Over the years ADAMAS had assisted clients in a number of paediatric projects covering various therapeutic areas. These have included studies related to treatment of infections in neonates, prevention of bleeding during surgery, treatment of ADHD and a vaccine study, to name but a few. The investigator sites have been located in Europe, the United States and South East Asia. Dr Mandy Hall Dryden, ADAMAS’ subject matter expert on paediatric studies, recently ran a training session for all our Consultants as a refresher and information sharing session.
It is estimated that between 60% and 80% of medicines used for children have not been evaluated formally. Children differ anatomically and physiologically; they are not just ‘small adults’. The route of administration and dosage form are important considerations, for instance acceptable tablet size is related to the age of the patient, as are dosing volumes for those drugs administered orally. The relevant guideline on pharmaceutical development of medicines for paediatric use is EMA/CHMP/QWP/180157/2011 and there are a host of other relevant regulations and guidelines. The EU paediatric regulation 1901/2006 & 1902/2006 defines its overall policy objective as to improve the health of the children of Europe by increasing the research, development and authorisation of medicinal products for use in children. This overall objective is supported by a number of general objectives including one which aims to ensure that medicines used to treat children are subject to high quality research and appropriately authorised. All the objectives listed in the regulation should be achieved ‘without subjecting children to unnecessary clinical trials and in full compliance with the EU Clinical Trials Directive’. The paediatric population represents a vulnerable sub-group and special measures are needed to protect their rights. Areas to be considered are composition of, or advice received by, the ethics committee, consent/assent, minimising risk and distress, and adverse event reporting.
Audits of paediatric studies are particularly important to ensure that the above areas are fully complied with in the interests of the patients, but also to ensure ethical and scientific integrity.