Page 13 - CF Roundtable - Winter/Spring 2026

as well as people with double lung
transplants whose genotypes do fit the
initial eligibility profile for that therapy.
Some of them have wound up taking
Trikafta steadily and benefiting from
it substantially. Others have regretted
ever taking it in the first place.
Concern about potential side effects
that would reduce my quality of life
looms large in my own thinking about
what I would and would not be willing
to take for experimental purposes.
Trying a completely new drug always
involves a bit of a gamble before
one even considers that a given trial
participant may not get the study drug
as opposed to a placebo or what we
call standard care in medical research.
A “standard care” scenario for a drug
trial usually means participants in the
control group—that’s the group of folks
who get compared to people taking
the new therapy—continue taking
whatever medication they were using
before. Getting the study drug raises
a whole host of additional concerns
about unanticipated adverse events,
which may range from temporary brain
fog or minor stomach upset to severe
allergic reactions or permanent organ
damage.
For me to take a totally new drug,
I’d have to weigh the potential risks
carefully and do a lot of background
reading on the active ingredients. I’d
need to consult with my CF team and
get their input while discussing my
own concerns with the providers who
know me and my body best. Ideally, I
would also be able to coordinate my
participation in a trial through my CF
clinic. In many cases this isn’t an option,
which I’ll address in more detail along
with other instrumental barriers like
transportation and availability. I’d need
to consider the broader social context
of both the initial drug development
and the planning of human trials for
later stages of testing its safety and
efficacy—how well the treatment
works under ideal conditions closely
controlled by researchers. I’d also want
to know how thoroughly the research
team planned intentionally for use of
the drug by a racially and ethnically
diverse population of patients, as well
as patients of all sexes and genders.
I should note that clinical trials aren’t
always testing drugs. Medical devices,
including those that many of us use
for routine airway clearance, also go
through clinical trials before getting
authorized for widespread use. Having
been an early adopter of a couple
different physiotherapy devices over
the years that have helped me with my
own health maintenance, I’ve often felt
more open to the idea of participating
in a device trial than a drug trial. But my
interest and willingness will ultimately
depend on the specifics of a given trial
and that all-important ratio of potential
benefits to known risks. After many
years both conducting and reviewing
research in the health sciences,
including providing human subjects
review for drug and device trials, I think
about those same considerations for
myself as a potential study participant.
Of course, work commitments
introduce several other considerations
as well for participation in clinical
trials. Medical research studies can
differ quite vastly in their logistics and
time requirements. And as several of
my USACFA colleagues noted while we
were planning the Focus Topic for this
issue, not all CF centers in the United
States participate equally in trials
for new drugs or devices. Some do
not participate at all! If a center does
participate in trials, the many people in
today’s adult CF community who work
in paid or volunteer positions must
consider how the design of a given trial
fits with our responsibilities for those
commitments.
All the usual logistical factors involved
in routine care also apply here. If we
are going to a clinic site physically,
how are we getting there and is that
transportation affordable? Do buses or
trains run when we need them to get
where we’re going? And if we’re driving,
is parking available and accessible? If
we are self-administering medication
or using a device at home, how do
those activities impact our ability to do
other daily tasks? Do we have adequate
instructions available in our native
languages to ensure that we’re taking
the medication or using the device as
intended? Can we have someone else
at home with us in case we experience
a serious adverse event and need
immediate assistance? What about
our own responsibilities at home and
how our health care activities intersect
with those? Can we participate in the
trial and still provide needed care for
children, elders, pets? And how do all
these nuances, along with whatever
type of observation and monitoring we
are under as trial participants, impact
our mental and social well-being?
Thanks to many advancements in care
that got their start in clinical trials,
including but certainly not limited to
CFTR protein modulators, more adults
with CF than ever before are living
dreams that might once have seemed
unattainable to us. We’re building our
careers, furthering our education,
buying our homes, expanding our
families, caring for others, exploring
new hobbies, and more. This
necessarily involves thinking about
what we want for our futures as well
as the day-to-day of our lives in the
present. How clinical trials fit into that
can depend on a great deal of factors.
I’ve seen promising innovation and
creativity from medical researchers
in recent years, especially those who
partner closely with patient scientists
and patient advisors from outside the
scientific community in planning their
drug and device trials, aimed at making
research participation more feasible
and accessible for people with CF
and a host of other chronic diseases.
Thanks to digital technology and vastly
improved connectivity, today’s clinical
trials often present fewer barriers
related to transportation and time. Fair
 Pearls of Wisdom continued on page 14
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