Research progress on rare real-world data-driven target trial emulation for drug repurposing
10.12173/j.issn.1005-0698.202501061
- VernacularTitle:稀有真实世界数据驱动的目标试验模拟在药物再利用中的研究进展
- Author:
Bosheng LI
1
;
Xuan HUANG
1
;
Wenxuan WANG
1
;
Wenyun YANG
1
;
Fangrong YAN
1
Author Information
1. 中国药科大学理学院生物统计系(南京 210009)
- Publication Type:Journal Article
- Keywords:
Target trial emulation;
Real-world data;
Propensity Score;
Drug repurposing;
Digital intelligence technology
- From:
Chinese Journal of Pharmacoepidemiology
2025;34(8):926-937
- CountryChina
- Language:Chinese
-
Abstract:
For rare real-world data involving off-label drug use or comorbidity-associated polypharmacy,researchers have increasingly adopted target trial emulation to investigate drug repurposing for target indications.The success of such studies hinges on rigorous trial design and strict adherence to predefined protocols and standardized pipelines.Key elements in the trial design include the precise definition of inclusion and exclusion criteria,the selection of trial and control drugs and determination of treatment allocation time,the determination of appropriate efficacy endpoints for the target indication,the identification of causal estimands,and the development of robust strategies for confounding adjustment.The execution of the trial follows a structured process:screening eligible subjects,extracting relevant drug exposure data,constructing treatment and control groups,emulating the target trial,and ultimately generating hypotheses for drug repurposing through statistical inference.Propensity score methods,including stratification,matching and weighting techniques,are critical tools for addressing confounding bias and ensuring accurate estimation of causal effects.In recent years,creative progress has been made in target trial emulation,particularly in the calculation of propensity scores.Researchers have adopted advanced machine learning techniques,to enhance variable selection and have actively explored the use of innovative methods of digital intelligence technology like classification and regression trees,support vector machines,and deep learning for the application of propensity score calculation.Target trial emulation based on real-world data has achieved remarkable advancements in drug repurposing,demonstrating broad application prospects,particularly in cardiovascular diseases,metabolic disorders,Alzheimer's disease,and cancer.
