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Saturday, May 17, 2008
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Taking
Part in Clinical Trials: What Cancer Patients Need to
Know
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Additonal Resources for Cancer Information
Glossary
Purpose of This
Article
This article is for people with cancer,
their families, and others who care about them.
It is divided into three sections. Each section
answers questions many people have about clinical
trials:
- 1.
What are clinical trials?
- 2.
What happens in a clinical trial?
- 3.
Should I take part in a clinical trial?
The first two sections provide background information
on the important role clinical trials play in improving
cancer care. They also explain some of the technical
terms you may hear from your doctor or nurse. Words
that appear in bold are defined in the Glossary. The third section of the
booklet is designed to help you answer question 3
for yourself. It raises issues to think about as you
decide whether a clinical trial is right for you.
For example, what are the pros and cons of being in
a clinical trial from the patient's point of view?
This section also lists some questions to ask the
doctor or nurse about any study you are considering.
The resources section lists other sources of information
about cancer and treatment studies.
This booklet is a part of the patient education program
of the National Cancer Institute (NCI). The NCI sponsors,
conducts, and oversees clinical trials and other cancer
research, and provides research-based information
to health professionals, patients, and the public.
What Are Clinical Trials?
Clinical trials, also called cancer treatment
or research studies, test new treatments in people
with cancer. The goal of this research is to find
better ways to treat cancer and help cancer patients.
Clinical trials test many types of treatment such
as new drugs, new approaches to surgery or radiation
therapy, new combinations of treatments, or new methods
such as gene therapy.
A clinical trial is one of the final stages of a long
and careful cancer research process. The search for
new treatments begins in the laboratory, where scientists
first develop and test new ideas. If an approach seems
promising, the next step may be testing a treatment
in animals to see how it affects cancer in a living
being and whether it has harmful effects. Of course,
treatments that work well in the lab or in animals
do not always work well in people. Studies are done
with cancer patients to find out whether promising
treatments are safe and effective.
Why Are Clinical
Trials Important?
Clinical trials are important in two ways.
First, cancer affects us all, whether we have it,
care about someone who does, or worry about getting
it in the future. Clinical trials contribute to knowledge
and progress against cancer. If a new treatment proves
effective in a study, it may become a new standard
treatment that can help many patients. Many of
today's most effective standard treatments are based
on previous study results. Examples include treatments
for breast, colon, rectal, and childhood cancers.
Clinical trials may also answer important scientific
questions and suggest future research directions.
Because of progress made through clinical trials,
many people treated for cancer are now living longer.
Second, the patients who take part may be helped personally
by the treatment(s) they receive. They get up-to-date
care from cancer experts, and they receive either
a new treatment being tested or the best available
standard treatment for their cancer. Of course, there
is no guarantee that a new treatment being tested
or a standard treatment will produce good results.
New treatments also may have unknown risks. But if
a new treatment proves effective or more effective
than standard treatment, study patients who receive
it may be among the first to benefit. Some patients
receive only standard treatment and benefit from it.
In the past, clinical trials were sometimes seen as
a last resort for people who had no other treatment
choices. Today, patients with common cancers often
choose to receive their first treatment in a clinical
trial.
What Happens in a
Clinical Trial?
In a clinical trial, patients receive treatment and
doctors carry out research on how the treatment affects
the patients. While clinical trials have risks for
the people who take part, each study also takes steps
to protect patients.
What Is It Like To
Receive Treatment in a Study?
When you take part in a clinical trial, you receive
your treatment in a cancer center, hospital, clinic,
and/or doctor's office. Doctors, nurses, social workers,
and other health professionals may be part of your
treatment team. They will follow your progress closely.
You may have more tests and doctor visits than you
would if you were not taking part in a study. You
will follow a treatment plan your doctor prescribes,
and you may also have other responsibilities such
as keeping a log or filling out forms about your health.
Some studies continue to check on patients even after
their treatment is over.
How Is the Research
Carried Out? How Are Patients Protected?
In clinical trials, both research concerns and patient
well-being are important. To help protect patients
and produce sound results, research with people is
carried out according to strict scientific and ethical
principles. These include:
1. Each clinical trial has an action plan (protocol)
that explains how it will work.
The study's investigator, usually a doctor,
prepares an action plan for the study. Known as a
protocol, this plan explains what will be done
in the study and why. It outlines how many people
will take part in the study, what medical tests they
will receive and how often, and the treatment plan.
