在观察性研究中使用质量管理系统和基于风险的方法,以获得强大的现实世界的证据
Reo Tanoshima1,2, Naoko Inagaki3,4, Manabu Nitta3,5
1YCU Center for Novel and Exploratory Clinical Trials, Yokohama City University Hospital, 1-1- 1 Fukuura, Yokohama, Kanagawa, Japan. reotano@yokohama-cu.ac.jp.
Therapeutic innovation & regulatory science
|September 3, 2024
概括
在使用真实世界数据 (RWD) 的观察性研究中,实施基于风险的方法是可行的. 这种质量管理系统确保了药物开发和政策决策的强有力的现实世界证据 (RWE).
科学领域:
- 药监和药物开发 药物监督和药物开发
- 卫生信息学和数据科学
背景情况:
- 从观察性研究中获得的真实世界证据 (RWE) 越来越多地用于药物开发和政策制定.
- 有效的质量管理系统对于产生可靠的RWE至关重要.
- 在干预研究中常见的基于风险的方法可以提高RWE质量.
研究的目的:
- 评估基于风险的质量管理系统实施的可行性和有效性,在使用真实数据的观察性研究中.
- 系统地评估和减轻与数据库选择和研究规划相关的风险,以产生RWE.
主要方法:
- 一个多学科的团队应用了基于风险的七步方法来管理质量.
- 为数据库选择和研究规划进行了定量风险评估.
- 评分了候选数据库,并选择了日本指定难治疾病的国家数据库.
- 研究计划和结果根据优先考虑的风险进行了修订.
主要成果:
- 基于风险的方法在一项针对指定难治性疾病的观察性研究中成功实施.
- 综合风险评估确定并优先考虑了数据库选择和研究规划中的潜在问题.
- 选择的数据库和修订的研究计划证明了基于风险的方法的可行性.
结论:
- 实施基于风险的方法是一种可行且必不可少的策略,用于从观察性研究中产生强有力的真实世界的证据.
- 对数据库质量和研究规划进行系统评估对于监管决策至关重要.
- 这种方法提高了现实世界的数据和证据的可靠性和有效性.
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