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Considering the Patient in Pediatric Drug Development - 1st Edition - ISBN: 9780128238882, 9780128242056

Considering the Patient in Pediatric Drug Development

1st Edition

How Good Intentions Turned Into Harm

Author: Klaus Rose
eBook ISBN: 9780128242056
Paperback ISBN: 9780128238882
Imprint: Academic Press
Published Date: 19th November 2020
Page Count: 414
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Considering the Patient in Pediatric Drug Development: How Good Intentions Turned into Harm addresses a fundamental challenge in drug development and healthcare for young patients. In clinical trials and clinical practice, the term "children" is used ambiguously to confer physiological characteristics to a chronological age limit, which in reality does not exist. This book outlines why the United States (US) and European Union's (EU) regulatory authorities, pediatric academia, and the pharmaceutical industry demand, support and perform pediatric drug studies, along with the key flaws of this demand that blurs the different administrative and physiological meanings of the term "child."

In addition, the book covers why most pediatric regulatory studies lack medical sense and many even harm young patients and the conflicts of interest behind pediatric drug studies. It includes relevant information about the maturation of the human body regarding absorption, distribution, metabolism and excretion of food and drugs as well as key differences between newborns, infants, older children and adolescents.

Key Features

  • Explains relevant information about the maturation of the human body regarding absorption, distribution, metabolism and excretion of food and drugs, including key differences between newborns, infants, older children and adolescents
  • Discusses historical roots of separate drug approval in officially labeled "children" and conflicts of interest in performing and publishing "pediatric" research
  • Helps to decipher justifications for pediatric studies to help people navigate the relevance of the information


Pharmaceutical personnel, physicians, nurses, pharmacologists, regulatory authorities, other healthcare professionals and those involved in clinical trials. Parents, patients and patients advocacy groups

Table of Contents

Rumpelstiltskin and the miller’s daughter
Conclusion and outlook
Abbreviations and technical explanations
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 medicinal
4.2 Suicide, depression, and mental disorders in young
4.3 Cancer and malignancies in young patients
4.4 Chronic arthritis in young and adult patients, and “Juvenile Idiopathic Arthritis” (JIA)
4.5 Diabetes
4.6 Allergies and allergen-specific immunotherapy (ASIT) in young patients
4.7 Epilepsy
4.8 Multiple sclerosis
4.9 Atopic dermatitis and psoriasis
4.10 Cystic fibrosis
4.11 Vaccines and antiinfectives
5. 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 patients
6.2 Developmental pharmacology and the globalization of the children-are-therapeutic-orphans mantra
6.3 Extrapolation
6.4 Daily deceit in “pediatric” studies
6.5 Myths in today’s medical world and “pediatric” studies
7. Juvenile animal studies
8. Child-friendly formulations: tablets, quick-dissolving formulations, liquids, and more
9. What do young patients really need?


No. of pages:
© Academic Press 2020
19th November 2020
Academic Press
eBook ISBN:
Paperback ISBN:

About the Author

Klaus Rose

Dr. Rose is a medical doctor by training and profession. In the mid-90’s, after clinical training, he joined the pharmaceutical industry. While at Novartis, in Switzerland, in 1997 he came across clinical studies in children and was intrigued. His eldest daughter suffers 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 was then global head of pediatrics at Novartis from 2001-2005 and moved to the same position at Genentech/Roche 2005-2009 in Basel. After one more year at a regulatory company he became an independent consultant in 2011. He has advised companies on European Medicines Agency (EMA) “pediatric investigation plans” (PIPs) and Food and Drug Administration (FDA) pediatric requests/demands.

Affiliations and Expertise

Klausrose Consulting, Pediatric Drug Development & More; Riehen (Basel), Switzerland


"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

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