FAQs
About Clinical Trials
Learning
if a Trial is Sound and Well Run:
What
is a clinical trial?
In
cancer research, a clinical trial is a study conducted with cancer patients,
usually to evaluate a new treatment. Each study is designed to answer
scientific questions and to find new and better ways to help cancer
patients.
The
search for good cancer treatments begin with basic research in laboratory
and animal studies. The best results of that research are tried in
patient studies, hopefully leading to findings that may help many
people.
Before
a new treatment is tried with patients, it is carefully studied in
the laboratory. This research points out the new methods most likely
to succeed and, as much as possible, shows how to use them safely
and effectively. But this early research cannot predict exactly how
a new treatment will work with patients. With any new treatment there
may be risks as well as possible benefits. There may also be some
risks that are not yet known. Clinical trials help us find out if
a promising new treatment is safe and effective for patients. During
a trial, more and more information is gained about a new treatment,
its risks, and how well it may or may not work.
Standard
treatments -- those now being used -- are often the base for building
new, hopefully better treatments. Many new treatments are designed
on the basis of what has worked in the past, in efforts to improve
on this.
Only
patients who wish to, take part in a clinical trial. You may be interested
in or asked to enter a trial. Learn as much as you can about the trial
before you make up your mind.
Advances
in medicine and science are the results of new ideas and approaches
developed through research. New cancer treatments must prove to be
safe and effective in scientific studies with a certain number of
patients before they can be made widely available.
Through
clinical trials, researchers learn which approaches are more effective
than others. This is the best way to test a new treatment. A number
of standard treatments were first shown to be effective in clinical
trials. These trials help us find new and better treatments.
Patients
take part in clinical trials for many reasons. Usually, they hope for
benefits for themselves. They may hope for a cure of disease, a longer
time to live, a way to feel better. Often they want to contribute to
a research effort that may help others.
Based
on what researchers learn from laboratory studies, and sometimes earlier
clinical studies and standard treatments as well, they design a trial
to see if a new treatment will improve on current treatments. The
hope is that it will. Often researchers use standard treatments as
the building blocks to try to design better treatments.
Many
trials have turned out to be better than standard treatments; others
have either been not as good as or no better than the treatments already
being used. Although there is always a chance that a new treatment
will be disappointing, the researchers involved in a study have reason
to believe that it will be as good as, or better than, current treatments.
There is
the potential for immediate personal benefit including:
- Standard
therapy is either unavailable or offers little hope of a good outcome:
You might have a positive response to the experimental treatment.
- Available
standard therapy has not produced a good response: You might
have a positive response to the experimental treatment.
- Early access
to new treatments: Considerable time can elapse between the
initial testing of a new treatment and the point of availability
to the general public.
- Review by
leading doctors and scientists: As a result of participation
in a clinical trial, your health condition may be reviewed by some
of the leading doctors and scientists in the field.
- In depth
monitoring of your illness: As a result of participation in
a clinical trial, your response to the therapy will be carefully
monitored.
- Possible
cost savings: The cost of some of your treatment may be covered
by the study.
Patients
in these studies receive the benefit of this vast expertise. At cancer
centers, patients receive care from a special research team. Through
new programs, community hospitals and doctors are also coming more
and more into the research network.
There
is also the potential to benefit others:
Patients participating
in a clinical trial contribute to a research effort that will help
current and future patients. Medical knowledge related to your specific
illness is expanded by your participation. This may eventually translate
into more effective drug treatments and therapies.
The knowledge
gained about improved treatment methods may also be directly applicable
to your loved ones. For example, when a women with breast cancer participates
in a trial, her daughters, sisters, nieces, and granddaughters --
who may be at risk of developing the disease in the future -- stand
to benefit directly from the new knowledge gained from her participation
in the trial.
Yes.
The treatments used in clinical trials can cause side effects and risks
depending on the type of treatment and the patient's condition. Side
effects vary from patient to patient.
Because
clinical trials are research into new areas of treatment, the risks
involved are not always known ahead of time, though efforts have been
made to find out what these risks might be. For this reason, trials
can carry unknown dangers and side effects as well as hoped-for benefits.
Patients need to know what is involved in a study -- what side effects
may be expected -- and, as much as possible, what "unknowns"
or uncertainties they may be facing.
