结合外部数据的混合设计评估地塞米松内窥镜给药暂停治疗白内障手术后炎症。

IF 5.5 2区 医学 Q1 PHARMACOLOGY & PHARMACY
Yuanyuan Zhao, Keer Chen, Jun Zhou, Jun Zhao, Dingheng Zhang, Junyue Wan, Weihong Yuan, Xi Chen, Ming Tan, Fuqiang Cui, Shein-Chung Chow, Ying Wu
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引用次数: 0

摘要

传统的随机对照试验(RCTs)由于招募时间长、成本高而面临越来越大的挑战。监管机构鼓励使用外部数据和真实世界证据(RWE)来提高效率,但由于对异质性和偏见的担忧,在验证性环境中的采用仍然受到限制。我们进行了一项概念验证研究,以评估混合贝叶斯借鉴设计在中国支持地塞米松内窥镜给药混悬液(DEXYCU) III期RCT的可行性和监管价值。采用等效概率倾向得分元分析-预测(EQPSMAP)方法,我们整合了三个数据源-全球RCT,中国区域III期RCT和中国真实世界数据(RWD)。该方法的性能通过主要疗效终点的点估计和95%可信区间进行评估。基于EQPSMAP的混合设计在存在基线不平衡和异构数据时显示出更高的鲁棒性和准确性。与传统的RCT相比,混合设计将所需的样本量减少了41至158例患者,在保持内部有效性的同时将试验时间缩短了约2至5个月。本研究证明了混合贝叶斯设计在后期试验中的可行性和调控价值。该方法为将外部数据整合到区域药物开发和监管决策中提供了一个实用的、偏倚控制的框架。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
A Hybrid Design Incorporating External Data to Evaluate Dexamethasone Intracameral Drug Delivery Suspension for Post-Cataract Surgery Inflammation.

Traditional randomized controlled trials (RCTs) face increasing challenges due to lengthy recruitment and high costs. Regulators have encouraged the use of external data and real-world evidence (RWE) to improve efficiency, yet adoption in confirmatory settings remains limited by concerns over heterogeneity and bias. We conducted a proof-of-concept study to assess the feasibility and regulatory value of a hybrid Bayesian borrowing design to support a Phase III RCT of Dexamethasone Intracameral Drug-Delivery Suspension (DEXYCU) in China. Using the Equivalence Probability Propensity Score Meta-Analytic-Predictive (EQPSMAP) approach, we integrated three data sources-a global RCT, a regional Phase III RCT in China, and a real-world data (RWD) in China. The method's performance was evaluated via point estimates and 95% credible intervals for the primary efficacy endpoint. The hybrid design based on EQPSMAP demonstrated greater robustness and accuracy in the presence of baseline imbalances and heterogeneous data. Compared to a traditional RCT, the hybrid design reduced the required sample size by 41 to 158 patients and shortened trial duration by approximately 2 to 5 months while preserving internal validity. This study demonstrated the feasibility and regulatory value of hybrid Bayesian designs in late-phase trials. The approach offers a practical, bias-controlled framework for integrating external data into regional drug development and regulatory decision making.

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来源期刊
CiteScore
12.70
自引率
7.50%
发文量
290
审稿时长
2 months
期刊介绍: Clinical Pharmacology & Therapeutics (CPT) is the authoritative cross-disciplinary journal in experimental and clinical medicine devoted to publishing advances in the nature, action, efficacy, and evaluation of therapeutics. CPT welcomes original Articles in the emerging areas of translational, predictive and personalized medicine; new therapeutic modalities including gene and cell therapies; pharmacogenomics, proteomics and metabolomics; bioinformation and applied systems biology complementing areas of pharmacokinetics and pharmacodynamics, human investigation and clinical trials, pharmacovigilence, pharmacoepidemiology, pharmacometrics, and population pharmacology.
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