See All Projects

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 is convening a workgroup 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 generate and advance a comprehensive series 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: Development phase

Impact:  The research community will have tools to facilitate multi-national pediatric drug development

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 identify and characterize the relevant ethical considerations in designing, recruiting, conducting, and reporting multi-regional pediatric trials and to delineate universal ethical considerations from those that are culturally and/or geographically specific.
  • To promote both the harmonization of existing regulatory frameworks for pediatric research and the promulgation of new, complementary pediatric regulations around the globe.

Project Leadership:

  • Enrica Alteri, MD. Head of Human Medicines Research and Development Support Division, 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

Contact Information: mrct@bwh.harvard.edu