Q: Why Would I Want My Child to Participate in a Research Trial?
A: Dr. Nora McNamara, University Hospitals Case Medical Center, Child & Adolescent Psychiatry Principal Investigator of ResearchThe United States Food and Drug Administration (FDA) has established a continuum of events necessary for approval of any new medical treatment or device that begins with animal studies, moves to small Phase I studies with healthy adult volunteers, and if the investigational treatment is deemed safe, continues with Phase II studies in affected individuals, and then Phase III studies that compare the treatment under study to either a placebo (something that looks like the investigational treatment but is inactive) or to an already established/FDA indicated treatment. This process takes years, yet it is essential as the best and safest way to determine if new treatments are helpful for many childhood conditions. I have been the Principal Investigator at the Discovery and Wellness Center for Children (DWCC), the University Hospitals Case Medical Center’s child and adolescent psychiatry research group, since 2012. Since that time, the DWCC has participated in Phase II and Phase III trials for new medications for children and adolescents with depression, bipolar spectrum disorders, schizophrenia, ADHD, Tourette’s Syndrome, anxiety disorders, and Autism Spectrum Disorder. More than half of the medications tested were not approved by the FDA, because there was no observed or recorded improvement in the group of children who received the medication compared to the group of children treated with a placebo.
So why, knowing this, would anybody ever want to enroll a child or adolescent in a clinical trial of a proposed new treatment? There is actually only one legitimate reason for anybody to volunteer to put themselves or their child at risk (risk of getting a placebo, risk of having potential side effects from the study medication, risk of having one’s blood drawn repeatedly, risk of boredom from long visits at our center answering repetitive/dry questions, among many other risks). That only legitimate reason is ALTRUISM. The youngsters that volunteer to be in clinical trials almost 100% of the time do so because they want to help other children in the future, children they will never even meet. That is a particular behavior that only the most caring human beings ever get a chance to practice. At the DWCC, we have dozens of children and adolescents and young adults every week who freely give us their assent to participate in a long and potentially uncomfortable process that may not even help them as individuals feel any better, because they want to help out their fellow human beings!
Every day, we DWCC researchers are awed by the bravery, the honesty, and the determination of our young subjects. We are grateful to have the opportunity to get to know them and to work with their families, we are constantly amazed by their determination to do something so meaningful at such young ages, and we are forever in their debt for those just-a-hair-less-than 50% trials that find that the treatment was effective and safe, and lead to new FDA approved treatments for so many conditions. The next time you or your child take a medication that has been FDA approved for whatever you hope the medication will help with, you might like to say a silent “Thank you!” inside your head to all of the hundreds of the unknown, brave volunteers who tried that medication (or a placebo!) in those Phase I, II, and III studies years before your medicine was available for regular clinicians to prescribe, because those unknown heroes, they did it just for you!