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Clinical Trials in Children: A Challenging Need

In the European Union, the paediatric population (0-16 years old) represents about 75 million people, a fifth of the total population. Although this population may appear relatively large, the majority of medicines are still only developed and assessed for use in adult populations. The absence of suitable authorised medicinal products to treat diseases in children has been an issue of concern for some time.

This is mainly a result of the failure of pharmaceutical companies to perform the necessary tests and trials to adapt medicinal products to the needs of children. It is estimated that over two thirds of patients admitted to paediatric wards in European hospitals received drugs prescribed in an unlicensed or off-label manner, not having been specifically evaluated for use in children. Consequently it leads to risks of inefficacy and/or adverse effects.

Pharmaceutical companies have traditionally been reluctant to invest in developing specific treatments or adapting existing medicines to meet the needs of the paediatric population, mainly because the market is small and therefore of lower priority to them - and the risks associated with paediatric treatments are generally higher (for example, need for long-term follow-up of adverse effects). In addition, the pharmaceutical industry tends not to develop specific paediatric formulations.


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By Dr Carlo Giaquinto, Department of Paediatrics at the University of Padova, Italy
 
Carlo Giaquinto is Adjoint Professor at the Department of Paediatrics of the University of Padova in Italy. Since 1984 he has been responsible for the existing paediatric AIDS referral centre and has been a founder member of the European collaborative study. In 1993 he became the European Co-ordinator of the Paediatric European Network Treatment AIDS (PENTA) group.

He is involved in several international projects in both developed and developing countries, mainly related to the management of children with HIV infection. Carlo is also the Project Leader of a large network (Pedianet) of family paediatricians in Italy. He has recently been appointed Honorary Senior Lecturer at the Centre for Paediatric Epidemiology and Biostatistics of the Institute of Child Health in London.


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Dr Carlo Giaquinto
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