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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

Objectives:

  • 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:

  • October 2019: Project launch with first workgroup meeting of diverse stakeholders
  • January 2020: Core leadership team in-person meetings
  • 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
  • July 2020: Submitted public comments to FDA in response to rare disease clinical trial networks

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