Access, Efficacy and Collaboration: Unlocking the Potential of Gene Therapies

Host:
Welcome to Insight Exchange, presented by L.E.K. Consulting, a global strategy consultancy that helps business leaders seize competitive advantage and amplify growth. Insight Exchange is our forum dedicated to the free, open, and unbiased exchange of the insights and ideas that are driving business into the future. We exchange insights with the brightest minds of the day, the most daring innovators, and the doers who are right now rebuilding the world around us.

Matt Mancuso:
Hello everyone, welcome and thanks for joining us today. In this episode we'll be discussing gene therapies and building sustainable strategies to commercialize these products. 2020 started with a flurry of activity in the space, and there's a lot to cover. Today we'll be going behind the scenes of a highly exclusive gathering that L.E.K. and Real Endpoints recently hosted in Phoenix, where we brought together 50 influential individuals, including gene therapy executives, payer, CMS leaders, and other key stakeholders in the gene therapy market. What made this gathering unique was its intimate nature, deliberately kept small to encourage candid conversation. The meeting room itself required no microphones, did not allow slides, and featured no formal speeches.

The distinctions between panelists and the audience blurred, setting the stage for an engaging, lively and sometimes controversial conversation. In this podcast we'll aim to provide you with some of the key takeaways from these discussions. Join us as we unravel the insights and valuable perspectives that were shared during this exclusive gathering, and review a glimpse into the future of sustainable commercial strategies in the world of gene therapies. I'm Matt Mancuso, managing director at L.E.K. Consulting, and I'll host today's discussion. I was once a scientist by training and I lead much of our firm's advanced modalities work. I'm really excited about the amount of new therapies and technologies on the way for patients and the major needs that they address.

I'm joined today by one of my colleagues, Alex and two of our friends from Real Endpoints, Roger and Ellen. Alex, would you mind introducing yourself real quick? And Roger, Ellen, thank you both so much for joining us today, it's been great to see you guys again since the conference a few months ago. Would you introduce yourselves as well, and when you go through, could you also provide a little background on Real Endpoints.

Alex Guth:
Thanks, Matt. I'm happy to start us off. I'm Alex Guth, I'm a managing director with L.E.K., I co-lead with much of our work in pricing and market access for biopharma. I'm also a virologist by training, so I'm always excited to talk gene therapy and cell therapy. I love to see viruses, or at least parts of them at work for good in the world finally.

Ellen Licking:
I'm Ellen Licking, I am a VP of client services here at Real Endpoints. Real Endpoints is a market access consultancy. We focused in three domains, one is market access strategy, another is contracting strategy and services, and the third area is patient support. I think we'll talk about how all of those come together in the gene therapy space today.

Roger Longman:
And I'm Roger Longman. Matt, thanks so much for having Ellen and me on the show today, really pleased to be here. My background is frankly as a reporter in the pharmaceutical and biotech world, started a company focused on that and then started Real Endpoints where I'm chairman several years ago to focus on the issues around how to pay for innovative therapies.

Matt Mancuso:
Great, thank you all so much. Roger, Ellen, again, thanks for coming on, it's great having some specialists in the commercial and reimbursement space as we chat today and go through this meeting and what's happened since. I appreciate the introductions, I mean, since we hosted this meeting in May I've been kind of surprised by the amount of movement in the gene therapy field. We've seen something like three major approvals and their launches depending on how you count by product and by geography and whatnot. We've seen a ton of new data clinically commercially. What's changed in your opinion since the meeting? I don't know, maybe Ellen over to you first, but what have you observed or what's going on since May and how's the world changed?

Ellen Licking:
When we were getting ready for the meeting in May, we knew that 2023 was going to be a big year for gene therapy, but I don't think we knew how active this spring was going to be. I think one of the things that we saw in the three approvals that happened since May, it's just the scale of diseases that can be treated with gene therapy. We've got dystrophic epidermolysis bullosa, we've got Duchenne muscular dystrophy, hemophilia A. That's generating a lot of excitement about how this new modality can actually be applied to help treat diseases where a lot of the treatment has been symptomatic.

