Introduction
Why Do We Need to Conduct Clinical Trials?
What Are Preclinical Studies And Clinical
Trials?
What Are the Phases of Clinical Trials?
Who Sponsors Clinical Trials? Who Conducts
Them?
Should I Think About Taking Part in a
Clinical Trial?
Who Will Look Out for Me as a Participant?
What's Out There? Finding Clinical Trials
How Do I Figure Out Which Study Is for Me?
What About Cost? Will My Insurance Cover It?
What Would Participating Involve for Me?
What If I'm Not Eligible for a Clinical
Trial?
References
Introduction
Choosing whether or not to take part in a clinical trial can be one of
the toughest decisions facing a person with cancer.
Clinical trials are people-based studies -- as opposed to animal or lab
studies -- of new drugs or procedures. Doctors use clinical trials to
learn whether a new treatment is safe and effective in patients. Such
studies are vital to the development of new treatments for diseases
such as cancer.
The doctors in charge of a clinical trial don’t know ahead of time how
things will turn out. If they did, there would be no need for the study
in the first place. Because of this, there’s no simple answer to the
question, "Should I take part?"
Most people don’t pay much attention to clinical trials until they are
diagnosed with a serious illness such as cancer. Medical breakthroughs
(the results of clinical trials) often make the news, but you usually
don’t hear about clinical trials themselves unless something has gone
wrong in a clinical trial. The media is quick to pick up on an instance
when a volunteer in a study is harmed. While it is very rare, people
have been harmed, and have even died, while taking part in clinical
trials. Reports of these tragic outcomes are important, because they
help to expose problems in the system, which are then corrected to
protect others.
What you usually don’t hear about, however, are the thousands of people
who are helped each year because they decided to take part in a
clinical trial, not to mention the millions who ultimately benefit from
others’ participation in clinical trials.
There is no right or wrong choice when it comes time to decide on
taking part in a clinical trial. The decision is a very personal one
and depends on many factors, including the benefits and risks of the
study, what the person hopes to achieve by taking part, and other
preferences.
Knowing all you can about clinical trials in general -- and ones you
are considering in particular -- can help you feel more at ease with
your decision. If you do decide to participate, knowing what to look
for and what to expect ahead of time can help you.
This guide should address many basic questions and concerns so that you
are better prepared to discuss the subject with your doctor and family.
It should clarify which questions need to be asked beforehand and what
the answers may mean for you. In the end, however, only you can decide
if taking part in a clinical trial is right for you.
One last note: this guide focuses on studies for people who are being
treated for cancer. But most of the information here applies to other
types of clinical trials as well.
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Why Do
We Need to Conduct Clinical Trials?
Clinical trials show us what works (and what doesn’t) in
medicine. They are the best way for doctors to learn what is safe and
effective in treating diseases such as cancer.
Some doctors and scientists conducted what would now be considered
clinical trials as far back as the late 1700s, but clinical trials were
not widely used until the middle of the 20th century. Up until that
time, doctors relied on their own experience in particular cases and on
the teachings of those who came before them. Progress was slow, and
very few medicines existed that could even be tested.
With the discovery of the first antibiotics and other drugs, doctors
needed a reliable way to tell what worked from what didn’t. They also
needed ways to find out which of the countless remedies available at
that time were safe for people to use. So they developed studies that
tested, and often compared, treatments in a specific group of people.
The results of these clinical trials proved to be more useful than
relying on whether or not something worked in one or a few people.
In the US, new drugs and medical devices (but not dietary supplements)
must be approved by the US Food
and Drug Administration (FDA) before they can be advertised or sold to the general public. The FDA
began overseeing the safety of new treatments in the late 1930s but
didn’t require proof of effectiveness until the early 1960s. Today,
drugs and medical devices must go through several phases of clinical
trials (discussed below) before being approved for use.
Based on what we have learned about cancer in recent years, researchers
can now develop new treatments in a more logical way and much faster
than in the past. But it's still a time-consuming and difficult process.
Clinical trials are only a small part of the research that goes into
developing a new treatment. Potential drugs, for example, first have to
be discovered, purified, characterized, and tested in labs (in cell and
animal studies) before ever reaching clinical trials. In all, about
1,000 potential drugs are tested before just one reaches the point of
being tested in a clinical trial. On average, a new cancer drug has at
least 6 years of research behind it before it even makes it to clinical
trials. But the major holdup in making new cancer drugs available is
the time it takes to complete clinical trials themselves. On average,
about 8 years pass from the time a cancer drug enters clinical trials
until it is approved.
Why so long? To establish safety and effectiveness, researchers look at
each new treatment in several different studies. Only certain people
are eligible to take part in each clinical trial. And cancer trials, by
their very nature, take years to complete. It takes months, if not
years, to see if a cancer treatment works in any one person.
The biggest barrier to completing studies is the shortage of people who
take part. Fewer than 5% of adults with cancer will do so. According to
the Pharmaceutical Research and Manufacturers of America (PhRMA), about
400 cancer medicines were being tested in clinical trials in 2005. Not
all of these will prove to be useful, but those that are may be delayed
in getting approved because the number of participants is so low.
The number one reason people give for not taking part in a clinical
trial is that they didn’t know the studies were an option for them. But
there are many other reasons. Some people may want to take part but
aren’t eligible for some reason. Some people are uncomfortable with the
idea of being a "subject" in a study. Others worry that they won’t be
treated fairly or could be harmed by an unproven treatment. Certainly
these are understandable concerns.
One of the most important points in deciding if a clinical trial can be
done is whether or not it would be ethical to ask patients to volunteer
for an experimental treatment. Has the study been designed, as much as
possible, to ensure the safety of the people involved? Would the
participants receive a treatment that is at least as good as, and
possibly better than, what they would get if they did not volunteer for
the study? Scientific panels are set up to review and approve all
clinical trials to make sure questions like these are answered before
the researchers are allowed to enroll patients.
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What
Are Preclinical Studies and Clinical Trials?
Clinical trials are medical research studies involving people.
They are done only after "preclinical" studies suggest that the
proposed treatment is likely to be safe and effective in people.
Preclinical studies, also called laboratory studies, include:
- Cell studies: These are often the first
tests done when looking at a new form of treatment. To see if it is
effective, researchers look for effects of the new treatment on cancer
cells on a laboratory dish or in a test tube.
- Animal studies: Treatments that appear
promising in cell studies are next tested on tumors in lab animals.
This gives the researchers an idea of how safe the new treatment is in
a living organism.
Although preclinical studies provide a lot of useful information, they
do not give us all the answers. After all, humans and mice are
different. A treatment that works against cancer in a mouse may or may
not work in people. And human bodies sometimes process drugs
differently. This may result in side effects that were not obvious when
studying the treatment in mice.
Clinical trials are therefore needed to answer 2 important questions:
- Is the new treatment effective?
Is it better than what’s now available to treat a specific disease? If
it’s not better, is it at least as good, perhaps while causing fewer
side effects? Or does it work in some people who don't benefit from
current treatments? In other words, is it a step forward? A treatment
that doesn’t offer anything new probably isn’t worth studying.
- Is the new treatment safe?
This must be answered while realizing that no treatment or
procedure—even one already in use—is entirely without risk. But do the
benefits of the new treatment outweigh the possible risks?
Answering these questions, while exposing as few people as
possible to an unknown treatment, often requires several different
clinical trials. These are usually divided into "phases." Each phase is
designed to answer certain questions, while trying to ensure the safety
of the people taking part. Each new treatment is tested in several
phases of clinical trials before being considered reasonably safe and
effective. These phases are discussed in the next section.
