Pediatric Formulations: A Roadmap

ISBN-13: 9781489980106 / Angielski / Twarda / 2014 / 439 str.

Daniel Bar-Shalom; Klaus Rose

Opis

Until the 1990s, it was generally accepted that medicines were first developed for adults and their use in children was investigated later, if at all. One of the main tasks of hospital pharmacies was the manufacturing of child-appropriate formulations in a more or less makeshift way. The first change came in 1997 with U.S. legislation that rewarded manufacturers to do voluntary pediatric research. Ten years later, the European Union passed legislation that required manufacturers to discuss all pediatric aspects, including formulations, with the regulatory authorities as a condition of starting the registration procedure. In consequence, manufacturers must now cover all age groups, including the youngest ones. So far, pediatric formulations were more a focus for academic researchers. Through the changed regulatory environment, there is now a sudden high commercial demand for age-appropriate formulations. This book begins by highlighting the anatomical, physiological and developmental differences between adults and children of different ages. It goes on to review the existing technologies and attempts to draw a roadmap to better, innovative formulations, in particular for oral administration. The regulatory, clinical, ethical and pharmaceutical framework is also addressed.

Recenzje

From the reviews:

"This book can be useful not only for those in drug development, but also for pharmacists who work in pediatrics (either hospital or community pharmacists), compounding pharmacists, pediatricians, nurses who specialize in this area, and certainly students or residents in any of these disciplines who plan to focus on pediatrics. ... I have reviewed and used many books on drug formulations and pharmaceutics, but I have not seen such a complete book devoted specifically to pediatrics." (Patrick J. McDonnell, Doody's Book Reviews, July, 2014)

Spis treści

Part I: The Patient

1. Pediatric Development: Anatomy. Age, Weight, Body Surface and Stature, Organ Development

Hannah Bachelor 

2. Pediatric Development: Physiology. Enzymes; Drug Metabolism; Pharmacokinetics & Pharmacodynamics

Geert ‘t Jong

3. Pediatric Development – Gastrointestinal 

Hannah Batchelor

4. How to Estimate the Dose to be Given for the First Time to Pediatric Patients

Gerard Greig

5. The Clinical Relevance of Pediatric Formulations

Karel Allegaert , Pieter De Cock , Johannes N van den Anker

Part II: Formulating for Children I, The Oral Route

6. General Considerations for Pediatric Oral Drug Formulation

Valentina Shakhnovich and  Susan M. Abdel-Rahman

7. Orosensory Perception

Per Møller

8. Flavor Is Not Just Taste: Taste Concealing

Charles R. Frey

9. Liquid Formulations

Charles R. Frey and J. Scott Madsen

10. Paediatric Solid Formulations

Sejal Ranmal and Catherine Tuleu

11. Semi-Solid Formulations

Daniel Bar-Shalom and Irwin C. Jacobs

12. The Challenge of Automated Compounding

Daniel Bar-Shalom

13. Pediatric Formulations in Clinical Testing and the Challenge of Final Market Formulation

Norbert Pöllinger

14. Buccal/Sublingual Drug Delivery for the Paediatric Population

Viralkumar F. Patel, Darragh Murnane, Marc B. Brown

Part III: Formulating for Children II, Non-Oral Routes

15. Topical and Transdermal

William McAuley, Matthew Traynor and Marc Brown

16. Parenteral Liquids for Intravenous  and Transdermal Use

Utpal U. Shah and Matthew Roberts

17. The Challenges of Paediatric Pulmonary Drug Delivery

Darragh Murnane and Marc B Brown

18. Nasal, Ocular,and Otic Drug Delivery

Hannah Batchelor

19. Rectal Drug Delivery

Hannah Batchelor

20. Intraosseous Infusions in Infants and Neonates

Jose Ramet, Maria A.L.J. Slaats Mst, Catharina J. Elsing Mst

Part IV: Compounding

21. Compounding for Children – The Compounding Pharmacist

Linda F. McElhiney

Part V: Materials

22. Food Ingredients

Parnali Chatterjee and Marie Ojiambo

23. Excipients and Active Pharmaceutical Ingredients (APIs)

Parnali Chatterjee and Mohammed M Alvi

Part VI: Clinical Development and Regulatory Aspects

24. Clinical Testing in Children

Klaus Rose 

25. Pediatric Formulations and Dosage Forms and Future Opportunities: Impact of Regulations in the United States and Implementation of Quality by Design    

Arzu Selen

26. Pediatric Pharmaceutical Legislation and its impact on Adult and Pediatric Drug Development: The EU Regulatory View
Siri Wang & Karl-Heinz Huemer

