Children need new treatments

There are not yet enough safe and effective treatments for diseases in children and adolescents.

There are around 100 million children under the age of 18 in Europe (about 1 million in Finland) who do not have enough suitable treatments for different age groups. Children can be diagnosed with thousands of different diseases if all currently identified diseases and rare diseases are put together. Worldwide, children under the age of 5 are the group most exposed to serious, and often deadly, infectious diseases (e.g. pneumonia) and rare and ultra rare diseases are often also found in this age group, which, if untreated, can quickly lead to death, or maim the child and cause much human suffering to both the child and the family during the child’s lifetime.

About 6% of the world’s total population suffers from more than 7,000 different rare diseases. More than half of these rare diseases are found in childhood, of which about 70-80% are genetic in origin, i.e. genetic disorders. It is estimated that about 30% of children with rare diseases die before their fifth birthday, as delayed diagnoses often occur in pediatric patients. Many doctor visits may be needed to identify the right disease, as rare diseases may have unclear or overlapping symptoms and may touch several medical specialties.

Children and adolescents also experience many chronic, multisystem diseases, as well as developmental disorders, such as various neurological, motor or hormonal delays. Type 1 diabetes, celiac disease, and rheumatoid arthritis are some of the most well-known autoimmune diseases and metabolic diseases. Many children’s diseases require the use of drugs for adults, without the studies and knowledge about their suitability and long-term side effects in children. As a result, there is still a need for completely new or existing safer and more effective treatments for children’s and young people’s diseases, which must also be studied in the right target group.