Training and Education
Patient Education
Clinical Trials 101
What is a Clinical Trial?
A clinical trial is a research study involving human volunteers to evaluate new ways to prevent, diagnose, or treat
diseases. Clinical trials help determine if experimental
treatments are safe, effective, or better in some way than
standard treatments.
Because disease affects everyone—but not in the same
way—it is important to have people of all races, ages,
backgrounds, and genders participate in clinical trials,
so that researchers can find the best ways of preventing,
diagnosing, and treating every kind of disease for every
kind of person.
Are There Different Types of Clinical Trials? Should You Participate in a Clinical Trial?
Clinical trials have four distinct steps, or “phases” that are
designed to answer different questions:
Phase I: Is the treatment safe? These are usually
the first studies of a new drug in people. Safety is
the main concern at this point, and investigators
are trying to find the best way to give a new
treatment, as well as the highest and safest dose.
Phase II: Does the treatment work? After
determined to be safe, the new treatment must be
proven effective. These trials are typically longer
and involve more people than Phase I trials.
Phase III: Is it better than what is already
available? Typically, in this stage of large-scale
testing, several thousand participants may be
“randomized” to receive either the standard
treatment or experimental treatment. If the drug
is shown to be better than standard care, the
drug may be submitted to the U.S. Food and
Drug Administration (FDA) for approval.
Phase IV: What else do we need to know? At
this stage, investigators will ask questions about
the full effects of a new treatment, including
such issues as cost-effectiveness, long-term
effectiveness, or how a drug affects a patient’s
quality of life.
Why are Clinical Trials Important?
Clinical trials are essential to the advancement of medicine. By participating in a trial, you may have access to a
new treatment that is better than the standard treatment.
You are also helping others who may benefit from the
trial’s findings—both today and in the future.
Participation in a clinical trial is completely voluntary,
and your health-care provider can help you weigh your
options. See the SIDEBAR for some questions to help guide
your discussion with your health-care provider. Remember, no question about your care is unimportant.
Before participating in a clinical trial, you must be
provided with an Informed Consent document explaining the risks and potential benefits of the trial. Be sure to
read over this information carefully. It is important to fully
understand the purpose of the trial and what to expect.
Are Clinical Trials Safe?
Before an experimental treatment can be applied to people, it is carefully studied in the laboratory to determine
its effectiveness and safety. Clinical trials are reviewed at
both the national level (by the FDA) and at the local level
(by an institutional review board or “IRB”).
Clinical trials are conducted according to a plan or
protocol, which describes the types of patients who may
enter the study; outcomes that will be measured; and
schedules of tests and procedures, drugs, dosages, and
length of study.
Possible participants are also carefully screened – by
thorough analysis of a patient’s medical history, physical examinations, and possibly other tests – to ensure
that they are the best possible candidates for the experimental treatment. During the trial, patients are carefully
monitored to track how the treatment is affecting their
condition. Since participation in a clinical trial is voluntary, a patient can stop at any time for any reason.
Questions to Ask Your
Health-Care Provider
• What is the purpose
of the trial?
• Who can participate in this
clinical trial?
• What type of tests or
procedures will be done (i.e.,
biopsies or blood draws)?
• Do I have to pay for any of the
treatments or tests?
• Will I be able to see my own
doctor? Who will monitor my
care and safety?
• How much time is involved
in participating? Will it fit
with my work schedule and
personal life?
• How does the treatment I
would receive in this trial
compare with other treatment
choices?
• How will the treatment and its
possible side effects affect my
daily life?
• Who can I speak with about
questions I have during and
after the trial? +