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Review
. 2024 Jan;33(1):e5695.
doi: 10.1002/pds.5695. Epub 2023 Sep 10.

Overview of global real-world data sources for pediatric pharmacoepidemiologic research

Gerold T Wharton  1 , Claudia Becker  2 , Dimitri Bennett  3   4 , Mehmet Burcu  5 , Greta Bushnell  6 , Carmen Ferrajolo  7   8 , Sigal Kaplan  9 , Ann W McMahon  1 , Naimisha Movva  10 , Sudha R Raman  11 , Oliver Scholle  12 , Mina Suh  10 , Jenny W Sun  13 , Daniel B Horton  6   14
Affiliations
Review

Overview of global real-world data sources for pediatric pharmacoepidemiologic research

Gerold T Wharton et al. Pharmacoepidemiol Drug Saf. 2024 Jan.

Abstract

Purpose: Given limited information available on real-world data (RWD) sources with pediatric populations, this study describes features of globally available RWD sources for pediatric pharmacoepidemiologic research.

Methods: An online questionnaire about pediatric RWD sources and their attributes and capabilities was completed by members and affiliates of the International Society for Pharmacoepidemiology and representatives of nominated databases. All responses were verified by database representatives and summarized.

Results: Of 93 RWD sources identified, 55 unique pediatric RWD sources were verified, including data from Europe (47%), United States (38%), multiregion (7%), Asia-Pacific (5%), and South America (2%). Most databases had nationwide coverage (82%), contained electronic health/medical records (47%) and/or administrative claims data (42%) and were linkable to other databases (65%). Most (71%) had limited outside access (e.g., by approval or through local collaborators); only 10 (18%) databases were publicly available. Six databases (11%) reported having >20 million pediatric observations. Most (91%) included children of all ages (birth until 18th birthday) and contained outpatient medication data (93%), while half (49%) contained inpatient medication data. Many databases captured vaccine information for children (71%), and one-third had regularly updated data on pediatric height (31%) and weight (33%). Other pediatric data attributes captured include diagnoses and comorbidities (89%), lab results (58%), vital signs (55%), devices (55%), imaging results (42%), narrative patient histories (35%), and genetic/biomarker data (22%).

Conclusions: This study provides an overview with key details about diverse databases that allow researchers to identify fit-for-purpose RWD sources suitable for pediatric pharmacoepidemiologic research.

Keywords: global databases; pediatric research; pharmacoepidemiology; real-world data sources; real-world evidence.

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Conflict of interest statement

Conflict of Interest:

GTW, AWM, OS, CB declare no conflict of interest. DB is an employee of Takeda. MB is a full-time employee of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA, and holds stock in Merck & Co., Inc., Rahway, NJ, USA. NM and MS are employees of EpidStrategies, a Division of ToxStrategies and have received various research grants to conduct pharmacoepidemiology studies using the databases described in this study. CF is a member of academic Spin-off "INSPIRE SRL" INnovative Solutions for medical Prediction and big data Integration in REal world setting" SRL which has received funding for conducting observational studies from various pharmaceutical companies. SK is an employee of Teva Pharmaceutical Industries Ltd. JWS is currently an employee at Pfizer Inc. DBH receives salary support and research funding from the Childhood Arthritis and Rheumatology Research Alliance.

Figures

Figure 1.
Figure 1.
Questionnaire Responses and Verification Outcomes (N=93)
See this image and copyright information in PMC

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