目的:推荐预防和处理以患者为中心的主要结局研究(PCOR)缺失数据的方法学标准。
方法:我们在2012年1月搜索了美国国家医学图书馆的书架和目录以及监管机构和组织网站,以获取有关缺失数据的正式建议的指导文件。我们提取了包含的指导文件和建议的特征。使用两轮修改的Delphi调查,一个多学科小组提出了预防和处理PCOR缺失数据的强制性标准.
结果:我们确定了1,790条记录,并评估了30条有相关建议。我们提出了10项强制性标准,涵盖三个领域。首先,唯一最好的方法是前瞻性地防止缺失数据的发生。第二,在分析缺失数据时,使用正确反映多种不确定性来源的有效统计方法至关重要。第三,对缺失数据的透明和彻底报告使读者能够判断调查结果的有效性。
结论:我们敦促研究人员采用严格的方法,并通过将最佳实践应用于预防和处理缺失数据来促进良好的科学。为观察性研究和使用现有记录的研究制定预防和处理缺失数据的指导是未来研究的优先事项。
OBJECTIVE: To recommend methodological standards in the prevention and handling of missing data for primary patient-centered outcomes research (PCOR).
METHODS: We searched National Library of Medicine Bookshelf and Catalog as well as regulatory agencies\' and organizations\' Web sites in January 2012 for guidance documents that had formal recommendations regarding missing data. We extracted the characteristics of included guidance documents and recommendations. Using a two-round modified Delphi survey, a multidisciplinary panel proposed mandatory standards on the prevention and handling of missing data for PCOR.
RESULTS: We identified 1,790 records and assessed 30 as having relevant recommendations. We proposed 10 standards as mandatory, covering three domains. First, the single best approach is to prospectively prevent missing data occurrence. Second, use of valid statistical methods that properly reflect multiple sources of uncertainty is critical when analyzing missing data. Third, transparent and thorough reporting of missing data allows readers to judge the validity of the findings.
CONCLUSIONS: We urge researchers to adopt rigorous methodology and promote good science by applying best practices to the prevention and handling of missing data. Developing guidance on the prevention and handling of missing data for observational studies and studies that use existing records is a priority for future research.