Methodological guidance for the use of real-world data to measure exposure and utilization patterns of osteoporosis medications.
Methodological guidance for the use of real-world data to measure exposure and utilization patterns of osteoporosis medications.
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DOI：
10.1016/j.bonr.2023.101730
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发表时间：
2024-03
期刊：
影响因子：
2.5
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中科院分区：
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骨质疏松症药物的观察性研究可以提供关键的真实世界证据（RWE），填补临床试验留下的知识空白。然而，需要仔细考虑研究设计，以产生可靠的关联估计。特别是，由于不同的药物类别、配方和给药途径（每种药物具有不同的药理学），从真实世界数据（RWD）来源获得骨质疏松症药物暴露的有效测量值非常复杂。需要特别注意延长二膦酸盐的半衰期以及延长地舒单抗和唑来膦酸的给药。此外，处方模式和用药行为通常导致治疗、转换和合并使用骨质疏松症治疗的差距。在这篇综述中，我们提出了重要的考虑因素，并提供专门的指导，测量骨质疏松症的药物暴露在RWD。首先，我们比较了用于骨质疏松症药物研究的RWD的不同来源，并提供了在这些数据来源中识别骨质疏松症药物使用的指导。接下来，我们提供了骨质疏松药理学的概述，以及它如何影响RWD中暴露测量的决策。最后，我们提出了使用RWD测量骨质疏松症药物暴露、依从性、转换、长期暴露和休药期的考虑因素。最终，全面了解RWD来源的差异和骨质疏松症药物的药理学对于获得骨质疏松症药物与结局（如骨折）之间关系的有效估计至关重要，而且对于改善已发表研究的关键评价也至关重要。真实世界数据（RWD）的使用对于骨质疏松症研究至关重要。在RWD中测量骨质疏松症药物暴露是复杂的。研究人员必须考虑不同的药物类别，配方和药理学。特定于数据源的注意事项（例如，缺失数据）也应该考虑。正确的暴露测量是在RWD中获得可靠估计的关键。
Observational studies of osteoporosis medications can provide critical real-world evidence (RWE) that fills knowledge gaps left by clinical trials. However, careful consideration of study design is needed to yield reliable estimates of association. In particular, obtaining valid measurements of exposure to osteoporosis medications from real-world data (RWD) sources is complicated due to different medication classes, formulations, and routes of administration, each with different pharmacology. Extended half-lives of bisphosphonates and extended dosing of denosumab and zoledronic acid require particular attention. In addition, prescribing patterns and medication taking behavior often result in gaps in therapy, switching, and concomitant use of osteoporosis therapies. In this review, we present important considerations and provide specialized guidance for measuring osteoporosis drug exposures in RWD. First, we compare different sources of RWD used for osteoporosis drug studies and provide guidance on identifying osteoporosis medication use in these data sources. Next, we provide an overview of osteoporosis pharmacology and how it can influence decisions on exposure measurement within RWD. Finally, we present considerations for the measurement of osteoporosis medication exposure, adherence, switching, long-term exposures, and drug holidays using RWD. Ultimately, a thorough understanding of the differences in RWD sources and the pharmacology of osteoporosis medications is essential to obtain valid estimates of the relationship between osteoporosis medications and outcomes, such as fractures, but also to improve the critical appraisal of published studies. The use of real-world data (RWD) is critical for osteoporosis research. Measurement of osteoporosis medication exposures in RWD is complex. Researchers must consider different drug classes, formulations, and pharmacology. Data source-specific caveats (e.g., missing data) should also be considered. Proper exposure measurement is key to obtaining reliable estimates in RWD.