A
clinical trial is a research study designed to determine whether
new drugs or treatments are both safe and effective. Carefully conducted
clinical trials are the fastest and safest way to find treatments
that work in people. Clinical trials are conducted in phases. The
trials at each phase have a different purpose and help scientists
answer different questions:
Phase
I Trials - researchers test a new drug or treatment in a small
group (20-80) for the first time to evaluate its safety, determine
a safe dosage range, and identify side effects.
Phase
II Trials - expands the study to a larger group of people (100-300)
to see if it is effective and to further evaluate its safety.
Phase
III Trials - expands the study to large groups of people (1,000-3,000)
to confirm its effectiveness, monitor side effects, compare
it to commonly used treatments, and collect information that
will allow the drug or treatment to be used safely.
Phase
IV Trials (Post-Marketing Studies) - takes place after the drug
or treatment has been licensed and marketed to gather additional
information including the drug's risks, benefits, and optimal
use.
Who
can participate in a clinical trial?
All
clinical trials have guidelines about who can participate. Using
inclusion/exclusion criteria is an important principle of medical
research that helps to produce reliable results. The factors that
allow someone to participate in a clinical trial are called "inclusion
criteria" and those that disallow someone from participating
are called "exclusion criteria". These criteria are based
on such factors as age, gender, the type and stage of a disease,
previous treatment history, and other medical conditions. Before
joining a clinical trial, a participant must qualify for the study.
Some research studies seek participants with illnesses or conditions
to be studied in the clinical trial, while others need healthy participants.
It is important to note that inclusion and exclusion criteria are
not used to reject people personally. Instead, the criteria are
used to identify appropriate participants and keep them safe. The
criteria help ensure that researchers will be able to answer the
questions they plan to study.
Why
would someone want to participate in a clinical trial?
People
take part in clinical trials for many reasons. Participants in clinical
trials can play a more active role in their own health care, gain
access to new research treatments before they are widely available,
and help others by contributing to medical research.
Although
remarkable progress has been made in defining and treating mental
illnesses, some treatments are not effective for all persons or
may have significant side effects. Thus, most people who agree to
take part in studies of mental illness hope the research will produce
knowledge about the disease itself — for example, the role
of genetics in illness — or about treatments that will benefit
them directly.
How
a treatment will work for a patient in a trial can't be known ahead
of time. Even standard treatments, although effective in many patients,
do not carry sure benefits for everyone. But patients should choose
if they want to take part in a study or not, only after they understand
both the possible risks and benefits.
How
is the safety of the participant protected?
Many
"checkpoints" ensure that clinical trials meet strict
scientific guidelines and follow Food and Drug Administration (FDA)
regulations designed to protect the subject. Several groups who
are not part of the research team examine both the scientific plan
and procedures to protect the interests of participants before an
investigator may begin the research.
Each
proposed study, including its provisions for the protection of human
subjects and its consent form, must be approved by an Institutional
Review Board (IRB). Every organization that conducts research, for
example a university or hospital, must have an IRB. The membership
of these boards includes scientists, persons who are not scientific
experts, and at least one "public" member who is not associated
with the organization.
An
important IRB responsibility is to review the informed consent materials
that an investigator develops for those who take part in the study.
This information allows the IRB and — more critically —
you to judge the value, risks, and potential benefits of a research
project. If an IRB has concerns about any part of the research proposal,
the committee will tell the director of the study. The researcher
must attend to these concerns before submitting the research proposal
to a funding agency.
A
funding agency, such as the National Institute of Mental Health
(NIMH), provides the next review of human subject provisions for
clinical research proposals. The funding agency also judges the
scientific importance of a research proposal, and how the researcher
will learn from it. Both the IRB and the funding agency conduct
regular reviews to be sure that the researchers are meeting all
the rules for the protection of human subjects.
What
is a protocol?
A
protocol is a study plan on which a clinical trial is based. The
plan is carefully designed to safeguard the health of the participants
as well as answer specific research questions. A protocol describes
what types of people may participate in the trial; the schedule
of tests, procedures, medications, and dosages; and the length of
the study. While in a clinical trial, participants following a protocol
are seen regularly by the research staff to monitor their health
and to determine the safety and effectiveness of their treatment.
What
is informed consent?
To
help you decide if you want to be a volunteer in a study, FDA requires
that you be given complete information about the study before you
agree to take part. This is known as informed consent. FDA requires
that volunteers be told:
that the study involves research of an unproven drug or device
the
purpose of the research
how
long the study is expected to take
what
will go on in the study and which parts of the study are experimental
possible
risks or discomforts
possible
benefits
other
procedures or treatments that you might want to consider instead
of the treatment being studied
that
FDA may inspect study records, but the records will be kept confidential
whether
any medical treatments are available if you are hurt, what those
treatments are, where they can be found, and who will pay for
the treatment
the
person to contact with questions about the study, your rights,
and injuries related to research
being
in the study is voluntary and you can quit at any time.
Informed consent information should be written so you can understand
it. If you don't, be sure to ask the doctor or other medical person
to explain it. Make sure you understand all of it before you agree
to be in the study.
Before
you can participate in the trial, you must sign the informed consent
form, showing that you have been given this information and understand
it. The informed consent form is NOT a contract and you can leave
the study at any time, for any reason.
Informed
consent is not a one-time event, but a continuing process. Throughout
a study, the research team must continue to provide information
about participating in the study. They must respond to any questions
you have about the research and inform you if any new risks are
identified. You may, at any time, reappraise your decision to take
part in the project and withdraw your consent. It is advisable to
discuss any concerns with the director of the study.
What
are the potential risks and benefits of clinical trials?
Potential
benefits include:
Health
care provided by leading physicians in the field of mental health
research.
Access to new drugs and interventions before they are widely available.
Close monitoring of your health care and any side effects.
A more active role in your own health care.
If the approach being studied is found to be helpful, you may
be among the first to benefit.
An opportunity to make a valuable contribution to mental health
research.
The potential risks include:
New drugs and procedures may have side effects or risks unknown
to the doctors.
New
drugs and procedures may be ineffective, or less effective, than
current approaches.
Even if a new approach has benefits, it may not work for you.
The
protocol may require more time and attention than would a non-protocol
treatment, including trips to the study site, more treatments,
hospital stays or complex dosage requirements
What
should people consider before participating in a trial?
People
should know as much as possible about the clinical trial and feel
comfortable asking the members of the health care team questions
about it, the care expected while in a trial, and the cost of the
trial. The following questions might be helpful for the participant
to discuss with the health care team. Some of the answers to these
questions are found in the informed consent document.
What
is the purpose of the study? What is the study trying to find
out?
What kinds of test and exams will I have to take while I'm in
the study? How much time do these take? What is involved in each
test?
How
do the possible risks, side effects, and benefits in the study
compare with my current treatment?
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?
How
long will the study last?
What follow-up will there be?
What
will happen at the end of the study?
What
are my other treatment choices? How do they compare with the treatment
being studied?