Questions to ask about clinical trials

To fully understand the clinical trial that has been proposed, here are some impor-
tant questions to ask the oncologist:
• What is the purpose of the study?
• Who is sponsoring the study? Who reviews it? How often is it reviewed? Who
monitors patient safety?
• What tests and treatments will be done during the study? How do these differ
from standard treatment?
• Why is it thought that the treatment being studied may be better than standard
treatment?
• What are the possible benefits?
• What are all possible disadvantages?
• What are the possible side effects or risks of the study? What are the side effects
of the study compared to those of standard treatment?
• How will the study affect my child’s daily life?
• What are the possible long-term impacts of the study compared to the standard
treatment?
• How long will the study last? Is this shorter or longer than standard treatment?
• Will the study require more hospitalization than standard treatment?
• Does the study include long-term follow-up care?
• What happens if my child is harmed as a result of the research?
• Compare the study to standard treatment in terms of possible outcomes, side
effects, time involved, costs, and quality of life.
• Have insurers been reimbursing for care under this protocol?
When discussing the clinical trial with the oncologist, you’ll need the information to
review later. Many parents bring a tape recorder or a friend to take notes. Some
parents write down all of the doctor’s answers for later reference.
Informed consent
True informed consent is a process—not merely an explanation and signing of docu-
ments. Informed consent requires that:
1. All the treatments available to the child have been laid on the table and discussed—
not just the treatment available at your hospital or through your doctor, but all the
treatments that could be beneficial, wherever they are given.
2. The parents and, to the extent possible, the child, have discussed these options
and decided that they want to consider one of them.
3. The option selected is thoroughly discussed, with all its benefits and risks clear
explained.
4. Those aspects of the study that are considered experimental and those that a
standard are clearly described.
A fully informed medical decision weighs the relative merits of a therapy after fu
disclosure of benefits, risks, and alternatives.
During the discussions between the doctor(s) and family, all questions should b
answered in language that is clearly understood by the parents and child, and the
should be no pressure to enroll the child in the study. The objective of the informe
consent process is that the participants are comfortable with their choice and ca
comply with it.
We had many discussions with the staff prior to signing the informed
consent to participate in the clinical trial. We asked innumerable
questions, all of which were answered in a frank and honest manner.
We felt that participating gave our child the best chance for a cure,
and we felt good about increasing the knowledge that would help other
children later.
The form that parents sign will have language similar to the following: “The stud
described above has been explained to me, and I voluntarily agree to have my chil
participate in this study. I have had all of my questions answered, and understan
that all future questions that I have about this research will be answered by the inve
tigators listed above.”
The study that our institution was participating in at the time of my
daughter’s diagnosis was attempting to lessen the treatment to reduce
neurotoxicity yet still cure the disease. My family began a massive
research effort on the issue, and we had several family friends who were
physicians discuss the case with the heads of pediatric oncology at their
institutions. The consensus was that since my daughter was at the high
end of the high-risk description, it was advisable to choose the standard
care, which was more aggressive.
Although we believe strongly in clinical trials, we decided to opt for
the standard treatment because she had a poor prognosis, and we felt
that she would need the cranial radiation, despite the possibility of late
side effects.
By the time a study is published in the literature, doctors on the cutting edge of treat-
ment are two to four years into improving that treatment or learning of its shortcom-
ings. For this reason, it is best to make decisions in partnership with knowledgeable
medical caregivers, rather than in isolation.
No matter how comfortable you are with your child’s treating oncologist, it may be
helpful to have another medical caregiver help sort out your options. Often, that per-
son will be the family’s pediatrician or family doctor. Second opinions can be obtained
from physicians at other institutions in your community or region, or any of the larger
pediatric cancer centers. They will arrange to review the information and outline rec-
ommendations. It is most useful to get a second opinion from a center that treats sig-
nificant numbers of patients with your child’s diagnosis. Most pediatric oncologists
are happy to facilitate this process for you.
When my son was diagnosed, we were told we had two options:
a clinical trial or standard treatment. We decided to get a second
opinion before making our decision. Our pediatrician, my husband,
and I met in the doctor’s office for a telephone conference with a
pediatric oncologist from a major treatment center. We each presented
our concerns. Our pediatrician thought of some issues neither my
husband nor I had considered. I think we all came away better informed
of our options.
Second opinions can also be arranged through the Childhood Cancer Ombudsman
Program, which uses volunteer specialists to provide free help to families consider-
ing the range of treatment options and engaged in the informed consent process. (See
Appendix C, Resource Organizations, for contact information.)