Welcome to Boundless Bee, the Hemophilia Bee podcast,
the podcast standing with you from a family
like yours, presented by the Coalition for Hemophilia
Bee and in partnership with Balancing Life's Issues.
In a time when science is advancing faster
than ever and information is just a click
away, it's easy for patients and families affected
by hemophilia b to find themselves overwhelmed or
even misled. When you're navigating a complex condition,
trying to understand new therapies, or advocating for
care,
getting accurate science based information is absolutely critical.
That's why today's guest is so important. Eric
Paulson understands not only the power of medical
innovation, but also the inner workings and the
frustrations of government. As a former congressman and
now chairman of the Institute for Gene Therapies,
he's uniquely positioned to help bridge the gap
between cutting edge treatments and the policies that
make them accessible. He's here today to talk
about how we ensure the right voices, those
grounded in research facts and patient experience, are
the ones help shaping the future.
Eric, welcome to Boundless b. It's great to
be with you. Thanks for having me. Of
course. And you spent a decade in congress,
so I just wanna start there. That's a
really long time to be in congress. Can
you give us a little sneak peek about
what that time was like? Yeah. And, well,
challenging times to be sure, but I will
say this, that one thing that I did
during my tenure in congress is that, I
was always a passionate advocate for medical innovation
and the life improving and life saving technologies
that were being made available for patients. So
I co chaired the medical technology caucus. I
served on several different committees that had jurisdictions
over health care, and and that kinda gave
me a front row seat, an opportunity
advocating for some, you know, different policies to
foster foster advances in healthcare. But I really
did enjoy my meetings with constituents and their
patients and their families who came to Capitol
Hill for meetings, especially during rare disease week.
They would share their personal stories or perspectives.
So, you know, while I always had that
opportunity to be a champion for new life
sciences and innovation,
it was those interactions,
I think, that really guided me and some
of my staff with an extra purpose to
know, engage and advocate on behalf of patients.
And now I get to do that in
a new role at the Institute of Gene
Therapies, which is really exciting after I left
Congress. A lot of the reason we're here
is because you help bring to light these
stories that end up I don't know. Is
it create empathy? Does it help create emotional
intelligence to people who are resistance to helping
get these advances in medicine to the people
who need it? Like, you talked to us
more about how
you bringing those stories to life has helped.
Yeah. There's no doubt. I mean, from a
personal motivation perspective, you know, rare disease patients
and the stories and they were constituents. Right?
I mean, they shared their personal side and
and and and I think having engagements with
those types of different stakeholders
and then bringing all those different voices together
just kinda helped,
you know, coalesce sort of in interest or
a motivation
to continue
helping, you know, this new frontier of medicine,
if you will, in in in a different
way. And this is where, you know, when
I look at gene therapies, I kind of
look at where we are right now in
that field, kind of where medical devices were
three decades ago. So we're sort of at
the infancy, but a very exciting stage, you
know, for hemophilia b patients, but also a
lot of different rare disease patients, which is
exciting. Yeah. But just to clarify, I mean,
at the Institute of Gene Therapies where you
are now, it's is it only about hemophilia
b, or is it about all kinds of
gene therapies? All kinds of therapies. I mean,
and I'll just tell you, you know, about
our mission essentially. But there's lots of, you
know, neuromuscular diseases.
You know, there's just 10,000 different rare diseases,
right, that a lot of patients have. Some
are ultra rare diseases. But, you know, we
were created and started up at the institute
five years ago. It's been an exciting time
over a five year period, because we've seen
numerous gene therapy approvals in that time frame,
including inherited blindness, right, that has a new
therapy for it. We've had policy initiatives advance
during our time now as an organization. And
so, you know, our mission has been now
really working to ensure that the health care
system and the policies around healthcare are keeping
up to speed with the science of gene
therapies. And so we can maximize the potential
of gene therapies by modernizing the regulatory system,
the reimbursement frameworks, so that patients are ultimately
able to realize this new promise. And and
that's really important, again, as I mentioned, in
this frontier of of medicine. Yeah. And if
we could just take a step back because
I think for a lot of people, it's
it's about simplifying this process. Is there anything
to be said about navigating health care? It
could be challenging. It'd be very confusing, and
there's there's jargon and there's semantics you just
don't understand. So is that is that part
of the work that you do? Well, I
think part of the work comes into place
there when we when we work with our
different advisory councils.
