Why Do Research in Children? (Clinical Trial Guidelines)

One or more medications are prescribed at nearly half of all children’s doctor visits in this country yet upwards of 30% of these medicines have not undergone the rigors of clinical testing in children.

Even though pediatricians agree that children and adolescent are not just “mini adults”, the off-label use of adult-approved drugs, adjusted for weight, is common in the treatment in these populations. While this may sound like “guesswork,” it’s really more of a ‘hand-me-down’ approach and often the only real choice available to physicians when treating childhood conditions that also affect adults.

Without controlled clinical research in children, a child becomes an experiment of one each time a drug or device is prescribed off-label – only unlike a controlled scientific “experiment”, no statistically tested data are collected that might inform the larger medical community.

To address the gap in knowledge about the specific action of FDA approved drugs for adults prescribed off-label for the treatment of children, pediatricians, the FDA, the National Institutes of Health, and other federal entities are encouraging FDA approved drug and device studies in children.

With easy access to on-line information, parents are likely to hear about clinical trials and want to know more. The Q&As below are intended to aid clinicians in discussing clinical trial options with patients and parents:

Why is clinical research in children and adolescents necessary?

Clinical research in children and adolescents allows physicians to better treat these populations by:

• Finding the best dose of medicines to prevent harmful effects or under-treatment.

• Making chewables, liquids or tablets that are easier for children to take, yet still safe.

• Finding treatments for problems that occur only in children, like prematurity.

• Finding treatments for diseases or conditions that occur in children, adolescents and adults but which can act differently in each sub-population, like arthritis or heart disease.

• Finding 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.

• Understanding how medicines affect children and adolescent brains and bodies as they grow and develop.

Can you point to some of the successes with children from clinical trials research in the past?

Yes, here are several important examples:

• Clinical trials showed that surfactant—a substance that keeps air sacs in the lungs inflated—helps premature infants breathe. As a result, the lives of thousands of babies who would otherwise die of respiratory failure are saved each year.

• As a result of repeated clinical trials in children with cancer, most children who develop leukemia survive. Only 50 years ago, acute leukemia was almost universally fatal in young children.

• Prior to the development of the polio vaccine, 15,000 American children developed paralytic polio each year. In the last five years, there have been no cases of paralytic polio in the United States. This advance was only possible through clinical trials in children.

Will my child benefit from participating in a clinical trial?

In general terms, there are some data to suggest that people who participate in clinical research have better outcomes compared to similar patients with the same disease.The reason why research participants tend to fare better is unclear. Improved outcomes may relate to the close observation often demanded by clinical trials, or closer follow-up after care that is often dictated by the research. Children may benefit from participating in clinical research of new medicines because these medicines are usually tested and proven to be safe and effective in adults before they are studied in clinical trials in children.

These short-term benefits are small in comparison to the benefits that will certainly be recognized by future children.

Who is watching out for my child in a study?

The answer is – lots of people. Many qualified professionals review a study in detail before a single person is enrolled. These people include:

•  the scientists who focus on identifying the right treatment,

•  the statisticians who help to design the right study with the right number of participants to get good results,

•  the medical investigators (nurses, doctors, pharmacists, psychologists, and technicians) who will make sure that the study is done in the right way with the right participants, and

•  Institutional Review Boards (IRB) are independent committees that review research plans and consent forms to make sure that people in a study are informed and protected. They review studies both before they start and throughout the study.

What Is “Informed Consent”?

Before you decide whether or not your child should participate in a clinical trial, you should learn the key facts about it. To help you decide, the investigators involved in the study explain its details. If you agree to participate, you give your informed consent. Informed consent is not a contract. You may withdraw from the trial at any time and for any reason.

If I find a clinical trial on-line, how do I discuss it with my doctor?

• Write down a list of questions at home and take it with you when you meet with your doctor. Make sure you get all of your questions answered.

• Take notes during the discussion. Read the notes back to the doctor to make sure you have heard everything correctly.

• Ask what information is already known about the medical product that your child may be given. Studies may be in progress elsewhere and some data may be available.

• Ask for contact information if you would like to talk to other parents whose child is participating in the study. The study team can provide this information if they get permission from other parents to have you contact them.

• After being presented with information and an informed consent document, you do not have to sign it right away. Go home and think about it, talk it over with your family, your child’s pediatrician, and other trusted people.

How do I find out more about clinical trials?

More information about having your child participate in a clinical trial is available at government Web sites, such as:

• http://www.clinicaltrials.gov

• http://www.fda.gov/ScienceResearch/SpecialTopics/PediatricTherapeuticsResearch/default.htm

• http://www.childrenandclinicalstudies.nhlbi.nih.gov

• http://www.cancer.gov/clinicaltrials/

http://www2.niddk.nih.gov/Research/ScientificAreas/Epidemiology/KPCT.htm

CHOC Children’s currently has 378 open studies across multiple diseases.

For more information, please contact the CHOC Children’s Research Institute at (714) 590-4341 or visit our Web site at http://www.choc.org/research/

Sources:

http://www.childrenandclinicalstudies.nhlbi.nih.gov

http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm048699.htm