Participating in a Clinical Study
Why is participation in Clinical Trials important?
Participation in clinical trials has become vital in contributing to medical research by allowing participants to assist in their own health care, gain access to new research treatments before they are widely available, and help others through the advancement of research.
What is a Clinical Trial?
A clinical trial is an evaluation of an investigational medication to determine if the medication is sufficiently safe and well tolerated so that further study is warranted to test its effectiveness in participants.
Before an investigational medication is used in a clinical trial, it must have been extensively studied in animals. Once a drug has been determined to be reasonably safe in animals, evaluations can then be conducted in healthy human volunteers. In these studies, a small number of healthy volunteers are given an experimental drug or treatment for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. Once safety and tolerability have been established in healthy individuals, the drug goes into further trials in individuals with the illness the drug was designed to treat.
In such a trial, participants with an illness are treated with either the investigational medication or a comparison agent which may be either a placebo (inactive substance) or a marketed medication known to be effective in treating the condition being studied. The purpose is to establish whether the investigational medication is better than placebo treatment and is either as good as or better than existing treatments in terms of effectiveness, tolerability and safety.
The results from such formal treatment studies are submitted to the Food and Drug Administration (FDA) for a decision as to whether the investigational medication should be approved for general release. Until that decision is made, the investigational medication can only be received in a clinical trial. Research physicians and clinical staff working in university medical centers, hospitals, or research clinics such as CRI, conduct such treatment studies.
How Do Trials Work?
Clinical trials are conducted according to a protocol, which is a plan of action developed by the researchers and approved by the FDA. The protocol defines who will be enrolled, how the investigational medication will be administered and how its effects will be monitored.
Each protocol contains criteria for selecting participants. For example, some research studies only accept participants who are in a particular stage of an illness. A protocol will also specify the age and sex of participants to be enrolled. It may exclude potential participants with certain medical conditions, such as kidney disease or heart conditions. Other medications taken while in a study are carefully controlled and restricted. Such restrictions are to protect potential participants from harmful effects which might occur when different medications are taken at the same time.
Clinical trials are often “randomized” and “blinded”. In randomized studies, participants are randomly assigned to a treatment group which may get the investigational medication, placebo or comparative medication. In blinded studies, neither the researcher nor the participant know which treatment group they are assigned to. This approach is taken to ensure that the researchers remain unbiased to the possible effects of the investigational medication when assessing participants. In the event that the researchers need to know what the participants have received, the Clinical Research Institute has access to the necessary codes, but such “breaking of the blind” is rarely needed and hence rarely done.
