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Promoting Global Clinical Research in Children

Focus Area: Current Project

Focus Area: Global Regulatory Engagement

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. Although some countries have well-established regulatory frameworks for research involving pediatric populations, many countries do not. In turn, large-scale pediatric trials often struggle to complete enrollment; indeed, some trials involving neonates, infants, children, and adolescents are not initiated at all. As a consequence, pediatric patients are often prescribed therapeutic products for which information regarding safety or efficacy for those age groups and/or those indications is inadequate. Given the practical challenges of executing multi-national clinical trials, the regulatory challenges of working across jurisdictions, and the ethical challenges of enrolling children in research, global innovation in pediatric drug development is lacking.

Medicines—including drugs, biologics and devices—must be more frequently, rigorously, and carefully evaluated for safety and efficacy in the global pediatric population. Realizing 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 this goal, 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 to identify and analyze ethical and regulatory differences and to create a comprehensive set of harmonized tools for global pediatric research.

We do not seek to duplicate efforts that are already underway; rather, we hope to facilitate and advance further development in the field.

Status: Active Project

Impact: To recommend harmonized approaches and tools to regulate and facilitate pediatric research around the globe


  • To understand and assess the current global landscape of pediatric research regulations, with a focus on illuminating legislative and regulatory gaps and inconsistencies
  • To describe existing initiatives related to pediatric clinical research and drug development, including efforts to harmonize ethical and pragmatic approaches in pediatric clinical research
  • To identify the challenges related to decision making for individual children that impede the conduct of transnational pediatric clinical trials.
  • 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.

Key Milestones:

  • February 2022: Hosted the second in the 5-part webinar series, Time to Listen: Hearing from Young People in Clinical Research
  • November 2021: Panel discussion (Talking with teens: Clinical research educational brochures for youth) and presentation of 2 posters: Talking to Teens About Clinical Research: The Development of Educational Brochures By and For Youth and Engaging Youth Throughout Product Development: Why, When, Who, and How
  • October 2021: Hosted the first in the 5-part webinar seriesAdvancing 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: Awarded a 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 MeetingEmerging 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”
  • February 2020: Kickoff of monthly teleconference meetings for 3 smaller workstreams
    1. Decision making for children’s participation in biomedical research
    2. Risk/benefit considerations for pediatric research
    3. Challenges in implementation of global pediatric clinical trials, including those encountered and remedied during the COVID-19 pandemic
  • January 2020: Core leadership team in-person meetings
  • October 2019: Project launch with first workgroup meeting of diverse stakeholders

Project Leadership:

  • Dominik Karres, MD, CPM. Paediatric Medicines Office, Scientific Evidence Generation Department, European Medicines Agency
  • 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
  • Robert “Skip” Nelson, MD, PhD. Senior Director, Pediatric Drug Development, Johnson & Johnson

Program Manager:

  • Lisa Koppelman, MSW, LICSW, MPH
  • Walker Morrell, MBe