Modern Pregnancy Pharmacoepidemiology: Methodological Challenges and Solutions in Study Design

1. Course Aim

In this course, faculty will present and discuss the unique challenges of designing studies on the safety of medical products in pregnancy. A standard protocol template that meets regulatory requirements will be used to guide the discussion. The emphasis will be on methods. Dr Leyla Sahin will discuss what are considered important aspects of study design and protocol writing from a regulatory perspective. Drs. Bateman, Hernandez-Diaz, Huybrechts and Margulis will each focus on specific study design and analysis challenges, including but not limited to selection into the population, selection into the analysis, left and right truncation, the role of mediators (birth weight paradox), confounding adjustment in light of uncommon exposure and outcomes, when to move to reporting measures of association. A question-and-answer session will follow.

The overarching goal of the Modern Pregnancy Pharmacoepidemiology course is to keep up with advances in the field. We cover new topics each year, from overviews of issues that are particular to research on the safety of medical products in pregnancy (2015, 2020) to deeper dives into specific aspects, such as study design (2018), exposure measurement (2016), various maternal, neonatal and offspring outcomes (2017, 2021), and significance testing and analytical approaches to address biases (2019). In 2022, the course discussed pregnancy pharmacoepidemiologic research within the framework of target trial emulation. In 2023, the course focused on non-live birth outcomes. This year, the course will delve into important design and analysis considerations when developing a study protocol.

2. Requisites Statement

This is an intermediate level pharmacoepidemiology course.

3. Course Objectives

1. To gain insight in the regulatory perspective on drugs safety in pregnancy studies
2. To be better equipped to develop or review a protocol for a study on the safety of medical products in pregnancy that meets regulatory requirements
3. To provide an understanding of what makes causal inference harder during pregnancy
4. To be better equipped to critically assess the validity of studies on the safety of medical products in pregnancy

4. Syllabus Outline

1. Regulatory considerations for post approval pregnancy safety studies

2. Guidance on addressing the unique challenges in the design and analyses of studies on drug exposures during pregnancy