PHILADELPHIA, Aug. 4 /PR Newswire/ -- "When parents give children a medication, they're often engaging in a mini clinical trial of their own," says Mark S. Schreiner, M.D., medical director of the Children's Clinical Research Institute at The Children's Hospital of Philadelphia. "That will change in December 2000 when a new law goes into effect requiring that all new drugs and new formulations of existing drugs be tested for use in children."
A clinical trial is a carefully designed study that tests the safety and effectiveness of a medication, medical treatment or device in a group of volunteers. Dr. Schreiner explains that there is a tremendous amount of variability from person to person in the way drugs are absorbed and metabolized in the body. There are also genetic differences among people that affect how certain drugs do or don't work in diverse populations. "Anytime a patient gets a drug, it's an experiment to see if it works for that particular patient. But with children, we're even more in the dark because we don't have the same quality information as we do for adults. Currently, only about 20 percent of the drugs on the market are well studied in children."
The Food and Drug Administration (FDA) has been concerned about this issue for more than 20 years. On a national level, some 180 pediatric drug trials are typically running every year. Based on the number of drugs approved in the last five years with potential benefit for children, the FDA approximates that an additional 375 pediatric trials should be conducted on an annual basis.
This dramatic increase in the number of pediatric clinical trials raises a number of issues for parents. Many more parents will be approached by physicians, asking for permission to enroll their children in drug trials. Parents will have a number of questions: "Is it safe to give my child a drug for which there is inadequate information? Or is it safer to enroll my child in a clinical trial?"
The issues of safety and ethics
"When we conduct clinical trials, the safety of the child is our utmost concern," states Dr. Schreiner. "All aspects of a clinical trial are pre-approved by the FDA and by an institutional review board. That means all of us are performing the same procedures in the same way. And from an educational point of view, everyone involved in conducting the trial shares information and experiences, and contributes to problem solving. The level of surveillance in terms of number of visits and attention to the individual child is usually much higher than during routine medical care. The result? A well-written clinical trial reflects the best care."
Dr. Schreiner points out another important safety factor. "We usually do not test drugs in children unless the drugs have first been tested in adults. So we do know more about the drug and its likely benefits before we start. An exception would be when the drug affects only children. For example, a clinical trial for a new vaccine may be tested in babies."
Children's studies also broach ethical questions. Parents must make decisions as trustees for their children because children are not legally or developmentally able to make those decisions on their own. "Pediatricians should become involved in this decision-making process," urges Dr. Schreiner. "They should scientifically review the study and in their own minds come to a decision about its value for their patients."
The balance between benefits and risks
"From my perspective, there is never a justification for any individual patient to take on undue risk to benefit the rest of society. On the other hand, if children do not participate in trials, then children as a group will not benefit," he says.
"One of the principles behind all research is the idea of distributive justice. That is, there must be a balance between the burdens of research and the benefits of research. We have inadequate information regarding the safety and effectiveness of drugs for children. As a result, children have not been equal recipients of the great advances in medicine and drug research. If we want to see children partake of the benefits, then we, as parents and society, must accept a communal obligation to bear some of the burden.
"We were all children, many of us will have children, and our children will have children. So we all have a stake in this. It's ironic that by protecting children from risks, we have also denied them the benefits," Dr. Schreiner concludes.
The Children's Hospital of Philadelphia, the nation's first children's hospital, is a leader in patient care, education and research. This 373-bed multispecialty hospital provides comprehensive pediatric services, including home care, to children from birth through age 19. The hospital is second in the United States among all children's hospitals in total research funding from the National Institutes of Health.
The Children's Clinical Research Institute (CCRI) is an academic contract research organization that designs and conducts comprehensive pediatric clinical trials.
About the Expert
A pediatric anesthesiologist at The Children's Hospital of Philadelphia since 1984, Mark S. Schreiner, M.D. is medical director of the Children's Clinical Research Institute. He has served as director of research for the Department of Anesthesiology and Critical Care Medicine and as chairman of the Hospital's Committee for the Protection of Human Subjects Institutional Review Board. He has designed and conducted clinical trials of drugs used in children and has been an advocate with federal granting agencies for incorporating the special needs of children in designing clinical trials. Dr. Schreiner graduated from St. Louis University School of Medicine and completed residencies in pediatrics at The Children's Hospital of Philadelphia and in anesthesia at the Hospital of the University of Pennsylvania. He also completed subspecialty training in Pediatric Anesthesia and Critical Care Medicine at The Children's Hospital of Philadelphia.
Pediatric Clinical Trials: What Parents Should Know
In December 2000 a new law goes into effect requiring that every new drug and new drug formulation be tested for use in children. Mark S. Schreiner, M.D., medical director of the Children's Clinical Research Institute at The Children's Hospital of Philadelphia, predicts a dramatic increase in the number of pediatric clinical trials that will occur next year. This means that more and more parents will be approached for permission to enroll their children. Dr. Schreiner, a nationally recognized expert in the field of pediatric clinical research, answers the questions parents will most likely ask when confronted with this opportunity.
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 a hospital setting, 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? Children have a great sense of altruism and they will benefit by understanding that they're helping others.
What is a protocol?
A protocol is the document by which all research studies flow. It's essentially the research "bible" that outlines the exact procedures for the investigator to follow. There must be no deviation. A protocol begins by explaining what is being studied and why. It will also include the name of the sponsor and the 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, what laboratory tests will be done and how often. It will also explain the risks and potential benefits of the study. The protocol also 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 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.
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 the doctor experienced? Has he or she conducted studies previously?
You have a right to ask for the credentials of the principal investigator and of anyone else involved in the trial.
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 course of 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?
You should understand that you can drop out of the trial at any time, for any reason, and without a penalty.
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?
In some instances, parents are 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 present to 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.
For more information, contact Kajsa Haracz of The Children's Hospital of Philadelphia, 215-590-7092, or haracz@email.chop.edu.
SOURCE: Children's Hospital of Philadelphia
WEB SITE: http://www.chop.edu/
ST: Pennsylvania
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