Director, Pediatric Infectious Diseases
Stony Brook Children’s Hospital
Director, Office of Clinical Trials
Stony Brook Medicine
Research is the lifeblood of medicine and helps us move the practice of medicine forward. Dr. Sharon Nachman, an expert in infectious diseases and an active researcher for clinical trials and promising medicines, helps parents gain a better understanding of what’s important to know before making the decision to participate or have your child participate in a research study.
Why is research important?
Dr. Nachman: Discoveries made in labs (basic science) only help people if we know how to apply what we’ve learned from our discoveries. Each new discovery will need to be tested in people. ‘Clinical trials’ are how we refer to those studies.
Why do people in studies sometimes get a ‘placebo’ and what is that?
Dr. Nachman: A placebo is something that contains no active medication. Salt water is an example of a placebo. We use placebos in studies when there are no existing therapies for a disease. We then compare the placebo to our new therapeutic medication.
We might want to learn if the new treatment leads to more side effects compared to a placebo, or if people who are given a new vaccine have lower, higher or the same chance, for example, of getting COVID-19, as when they are given a placebo. Comparing them helps us see if our new ideas benefit our patients.
Pretty much all vaccines in use today were tested or compared to a placebo, including all of the COVID-19 vaccines.
Does research only involve sick children or sick adults?
Dr. Nachman: Research can involve healthy as well as sick children or adults. For each new medicine, we must evaluate across ages and for safety and best doses. Then we compare each new medicine to one that is currently the standard of care.
Why do we test medicines in children?
Dr. Nachman: While children may get the same diseases that adults do, they may need to be treated differently. If we ignore studying medicines in children, they would never have access to these medications and we wouldn’t know how to treat children effectively.
What happens before you ever enroll in a study?
Dr. Nachman: Once a researcher develops a clinical trial study plan, the Food and Drug Administration (FDA) reviews the study and approves it. Then it’s up to the treating physician’s institution to take the next critical step. Every institution has an Institutional Review Board, or IRB. No study is allowed to enroll a patient before the IRB says yes or no.
There are several questions that the IRB is responsible for making a determination about for a medical study. For example, is it a correct interpretation of what is accepted treatment for a current illness? Are there enough safety measures in the study so that you, the patient, are kept safe? In medicine, things change. The IRB looks at a study every single year the study is open. What looked like a great therapy a year ago may not look like a good therapy now.
Because the goal of the IRB is to protect all patients, the IRB could, at any time, say to continue a study or stop it.
What is informed consent?
Dr. Nachman: Informed consent is the conversation between you as the patient, (or in the case of children, you as the patient’s parent), and the researcher about the study, plus your final decision of whether or not to participate. For kids, the parents must give the consent and the children give “assent.” Kids ages 12 through 18 assent to a clinical research study. They must decide to either agree to participate or say no. If they say no, we can’t enroll them in a study. Researchers must confirm that a child understands what they are being asked to be involved in.
Children under age 12 often don’t understand what we are asking them and for them the assent process is a little more complicated. We sit with them and their parents, explain what we are studying, why we want them to be enrolled, and allow the child to say either “yes” they want to participate, or “no.”
To learn more about research, visit: stonybrookmedicine.edu/research/community_education