research FAQs for families

A research study is a scientific way to improve or develop new methods of health care. Studies are designed to answer specific questions on how to prevent, diagnose or treat diseases and disorders. Clinical research studies, or clinical trials, involve people. They may test new medicines or devices, and study ways to screen, prevent, diagnose and treat disease. Every clinical research study must follow strict guidelines under the Food and Drug Administration (FDA). All studies are carefully researched before a drug is given to a patient.

At Dayton Children's, the majority of clinical trials are Phase II through IV trials, which means that the drugs being studied have already been through extensive testing and are considered very safe. In most cases, the drug already has been tested and approved for use in adults before any study with children.

Clinical research studies are the best way for medical professionals to identify the most effective therapies for children. Studies can:

• Provide information about dosing to prevent harmful effects or under-treating of disease;
• Help companies develop chewables, liquids or tablets that are easier for children to take, yet still safe;
• Lead to the development of therapies for conditions that occur only in children, such as prematurity;
• Lead to treatments for conditions that occur in both children and adults but act differently in children and adults, such as arthritis or heart disease;
• Help us understand how medicines affect children's brains and bodies as they grow and develop.

The health and safety of children is our number one priority. Clinical research is federally regulated with built-in safeguards to protect participants. The clinical research studies follow carefully controlled protocols, which are reviewed by an Institutional Review Board. An Institutional Review Board is an independent group of medical, scientific and nonscientific members, whose responsibility is to ensure the protection and safety of the participants involved in a trial. In addition, the ethical and legal codes that govern medical practice also apply to clinical research studies.

The U.S. Department of Health and Human Services (HHS) recently launched a new public outreach website to help people learn more about what participation in a research study involves.

Some reasons include:

• Clinical research studies often include no-cost diagnostic testing, such as blood tests and X-rays, which might not be done otherwise.
• Participants receive free physical examinations and may receive free study medications.
• Research participation can help advance treatment options for other people.
• Clinical research staff often is able to spend more one-on-one time with patients who participate in research.
• Families often feel they receive more personalized attention and education when their child is participating in a research study.
• Participants may receive investigational research medication before it is available to everyone.

If a study is being conducted in the specialty area at Dayton Children's where your child is treated—cancer care, for example you may receive information about the study from that department. If you visit your child's primary care physician and his or her office is participating in a study, the office staff may provide you with information about the study.

Most studies are conducted at Dayton Children's. Other studies are conducted in participating community physician offices, or based at other hospitals in our region.

• Double-Blind Study: A research study in which neither the patient nor the physician knows who is receiving a particular treatment.
• Placebo: A pill, liquid or powder that contains no medicine. Clinical trials often compare the effectiveness of the drug being tested to the effectiveness of a placebo.
• Phase 1 Trial: A research study in which a small group of people participates in order to test a new drug or treatment for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
• Phase 2 Trial: A research study in which a larger group of people participates in order to further test the new drug or treatment to evaluate whether it is safe and effective.
• Phase 3 Trial: A research study in which a large group of people participates to confirm the drug or treatment’s effectiveness, monitor its side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
• Phase 4 Trial: A research study that typically is conducted after a drug or treatment has been approved. The purpose is to study new uses for the drug or treatment, compare the approved drug with other treatments, or study its long-term effects.