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Welcome to Boundless Bee, the Hemophilia Bee podcast,

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the podcast standing with you from a family

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like yours, presented by the Coalition for Hemophilia

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Bee and in partnership with Balancing Life's Issues.

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In a time when science is advancing faster

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than ever and information is just a click

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away, it's easy for patients and families affected

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by hemophilia b to find themselves overwhelmed or

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even misled. When you're navigating a complex condition,

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trying to understand new therapies, or advocating for

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care,

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getting accurate science based information is absolutely critical.

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That's why today's guest is so important. Eric

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Paulson understands not only the power of medical

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innovation, but also the inner workings and the

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frustrations of government. As a former congressman and

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now chairman of the Institute for Gene Therapies,

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he's uniquely positioned to help bridge the gap

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between cutting edge treatments and the policies that

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make them accessible. He's here today to talk

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about how we ensure the right voices, those

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grounded in research facts and patient experience, are

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the ones help shaping the future.

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Eric, welcome to Boundless b. It's great to

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be with you. Thanks for having me. Of

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course. And you spent a decade in congress,

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so I just wanna start there. That's a

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really long time to be in congress. Can

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you give us a little sneak peek about

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what that time was like? Yeah. And, well,

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challenging times to be sure, but I will

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say this, that one thing that I did

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during my tenure in congress is that, I

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was always a passionate advocate for medical innovation

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and the life improving and life saving technologies

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that were being made available for patients. So

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I co chaired the medical technology caucus. I

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served on several different committees that had jurisdictions

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over health care, and and that kinda gave

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me a front row seat, an opportunity

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advocating for some, you know, different policies to

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foster foster advances in healthcare. But I really

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did enjoy my meetings with constituents and their

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patients and their families who came to Capitol

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Hill for meetings, especially during rare disease week.

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They would share their personal stories or perspectives.

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So, you know, while I always had that

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opportunity to be a champion for new life

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sciences and innovation,

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it was those interactions,

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I think, that really guided me and some

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of my staff with an extra purpose to

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know, engage and advocate on behalf of patients.

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And now I get to do that in

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a new role at the Institute of Gene

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Therapies, which is really exciting after I left

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Congress. A lot of the reason we're here

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is because you help bring to light these

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stories that end up I don't know. Is

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it create empathy? Does it help create emotional

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intelligence to people who are resistance to helping

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get these advances in medicine to the people

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who need it? Like, you talked to us

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more about how

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you bringing those stories to life has helped.

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Yeah. There's no doubt. I mean, from a

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personal motivation perspective, you know, rare disease patients

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and the stories and they were constituents. Right?

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I mean, they shared their personal side and

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and and and I think having engagements with

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those types of different stakeholders

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and then bringing all those different voices together

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just kinda helped,

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you know, coalesce sort of in interest or

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a motivation

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to continue

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helping, you know, this new frontier of medicine,

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if you will, in in in a different

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way. And this is where, you know, when

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I look at gene therapies, I kind of

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look at where we are right now in

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that field, kind of where medical devices were

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three decades ago. So we're sort of at

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the infancy, but a very exciting stage, you

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know, for hemophilia b patients, but also a

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lot of different rare disease patients, which is

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exciting. Yeah. But just to clarify, I mean,

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at the Institute of Gene Therapies where you

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are now, it's is it only about hemophilia

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b, or is it about all kinds of

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gene therapies? All kinds of therapies. I mean,

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and I'll just tell you, you know, about

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our mission essentially. But there's lots of, you

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know, neuromuscular diseases.

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You know, there's just 10,000 different rare diseases,

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right, that a lot of patients have. Some

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are ultra rare diseases. But, you know, we

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were created and started up at the institute

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five years ago. It's been an exciting time

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over a five year period, because we've seen

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numerous gene therapy approvals in that time frame,

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including inherited blindness, right, that has a new

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therapy for it. We've had policy initiatives advance

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during our time now as an organization. And

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so, you know, our mission has been now

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really working to ensure that the health care

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system and the policies around healthcare are keeping

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up to speed with the science of gene

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therapies. And so we can maximize the potential

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of gene therapies by modernizing the regulatory system,

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the reimbursement frameworks, so that patients are ultimately

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able to realize this new promise. And and

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that's really important, again, as I mentioned, in

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this frontier of of medicine. Yeah. And if

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we could just take a step back because

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I think for a lot of people, it's

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it's about simplifying this process. Is there anything

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to be said about navigating health care? It

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could be challenging. It'd be very confusing, and

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there's there's jargon and there's semantics you just

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don't understand. So is that is that part

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of the work that you do? Well, I

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think part of the work comes into place

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there when we when we work with our

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different advisory councils.

