What Is a Clinical Trial?
One option for someone who can't hit on the right medication is participation in a test of a medication still in the development stage. Clinical trials are experiments that are designed to find out how safe and effective new treatments are in humans. They are sponsored by private parties such as medical researchers, drug developers, and medical institutions. Government agencies such as the National Institutes of Health also run clinical trials. Some are conducted in just one location and some are conducted in multiple hospitals, universities, or clinics. Some studies don't test new treatments but instead observe and document changes in a population of people. Patients who participate are closely monitored and guided by a team of researchers and health care workers.
Benefits and Risks
Why do people participate in clinical trials? One reason is to help others. By participating, subjects provide valuable scientific information that can improve medical treatments for many people. Another reason is to seek benefits that are not available from current, approved medical care.
Reasons to Join a Clinical Trial
To allow the patient to assume a different type of active role in her own treatment
To receive potentially cutting-edge treatments long before they become generally available
To help advance medical progress for others
To benefit from expert medical care at little or no cost
Sources: The National Institutes of Health and ClinicalTrials.gov
A patient who cannot take or benefit from any available antipsy-chotic drugs, for example, may want to volunteer to test a new experimental drug to see if it can help her. In this way, she may receive treatment that she would not otherwise be able to obtain.
You should consider the potential downside of clinical trials before you sign up for one. Because new and unapproved drugs are experimental, they may have unknown side effects that were not seen when they were tested on animals. In very rare cases, they have been fatal.
Of course, the experimental treatment may be perfectly safe, but it may not work for everyone. Finally, participation in a clinical trial might be less convenient than a traditional treatment that is available at a home clinic or from a psychiatrist. There is a good chance it will take more of your time. Instead of taking pills once a day and attending therapy, treatment might be more involved and require more on-site observation.
These factors vary depending on the nature and design of individual clinical trials. Be sure to know exactly what is required before agreeing to participate. Patients who participate in clinical trials adhere to treatment that is spelled out in a protocol. This is another name for the action plan the researchers developed and must follow as they conduct the trial. It states the trial's purpose and details concerning the design of the study.
Types of Clinical Trials
Clinical studies have different goals depending on what is being tested. Some trials compare two approaches for treating a disease to determine which is better. Other trials aim to identify or prevent disease in a certain population.
Many clinical trials test experimental drugs, drug combinations, or new therapies. Not all studies are treatment trials, however. Screening trials, for example, try out different ways to spot diseases in an appropriate group of people. Diagnostic trials look for improved ways to diagnose a health problem. Prevention trials seek clues that may help people avoid getting a disease or prevent it from striking again.
Clinical Trial Phases
Clinical trials are divided into four steps or levels, called phases, based on how advanced an experimental drug is in its development. You should know the phase of any trial you apply for. It may influence the benefit you can derive from the study.
Phase I trials are the first step in the long road toward approval of a new drug or treatment. These studies are designed to learn how the body handles the drug. They look for side effects at different doses and for signs of effectiveness in twenty to eighty subjects. Some Phase I trials study only people without disease, while others include patients as well.
Phase II trials look more closely at the ability of the new drug's effectiveness in 100 to 300 patients. They also track any side effects.
Phase III trials follow only if Phase II results are encouraging. They are larger trials with up to 3,000 subjects and are designed to learn more about the benefits and side effects. The results help determine what will be included on the drug's label.
Phase IV trials are conducted after the drug has been approved and marketed. Once it is released to all patients, the number of subjects who are exposed to a new drug increases far beyond the number that could be tested in the clinic. Monitoring the drug's effect in so many patients can help determine the most effective and safest doses and identify previously unseen or rarely seen side effects.
Interventional versus Observational Studies
Some clinical trials observe patients. The participants are not treated with any new or experimental drugs or procedures. They often continue to take any standard, already approved medications they normally use. The purpose of observational studies is to watch for and measure changes in medical conditions of the participants.
Investigational trials are different. They compare the effects of new drugs or treatments with either standard treatments or with placebo treatments. The control group, about half of the total number of participants, receive a placebo or their usual medication. Their outcomes are compared to those of the experimental group, which receives the new drug or treatment.
Question
What are placebos?
Placebos, sometimes called sugar pills, are look-alike medications designed to prevent subjects from knowing whether they are taking real or inactive medications. Their use is an effective way to eliminate psychological factors that can confuse results. Many people may believe they are being affected by a treatment even when the treatment has no biological action.
The well-documented placebo effect can make people react differently simply because they believe they are being treated. Placebos can make someone feel better for a while, but most of the time they don't cure or treat severe disease for long. In double-blind studies, neither the patients nor the researchers are supposed to know who receives the placebo and who gets the test substance. Only the institutional review board, a committee consisting of independent researchers, physicians, and other overseers, has the key that reveals who is getting what.
A review board is established for each trial to approve and monitor its progress. The board evaluates the purpose of the study and decides whether its potential benefits outweigh potential risks. It is charged with maximizing the safety of all participants and will step in and stop the trial if it detects any health threats. It may also stop a trial if a new drug is working so well it would be unethical to deprive the other patients in the study, the control group, who are not being treated with it.
Essential
Signing an informed consent document does not mean you are obligated to participate in a study. It simply means that you have read it and acknowledge that you know the purpose, potential benefits, and risks that come with participating in the trial. It will weaken the study if you quit without a good reason, but can leave any time you like.
Before you sign up for a study, you will get a detailed explanation of the clinical trial. A member of the clinical trial staff will go over an informed consent document with you. This is not a contract. It is a document that spells out the trial's purpose, the impact of your participation on your treatment, and the potential side effects, if any. Patient confidentiality applies in a clinical trial as it does in a standard medical practice; your name can never be released to anyone outside the staff conducting the study.
