Clinical trials are
research studies in which people help doctors find ways to
improve health care. Each study tries to
find better ways to prevent, diagnose, or treat health problems.
purpose of a clinical trial is to find out whether a medicine or treatment
is safe and effective for treating a certain condition or
disease. Clinical trials compare the effectiveness of this medicine or
treatment against standard, accepted treatment or against a
placebo if there is no standard treatment.
Taking part in a clinical trial
is voluntary. No one can make you participate. If you choose not to take part, you will be offered the standard
treatment for your health problem. You can talk to your doctor if you have questions about clinical trials.
Why are they important?
are important because they compare new treatments with accepted treatments.
They allow researchers to find out if a new treatment works as well as or better than
accepted treatments. The new treatment might have fewer or less serious side effects.
The new treatment also might not work as well or might cause more
side effects than standard treatments.
Clinical trials help drug companies make medicines that are safer and
more effective with fewer side effects. Clinical trials also help these
companies decide whether it is worthwhile to seek approval from the U.S. Food
and Drug Administration (FDA) for a certain medicine. If a medicine doesn't
work as well as standard treatment, then the FDA isn't likely to approve
Clinical trials are also important in finding treatments if no
standard treatment exists.
Taking part in a clinical trial may
not benefit you directly. But in the future it may help other people who have the
How do clinical trials work?
Your doctor will help
you find out if you are eligible to take part in a clinical trial. The
company sponsoring the trial will have a very strict set of standards, or criteria, that all
participants must meet.
If you meet the criteria, you may be "randomized" to
get either the new medicine, a medicine that is considered standard therapy, or a
placebo. That means that a computer is used to randomly assign you to one of
the treatments. In many studies, neither you nor your doctor knows which treatment you are
getting. But not all clinical trials randomize people. And if you have a
serious disease, such as cancer, you will not be given only a placebo, unless no
effective treatment is known.
After you are accepted by the clinical trial and you give your
consent to take part:
You will be given a structured program to follow.
will have a schedule of tests, doctor appointments, and treatments.
be asked to keep a diary of your experience during this time.
Doctors, nurses, social workers, and
other health professionals may be part of your treatment team.
Be sure to carefully follow instructions. If you don't
know what you are supposed to do next, call your doctor. Or call the person
responsible for your trial.
medicine or treatment must go through three phases before it is
approved for use by the U.S. Food and Drug Administration (FDA).
What happens when the clinical trial is finished?
After a clinical trial is completed and the results are studied,
the FDA decides whether to approve continued development of the medicine. If
the medicine that you received remains in development, you may be able to
get more doses as an extension of the study.
results of the clinical trial show that the new medicine or combination of
medicines works much better than standard treatment, the new medicine may
become available to the general public.
team may continue to check on you after your trial is over.
What are the risks?
You should be fully informed about the possible
risks of the trial before you agree to participate.
Although the purpose of trials is to find new and better treatments, the new treatment may not work as well as
You may have unpleasant, serious, or
even life-threatening side effects from the treatment.
treatment may not work for you.
The trial may require more of your
time than standard treatment. You may have to:
Make more trips to the study
Have more treatments.
Receive your treatment in a
Take more medicine more often or at very specific times.
Keep a written diary of your experience.
How is your safety protected?
Every clinical trial
in the United States must be approved and monitored by an
institutional review board (IRB) to make sure that the
risks are as low as possible and are worth any potential benefits.
The ethical and legal rules for medical practice also apply to
clinical trials. Most clinical research is regulated by the U.S. government,
with specific rules to protect the participants. Clinical trials follow a
carefully controlled study plan (protocol) that explains what everyone will do
in the study. During the clinical trial, researchers report the results of the
trial at scientific meetings, to medical journals, and to government agencies.
Your name will remain secret and will not be mentioned in these reports.
Who pays for clinical trials?
Sometimes the group sponsoring your trial will be responsible for the
cost of the medicine as well as the costs of medical tests that are required
while you are in the trial. (Clinical trials usually require you to have more
medical tests than you would have if you were not in the trial.) But in other
trials, the cost of the medicine and only some of the medical tests will be
covered. Some studies will reimburse you for the cost of traveling to and from
your medical visits.
How can you find out about clinical trials?
National Institutes of Health, through its National Library of Medicine, has
developed ClinicalTrials.gov to provide information about clinical
research studies to patients, family members, and members of the public. You
can contact this service on the Internet at www.ClinicalTrials.gov. Or you can
get information over the phone by calling 1-888-346-3656 or (301) 594-5983.
There may or may not be a clinical trial available in your area that relates to
your particular disease or stage of disease.
How this information was developed to help you make better health decisions.