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Patients. And and and there's organizations

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such as, you know, the hemophilia beat, you

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know, coalition, but we also have individual parents

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and their family perspectives that are members of

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our patient advisory council. And those perspectives and

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stories bring sort of the real life component

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of the challenges that are out there, the

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value that needs to be focused on in

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terms of opportunities and with reforming and modifying

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our health care system system and and making

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progress along those fronts. So we are the

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most impactful when we are centered around sort

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of the patient needs and efforts and and

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stories and experiences that that that motivate and

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drive us and our other members. Are are

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there any specific stories that kinda leap to

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mind when you you say that? Well, you

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know, we have Capitol Hill briefings, for instance,

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that, we try to bring in a patient

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perspective periodically to educate policymakers.

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And, you know, we had someone recently who

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was the parent of a Duchenne,

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child. And, there was not a dry eye

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in

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in the audience of different congressional staff after

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hearing some of the stories and challenges that

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this family had faced, but also the hope

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that a new therapy was bringing for change

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for this family and and the opportunity to

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really bring a value in in a in

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a different way. And and, again, these are

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new exciting

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treatments that are being approved by the FDA,

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but that also means that there are regulatory

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and other policy hurdles that have to be

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addressed and identified

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and dealt with, to make sure that these

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these these hopes are are actually realized. When

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it comes to making sure these hopes are

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realized, I mean, this is this feels like

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a very strange time with in this administration

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and especially when it comes to, like, cuts,

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budgetary things. I guess, from your perspective, your

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from your years in congress and where you

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sit today, is there a message of of

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hope around stories

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from patients, like, being able to affect what's

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happening in congress in in a positive direction

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to get families the the care they need?

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Yeah. Absolutely.

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A couple of things here. I I will

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say number one is that we're blessed to

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have very good bipartisan

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policymakers

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on both sides of the aisle that are

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engaged in this space, that are are learning

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the value of gene therapies and what that

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means to patients. And that all comes, I

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think, from the grassroots

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involvement,

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of folks that come to Capitol Hill. Again,

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these are constituents, right, that and policymakers

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wanna help their constituents.

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I think once there is an education level

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with these different stakeholder groups and others that

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engage in with policymakers and especially those patient

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stories, some of these folks wanna become leaders

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in this field. And so we've had various

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pieces of legislation introduced. We've had advocates with

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the first Trump administration, the Biden administration, and

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now the second Trump administration.

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So, again, very bipartisan background and support, which,

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again, helps realize the hope. So, again, I'm

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optimistic. I think one of the challenges that

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we do see also is that we wanna

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make sure there's continued research and development in

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this space, right, which means investment in at

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the NIH, for instance, National Institutes of Health.

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We we wanna make sure that, you know,

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if there are reductions that are happening in

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government spending, that those are carefully targeted and

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that we're able to be lean and mean,

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but we're not cutting our nose off to

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spite our face, essentially. Right? That we're actually

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gonna be able to continue the innovation here

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in The United States, here at home, become

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FDA approved, and then move forward with the

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treatments. Yeah. And I guess, could we could

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we talk about advocacy for a second? Because

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I think for a lot of people, it's

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a scary word. And maybe for, you know,

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the the overwhelmed parent with so much else

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going on, it might feel like going to

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Capitol Hill

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is

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not an option. Like so can you talk

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to us a little bit about what advocacy

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means to you and how you've seen it

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work from,

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I don't know, from as simple as writing

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a letter to

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actually being on Capitol Hill speaking to congress.

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Yeah. I mean, advocacy again, this is part

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of when I think of our patient advocacy

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advisory council, for instance, and in organizations like

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hemophilia and others that have grassroots organizations. So

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number one, as we mentioned before, patient stories

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are essential because those are real life experiences.

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And for elected officials and policymakers

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helping their constituents, those are huge opportunities. Letters,

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phone calls, emails,

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those are all impactful. You don't have to

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take the time to fly to Washington to

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have a visit with your member of congress

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on Capitol Hill. You can also ask for

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a visit though in the home district office.