On the flip side though, I think we have this environment where we've had some gene therapies that were approved last fall in 2022 where we haven't necessarily seen the uptake from a patient perspective that was anticipated at least by Wall Street. And so it's a little bit bipolar in that regard, in that we're not seeing this uptake in terms of the addressable market that was anticipated. And for companies, it really underscores how long the commercial road is for them, that that approval is a first step in a longer series of steps in building a commercially successful product.

Alex Guth:
I completely agree with Ellen though, it's been an exciting time in the last few months, but it's really been a story in gene therapy of high highs and low lows this year. As we said, the pace of new approvals has been unprecedented. At the same time, on the low side we've seen numerous notifications that companies are laying off employees, the financial environment isn't helping, a number of companies that were originally very well funded have shut down. You see things like summation, CODA has gone through this, others are teetering on the itch of a shutdown for lack of funding. One driver is certainly technology and some of those platforms struggling, but there's also from a market perspective, an unwillingness to fund some early stage companies, particularly in categories where competitors are further along. 

Ellen Licking:
 Alex, to build on that, I think one of the concerns is that it's not a scientific issue that they're wrestling with. It really is a commercial challenge. And that was a key topic that we talked through at this meeting in May, is thinking through what are the different elements you have to have in the roadmap to drive that commercial success?

Alex Guth:
I think the challenges were anticipated, but gene therapy manufacturers are really feeling the scale of them just now. And that really ranges across multiple stakeholders, it’s building the infrastructure, doing the patient finding, building comfort amongst physicians for this very new modality. And then of course there’s the question of payment, the very large upfront payments, how to think about reimbursement, and rebating based on these therapies that can have efficacy for many, many years. And that was a key topic during the meeting.

Matt Mancuso:
Ellen, Alex, to those points, on one hand it’s amazing how much has changed since just our May meeting. On the other hand, I was looking at an old set of slides the other day, I had a 2019 forecast in front of me of the gene therapy market and it was estimated at something like 7 billion by 2023. We’re not there today, right? And some of that was due to clinical and manufacturing challenges, and not to mention there was a pandemic in between. But even with the recent approvals now uptake’s still slower. I mean, I hear you both talking about the commercial side, do you think we’ve kind of solved some of these clinical and manufacturing challenges at this point and really it’s a commercial play now? What’s the state of the field in your opinion?

Roger Longman:
I think that what Alex and Allen have already said is true of course, the uptake has been slow. There are certainly clinical issues, scientific issues that have slowed things down. There’s certainly manufacturing issues that have slowed things down more, and I think the cell therapy than on the gene therapy side. But if I’m to look at what has caused this slower than expected uptake, I think about three or four different factors. The first one is what Ellen referred to, and that is patient identification. Finding truly eligible patients in the first place, which is a lot more difficult than many people have expected, certainly than I expected. And then finding those people who are actually willing to undergo the treatment, which sometimes includes hospitalization, includes long distance travel to the centers of excellence. Sometimes it just simply means that sometimes people are just concerned about having their genes messed with, particularly if there are pretty good, certainly less complex therapies available.

Hemophilia’s probably the best example where you’ve got therapies like hemlibra from Roche, you’ve got next generation factor replacements like Altuviiio from Sanofi, and those do a pretty good job pretty conveniently. So, there’s that kind of competition. The second big issue is again, something that I don’t think everybody recognizes, that a lot of these people, a lot of the patients are in difficult to reach populations. So, if you think about sickle cell, that’s largely a Medicaid population or Medicaid is a big portion of it, and folks historically mistrust the medical system because the medical system has in fact failed them. So, that’s the second issue. Ellen and Alex have both mentioned reimbursement, which is a particular problem. 