You
will be told about the treatments being tested and will be given a
form to read that discusses the risks and hoped-for benefits. If you
agree to take part, you will be asked to sign a document, called the
informed consent form. Before you sign, be sure you understand what
risks you face. Ask the doctor or nurse to explain any parts of the
form or the trial that are not clear. If you do not want to be in
the trial, you may refuse. Even if you sign the consent form, you
are free to leave the trial at any time and can receive other available
medical care.
In
clinical trials, most side effects are temporary and will gradually
go away once treatment is stopped. For example, some anticancer drugs
cause hair loss and nausea and some do not. They can also affect the
bone marrow which produces blood cells. During treatment, the number
of blood cells, called blood counts, may fall too low. Since this could
lead to possible infection or other problems, patients have their blood
counts checked often. Luckily, bone marrow has a great ability to replace
blood cells, so that blood counts can usually return to normal.
Some
side effects in clinical trials can be permanent and serious, even life-threatening.
Also, certain side effects may not appear until later, after the treatment
itself is over. (These "late" effects may include damage to
a major organ like the heart, lungs, or kidneys; sterility; or a second
cancer.) Many cancer patients are now living longer, largely because
of better treatments. Researchers are concerned and trying to prevent
late complications of treatments.
As
a patient, it can be hard to decide about your treatment. There are
a number of things to consider. Cancer is a life-threatening disease
which causes symptoms of its own that are not related to treatment.
In each case, the unavoidable risks of the cancer itself, and your
condition, should be weighed against the potential risks and benefits
of a new research treatment. Standard treatments, as well as treatments
in clinical trials, can also cause side effects and risks.
Any
medical treatment can carry the potential for side effects in some
patients. Cancer treatment is particularly powerful because it is
designed to destroy constantly dividing cancer cells. It can also
affect healthy dividing cells and this can cause side effects. The
challenge to researchers has been to develop treatments that destroy
cancer cells but do not harm healthy cells.
What
is being done to lessen side effects of treatment?
Cancer researchers are trying to make cancer treatment more effective
and lessen its side effects for the cancer patient, including:
- new
anticancer drugs with less side effects;
- better
antinausea medicine;
- some
shorter periods of time on anticancer drugs;
- special
ways to protect normal tissues during radiation therapy;
- new
methods of surgery that are less extensive and less damaging to
the body; and
- psychological
support programs and information on ways to cope during difficult
times. How patients feel during and after treatment is important.
If
You Are Thinking of Entering a Clinical Trial . . .
Every
clinical trial is designed to answer a set of research questions.
If you fit the guidelines for a trial, you may be eligible to take
part. Each study enrolls patients with certain types and stages of
cancer and certain health status. A study that involves two or more
treatments can yield reliable answers only if all the patient cases
are the same so they can be compared with each other.
Depending
on the study, you might be asked for such personal information as,
your:
- gender (sex)
- age
- ethnicity
- education
- occupation
- lifestyle
You will also
be asked for a variety of health/illness related information, such
as:
- site of cancer
- stage of cancer
- previous treatments
- previous treatment
outcomes
- health and
wellness background
- allergy history
physical/functional status
- family history
Before
a decision is made about your treatment (whether it is in a clinical
trial or not), your type of cancer will be diagnosed and "staged."
Staging tells how far the disease has spread. Deciding on treatment
depends on many things, including the stage of the disease and your
general condition. You would most likely be referred to a trial by
your own doctor or by a doctor who knows your case. Some patients
find out about trials from other sources. In any case, you must have
a reasonable understanding of your role in a research study and be
freely willing to take part in it. Ask what you can expect if you
take part in a trial.
There
are many ways to find out what your treatment choices are. Talk with
your doctors and get the opinion of cancer specialists (oncologists).
You should not be afraid to ask for a second opinion. A helpful, new
computer information system, called PDQ, is supported by the National
Cancer Institute. PDQ can give your doctor the latest information
on clinical trials being offered around the country for each type
and stage of cancer. This reference is updated regularly, and is featured
on our website
for our convenience. Your doctor can check it from a library or personal
computer.
The
Cancer Information Service (CIS) is another source of information.
This program, also sponsored by the National Cancer Institute, answers
cancer-related questions from the public, cancer patients and their
families, and health professionals. If you have questions, call the
toll-free number: I-800-4-CANCER, and you will be connected to the
CIS office serving your area. For the benefit of Moores UCSD cancer
patients, we have included this the NCI
CIS information on our website.
