|
An Introduction to
Clinical Trials
Choosing to participate in a
clinical trial is an important personal decision. The following frequently asked
questions provide additional information about clinical trials. In addition, it
is often helpful to talk to a physician, family members, or friends about
deciding to participate in a clinical trial. If you identify a trial and you
believe that you qualify to participate, the next step is to contact the
research study team and ask any questions that you may have.
Back to Top
What is a clinical trial?
Although there are many definitions of clinical
trials, they are generally considered to be biomedical or health-related
research studies in human beings that follow a pre-defined protocol.
ClinicalTrials.gov includes both interventional and observational types of
studies. Interventional studies are those in which the research subjects are
assigned by the investigator to a treatment or other intervention, and their
outcomes are measured. Observational studies are those in which individuals are
observed and their outcomes are measured by the investigators.
Back to Top
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.
Back to Top
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.
Back to Top
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.
Back to Top
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.
Back to Top
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.
Back to Top
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.
Back to Top
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)
Back to Top
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?
Back to Top
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.
Back to Top
Does a participant continue to work with a
primary health care provider while in a trial?
Yes. Most clinical trials provide short-term
treatments related to a designated illness or condition, but do not provide
extended or complete primary health care. In addition, by having the health care
provider work with the research team, the participant can ensure that other
medications or treatments will not conflict with the
protocol.
Back to Top
Can a participant leave a clinical trial
after it has begun?
Yes. A participant can leave a clinical trial, at
any time. When withdrawing from the trial, the participant should let the
research team know about it, and the reasons for leaving the study.
Back to Top
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 an experimental treatment, its risks
and how well it may or may not work.
Back to Top
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.
Back to Top
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.
Back to Top
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.
Back to Top
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.
Back to Top
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.
Back to Top
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 an 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.
Back to Top
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.
Back to Top
See "FDA Finds New Ways to Speed
Treatments to Patients" for more details. Link to:
http://www.fda.gov/fdac/special/newdrug/speeding.html
The preceding has come directly from
ClinicalTrials.gov.
Questions, concerns, or feedback about human research at
the Washington University in St. Louis, can be directed to the Human Research
Protections Office at Washington University in St. Louis, 660 South Euclid, Box
8089, St. Louis, MO 63110; Telephone: 314-633-7400; for long-distance, call toll
free: 1-800-438-0445.
Back
to Top
|
