Clinical Research Information
Did you know that about 80% of all drugs used by pediatricians have never been formally tested in children? This has resulted in many doctors using drugs in children based upon information from adult studies without adequate information about the safety, effectiveness, and actions during the different stages of development in children (infancy, childhood and adolescence).
In December 2000 another new law went into effect requiring that the U.S. Food and Drug Administration (FDA) ask doctors and pharmaceutical companies to test every new drug and new drug formulation for their use in children. This is a great leap forward in improving the health care of our children. If done safely and carefully, these tests will result in new and better drugs to treat childhood disease and illness. The first step in evaluating new drugs for children is to educate parents. As new and better drugs are developed to treat childhood diseases, more and more parents will be approached for permission to enroll their children in studies.
Here are answers to parents’ frequently asked questions about clinical trials and research involving children. Much of this information is taken from established and well-respected pediatric research organizations that are listed on the “Web Sites of Interest” page.
- What is a clinical study?
- Why is it necessary to do clinical research studies in children?
- Will my child be safe?
- Why would a child enroll in a clinical study?
- How and why would we be approached to have our child participate in a clinical trial?
- Does my child have the disease or problem that the study targets?
- Will my child be able to cope with a trial?
- What is a protocol?
- What is involved in the study?
- What are the doctor's hours?
- Will we be compensated for participating?
- What if my child is a girl of childbearing age?
- Is the doctor experienced?
- Is there an experienced study coordinator, nurse, or other resource person who is going to be available to me?
- Has the protocol been reviewed by an Institutional Review Board (IRB)?
- If my child is injured during the trial, who is responsible for paying for the cost of his or her treatment?
- Can we drop out of the trial at any time?
- Do I understand the consent form?
- What if I have trouble making a decision?
What is a clinical study?
A clinical research study provides patients with an experimental treatment. Conducted by qualified physicians, and under strict guidance of the United States’ Food and Drug Administration (FDA), the main objective of a clinical research study is to evaluate novel treatments for a specific disease. All studies are carefully researched in a laboratory before a drug is given to the patient. In many cases, the drug is also researched in adults before being given to children.
Why is it necessary to do clinical research studies in children?
Most medications currently available provide inadequate instructions on dosages for children (labeling). In the past, medical research has been done primarily with adults. Children, however, are not miniature adults. As a result, adult treatments may not always have the same effect in children.
Will my child be safe?
The FDA has established strict guidelines for how clinical trials are to be conducted. It requires the sponsor of the trial to scrutinize the credentials of the clinical investigators who conduct the trial. Your child will be monitored closely during the trial and may need to be seen more often.
Some reasons parents enroll their children into studies include:
· Clinical studies often include no-cost diagnostic testing (such as blood tests and X-rays) that may not otherwise be done.
· Most participants receive a free physical examination and medication.
· Parents and children with chronic illnesses want to help improve care for their disease.
· Research caregivers are often able to spend more one-on-one time with their patients. Parents often feel they receive more personalized care and education.
· Many parents would like their children to receive research medication before it is commercially available.
How and why would we be approached to have our child participate in a clinical trial?
Your child’s pediatrician may ask you based upon his or her knowledge of your child’s medical history. If your child is hospitalized or is being seen in a clinic, you may be approached directly by an investigator or a clinical coordinator. Some hospitals maintain a database of patients and diagnoses. The database may be searched if researchers are looking for a particular disease or condition. In hospitals, physicians meet at least once a week to review challenging cases and your child’s case may be one of them. It’s very important that you do not feel pressured into making a decision, even if it is your child’s pediatrician asking you to participate.
Does my child have the disease or problem that the study targets?
A drug study should always be a match for the child. Obviously, a child should not participate in the study of an epilepsy drug unless the child has epilepsy. However, some conditions are so common that all children will benefit by having your child participate. For example, researchers may be investigating a new drug to treat fever or ear infections in children.
Will my child be able to cope with a trial?
You know your child better than anyone else. Does your child have the temperament and personality to cope with the disciplined regimen required by a clinical trial? Most children will benefit from understanding that they’re helping others.
What is a protocol?
A protocol is the document from which all research studies flow. It’s the research “bible” that outlines exact procedures for the investigator to follow. There must be no deviation. A protocol explains what is being studied and why. It includes the name of the sponsor and names of the principal investigators. The sponsor may be a pharmaceutical company, the National Institutes of Health, or a foundation. The protocol will describe the study design, how many people will be in the study, how many times each patient will be seen, which laboratory tests will be done, and how often. It will explain the risks and potential benefits of the study. The protocol contains all the information required by the Food and Drug Administration (FDA).
What is involved in the study?
You should receive a verbal explanation of the study and the details of the protocol should be included in a written consent form. Although the consent form should give you most of the details, there are a few extra items you may want to ask about. For example, will you be actively involved with collecting and reporting data? If your child is older, will he or she be required to keep a diary or to phone in reports? How many visits will there be? Will blood be drawn? What are the risks? The expected benefits?
What are the doctor’s hours?
Does the investigator have evening and weekend hours? You’ll want to minimize disruptions to your work schedule and to your child’s school day. When you arrive at the doctor’s office, will you be seen promptly? When you are participating in a trial, you should be seen right away without waiting as you might for a routine office visit.
Will we be compensated for participating?
If there are no extra visits or procedures, you should not expect to receive any compensation. However, if there are, parents should expect to be compensated for the trouble they take to participate in a study. This includes reimbursement for transportation, meals and overnight accommodations. Your child may also be offered a small gift, such as a savings bond or a gift certificate for toys or books. However, be wary of expensive gifts as a lure to participate in a study.
What if my child is a girl of childbearing age?
Because there is so little information about the effects of drugs on pregnant women and developing fetuses, drug companies and clinical investigators will want to be sure that your daughter is not pregnant and will not become pregnant during the course of the trial. This may be a difficult issue for parents of young girls who are capable of child bearing but are not yet sexually active. A pharmaceutical company may require your daughter to take a pregnancy test and may also require her to use some form of birth control. Some companies may accept abstinence as a means of birth control.
Is there an experienced study coordinator, nurse, or other resource person who is going to be available to me?
Ask to meet the principal investigator, the study coordinator, and anyone else who will work with you during the study. It’s important for you to feel at ease with all of them and to be confident they will be available whenever you have questions or problem.
Has the protocol been reviewed by an Institutional Review Board (IRB)?
The Institutional Review Board is a committee within the organization or hospital that ensures the protection and ethical treatment of all participants in a clinical study. The IRB is an impartial, independent group that has no relationship with the individual clinical trial.
If my child is injured during the trial, who is responsible for paying for the cost of his or her treatment?
Usually this issue is addressed in the consent form. You should discuss this topic with the investigator before you sign the form.
Can we drop out of the trial at any time?
Yes. You should understand that you can drop out of the trial at any time, for any reason, and without a penalty. However, there are some trials (for example some chemotherapy trials for cancer) where dropping out may be risky or dangerous.
Do I understand the consent form?
You will be given a detailed consent form to sign. It should be written in a language you speak with words that you can understand. Informed consent is not a single event. It is an ongoing process during which you must have time to understand, to make an informed decision and to continually reevaluate your willingness to participate. You should be given a copy of the consent form so that you can reread it to refresh your memory about the study details.
What if I have trouble making a decision?
Sometimes parents feel overwhelmed, especially if they have just learned that their child has a serious illness. Sit back and listen to what your doctor and the clinical investigator have to say. Try to take in as much information as you can. Read through the materials they give you. Feel free to consult with your child's pediatrician and with other health care professionals. Remember, it's OK to say "no" if the study is not a good match for you or your child.