It seems as if everybody wants information about you
these days. Try to get that rebate the salesman promised on your
new computer, and the company wants to know how old you are, how
much money you make and whether you went to college.
Phone surveys want to know what you think of your
congressional representative, whether you’re planning to sell
your house and what radio stations you listen to.
If you have a neuromuscular disease, there’s
a good chance that you’ve been asked to participate in some
sort of data collection, known as a registry, database or survey,
about you, your family and your disease. And understandably, not
everyone feels entirely comfortable going on record with this information.
Registries are proliferating these days, and we asked
a few of the people who oversee them or participate in them what
advice they have for those who are thinking of signing up.
Estimate how much of your time participation will
take.
Filling out forms, online or on paper, or answering
questions orally, can be time-consuming.
Joining a registry usually takes minutes to a few hours, but sometimes
more if physician exams or tissue samples are part of the process.
“There’s a figurative line you have to
draw to make sure that things of substance are in the registry,”
says Michael Shy at Wayne State University in Detroit about designing
a registry. He oversees the Charcot-Marie-Tooth (CMT) Disease North
American Database, through an MDA grant.
“If you make it easier for the patient, you
get less information. Our database takes a couple of hours to fill
out, because it requires a detailed medical history, and it asks
about symptoms and signs and if they’re taking this or that
medication.
“You can make [the survey] shorter, but then
you don’t get as much data. We chose to go the inclusive route.”
Assure yourself that your privacy will be protected.
This is probably the area of greatest concern for
families. HIPAA, the Health Insurance Portability and Accountability
Act, went into effect in 2003 and is designed to protect health
information from misuse.
When Diane Hardin, 57, of Madison Heights, Mich.,
was deciding whether to participate in the CMT North American Database,
privacy was a major concern. She did participate, but only after
assuring herself that “only CMT-type information would be
used, not personal information.”
A reputable survey will go to great lengths to ensure
that participants’ privacy is protected and to tell them how
this will be accomplished. For instance, the information about the
CMT database clearly states, “Your name and identifying information
will NEVER be released without your written consent.”
One way to ensure privacy is for participants to enter
identifiable data, such as names and addresses. Then a small number
of database administrators will turn this information into numbers
and release it only as blocks of statistics that can be used for
research but can’t be used to identify any particular person.
If a researcher is looking for people to take part
in a clinical trial, he or she has to go to the administrators,
who will then contact qualifying participants and invite them to
apply for the trial.
Some databases, such as Genzyme’s Pompe Registry,
assure privacy by never collecting any identifiable information.
Understand the purpose of the registry or survey.
Most people who take the time to participate in a
registry want to know how the information will ultimately be used.
Physicians, says Jennifer Farmer, registry coordinator
for the Friedreich’s Ataxia Research Alliance (FARA), based
in Springfield, Va., can “learn more about the range of clinical
symptoms, or how people with the same condition change over time,”
information that’s often lacking for rare diseases.
And, she notes, if they want to conduct a research
study, a registry can dramatically reduce the time and effort they
have to spend finding participants.
Richard Finkel, an MDA clinic co-director at Children’s
Hospital of Philadelphia, had clinical trials in mind when he designed
a survey for parents of children with spinal muscular atrophy. (The
North American part of this survey closed last month.)
He says that at a meeting with the Food and Drug Administration
last year, speakers emphasized that the FDA is looking for trials
that measure “clinically meaningful” changes, differences
that mean something to patients and parents, not just to statisticians.
“If a drug improved a 10-meter walk test by
one second, that might be statistically significant compared to
the placebo,” Finkel says. “But what difference does
that make to a child?”
The FDA wants to see meaningful measures built into
trials from the start. “I wanted to see what benefit [parents]
considered meaningful, so that investigators contemplating drug
trials would know what to go after,” Finkel says.
He also wanted the parent survey to help him design
trials that would enroll and retain adequate numbers of subjects.
