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What is a clinical trial?
A clinical trial (also clinical research) is a research study in human
volunteers to answer specific health questions. Carefully conducted clinical
trials are the fastest and safest way to find treatments that work in people and
ways to improve health. Interventional trials determine whether experimental
treatments or new ways of using known therapies are safe and effective under
controlled environments. Observational trials address health issues in large
groups of people or populations in natural settings.
Why participate in a clinical
trial?
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
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.
What happens during a clinical
trial?
The clinical
trial process depends on the kind of trial being conducted (See
What are the different types of clinical trials?)
The clinical trial team includes doctors and nurses as well as social workers
and other health care professionals. They check the health of the participant at
the beginning of the trial, give specific instructions for participating in the
trial, monitor the participant carefully during the trial, and stay in touch
after the trial is completed.
Some clinical
trials involve more tests and doctor visits than the participant would normally
have for an illness or condition. For all types of trials, the participant works
with a research team. Clinical trial participation is most successful when the
protocol is carefully followed and there is frequent contact with the
research staff.
What is informed consent?
Informed
consent is the process of learning the key facts about a clinical trial before
deciding whether or not to participate. It is also a continuing process
throughout the study to provide information for participants. To help someone
decide whether or not to participate, the doctors and nurses involved in the
trial explain the details of the study. If the participant's native language is
not English, translation assistance can be provided. Then the research team
provides an informed consent document that
includes details about the study, such as its purpose, duration, required
procedures, and key contacts. Risks and potential benefits are explained in the
informed consent document. The participant then decides whether or not to sign
the document. Informed consent is not a contract, and the participant may
withdraw from the trial at any time.
What are the benefits and risks of participating in a clinical trial?
Benefits
Clinical trials
that are well-designed and well-executed are the best approach for eligible
participants to:
·
Play an
active role in their own health care.
·
Gain
access to new research treatments before they are widely available.
·
Obtain
expert medical care at leading health care facilities during the trial.
·
Help
others by contributing to medical research.
Risks
There are risks
to clinical trials.
·
There
may be unpleasant, serious or even life-threatening side effects to experimental
treatment.
·
The
experimental treatment may not be effective for the participant.
·
The protocol may require more of their
time and attention than would a non-protocol treatment, including trips to the
study site, more treatments, hospital stays or complex dosage requirements.
What
are side effects and adverse reactions?
Side effects are any undesired actions or effects of the
experimental drug or treatment. Negative or adverse effects
may include headache, nausea, hair loss, skin irritation, or
other physical problems. Experimental treatments must be
evaluated for both immediate and long-term side effects.
How
is the safety of the participant protected?
The ethical and legal codes that govern medical practice
also apply to clinical trials. In addition, most clinical
research is federally regulated with built in safeguards to
protect the participants. The trial follows a carefully
controlled protocol, a study plan which details what
researchers will do in the study. As a clinical trial
progresses, researchers report the results of the trial at
scientific meetings, to medical journals, and to various
government agencies. Individual participants' names will
remain secret and will not be mentioned in these reports
(See Confidentiality
Regarding Trial Participants).
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?
·
Who is going to be in the study?
·
Why do researchers believe the experimental treatment being
tested may be effective? Has it been tested before?
·
What kinds of tests and experimental treatments are
involved?
·
How do the possible risks, side effects, and benefits in the
study compare with my current treatment?
·
How might this trial affect my daily life?
·
How long will the trial last?
·
Will hospitalization be required?
·
Who will pay for the experimental treatment?
·
Will I be reimbursed for other expenses?
·
What type of long-term follow up care is part of this study?
·
How will I know that the experimental treatment is working?
Will results of the trials be provided to me?
·
Who will be in charge of my care?
What kind of preparation should a potential participant make
for the meeting with the research coordinator or doctor?
·
Plan ahead and write down possible questions to ask.
·
Ask a friend or relative to come along for support and to
hear the responses to the questions.
·
Bring a tape recorder to record the discussion to replay
later.
Every clinical trial in the U.S. must be approved and
monitored by an Institutional
Review Board (IRB) to make sure the risks are as low
as possible and are worth any potential benefits. An IRB is
an independent committee of physicians, statisticians,
community advocates, and others that ensures that a clinical
trial is ethical and the rights of study participants are
protected. All institutions that conduct or support
biomedical research involving people must, by federal
regulation, have an IRB that initially approves and
periodically reviews the research.