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You'll often find those staff members at home

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or the legislative official when they're back in

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their district very willing to meet on hand

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in in the home district. So lots of

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opportunities to engage, and there should be no

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hesitation about having a personal voice shared with

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a member of congress, for instance, or or

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a local state legislator, you know, advocating for

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newborn screening and some of these initiatives at

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the state level because it can absolutely be

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very impactful because ultimately these policymakers wanna be

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helping their constituents. Can you recall a moment

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in your tenure where

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your mind was changed in a certain direction

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because of these letters you got or these

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phone calls that you got or, you know,

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people who, like, came to your office in

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in to advocate for for something? Well, I

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I will say this is that, on a

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regular basis, I I would I would ask

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my staff to keep track of different tallies

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of, you know, what are we hearing about

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today? Are there certain issues we're hearing about

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from constituents? Because, you know, there could be

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a news story that's driving a lot of

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attention, right, with phone calls or letters or

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or emails. But I do remember one experience

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in particular that was with a rare disease

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patient that I never expected to get involved

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00:10:02,774 --> 00:10:04,375
in. And this was someone who actually came

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to visit me who was suffering from pulmonary

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fibrosis, you know, a special disease of the

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lungs where there's no known treatment, no known

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cure. And his ask or request is like,

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hey, we should make sure that the National

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Institute of Health is creating a registry

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to collect data on all the different patients

279
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that have this disease because it actually is,

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very prominent as much, killing as many people

281
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as breast cancer. And so we need more

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focus and attention on this. So that prompted

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me to say, hey, we should be introducing

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legislation. I got some bipartisan authors. We got

285
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house and senate engaged, and we were able

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to make some progress and then drive some

287
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attention on this. So that's one story

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that I never expected

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to come across or to actually engage in

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00:10:42,440 --> 00:10:45,000
on the legislative level, but it was meaningful

291
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and impactful because I was able to help

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someone and and move something forward. Cool. And

293
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I just want our listeners to hear that.

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Our congressmen,

295
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you know, are people with families at the

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end of the day, and and they need

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to hear from you, though, in order to

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make sure, especially for the voices of the

299
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hemophilia b community, that the policies

300
00:11:02,595 --> 00:11:05,334
and all these kinds of things are shifting

301
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in a favorable direction.

302
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And I guess if you could just speak

303
00:11:08,950 --> 00:11:10,549
a little bit to maybe what are some

304
00:11:10,549 --> 00:11:12,549
of the major hurdles you're facing right now

305
00:11:12,549 --> 00:11:15,029
when it comes to access to to gene

306
00:11:15,029 --> 00:11:18,070
therapy and just to make sure patients can

307
00:11:18,070 --> 00:11:20,475
begin to, benefit from the treatments. Yeah. You

308
00:11:20,475 --> 00:11:22,634
know, the very nature of gene therapies, you

309
00:11:22,634 --> 00:11:24,794
know, makes for some unique challenges with our

310
00:11:24,794 --> 00:11:27,674
traditional regulatory paradigms that exist out there because

311
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these are treatments that are relatively new. They're

312
00:11:30,154 --> 00:11:33,754
relatively incredibly unique. They're potential onetime treatments that

313
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can have long lasting benefits for both patients

314
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and the health care system alike. So we

315
00:11:38,100 --> 00:11:39,240
do need to be rethinking

316
00:11:39,620 --> 00:11:42,500
our policy and our regulatory landscape. So that

317
00:11:42,500 --> 00:11:44,899
means our current reimbursement system, for example, is

318
00:11:44,899 --> 00:11:47,220
not really set up to accommodate the influx

319
00:11:47,220 --> 00:11:49,139
of these new therapies that are currently going

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00:11:49,139 --> 00:11:51,325
through the FDA approval process, and and that

321
00:11:51,325 --> 00:11:53,184
impacts patient access. So

322
00:11:53,884 --> 00:11:56,625
we've also seen threats to the FDA's accelerated

323
00:11:56,764 --> 00:11:59,565
approval pathway, for instance, and the coverage of

324
00:11:59,565 --> 00:12:01,804
treatments that move through that process. And that's