So, there’s some intriguing solutions to those issues, but it’s still early days. And finally, back to the provider side, there’s the centers of excellence challenge. You have to get the centers up and running. There are economic issues to be worked out with payers. It’s not just the manufacturer that’s a problem, some of these centers of excellence want what are essentially admin fees, which double or more the cost of the drug. And then there are certain providers which are competitive with the gene therapy providers. So for example, in hemophilia, again, you have the hemophilia treatment centers, which have a significant financial incentive to keep the patients in the HDCs on factor replacement.

Matt Mancuso:
Roger, I think if I try and summarize what you’re saying, I’m hearing big issues across what I think of are probably the three or four different key customers in the healthcare ecosystem. You’ve got challenges along the patient access, challenges along the payer employer access, and the challenges along the provider center of excellent access, which we could either call one or two different axis. Do you have any feel for which of these has been the biggest challenge so far? 

Roger Longman:
In the launches so far I think it’s patient identification. And patient identification broadly considered finding the patient, finding the eligible patients, finding the patients willing to do something, willing to take the gene therapy.

Alex Guth:
Roger, I’d like to pick up on that, because that patient willingness is the one that may be caught me most by surprise, and I think that’s true of a lot in the field. I’m a scientist by training, the data on gene therapies is often really compelling, something we’re very excited about. But you hit on something that I think is underappreciated, which is that to some of the specific patient populations that are being targeted, they really have been failed by the medical system in the past. You mentioned sickle cell, medical equity for those patients has been an enormous challenge for the whole of modern history in the development of therapies for sickle cell. Hemophilia can be easy to forget, but that is a patient population that was absolutely decimated by HIV in the ‘80s and early ‘90s, and the scars from that, they last a long time. I’ve had an opportunity recently to speak with patients from both of those groups, and at times I’ve been surprised the degree to which there remains really significant trepidation about new modalities, even as there’s great enthusiasm on the scientific and prescriber communities.

Ellen Licking:
Alex, I’m glad you mentioned that, if we're being really honest, these therapies have a lot of uncertainty associated with them. There's not long-term data around the durability of these therapies. There's also the fact that we're in the midst of this renaissance that we're seeing new science in terms of the backbones that are used to deliver them. But right now some of those therapies, if you get it now, you're not going to be able to get one in the future. So, patients are also having to weigh this very difficult choice, I'm stable on these other drugs that control my symptoms pretty well. The field is evolving quickly, and there might be something better in the future, but if I take an option that turns out to be not quite so good now, I'm not going to be able to get that option. And so I think it creates a natural hesitancy for patients and then the providers who are advising them.

Matt Mancuso:
A few things to unpack there, I think Alex, on the hemophilia point, it's amazing to think about how much distrust there can be in some of these populations, I think to Roger's point on sickle cell, that one comes up often. But there was something like 5,000 patients who had contaminated blood products in the hemophilia community if I recall correctly. And when you consider there are only tens of thousands of them, I mean the impact is tremendous. Ellen, to the points you were just making, do you have any feel for what it would take to get patient acceptance higher? 

Ellen Licking:
It's a data issue and it's going to take time. I think you're going to see adoption in the patients who are the sickest and have the severest forms of disease who aren't being well controlled because they need other options. But I think one of the things companies who are developing these therapies need to do is think about their patient support almost on steroids. Those things that make sure they can get to the treatment center, they have access to transportation and an appropriate kind of work environment that allows them to take time off for the therapy, or allows them to take time off for the therapy when their children or loved ones are having the therapy.

Those are all these kinds of things that support the access to care that aren't typically thought about as healthcare, but we know based on lots of other studies elsewhere really impact access to care. To take a page from some of the work that's been done in the rare disease community where they're going out and finding the individual patients and thinking through holistically what they need to be able to access the therapy, that's probably something that's going to have to be done. And frankly, at the price tags the therapies are being offered, you can see why stakeholders across the healthcare ecosystem would expect those services to be on board.

Matt Mancuso:
Yeah. You mentioned a lot of good points in there, Ellen. One that just stuck with me was the fact that a lot of these decisions are being made by caregivers for junior patients. And I think maybe one example more than others where that's the case is zolgensma of course, where the patients are quite young. One of the things that struck me from the conversations at the meeting was that a good number of these patients may be ending back up on other therapies. I think of gene therapy as having the promise of being one and done, are we really observing that today? What's going on in reality with spinal muscular atrophy? What's going on in hemophilia?

