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  • Pediatric Clinical Trials are on the Rise as New Law Goes Into Effect

    August 9, 2000 MedscapeWire

    "When parents give children a medication, they're often engaging in a
    mini clinical trial of their own," says Mark S. Schreiner, MD, medical director of
    the Children's Clinical Research Institute at The Children's Hospital of
    Philadelphia. "That will change in December...when a new law goes into effect requiring that all new drugs and new formulations of existing drugs be tested for use in children."

    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% of the drugs on the market are well studied in children."

    The US 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 conducted every year. Based on the number of drugs approved
    in the last 5 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?"

    "When we conduct clinical trials, the safety of the child is our utmost concern,"
    states Dr. Schreiner. "All aspects of a clinical trial are preapproved
    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.... 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."

    "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.

    Dr. Schreiner advises pediatric clinical researchers to ensure that parents have the answers to the following questions before signing a consent form.

    How and why would we be approached to have our child participate
    in a clinical trial? A child's pediatrician may ask for a child's participation based
    on the pediatrician's knowledge of the child's medical history. If a child is
    hospitalized or is being seen in a clinic, parents 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 children with a particular disease or
    condition. In a hospital setting, physicians meet at least once a week to
    review challenging cases. It's very important that parents do not feel pressured
    into making a decision, especially if the person making the request is the
    child's pediatrician.

    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 any child participate. For example, researchers
    may be investigating a new drug to treat fever or ear infections in

    Will my child be able to cope with a trial?
    Parents know their children better than anyone else. Listen to
    parents when they express doubts that their child may not have the temperament
    or personality to cope with the disciplined regimen required by a clinical trial.
    However, it is important to remind parents that 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 FDA.

    What is involved in the study?
    Parents 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 provide most of the details, there are a few extra
    items parents may want to ask about. For example, will a parent be actively
    involved with collecting and reporting data? If the 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? Parents will want to
    minimize disruptions to their work schedule and to their child's school day.
    When parents arrive at your office, or the office of the clinical investigator,
    they'll want to know that they will you be seen promptly. Parents should be
    reassured that when participating in a trial, they should be seen right away
    without waiting as they 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. The children being studied in
    the trial 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, parents 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. The child may also be offered a small gift, such as a savings bond or a gift certificate for toys or books. However, parents should 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 a young woman who might participate 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 her 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?
    Emphasize to parents that they 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? Parents should ask to meet the principal investigator, the study coordinator,and anyone else who will work with them during the study. It's important for them to feel at ease with everyone involved and to be confident they will be available whenever there are questions or problems.

    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. Parents should discuss this topic with the investigator before signing the form.

    Can we drop out of the trial at any time?
    Parents should understand that they can drop out of the trial at any time, for
    any reason, and without a penalty.  Do I understand the consent form?
    Parents will be given a detailed consent form to sign. Remind them that the
    form should be written in a language they speak with words that they can
    understand. Informed consent is not a single event. It is an ongoing process
    during which parents must have time to understand, to make an informed
    decision, and to continually reevaluate their willingness to participate. Parents
    should be given a copy of the consent form so that they can reread it for
    more information 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. Be relaxed and considerate with
    parents; ask them to sit back and listen to what the clinical investigator has to
    say. They should try to take in as much information as they can and read
    through the materials presented. Remind parents that they can consult with
    the child's pediatrician and other healthcare professionals.
    Parents should know that it is all right to decline if the study is not a good
    match for them or their child.

    Medscape Wire is prepared by Deborah Flapan, associate editor at
    Medscape. Send press releases and comments to

    Jane Sheppard
    Future Generations
    Publisher of Healthy Child Newsletter
    Vital, In-depth Information on Children's Health Issues
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