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Pediatric Drug Development – MasterClass
Wednesday, 2nd September, 2020

Klaus Rose

klausrose Consulting,
Riehen, Switzerland


Registration & Coffee




Introduction: Pediatric Drug Development: clinical need, regulatory requirements US + EU, impact of US & EU pediatric legislation on drug development


EU Pediatric Investigation Plans (PIPs) and US initial Pediatric Study Plans (iPSPs)




Pediatric oncology: current PIP requirements/ US RACE for children act


FDA/EU expectations multiple sclerosis, epilepsy, dermatology




Regulatory strategies: PIP negotiations, PIP modifications, strategic outlook




End of Masterclass

Drug development in general is large, complex, and commercial. It has created modern drugs and continues to to so, has changed the course of many diseases; has created great wealth and many jobs. Many diseases face today profound treatment revolutions.

Pediatric drug development is not just a part of drug development. It emerged out of perceived discrimination of children in drug treatment and -development. It became highly relevant for pharmaceutical industry when the US began to reward pediatric studies with patent extension. The EU expanded this and demands pediatric investigation plans (PIPs) and pediatric studies for drug approval.

The US Food and Drug Administration (FDA) defines children as under 17, the EU as under 18 years old. These definitions are administrative and flawed. Newborns are immature and vulnerable, but they mature. Regarding drug metabolism, the body is mature long before the 17/18th birthday. US and EU pediatric laws pretend that "children" remain as vulnerable as newborns until they become adult. US and EU approve drugs separately in "children" as if they were another species. From 2020 on, the FDA will demand separate pediatric studies for new anticancer drugs. Most FDA/EMA-requested/demanded "pediatric" studies are medically needless, many harm patients by substandard treatment or no treatment at all in placebo groups.

US/EU pediatric laws have created many hidden interests. Companies must pay "pediatric" studies to get adult drug approval. These studies support research infrastructures and careers in academia, FDA/EMA, and industry. At this interface of law, medicine and regulatory affairs, everybody involved in hands-on care of children, drug development, regulatory affairs, ethics committee or is otherwise interested in child healthcare need a compass to navigate confusing information and regulatory requirements.

For pharmaceutical companies, these challenges include: (1) Negotiating "pediatric" studies with FDA and EMA; separate "pediatric" studies that lack sense or harm patients should be avoided. (2) Many ongoing "pediatric" studies cost millions per year, are questionable, and cannot recruit. The EMA insists on continuation. New ways to negotiate study modification or termination need to be explored. (3) Patient advocacy groups, Institutional Review Boards/ ethics committees, the media, the courts, scientific journals and the social media have started to discuss this challenge.

The pediatric drug development masterclass offers key information on regulatory "pediatric" challenges, relevant parts of developmental pharmacology, science, the history of pediatric laws & FDA/EMA decisions and the outcome of FDA/EMA-demanded studies.