Pediatric regulations enacted in both Europe and the USA have disrupted the pharmaceutical industry, challenging business and drug development processes, and organizational structures. Over the last decade, with science and innovation evolving, industry has moved from a reactive to a proactive mode, investing in building appropriate structures and capabilities as part of their business strategy to better tackle the challenges and opportunities of pediatric drug development.
The EFGCP Children’s Medicines Working Party and the IQ Pediatric working group have joined their efforts to survey their member company representatives to understand how pharmaceutical companies are organized to fulfill their regulatory obligations and optimize their pediatric drug development programs.
Key success factors and recommendations for a fit-for-purpose Pediatric Expert Group (PEG) were identified.
Pediatric structures and expert groups were shown to be important to support optimization of the development of pediatric medicines.
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The authors thank Dimitrios Athanasiou, Jack Cook, Begonya Nafria Escalera, Claudio Fracasso, Johannes van den Anker, and Katie Rizvi for their review of the draft manuscript. Special thanks to the following colleagues for revealing the structure of their respective PEG: Ensio Norjavaara & Solange Corriol-Rohou (AstraZeneca); Corina Becker (Bayer); Sebastian Haertter (Boehringer-Ingelheim); Leslie Dickman & Sabine Fuerst-Recktenwald (Roche/Genentech); Christina Bucci-Rechtweg (Novartis); Kristina an Haack, Ashley Strougo, & Sebastien Bernard (Sanofi); Sam Maldonado & Katherine Cheng (Johnson & Johnson); Angela James (Astellas); and Jing Liu, Carlo Bello, & Claudio Fracasso (Pfizer).
No funding sources.
Conflict of interest
Conflict of Interest forms have been uploaded for each author. Thomas Severin is employee of Novartis Pharma AG, Basel, Switzerland. Solange Corriol-Rohou is employee of AstraZeneca, Paris, France. Christina Bucci-Rechtweg is employee and stockholder of Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA. Kristina an Haack is employee of Sanofi/Genzyme, Chilly-Mazarin, France. Sabine Fuerst-Recktenwald is employee of F. Hoffmann-La Roche Ltd., Basel, Switzerland. Pirkko Lepola is employee of Helsinki University Hospital, Department of Children and Adolescents, Helsinki, Finland and has no disclosures. Ensio Norjavaara is employee of AstraZeneca, Mölndal, Sweden. Martine Dehlinger-Kremer is employee of Synteract, & EUCROF, Munich, Germany. Sebastian Haertter is employee of Boehringer-Ingelheim, Ingelheim, Germany. S.Y. Amy Cheung is employee of Certara Strategic Consulting, Princeton, New Jersey, USA.
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In this article, ‘Pediatric Expert Group’ (PEG) is a term used as a common denominator when talking more generally about a pediatric structure used within a company or companies.
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Severin, T., Corriol-Rohou, S., Bucci-Rechtweg, C. et al. How is the Pharmaceutical Industry Structured to Optimize Pediatric Drug Development? Existing Pediatric Structure Models and Proposed Recommendations for Structural Enhancement. Ther Innov Regul Sci (2020). https://doi.org/10.1007/s43441-020-00116-4
- Medicines for children
- Pediatric medicines
- Drug development
- Pediatric structures
- Expert group