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What Cancer Patients Need to Know
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.)
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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
- Web Sites
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 this article 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.)
Source: National Institutes of Health
April 1998
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