Ellen Licking:
I think for zolgensma it's pretty clear it's not one and done. You're seeing patients on spinraza or evrysdi. That's concerning to stakeholders, both patients and the providers and the payers. From a patient perspective, what happens if you're on a therapy that's not truly one and done, and because of an immune reaction, you can't get something better in the future? That creates that hesitation and a natural desire for wait and see approach. Then on the payer side, I think we all have to acknowledge that one of the challenges with gene therapy is payers have been sort of told that the value of these therapies is that they are a one and done situation.

So, the price tag for some of these is actually worth it because you're not going to be on other expensive therapies in perpetuity. But that math no longer works if they're going on expensive therapies in two or three years. And so you're seeing payers really try and push discussions around, what are alternative value-based agreements? Whether they're outcomes-based agreements or alternative findings arrangements that can help them manage the uncertainty around the durability. And that was a topic that came up repeatedly in our May meeting, at a certain threshold does a value-based agreement need to be part of the equation for these gene therapies until we've got more data that really shows that durability?

Matt Mancuso:
Ellen, thanks for sharing all that. I'm going to park the value-based agreement point for a second, I want to come back to that in more detail. But quickly before we go there, Alex, you'd mentioned to me the other day something about the patient switching dynamics in hemophilia and patients going back on factor. I don't remember the specifics, but it felt similar to the zolgensma situation. Can you elaborate on that?

Alex Guth:
Yeah, absolutely. This concern about durability and the addition of further therapy is already impacting how these therapies are being evaluated. The recent example I was mentioning was in hemophilia, BioMarin's gene therapy in hemophilia A. Some background, in hemophilia efficacy is measured by bleed rates, and patients on gene therapy typically report a bleed requiring treatment about once per year. However, in about 12% of patients during the trial another therapy was used after gene therapy as a supplement when the gene therapy's efficacy waned. In a traditional trial, you'd note the discontinuation and switch to another therapy and that's it, that's the end of the efficacy evaluation.

But in gene therapy, the FDA has said, well, these patients have received the gene therapy infusion, they're still in the trial, they're still being evaluated for efficacy. And what's more, since we can't know how much they would've bled without the additional therapy, the FDA's required that we count theoretical bleeds, the bleeds that they may have received if not for the additional therapy and that be imputed in the analysis. So, for the 12% or self patients who use combo therapy, they were accounted as though they were experiencing nearly 40 bleeds per year for any time they were on a secondary therapy. When you compare that to the actual bleed rate, which was about one per year, you can understand the outsized impact this potential for multi-agent use has on reported efficacy for trials.

In the end, as a result of this statistical analysis on this 12% of patients that used another therapy, the total efficacy reported on the gene therapies label, which included these imputed bleeds, is about two and a half times worse than what was published in the clinical trials, which only counted the bleeds those patients actually experienced. It's an enormous penalty to the reported efficacy that stems from using another agent in a minority of patients. So, clearly the FDA is very attuned to the potential that gene therapy is being pitched as a single agent, but in practice ends up in combination with other therapies.

Matt Mancuso:
That's really interesting, Alex. I don't know that I've seen that before. Have any of you guys encountered that in other situations.

Ellen Licking:
No, I actually think it's new and I think it's going to be a real challenge for gene therapy companies, especially if we go back to value-based agreements for a minute. We think about how they're constructed, because typically one of the ways they'll be constructed is using the failure rates in the label to get at what kind of a payment or other metric you would use to monitor with payers. 

Alex Guth:
Ellen, building on that a bit, we've talked about this value-based agreements that may result in rebates if a gene therapy isn't effective, and I think in discussions with many gene therapy makers, that's something that they're prepared for and thinking about. But ultimately they don't want to lose payment because say a physician wanted to use two therapies.