Some Important Points to Keep in
Mind
Clinical trials are vital in studying all aspects of medicine,
not just cancer. The stakes may seem higher when researching medicines
to treat cancer, but all new treatments (drugs and medical devices)
must go through clinical trials before being approved by the FDA for
general use.
When most people think about clinical trials, they have
certain ideas about what they involve. But not all clinical trials are
the same.
Not all clinical trials study treatments.
Many clinical trials study new ways to detect, diagnose, or determine
the extent of disease. Some even look at ways to prevent the disease
from happening in the first place.
Even among clinical trials that do study treatments,
not all of them study drugs. Many clinical trials test
other forms of treatment, such as new surgery or radiation therapy
techniques, or even complementary or alternative medicines.
When clinical trials do look at drugs, not all of
them study new ones. Even after a drug has been approved for
use against a type of cancer, doctors sometimes find it works better
when given a certain way or when combined with other treatments. It may
even work on a different kind of cancer. Clinical trials are needed to
study these possibilities as well.
Very few cancer clinical trials involve a placebo.
A placebo is an inactive ingredient or pill used in some types of
clinical trials to help make sure results are unbiased. A placebo is
sometimes called a "sugar pill." Over the years, doctors have observed
that some people begin to feel better even if they just think
they’re being treated. With the possibility of getting a placebo,
people can’t tell whether they are receiving the treatment being
studied or not, which makes the results more likely to be valid.
Placebos are rarely used alone in cancer research, unless no
known effective treatments exist. It’s certainly not ethical to have
someone take a placebo if an effective standard treatment is already
available. When cancer clinical trials compare treatments, they compare
the new treatment against the current standard treatment. At times, a
study may be designed so that patients may not be told which one they
are receiving, but they know they are at least getting the current
standard of care.
In some clinical trials, the doctors want to learn whether
adding a new drug to the standard therapy improves its effectiveness.
In these studies, some patients receive the standard drug(s) and a new
one, while other patients receive the standard drug(s) and a placebo.
But none of the patients would receive a placebo only. Everyone
receives standard treatment if there is a standard treatment available.
(See "What Are the Phases of Clinical
Trials?" for an example of a phase III study involving a
placebo.)
All clinical trials are voluntary. You
always have the right to choose whether or not you will take part in a
clinical trial. The level of care you receive should not be affected by
your decision. And you have the right to leave a clinical trial at any
time, for any reason. If you decide to leave, your health care team may
ask that you continue to be monitored for a period of time to look for
any long-term effects of treatment. We discuss these issues further in
the section "What Would Participating
Involve for Me?"
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What
Are the Phases of Clinical Trials?
Clinical trials are usually conducted in distinct phases. Each
one is designed to answer specific questions. Knowing the phase of the
clinical trial you are considering is important because it may give you
some idea about how much is known about the treatment being studied.
There are advantages and disadvantages to taking part in each phase of
clinical trial.
Phase I Clinical Trials – Is the
Treatment Safe?
These studies are the first ones to involve people as participants.
Although the treatment has been tested in lab and animal studies, the
side effects in people can’t always be predicted. For this reason,
these studies usually include small numbers of people (15 to 50) and
are generally reserved for those who do not have good treatment options
left to them. Often, people with different types of cancer are eligible
for the same study. These studies are usually done in major cancer
centers.
The main reasons for doing phase I studies are to determine the highest
dose of the new treatment that can be given safely (without serious
side effects) and to decide on the best way to give the new treatment.
The first few people in the study often receive a low dose of the
treatment and are then watched very closely. If there are no or only
minor side effects, the next few patients receive a higher dose. This
process continues until doctors find the dose that is most likely to
work while having an acceptable level of side effects.
Safety is the main concern because it is the first time the treatment
has been used in people. Doctors keep a close eye on how the people in
the study are doing. Special tests, such as blood tests to measure
levels of the drug in the body at certain time points, are often a part
of these trials. Some studies may require time in a hospital. Placebos
(inactive pills or ingredients) are not part of phase I trials.
These studies are not designed to look at how effective the new
treatment is. Overall, these trials are the ones with the most
potential risk and the least likelihood of potential benefit. But they
do help some patients. For those with life-threatening illnesses,
weighing the potential risks and benefits carefully is crucial.
Here’s an example of a typical phase I clinical trial, but keep in mind
that each study is slightly different:
Bruce was diagnosed with cancer 4 years ago. He was first
treated with radiation therapy, but the cancer was later found to have
spread to distant parts of his body. His doctor told him two
chemotherapy drugs, “A” and “B,” might help him. Bruce’s cancer shrank
for a short time while he was taking drug A, but then it began to grow
again. Drug B did not work in his case.
Because Bruce is still relatively young, his doctor suggests
he might want to consider trying a new form of treatment, “EXP1,” which
is being studied in a phase I clinical trial at the nearest university
hospital.
Bruce talks with the university doctor conducting the study.
The doctor explains that the drug being studied showed some promise in
lab tests, but exactly how well it will work in people is still
unknown. What’s more, it may have side effects that haven’t been seen
yet. After getting all of his questions answered and weighing his
options, Bruce decides to take part in the study.
Because 3 people have already enrolled in the study and have
had no major side effects, Bruce will be the first to get a higher dose
of the treatment. He will need to stay in the hospital overnight on the
first night. This is both to watch for any unexpected reactions and to
take blood samples every few hours so that doctors can figure out how
long the treatment stays in his body. He will be allowed to go home the
next day but must return regularly over the next few weeks to be
monitored, until it is time for the next treatment.
Phase II Clinical Trials – Does
It Work?
If a new treatment is found to be reasonably safe in phase I clinical
trials, the treatment can then be tested for effectiveness in a phase
II clinical trial.
Usually, a group of anywhere from 25 to 100 patients with the same type
of cancer receive the new treatment in a phase II study. They are
treated using the dose and method that were determined most likely to
be safe and effective in phase I studies. In a typical phase II
clinical trial, participants all receive the same dose, and no placebo
is involved.
Some phase II studies do, however, involve randomizing
participants into 1 of 2 treatment groups, similar to what is done in
phase III trials (see below). These groups may get different doses or
get the treatment in different ways to see which provides the best
balance of safety and effectiveness. Phase II studies are often
conducted at major cancer centers but may also be done in community
hospitals or even doctors’ offices.
Doctors look for some evidence that the treatment is effective. The
type of benefit they look for depends on the goals of the clinical
trial. This may involve a response
(where the tumor shrinks or disappears). Or it may be an extended
period of time where the tumor does not get any bigger or an increase
in the length of time before a cancer comes back. In some studies the
benefit may be an improved quality of life. Many studies look to see if
people getting the new treatment live longer than they would have been
expected to without the treatment.
If a certain percentage of the patients benefit from the treatment (and
the side effects aren’t too severe), it is considered to be active
enough against that type of cancer to allow it to go on to a phase III
clinical trial. In addition to watching for responses, doctors continue
to look for any side effects. Larger numbers of patients receive the
treatment in phase II studies, so there is a better chance that less
common side effects may be seen.
Here’s an example of a phase II clinical trial. Again, some phase II
studies may be slightly different:
Angela was diagnosed with cancer several months ago. Only
one form of treatment, drug “C,” is known to work for people with her
type of cancer, and it only works in about half of the people who try
it. After several months of this treatment, Angela’s doctor told her
that it did not appear to be helping in her case.