Where do
the ideas for trials come from?
Ideas for clinical trials usually come from researchers.
After researchers test new therapies or procedures in the
laboratory and in animal studies, the experimental
treatments with the most promising laboratory results are
moved into clinical trials. During a trial, more and more
information is gained about a experimental treatment, its
risks and how well it may or may not work.
Who sponsors clinical
trials?
Clinical trials are sponsored or funded by a variety of
organizations or individuals such as physicians, medical
institutions, foundations, voluntary groups, and
pharmaceutical companies, in addition to federal agencies
such as the National Institutes of Health (NIH), the
Department of Defense (DOD), and the Department of Veteran's
Affairs (VA). Trials can take place in a variety of
locations, such as hospitals, universities, doctors'
offices, or community clinics.
What is a protocol?
A protocol is a study plan on which all clinical trials are
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 a placebo?
A placebo is an inactive pill, liquid, or powder that has no
treatment value. In clinical trials, experimental treatments
are often compared with placebos to assess the experimental
treatment's effectiveness. In some studies, the participants
in the control group
will receive a placebo instead of an active drug or
experimental treatment.
What is a
control or control group?
A control is the standard by which experimental observations
are evaluated. In many clinical trials, one group of
patients will be given an experimental drug or treatment,
while the control group is given either a standard treatment
for the illness or a placebo.
What are the different types of clinical trials?
Treatment trials test
experimental treatments, new combinations of drugs, or new
approaches to surgery or radiation therapy.
Prevention trials look
for better ways to prevent disease in people who have never
had the disease or to prevent a disease from returning.
These approaches may include medicines, vitamins, vaccines,
minerals, or lifestyle changes.
Diagnostic trials are
conducted to find better tests or procedures for diagnosing
a particular disease or condition.
Screening trials test
the best way to detect certain diseases or health
conditions.
Quality of Life trials
(or Supportive Care trials) explore ways to improve comfort
and the quality of life for individuals with a chronic
illness.
What are
the phases of clinical trials?
Clinical trials are conducted in phases. The trials at each
phase have a different purpose and help scientists answer
different questions:
In Phase I trials,
researchers test a experimental drug or treatment in a small
group of people (20-80) for the first time to evaluate its
safety, determine a safe dosage range, and identify side
effects.
In Phase II trials,
the experimental study drug or treatment is given to a
larger group of people (100-300) to see if it is effective
and to further evaluate its safety.
In Phase III trials,
the experimental study drug or treatment is given 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
experimental drug or treatment to be used safely.
In Phase IV trials,
post marketing studies delineate additional information
including the drug's risks, benefits, and optimal use.
What is an
"expanded access" protocol?
Most human use of
investigational new drugs takes place in
controlled clinical trials
conducted to assess safety and
efficacy of new drugs.
Data from the trials can serve as the basis for the drug
marketing application. Sometimes, patients do not qualify
for these carefully-controlled trials because of other
health problems, age, or other factors. For patients who may
benefit from the drug use but don't qualify for the trials,
FDA regulations enable
manufacturers of investigational new drugs to provide for
"expanded access" use of the drug. For example, a treatment
IND (Investigational New Drug application) or treatment
protocol is a relatively unrestricted study. The primary
intent of a treatment IND/protocol is to provide for access
to the new drug for people with a life-threatening or
serious disease for which there is no good alternative
treatment. A secondary purpose for a treatment IND/protocol
is to generate additional information about the drug,
especially its safety. Expanded access protocols can be
undertaken only if clinical investigators are actively
studying the experimental treatment in well-controlled
studies, or all studies have been completed. There must be
evidence that the drug may be an effective treatment in
patients like those to be treated under the protocol. The
drug cannot expose patients to unreasonable risks given the
severity of the disease to be treated.
Some investigational drugs are available from pharmaceutical
manufacturers through expanded access programs listed in
ClinicalTrials.gov.
Expanded access protocols are generally managed by the
manufacturer, with the investigational treatment
administered by researchers or doctors in office-based
practice. If you or a loved one are interested in treatment
with an investigational drug under an expanded access
protocol listed in
ClinicalTrials.gov,
review the protocol
eligibility criteria and location information and
inquire at the Contact Information number.
See "FDA Finds New Ways to Speed Treatments to Patients" for
more details. Link to:
http://www.fda.gov/fdac/special/newdrug/speeding.html
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