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Choosing to participate
in a clinical trial is an important personal decision. The following frequently
asked questions provide detailed information about clinical trials. In addition,
it is often helpful to talk to a physician, family members, or friends about
deciding to join a trial. After identifying some trial options, the next step is
to contact the study research staff and ask questions about specific trials.
Frequently asked questions about clinical trials:
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.
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.
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.
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.
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.
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, vaccines, vitamins,
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 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.
What is "expanded access"?
Expanded access is a means by which manufacturers make
investigational new drugs available, under
certain circumstances, to treat a patient(s) with a serious disease or condition
who cannot participate in a
controlled clinical trial.
Most human use of
investigational new drugs takes place in
controlled clinical trials conducted to assess the safety and
efficacy of new drugs. Data from these
trials are used to determine whether a drug is safe and effective, and serve as
the basis for the drug marketing application. Sometimes, patients do not qualify
for these controlled trials because of other health problems, age, or other
factors, or are otherwise unable to enroll in such trials (e.g., a patient may
not live sufficiently close to a clinical trial site).
For patients who cannot participate in a clinical trial of an investigational
drug, but have a serious disease or condition that may benefit from treatment
with the drug,
FDA regulations enable manufacturers of such
drugs to provide those patients access to the drug under certain situations,
known as "expanded access." For example, the drug cannot expose patients to
unreasonable risks given the severity of the disease to be treated and the
patient does not have any other satisfactory therapeutic options (e.g., an
approved drug that could be used to treat the patient's disease or condition).
The manufacturer must be willing to make the drug available for expanded access
use. The primary intent of expanded access is to provide treatment for a
patient's disease or condition, rather than to collect data about the study
drug.
Some investigational drugs are available for treatment use from pharmaceutical
manufacturers through expanded access programs listed in ClinicalTrials.gov. If
you or a loved one is interested in treatment with an investigational drug under
an expanded access protocol listed in ClinicalTrials.gov, review the protocol
eligibility criteria and inquire at the Contact Information number. If there is
not an expanded access protocol listed in ClinicalTrials.gov, you or your health
care provider may contact a manufacturer of an investigational drug directly to
ask about expanded access programs.
____________
Resources:
www.ClinicalTrials.gov
Medical review by
John Claude Krusz, PhD, MD
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© Teri Robert, 2010. Last updated February 15, 2010.
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