27. Pediatric Pharmaceutical Legislation in USA and EU and their impact on Adult and Pediatric Drug Development

 Klaus Rose

28. Checks and Balances in the EU: The Role of the European Ombudsman, with a Focus on the Paediatric Regulation

Rosita Agnew

Part VII: Concluding Remarks - The Future of Pediatric Formulations

29. The Dangerous Business Of Predicting The Future

Daniel Bar-Shalom. Hannah Batchelor, Linda F. McElhiney, Klaus Rose

O autorze

Daniel Bar-Shalom is an Associate Professor in Drug Delivery at The Faculty of Health and Medical Sciences, University of Copenhagen. He has a degree in Biology from The Tel-Aviv University (1975), carried research in botany and then graduated from the School of Pharmacy, Hebrew University of Jerusalem (1982). He is a qualified pharmacist and has managed and owned a pharmacy in Haifa where he gained insight into the needs and idiosyncrasies of patients. He moved to Denmark in 1986 and founded Egalet, a drug delivery company where he served until 2007 as Vice President, R&D. He has been a consultant for various pharmaceutical and medical device companies. He is also the inventor of various technologies, holds many patents and has formulated marketed products. His main interests are controlled drug delivery and in particular oral pediatric formulations and geriatric formulations addressing the problem of polypharmacy.

Dr. Klaus Rose is CEO of Klausrose Consulting, Switzerland and advises on pediatric drug development and how to comply with FDA and EMA pediatric requirements. He has studied Latin languages, psychology and medicine. After postgraduate clinical training in general medicine in Germany and England, he joined the pharmaceutical industry. He has held various positions in R&D and medical affairs, was Global Head Pediatrics Novartis from 2001 to 2005 and was Global Head Pediatrics Roche from 2005 to 2009. After a year with a regulatory consultancy, he established his own business. Dr. Rose is a frequent speaker on international conferences on pediatric drug development, organizes trainings and publishes on a regular base.

Blurb

As the first book that specifically addresses pediatric formulations in the context of drug development, Pediatric Formulations: A Roadmap covers anatomy and physiology of children as well as the technical state of the art, gives hints about where to find inspiration, and provides a suitable background on the regulatory framework. Existing books on pediatric formulations mainly discuss compounding at the pharmacy. This is not a “how to formulate” textbook for pharmaceutical scientists; the subtitle “A Roadmap” indicates that we are on a path in largely unexplored territory. It is an attempt to equip the reader with necessary information on how to get started with the appropriate mindset.

Among medicinal specialties, pediatrics is a young discipline. The potential dangers of medicine were often underestimated in earlier times. Cough suppressants labeled as “suitable for children and adults” a hundred years ago could kill children as they contained opioids. Since then, a complex framework has evolved, with physicians, pharmacists, drug development companies, generic companies, academic scientists, regulatory authorities, reimbursement organizations, patients and children’s parents as key players. The position of children in our society has changed dramatically during the last century. Children were protected against clinical trials, yet thanks to clinical trials, many more children with cancer survive today. The development of age-adjusted drug formulations is not only a technical challenge. For new drugs today, there are laws both in the United States and the European Union that compel the research-based pharmaceutical industry to also consider children during drug development. One of the major demands of authorities is the development of age-appropriate formulations.

Daniel Bar-Shalom is an Associate Professor in Drug Delivery at The Faculty of Health and Medical Sciences, University of Copenhagen. He has a degree in Biology from The Tel-Aviv University (1975), carried research in botany and then graduated from the School of Pharmacy, Hebrew University of Jerusalem (1982). He is a qualified pharmacist and has managed and owned a pharmacy in Haifa where he gained insight into the needs and idiosyncrasies of patients. He moved to Denmark in 1986 and founded Egalet, a drug delivery company where he served until 2007 as Vice President, R&D. He has been a consultant for various pharmaceutical and medical device companies. He is also the inventor of various technologies, holds many patents and has formulated marketed products. His main interests are controlled drug delivery and in particular oral pediatric formulations and geriatric formulations addressing the problem of polypharmacy.

Dr. Klaus Rose is CEO of Klausrose Consulting, Switzerland and advises on pediatric drug development and how to comply with FDA and EMA pediatric requirements. He has studied Latin languages, psychology and medicine. After postgraduate clinical training in general medicine in Germany and England, he joined the pharmaceutical industry. He has held various positions in R&D and medical affairs, was Global Head Pediatrics Novartis from 2001 to 2005 and was Global Head Pediatrics Roche from 2005 to 2009. After a year with a regulatory consultancy, he established his own business. Dr. Rose is a frequent speaker on international conferences on pediatric drug development, organizes trainings and publishes on a regular base.