After doing a little research online, Angela and her doctor
decide her best bet may be to enroll in a clinical trial. They find a
phase II study being conducted by a doctor nearby, who is testing a new
type of medicine, called “EXP2.” This medicine was already found to be
safe in phase I studies. Although not many people have tried it, a
couple of people with Angela’s type of cancer were helped by it.
Angela, like all of the other people in this study, will
receive EXP2 once a week as an outpatient at a local hospital. Before
receiving the drug each week, she will have physical examinations and
blood tests done to see how her body is reacting to the medicine. She
will also have scans done after several weeks to see if the drug is
affecting the cancer. She had similar tests while receiving drug C, but
this time the tests and exams are a little more frequent. Also, her
doctor asks more specific questions about the side effects she
experiences.
Phase III Clinical Trials – Is
It Better Than What’s Already Available?
Treatments that show a certain level of effectiveness in phase II
studies usually must go through one more stage of testing before being
approved for general use. Phase III clinical trials compare the safety
and effectiveness of the new treatment against the current standard
treatment.
Phase III clinical trials require a large number of patients, usually
at least several hundred. These studies are generally conducted in many
places across the country (or even across the world) at the same time.
Because doctors do not yet know which treatment is better, patients are
often randomized
(chosen at random, like flipping a coin) to receive either the standard
treatment or the new treatment. When possible, the study is double-blinded.
That is, neither the doctor nor the patient knows which of the
treatments the patient is getting. Randomization and blinding are
discussed in more detail below.
As with other studies, patients in phase III clinical trials are
watched closely for side effects, and treatment is stopped if they are
too severe. Placebos may be used in some phase III studies, but they
are never used alone if there is already a treatment available that may
work.
Here’s an example of a phase III clinical trial that could possibly
involve a placebo:
Joe has just been diagnosed with cancer. His surgeon
was able to remove the tumor, but tells Joe that this form of cancer
returns in about one-third of people. For this reason, doctors usually
recommend giving a short course of chemotherapy drug "D." While this is
the best drug available for reducing the likelihood of recurrence, some
cancers still return.
Joe’s doctor tells him that a new type of therapy, called "EXP3," is
now being studied. EXP3 was designed to be given along with drug D.
Earlier studies in animals and people have shown that the combination
of drug D and EXP3 seems to be safe and effective. But it is not yet
known if this combination will be better than the current standard of
drug D alone in reducing the risk of recurrence. Therefore, doctors are
testing it in a phase III clinical trial.
To do this, they’ve designed a study that involves treating people with
this cancer in 1 of 2 groups (that is, there are 2 "arms" of the
study): one group will get drug D plus EXP3, while the other group will
get drug D plus a placebo. The patients will be blinded as to which
arm they are on -- that is, they will not know whether they are getting
EXP3 or the placebo. But all of the patients will be getting drug D,
which is the accepted standard of care. The people who get EXP3 may do
better than those who get the placebo. On the other hand, they may do
worse because of things such as unknown side effects. Or both groups
may do equally well, in which case EXP3 would not be any better than a
placebo.
Joe, in deciding whether to take part in the clinical trial, needs to
understand that he will be randomized to 1 of the 2 treatment arms, and
neither he nor his doctor will have control over this. He also needs to
understand that while on this study, he will not know which arm he has
been assigned to (whether he is getting EXP3 or a placebo).
Randomization and blinding are used in many phase III studies because
they help make the study results more credible.
When possible, participants are randomized to ensure that the two 2
study groups have the same traits -- for example, to make sure that
they both contain people in similar states of health, so the results
are not skewed in favor of one group. If people who were allowed to
choose which treatment they got, the study results might not be as
accurate. For example, people who were sicker might tend to choose one
treatment over the other. If this treatment was then found not to work
as well, doctors couldn’t be sure if this was because the treatment
wasn’t as good or because it was tested in sicker people.
Often people have a 50:50 chance of ending up in one group or the
other. In some cases, the study may allow for a different ratio, such
as 2 out of /3 of the people receiving the new treatment and only 1 out
of /3 getting the standard treatment.
Some people find the concept of randomized studies to be distressing,
since neither the patient nor the doctor can choose which group the
patient falls into. This can be especially true if a study is looking
at two totally different treatments, and a person sees one as possibly
better than the other. But remember, doctors are conducting doing the
study because they really don’t know which one is better.
Unfortunately, taking part in such a study is sometimes the only way a
person may have a chance of getting a new form of treatment. But even
then, that treatment may or may not be the best one for him or her.
Blinding is
another area of concern for some people. In a blinded study, the
patient doesn’t know which treatment he or she is getting. In a
double-blinded study, neither the patient nor the doctor knows which
treatment is used. Not knowing what you are receiving can be difficult.
Your doctor can always find out which group you are in if medically
necessary (such as during a possible drug reaction), but although it
may result in your being removed from the study.
The possibility of receiving a placebo
can also be upsetting to some. But this very rarely means you would get
no treatment, unless there was no effective standard treatment to
compare the new drug to. Again, in the example above, Joe will
definitely get drug D, but he will also get either EXP3 or a placebo.
Submission for FDA Approval
In the United States, when clinical trials show a new treatment to be
more effective and/or safer than the current standard treatment, it is
submitted for approval to the Food and Drug Administration (FDA). The
FDA then takes several months to review the data.
Based on the results of the clinical trials, the FDA decides whether or
not it is appropriate for use in the general public. When this happens,
the treatment often becomes the new standard of care, and newer drugs
must be tested against it before being approved.
Phase IV Clinical Trials – What Else Do
We Need to Know?
Even after testing a new medicine on thousands of patients, the full
effects of the treatment may not be known, and some questions may still
need to be answered. For example, a drug may get FDA approval based on
the fact that it was shown to reduce the risk of cancer recurrence. But
does this mean that those who get it are more likely to live longer?
Are there rare side effects that haven’t been seen yet, or side effects
that only show up after the drug is used for a long time? These types
of questions may take many years to answer fully, and may not be
critical for getting a medicine to market. They are often addressed in
what are known as “phase IV” clinical trials.
Phase IV studies look at drugs that have already received FDA approval.
Therefore they are already available for doctors to give to patients.
But these studies are still needed to answer important questions.
When considering taking part in a phase IV trial, patients should know
that the drug has already been approved for use. They do not need to
enroll in the study to get the medicine. At the same time, the care
they would receive in these types of studies is often very similar to
what they could expect if they were to get the treatment outside of a
clinical trial. They should be reassured that in participating they
would be receiving a form of treatment that has already been studied
extensively and that they would be doing a service to future patients.
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Who
Sponsors Clinical Trials? Who Conducts Them?
Of the thousands of cancer clinical trials going on at any one
time, the US National
Cancer Institute (NCI), a part of the National Institutes of
Health (NIH), sponsors (pays for the costs of) a good portion of them.
These studies are often run by NCI-sponsored cancer cooperative groups,
which are networks of doctors and institutions across the country who
specialize in a particular aspect of cancer.
In the Unites States, there are currently 10 major cooperative
groups conducting studies:
- American College of Radiology Imaging Network (ACRIN)
- American College of Surgeons Oncology Group (ACOSOG)
- Cancer and Leukemia Group B (CALGB)
- Children's Oncology Group (COG)
- Eastern Cooperative Oncology Group (ECOG)
- Gynecologic Oncology Group (GOG)
- National Surgical Adjuvant Breast and Bowel Project (NSABP)
- North Central Cancer Treatment Group (NCCTG)
- Radiation Therapy Oncology Group (RTOG)
- Southwest Oncology Group (SWOG)
Other government agencies, including parts of the Department
of Veterans Affairs and the Department of Defense, also sponsor some
cancer clinical trials.