Ellen Licking:
Absolutely, and I think what it also will do for gene therapy companies is as they're thinking about the stakeholders they're working with, they're going to be looking at the providers and how they vet the providers, and this makes the decisions about which centers of excellence even more important. They're going to want to make sure that protocols being followed are the ones that were studied in the trials and have the most data around them, because that's where they have the most data themselves about how the product works. Otherwise there's a risk they would be taking on that they have no control over as the companies.

Matt Mancuso:
That's great. Thank you guys. I think I'm probably the least deep of all of us on this call when it comes to the payer and reimbursement side. One of the things that caught my attention when we were in Phoenix was hearing how CMS had changed how it's calculating best price to enable VBAs more broadly. I kind of expected value-based agreements to be growing, but I didn't know what was slowing it down and what was really happening. Taking the conversation you're having around the labels and building on it, just broadly, can you help summarize for me what's going on with value-based agreement?

Roger Longman:
A lot, but at the macro level I think what you need to understand is what Ellen has articulated quite well, which is how do you deal with the uncertainty of efficacy or of durability? And how do you mitigate those uncertainties? Usually that's going to be some kind of an innovative contract, some kind of a performance guarantee. Now, it is true that ultra-rare, ultra ultra-rare gene therapies are probably not going to require a performance-based contract in order to get coverage. But to your point, Matt, at the meeting it was pretty clear from the payers they expect VBAs, that those VBAs are going to be table stakes. The question is what flavor of VBA? You mentioned just now the idea of a provider wanting to use two therapies, if the gene therapy doesn't work he wants to be able to add on something else.

Well, there are now structures, risk sharing structures which kind of allow that, one of which is the warranty structure, which is in effect an insurance policy that the manufacturer buys through a third party, and which can then be structured to allow all sorts of, or a variety of different remedies, including the remedy of paying for an additional therapy if the gene therapy isn't perfectly effective. These warranties also address another issue which you were getting at, Matt, which is around Medicaid best price, because a warranty is an insurance policy, effectively, the Medicaid best price is determined not by the total dollar value of the remedy, but in fact by the premium that the manufacturer pays to the third party insurer.

There are other kinds of structures as well, and I don't want to simply limit it to warranties, which are still relatively rare in the marketplace. But the point is that there are structures which can be used to address those original uncertainties. The other thing that's important here is that the government, the federal government is increasingly interested in VBAs as you point out, Matt, and as was discussed at the meeting by indeed CMS. There is a major experiment going on now in which CMMI is trying to structure a way of its itself being the negotiator of VBAs for any and all state Medicaid organizations. That would solve certain problems, it would make, the idea would be, can I address the complexity and cost issues for Medicaid of doing VBAs, which are challenging with a quid pro quo from manufacturers?

So, you get an efficient process for faster coverage in Medicaid, which one should note is a major market in gene therapy, and that would be in return for meaningful risk shares. There's a lot of work to do here. I don't want to pretend that this is a done deal, the states have to want to go along, the rich states have to want to be able to work with the poorer states. The pharmaceutical companies have to want to go along, and they are increasingly skeptical of government involvement in pricing issues such as value-based agreements, most obviously given the reaction they've had to the IRA negotiation program.

Matt Mancuso:
Roger, if you had to estimate what percentage of patients we might see covered on VBAs in the future, just give any sort of directional idea to me. Is it going to be used in a minority of situations? The majority? The vast majority? What's the future look like in your view?

Roger Longman:
In my view, when it comes to gene therapy it will be the majority of patients, indeed the vast majority of patients, granted the gene therapies are used as, come out and are adopted with the vigor that I think they should be. I do expect it across all channels, I think that certainly the Medicaid payers that we have talked to are extremely interested in risk sharing agreements. Commercial is obvious, they're very familiar with VBAs now and are sort of demanding that idea from manufacturers as we speak. I don't think that there's a payer that we have talked with in a commercial insurance program that hasn't said, "We want risk sharing programs in gene therapy."

