Pediatric Drug Development: Trends and Perspectives in the European Union

SUMMER 2022, THE EVIDENCE FORUM, WHITE PAPER

Summary

Specialty areas of unmet need, such as pediatrics, have not always been a priority focus of pharmaceutical companies due to low financial incentives compared to the research and development (R&D) investment required. As a result, it is estimated that greater than fifty percent of medicines used in children in the EU have never actually been studied in a pediatric population. In 2006, the European Medicines Agency’s (EMA) responsibilities expanded to include pediatrics, and in 2007, a single legislation, the Paediatric Regulation (EC No. 1901/2006), was implemented. The purpose of the Regulation is to improve the health of children in Europe by facilitating the development and availability of new medicines for children from neonates to 17 years. The Regulation dramatically changed the regulatory environment for pediatric medicines in the EU, primarily by establishing the Paediatric Committee (PDCO) which is responsible for coordinating the EMA’s work on medicines for children.

In this paper, we discuss:

• Pediatric Investigation Plans (PIPs), including rewards, incentives, and mandates on pediatric research programmes
• The Commission’s 10-year report on the impact of the Regulation, and a re-evaluation of the impact over the last 5 years based on current trends in pediatric medicine marketing approvals
• Challenges in conducting pediatric clinical trials and solutions to help companies develop effective pediatric studies

Authors

Andrew Bevan, MRes, CSci, CBiol, MRSB, Executive Director, Peri- and Post-Approval Interventional Studies
Evidera, a business of PPD, part of Thermo Fisher Scientific

Alan Nelson, PhD, Senior Director, Peri- and Post-Approval Interventional Studies
Evidera, a business of PPD, part of Thermo Fisher Scientific

Catriona Roscoe-Cutting, FTOPRA, Executive Director, Strategic Regulatory Consulting
PPD, part of Thermo Fisher Scientific

Susan McCune, MD, Vice President, Pediatrics & Clinical Pharmacology, Rare Disease and Pediatric Center of Excellence, Medical and Scientific Strategy Consulting
PPD, part of Thermo Fisher Scientific