Begonya Nafria Escalera
Begonya Nafria
Share4Rare Coordinator

Clinical trials in paediatric research

Child undergoing medical test
When a pathology has no known treatment, clinical trials represent the only opportunity to improve the quality of life of patients. These studies are highly regulated and have become essential to move biomedical research forward.

A clinical trial is a research study that investigates how a drug or medical treatment works on people, including children. In the case of rare diseases, this is the only way that patients and families may be able to improve their situation, as there are practically no treatments available.

In a clinical trial, for instance, researchers may compare two treatments, a drug that needs to be studied and an already known treatment, or an inactive substance called placebo. Treatments are assigned to participants through a randomization process, in such a way that neither parents, nor children, nor the researcher chooses the treatment group.

In order to authorize a drug for its use on people, it must necessarily undergo this type of research, which is the most rigorous and reliable. In this way, researchers can find out what are the optimal dose, efficacy, safety and side effects of a drug.

Why do we need to perform clinical trials in children?

Children are not small adults.

Most of the drugs and treatments used on children have not been tested before in this group of age. Therefore, it is difficult to know the correct dose, safety and side effects. Furthermore, children are always growing up and their bodies change very quickly, so we must consider the different stages of growth when studying the dose of a treatment: an 8-month-old child is completely different from an 8-year-old child, who, at the same time, is completely different from an 18-year-old. At each stage of growth we may have to apply a different dose of a drug, a different measure of a device or a different therapy.

Clinical trials are important because they can help us:

  • Understand the differences in children as they grow up and develop.
  • Identify what is the best dose without side effects.
  • Produce drugs that children can easily take (syrups, chewing gum, etc.).
  • Find treatments for diseases that only occur in children.
  • Find treatments for diseases that also occur in adults but that have different symptoms or manifestations in children.
  • Find treatments that have an impact on improving the quality of life of children.

For this reason, the European Union health authorities have decided to increase the number of clinical trials in children in a regulated environment.

In rare diseases, given the small number of patients, it is not always possible to administer a placebo to a control group. For this reason, it is very important to describe, prior to any clinical trial, what in medical science is called the natural history of the disease, which studies the genetic basis that produces the disease (specific mutations and their typology), and its clinical symptoms over the years. This is what we try to do in Share4Rare. Will you help us?