This
is a big question. Finding answers and making decisions are often
hard for a cancer patient. The diagnosis of cancer and deciding what
to do about it can be overwhelming, and you may be confused and upset.
It is important to discuss your options with medical experts -- including
your own doctor -- and with those close to you. Your personal doctor
and cancer specialists can counsel you about your choices for standard
treatment or clinical trials.
Talk
to them and ask questions about the problems you are facing. If you
understand what is going on, you can help your doctor work with you
more effectively. You may want to take a friend or relative along
with you when talking to your doctor about your case.
Take
time to ask your questions and to discuss what you want to know. It
may help you and your doctor if you plan what to ask and write questions
down ahead of time. No question is foolish. Learn what is available
to you. Find out your choices and the risks and benefits of each.
Each patient is different. You are an individual with individual needs,
and your health is important. If you are a parent of a child with
cancer, of course, you have great concerns about making the best decision
for your child's care.
As
you decide about treatment and whether or not to participate in a
clinical trial, remember that you are not alone. There are many people
to help you -- doctors, nurses, social workers, clergy, your family,
friends, and other patients. Although it is YOUR decision, they can
help you think about it to decide what is best for you.
- What
is the purpose of the study?
- What
does the study involve? What kinds of tests and treatments? (Find
out what is done and how it is done.)
- What
is likely to happen in my case with, or without, this new research
treatment? (What may the cancer do and what may this treatment
do?)
- What
are other choices and their advantages and disadvantages? (Are
there standard treatments for my case and how does the study compare
with them?)
- How
could the study affect my daily life?
- What
side effects could I expect from the study? (There can also be
side effects from standard treatments and from the disease itself.)
- How
long will the study last? (Will it require an extra time commitment
on my part?)
- Will
I have to be hospitalized? If so, how often and for how long?
- Will
I have any costs? Will any of the treatment be free?*
- If
I am harmed as a result of the research, what treatment would
I be entitled to?
- What
type of long-term followup care is part of the study?
Clinical trials
often contain technical medical language, not used in everyday conversation.
Many trials therefore require that the patient be fluent in English.
Some trials will also be conducted in a second language. If you do
not speak English fluently, it still may be possible to participate
in the study if you have a friend or a family member who is readily
available to act as a translator between you and your health care
providers. You should discuss this possibility with your doctor.
*Costs are a major concern of patients and families. Different arrangements
and policies exist at different institutions and, of course, insurance
coverage varies. Patients should freely discuss what costs are involved
in their cases ahead of time. If you need financial aid, contact the
hospital social services office, the Cancer Information Service, or
the local chapter of the American Cancer Society. They may be able
to direct you to a source of help.
Informed
consent, a key part of a good trial, is required in studies that are
federally regulated or funded as well as being subject to state laws.
Informed consent means that as a patient, you are given information
so you can understand what is involved in a trial, including its potential
benefits and risks, and then decide freely to take part in it or not.
The nature of the treatment is explained by the doctors and nurses
who are conducting the trial. You are given an informed consent form
to read and consider carefully. The
informed consent document can be unsettling. It is important to realize
that this consent procedure is done to protect you, not to frighten
you. You should fully understand both the benefits and the risks involved
in a particular trial so that you can make your own informed decision
about whether you wish to participate. Ask
any questions you may have. Then, if you agree to take part, you can
sign the form. Of course, you may also refuse.
The
informed consent process is ongoing.
Tthe goal of clinical trials is the development of new treatment options
which may eventually become the accepted new standard treatment. However,
in your course of the clinical trial, if you experience side effects
that are more than you can, or are willing, to tolerate, you or your
doctor would decide either to decrease the treatment or stop it entirely.
Similarly, should your disease appear to progress in spite of the
treatment, your participation in the trial would stop, in order to
permit you or your doctor to consider other treatment choices.
If you enter a trial, you will continue to receive any new information
about your treatment that may affect your willingness to stay in the
trial. Signing a consent form does not bind you to the study.
Whether
cancer patients are in a research study or not, they face a new world
of medical terms and procedures. For some people, myths and fears
of "experimentation" or of being a "guinea pig"
are associated with clinical trials. And, surely, there are fears
of the unknown. Understanding what is involved can ease some of your
anxieties. Patients in a clinical trial, for example, receive their
care in the same places that standard treatments are given; cancer
centers, hospitals, clinics, or doctors' offices.
