Author: Mia James
You have been delivered a cancer diagnosis.
The next step? Creating a treatment plan that
truly represents your unique needs and goals.
As this process evolves, you may discover that
participation in a clinical trial is right for you.
This decision, which should be made together with
your health care team and take into account the specifics
of your diagnosis and other factors (personal as well as
treatment- and disease-related), is ultimately yours alone,
but you’ll be better able to make an appropriate decision
with an understanding of the clinical trials process.
What are clinical trials?
Clinical trials are medical research studies that test the safety and the
effectiveness of new therapies for screening, prevention, diagnosis, or
treatment of a disease in human participants. These trials, which test
therapies that have already been evaluated for safety in pre-clinical
trials, help determine the benefits as well as the side effects of potential
new methods of cancer treatment and prevention.
Why choose a clinical trial?
When it comes to patient participation in clinical trials, Maurie
Markman, MD, senior vice president of clinical affairs and national director of medical oncology at Cancer Treatment Centers of America® (CTCA), says that
patient needs should always come before research
findings. “Individuals who participate in trials
are hopefully helping themselves and at the same
time helping future patients,” he explains.
For this reason Dr. Markman supports patients’
decisions to enroll in trials only if participation
truly serves their treatment needs and goals. The
possibility of contributing to useful research
findings is a secondary concern. “The only
reason we would even consider offering a trial is
if it’s in the patient’s best interest,” he explains.
To establish whether a specific trial is a good
choice, Dr. Markman suggests that you ask
yourself a basic question: Why should I do this?
The question is critical, he says, to force you to
weigh the risks and the benefits and because
a trial may add more time and more tests to a
The potential benefit of participating in a trial
is that the trial therapy will be effective—that
it will shrink the tumor, improve symptoms,
or extend survival. Risks, on the other hand,
include possible side effects, the potential that
the trial will extend treatment time and add to
the number of tests to undergo, and, ultimately,
the possibility that the therapy won’t be effective.
Also important to add to the potential risk
category, Dr. Markman says, is the fact that
there may not yet be much information about
the therapy. “With some of the experimental
drugs, we actually don’t know a lot about the side
effects,” he explains.
Learning as much as possible about a trial will
help you decide whether participation is in your
best interest. Depending on the product being
investigated, there may be information available
online about previous results and known side
effects. At many treatment centers, Dr. Markman
says, including CTCA, patients can also access
information about current trials in documents
produced by an institutional review board
(a group responsible for reviewing each trial
before it is accepted by the treatment center)
that explain in patient-friendly language the
purpose of the trial, possible side effects, and
time and effort required by participants. After
reviewing all available information, you should
discuss the trial with your nurses, doctors, and
other members of your health care team, Dr.
Paying for clinical trials
An additional consideration in your decision
will be the cost of the trial and how expenses will
be covered. “If there’s an experimental drug—
one that has not been approved by the US Food
and Drug Administration—there will be no
charge for that drug,” Dr. Markman says. In this
case, the trial’s sponsor—the pharmaceutical
company behind the drug—would cover those
costs. The sponsor would also be responsible for
any tests that would not be part of standard care
for the disease in question (care that you would
receive outside of the trial).
When it comes to tests or drugs that would
be part of standard care, you or your insurer are
responsible for those costs. This can get tricky,
says Dr. Markman, because some insurers may
consider this an opportunity not to pay.
In some cases, states have stepped in to create
laws related to this scenario. “Some states have
basically mandated that if an insurer is licensed
and registered in that state and would otherwise
have paid, they’re going to have to pay if the
patient is on a trial,” Dr. Markman says. (Stateby-
state information is available through the
National Cancer Institute website at cancer.gov/
To avoid complications and unexpected
expenses, Dr. Markman suggests learning about the financial aspects of a trial before enrolling—then have the
information evaluated before beginning the trials process and
work with insurers to determine what will be covered.
Questions to ask
Before enrolling in a trial, Dr. Markman recommends asking
your health care team the following questions:
- Why should I pursue this trial versus some other
form of therapy? (What are my other choices?)
- What side effects are anticipated?
- What is known about the potential benefits?
- What extra time and effort will be required of me
(and my family) compared with other available
- Are there any additional financial costs? What
part will be covered by somebody other than my
insurance company? What part will be covered by
my insurance? What part am I expected to cover?
It’s a personal decision
Though there are many important considerations in
choosing whether to enroll in a clinical trial, the primary
question remains constant: Is the trial in your best interest?
Remaining focused on this critical question will help form a
truly personal treatment plan based on your individual needs
A Patient Perspective
A survivor and three-time clinical trial
participant shares her insights.
As a three-time survivor of melanoma and a three-time clinical
trial participant, Meg Monday of Hailey, Idaho, qualifies as
an expert on the trials process from a patient’s perspective.
Though each diagnosis and subsequent clinical trial was
unique, Meg’s motivation to participate was similar each
time around: she wanted the chance to receive a therapy that
might be more effective than the current standard of care.
Meg found effective therapy for all three melanoma diagnoses
in three separate trials. She entered the first trial (for
early-stage disease) knowing that she’d receive the standard
of care and also have the chance of getting a superior therapy.
With the two subsequent later-stage diagnoses, Meg says that
trials made sense because there was no effective treatment on
the market at that time. “For the disease I had,” she explains,
“there really wasn’t a standard of care.” Fortunately, Meg
found effective treatment in all three of the trials she entered.
Despite Meg’s positive clinical trials experiences, she acknowledges
that trials aren’t for everyone. Anyone thinking
about enrolling in a trial, she says, should consider carefully
some key points and of course discuss these and all concerns
with a doctor.
A clear understanding of potential risks and benefits is critical
when you’re deciding whether or not to enroll in a trial,
Meg says. This entails knowing the effectiveness of the standard
of care for your diagnosis and the potential benefits and
risks of the trial.
“Really ask your doctor,” Meg says, “‘What can I expect from
this?’” Ask also what the goals of this trial are for you, such as
how much your outcomes might be improved with the trial
Meg also recommends asking yourself how comfortable
you are with what you might experience in the trial. For example,
if you’re considering a placebo-controlled trial (where one
group receives the therapy and the other receives a placebo),
think about whether you’re comfortable receiving a placebo
instead of the therapy (even though participants receiving
placebo are able to switch to therapy if it’s proven effective).