The same protocol is used by each doctor that takes
part.
For patient safety, each protocol must be approved
by the organization that sponsors the study (such
as the National Cancer Institute) and the Institutional
Review Board (IRB) at each hospital or other study
site. This board, which includes consumers, clergy,
and health professionals, reviews the protocol to
try to be sure that the research will not expose patients
to extreme or unethical risks.
2. Each study enrolls people who are alike in
key ways.
Each study's protocol describes the characteristics
that all patients in the study must have. Called eligibility
criteria, these guidelines differ from study to study,
depending on the research purpose. They may include
age, gender, the type and stage of cancer,
and whether cancer patients who have had prior cancer
treatment or who have other health problems can take
part.
Using eligibility criteria is an important principle
of medical research that helps produce reliable results.
During a study, they help protect patient safety,
so that people who are likely to be harmed by study
drugs or other treatments are not exposed to the risk.
After results are in, they also help doctors know
which patient groups will benefit if the new treatment
being studied is proven to work. For instance, a new
treatment may work for one type of cancer but not
for another, or it may be more effective for men than
women.
3. Cancer clinical trials include research at
three different phases. Each phase answers different
questions about the new treatment.
- Phase
I trials are the first step in testing a new
treatment in humans. In these studies, researchers
look for the best way to give a new treatment
(e.g., by mouth, IV drip, or injection? how many
times a day?). They also try to find out if and
how the treatment can be given safely (e.g., best
dose?); and they watch for any harmful side
effects. Because less is known about the possible
risks and benefits in Phase I, these studies usually
include only a limited number of patients who
would not be helped by other known treatments.
- Phase
II trials focus on learning whether the new
treatment has an anticancer effect (e.g., Does
it shrink a tumor? improve blood test results?).
As in Phase I, only a small number of people take
part because of the risks and unknowns involved.
- Phase
III trials compare the results of people taking
the new treatment with results of people taking
standard treatment (e.g., Which group has better
survival rates? fewer side effects?). In most
cases, studies move into Phase III testing only
after a treatment shows promise in Phases I and
II. Phase III trials may include hundreds of people
around the country.
4. In Phase III trials, people are assigned at
random to receive either the new treatment or standard
treatment.
Researchers assign patients by chance either to a
group taking the new treatment (called the treatment
group) or to a group taking standard treatment
(called the control group). This method, called
randomization, helps avoid bias: having
the study's results affected by human choices or other
factors not related to the treatments being tested.
In some studies, researchers do not tell the patient
whether he or she is in the treatment or control group
(called a single blind study). This approach
is another way to avoid bias, because when people
know what drug they are taking, it might change the
way they react. For instance, patients who knew they
were taking the new treatment might expect it to work
better and report hopeful signs because they want
to believe they are getting well. This could bias
the study by making results look better than they
really were.
Why Do Phase III Clinical Trials
Compare Treatment Groups?
Comparing similar groups of people taking different
treatments for the same type of cancer is another
way to make sure that study results are real and caused
by the treatment rather than by chance or other factors.
Comparing treatments with each other often shows clearly
which one is more effective or has fewer side effects.
Another reason Phase III trials compare the new treatment
with standard treatment is so that no one in a
study is left without any treatment when standard
treatment is available, which would be unethical.
When no standard treatment exists for a cancer, some
studies compare a new treatment with a placebo
(a look-alike pill that contains no active drug).
However, you will be told if this is a possibility
before you decide whether to take part in a study.
Your Doctor Can Tell You More
If you have any questions about how clinical trials
work, ask your doctor, nurse, or other health professional.
It may be helpful to bring this booklet and discuss
points you want to understand better.
Should I Take Part
in a Clinical Trial?
This is a question only you, those close to you, and
your health professionals can answer together. Learning
you have cancer and deciding what to do about it is
often overwhelming. This section has information you
can use in thinking about your choices and making
your decision.
Clinical Trials: Weighing the Pros
and Cons
While a clinical trial is a good choice for some people,
this treatment option has possible benefits and drawbacks.
Here are some factors to consider. You may want to
discuss them with your doctor and the people close
to you.
Possible Benefits
- Clinical
trials offer high-quality cancer care. If you
are in a study and do not receive the new treatment
being tested, you will receive the best standard
treatment. This may be as good as, or better than,
the new approach.
- If
a new treatment approach is proven to work and
you are taking it, you may be among the first
to benefit.
