Building global consensus on definitions for drug safety studies in pregnancy to improve harmonized evaluation of real-world data

Background: There is increasing interest among regulatory agencies, academics, industry, and payers regarding the use of real-world data (RWD) to study drug safety in pregnancy. A core challenge faced by investigators, however, is the lack of alignment across regulatory agencies and clinical societies regarding definitions of time anchors and common outcomes. As the use of RWD for pregnancy studies continues to increase, it is essential to develop recommendations for alignment of definitions to increase the external validity of studies in this field.

Objectives: This workshop will be aimed at all researchers working with drug utilization and safety in pregnancy.
Objective 1) To summarize and evaluate gaps in definitions and misalignment of existing guidance.
Objective 2) To explore and discuss the feasibility and suitability of proposed consensus of definitions for pregnancy studies.

Description: Intro and chair (Anne Broe), Brief presentation of existing guidance (Leyla Sahin) followed by interactive group discussions: Participants will review current definitions for key variables that are fundamental to the design of pregnancy studies: time anchors (start of pregnancy, trimester boundaries) (Sydney Willis), pregnancy loss (miscarriage and stillbirth) (Marleen van Gelder), and congenital malformations (classifications and differences) (Eileen Dareng). Using case study examples, participants will evaluate how the heterogeneity of these definitions impact results and interpretation of RWE studies. Participants are encouraged to share examples from their own research.

Presentation of proposed definitions as outlined by the MiPal consensus network, followed by interactive group discussions: During this exercise, participants will have the opportunity to review the recommendations and discuss the implications of the adaptation of these recommendations to inform design of RWD studies for drug safety in pregnancy.

Conclude with plenary summary of participant discussion.

Key takeaways – global alignment of definitions for pregnancy drug utilization and safety studies. Cross functional input including academic, industry, and regulators’ views on the importance of alignment in pregnancy terminology and definitions. The outcome of the workshop will inform a publication.

Participants will leave this workshop with an understanding of the breadth of definitions used in pregnancy studies and the challenges this heterogeneity poses for results and interpretation of real-world evidence (RWE). Additionally, participants will learn how to leverage recommendations from the Medication in Pregnancy and Lactation (MiPal) SIG working group to better align RWE from pregnancy studies.