Page 8 - CF Roundtable - Winter/Spring 2026

FOCUS TOPIC: CF CLINICAL TRIALS
By: Caleigh Haber and Keith Berelowitz
HOPE FOR TODAY,
CARE FOR TOMORROW:
UNDERSTANDING CF CLINICAL TRIALS
Living with cystic fibrosis (CF) can sometimes feel
isolating. Between daily treatments, hospital visits, and
just trying to live life, the idea of a “clinical trial” might
sound like one more thing to juggle. But here’s the truth:
you’re not alone in this, and clinical trials are one of the
reasons we have the treatments we do today.
This article is here to break it down, not in medical
jargon, but in real, plain language. We’ll look at how trials
are designed, why they matter, what it’s like to take part,
and how researchers are working to make them easier
and more accessible for everyone with CF. My hope is
that by the end, you’ll feel a little less intimidated and
a little more empowered to see if a trial could be right
for you.
Where We’re At Right Now
Let’s start with the big picture.
•
•
•
162,428 people worldwide are estimated to be
living with CF, as of 2022. (Guo et al., 2022,1)
Only about 65% are diagnosed, which means a big
chunk of people may still not know they have CF.
(Guo et al., 2022,1)
And just 12% are receiving triple combination
therapies. (Guo et al., 2022,1)
If you’ve been on a triple-combo, you already know how
life-changing it can be. These therapies don’t just treat
symptoms, they go after the root problem, the faulty
CFTR protein. For people with at least one F508del
mutation, they can mean stronger lungs, better quality
of life, and slower disease progression.
The reality is, these breakthroughs don’t happen by
accident, they’re the result of years of hard work, not
just from researchers, but from patients who signed up
for trials. Every advance in CF care exists because real
people with CF were willing to step up, even when they
weren’t sure if they’d benefit directly.
What Trials Really Look Like
So, what actually happens in a trial? An American
Thoracic Society (ATS) study followed 8,735 people
with CF over a six-year period using data from the
Cystic Fibrosis Foundation Registry database (Goss et
al., 2005,2). Here’s what they found:
•
About 1 in 3 people with CF took part in at least one
official clinical trial.
•
Those who joined tended to be sicker, with lower
lung functions [FEV1% predicted (68 vs. 77%, p
< 0.001)] and more infections [Pseudomonas
aeruginosa infection (71 vs. 65%, p < 0.01)].
•
They were also more likely to have private insurance
and nearly twice as likely to be white.
What’s this all mean? Even though people in trials
were generally sicker to begin with, their lung function
declined more slowly than those who didn’t take part.
That means these new treatments are powerful, and
being in a trial can sometimes offer real benefits.
What else does it mean? People with cystic fibrosis
who are not white and are uninsured may have more
difficulty accessing clinical trials. That’s not right, and it
needs to change.
So how can underserved communities participate
without being overwhelmed by logistics or finances?
The answer starts with clarity. Finding and evaluating
trials must be explained in plain language with support
that makes participation realistic. Researchers can go
further by offering resources and working with patient-
centered platforms, ensuring trials meet people where
they are, instead of asking them to fit into a system that
leaves too many behind.
Why Access Matters
If trials are so important, why isn’t everyone signing up?
A lot of it comes down to access.
8 CF Roundtable Winter/Spring 2026