In order to allow a new medicine or device to be given to many patients, it must first be tested to be sure that it is safe and works. Clinical trials are used to test the effect of a particular treatment in a strictly scientifically-controlled way.
Clinical trials are carefully evaluated by designated ethical committees and State drug regulatory agencies to ensure that the trial provides a valid answer to the question it is supposed to answer (usually whether the treatment works without too many side effects). Ethical committees will also check that the trial is performed according to Good Clinical Practice (that is, a number of internationally accepted rules designed to protect people involved in a trial). Usually an independent Data Safety Monitoring Committee is established that continually receives reports on events during the entire trial. This Committee has as its primary task protection of the participating patients during the trial.
Safer and more effective drugs are continuously being developed; clinical trials in real patients are needed to demonstrate these benefits before the regulatory authorities can judge a medicine to be safe and effective and give it a licence for use in humans. If you decide to participate in a clinical trial, you will be assisting with the research of new drugs developed to be safer and more effective than existing therapies.
Physicians involved in clinical trials are often especially interested in the condition being studied. They will ensure your treatment is explained before entering the trial. Investigators and research nurses should also be readily available to you if your symptoms worsen. Often, you may get access to a treatment years before it would be available for routine management.
If your doctor considers you suitable to take part in a clinical trial, he/she will explain the purpose and what the clinical trial will involve. A thorough physical examination and medical history review will be needed to make sure you are suitable for the clinical trial in question. If you are suitable, you will be assigned a treatment being tested and given a treatment plan to follow. You may also be asked to complete a diary to note any changes in your health, and you may be asked to return to your clinic for regular assessments. These extra follow-up visits frequently serve to improve your long-term care..
Your primary care physician/GP will be involved and informed when you enter the study and will receive a written summary at the end of the trial.
When two drugs are being compared, the clinical trial may be blinded; this means that either you or your doctor (or both - known as double- blind) will not know which specific medication you are on. This is to eliminate any bias that may result from knowing that you are/are not receiving the drug being investigated by the trial. The Data Safety Monitoring Committee is unblinded throughout the trial to assure patient safety.
You may also be given an inactive agent, known as a placebo, or dummy pill. This will be used to see if there is any effect of the new treatment compared to standard treatment.