Because
a growing number of cancer specialists are now in private community
practice, most cancer care can be given in an area near your home.
Doctors, nurses, social workers and other health professionals from
many different specialties may help care for you. They are working
together for your good, and there is consideration for your privacy
and well-being.
If
you join a research study, you will be watched closely and data on your
case will be carefully recorded. You may receive more examinations and
tests than are usually given. (These are to follow your progress as
well as to collect study data.) Of course, tests can carry certain risks
and benefits or discomforts of their own. Although they can be inconvenient,
these tests can assure an extra ounce of observation along the way.
During
the course of a study, if it is clear that a treatment is not in your
best interest, you will be removed from the study and you can discuss
other options.
Yes.
Just as you can refuse to join a study, you may leave a study at any
time. Your rights as an individual do not change because you are a patient
in a clinical trial. You may choose to take part or not, and you can
always change your mind later, even after you enter a trial.
You
may also refuse to take part in any aspect of the research. If you
have questions at any time about any part of the study, be sure to
ask your doctors. If you are not satisfied with the answers, you may
consider leaving the study. If you decide to leave, it will not be
held against you. Don't be afraid that you will receive no further
care.
People
who consider terminating their participation in a clinical trial,
usually do so for a specific reason or reasons. The ideal way to make
your decision about whether to continue participating in a study is
to talk with the physician or study coordinator to be sure you have
all the information you need to make the right decision for you. This
opportunity to talk also gives the study coordinator the opportunity
to evaluate whether there is a way to correct whatever is leading
you to contemplate stopping your participation in the study.
The
ethical and legal codes that govern medical practice apply to clinical
trials. In addition, most clinical research is federally regulated or
federally funded (at least in part), with built-in safeguards to protect
patients. These safeguards include regular review of the protocol (the
study plans) and the progress of each study by researchers at other
places.
For
example, federally funded and federally regulated clinical trials must
first be approved by an Institutional Review Board (IRB) located at
the institution where the study is to take place. IRBs, designed to
protect patients, are made up of scientists, doctors, clergy and other
people from the local community. An IRB reviews a study to see that
it is well designed with safeguards for patients, and that the risks
are reasonable in relation to the potential benefits.
Federally
supported or regulated studies also go through reviews by a government
agency, such as the National Cancer Institute,* which sponsors and
monitors many trials around the country.
Any
well-run clinical trial, whether federally supported or not, is carefully
reviewed for medical ethics, patient safety, and scientific merit by
the research institution. Every study should provide for monitoring
the data and the safety of patients on an ongoing basis.
As
discussed earlier, informed consent is also an important process that
helps to protect patients.
After
patients join a clinical trial and it progresses, the doctors report
the results of the trial to scientific meetings, to medical journals
whose articles are approved by experts, and to various government
agencies.
*The National Cancer Institute (NCI) is the Federal Government's chief
agency for cancer research. Located at the National Institutes of
Health (NIH) in Bethesda, Maryland, the NCI funds cancer research
across the country and conducts research at its own facilities. For
information about NCI trials, call: 800-638-6694 (in Maryland, call
800-492-6600).
Things
that make a sound, well-run trial to safeguard patients include items
previously discussed above. Keeping these items in mind, here are
some important questions for you to ask to find out if a study is
well run:
- What
is its purpose?
- Who
has reviewed and approved the study?
- Who
is sponsoring the study?
- How
are the study data and patient safety being checked?
- Where
will information from the study go? (In government- related research,
for example, reports might go to the NCI and/or the Food and Drug
Administration.)
For
your own protection, be sure to get satisfactory answers to these
questions before you agree to take part.
There
are many kinds of clinical trials. They range from studies of ways
to prevent, detect and diagnose, control and treat cancer, to studies
of the psychological impact of the disease and ways to improve the
patient's comfort and quality of life (including pain control).
Most
cancer clinical trials deal with new treatments. These treatments
often involve surgery, radiation therapy (the use of X-rays, neutrons
or other types of cell-destroying radiation), and chemotherapy (the
use of anticancer drugs). Alone, or in combination, these types of
treatments can cure many cancer patients and prolong the lives of
many others. A fairly new area of cancer treatment is biological therapy
-- the use of biologicals (substances produced by the body's own cells)
and biological response modifiers (substances that affect the body's
natural defense systems against disease).
