物管理局（FDA）鼓励医疗器械制造商提交真实世界证据（RWE）以支持监管决策，但真实世界数据（RWD）生成适合决策的证据的能力仍不清楚。2017 年医疗器械用户收费修正案（MDUFA IV）授权国家卫生技术评估协调中心（NESTcc）开展试点项目，即“测试案例”，以评估当前 RWD 是否捕获了行业利益相关者提出的研究问题所需的信息。我们综合了 18 个在 2020 年至 2022 年期间进行的测试案例中有关使用 RWD 进行研究的挑战以及研究团队为提高从 RWD 生成证据的能力而采用的策略的关键经验。我们审查了每个测试案例团队的研究方案和报告，并对参与组织的代表进行了 49 次半结构化访谈。对访谈记录进行了编码和主题分析。利益相关者在使用 RWD 时遇到的挑战包括缺乏独特的设备标识符、捕获结构化数据中的关键数据元素及其适当含义、结构化数据中诊断和程序代码的可靠性有限、从非结构化电子健康记录（EHR）数据中提取信息、长期研究终点的捕获有限、数据缺失和数据共享。成功的策略包括使用制造商和供应链数据、利用临床登记处和登记处报告流程来收集和汇总数据、查询标准化的 EHR 数据、实施自然语言处理算法以及使用多学科研究团队。测试案例确定了在使用 RWD 时遇到的许多挑战，但也为解决这些挑战和提高研究人员使用 RWD 生成医疗器械证据的能力提供了机会。