The other main sponsors of clinical trials are pharmaceutical
and biotechnology companies, which must prove their medicines are safe
and effective before they can be marketed. Some nonprofit organizations
also sponsor clinical trials.
Researchers conduct clinical trials in many different
settings. Major cancer centers are often the focal points of clinical
trials research. Because they usually have the most advanced facilities
and highly trained staffs, they can conduct all phases of clinical
trials. But they are not the only places where these studies take
place.
Community hospitals across the country also participate in
clinical trials, although these are usually phase II or III studies.
Many of these hospitals are part of the NCI’s Community Clinical
Oncology Program (CCOP). CCOP members conduct the same clinical trials
across the country. Community hospitals may conduct other, privately
sponsored, studies as well.
Doctors in private practice can also be involved in clinical
trials, either as members of cooperative groups or by being actively
involved in privately sponsored research. But many doctors decide not
to conduct clinical research, for a variety of reasons.
What This May Mean for You
At one time, clinical trials were done only at major medical
centers. This often meant that patients had to travel long distances
and were treated by doctors they did not know very well. This is
sometimes still the case, especially with phase I and some phase II
studies. Of course, this is not necessarily a bad thing. Many people
prefer to be treated in major cancer centers because of their
experience, reputation, and resources. Ultimately, the hassles of
traveling must be weighed against the chance of benefiting from the
treatment.
Patients now have more options. This may include staying
closer to home during a study or even staying with their own doctor.
Your doctor may or may not be involved in clinical trials. If he or she
is, you may be eligible for one of them. Whether this is the right
study for you is, of course, a question worth asking.
Although clinical trials are now done in many different
settings, this should not affect the quality of care you receive. No
matter where a study is conducted, the same rules are in place to
protect patients.
Having so many options can be a burden in and of itself. With
the thousands of clinical trials underway across the country, how can
you—or even your doctor—decide which one is best for you? At this time,
there is no complete list of all the cancer clinical trials. There are,
however, several good places to start looking if you’re interested.
We’ll explore these in the section "What's
Out There? Finding Clinical Trials".
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Should
I Think About Taking Part in a Clinical Trial?
This is one of the toughest questions many people with cancer
will face. The same answer won’t be right for everyone. When trying to
decide, first ask yourself some basic questions:
- Why do I want to take part in a clinical trial?
- What are my goals and expectations if I decide to
participate? How realistic are these?
- How sure are my doctors about what my future holds if:
- I decide to participate?
- I decide not to participate?
- Have I considered:
- The chance of benefit vs. risk?
- Other possible factors, such as time and money?
- My other possible options?
Some of these questions may not have clear-cut answers, but
they should help you start thinking about some important issues. Each
person’s situation is unique, and each person’s reasons for wanting or
not wanting to take part in a study may be different.
Risk vs. Benefit
Each clinical trial offers its own opportunities and
risks, but most have some things in common.
Generally, clinical trials have some of the same potential
benefits:
- You may help others who have the same condition in the
future by contributing to cancer research.
- You may have access to treatment that is not otherwise
available, which might be safer or more effective than current
treatment options.
- You may increase the total number of treatment options
available to you, even if you haven’t yet received all of the standard
treatments.
- You may feel you have more control over your situation and
are taking a more active role in your health care.
- You will likely receive more attention from your health
care team and more careful monitoring of your condition and the
possible side effects of treatment.
- Some study sponsors may pay for part or all of your medical
care and other expenses during the study. This is not true for all
clinical trials. Be sure you know who is expected to pay for your care
before you enroll in the study.
Some of the potential downsides of being in a study can
include the following:
- The new treatment may have unknown side effects or other
risks, which may or may not be more severe than those from existing
treatments. This is especially true of early phase trials.
- As with other forms of therapy, the new treatment may not
work for you, even if it helps others.
- There may be inconveniences such as more frequent testing,
as well as time and travel commitments.
- If you take part in a randomized clinical trial, you may
not have a choice about which treatment you receive. If the study is
blinded, you (and possibly your doctor) will not know which one you are
getting (although this information is available if needed). This will
be explained to you before you decide to take part.
- Insurers may not cover all of the costs of taking part in a
clinical trial (although they usually cover the costs for what would
normally be standard care). Be sure to talk to your insurance provider
and to someone involved with the study before you decide to take part,
so you know what you may have to pay for.
Answers to Some Common Questions
About Clinical Trials
Most people have some concerns about taking part in a clinical
trial, often because they’re not really sure about what it involves.
Gathering as much information as possible beforehand is the best way to
ensure that you will make the choice that is right for you.
Will there be risks?
Yes, risks are involved in clinical trials. By the same token, risks
are a part of any medical test, drug, or procedure. The risk may be
greater in a clinical trial because some aspects of any new treatment
are unknown. This is especially true of earlier phase clinical trials,
where the treatment has been studied in fewer people.
Perhaps a more important question is whether the risks are outweighed
by the possible benefits. People with cancer are often willing to
accept a certain amount of risk for a chance to be helped, but it is
always important to have a realistic idea about what this chance is.
Ask your doctor if he or she can give you a reasonable idea of what the
chance of benefit is, and exactly what this benefit is likely to be in
your case.
With this in mind, you can make a more informed decision -- one that is
right for you. Some people may decide that any chance of being helped
is worth the risk, while others may not.
Will I be a “guinea pig”?
There’s no denying that the ultimate purpose of a clinical trial is to
answer a medical question. People who take part in clinical trials may
need to do certain things or have certain tests done to stay in the
study.
But this does not mean that you will not receive excellent,
compassionate care while in the study. In fact, most people enrolled in
clinical trials appreciate the extra attention they receive from their
health care team. In 2005, the Coalition of Cancer Cooperative Groups
surveyed over 1,700 people with cancer concerning their awareness and
attitudes about clinical trials. Only a small portion took part in
clinical trials. But most of those who did were very satisfied: 96%
said they were treated with dignity and respect, 92% said they had a
positive experience, and 91% would recommend that family or friends
take part in a clinical trial if faced with cancer.
Will I get a placebo?
The vast majority of cancer clinical trials do not involve a placebo
unless it's in combination with another, active, drug. It would be
unethical to give someone an inactive medicine if it would deny him or
her the chance to get one that has already been shown to work.
Unfortunately with cancer, there are some situations in which no
effective treatments are known. In rare cases, testing a new treatment
against a placebo might be needed to prove that the treatment is better
than nothing at all.
The very least you should expect from any clinical trial is to be
offered the standard of care already in use. (See the section "What Are
the Phases of Clinical Trials?" for an example of a phase III study
involving a placebo.)
Will my information be kept confidential?To
the extent possible, all of your personal and medical information is
kept confidential. Of course, your health care team needs to have
access to this information to provide you with the best possible care,
just as they would if you were not a clinical trial.
Medical information that is important for the study, such as test
results, is usually transcribed onto special forms, which are then
given to the people analyzing the study results. Your information is
assigned a specific number or code - your name does not appear on the
forms. Occasionally, some members from the research team or from the
FDA may need to look at your medical records to verify the information
they were given is correct. But your personal information is not
submitted to them and is not used in any publications of study results.