- By
looking at the pros and cons of clinical trials
and your other treatment choices, you are taking
an active role in a decision that affects your
life.
- You
have the chance to help others and improve cancer
treatment.
Possible Drawbacks
- New
treatments under study are not always better than,
or even as good as, standard care. They may have
side effects that doctors do not expect or that
are worse than those of standard treatment.
- Even
if a new treatment has benefits, it may not work
for you. Even standard treatments, proven effective
for many people, do not help everyone.
- If
you receive standard treatment instead of the
new treatment being tested, it may not be as effective
as the new approach.
- Health
insurance and managed care providers do not always
cover all patient care costs in a study. What
they cover varies by plan and by study. To find
out in advance what costs are likely to be paid
in your case, talk to a doctor, nurse or social
worker from the study.
Your Rights, Your Protections
Before and during a cancer treatment study, you have
a number of rights. Knowing these can help protect
you from harm.
- Taking
part in a treatment study is up to you. It may
be only one of your treatment choices. Talk with
your doctor. Together, you can make the best choice
for you.
- If
you do enter a study, doctors and nurses will
follow your response to treatment carefully throughout
the research.
- If
researchers learn that a treatment harms you,
you will be taken off the study right away. You
may then receive other treatment from your own
doctor.
- You
have the right to leave a study at any time.
One of your
key rights is the right to informed consent.
Informed consent means that you must be given all
the facts about a study before you decide whether
to take part. This includes details about the treatments
and tests you may receive and the possible benefits
and risks they may have. The doctor or nurse will
give you an informed consent form that goes over key
facts. If you agree to take part in the study, you
will be asked to sign this informed consent form.
The informed consent process continues throughout
the study. For instance, you will be told of any new
findings regarding your clinical trial, such as new
risks. You may be asked to sign a new consent form
if you want to stay in the study.
Signing a consent form does not mean you must
stay in the study. In fact, you can leave at any time.
If you choose to leave the study, you will have the
chance to discuss other treatments and care with your
own doctor or a doctor from the study.
Questions You Should
Ask
Finding answers and making choices may be hard for
people with cancer and those who care about them.
It is important to discuss your treatment choices
with your doctor, a cancer specialist (an oncologist)
to whom your doctor may refer you, and the staff of
any clinical trial you consider entering.
Ask questions about the information you receive during
the informed consent process and about any other issues
that concern you. Getting answers can help you work
better with the doctor. You may want to take a friend
or relative along when you talk to the doctor. It
also may help to write down your questions and the
answers you receive, or bring a tape recorder to record
what is said. No question about your care is foolish.
It is very important to understand your choices.
Here are some questions you may want to ask about:
The Study
- What
is the purpose of the study? In what phase is
this study?
- Why
do researchers believe the new treatment being
tested may be effective? Has it been tested before?
- Who
sponsors the study, and who has reviewed and approved
it?
- How
are the study data and patient safety being checked?
- When
and where will study results and information go?
Possible
Risks and Benefits
- What
are the possible short- and long-term risks, side
effects, and benefits to me?
- Are
there standard treatments for my type of cancer?
- How
do the possible risks, side effects, and benefits
in the study compare with standard treatment?
Your
Care
- What
kinds of treatments, medical tests, or procedures
will I have during the study? Will they be painful?
How do they compare with what I would receive
outside the study?
- How
often and for how long will I receive the treatment,
and how long will I need to remain in the study?
Will there be follow-up after the study?
- Where
will my treatment take place? Will I have to be
in the hospital? If so, how often and for how
long?
- How
will I know if the treatment is working?
- Will
I be able to see my own doctor? Who will be in
charge of my care?
Personal
Issues
- How
could the study affect my daily life?
- Can
you put me in touch with other people who are
in this study?
- What
support is there for me and my family in the community?
Cost
Issues
- Will
I have to pay for any treatment, tests, or other
charges?
- What
is my health insurance likely to cover?
- Who
can help answer any questions from my insurance
company or managed care plan?
Other
Questions
(Use this space to write down other questions you
have.)
________________________________________________
________________________________________________
________________________________________________
________________________________________________
________________________________________________
________________________________________________
Others Can Help
As you make your treatment decisions, remember that
you are not alone. Doctors, nurses, social workers,
other people with cancer, clergy, family, and those
who care about you can help and support you. The resources
in the next section also can provide more information
and links to other contacts in your community.
