Frequently Asked Questions About Pediatric Clinical Trials

• Could a Clinical Study Help My Child?
• Why is research with children important?
• What is a clinical study?
• What Happens in a Clinical Study?
• How do I know if my child qualifies for a study?
• What is a study protocol?
• Are there benefits to my child participating in a clinical study?
• How can I weigh the benefits and risks of placing my child in a study?
• Can my child leave a clinical study after it begins?
• Are there additional charges or financial considerations?
• Who sees my child's medical records?

Could a Clinical Study Help My Child?

Akron Children's offers patients many opportunities to receive the latest medicines, treatments, and procedures through clinical study participation. By facilitating these studies, the Rebecca D. Considine Research Institute contributes to improving healthcare for children of every age. For the most part, new drugs and treatments are proven safe and effective in adult studies. Yet infants and children are quite different physically and mentally. They are growing and maturing all the time. Therefore, studies may often establish a medicine’s effectiveness, safety, and the best dose to give children of different ages. Other clinical studies seek to assess whether a new treatment has certain advantages over current therapies. Watch this National Institutes of Health video about the importance of children in clinical studies.

Why is research with children important?

Most medications doctors prescribe were tested and approved for adults. Such treatments may not work the same way in infants, children, or teens. Children are constantly growing and developing. There are a host of similar reasons for conducting research with children.

Doctors undertake studies to: 

• Understand differences in children as they grow and develop.
• Find treatments for problems that occur only in children, like prematurity. 
• Find treatments for certain diseases or conditions that affect both children and adults but act differently in children, such as arthritis or heart disease. 
• Identify the best dose of a medicine to prevent under-treatment or side effects. 
• Produce chewable, liquid or tablet formulas that are easier for children to take. 
• Find treatments for new or existing diseases to improve the health of children in the future, such as vaccines to help children stay healthier.
   

What is a clinical study?

A clinical study evaluates the effectiveness and/or safety of new treatments or medical devices on patients with a known health problem. A study might also test medical procedures (better surgical techniques, etc.).

The goal is to better understand, diagnose, treat or cure a disease or condition. Well-designed, closely monitored studies are often the fastest, safest way to find new or better therapies. When a study shows a new therapy is effective and safe for disease prevention, treating a specific medical condition, or provides treatment benefits, the Food and Drug Administration (FDA) approves the medicine or device for all doctors to offer to their patients.

What Happens in a Clinical Study?

Carefully planned and closely monitored, studies run the gamut in terms of what is required from patients or parents. Some are as simple as asking a parent (or child) to complete a survey. Other studies may require diagnostic tests, entering information in a home diary, or periodic visits to the hospital.

Should you find a study that might help your child, learn as much as possible about the study’s purpose and the activities involved. Find out what you will be asked to do. Write down your questions. The doctor leading the study, or a qualified study team member, will explain the study in detail. If you don’t understand, continue to ask for further explanation.

Many parents also seek advice from their child’s pediatrician or family doctor. We encourage you to talk it over at home as well. To enroll your child, he or she will need to meet specific study criteria (age, gender, weight, medical history, etc.).

How do I know if my child qualifies for a study?

Every study specifies who can participate. Known as “inclusions” and “exclusions,” these factors are determined by the specific illness or medical condition and a child’s age, weight, medical history and other traits relating to the study’s goals.

Some studies need healthy volunteers, such as tests for new vaccines. If you are interested in a study but your child is ineligible, explore whether an alternate study may benefit your child’s health.
 

What is a study protocol?

Doctors conducting a clinical study must follow detailed pre-approved procedures and guidelines. For instance, the protocol may specify how often a patient must take a specific dose of medicine.

The protocol also includes a schedule for any special tests, X-rays, or examinations that are required. Sometimes a protocol may also alert patients and their parents to watch for potential side effects.

This written “recipe” forms the foundation for all protocols. It ensures every study physician follows identical practices no matter where the study takes place.

Are there benefits to my child participating in a clinical study?

Study participation offers several potential benefits:

• You play an active role in your child’s healthcare.
• Your child may gain access to new treatments not yet available to the public.
• Your child will have expert medical care at a leading healthcare facility.
• Everyone in a clinical study contributes in some fashion to medical knowledge that will benefit others.

 

How can I weigh the benefits and risks of placing my child in a study?

There is no one answer to this question. Some things to consider include:

• Is your child doing well on his or her current treatment?
• If not, are there any treatment options other than study participation? 
• How serious is your child’s condition and what is the long-term prognosis? 
• Do you feel you understand all potential benefits and risks?

You should also seek advice from your pediatrician or family doctor. Talk it over at home. If you have more questions, continue to ask the doctors or study team members who are conducting the research. They encourage you to ask questions at any time throughout the course of your child’s study participation. If their response is unclear to you—ask them to provide a different explanation.

Before putting your child in the study, you must feel at ease about the goals, requirements, and potential treatment results. Having all your questions answered is critical to reaching such a comfort level.

Can my child leave a clinical study after it begins?

Yes. You may remove your child from the study at any time.

Are there additional charges or financial considerations?

Sometimes a study sponsor may reimburse expenses such as travel, meals, and similar costs that could be required for being in the study. Each study varies. You should also find out what insurance may or may not cover.

Who sees my child's medical records?

Research investigators collect only the data or patient information needed to meet the study goal. Investigators and research sponsors (governmental agencies, universities, healthcare companies, etc.) must adhere to strict guidelines to ensure a child’s medical and research information stays confidential.

Known as “Protected Health Information,” this includes items such as name, address, medical record number, and similar individual data. Sometimes medical journals publish study findings to inform the wider medical community. However, patient names or other identifiable information is never published without the parent’s permission.