Other
Questions You Should Ask Your Research Team
Each clinical trial is unique, with its own potential benefits
and risks. Before you decide to take part in a clinical trial, make
sure you have answers to the following questions:
- Why is this study being done?
- What is likely to happen in my case if I decide to take
part or decide not to take part in the study?
- What are my other options (standard treatments, other
studies)? What are their advantages and disadvantages?
- How much experience do you have with this particular
treatment? With clinical trials in general?
- What were the results in previous studies of this
treatment? How likely are they to apply to me?
- What kinds of treatments and tests would I need to have on
this study? How often are they done?
- Will this require an extra time or travel commitment on my
part?
- How could the study affect my daily life?
- What side effects might I expect from the study? (Remember
that there can also be side effects from standard treatments and from
the disease itself.)
- Will I have to be in the hospital for any parts of the
study? If so, how often and for how long?
- Will I still be seeing my regular doctor?
- Will I have any costs? Will any of the treatment be free?
Will my insurance cover the rest?
- If I am harmed as a result of the research, what treatment
will I be entitled to?
- How long will I remain in the study?
- Are there reasons I would be removed from the study? Are
there reasons the study might be stopped early?
- Is long-term follow-up care part of the study? What would
it involve?
- If the treatment is working for me, can I choose to
continue getting it even after the study ends?
- Are there others already taking part in the study whom I
could speak to?
- Will I be able to find out about the results of the study?
Involving trusted friends and family members may be a good
idea. They can help reassure you that you’re making a decision that’s
right for you. In addition, getting a second opinion from a doctor who
is not associated with the study can give you a broader sense of
whether this particular study is the best one to consider.
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Who
Will Look Out for Me as a Study Participant?
Several levels of safeguards are in place to help protect the
welfare of people who take part in clinical trials. There are still
risks involved with any study, but these safeguards try to reduce the
risk as much as possible.
Three basic principles, as outlined in the Belmont Report from
the late 1970s, provide the basis for research involving humans:
- Respect for persons: Recognizing that
all people should be respected and have the right to choose what
treatments they receive
- Beneficence: Protecting people from harm
by maximizing benefits and minimizing risks
- Justice: Trying to ensure that all
people share the benefits and burdens of research equally
These principles are upheld by individuals and groups at the
sites conducting research and by government agencies charged with
overseeing clinical trials. A very important part of patient protection
is the informed consent process, which is described
in detail in the section "What Would
Participating Involve for Me?"
Safeguards in Institutions
Centers conducting clinical trials have committees that review
all potential and ongoing clinical trials to protect the safety of
those in the study. These are required for all federally funded
clinical trials, but even privately sponsored studies typically undergo
such reviews.
Institutional review boards (IRBs): IRBs
are groups of people responsible for protecting the welfare of study
participants and making sure that studies comply with federal laws.
They are often made up of medical experts (doctors, nurses), other
scientists, and non-medical people. Not all of the people on the IRB
can come from only one of these groups. (In other words, an IRB
couldn’t be composed of just doctors.) Many institutions have their own
IRBs, although some smaller centers may use larger, "central" IRBs. The
federal Office of Human Research Protections (OHRP; see below) oversees
the activities of IRBs.
Researchers wanting to start a study must first submit the study
protocol (the plan describing the study in detail) to the IRB for
review. The IRB must decide if the study would be acceptable on
medical, ethical, and legal grounds. In other words, is the study
addressing a worthwhile question, and is it doing so in a way that
ensures the safety of those talking part as much as possible? One of
the most important functions of an IRB is to make sure the informed
consent form that people entering the study must sign is accurate,
complete, and easy to understand. Once a study begins, the IRB also
follows its progress on a regular basis to look for potential problems.
If you take part in a clinical trial, you can contact the study’s IRB
directly with any questions or concerns regarding safety.
Data safety monitoring boards (DSMBs): DSMBs
are used for phase III (and some earlier phase) studies. They are
committees comprised of doctors and other scientists not involved in
the study. Their job is to look at study statistics. They monitor the
results of the clinical trial at different time points and can stop a
study early (before all of the intended participants have been enrolled
or completed the study) if:
- it becomes clear that the new treatment is much more (or
much less) effective, so as to allow all study participants to get the
better treatment
- safety concerns arise (such as risks of the new treatment
clearly outweighing the benefits), so that no more people are exposed
to possible harm
The principal investigator (PI): The PI is
the doctor who is in charge of all aspects of a particular study.
Ultimately, the responsibility for patient safety in a clinical trial
lies with the PI. Part of this responsibility is letting the study
sponsor know right away when serious side effects occur.
PIs generally have years of experience in running clinical trials. They
are required to submit their credentials to the FDA before being
allowed to run any study.
Government Agencies
Several government agencies play roles in ensuring that all
research is conducted with patient safety in mind. These include:
Office of Human
Research Protections (OHRP): The OHRP is the government’s
main guardian of people’s safety and welfare in clinical trials. It was
established in 2000 to coordinate efforts to protect all people
involved in federally funded research. It enforces regulations
regarding the informed consent process, institutional review boards
(IRBs), and the participation of special populations in clinical
trials, such as children and those with mental disabilities.
The OHRP has temporarily suspended research activities at several
institutions in the last few years, including those in some major
research centers, until system flaws were corrected.
The OHRP also educates research centers and individuals to help them
comply with established clinical trials standards.
Food
and Drug Administration (FDA): The FDA has the
final say about whether or not a new treatment can be marketed to the
public; that is, whether or not doctors can begin prescribing it to
their patients. Once all phases of clinical trials on a new treatment
are completed, the FDA reviews the information and decides if it is
safe and effective enough to be approved.
But the FDA’s role in many clinical trials begins long before this. Any
sponsor seeking approval for a new treatment must submit all study
protocols to the FDA before the clinical trials are allowed to proceed.
Researchers who will be involved in the study must submit their
credentials for review as well.
The FDA also inspects (audits) sites conducting clinical trials,
especially if there is reason to think that proper procedures are not
being followed. If serious problems are found, the FDA can forbid a
particular site or doctor from doing any further research.
The authority of the FDA is not absolute, however. Clinical trials that
study treatments already on the market are not subject to the same FDA
regulations (although many are still done in a similar manner). And
substances considered to be "dietary supplements" do not need FDA
approval to be sold in the first place. Dietary supplement makers
aren’t required to prove that their products are safe or effective.
Therefore, they usually do not bother with the expense of conducting
clinical trials. A relatively small number of clinical trials are done
to study the effectiveness of dietary supplements; most of these are
funded by the National Institutes of Health (NIH).
National
Cancer Institute (NCI): The National Cancer
Institute, part of the NIH, sponsors many of the cancer clinical trials
going on at any one time, including those being conducted by
cooperative groups. Proposals for such studies must be approved by the
NCI before funding is granted. The NCI also inspects (audits) each site
involved in NCI-sponsored research at least once every 3 years.
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What’s
Out There? Finding Clinical Trials
People find out about clinical trials in different ways. Most
people who enter clinical trials do so after hearing about them from
their doctor. Many cancer patients actively look for clinical trials on
the Internet or in other places, hoping to find more options for
treatment. Some clinical trials are advertised directly to patients.
If you already have a particular clinical trial in mind, you
may want to go to the next section ("How Do
I Figure Out Which Study Is for Me?") to learn what you
should know about the study.
Types of Clinical Trial
Information
At this time there is no single place to get information on
all of the cancer clinical trials now enrolling patients. But there are
several resources you should be aware of. These resources can be
divided into2 main types -- clinical trials lists and clinical
trials matching services.