“You need to design a trial that’s going
to be something that parents or patients find acceptable. If you
design a very difficult trial that asks too much, you may get a
very low enrollment, or you may find that retention of patients
in the trial may be poor.”
With that in mind, he built into the survey questions
about how much risk parents were willing to take, how long they
were willing to participate, how far they would travel to a study
site, and whether they would enter their child into a study with
a placebo group.
Robert Miller, who directs the MDA/ALS Center at California
Pacific Medical Center in San Francisco, chairs the advisory board
for the ALS CARE registry and has an MDA grant to oversee the ALS
Connection.
The CARE registry, in operation since 1996, now includes
data on more than 6,000 people with ALS, most of whom are being
cared for at major medical centers. It’s paper-based (although
the forms can be downloaded from the Web) and requires participation
by the patient, his or her physician, and his or her caregiver.
“We’ve learned a lot about what treatments
patients are using, whether the treatments were useful, and also
whether the evidence-based guidelines approved by the American Academy
of Neurology are being followed,” Miller says.
He adds that leads for new research projects have
come from the ALS CARE program.
“For example, the finding that 35 percent of
ALS patients in the program were veterans was a big surprise and
led to a whole series of studies about the risk of ALS in veterans
of military service. These have been very fruitful.”
The ALS Connection registry is newer and is designed
to reach those who aren’t attending large specialty clinics.
“They can participate in this research effort and help us
to learn more about the standard of care and the satisfaction with
care,” Miller says.
Find out who’s funding and overseeing the registry
or survey.
Diane Hardin says she had a great advantage over most
potential registrants when she signed on to the CMT Database because
neurologist Michael Shy oversees it, and he’s one of her doctors.
“I need to know that the person who is doing it is honest,
and I think Dr. Shy is,” she says.
Not everyone has that advantage, of course, but the
registry’s Web site or paper information should clearly tell
you what organization, government agency or company is sponsoring
the research. It also should provide you with contact information
in case you have further questions.
Find out what direct benefits participation may have
for you.
Biomedical engineer Chris Dandrea is the project manager
for a Pompe’s disease (acid maltase deficiency) registry at
the bio-pharmaceutical company Genzyme, which received market approval
this spring for Myozyme, a treatment for Pompe’s disease.
The Pompe Registry enables a physician to answer questions
about a patient’s clinical status and quality of life, via
a secure, Web-based system, after the person has provided written
permission.
Dandrea says that roughly a third of physicians who
participate in the registry manage multiple Pompe’s patients
in all aspects of the disease. The remaining doctors are typically
focused on managing one or more symptoms of the disease, based on
their specialties.
Because Pompe’s disease is so rare, Dandrea says, “educating
the medical community is critical. The registry is one way to connect
the experience of Pompe disease experts with those physicians who
may be managing their first Pompe patient.
“One of the benefits of registry participation
to the patient is that their physician is now a member of a network
of Pompe disease experts around the world.”
He also notes that Genzyme sponsors an annual meeting
of medical professionals involved with the registry.
“They can talk about new developments, new uses
for the registry, and new analyses and publications using registry
data. And we have also had leaders of patient organizations share
their perspective about their quality of life and experiences with
treatment.”
Since the approval of Myozyme in April, Dandrea says,
“the big question is, because new treatment and support measures
will impact the natural course of the disease over time, what are
the health consequences? What new medical trends emerge as patients
live longer?
“What will further improve patient care? Because
at the end of the day, for these patients, that’s what you
want to do. And one key to improving patient care is to collect
and analyze as much patient data as possible over the long term.”
For people with disorders such as Friedreich’s
ataxia, for which disease-modifying treatments are lacking, a desire
to accelerate clinical research or to take part in it can be the
main motivation for joining a registry.
The FARA Patient Registry is one way for people with
Friedreich’s ataxia to learn about clinical trials they might
want to take part in, says Farmer, although not all registrants
will be eligible for all clinical trials. (It’s also important,
she notes, to understand that joining a registry doesn’t mean
that you have to participate in future research studies.)
