Following an increase in cooperation between United States, Japanese and European regulators, clinical research trials worldwide are increasingly involving children. In December 2007, the World Health Organisation launched a campaign called “Make medicines child size” targeting a range of antibiotics, asthma and pain medication. They also called for further research into combination pills for HIV/AIDS as well as appropriate child therapies for a number of neglected tropical diseases.
The need for paediatric clinical trials
All clinical trial participants are vulnerable, none more so than children, which is why Phase I studies in healthy volunteers are not carried out on this age group as it is considered ethically unacceptable.
This book begins by explaining how current practice in paediatric clinical pharmacology is only in its early stages and does not reflect existing scientific medical knowledge. At present, most drugs prescribed to children are, in fact, adult drugs extended to treat paediatrics off-label, as not enough trials have involved children to officially approve the medications for their specific use in children.
The book comprises eight sections with contributions from 63 authors worldwide, all specialists in this field, with a wealth of knowledge and experience. These cover topics such as The Global Framework of Paediatric Drug Development, Paediatric Clinical Pharmacology Practical and Ethical Challenges and The
Future of the Paediatric Pharmaceutical Market.
There are some lengthy initial chapters on regulatory requirements that would likely be challenging even to an experienced researcher. Too many abbreviations begin to make it difficult to understand, but progress beyond these and there are some excellent chapters.
When covering the practical challenges faced by potential organisers of clinical trials, the book describes the key differences to consider between research on adults and children. It highlights the range of personnel that need to be involved and the current gap that exists between the clinical knowledge of paediatricians and the knowledge of research trial organisers. It goes on to describe the unique challenges to consider in making clinical trials not only justifiable but feasible, and acknowledges that ethics committees may have less experience with children which may slow down the approval process.
A chapter containing definitions and practical advice on paediatric consent/assent including regulatory rules details the challenges and timings of
the process in acute and chronic conditions and how to tackle them.
A further chapter covers issues surrounding the formulation and delivery method of new medicines and explores important differences to consider when dosing children as opposed to adults. For example using different formulations of the same drug, that would be easier to administer to children, can have an impact on the stability and shelf life of an Investigational Medicinal Product (IMP).
Guidance in line with the International Conference on Harmonisation defines the five different age categories for trials in children explaining that the different stages of child development over time may not be age-defined in a sick child. Understanding of these stages is essential in dose selection.
There is also a specialist section on disease specific challenges including a chapter on familial dyslipidaemia and even paediatric cancer treatment in Africa.
The book concludes with plans and predictions for the future. Clinical trials in children need strong support including the global cooperation and sharing of information through the internet on portals, collaboration of US and European regulators and specialist paediatric research networks. It also suggests raising awareness to parents and the public of the importance of paediatric trials through campaigns by patient organisations and the pharmaceutical industry.
All in all, this book is an essential reference guide for all practitioners involved in paediatric care as well as those involved in paediatric trials from the planning and protocol development stage right through ethical and practical considerations to consent/assent issues. I highly recommend a copy for the workplace.
Alex Hodges (email@example.com) is a Senior Research Nurse at Research Nurses Limited, trading as ResearchNurses.co, a specialist provider of Research Nursing services in the homecare setting.