Clinical Trials Lists
These sources can provide the names and descriptions of clinical trials
of new treatments. If there is a particular study you are interested
in, you will probably be able to find it in a list. It will often
include a description of the study, the criteria for which patients are
eligible, and whom to contact. If you (or your health care providers)
are willing and able to read through descriptions of all the studies
listed for your cancer type, then a list may be all you need. Some
organizations that provide lists can help you narrow the list a little,
according to the kind of treatment you are looking for (chemotherapy,
immunotherapy, radiation therapy, etc.) and the stage of your cancer.
Clinical Trials Matching
Services
Over the past few years, several organizations have developed
computer-based systems to match patients with studies they may be
eligible for. These organizations generally offer their services online.
Each may differ somewhat in how it works. Some of the services allow
you to search for clinical trials without registering at the site. Even
if registration is required, they usually assure you that the
information will be kept confidential. Either way, you will likely have
to enter certain details, such as the type of cancer, the stage of the
disease, and any previous treatments you may have had. When provided
with this information, these systems can identify clinical trials for
which you are likely eligible, and save you the time and effort of
reading descriptions of studies that are not relevant to you. Some
firms also allow you to subscribe to mailing lists so that you are
informed as new studies open up.
While they are usually free to users, the business of most clinical
trials matching firms is based on receiving a fee for listing studies
or getting a "finder’s fee" from those running the studies when someone
enrolls. Because of this, there may be some differences in the way they
"rank" or present the studies to you.
How to Choose a Clinical
Trials Matching Service
Because different services vary in the way they work, be sure you
understand how the service you are looking at operates. Ask the
following questions. Note that the answers do not necessarily mean that
the service is not worth using:
- Is there a fee for using the service?
- Do I have to register to use the service?
- Does the service keep my information confidential?
- Where does the service get its list of clinical trials?
- Does the service rank the studies in any particular order?
Is this based on fees they are receiving?
- Can I contact the service via the Internet or by telephone?
The American Cancer
Society's Clinical Trials Matching Service
After reviewing the available matching services, the American Cancer
Society chose to work with the Coalition of Cancer Cooperative Groups
in an effort to provide a free, confidential, and reliable matching and
referral service to patients looking for clinical trials. The Coalition
of Cancer Cooperative Groups is a non-profit service organization
formed in 1997 with the goal of assuring access to cancer clinical
trials.
ACS offers TrialTracker, the screening tool developed by the
Coalition of Cancer Cooperative Groups, to help patients find high
quality care in clinical trials most appropriate for their medical
needs and personal preferences, while helping researchers study more
effective treatments for future patients.
The TrialChecktm database, developed
and maintained by the Coalition of Cancer Cooperative Groups, is a
comprehensive database that includes cooperative group, National Cancer
Institute, and industry trials. To our knowledge, this is the most
comprehensive matching database of cancer clinical trials available.
The clinical trials information provided by the American
Cancer Society is not biased in any way by whether the trial is funded
by the government or by pharmaceutical or biotechnology firms. It is
updated continually, as is the contact information that allows patients
to get in touch with the doctors and nurses at cancer centers running
each of the studies.
The American Cancer Society provides access to the TrialChecktm
system for our constituents through our Web site, http://www.cancer.org
(click on "Find
a Clinical Trial") and through a toll-free number
(1-800-303-5691) at our National Cancer Information Center. For
information about the Coalition of Cancer Cooperative Groups and the
TrialChecktm suite of products used by the
American Cancer Society’s clinical trials matching service, visit http://www.cancertrialshelp.org/patientsCaregivers/patientsCaregivers.jsp.
Other Clinical Trials Lists and
Matching Services
The National Cancer Institute (NCI)
sponsors most government-funded cancer clinical trials. The
NCI maintains a list of active studies (those
currently enrolling patients), as well as some privately funded
studies, in the Physician Data Query (PDQ) database. You can access the
list via their Web site at www.cancer.gov/clinialtrials
or by calling 1-800-4-CANCER. You can search the list by the type and
stage of cancer, by the type of study (for example, treatment or
prevention), or by geographic location.
The National Institutes of Health (NIH)
maintains an even larger database of clinical trials at www.clinicaltrials.gov,
but not all of these are cancer specific.
EmergingMed provides a free and confidential
matching and referral service to patients looking for clinical trials
at www.emergingmed.com.
CenterWatch (www.centerwatch.com)
is a publishing and information services company that maintains a list
of both industry-sponsored and government-funded clinical trials for
cancer and other diseases.
Major cancer centers
(and even some community hospitals and doctors’ offices) usually offer
lists on their Web sites of the clinical trials being conducted there.
You can find the major cancer centers closest to you on the American
Cancer Society Web site at www.cancer.org/docroot/FTC/ftc_0.asp.
Private companies, such as pharmaceutical
or biotechnology firms, may list the studies they are
sponsoring on their Web sites or offer toll-free numbers to call. Some
of these firms also offer matching systems for the studies they
sponsor. This can be helpful if you are interested in research on a
particular experimental treatment and know the company developing it
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How Do
I Figure Out Which Study Is for Me?
The study protocol is the written plan for how a clinical trial is to
be conducted. It is what is submitted to the FDA and to an
institutional review board (IRB) before a new treatment can be studied.
A protocol contains the following information:
- Why the study is being done (including the goals of the
study)
- Information about the treatment being tested, often
including results of previous studies
- The phase of the study and how many people will be enrolled
- Who is eligible for the study
- How the treatment is to be given
- What tests people will have done during the study and how
often
- Other information that will be collected on participants
Actual study protocols can be 100 pages or more in length, and they can
be very technical. Because these protocols are not written with
patients in mind, making sense of their language is not always easy.
The clinical trials lists available on the Web often include summaries
of these protocols, just highlighting some key points. Research team
members may have also have protocol summaries or other information
about the study they can share with you. Often, the most important
parts for patients looking for studies are the eligibility criteria and
any information available about the new treatment.
Eligibility Criteria (Inclusion
Criteria)
Each clinical trial has certain conditions, or criteria,
that patients must meet before they can enter the study. These are
necessary to ensure that the study can answer the specific questions it
was designed to answer. For cancer clinical trials, these criteria
usually have to do with:
- The type of cancer a person has
- The stage (extent) of the cancer
- Previous treatments a person must or must not have had
- The length of time since a person last received treatment
- Results of certain lab tests
- The medicines a person is taking
- Other medical conditions the person has
- Any previous history of another cancer
- A person’s activity level (also known as performance
status)
Other factors, such as a person’s age and sex, may also be
part of the criteria. There may be other conditions that are more
specific to each study, as well.
Advertisements andclinical trails lists may not contain all of
a study’s
eligibility criteria. If you’ve found a study you think you might
qualify for, you can usually contact someone invloved with the study
to get a full list of the eligibility criteria.
I Think I’m Eligible. Now What?
Once you’ve found a study that you think you’re eligible for, deciding
if it’s the right one for you can still be hard. There may even be more
than one that looks promising. Again, learning as much as you can is
key.
One option is to talk with someone connected to the study. This could
be the principal investigator (PI) -- the doctor in charge of the study
-- or a research coordinator. Research coordinators are usually nurses.
One of their jobs is to check to see if people meet eligibility
criteria before entering a study. They also make sure that the study
protocol is followed for each patient. Often they serve as a link
between study patients and their doctors.
