Considering the Patient in Pediatric Drug Development

Considering the Patient in Pediatric Drug Development

How Good Intentions Turned Into Harm

1st Edition - November 19, 2020

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  • Author: Klaus Rose
  • eBook ISBN: 9780128242056
  • Paperback ISBN: 9780128238882

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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?

Product details

  • No. of pages: 414
  • Language: English
  • Copyright: © Academic Press 2020
  • Published: November 19, 2020
  • Imprint: Academic Press
  • eBook ISBN: 9780128242056
  • Paperback ISBN: 9780128238882

About the Author

Klaus Rose

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.

Affiliations and Expertise

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

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