Matt Mancuso:
That's really interesting. It sounds like an awesome way to align value to what we're actually delivering here or maybe set another way, put our money where our mouth is. 

Ellen Licking:
I think one of the things that's interesting about CMMI's initiative is it shows the challenges that we're trying to overcome that we've actually already talked about in this podcast. One of the reasons this is a Medicaid initiative is there's a great concern that there's this new modality that patients will not have access to because of the limitations of Medicaid state budgets, and that it's going to actually increase inequities in care. So, I think this is one element. I think the second element is the nascent state of the infrastructure required to really do VBAs. While I agree with Roger that we will get to a point where VBAs are used will be a majority, I think the challenge is we don't really have an infrastructure to do this well and do it repeatedly.

And we heard that at the meeting as well, if you think about where we see VBAs working well, it's where you can track them with a claim because we have a very good claim infrastructure. So, that's one of the ones where hemophilia is a great one for a value-based agreement because there's a claim when there's a bleed. But in other cases we don't have those claims data. And so one of the things that companies and payers who are interested in this space are really thinking about is how they can be using claims data and eventually then get to some of the more outcomes data they would like to be measuring that are difficult to get right now the way our system is set up. And we still have a ways to go for that to really come into being.

Roger Longman:
That's true, Ellen. The one caveat I'd have on that is that these are still rare diseases, and rare diseases are by definition, easier to get clinical data from because there are fewer patients than fewer providers providing those therapies. So, it's not the block that it would be for much more prevalent conditions, hypertension or diabetes or things like that where insurers just cannot get anything, cannot really do value-based agreements without anything other than a claims metric.

Ellen Licking:
But we do have gene therapies in those more prevalent areas that will be coming in the not too distant future.

Roger Longman:
They will. You're right. Yep.

Ellen Licking:
I think it's a great goal. I think one of the key things is structuring VBA is to think about how you get rid of a claim, and then that becomes the success right now. But then moving towards what is the infrastructure that allows that sharing of the more clinical data? And frankly, right now, that's one of the reasons, Roger, we're seeing warranties as coming to be a more exciting option in certain cases, because you don't need the claims data, you can get it through the provider at attestation of what the clinical value of the product was or wasn't.

Roger Longman:
That's true.

Ellen Licking:
The other place I would look, Matt, is what's happening around accelerated approval and the whole dialogue we've seen around accelerated approval. We know in general payers are not big fans of drugs approved under an accelerated approval channel because they don't necessarily have the data that shows the value. And I think depending on the price and budget impact for gene therapies that are approved under that channel, you're going to see a push from payers for some kind of value-based agreement or risk share. 

Matt Mancuso:
That's really exciting. Awesome. Well, thank you guys for that coverage on value-based agreements. The meeting just continues to get me excited about what's going on in the gene therapy field, and honestly, I don't even know how to put bounds on what that means. There's probably something like 2,000 in vivo or ex vivo genetically modified therapies in development today.I think the excitement's there, I mean, no doubt our meeting focused a lot on the emerging commercial challenges. But it felt like a lot of the earlier clinical and manufacturing challenges are fading into the past.

As we get near the end of our time here, I was hoping to open it up to each of you one more time. I guess a quick question for you all, where do you see the market going over the near term? And in just a simple one word answer to the next one, are you optimistic about the next few years of gene therapy here? Do you think we'll have more hurdles in the future? 

Alex Guth:
Absolutely. I'll give more than one word, I'll start with optimistic. But what I'm optimistic about is that we are going to advance enough that we can face some of these hurdles. Some of the development challenges that have prevented us from reaching these commercial questions in the past have been resolved. The pace of development and approval I think is really staggering and thrilling. We talked earlier about the multiple approvals that there have been in just the last few months, and the upcoming calendar is filling up with upcoming FDA meetings and data. One element that is supportive of this is the FDA has been amenable to clinical trial designs which permit a feasible study in these rare disease populations, including permitting external controls like natural history studies in lieu of just randomized study design.