Patients. And and and there's organizations
such as, you know, the hemophilia beat, you
know, coalition, but we also have individual parents
and their family perspectives that are members of
our patient advisory council. And those perspectives and
stories bring sort of the real life component
of the challenges that are out there, the
value that needs to be focused on in
terms of opportunities and with reforming and modifying
our health care system system and and making
progress along those fronts. So we are the
most impactful when we are centered around sort
of the patient needs and efforts and and
stories and experiences that that that motivate and
drive us and our other members. Are are
there any specific stories that kinda leap to
mind when you you say that? Well, you
know, we have Capitol Hill briefings, for instance,
that, we try to bring in a patient
perspective periodically to educate policymakers.
And, you know, we had someone recently who
was the parent of a Duchenne,
child. And, there was not a dry eye
in
in the audience of different congressional staff after
hearing some of the stories and challenges that
this family had faced, but also the hope
that a new therapy was bringing for change
for this family and and the opportunity to
really bring a value in in a in
a different way. And and, again, these are
new exciting
treatments that are being approved by the FDA,
but that also means that there are regulatory
and other policy hurdles that have to be
addressed and identified
and dealt with, to make sure that these
these these hopes are are actually realized. When
it comes to making sure these hopes are
realized, I mean, this is this feels like
a very strange time with in this administration
and especially when it comes to, like, cuts,
budgetary things. I guess, from your perspective, your
from your years in congress and where you
sit today, is there a message of of
hope around stories
from patients, like, being able to affect what's
happening in congress in in a positive direction
to get families the the care they need?
Yeah. Absolutely.
A couple of things here. I I will
say number one is that we're blessed to
have very good bipartisan
policymakers
on both sides of the aisle that are
engaged in this space, that are are learning
the value of gene therapies and what that
means to patients. And that all comes, I
think, from the grassroots
involvement,
of folks that come to Capitol Hill. Again,
these are constituents, right, that and policymakers
wanna help their constituents.
I think once there is an education level
with these different stakeholder groups and others that
engage in with policymakers and especially those patient
stories, some of these folks wanna become leaders
in this field. And so we've had various
pieces of legislation introduced. We've had advocates with
the first Trump administration, the Biden administration, and
now the second Trump administration.
So, again, very bipartisan background and support, which,
again, helps realize the hope. So, again, I'm
optimistic. I think one of the challenges that
we do see also is that we wanna
make sure there's continued research and development in
this space, right, which means investment in at
the NIH, for instance, National Institutes of Health.
We we wanna make sure that, you know,
if there are reductions that are happening in
government spending, that those are carefully targeted and
that we're able to be lean and mean,
but we're not cutting our nose off to
spite our face, essentially. Right? That we're actually
gonna be able to continue the innovation here
in The United States, here at home, become
FDA approved, and then move forward with the
treatments. Yeah. And I guess, could we could
we talk about advocacy for a second? Because
I think for a lot of people, it's
a scary word. And maybe for, you know,
the the overwhelmed parent with so much else
going on, it might feel like going to
Capitol Hill
is
not an option. Like so can you talk
to us a little bit about what advocacy
means to you and how you've seen it
work from,
I don't know, from as simple as writing
a letter to
actually being on Capitol Hill speaking to congress.
Yeah. I mean, advocacy again, this is part
of when I think of our patient advocacy
advisory council, for instance, and in organizations like
hemophilia and others that have grassroots organizations. So
number one, as we mentioned before, patient stories
are essential because those are real life experiences.