325
00:12:01,804 --> 00:12:04,365
critical for the development of these new gene

326
00:12:04,365 --> 00:12:06,870
therapies because of of all these different variables

327
00:12:07,409 --> 00:12:10,549
across disease populations that gene therapies can benefit

328
00:12:10,610 --> 00:12:12,769
and can provide benefit and the unique benchmarks

329
00:12:12,769 --> 00:12:15,169
of these therapies themselves. And let me take

330
00:12:15,169 --> 00:12:16,450
a step back here too because I do

331
00:12:16,450 --> 00:12:18,470
think one of the biggest priorities and hurdles

332
00:12:18,529 --> 00:12:21,054
that we face in general is also ensuring

333
00:12:21,054 --> 00:12:23,235
that all policy makers and regulators

334
00:12:23,695 --> 00:12:25,855
and and even the general public understand again

335
00:12:25,855 --> 00:12:28,174
this unique value of gene therapies because we

336
00:12:28,174 --> 00:12:31,295
have an opportunity knowing that chronic, disease and

337
00:12:31,295 --> 00:12:33,379
illness consumes about 85¢

338
00:12:33,379 --> 00:12:35,139
of every health care dollar when you manage

339
00:12:35,139 --> 00:12:37,539
these symptoms and chronic illnesses over a patient's

340
00:12:37,539 --> 00:12:39,940
lifetime. We've really got an opportunity now to

341
00:12:39,940 --> 00:12:41,639
disrupt that whole process,

342
00:12:42,019 --> 00:12:44,019
with gene therapies. And so you might have

343
00:12:44,019 --> 00:12:45,954
an expensive treatment right up front, but if

344
00:12:45,954 --> 00:12:48,274
you have something that lasts a lifetime for

345
00:12:48,274 --> 00:12:50,355
a cure or potential treatment, you can improve

346
00:12:50,355 --> 00:12:51,954
and save lives for patients, but you can

347
00:12:51,954 --> 00:12:53,954
also be saving our health care system a

348
00:12:53,954 --> 00:12:55,714
lot of money down the road. So that's

349
00:12:55,714 --> 00:12:58,355
the excitement and opportunity, I think, across the

350
00:12:58,355 --> 00:13:00,115
board. Yeah. And, like, I guess, what are

351
00:13:00,195 --> 00:13:02,059
are there some, like, general myths that we

352
00:13:02,059 --> 00:13:04,059
could bust here for a couple minutes in

353
00:13:04,059 --> 00:13:05,200
terms of gene therapy?

354
00:13:05,980 --> 00:13:07,899
Because I know especially for the hemophilia b

355
00:13:07,899 --> 00:13:10,459
community, there are things like abstinence and alcohol

356
00:13:10,459 --> 00:13:12,480
use kind of are some of the hesitations

357
00:13:12,940 --> 00:13:15,179
for people to look at that therapy as

358
00:13:15,179 --> 00:13:17,054
a possible treatment. You know, in general, I

359
00:13:17,054 --> 00:13:19,454
mean, it's always important for patients to be

360
00:13:19,454 --> 00:13:22,495
talking individually with their physician, with their doctor,

361
00:13:22,495 --> 00:13:25,134
with their care providers to work through all

362
00:13:25,134 --> 00:13:27,855
of the backgrounds and steps to take if

363
00:13:27,855 --> 00:13:30,014
this is a therapy that would make sense

364
00:13:30,014 --> 00:13:32,019
for them. Right? And so I know at

365
00:13:32,019 --> 00:13:32,600
the Hemophilia

366
00:13:33,539 --> 00:13:36,019
B Coalition at the symposium, for instance, that

367
00:13:36,019 --> 00:13:37,539
just took place in Orlando where I had

368
00:13:37,539 --> 00:13:40,019
an opportunity to join patients and a lot

369
00:13:40,019 --> 00:13:41,700
of providers there is that there was a

370
00:13:41,700 --> 00:13:43,940
special panel between a gene therapy doctor and

371
00:13:43,940 --> 00:13:46,360
a patient there. And, it was a great

372
00:13:46,524 --> 00:13:49,024
give and take about how this patient understood

373
00:13:49,084 --> 00:13:51,084
and went through the process of getting a

374
00:13:51,084 --> 00:13:53,245
new treatment. Not sure if this was gonna

375
00:13:53,245 --> 00:13:55,084
be the right step at first, but in

376
00:13:55,084 --> 00:13:57,264
that consultation with the health care providers

377
00:13:57,644 --> 00:14:00,445
that served this patient, it it made sense.

