A wide range of people patriciate in clinical trials, for many different reasons. Although some participate after they’ve exhausted all approved treatment options, others join to test new combinations of drugs, new surgical procedures, or so researchers can look at other areas of care, like improving quality of life. Many healthy volunteers also participate in clinical trials!
New drugs and treatments need to be studied in children in the same way they need to be studied in adults. Clinical trials in children help to gather important information on safety, efficacy, and dosing. Children are enrolled in clinical trials with the consent of their parent or guardian.
There is a risk of experiencing serious adverse side effects when participating in a clinical trial, but several safeguards are in place to make every clinical trial as safe as possible. An Institutional Review Board made up of doctors, scientists, and laypeople must approve all clinical trials in the United States and are dedicated to ensuring participants are not exposed to unnecessary risks. Before joining a clinical trial, potential participants must undergo the informed consent process where they are made aware of the risks, benefits, and alternatives to participating.
Depending on the type of study, participants may be randomly assigned to a test group receiving the experimental treatment or a control group receiving a placebo/standard care. This allows researchers to compare the results from the two groups and determine if they happened by chance or because of the intervention. Patients who are randomly assigned to a placebo (often referred to as the control group), cannot be more likely to die or experience substantial harms or severe discomfort, than those in the treatment group. Furthermore, patients who receive a placebo at the beginning of a trial may have the opportunity to receive the study drug at a later time.
Even after providing Informed Consent and starting treatment, a participant can withdraw at any time, for any reason.
When participating in a clinical trial, most patients won’t have an increase in their typical cost of care. Generally, a participant’s health insurance will cover charges related to tests and treatments for standard of care, and participants are often not billed for things done solely for research purposes. A section of the informed consent will breakdown what costs patients can expect.
Doctors are not always aware of all clinical trials, so they may not know if a patient is potentially eligible for one. Patients can learn about current clinical trials at www.clinicaltrials.gov.
After undergoing pre-clinical research to determine if a new drug or treatment has the potential to improve the care of patients, it enters into clinical trials. Clinical trials assess the safety and efficacy of a drug or treatment in people.
Phase 1 trials are primarily done to test a treatment of drug’s safety. A small group of 15 to 30 patients are given a new drug or treatment to help researchers identify side effects and determine a safe dosing range. If the treatment or drug is deemed safe, it can be tested in a Phase 2 trial.
In Phase 2 trials a larger group of patients are given the treatment. Researchers use this phase to further evaluate safety and determine if the treatment is effective. The new drug or treatment is not usually compared to the standard-of-care treatment at this point in the process. If the treatment is found to be effective, it can be tested in a Phase 3 trial.
Phase 3 trials enroll a large number of patients—usually 100 or more. Researchers continue to monitor treatment safety and efficacy and compare the new treatment or drug to the standard-of-care treatment to assess which works better. If one treatment is found to have much better results, the study will be stopped early.
Phase 4 trials take place after a treatment or drug is approved by the U.S. Food and Drug Administration. By testing the treatment in larger groups of patients, researchers can gather additional information on safety and side effects as well as how well it works and how it can best be used.