Promoting Global Clinical Research in Children

Critical to the health of a population is the development and administration of safe and effective therapeutic products for children. Global innovation in pediatric drug development lags behind adult product development due to myriad practical challenges of executing multi-national clinical trials, regulatory challenges of working across jurisdictions, and ethical challenges of enrolling children in research. As a consequence, pediatric patients are often prescribed therapeutic products for which safety or efficacy information for those age groups and/or those indications is inadequate.

Medicines—including drugs, biologics and devices—must be more frequently, rigorously, and carefully evaluated for safety and efficacy in the global pediatric population. This vision depends, in part, on identifying and overcoming existing barriers to conducting multi-regional pediatric trials, and then motivating their enrollment, conduct, and completion.

In pursuit of the goal to further harmonize approaches and offer tools to regulate and facilitate pediatric research around the globe, the MRCT Center convened a workgroup in 2019 of diverse stakeholders from academia, industry, regulatory agencies, not-for-profit organizations, trial networks, patients and patient advocacy organizations, and existing pediatric research initiatives. With our workgroup activities now complete, we are focused on disseminating our findings and continuing to actively engage in various international projects.

We offer a comprehensive suite of resources tailored for stakeholders who are directly involved or have an interest in pediatric clinical research. This includes our Including Young People in Research toolkit, our Guiding Principles, and a curated selection of pediatric-focused webinars, complete with recordings and summaries.

In 2024, we released a comprehensive interactive webpage to summarize the work and link to key deliverables.

Objectives

To identify the challenges related to decision making for individual children that impede the conduct of transnational pediatric clinical trials, including issues related to assent, consent, and inclusion of the voice of young people.
To address the lack of consensus and awareness on key issues around benefit and risk considerations that create barriers to and inefficiencies in transnational research with children.
To define and address the operational barriers to conducting transnational pediatric trials and propose solutions, including the promotion of a model of global pediatric regulatory cooperation.

Key Milestones

January 2024: A comprehensive interactive overview of the Pediatrics project and key deliverables was released.
March 2023: Released the Including Young People in Research toolkit.
March 2023: Hosted the fifth in the 5-part webinar series, Informing the Future.
November 2022: Hosted the fourth in the 5-part webinar series, Facilitating Pediatric Medicines Development: Models of Global Cooperation
June 2022: Hosted the third in the 5-part webinar series, Assent and Consent in the Field: Culture, Context, and Respect
April 2022: Release of 3 additional pediatric educational brochures for young people contemplating participation in a clinical trial; these proudly carry the iCAN Seal of Approval
February 2022: Hosted the second in the 5-part webinar series, Time to Listen: Hearing from Young People in Clinical Research
October 2021: Hosted the first in the 5-part webinar series, Advancing international Pediatric Clinical Research: Informing the future form COVID-19 Lessons Learned.
July 2021: Panel discussion at DIA-Pediatric engagement in research: Young people have a voice
March 2021: Dissemination of “Biobank” comic book, a collaboration of Boston Children’s Hospital, Harvard Catalyst and Precision Link
February 2021: Project focus pivots to deliverable preparation
January 2021: Received an FDA R13 scientific conference grant to support a series of meetings, “Global Cooperation to Promote Clinical Research in Children” to be hosted virtually during 2021-2022
December 2020: Panel Discussion at MRCT Center Annual Meeting–Emerging Issues in Global Pediatric Clinical Trials: Perspectives on patient/family/caregiver engagement
November 2020: Released “Assent to Consent” flyer which earned the iCAN (International Children’s Advisory Network) “Seal of Approval”
January 2020: Core leadership team in-person meetings
October 2019: Project launch with first work group meeting of diverse stakeholders

Project Leadership & Staff

Barbara E. Bierer, MD. Faculty Director, MRCT Center
Steven Joffe, MD, MPH. Chief, Division of Medical Ethics, Perelman School of Medicine at the University of Pennsylvania
Dominik Karres, MD, CPM. Paediatric Medicines Office, Scientific Evidence Generation Department, European Medicines Agency
Robert “Skip” Nelson, MD, PhD. Senior Director, Pediatric Drug Development, Johnson & Johnson
Lisa Koppelman, MSW, LICSW, MPH. Program and Team Director, MRCT Center

CLINICAL TRIALS & RESEARCH

Promoting Global Clinical Research in Children