Both PIs and research coordinators should be able to answer any
questions you have about the study. See the section "Should I Think
About Taking Part in a Clinical Trial?" for a list of questions you
should ask. Although they can give you answers about their particular
clinical trial, they are not likely to be helpful in discussing other
studies you may be considering. What’s more, they may be biased (even
if unintentionally) toward their own study.
If you haven’t already, talk to your own doctor about studies you are
looking at. Bring in whatever information you can, so that your doctor
can help you judge what might be right for you. While no doctor knows
about every clinical trial being done, yours is most familiar with your
medical situation and can probably tell you whether the study is worth
considering. This discussion can be time consuming, so you may need to
make a special appointment to allow him or her enough time to look over
the information you provide.
You may also want to get a second opinion from a doctor not connected
to the studies you are considering. Doctors who are well known in their
fields are usually up on the latest experimental treatments, and they
may be able to point to ones that appear more promising.
If you have access to the Internet, you can do some research on your
own. Try to find out if the new treatment has been studied before or if
it is being studied now in other diseases, as well as if any results
are available. If this is hard for you, have someone close to you help
or do it for you. People with a medical background may have an easier
time sorting through such information.
Finally, talk to friends and family members you trust. While the
decision is ultimately yours, their opinions may provide you with
insight into things you had not considered.
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What
About Cost? Will My Insurance Cover It?
This is a crucial question to ask before deciding to take part in a
clinical trial. Recent studies have shown that the overall costs of
taking part in a clinical trial are not much more than the costs of
treatment outside of a study. Still, insurance coverage can vary
widely.
When insurers do cover costs related to clinical trials, it is usually
only for tests, treatments, or doctor’s visits that would have been
part of your treatment plan if you were not taking part in a study. In
other words, they are not likely to pay for special tests or treatments
you are getting solely because you’re in the study.
The study sponsor (whether it is the government or a pharmaceutical or
biotechnology company) usually provides the new treatment at no cost
and pays for special testing or extra doctor visits. Some sponsors may
pay for more than this; for example, some may offer to compensate for
travel time and mileage. It is important to find out what they will pay
for before entering the study.
Private Insurers
In the past, insurers were sometimes reluctant to pay for any of the
costs related to a clinical trial. Their concern was that they would be
paying for treatments that had not been proven to be effective.
In recent years, many (but not all) major insurance providers have
volunteered to cover some of the costs of clinical trials. However,
they may limit which types of trials they will cover. They are more
likely to pay for costs from phase II or III clinical trials, but they
generally look at each request on a case-by-case basis.
Medicare
Medicare covers any cancer care normally covered by Medicare
when it is part of either 1) a clinical trial for the diagnosis and
treatment of cancer; or 2) a clinical trial funded by the National
Cancer Institute (NCI), NCI-Designated Cancer Centers, NCI-Sponsored
Clinical Trials Cooperative Groups, or another federal agency that
funds cancer research. This care may include the following:
Routine tests, procedures, and doctor visits
- Services or items that are part of the experimental
treatment, such as costs to administer investigational drugs
- Health care associated with being in a clinical trial,
such as a test or a hospitalization due to a side effect or
complication
What costs are not covered?
- Investigational drugs, items, or services that are being
tested as part of the clinical trial
- Items or services used only to collect data for the
clinical trial
- Anything being provided free by the sponsor of the
clinical trial
- Any coinsurance and deductibles
Cancer prevention trials are not currently covered by
Medicare. If you are not sure whether your trial meets all of the
requirements, discuss these concerns with your doctor or call the
Medicare information number (1-800-633-4227). Other trials may be
covered, so be sure to ask about any clinical trial before you begin
taking part in one that may not be covered.
Laws Regarding Clinical Trials
Coverage
In recognizing the importance of clinical trials, many states
have passed laws about insurance coverage for research studies. Several
more states are now considering legislation. A few states have worked
out voluntary agreements with insurance companies to provide clinical
trials coverage.
The types of studies and exact coverage required by these laws vary
from state to state -- some cover all clinical trials, while others may
cover only certain phases of clinical trials. For a list of state
clinical trials insurance laws, see our document, "Clinical
Trials: State Laws Regarding Insurance Coverage".
The federal government may soon become involved as well. Bills
have recently been introduced in both houses of Congress that, if
passed, would require insurers to provide coverage for certain clinical
trials. This would allow more people to participate.
What You Can Do
If possible, find out what your insurer will cover before you get
involved in a clinical trial.
Find out if laws have been passed in your state that require coverage
of routine costs of clinical trials. Then gather as much information as
you can about the study and contact your insurance provider to find out
about coverage. Many providers may not be able to give you a simple
"yes" or "no" answer up front, as they may review claims on a
case-by-case basis. You may, however, be able to find out if they’ve
covered costs for clinical trials similar to yours (or ones that
studied the same treatment) in the past.
Have a summary of your study available, and, if possible, any results
of previous studies of the treatment. You may need to ask your doctor
or the study’s research coordinator for help in gathering this
information. If needed, your doctor may be able to further justify to
your insurer as to why this study is appropriate for you.
Study sponsors are often eager to recruit eligible patients for their
clinical trials, and they may be willing to cover costs if your
insurance does not. If needed, ask your doctor or the research
coordinator to contact the study sponsor on your behalf.
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What
Would Taking Part in a Clinical Trial Involve for Me?
Having an idea of what you can expect from taking part in a
study can help relieve some of your concerns and make things go more
smoothly. The first thing you will need to do is give your informed
consent to participate.
Informed Consent
The people running the study are required to get your written, informed
consent before you take part in any way (often even before you have any
needed tests to see if you are eligible for the study). In the informed
consent process, the researchers (doctors or nurses) will explain the
details of the study to you and answer all of your questions and
concerns.
You are then given a written consent form to sign. While consent forms
are not all the same, they should include the following:
- the reason for the study (what the researchers hope to find
out)
- who is eligible to take part in the study
- what is known about the new type of treatment
- the possible risks and benefits of the new treatment (based
on what is known so far)
- alternative treatments that may be an option for you
- the design of the study (whether it is randomized, double
blinded, etc.)
- how many and what types of tests and doctor’s visits are
involved
- who is responsible for the costs of the clinical trial
(tests, doctor’s visits, etc.) and for the costs if you need additional
care as a result of the clinical trial
- a statement about how your identity will be protected
- a statement about the voluntary nature of the study and
your right to leave the study at any time without fear of compromised
care
- contact information if you have further questions
Before you sign the consent form, ask questions. Be sure someone from
the research team goes over the form with you in detail. Efforts are
made to make consent forms as understandable as possible, but there
still may be terms or ideas that are confusing to you. You may want to
bring someone along with you to the meeting to make sure all your
concerns are addressed.
Be sure you understand what is involved and what is expected of you.
You may want to explain what you heard back to your doctor or nurse to
make sure you got it right. Recent surveys have shown that while most
people are satisfied with the informed consent process, over half do
not understand some of the main points on the consent form.
Finally, don’t be rushed into making a decision. Take the consent form
home with you if needed. Get opinions from trusted family members and
friends. If possible, you may want to get a second opinion from another
doctor as well.
Taking Part in the Study
Once you’ve signed the consent form, you are ready to take part in the
study. You will most likely need to have blood tests or imaging tests
done before you start treatment (if you haven’t had them recently). A
full history and physical exam are also usually required. The results
are needed beforehand to confirm that you meet the eligibility criteria
and to help ensure your safety.
As mentioned earlier, some studies may require you to stay in a
hospital for a day or two to get treatment. In other studies the
participants are treated much the same way as other patients getting
treatment outside of a clinical trial.
