# The development of a systematic approach to harness real-world evidence for the evaluation of medication safety and effectiveness in children

> **NIH NIH K08** · BRIGHAM AND WOMEN'S HOSPITAL · 2024 · $168,051

## Abstract

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 pro...

## Key facts

- **NIH application ID:** 10890198
- **Project number:** 5K08HD110600-02
- **Recipient organization:** BRIGHAM AND WOMEN'S HOSPITAL
- **Principal Investigator:** Timothy John Savage
- **Activity code:** K08 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $168,051
- **Award type:** 5
- **Project period:** 2023-08-01 → 2028-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10890198

## Citation

> US National Institutes of Health, RePORTER application 10890198, The development of a systematic approach to harness real-world evidence for the evaluation of medication safety and effectiveness in children (5K08HD110600-02). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10890198. Licensed CC0.

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