National Cancer Institute
Information Resources
You may want more information for yourself, your family,
and your doctor. The following National Cancer Institute
(NCI) services are available to help you.
Telephone...
Cancer
Information Service (CIS)
Provides accurate, up-to-date information on cancer
to patients and their families, health professionals,
and the general public. Information specialists
translate the latest scientific information into
understandable language and respond in English,
Spanish, or on TTY equipment. Toll-free: 1-800-4-CANCER
(1-800-422-6237) TTY: 1-800-332-8615
Internet... these web sites may be useful:
http://www.nci.nih.gov/
NCI's primary web site; contains information about
the Institute and its programs.
http://cancertrials.nci.nih.gov/
Cancer Trials; NCI's comprehensive clinical trials
information center for patients, health professionals,
and the public. Includes information on understanding
trials, deciding whether to participate in trials,
finding specific trials, plus research news and
other resources.
http://cancernet.nci.nih.gov/
CancerNetTM; contains
material for health professionals, patients, and
the public, including information from PDQ¨ about
cancer treatment, screening, prevention, supportive
care, and clinical trials, and CANCERLIT¨, a bibliographic
database.
http://rex.nci.nih.gov/
Includes news, upcoming events, educational materials,
and publications for patients, the public, and the
mass media.
http://chid.nih.gov/ncichid/
Cancer Patient Education Database; provides information
on cancer patient education resources for health
educators and other health professionals.
E-mail...
CancerMail
Includes NCI information about cancer treatment,
screening, prevention, and supportive care. To obtain
a contents list, send e-mail to cancermail@icicc.nci.nih.gov
with the word "help" in the body of the message.
Fax...
CancerFax®
Includes NCI information about cancer treatment,
screening, prevention, and supportive care. To obtain
a contents list, dial 301-402-5874 from a fax machine
hand set and follow the recorded instructions.
Glossary
This glossary contains a list of words used in the
booklet and their definitions. It also explains
some other terms related to treatment studies that
you may hear from your doctor or nurse.
Bias: Human choices or any other factors
beside the treatments being tested that affect a
study's results. Clinical trials use many methods
to avoid bias, because biased results may not be
correct.
Clinical trials: Research studies that involve
people. Each study tries to answer scientific questions
and to find better ways to prevent or treat cancer.
Control group: In a clinical trial, the group
of people that receives standard treatment for their
cancer. (See Treatment group.)
Informed consent: The process in which a
person learns key facts about a clinical trial or
research study and then agrees voluntarily to take
part or decides against it. This process includes
signing a form that describes the benefits and risks
that may occur if the person decides to take part.
Institutional Review Board (IRB): Groups
of scientists, doctors, clergy, and consumers at
each health care facility at which a clinical trial
takes place. Designed to protect patients who take
part in studies, IRBs review and must approve the
protocols for all clinical trials funded by the
Federal Government. They check to see that the study
is well-designed, does not involve undue risks,
and includes safeguards for patients.
Investigator: A researcher in a treatment
study.
Oncologist: A doctor who specializes in treating
cancer.
Placebo: A tablet, capsule, or injection
that looks like the drug or other substance being
tested but contains no drug.
Protocol: An action plan for a clinical trial.
The plan states what will be done in the study and
why. It outlines how many people will take part
in the study, what types of patients may take part,
what tests they will receive and how often, and
the treatment plan.
Randomization: A method used to prevent bias
in research. People are assigned by chance to either
the treatment or control group.
Remission: When the signs and symptoms of
cancer go away, the disease is said to be "in remission."
A remission can be temporary or permanent.
Side effects: Problems that occur when treatment
affects healthy cells. Common side effects of standard
cancer treatments are fatigue, nausea, vomiting,
decreased blood cell counts, hair loss, and mouth
sores. New treatments being tested may have these
or other unknown side effects.
Single blind study: A method used to prevent
bias in treatment studies. In a single blind study,
the patient is not told whether he/she is taking
the standard treatment or the new treatment being
tested. Only the doctors know.
Stage: The extent of a cancer and whether
the disease has spread from the original site to
other parts of the body. Numbers with or without
letters are used to define cancer stages (e.g.,
Stage IIb).
Standard treatment: The best treatment currently
known for a cancer, based on results of past research.
Treatment group: The group that receives
the new treatment being tested during a study. (See
Control group.)
NIH Publication No. 98-4250 (April 1998)
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