Clinical
trials are carried out in phases, each designed to find out certain
information. Patients may be eligible for studies in different phases
depending on their general condition and the type and stage of their
cancer. More patients take part in the later phases of studies than
in the earlier ones.
Phase
I studies establish the safety of a new treatment or procedure,
and is given to a small number of patients. The researchers must find
the best way to give a new treatment and how much of it can be given
safely. They watch carefully for any harmful side effects. The research
treatment has been well tested in laboratory and animal studies, where
they have been demonstrated to be effective, but no one knows how
patients will react. Phase I studies may involve significant risks
for this reason. They are offered only to patients whose cancer has
spread and who would not be helped by other known treatments. Phase
I treatments may produce anticancer effects, and some patients have
been helped by these treatments.
Phase
II studies determine the effectness of a research treatment on
various types of cancer among patients who share common illness-related
characteristics. Each new phase of a clinical trial depends on and
builds on intormation from an earlier phase. A Phase II study usually
requires a greater number of participants than a Phase I study.
Phase III studies compares the research treatment with the standard
treatment to see which is more effective. Often researchers use
standard therapy as the base to design new, hopefully better treatments.
Phase IV is when the new research treatment becomes part of standard
treatment in patient care. For example, a new drug that has been
found effective in a clinical trial may then be used together with
other effective drugs, or with surgery, and/or radiation therapy.
The
doctors who conduct a clinical trial follow a carefully designed treatment
plan called a "protocol." This spells out exactly what will
be done and why. Studies are planned to safeguard the medical and
psychological health of patients as well as to answer research questions.
Some
clinical trials test one research treatment in one group of patients.
Other trials compare two or more treatments in separate groups of patients
who are similar in certain ways, such as the extent of their disease.
This way, the treatment groups are alike and the results from each can
validly be compared.
One
of the groups may receive standard (the most accepted) treatment so
the new treatments can be directly compared to it. The group receiving
the standard treatment is called the "control" group. For
example, one group of patients (the control group) may receive the usual
surgical treatment tor a certain cancer, while another patient group
with the same type of cancer may receive surgery plus radiation therapy
to see if this improves disease control.
Sometimes,
no standard treatment yet exists for certain cancer patients. In drug
studies for such cases, one group of patients might receive a new drug
and the control group, none. But no patient is placed in a control group
without treatment if there is any known treatment that would benefit
that patient. The control group is followed as often and carefully as
the "treatment" group.
One
of the ways to prevent the bias of a patient or doctor from influencing
study results is "randomization." If a patient agrees to be
randomized, this means he or she is selected by chance to be in one
group or another. The researchers do not know which treatment is best.
From what is known at the time, any one of the treatments chosen could
be of equal benefit to the patient.
If
the treatment in a trial is not helping the patient, the patient's doctor
can decide to take him or her out of the study. Of course, the patient
can decide to leave, as well, and still receive other available care.
There are regular reviews of the results of a trial and the information
is shared. This is important, because if a treatment is found to be
too harmful or not effective, it is stopped. Also, when there is firm
evidence that one method is better than the others in a study, the trial
is stopped and all patients in the trial are given the benefit of the
new information. Such information may help present and future patients.
Throughout
a clinical study, a patient's personal doctor will be kept informed
of the patient's progress. Patients are encouraged to maintain contact
with their referring doctors.
A
nationwide effort to conquer cancer intensified with the National
Cancer Act of 1971. As a result of the National Cancer Program created
by that legislation, more cancer patients are being cured today than
ever before, and many others are living longer with improved quality
of life.
The
National Cancer Program brings together a network of researchers at
many public and private institutions around the country. These include
the NCI, cancer centers, universities, community hospitals and private
industry. Groups involving hundreds of researchers are working to
discover and put to use new knowledge to benefit the cancer patients
of today and tomorrow.
Knowledge
gained from research studies with patients --clinical trials -- has
been essential to overall progress in the war on cancer. Such studies
have led to increased survival for childhood cancers, Hodgkin's disease,
breast, uterine, testicular and bladder cancers, as well as others.
These studies continue to play a key role in progress against cancer.
Today,
major scientific discoveries in the laboratory are part of a revolution
in biology. New tools to unravel the process of cancer are
leading to exciting new approaches against cancer. Clinical
trials continue to be the link between such basic research and patients.
The goal is to translate the best of that research into findings that
directly help people.
A
portion of this information is provided by the National Cancer Institute.
