Medical Heroes Newsletter
A Patient's Story
The other side of the needle: An investigator’s experience as a patient
Jürgen Venitz, MD, Ph.D. thought he understood every
aspect of the clinical trial process.
During the 1980s, as a medical student in Germany, he'd participated in at least ten Phase I trials as a healthy
volunteer.
The experience sparked his interest in research and led him to a
career in clinical pharmacology. Having "experienced life on both
sides of the needle", Dr. Venitz thought he had a 360-degree view of
the process. But it wasn’t until he became gravely ill that Dr. Venitz
came to fully appreciate that therapeutic trials are days in a life.
Life in the ICU
Dr. Venitz first knew something was wrong when he began slurring
his speech during a lecture. Most people might have suspected
stroke, but Dr. Venitz suspected myasthenia gravis, an autoimmune
neuromuscular disorder characterized by fluctuating muscle weakness
in the voluntary muscles.
"My scientific training led me to rule out stroke," he says.
"Everything pointed toward myasthenia gravis." A neurologist
confirmed Dr. Venitz’ suspicions.
Although there is no cure for myasthenia gravis, treatment can
help relieve symptoms.
Despite treatment, his immune system continued to attack itself.
Six weeks after his symptoms appeared he was admitted to the
hospital’s intensive care unit (ICU): an experience that would
become all-too familiar over the course of the next three years.
Ultimately, his neurologist settled on a course of treatment that
forced Dr. Venitz to undergo plasmapheresis. The process is similar
to dialysis. But the catheter often became infected, resulting in
extended stays in the ICU.
Then what?
Not surprisingly, when Dr. Venitz’ neurologist approached him
about a therapeutic trial, after another visit to the ICU for a blood
infection, his interest was instantly piqued.
But there was a sticking point.
"I asked my doctor, 'So let’s assume this is
my miracle drug and it works. The trial lasts
a year. Then what?' But he didn’t have an
answer for me. There was no end game."
Assuming it worked; the disclosure form
didn’t say whether patients would be allowed
continued access to the drug on a compassionateuse
basis.
He explains, "I’ve been doing clinical trials for 25 years and
what happens after the investigation – was something I had never
thought about. But as a patient I wasn’t going to take the chance
that this might work for me but I might not be able to have access
to it after the trial, so I said no."
Instead, Dr. Venitz opted to participate in a non-interventional
trial to help clinicians establish a quality of life assessment tool
for myasthenia gravis patients. That study involved periodically
answering survey questions about how his condition was affecting
his quality of life and lead to the creation of an assessment tool.
"It helps focus the conversation," Dr. Venitz says. "The assessment
tool really forces you to figure out what changes have
occurred since your last visit in a much more formal, organized way."
Trials through a new lens
Today, Dr. Venitz' condition has stabilized. Although he takes
about 20 pills a day, he hasn’t had to undergo plasmapheresis
in nearly three years.
He's used his experience to affect what he can. For a decade,
Dr. Venitz has served as a member of an Institutional Review
Board (IRB) and he brings his experience as a patient to that role.
"Now, when I’m reviewing a trial, I understand how patients
might feel if they couldn’t have access to a drug after the trial
ended and we make sure it’s in the disclosure. They have to think
about the rest of their lives."
Understanding Clinical Trials
Can you take part in a trial while
working full time?
Many potential volunteers worry whether becoming a clinical trial participant will
interfere with their job or create problems
with their boss. While clinical trials
volunteers may need to do some careful
planning, you don’t need to be a super hero to hold a job and still volunteer.
"We want to dispel the idea that you will have to take a whole
day off from work to be in a clinical trial," says Lisa Pacitto, of
MassResearch. "Usually the first screening visit takes longer,
because there may be a medical exam and you have to read the
Informed Consent form. But in most studies, the other visits are
shorter and are easier to work around your schedule."
Often, people who work non-standard hours can talk with the
trial site staff to schedule visits around their available time. "For
example, shift workers can often get to their site visit before going
to work, or before going home at the end of their shift,” says Karri
Venn at LMC Endocrinology Centres. "However, it’s a little
trickier if their visit requires having fasting blood drawn, because
people want to have it done early in the morning."
If you’re worried about a potential conflict between work and
your trial commitments, it’s wise to consider these steps:
- Find out exactly what’s required.
Ask how often you’ll have to visit the site, how long each visit
is likely to last, the schedule of visits, and how long the trial
will go on.
Dr. Charles Wilcox of Pharmacology Research Institute (PRI),
advises asking these questions which can give you a sense of how
much time you’ll spend on the trials: What is expected of me? If I
arrive on time for my appointments, will I be seen within fifteen
minutes or less of my scheduled appointment time? Will my calls
always be answered by a “live voice” during business hours?
- Be aware that many research sites try hard to accommodate
volunteers.
Don’t be afraid to speak up about your schedule concerns.
Patricia Larrabee of Rochester Clinical Research (RCR) says,
"We do whatever we can to make visits convenient. Most patients
try to come in early in the day; at lunch hour, or at the end of the
day, so we are set up to see them at those times. If a trial requires
fasting blood, we open up more slots early in the morning."
Dedicated research sites also work to eliminate waiting time,
which helps to keep the visit short. "Coming for a clinical trial is
different from going to your regular doctor," says Larrabee. "We’ve
trained our staff that nobody sits in the waiting room longer than
a minute."
Pacitto agrees that working with the volunteer helps make visits
more convenient. "One gentleman who is a research subject
travels a lot for work. When he starts traveling, we ask him for
his travel dates. We look at his schedule and see how we can
work around it so that
he can get to his site
visits. Also, we can
start at 6:00 a.m. and
get people in and out
so they can get to work
before it starts."
Research sites also
try to accommodate
volunteers by providing
extra services that can
help them save time in
related areas and gain
other benefits.
Still, there are some
jobs, such as factory workers with very limited breaks and rigid
lunch schedules, that may not always lend themselves to clinical
trials. Other situations may make it stressful to volunteer. "Some
people’s place of employment is not respectful of the fact that
they need to take care of their health or wellness," says Venn.
"So
they keep their disease secret from their jobs." This may make
volunteers worry about making time for their site visits.
- Think honestly about your motivation.
"Maintaining your scheduled visits could seem burdensome if
the trial lasts months or years", says Wilcox. "If the trial has been
going on and the subject realizes or suspects that the treatment
isn't working for him, it becomes harder to stay motivated and do
all the things that are required for the trial protocol."
Motivation to improve your own condition and to assist in
developing new medical treatments helps offset some of these
challenges.
Since it’s important to the trial that volunteers complete their
scheduled visits, volunteers should weigh the challenges they
face in fulfilling their trial responsibilities and discuss them with
research site staff. These candid interactions will lead to the most
positive—and least frustrating—situation for volunteers.