You may have tests done more frequently, both to determine how well the
treatment is going and to look out for your welfare. You will likely
receive more attention as a study participant than you would otherwise.
The doctors and nurses may examine you more often and will want to know
if you experience any side effects (called "adverse events") while
getting treatment.
Because the possible complications may not fully be known, it is very
important to let them know about anything out of the ordinary. They can
then decide if what you are experiencing is related to the study and if
the symptom needs to be treated or your therapy needs to be
changed.Your participation in the study may end for any number of
reasons:
- You complete treatment on the study.
- The treatment does not appear to be working for you.
- You have serious side effects while in the study.
- The study itself is stopped early because the treatment
either has proven to be effective or has proven to be too harmful in
other participants.
- You decide to leave the study.
Once out of the study, you may still be monitored for a time so that
researchers can continue to get an idea of how you are doing.
Some studies allow you to continue to receive the new treatment even
after the study ends. This is known as "open enrollment." This option
varies among clinical trials, so be sure to ask about it before you
begin.
What If I Want to Leave the
Study Early?
You will be told several times before you enter the study that your
participation is always voluntary.
This is an important point. You have the right to leave the study at any time, for any reason.
Your doctor will still take care of you to the best of his or her
ability.
Regardless of when or why you leave the study, you may be asked if the
researchers can follow up with you from time to time to see how you are
doing. This may provide them with important information and can also
help ensure your safety, even after you leave the study.
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What
If I’m Not Eligible for a Clinical Trial?
While some people may be too ill or have other problems that exclude
them from clinical trials, most people will probably be eligible for
some type of study, even if they’ve had several treatments already. Of
course, not all studies you are eligible for may be a good fit for you.
It’s always important to understand the purpose of the study and to
have a realistic idea of the possible risks and benefits for you.
Clinical trials offer the best avenue of access to experimental
treatments. Study protocols, which are written based on prior study
results, are strictly followed and patients are monitored carefully.
Some people may be interested in a certain treatment that is only
available in clinical trials but may not meet the eligibility criteria
outlined for the studies. In some of these cases, a person’s doctor may
ask the study sponsor if they can get an eligibility waiver or
special exception
to allow the person into the study, even though he or she does not
strictly meet all of the criteria. This decision is usually made by the
study’s principal investigator, sometimes in consultation with others
involved in the study. If entered, the person is treated according to
the study protocol (the same tests, doctor’s visits, follow up, etc.),
although the results are not included in the final study results.
In other cases, the studies may have already enrolled enough people and
aren’t accepting more participants.
At times, there may be ways to get access to treatments that are in
late phase clinical trials but not yet FDA approved. These are usually
referred to as "expanded access" or "compassionate use" programs. In
recent years the FDA has expanded these programs to allow some patients
who urgently need these treatments to be able to get them.
But they are not always easy to get access to. The programs are
voluntary on the part of the company making the treatment. They are not
required to provide the treatment in these settings, and some companies
may decide not to for various reasons (manufacturing issues, excess
demand, etc.). Because of the amount of effort and paperwork involved,
the process can at times be slow (weeks to months).
Some of these programs are described below. All require your informed
consent, much the same as for any clinical trial.
Treatment Use of an Investigational New Drug (Treatment IND)
In some cases, if a treatment is showing promise in late phase clinical
trials, the maker may apply to the FDA for a treatment IND. This
is much like setting up a new study, but it is meant mainly as a way
for patients with no other options to get early access to the
treatment, rather than to help get the treatment FDA approved.
This can sometimes be used when a person would not have met the
eligibility criteria for the clinical trials or when the studies are
already closed to further enrollment. It requires that the person have
a "life-threatening" or "severely debilitating" condition for which
there are no other treatment options.
Your doctor would need to get in touch with the treatment manufacturer
to see if such a program exists and what would be needed for you to
enter it. As with clinical trials, these programs have to have a
protocol that meets FDA approval, as well as institutional review board
(IRB) approval in many cases.
With a treatment IND, the supplier may or may not charge for the
treatment in question. It is important to find out beforehand whether
you or your insurance company might be responsible for paying for the
treatment.
Single Patient and Emergency Use
of an Investigational New Drug (IND)
A single patient IND
is used to get access to an unapproved treatment for a single person
with a serious condition who is not eligible for a clinical trial. It
is similar to a treatment IND in some ways. It does not require that
the clinical trial protocol be followed, but it would likely require
that your doctor spell out in detail the proposed treatment plan.
To get a single patient IND, your doctor would need to contact the
treatment manufacturer to see if they would supply it. He or she would
then need to have the proposed treatment protocol approved by the IRB
and the FDA before treatment would be allowed to begin.
An emergency IND
can be used when there isn’t time to get approval from the IRB. Your
doctor would need to contact the manufacturer to see if the treatment
is available and then file the needed paperwork with the FDA. While IRB
approval is not needed before starting treatment, the IRB would have to
be notified of the situation and would have to approve future uses.
Conclusions
Clinical trials can offer benefits for many people during their cancer
experience. These may include access to newer or more treatment
options, getting more involved medical care, and having a greater sense
of control over one’s situation. But by their nature, clinical trials
involve some possible risks and downsides as well, and they may not be
right for everyone. Your decision on whether to seek or enter a
clinical trial should be based on a realistic understanding of these
possible risks and benefits.
If you are thinking about entering a clinical trial, there are many
groups, including the American Cancer Society, who can help guide you
through the experience.
Additional Resources
More Information from
Your American Cancer Society
The following information may also be helpful to you. These materials
may be ordered from our toll-free number, 1-800-ACS-2345.
Clinical Trials: State Insurance Coverage Laws
National Organizations
and Web Sites
In addition to the American Cancer Society, other sources of patient
information and support include:
National Cancer Institute
Telephone: 1-800-422-6237 (1-800-4-CANCER)
Internet address: www.cancer.gov
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References
Bennett C, Adams J, Knox K, et al. Clinical trials: Are they a
good buy? J Clin Oncol. 2001;19:4330-4339.
Coalition of Cancer Cooperative Groups. Most cancer patients
satisfied with clinical trial experience, yet, few aware of
opportunity: study shows improved physician-patient communications
could increase enrollment in cancer clinical trials [press release].
June 5, 2006. Available at:
www.cancertrialshelp.org/press/pressTemplate.jsp?ID=2120&VID=10009.
Accessed July 13, 2006.
ECRI: Should I Enter a Clinical Trial? A Patient
Reference Guide for Adults with a Serious or Life-Threatening Illness.
ECRI; February 2002. Available at
www.ecri.org/Patient_Information/Patient_Reference_Guide/prg.pdf .
Accessed July 19, 2006.
Food and Drug Administration. Access to unapproved drugs.
Available at: www.fda.gov/cder/cancer/access.htm. Accessed July 19,
2006.
Getz K, Borfitz D. Informed Consent: The Consumer’s
Guide to the Risks and Benefits of Volunteering for Clinical Trials.
Boston, Mass: CenterWatch; 2002.
National Cancer Institute: Learning About Clinical
Trials. National Cancer Institute.
Available at www.nci.nih.gov/clinicaltrials/learning. Accessed July 19,
2006.
Pharmaceutical Research and Manufacturers
of America (PhRMA) - Medicines in Developmnt for Cancer. May 2005.
Available at www.phrma.org/files/Cancer%20Survey.pdf.
Accessed Jul 12, 2006.
Revised: 08/03/2006
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