Pediatric Pharmacoepidemiology

1. Course Aim
The increased use of medications by children and the historical exclusion of children from clinical trials has created the need for pediatric pharmacoepidemiology, a sub-specialty within pharmacoepidemiology. Pediatric pharmacoepidemiology requires adapting the known pharmacoepidemiologic principles and frameworks to account for the unique challenges in studying young populations (e.g., neonates, infants, children, and/or adolescents). This course will introduce participants who have a good understanding of pharmacoepidemiology to the specific challenges arising in pediatric pharmacoepidemiology and the operational approaches to account for these challenges. Specific topics covered include the implications of growth and development in children, the use of pediatric real-world data, and the design of studies tailored to the pediatric population.

2. Requisites Statement
This course assumes knowledge of how to conduct pharmacoepidemiologic studies in adults. No prior experience in pediatrics is required.

3. Course Objectives
• To understand why pharmacoepidemiologic studies of young populations may differ from studies of adults and why they may differ by pediatric subgroup
• To understand how to adapt pharmacoepidemiologic principles to address the unique challenges in conducting pediatric pharmacoepidemiologic studies

4. Syllabus Outline

i. Overview of Real-World Data in Pediatric Pharmacoepidemiology: Unique Challenges and Opportunities (Mehmet Burcu)
This section will present the purpose and scope of pediatric pharmacoepidemiology and provide an overview of the challenges and opportunities of using real-world data in pediatric pharmacoepidemiology, highlighting the additional data elements that are needed when studying young populations, currently available data sources, and unique ways that such data can be used to generate real-world evidence in the pediatric population.

ii. Growth, Development, and Treatment Prescribing Challenges (Daniel Horton)
Following a biological and clinical perspective, the section will review the implications of growth and development on pediatric pharmacoepidemiologic studies. This section will also focus on the aspects of medication exposure (including dosage and off-label prescribing) that are unique to young populations.

iii. Study Design Considerations in Children vs. Adults (Jenny Sun)
When designing a pharmacoepidemiologic study, when and how would the design differ for studies of children vs. adults? This section walks through an application example and highlights the importance of pediatric-specific design considerations, including those related to confounding control, measurement of study outcomes, and selection of the target population.

iv. Pharmacoepidemiology in Neonates and Infants (Jonathan Slaughter)
Studies of young populations can be uniquely different for each pediatric subgroup. This section highlights additional challenges and considerations that are unique to studying neonates (first 28 days after birth) and infants (28 days to 23 months).

v. Breakout Sessions & Discussion (All)
In small groups, participants will work together on case studies to adapt an adult pharmacoepidemiologic study into a pediatric study. Groups will brainstorm how conducting this study would differ for adults vs. young people, as well as how such study may differ if adapted to different pediatric subgroups (neonates, infants, children, adolescents). Ideas will be shared and discussed with the entire group of students and course faculty.