And for elected officials and policymakers
helping their constituents, those are huge opportunities. Letters,
phone calls, emails,
those are all impactful. You don't have to
take the time to fly to Washington to
have a visit with your member of congress
on Capitol Hill. You can also ask for
a visit though in the home district office.
You'll often find those staff members at home
or the legislative official when they're back in
their district very willing to meet on hand
in in the home district. So lots of
opportunities to engage, and there should be no
hesitation about having a personal voice shared with
a member of congress, for instance, or or
a local state legislator, you know, advocating for
newborn screening and some of these initiatives at
the state level because it can absolutely be
very impactful because ultimately these policymakers wanna be
helping their constituents. Can you recall a moment
in your tenure where
your mind was changed in a certain direction
because of these letters you got or these
phone calls that you got or, you know,
people who, like, came to your office in
in to advocate for for something? Well, I
I will say this is that, on a
regular basis, I I would I would ask
my staff to keep track of different tallies
of, you know, what are we hearing about
today? Are there certain issues we're hearing about
from constituents? Because, you know, there could be
a news story that's driving a lot of
attention, right, with phone calls or letters or
or emails. But I do remember one experience
in particular that was with a rare disease
patient that I never expected to get involved
in. And this was someone who actually came
to visit me who was suffering from pulmonary
fibrosis, you know, a special disease of the
lungs where there's no known treatment, no known
cure. And his ask or request is like,
hey, we should make sure that the National
Institute of Health is creating a registry
to collect data on all the different patients
that have this disease because it actually is,
very prominent as much, killing as many people
as breast cancer. And so we need more
focus and attention on this. So that prompted
me to say, hey, we should be introducing
legislation. I got some bipartisan authors. We got
house and senate engaged, and we were able
to make some progress and then drive some
attention on this. So that's one story
that I never expected
to come across or to actually engage in
on the legislative level, but it was meaningful
and impactful because I was able to help
someone and and move something forward. Cool. And
I just want our listeners to hear that.
Our congressmen,
you know, are people with families at the
end of the day, and and they need
to hear from you, though, in order to
make sure, especially for the voices of the
hemophilia b community, that the policies
and all these kinds of things are shifting
in a favorable direction.
And I guess if you could just speak
a little bit to maybe what are some
of the major hurdles you're facing right now
when it comes to access to to gene
therapy and just to make sure patients can
begin to, benefit from the treatments. Yeah. You
know, the very nature of gene therapies, you
know, makes for some unique challenges with our
traditional regulatory paradigms that exist out there because
these are treatments that are relatively new. They're
relatively incredibly unique. They're potential onetime treatments that
can have long lasting benefits for both patients
and the health care system alike. So we
do need to be rethinking
our policy and our regulatory landscape. So that
means our current reimbursement system, for example, is
not really set up to accommodate the influx
of these new therapies that are currently going
through the FDA approval process, and and that
impacts patient access. So
we've also seen threats to the FDA's accelerated
approval pathway, for instance, and the coverage of
treatments that move through that process. And that's
critical for the development of these new gene
therapies because of of all these different variables
across disease populations that gene therapies can benefit
and can provide benefit and the unique benchmarks
of these therapies themselves. And let me take
a step back here too because I do
think one of the biggest priorities and hurdles
that we face in general is also ensuring
that all policy makers and regulators
and and even the general public understand again
this unique value of gene therapies because we
have an opportunity knowing that chronic, disease and
illness consumes about 85¢
of every health care dollar when you manage
these symptoms and chronic illnesses over a patient's
lifetime. We've really got an opportunity now to
disrupt that whole process,
with gene therapies. And so you might have
an expensive treatment right up front, but if
you have something that lasts a lifetime for
a cure or potential treatment, you can improve
and save lives for patients, but you can
also be saving our health care system a
lot of money down the road. So that's
the excitement and opportunity, I think, across the
board. Yeah. And, like, I guess, what are
are there some, like, general myths that we
could bust here for a couple minutes in
terms of gene therapy?
