In this episode, Erin Satterwhite, President & GM of Optum Frontier Therapies, explains why treating rare diseases is so difficult – and a bold new plan to address the challenge.
Many diseases are so rare that they are often misdiagnosed -- and, even if diagnosed properly, some therapies only treat the symptoms, not the underlying disease. But there's new hope for those who suffer from a rare disease, and the health care field is certainly looking at new approaches to treating them. Erin Satterwhite, President & GM of Optum Frontier Therapies, offers her insights.
SUMMARY KEYWORDS
therapies, rare disease, rare diseases, Optum, frontier, treating, treatments, specialty pharmacy, creating, gene therapies, pharmacy, disease, patients, cures, innovation, podcast, work, distribution, build, space
SPEAKERS
Ira Apfel, Erin Satterwhite
Ira Apfel00:05
Hello, and welcome to UnitedHealth Group’s Weekly Dose Podcast, where we'll get you up to speed on the latest trends shaping the future of healthcare. Don't forget the podcast is now available on Apple podcasts and Spotify, so be sure to subscribe so you never miss an episode. And now on to our show. Rare Diseases impact millions of people in the United States, and approximately two thirds are children. Fortunately, there are new specialty drugs, gene therapies and treatments coming online to address their needs. But specially drugs and treatments for rare diseases require special clinical settings. And on the cutting edge of rare disease treatment is a place called Optum Frontier Therapies. It's a new one-of-a-kind center that recently opened in Las Vegas. The goal of often frontier therapies is simple to be a full service, one stop model for advanced therapeutics and potentially change the way the industry treats people with rare diseases. Here to discuss rare diseases and Optum frontier therapies is Erin Satterwhite, she's the president and gm of Optum Frontier Therapies at Optum. Erin Satterwhite, welcome to the podcast.
Erin Satterwhite 01:16
Thank you, it's a pleasure to be here, Ira.
Ira Apfel01:19
So let's start at the 30,000-foot level and work our way down. What defines a rare disease and what makes it different and harder to treat the more common ailments give listeners a sense of the scope of the issue.
Erin Satterwhite 01:32
Rare Diseases is something that's actually defined by FDA. And the current definition is that it's a disease that affects less than 200,000 people in the United States. c.
Ira Apfel02:43
And what is the typical path for treatment for people with rare diseases, particularly for kids, assuming there even is a treatment,
Erin Satterwhite 02:53
a lot of these rare diseases are treated symptomatically, there's very few treatments that are cures, there are cures that are in the pipeline, or potentials for cures, and a lot of these are in the gene therapy or gene editing world. And the promise of these therapies is, you know, it's almost magical to think about the fact that you could adjust a default or a faulty gene and, and then have a healthy child, you know, versus, you know, having something that is going to be substantially life altering. And previously, this was science fiction, and now we're actually starting to see things like CRISPR come out, the current gene therapies are viral vectors that will insert a healthy gene and then allow that to be expressed in the body. And what that's doing is essentially putting a healthy protein out that that that could ameliorate a lot of the symptoms. And so we're not quite there yet with the current gene therapies in terms of a true cure, but a lot of the disease burden is being addressed. And so I think that the future is going to be really interesting for this space, especially as you start to see more advanced technologies like CRISPR come out. And
Ira Apfel04:21
I imagine to patients and their caregivers, parents and the like, they're really, the optimism must be really sky high now because instead of just treating the symptom, as you said, We are at least beginning to see possibilities of treating the actual disease, correct?
Erin Satterwhite 04:37
Yeah, absolutely. There's treatments coming out now. And a lot of it is because of the advocacy of parents. The patient advocacy world has been very involved in helping support clinical trials, and the advocacy at the National and policy level to create economic incentives for more treatments for these rare diseases. And so what you're seeing is a lot of passion from people who've been affected by these specific types of diseases that are creating environments where these cures can come about. And there's a sense of community and paying it forward for people who have lived with these diseases one way or another, trying to make it better for people that are coming after them.
Ira Apfel05:26
Describe a little bit more about Optum frontier therapies, what makes it unique, and how does it work?
