# TreeScan to Evaluate the Safety of New Drugs in Pediatric Populations

> **NIH NIH R01** · BRIGHAM AND WOMEN'S HOSPITAL · 2024 · $507,519

## Abstract

Children are not small adults; yet we heavily rely on information collected in randomized and
non-randomized studies in adult populations to inform treatment strategies for pediatric patients.
Historically, children have been protected from research for ethical reasons. However, this well-
intentioned protection from the risks and burdens of trials has paradoxically left children more
susceptible to the potential risks of drugs used in everyday practice, frequently off-label and
without high-quality evidence of efficacy, effectiveness, or safety. The importance of
pharmacoepidemiologic drug safety monitoring using routinely collected healthcare data has
been increasingly recognized by the FDA, EMA, and other regulators, and forms the backbone
for a major component of the nation’s drug safety surveillance system. However, to date, the
focus of these initiatives has been on drug safety in adult populations; there is currently no
systematic surveillance system targeting drug safety in pediatric populations. To address this
evidence gap – which puts pediatric patients at increased risk – we propose to develop and test
the performance of TreeScan based approaches for the systematic and simultaneous
evaluation of multiple potential adverse outcomes in pediatric populations, and to implement
these methods for prospective sequential surveillance of new pediatric medications. TreeScan
methods use a hierarchical tree comprising thousands of outcomes and account for multiple
testing of correlated hypotheses while systematically screening for potential adverse effects.
The methodology has never been used to evaluate drug safety in pediatric patients, where there
are unique challenges compared to adult populations. Thus, the methods require further
development and refinement. We will test the approach based on real-world examples of
established drugs with relatively well characterized safety profiles (Aim 1) as well as plasmode
simulations that explore a broad range of plausible clinical contexts in pediatrics (Aim 2). The
performance tested approach will then be implemented to prospectively monitor the safety of
recently approved drugs and new drugs that will be approved during the early years of the grant
(Aim 3). Potential signals will be further evaluated in full-scale epidemiological studies. This
project will provide a critically needed tool that could lead to early detection of unsuspected
adverse effects of drugs in pediatric populations if they exist and provide reassurance if no
safety issues with large effects are detected. The proposed studies are expected to have an
immediate and important public health impact by providing comprehensive assessments of the
safety profiles for new medications in pediatric populations.

## Key facts

- **NIH application ID:** 10851994
- **Project number:** 5R01HD110092-03
- **Recipient organization:** BRIGHAM AND WOMEN'S HOSPITAL
- **Principal Investigator:** Krista F Huybrechts
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $507,519
- **Award type:** 5
- **Project period:** 2022-07-28 → 2026-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10851994

## Citation

> US National Institutes of Health, RePORTER application 10851994, TreeScan to Evaluate the Safety of New Drugs in Pediatric Populations (5R01HD110092-03). Retrieved via AI Analytics 2026-06-11 from https://api.ai-analytics.org/grant/nih/10851994. Licensed CC0.

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