Because I know especially for the hemophilia b
community, there are things like abstinence and alcohol
use kind of are some of the hesitations
for people to look at that therapy as
a possible treatment. You know, in general, I
mean, it's always important for patients to be
talking individually with their physician, with their doctor,
with their care providers to work through all
of the backgrounds and steps to take if
this is a therapy that would make sense
for them. Right? And so I know at
the Hemophilia
B Coalition at the symposium, for instance, that
just took place in Orlando where I had
an opportunity to join patients and a lot
of providers there is that there was a
special panel between a gene therapy doctor and
a patient there. And, it was a great
give and take about how this patient understood
and went through the process of getting a
new treatment. Not sure if this was gonna
be the right step at first, but in
that consultation with the health care providers
that served this patient, it it made sense.
And it might not be for everybody, but
it is something you can work through a
lot of those myths or challenges or caution,
challenges with by having those types of conversations
upfront. Yeah. And I think just another shout
out to the symposium in any any event
that the Coalition for Hemophilia
b puts on because those are opportunities to
connect directly with people who are going through
these therapies, who are going through these treatments,
and are on the kind of the cutting
edge of the new treatments out there. And
because I think you're absolutely right. I believe,
actually, I think I was at that same
breakout session as you. And just hearing the
stories from these, you know, totally
from these whose lives have been transformed
because of advances in science and medicine is
is just so inspiring and but it does
take that effort to
to go to those things, right, to invest
in the education with the community.
And I think going back to the idea
of advocacy,
like, educating yourself on these things is a
form of that. Can you speak a little
bit to to, like, education and the Coalition
for Hemophilia B? Well, you know, in education,
I mean, it it comes from just, you
know, engaging with your podcast. Right? And just
taking the time to listen and hear new
perspectives and stories that that that other patients
of similar situations are going through, for instance.
Right? And so that's one thing I've observed
by attending some of these various symposiums with
patients of different rare diseases is that, you
know, there's there's a commonality and there's some
different experiences, obviously, but, you know, there's also
a huge support group that can be brought
together that,
can kinda lift people up.
And and so I guess that's one of
the exciting components of it. Yeah. Well, I
mean, thank you so much for your time
today. I know you're very busy, and the,
you know, the coalition is is very grateful
for your time and your energy and your
effort. And I guess if just maybe to
round this out, what what gives you hope
right now given given the current landscape? Yeah.
You know, again, I as I sort of
said earlier, I I think the future is
bright. I I think the science is really
exciting. We've got multiple new gene therapy approvals
that are expected each and every year. We've
got bipartisan support in congress, which is good.
We've got legislative initiatives right now that are
moving forward. The MVP act, bipartisan support, accelerated
care for kids act that is moving forward
with bipartisan support. So we've we've got good
legislative initiatives that have been introduced, and we've
got more and more policy makers through the
personalized medicine caucus and others that have sponsored
some of our briefings on Capitol Hill. So
I'm really optimistic,
but it ultimately means that
the grassroots that comes from our patient organizations
is gonna help to continue to move this
forward. And so for all of your listeners,
please visit our website at genetherapies.org
to learn more about what we do at
IGT.
And we're we're excited to stay active with
with different social media channels with you and
others. And so that engagement is really key
to success. So, well, thank you so much.
And and, again, a shout out to advocacy.
It does not have to be scary. It
could be as easy as visiting gene-therapies.org,
or the College for Hemophilia b website and
go to the community section to see how
you can get involved.
Share your stories and let Eric and the
Institute for Gene Therapy, be able to uplift
your voice in this community. So thank you
again so much for your time today, Eric.
Sounds good. Really enjoyed our conversation. Look forward
to staying in touch in the future. Fantastic.
Alright, everybody. Take care.
This has been a production of Balancing Life's
Issues produced by me, Kai. Follow along with
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Anything else to add, Miles?