Patients seeking to participate in a clinical trial often face
an obstacle: Their health insurer could limit their coverage when
they enter the experimental treatment program. That's about to end.
Starting January 1, a little-noticed provision of the Affordable
Care Act bans insurers from denying routine medical services to
clinical trial participants--and that could open the door for many
patients seeking potentially life-saving treatments for cancer,
heart disease and other serious medical conditions.
But many of these patients are likely to run into another
barrier: Chances are, they'll be on their own navigating the maze
of thousands of government, academic and private research
experiments. Many patients may not even know that a clinical trial
is an option, says Dr. Ted Gansler, director of medical content for
the American Cancer Society.
Gansler says that he expects the new health care law will raise
awareness of clinical trials. The law was intended "to remove an
obstacle for patients" to participate in clinical trials, he
A clinical trial helps determine whether drugs, medical devices,
and new therapies and vaccines that hold promise in the laboratory
are safe and effective for humans. Some trials examine whether
already approved drugs can be effectively used for other diseases
or work better when combined with other treatments. Still other
clinical trials test new surgeries or therapies that could prevent
disease or a recurrence of illness.
Retired engineer M. Dennis Sisolak, 72, of Bel Air, Md., entered
a 15-month clinical trial at the Sidney Kimmel Comprehensive Cancer
Center at Johns Hopkins Hospital in 2008, a year after being
diagnosed with advanced kidney cancer. Standard treatment had
failed to halt the cancer's progression.
Sisolak's trial tested an immunotherapy drug--one designed to
get rid of the cancer rather than slow its spread. "I'm feeling
great," says Sisolak, who has had clean scans. "There is no cancer.
I haven't taken any cancer meds for two years." Not everyone sees
such positive results. Treatments tested in clinical trials may
result in outcomes no better than established treatments. In some
cases, tested treatments can worsen the illness and even result in
death. Adverse reactions and unpleasant side effects also are
Depending on the needs of the researchers, you can enter a trial
at one of three phases. Phase I trials have typically 15 to 30
patients and determine safe doses and side effects. A Phase II
trial includes about 100 patients and helps determine how well a
drug or treatment works and whether it is safe. In Phase III
trials, a drug or treatment is given to several hundred to several
thousand participants to confirm its effectiveness compared with
the current standard treatments.
Several obstacles have discouraged patients from participating.
For one thing, doctors typically don't discuss a clinical trial
with patients as a treatment option. Also, many patients who enter
trials face significant out-of-pocket costs. The researchers pay
for such expenses as the drugs, medical devices and treatments that
are being tested. But insurance companies often deny coverage for
routine services related to the trial, such as temporary
hospitalization or monitoring. The new health care law requires
insurers to pay for these costs. Medicare already covers routine
costs related to most government-sponsored trials.
Moreover, several myths discourage participation, experts say.
One is that a patient in a Phase III trial will receive only a
placebo if they are placed in a control group, which is made up of
patients who do not get the experimental treatment. "You will never
get inferior care," says Patricia Haugen, a breast cancer survivor,
former clinical trial patient and consumer advocate in Sioux Falls,
S.D. Patients on the placebo receive the current standard
treatment, Haugen says.
Another fallacy: Clinical trials are a last-resort option. "That
misunderstanding can prevent a patient from getting the benefit of
a new treatment," says Dina Lansey, a registered nurse and clinical
research recruitment specialist at Hopkins' Kimmel cancer center,
in Baltimore, Md.
Although many patients seek out a trial when standard treatment
fails, clinical trials can be a first-choice treatment option, too.
"Many clinical trials test treatments for earlier forms of cancer,"
To be eligible for a trial, a patient generally must enroll
after diagnosis and before treatment begins--or when standard
treatment fails. Not all trials test new treatments on sick
patients, however. Some studies aimed at preventing a disease seek
healthy participants who may be at risk for a medical condition.
Many trials look at new ways to detect, diagnose or understand a
Among the 474 clinical trials ongoing at Hopkins' cancer center,
for example, some examine ways to better manage nausea or pain,
while several look at various approaches to support patients and
their families. Others focus on finding new ways to screen for a
Finding a Perfect Match
But looking for a clinical trial can be daunting--and sometimes
unsuccessful. Cynthia Solomon, 66, of Sonoma, Cal., in September
searched for a clinical trial for terminal stage 4 ovarian cancer
on ClinicalTrials.gov, a service of the National Institutes of
Health that lists more than 150,000 publicly and privately funded
Solomon, a retired business owner, was seeking a trial that was
testing a particular type of drug treatment. She says most of the
studies she found were outdated or closed. Solomon's doctors at the
University of California-San Francisco only knew of trials at the
research facilities there. "Doctors rarely bring up clinical trials
unless their institution has one to offer, and they are rarely
aware of other clinical trials," says Solomon. She gave up
searching for a clinical trial and is focusing on her quality of
Meanwhile, Sisolak says he only considered a clinical trial
after his hematologist called his former professor at Hopkins
medical school to ask about possible research studies. The
professor knew of the cutting-edge research at the university.
Despite the difficulties in navigating the system, there could
well be a trial for you. If your doctor does not know of any, turn
to the Internet. A good place to start your search, despite
Solomon's disappointment, is
. A recent search, for example, found that 307 trials for
Parkinson's disease were recruiting participants. You can narrow
your search by location, age, trial phase and other factors.
Another good resource is the nonprofit Center for Information &
Study on Clinical Research Participation (
For cancer trials, look into the American Cancer Society's free
Clinical Trials Matching Service at
. At the National Cancer Institute's Web site (
), you can search among the 12,000 trials that are accepting
patients, and you can review the results of completed research.
If you've been diagnosed with a specific condition, call its
professional advocacy organization. The TrialMatch program at the
Alzheimer's Association will try to put patients in touch with
studies. Alzheimer's patients and their families also should check
out the National Institute on Aging (
). Among 23 studies in California, researchers are recruiting
individuals with early-onset Alzheimer's to test the impact of
Gansler says that many databases will screen for patients'
clinical details. Often, the treatment being studied "is not at all
relevant to their type of cancer or the stage of their cancer," he
For example, Gansler says, initial human studies of new drugs
focus on finding a safe dose and studying side effects. These
trials could be a reasonable option for a patient with advanced
cancer that has returned after other treatments. If you are
accepted into a clinical trial, you will be given "informed
consent" documents, which will describe the details of the
treatment. Do not enroll until you've read the documents.
"Sometimes consent documents can be intimidating, but they spell
out the risks and benefits," Haugen says. Ask your doctor to go
over these documents with you.
Lansey says patients and their families need to understand the
number of visits that will be required, possible side effects,
risks and benefits, and what the treatment entails. "Patients and
their families are encouraged to ask questions throughout this
discussion," Lansey says.
Find out about any out-of-pocket expenses, including lodging and
food if you travel for a clinical trial. And, remember, clinical
trials are voluntary and you can drop out at any time.