378
00:14:00,445 --> 00:14:02,289
And it might not be for everybody, but

379
00:14:02,289 --> 00:14:03,730
it is something you can work through a

380
00:14:03,730 --> 00:14:05,910
lot of those myths or challenges or caution,

381
00:14:06,370 --> 00:14:08,789
challenges with by having those types of conversations

382
00:14:08,850 --> 00:14:10,929
upfront. Yeah. And I think just another shout

383
00:14:10,929 --> 00:14:13,009
out to the symposium in any any event

384
00:14:13,009 --> 00:14:14,384
that the Coalition for Hemophilia

385
00:14:14,785 --> 00:14:17,184
b puts on because those are opportunities to

386
00:14:17,184 --> 00:14:20,225
connect directly with people who are going through

387
00:14:20,225 --> 00:14:22,304
these therapies, who are going through these treatments,

388
00:14:22,304 --> 00:14:24,304
and are on the kind of the cutting

389
00:14:24,304 --> 00:14:26,465
edge of the new treatments out there. And

390
00:14:26,465 --> 00:14:28,230
because I think you're absolutely right. I believe,

391
00:14:28,230 --> 00:14:29,750
actually, I think I was at that same

392
00:14:29,750 --> 00:14:31,909
breakout session as you. And just hearing the

393
00:14:31,909 --> 00:14:33,850
stories from these, you know, totally

394
00:14:34,230 --> 00:14:36,250
from these whose lives have been transformed

395
00:14:36,629 --> 00:14:39,029
because of advances in science and medicine is

396
00:14:39,029 --> 00:14:40,949
is just so inspiring and but it does

397
00:14:40,949 --> 00:14:42,490
take that effort to

398
00:14:42,845 --> 00:14:44,845
to go to those things, right, to invest

399
00:14:44,845 --> 00:14:46,545
in the education with the community.

400
00:14:47,085 --> 00:14:48,764
And I think going back to the idea

401
00:14:48,764 --> 00:14:49,424
of advocacy,

402
00:14:49,804 --> 00:14:51,965
like, educating yourself on these things is a

403
00:14:51,965 --> 00:14:53,404
form of that. Can you speak a little

404
00:14:53,404 --> 00:14:55,559
bit to to, like, education and the Coalition

405
00:14:55,559 --> 00:14:57,811
for Hemophilia B? Well, you know, in education,

406
00:14:57,811 --> 00:15:00,063
I mean, it it comes from just, you

407
00:15:00,063 --> 00:15:02,315
know, engaging with your podcast. Right? And just

408
00:15:02,315 --> 00:15:04,567
taking the time to listen and hear new

409
00:15:04,567 --> 00:15:06,818
perspectives and stories that that that other patients

410
00:15:06,818 --> 00:15:09,070
of similar situations are going through, for instance.

411
00:15:09,070 --> 00:15:11,195
Right? And so that's one thing I've observed

412
00:15:11,195 --> 00:15:13,995
by attending some of these various symposiums with

413
00:15:13,995 --> 00:15:16,154
patients of different rare diseases is that, you

414
00:15:16,154 --> 00:15:18,154
know, there's there's a commonality and there's some

415
00:15:18,154 --> 00:15:21,355
different experiences, obviously, but, you know, there's also

416
00:15:21,355 --> 00:15:24,009
a huge support group that can be brought

417
00:15:24,009 --> 00:15:24,970
together that,

418
00:15:25,450 --> 00:15:26,990
can kinda lift people up.

419
00:15:27,529 --> 00:15:29,289
And and so I guess that's one of

420
00:15:29,289 --> 00:15:31,210
the exciting components of it. Yeah. Well, I

421
00:15:31,210 --> 00:15:32,730
mean, thank you so much for your time

422
00:15:32,730 --> 00:15:35,049
today. I know you're very busy, and the,

423
00:15:35,049 --> 00:15:36,970
you know, the coalition is is very grateful

424
00:15:36,970 --> 00:15:38,545
for your time and your energy and your

425
00:15:38,545 --> 00:15:40,545
effort. And I guess if just maybe to

426
00:15:40,545 --> 00:15:43,105
round this out, what what gives you hope

427
00:15:43,105 --> 00:15:45,904
right now given given the current landscape? Yeah.

