Necessity and limitations of paediatric research-a personal view

The objective of this review is to present a personal view on the need of paediatric research and its practical and ethical boundaries. The review is based on the personal experience of a senior paediatric neurologist and head of a research ethics committee, taking into account selected references. In summary, the physical and psychological immaturity and developmental potential of children, especially at a young age, require specific, age -appropriate research methods and topics that differ from research on adults. Worldwide, many children still die from common and treatable diseases such as measles and pneumonia. On the other hand, new lifestyle -dependent diseases such as allergies, attention deficit disorder and obesity are rapidly increasing in number in industrialised countries. With the gradual elucidation of the genetic causes of rare diseases, interest in their treatment is also increasing. The necessary research includes agerelated physiological and pathophysiological, pharmacological, psychosocial, educational, epidemiological and socio-economic studies. However, young children are not able to understand complex instructions and follow study protocols, which makes it difficult to conduct reliable and valid studies. In addition, the rarity of many paediatric diseases and the different age groups often require national and supranational research groups with the associated high costs. As children have limited capacity to understand and consent to the risks and benefits of their participation, they are considered particularly vulnerable persons who need to be protected in all types of research in accordance with international ethical regulations and laws. In conclusion, as research on children tends to be more complex and less often refinanced, public and private sponsors, regulators and public health systems are called upon to support the necessary research on children in any way they can. This is not only about developing new therapies for rare diseases in developed countries, but also affordable therapies for common diseases in poorer regions of the world.