Making new discoveries that improve understanding and treatment of childhood diseases is an important part of CHoR’s mission. Dr. Henry Rozycki, CHoR’s Vice Chair for Research, answers some common questions about research studies and how research affects health care.
What are research studies and why are they important to pediatric care?
Research is the process of gathering and analyzing information to answer a question. A research study is the process of analyzing this information, or data, with the purpose of providing results that can be useful for a larger group. Studying how well a doctor’s patients do after a visit is only considered to be a “study” when the results are analyzed and presented to an audience beyond the doctor’s own practice.
At CHoR, we conduct many types of research studies. We have team members who work in the laboratory studying cells to better understand how diseases happen. We look at how doctors provide care and better ways to deliver care that would help patients more directly. Many doctors at CHoR conduct clinical research which involves gathering information from patients to improve care. Recent research breakthroughs at CHoR include new drugs for the treatment of cystic fibrosis, the advancement of treatment for children with neuroblastoma (childhood cancer) and improvements in care for traumatic brain injuries.
How do research studies and clinical trials typical work?
There are several types of clinical research studies. To find out how patients respond to a treatment, physician researchers collect information from the medical records of patients with a specific disease or condition in ways that protect any personal or identifying information. These are called retrospective studies and they tell us how well treatment is working. Improving care and outcomes involves prospective clinical trials. In these types of studies, eligible patients are invited to participate in a trial that usually compares the current standard of care to a new and, hopefully, better method of care. Prospective clinical trials can involve a new drug, an old drug for a new use, or a new procedure, treatment or device.
A good example of the success of clinical trials is in children with cancer. The majority of children with cancer in the United States are enrolled in a clinical trial that is coordinated among a nationwide network of children’s cancer centers and supervised locally by one of our doctors. As a direct result of previous trials, children with the most common kind of leukemia have gone from having less than a 10 percent chance of surviving to a greater than 90 percent chance.
How are clinical trials conducted at CHoR?
All clinical trials at CHoR must be reviewed and approved by an independent review board and supervised by faculty member. If a child meets the eligibility requirements, both the parents and the child (if the child is over age 7) will be given the information needed to decide whether to participate, including risks as well as benefits. All participation is voluntary and we provide the best care for all children whether they participate in a clinical trial or not. Once everything is explained, questions are answered and consent forms are signed, the participant follows the study plan. Most often this involves filling out a questionnaire, a blood test or taking a drug. In some cases, the drug a child is given might be real or a placebo (a fake treatment sometimes called a “sugar pill,” although it doesn’t necessarily contain sugar). The purpose of the placebo is to make sure that any effect of the drug is due to the drug and not to individual variation. In many trials, participants continue to receive their current treatment (what they are already taking), while in others, the study drug replaces their current treatment. No matter what is involved in the study, everything is laid out in the beginning to the family and the participant. Expenses related to participating in the study, like parking or travel, are often covered and some studies also provide a small compensation to participants.
What types of trials are currently underway CHoR?
CHoR usually has more than 100 clinical trials open for enrollment at any given time. There are clinical trials at CHoR for outpatients as well as inpatients in almost every specialty area: cancer, allergies, infectious diseases, diabetes, and lung and kidney diseases to name a few. Since studies close to enrollment when complete and others start at different times throughout the year, it’s not possible to list all of the current trials.
How can patients learn more about research studies/clinical trials?
Most often, doctors are on the lookout for eligible patients and will approach a parent directly. Research nurses and coordinators also contact potential participants. It is also not uncommon for parents to ask the doctor if their child might be eligible for a clinical trial. This may be especially useful for children with uncommon conditions or children who do not respond to standard therapy/care.
If you would like to inquire about whether there are any clinical trials for your child and their condition, you may contact CHoR’s Research Office at (804) 828-0212 or email Melissa.firstname.lastname@example.org directly. It can also be helpful to search for clinical trials through researchmatch.org and at clinicaltrials.gov. Both sites enable users to refine searches by age and condition.
Results from clinical trials are meant to help children in the future. Participation in a trial may also help your child, but it may not, and it is a parent’s decision whether a child participates. The best way to make that decision is to get as much information as you can about your child and their illness. CHoR has a checklist of questions to ask that may be helpful in making a decision.