Pediatric treatments - a challenging field of research!

Children often have to accept treatments based on what is known to work in adults. Many efforts are being made to provide proper research for children, to find the best treatment and drugs. Drug companies face challenges: pediatric trials come with a high level of complexity and cost. Besides ethical, economic, regulatory and technical requirements when conducting clinical studies on children, cultural specificity should not be neglected. The experts in the Ethics Review Committee have the same status as experts performing the scientific evaluation, bound by the European Commission obligations concerning conflict of interest and confidentiality. For each pediatric clinical trial obtaining Informed consent, Parental permission, and in some cases, a patient assent is obligatory. The most difficult issue is to conduct clinical trial in neonates; therefore in this population many of the medicines included in the Clinical Guidelines go “off-label” before getting ready to study this population. Neonates, preterm or term, represent the most vulnerable paediatric population. When affected by serious diseases, they are multi-drug users with many potential interactions. Trial protocols for newborns should consider potential for long-term, including developmental effects. Data show that current number of clinical trials in Europe is ~20000, 14,29% of them conducted in paediatric population in EU countries, while only 10% are conducted in children of developing countries. In Macedonia, as a part of South-Eastern Europe, there are 3/36 clinical trials conducted in children up to 16-17 years. None of them is conducted in neonatal period or infancy. Compared to others in the region, these data are far from satisfactory (Serbia registered 21/215 as paediatric clinical trials, Croatia 20/303, Bulgaria 27/415). And why were these 3 studies completed? Because they were part of multicenter clinical trial in EU, funded by the pharmaceutical industry, and were together with almost all SEE countries. Instead of conclusion: Clinical trials, conducted in proper way, are necessary and must follow general principles of medical ethics, harmonized with the rights of the child:respect for life, beneficence(do some good), non-maleficence(do no harm). Researchers/clinicians should never forget that children are not “little adults, they are unique” (Dr R.Renkins), and “the research is really the only way to bring children into first class citizenship into the medical care” (Dr G.Pearson)