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National Institutes of Health
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Why Do Research in Children?
"If we can find a way to reverse some of the effects of the disease, we can make it better and maybe parents won't have to worry about it..."
Sawyer, child in Fabry disease study

Why Do Research In Children?

Medicines, devices and treatments are often not tested in children.

At nearly half of medical visits, children are given a medicine and 70% of those medicines have only been tested in adults.

The simple truth is...children are not little adults.

But without research in children themselves, we have no choice but to treat them that way.

Doctors and nurses often give medicines to children even though they have not been studied and approved by the Food and Drug Administration (FDA) for use in children. This is known as "off-label" use. Most of the time, this works well but when the adult dose is adjusted to the weight of a child, there is a chance that the dose used could be ineffective or even harmful.

While it may sound like "guesswork," it's really been more of a 'hand-me-down' approach. Without research in children though, it's all we have. We need to think about how a child's brain and body are well as the way that medicines and other treatments are handled in a child's body over time.


Children are growing. They are changing and maturing all the time. When thinking about the right dose for a child, we look at the stage of growth:

Stages of Growth
The Stages of Growth range from preterm newborn infants, to term infants (0-28 days), to infants and toddlers (more than 28 days to 23 months), to children 2 to 11 years, to adolescents 12 to 18 years.
An 8-month-old is completely different from an 8-year-old, who is completely different from an even among children, everyone is different. And at each of these stages of growth, they may need different doses of medicine, different sizes of devices or different types of therapy. For example, testing of one antibiotic showed that babies needed higher doses than older children to get rid of their infection. Many medicines are filtered out of the body and handled differently by a child's developing liver or kidneys - and because there is limited research, we don't know what the long term effects on these organs may be. So, we need to study them to find out.
"We would not know as much as we know today if people were not brave enough to be in clinical trials. So it helps future generations."
Jackie, mother of child in Fabry disease study

Clinical studies are important

Why? Because they can help us:
  • Understand the differences in children as they grow and develop.
  • Identify the best dose of medicines to prevent harmful effects or under-treatment.
  • Produce chewables, liquids or tablets that are easier for children to take.
  • Find treatments for problems that occur only in children, like prematurity.
  • Find treatments for certain diseases or conditions that occur in both children and adults but which act differently in children and adults, like arthritis or heart disease.
  • Understand how medicines are used in and filtered out of the body in children of all ages.
  • Find treatments for new or existing diseases to improve the health of children in the future, like vaccine studies that were done years ago help children stay healthier today.
  • Treat our children like children, rather than little adults.
"She's part of a different statistic that's going to help other babies..."
Nicole, mother of child in heart defect study