Children who have rare and serious diseases are often treated with medicines that were developed and tested in adults. For those whose young lives are affected by serious health problems, clinical trials may provide early access to potential treatments that could alleviate symptoms and contribute to critical data on how children will respond to treatment.
It’s a big decision to participate in a clinical trial. In consultation with their child’s medical team, parents and caregivers make that decision on a child’s behalf. As with all clinical trials, the process is highly regulated and safeguards are in place. But considering participating in research means weighing both the potential benefits and risks.
Here are five things to know about pediatric clinical trials:
1. Children and adults respond to treatments differently.
Children’s bodies respond differently to medicines, so research designed specifically for them is essential.
“Despite many similarities, pediatric patients can respond differently to the same treatments often used in adult medical practices,” according to Nicklaus Children's Hospital in Miami.
As one journal article put it, children are not little adults so doctors can’t simply extrapolate adult data to children. At different ages and stages, people absorb and metabolize medicines differently, the article said.
“Unlike adults, children are growing and developing, and their immune systems are evolving. The way medicines are absorbed and processed can be very different. If we understand these differences, we will learn more about how to treat younger patients appropriately,” said Moshe Vardi, CSL’s Global Head of Clinical Development.
2. The World Health Organization (WHO) says the lack of research about children is a global concern.
While mortality rates among children under 5 have dropped significantly since 2000, that progress is slowing, the WHO said in a report published in 2025. Children, especially newborns and infants, are underrepresented in research.
“Without focused research, child survival, growth and development will continue to be compromised. Investment in early childhood is widely recognized as providing high returns, reinforcing the need to give priority to research that supports evidence-informed interventions for this age group,” the WHO report said.
3. Parents and caregivers make the decisions.
Parents and caregivers are in the driver’s seat about whether to participate in a clinical trial and may withdraw a child from a trial at any time.
The U.S. Food and Drug Administration recommends that parents ask questions and take their time before deciding to enroll their child in a trial. The agency recommends asking:
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What is the evidence that the investigational product might help my child?
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How will you check to see if the investigational product is working?
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Are there any concerns that the investigational product could be unsafe?
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How closely will my child be monitored if there are problems?
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Who do I call if I’m concerned that there is a problem?
4. Trials involving children are carefully designed and regulated.
Clinical trials are conducted under strict guidelines, with strong safeguards in place. Children are invited to participate only if a study is suitable for them, and their rights, safety and well-being are always the priority.
Children’s hospitals around the world sponsor clinical trials to improve the health of their young patients.
In Australia, Melbourne’s Children’s Trial Centre, formed in 2014 “to deliver excellence in design and conduct all clinical trials” with child participants. The collaboration, including research, clinical and academic institutions, promises parents:
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Your child will only be invited to join a research study or clinical trial if it’s suitable for them.
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Saying no to a clinical trial will not affect the care or treatment your child receives.
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You’ll get clear information about the trial and have the chance to ask questions before deciding to join.
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Your child won’t be part of the trial unless you give written consent.
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You can change your mind and stop participating at any time, and your care will not be affected.
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Your rights as a patient will always be respected. This includes your right to access care, feel safe, be treated with respect, be involved in decisions, have your privacy protected and give feedback.
London’s Great Ormond Street Hospital for Children runs 200 clinical trials a year, the hospital recently reported.
The research staff describe their clinical studies this way: “This is where we work with our patients to try out new treatments, devices or procedures in a controlled way, to see if we can improve on the current options available.”
5. CSL made a recent announcement about its pediatric clinical research.
CSL invests in pediatric research that explores the value and effectiveness of treatments already approved for use in adults living with rare and serious diseases. Learn about a recent announcement following a pediatric clinical trial.
The research teams at CSL also recognize that children deserve special care when they’re participating in a clinical trial. Responding to feedback from caregivers, CSL was the first to offer child trial participants interactive teddy bears specially designed to be comforting during clinical trial visits and procedures.
More than just a stuffed toy, “Clinical Companions” from Empath Labs have their own app that simulates the medical procedures the child will experience. The bears offer support while also helping kids better understand medical procedures and feel more prepared for what to expect.
