The development of a systematic approach to harness real-world evidence for the evaluation of medication safety and effectiveness in children
开发一种系统方法来利用现实世界的证据来评估儿童用药的安全性和有效性
基本信息
- 批准号:10739628
- 负责人:
- 金额:$ 16.82万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2023
- 资助国家:美国
- 起止时间:2023-08-01 至 2028-07-31
- 项目状态:未结题
- 来源:
- 关键词:AddressAdultAdvisory CommitteesAgeAgreementAreaCalibrationCategoriesCellulitisCephalexinChildChildhoodClinicalClinical DataClinical TrialsComplementControlled StudyDataData FilesData ReportingData SetData SourcesDatabasesDevelopmentDiagnosticDiseaseDrug PrescriptionsEffectivenessElectronic Health RecordEpidemiologic MethodsEvaluationEventExposure toFDA approvedFoundationsGenerationsGoalsHealthcareHospitalsInfantInsulin-Dependent Diabetes MellitusKnowledgeLabelMachine LearningMeasuresMedicalMentorsMentorshipMethodologyMethodsModelingNational Institute of Child Health and Human DevelopmentOmeprazoleOutpatientsPatient CarePatientsPediatric ResearchPediatricsPharmaceutical EconomicsPharmaceutical PreparationsPharmacoepidemiologyPharmacologyPopulationProcessProtocols documentationPublic Health SchoolsPublishingQuality of CareRandomized, Controlled TrialsRanitidineReproducibilityResearchResearch DesignResearch PersonnelResearch PriorityRiskSafetySeriesSourceTherapeutic UsesTrainingTrimethoprim-SulfamethoxazoleVisitWarfarinWomanWorkage groupclinical careclinical practicecomparative effectivenesscomparative safetydesignhealth care service utilizationimprovedmedication safetynovelnovel therapeuticsoff-label usepediatric patientsprospectivesafety assessmentscaffoldsuccessvenous thromboembolism
项目摘要
Approximately 20% of pediatric outpatient visits result in a prescription. Half of these prescriptions are off label,
a number that rises to 80% among infants. The safe and effective prescribing of medications is essential to
providing the highest quality care, and yet many children are receiving medications that have not been
evaluated in their age group or in their disease area by rigorous randomized controlled trials (RCTs). There is a
tremendous amount of data collected in the routine clinical care of patients via electronic health records
(EHRs), claims data, and patient-reported data. These “real-world data” (RWD) can be harnessed with
advanced epidemiologic methods focused on causal inference to generate “real-world evidence” (RWE) of
medication safety and effectiveness. RWE studies, if conducted with scientific rigor, have the potential to
complement RCTs and address important evidence gaps in the use of medications in children.
This project aims to develop a framework for the rigorous conduct of RWE studies of pediatric
medication use through the following specific aims: (1) to emulate 10 RCTs of medication effects in children
using routinely collected healthcare data, (2) to evaluate alternative approaches to minimize bias when
emulating RCTs in pediatric populations, and (3) to conduct 3 pediatric RWE studies on medication effects in
clinical areas that lack RCT evidence. We will use RCTs as our gold-standard and use advanced causal
inference and pharmacoepidemiology methods to design RWE studies to emulate published RCTs as closely
as possible. Ten model RCTs will provide the scaffold for the evaluation of which methods perform best for the
emulation of RCTs using RWE, and under which conditions. We will identify and summarize the methods that
lead to the most successful emulation of RCTs. We will use best practice approaches to conduct three novel
RWE studies. We will use three large healthcare utilization databases, including publicly and commercially
insured children, as sources of RWD to conduct the RWE studies. These databases have detailed diagnostic
and prescription information, making them well suited to studying medication use in children.
The Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital
provides the ideal setting for this endeavor. I will work with a team of mentors with expertise in
pharmacoepidemiology, causal inference, comparative safety and effectiveness, pharmacology, and pediatric
research to successfully complete the proposed project. This project provides the ideal framework to develop
expertise in advanced pharmacoepidemiology and causal inference methods and support the overarching goal
of my research: to generate high-quality evidence of medication safety and effectiveness in pediatric patients
when no RCT data is available. Hands-on training and direct mentorship will be complemented by coursework
at the Harvard T.H. Chan School of Public Health. This endeavor will provide the necessary foundation for me
to become an independent investigator in pediatric pharmacoepidemiology.
大约20%的儿科门诊就诊导致处方。这些处方中有一半是标签外的
在婴儿中这个数字上升到80%。安全有效的药物处方是必不可少的,
提供最高质量的护理,但许多儿童正在接受的药物,
通过严格的随机对照试验(RCT)在他们的年龄组或他们的疾病领域进行评估。有一个
通过电子健康记录在患者的常规临床护理中收集的大量数据
(EHR)、索赔数据和患者报告数据。这些“真实世界数据”(RWD)可以通过以下方式加以利用:
先进的流行病学方法侧重于因果推理,以产生“真实世界的证据”(RWE),
药物的安全性和有效性。RWE研究,如果以科学的严谨性进行,有可能
补充随机对照试验,并解决儿童用药的重要证据缺口。
本项目旨在制定一个框架,以严格进行儿科RWE研究。
通过以下具体目标来评估药物使用:(1)模拟10项儿童药物效果的RCT
使用常规收集的医疗保健数据,(2)评估替代方法,以最大限度地减少偏倚,
在儿科人群中模拟RCT,以及(3)进行3项关于药物作用的儿科RWE研究,
缺乏RCT证据的临床领域。我们将使用随机对照试验作为我们的金标准,
推理和药物流行病学方法设计RWE研究,以模拟已发表的RCT,
越好. 10项随机对照试验模型将为评估哪种方法对治疗最有效提供依据。
使用RWE的RCT仿真,以及在何种条件下。我们将确定和总结的方法,
导致最成功的RCT仿真。我们将使用最佳实践方法进行三个新的
RWE研究。我们将使用三个大型医疗保健利用数据库,包括公共和商业
投保儿童,作为RWD的来源进行RWE研究。这些数据库有详细的诊断
和处方信息,使它们非常适合研究儿童的药物使用。
布里格姆妇女医院药物流行病学和药物经济学部
为这一奋进提供了理想的环境。我将与一个导师团队合作,
药物流行病学、因果推断、比较安全性和有效性、药理学和儿科
研究,以顺利完成拟议的项目。该项目提供了理想的框架来开发
先进的药物流行病学和因果推理方法的专业知识,并支持总体目标
我的研究目标:为儿科患者提供高质量的药物安全性和有效性证据
当没有RCT数据可用时。实践培训和直接指导将辅之以课程作业
在哈佛T. H.陈氏公共卫生学院。这一奋进将为我提供必要的基础
成为儿科药物流行病学的独立研究者。
项目成果
期刊论文数量(0)
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