Erin Satterwhite 05:33
Optum frontier therapies was an innovation that we recognized was necessary in the market as the growth of these complex therapies, treating very complex patients is only going to increase. And we took a step back and said, What do patients need? What do providers need what you pharmaceutical life science companies need, and what the payers need, as this marketplace starts to grow in an unprecedented way. And what we found was that the complexity of care and the cost of these therapies, were creating challenges in the existing sort of way of doing business, in specialty pharmacy, in distribution and in commercial services. So we actually designed the business by listening to the people that we're serving, we did about a year’s worth of market research and understanding the needs of what we call the four P's which are those four constituents I mentioned. And what we developed was a model that has ultra-specialized distribution with wholesale licensing, a rare disease pharmacy, that has a focus on the high touch White Glove service that's required both in the logistic support as well as in the patient care and an ongoing management.
Ira Apfel07:01
And I want to make clear, this is not the same as a specialty pharmacy, which is another term of art, if you will, or another type of pharmacy that you see offered by Optum and other companies as well. Is that correct?
Erin Satterwhite 07:18
It's partly a specialty pharmacy. But what we've done is we've taken some of the most complex aspects of managing specialty pharmacy and hyper concentrated it in opt in frontier therapies. And then we adjusted the business model to have co located specialty distribution and pharmacy dispensing. And that allows us to operate a little bit differently. And the way that I like to sort of simplify it is our business is not about volume, it's about managing high value complex needs. And a lot of specialty pharmacies do a lot of volume. And that is not the reason that we were built, we have a relatively small physical footprint compared to other bigger specialty pharmacies. And that co located nature of having distribution and pharmacy on the same site allows us to do things a little differently also with managing the supply chain. So while we have aspects of a specialty pharmacy and we are accredited as a specialty pharmacy, we additionally have some, some other bells and whistles that a lot of others do not. And that gives us more flexibility to work in the ecosystem.
Ira Apfel08:34
And I imagine that part of also what makes Optum frontier therapies unique is you must have to recruit different or trained staff differently.
Erin Satterwhite 08:45
Absolutely, we've really focused on bringing people in that have a high degree of empathy for what people experience in the rare disease space, and also a lot of either personal or professional expertise in the area of rare disease. And, and this has been mostly by focusing on our mission, we're on a mission to support people with compassion while creating access to therapies at the frontier of health care. And that mission is a calling and people answer. What we've found is that we have patients who have opted to come work with us, we have parents who work with us, we have people who have siblings or other deeply personal experiences who have really sought out employment at Optum Frontier Therapies. And that's really helped shape our culture. And that mission is something that we hold very dear. And it really builds our business from sort of first principle basis. And having empathy allows us to stay focused on why we're here. We're not here to just go make money we're here to be able to make a difference. And that's what puts our feet on the floor in the morning.
Ira Apfel10:03
I know come from tear therapies is pretty new. But how much of the game changer? Do you think it could be for the entire Rare Disease community and how its treated and how its approached?
Erin Satterwhite 10:19
I think of Optum frontier therapies as a foundational bet on what's going to be needed as the marketplace evolves. I don't think that it's the panacea, it's not going to be the end all be all, there's a lot of infrastructure that we're going to have to build around rare disease, if we're going to be able to manage the trillion dollars of economic burden that rare disease creates in an annual basis, the direct medical costs from rare diseases upwards of 500 million, I'm sorry, $500 billion. And so when you look at a problem of that magnitude, it's hitting in the same sort of stratosphere as diabetes and cardiovascular disease, you can't just have one business that is going to serve a problem that large. So what we're doing is by building up to frontier therapies, as we're creating a Nexus for expertise for addressing where some of the largest spend is going to be coming in rare disease, which is in the pharmaceutical space, and that learning and that that context is going to be a springboard for more innovation. And I fully anticipate that there's going to be innovation and benefit design in complex case management, and creating new ways of looking at supporting the diagnostic odyssey and bringing greater access to genomics for people.
Ira Apfel11:52
Erin Satterwhite, thank you so much for being on the podcast today.
Erin Satterwhite 11:55
Ira, it's been a pleasure. Thank you so much for inviting me.
Ira Apfel11:58
That's it for this episode of UnitedHealth Group’s Weekly Dose Podcast. Don't forget, please subscribe to the weekly dose podcast on Apple podcasts or Spotify. Thanks so much for listening and have a great rest of your week.