Children in Research
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, there is no choice but to treat them in the same way adults are treated.
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.
This approach may sound like guesswork, but without research in children, it's all we have. We need to think about how a child's brain and body are developing...as well as the way a child's body handles medicines and other treatments over time. Clinical research in children helps us to treat our children like children, rather than as little adults, in several ways:
- It uncovers the best dose of medicines to prevent harmful effects or under-treatment;
- It leads to the development of chewables, liquids, or tablets that are easier for children to take, yet still safe;
- It results in treatments for problems that occur only in children, like prematurity;
- It leads to treatments for diseases or conditions that occur in both children and adults but that act differently in children and adults, like arthritis or heart disease;
- It results in treatments for new or existing diseases that improve the health of children in the future, like vaccine studies that were done years ago and help children stay healthier today; and
- Clinical research in children help us understand how medicines affect children's brains and bodies as they grow and develop.
The human subject research regulations define “children” as follows:
“Children” are persons who have not attained the legal age for consent to treatments or procedures involved in the research, under the applicable law of the jurisdiction in which the research will be conducted (45 CFR 46.402(a)).
In the US, the legal age of adulthood is a matter of state and local law. Kentucky state law defines "Child" as any person, who has not reached his or her eighteenth birthday.
Eligibility for Emancipation: Although there’s no law specifying an emancipation procedure In Kentucky, it’s still possible to become emancipated before 18. This can happen any of the following ways:
- Marriage: A child can marry before 18 and be considered a legal adult, this requires parental consent or a court order granting the marriage license.
- Self-Support: Moving out of a parent’s home and becoming self-supporting will impliedly emancipate the child, if the parent doesn’t try to get the child back to the home Court Order – A minor can petition the court for emancipation and, possibly, be granted it by court order.
Minors of any age can consent to emergency care or treatment for pregnancy, drug or alcohol abuse, or sexually transmitted infections (STIs). Minors 16 or older can also consent to outpatient mental health treatment without parental consent.
Children are growing. They are changing and maturing all the time. An 8-month-old is completely different from an 8-year-old, who is completely different from an 18-year-old, so even among children, everyone is different. And at each of the stages of growth below, children 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 conduct studies to find out.
Clinical research can look a lot like regular, or standard, medical care. Sometimes it is hard to tell the difference. Here are some of the ways they may be similar:
- The researcher and your health care giver can be the same person.
- The setting may be your regular clinic.
- The treatments may seem the same.
Research is done to help find out if a treatment or procedure is good for a large group of people with a certain disease or condition. Research helps to answer questions for the future health of those populations. Standard medical care, however, focuses on individual needs in the present.
When considering enrolling your child in a study, make sure you understand the difference between the regular care your child gets at the doctor and what's involved in research. Even when the place and healthcare providers are the same as your regular health care team, find out what makes it a research study.
Make sure you ask:
- How is this different from standard care?
- Will I see different doctors and nurses for the study?
- Will I go to a different hospital or clinic for the study?
- Will the doctors and nurses ask me a lot more questions about my child's condition?
- Will there be more paperwork or additional tests when we are in the study?
- Will there be more rules and deadlines in the study?
- Here is an example of how some things in a clinical study are the same as in regular care, while some are different.
QUESTIONS/CONCERNS ABOUT A STUDY: The Consent form you receive when you are invited to be a research subject includes some contact information if you have questions or concerns about the study. You are encouraged to contact the researcher if you have questions.
QUESTIONS ABOUT RIGHTS AS A RESEARCH SUBJECT: Call the office at 502.852.5188 or toll free at 1.800.334.UofL (8635). If calling toll free, when the operator answers, ask to be connected to extension 503.852.5188. You can discuss any questions about your rights as a research subject, in private, with a member of the IRB.
ANONYMOUS QUESTIONS/COMPLAINTS: If you have concerns or complaints about the research or research staff and you do not wish to give your name, you may call toll free at 1.877.852.1167. This is a 24 hour hot line answered by people who do not work at the University of Louisville.
Parents who are asked to enter their child into a study will want to know, "Will being in this study help MY child?" It is very important to understand that research is done to gain information about a disease, condition, drug, or treatment that will benefit children in the future. It is different from regular medical treatment that is given to help a specific child.
So, while most studies are not done to help a specific child, does this mean there are no benefits to being in a study? In fact, there can be potential benefits when entering a clinical study.
One reason that parents say that they join a study is to help other families in similar situations. Today our children have more protection from death, disability and discomfort from many childhood diseases like polio or measles because parents in the past made a decision to allow their child to be in a study to test vaccines.
But, there are still many medicines and procedures that have not been tested in kids. And before they can be accepted for use, they must be tested to see if they are safe and effective.
Researchers test new drugs and treatments because they have reason to believe they might work better or be safer than the standard care. In a study, your child may have access to something that is not available yet. If the drug or treatment is found to be helpful, your child may be among the first to benefit.
Sometimes enrolling in a clinical study can give your child a chance to see extra doctors or find out more facts about your child's condition. The study team may be able to tell you about organizations, groups, or websites that deal with your child's condition. A clinical study may be able to put you in touch with families going through what you are going through.
A clinical study may offer closer monitoring or additional testing for your child, which may not be part of regular care. Sometimes a study asks parents to keep a diary or to bring a child in to be seen more often, such as weekly visits. Children in a clinical study will be watched closely for side effects and to understand how the treatment is working. Whatever the reason, remember that clinical studies are designed to test if a drug or procedure works and is safe. There may be benefits for your child, but there may not be.