Considering the Patient in Pediatric Drug Development

Dr. Klaus Rose is a medical doctor by training and profession. In the 1990s, after clinical training, he joined the pharmaceutical industry. In 1999 at Novartis in Switzerland, he came across clinical studies in children and was intrigued. His eldest daughter suffered from Sturge–Weber syndrome, which is serious and very rare. Dr. Rose became passionate about “pediatric drug development” and what the European Medicines Agency (EMA) called “Better Medicines for Children.” He became global head pediatrics at Novartis from 2001–2005 and moved to the same position 2005–2009 at Genentech/Roche. After one more year at a regulatory company, he became an independent consultant in 2011. He advises companies on EMA “pediatric investigation plans” (PIPs), and Food and Drug Administration (FDA) pediatric requests or demands. He detected fundamental flaws in “Pediatric Drug Development“ and publishes about this (and more) in peer-reviewed journals and medical textbooks.

"The book is educational and interesting. As a pharmacist who worked in a pediatric hospital for five years, I do think the book could have discussed some of the life-saving efforts of pediatric medicine as well." --© Doody’s Review Service, 2020, Judith Klevan, BA, BS(Einstein Medical Center Philadelphia), reviewer, expert opinion

• Rumpelstiltskin and the miller’s daughterConclusion and outlook Introduction Abbreviations and technical explanations Background 1. The process of modern drug development and the challenge of young patients 2. US and EU pediatric legislation 3. A paradigm shift 4. Introduction: specific disease areas 4.1 Tissue-engineered products (TEPs), advanced medicinal4.2 Suicide, depression, and mental disorders in young4.3 Cancer and malignancies in young patients4.4 Chronic arthritis in young and adult patients, and “Juvenile Idiopathic Arthritis” (JIA)4.5 Diabetes4.6 Allergies and allergen-specific immunotherapy (ASIT) in young patients4.7 Epilepsy4.8 Multiple sclerosis4.9 Atopic dermatitis and psoriasis4.10 Cystic fibrosis4.11 Vaccines and antiinfectives5. Neonatology 6. Challenges at the interface of science, drug development, and drug approval beyond specific disease areas 6.1 The on-label/off-label framework in adults and young patients6.2 Developmental pharmacology and the globalization of the children-are-therapeutic-orphans mantra6.3 Extrapolation6.4 Daily deceit in “pediatric” studies6.5 Myths in today’s medical world and “pediatric” studies7. Juvenile animal studies8. Child-friendly formulations: tablets, quick-dissolving formulations, liquids, and more9. What do young patients really need?