A clinical trial is a scientific study that seeks to answer important medical questions about a new investigational treatment:
- To see if it relieves symptoms
- To see if it improves a person’s health
- To see if it works better than other alternative treatments
- To see if it is safe and effective
Clinical trials enable patients around the world to benefit from the earliest possible access to cutting-edge medicines and technologies. In some clinical trials, a new drug or treatment is compared with the best-known standard therapy to see if it more effective or causes fewer side effects, whereas other studies look to determine whether an existing drug or treatment could be used in a new way. All clinical trials are conducted according to strict rules set by the Food and Drug Administration (FDA), which ensure that people who volunteer to be in the studies are treated as safely as possible.
Studies are done in four phases. Each phase is conducted to answer different questions. Phase I studies involve testing the treatment on a limited number of healthy people (20-80 people) to determine the treatment’s basic safety. Phase II studies involve testing the treatment on a small number of affected patients to determine effectiveness and the best starting dose. Phase III studies involve testing the treatment on a large number of affected patients to see if the treatment works as well as the standard or current treatment. These phases generally take many years. Phase IV studies occur after a drug is approved by the FDA and involve post-marketing surveillance and cost-benefit analysis.
A placebo is an inactive medication that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the true benefit or harm of the treatment.
Each study is different, but the benefits of participating may include any of the following:
- An opportunity to receive a medical treatment that is not widely available
- Access to quality medical care from doctors who specialize in the condition being studied
- A chance to advance medical knowledge
- The satisfaction of helping others who may be suffering from a chronic, serious or life-threatening illness
Some treatments that are tested might have known side effects that could be unpleasant, serious or even life-threatening, and some may have unanticipated side effects. Because the treatments being studied are new, doctors don’t always know what the side effects could be.
Some side effects are temporary and go away when the treatment is stopped, while others may last longer or, although rare, may be permanent. Some side effects may appear during treatment, while other may not show up until after the treatment is over. The risks of each trial depend on the medical condition and treatment under studied. All known risks will be fully explained to you by the researchers prior to volunteering.
The ethical and legal codes that govern medical practice also apply to clinical research. In addition, clinical research is federally regulated with built-in safeguards to protect the participants. Clinical trials follow carefully controlled protocols, or study plans, which detail what researchers will do in the study. Individual participant names and the personal health information of the participants will be kept confidential.
If your doctor has determined that you qualify to participate in the study and you agree to take part, your doctor will discuss the details of the study with you and answer any questions you might have. Paperwork will be completed to show that you have been informed of all aspects of the study and that you have agreed to participate.
To help you decide if you want to be in a study, the FDA requires that you be given complete information about the study before you agree to take part. This is known as informed consent. The FDA require that people be told:
- That the study involves research of an unproven drug or device
- The purpose of the research
- How long the study will take
- What will happen in the study and which parts of the study are experimental
- Possible risks and/or discomforts
- Possible benefits
- Alternative treatments that you may want to consider instead of the treatment being studied
- That the FDA may look at your study records, but that records will be kept confidential
- Whether any medical treatment are available if you are hurt, what those treatments are and who will pay for the treatment
- The person to contact with questions about the study and your rights as a participant
- That you have the option to quit at any time
Here are some questions to ask your doctor to help you decide if you want to take part in a clinical trial:
- What is the study trying to find out?
- What kind of tests and exams will I have to take while I’m in the study?
- How much time do these take?
- How often does the study require me to go to the doctor or clinic?
- Will I be hospitalized? If so, how often and for how long?
- What are the costs to me?
- Will my health insurance pay for it?
- What follow up will there be?
- How long will the study last?
- What will happen at the end of the study?
- What are my other treatment choices?
- How do they compare to the treatment being studied?
- What side effects can I expect from the treatment being tested?
- How do they compare with side effects of standard treatment?
Once in the study, you will be asked to follow the treatment plan and to attend your scheduled clinic visits. It is very important that you follow the study instructions. Depending on the study, the length of your participation will vary. If you choose, you may leave the study at any time without your usual health care being affected.
No. Any interested individuals that meet the established study criteria may participate.