Promoting Global Clinical Research in Children
Focus Area: Current Project
Focus Area: Global Regulatory Engagement
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 is lacking 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 harmonized approaches and 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. The MRCT Center is committed to ensuring children benefit from clinical research, as well as elevating the youth voice in all aspects of clinical trials and product development.
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:
- March 2023: Hosted the fifth and final webinar in the 5-part series, Promoting Global Clinical Research in Children: Informing the Future.
- January 2023: Presented “Aligning Stakeholders to Facilitate Pediatric Access to Medicines: From Product Development through HTA Review” at a Fondation Brocher conference in Hermance, Switzerland.
- 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 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 Director:
- Lisa Koppelman, MSW, LICSW, MPH
Project Manager:
- Lauren Otterman, MBHL