428
00:15:45,904 --> 00:15:47,745
You know, again, I as I sort of

429
00:15:47,745 --> 00:15:49,425
said earlier, I I think the future is

430
00:15:49,425 --> 00:15:51,440
bright. I I think the science is really

431
00:15:51,440 --> 00:15:54,320
exciting. We've got multiple new gene therapy approvals

432
00:15:54,320 --> 00:15:56,240
that are expected each and every year. We've

433
00:15:56,240 --> 00:15:58,720
got bipartisan support in congress, which is good.

434
00:15:58,720 --> 00:16:00,879
We've got legislative initiatives right now that are

435
00:16:00,879 --> 00:16:04,340
moving forward. The MVP act, bipartisan support, accelerated

436
00:16:04,544 --> 00:16:06,225
care for kids act that is moving forward

437
00:16:06,225 --> 00:16:09,024
with bipartisan support. So we've we've got good

438
00:16:09,024 --> 00:16:11,664
legislative initiatives that have been introduced, and we've

439
00:16:11,664 --> 00:16:13,664
got more and more policy makers through the

440
00:16:13,664 --> 00:16:15,985
personalized medicine caucus and others that have sponsored

441
00:16:15,985 --> 00:16:17,664
some of our briefings on Capitol Hill. So

442
00:16:17,664 --> 00:16:18,565
I'm really optimistic,

443
00:16:19,105 --> 00:16:20,659
but it ultimately means that

444
00:16:21,519 --> 00:16:23,700
the grassroots that comes from our patient organizations

445
00:16:23,839 --> 00:16:25,519
is gonna help to continue to move this

446
00:16:25,519 --> 00:16:27,620
forward. And so for all of your listeners,

447
00:16:27,759 --> 00:16:30,960
please visit our website at genetherapies.org

448
00:16:30,960 --> 00:16:32,399
to learn more about what we do at

449
00:16:32,399 --> 00:16:32,899
IGT.

450
00:16:33,200 --> 00:16:35,485
And we're we're excited to stay active with

451
00:16:35,485 --> 00:16:37,884
with different social media channels with you and

452
00:16:37,884 --> 00:16:41,245
others. And so that engagement is really key

453
00:16:41,245 --> 00:16:42,924
to success. So, well, thank you so much.

454
00:16:42,924 --> 00:16:45,264
And and, again, a shout out to advocacy.

455
00:16:45,884 --> 00:16:47,325
It does not have to be scary. It

456
00:16:47,325 --> 00:16:50,289
could be as easy as visiting gene-therapies.org,

457
00:16:50,909 --> 00:16:53,309
or the College for Hemophilia b website and

458
00:16:53,309 --> 00:16:54,909
go to the community section to see how

459
00:16:54,909 --> 00:16:55,730
you can get involved.

460
00:16:56,110 --> 00:16:58,110
Share your stories and let Eric and the

461
00:16:58,110 --> 00:17:00,429
Institute for Gene Therapy, be able to uplift

462
00:17:00,429 --> 00:17:02,269
your voice in this community. So thank you

463
00:17:02,269 --> 00:17:03,794
again so much for your time today, Eric.

464
00:17:03,875 --> 00:17:06,115
Sounds good. Really enjoyed our conversation. Look forward

465
00:17:06,115 --> 00:17:08,194
to staying in touch in the future. Fantastic.

466
00:17:08,194 --> 00:17:09,654
Alright, everybody. Take care.

467
00:17:21,509 --> 00:17:23,670
This has been a production of Balancing Life's

468
00:17:23,670 --> 00:17:26,390
Issues produced by me, Kai. Follow along with

469
00:17:26,390 --> 00:17:30,069
other BLI produced shows at balancinglife'sissues.com.

470
00:17:30,069 --> 00:17:31,394
Got an idea for the show?

471
00:17:32,115 --> 00:17:34,755
Email me, Kai, at balancing life's issues dot

472
00:17:34,755 --> 00:17:36,914
com. And be sure to follow and subscribe

473
00:17:36,914 --> 00:17:39,174
to this show wherever you listen to podcasts.

474
00:17:39,474 --> 00:17:40,934
Anything else to add, Miles?