At the same time, the ramp up for commercialization for many gene therapies as Roger discussed, has been slow. The elements we've talked about, patient finding, building physician comfort, payer contracting, those have all proven to be challenging in a very new modality. And to my mind, for companies that are launching a gene therapy this creates a real strategic challenge. Theoretically, there's a huge first mover advantage. For now gene therapy is a one shot treatment, and so first entrants are motivated to get in there to address the prevalent population before anyone else can enter.

It's a race to be first like we saw in HCV a decade ago. But in practice, gene therapy developers, they are squeezed. The commercialization, standard operating procedures, the infrastructure, those are slow to get up and running on one end, while the pace of development means competitors in many of these diseases may be soon nipping at your heels, and they can benefit from the market development you're investing in. So, companies are really having to rethink their approach for approval to ensure they're well positioned to have that rapid uptake.
 
Ellen Licking:
I agree with so much of what you said there. I am also optimistic about the field for two reasons. One, I think from a technology perspective, and if we're thinking about a manufacturing perspective, new technologies are coming on board that particularly for the ultra-rare diseases will make it more affordable to create therapies. 

And I think because of the original price tags, companies potentially thought of access to gene therapy as purely a payer issue. And as Roger has noted, there's this big demand issue and they need to get creative there. I would just say that companies, when they're working with the payers, the earlier they start to find out what it is that the payer's true concern is and they start thinking about how they're designing their clinical trials to address that concern so they come to market with the data, and are prepared potentially to do risk shares to mitigate any uncertainty, that will stand them in good stead. And so we won't necessarily be talking about payer access issues in the same way we have been today.

Roger Longman:
You know what? As I listen to Ellen and Alex, I agree, they're absolutely right. I think that anybody in this business has got to be optimistic because the challenges are so huge that you just give up if you weren't positive about the potential for what we're doing. But weirdly, I also see a kind of positivism or opportunity in the challenge that we've seen. Now, Alex pointed out that this is a first mover business, and he's absolutely right. The issue is that if in fact we've had these problems so far with uptake, with label changes, with payers, it sort of makes me wonder, have investors and companies moved a little too quickly to say, well, if we're a fast follower, we're not actually going to make it because the first guys will take this market for one and done therapies.

My sense is that in fact, there is going to be room and maybe more room than expected for fast followers or even not so fast followers, depending on how long this uptake challenge takes. And in fact, I just saw that Roche Spark is going to be moving ahead with one of its hemophilia therapies, which frankly I hadn't expected them to do. And my bet is that we're going to see other companies kind of reevaluating maybe their first decisions about moving ahead with programs that they were possibly going to cancel because of this first mover issue. So, optimistic, absolutely.

Matt Mancuso:
That's excellent. To Alex, Ellen, Roger, thank you all so much for your time today, it was great getting everyone together and being back in touch since our meeting out in Arizona. I'm looking forward to more of it in the future. Is there anything any of you would like to add as we close out here?

Roger Longman:
I think you've done a wonderful job, Matt, in herding us all together and eliciting some really interesting insights, at least from Alex and Ellen.

Alex Guth:
I just think we may need to make this weekly at the rate the field is evolving. It seems like there's a new approval and a big new study every couple of days, so to be continued for certain.

Matt Mancuso:
Absolutely. Well, to the listeners, we're happy to provide more detailed discussions on requests. We invite you to connect with us, Alex and I are here at L.E.K., we have extensive experience in providing strategic support to a number of different bio pharmaceutical and biotech companies across a wide variety of situations. On the commercial side over at Real Endpoints, I'm sure Roger and Ellen would welcome any calls, so please feel free to reach out. Thank you all for listening today, appreciate the time. And to anyone who was with us in Arizona, thanks for the insights and the conversation, it was great. Great being in touch with you all. Take care.

Host:
Thank you, our listeners, for joining us today at the Insight Exchange presented by L.E.K. Consulting. Links to resources mentioned in this podcast can be found in the show notes. Please subscribe or follow for future episodes wherever you listen to your podcasts. Also, we encourage you to submit your suggestions for future insights online at lek.com.