Rae Woods (00:02): From Advisory Board, we are bringing you a Radio Advisory, your weekly download on how to untangle healthcare's most pressing challenges. My name is Rachel Woods. You can call me Rae. (00:13): We are now in the final week of our series about bespoke care and innovation. We've talked to our experts on the future of cancer care, and the equity opportunity in precision medicine. But for today's conversation, I want to focus on an area of innovation that is perhaps on everyone's mind when it comes to bespoke care, and that is cell and gene therapies. (00:35): Look, a lot of the conversations that we're hearing on cell and gene therapies, frankly, a lot of the conversations we've had on this very podcast, have focused specifically on how purchasers and pharma can partner together around payment models. But those conversations can often feel theoretical. And in many cases, they actually exclude the voice of one of healthcare's most important stakeholders, which are the providers themselves. (00:58): So today, I want to pause on the payment model problem and I want to talk about the providers, specifically the providers who are, or who are considering actually delivering cell and gene therapies. To do that, I'm bringing Advisory Board pharmacy experts, Gina Lohr and Chloe Bakst to talk about cell and gene therapy delivery models, including who needs to get in the game today, who shouldn't, and why everyone who's listening to this episode has next steps. (01:27): Hey, Chloe. Hey, Gina. Welcome to Radio Advisory. Gina Lohr (01:30): Hi, Rae. Chloe Bakst (01:31): Hey, Rae. Rae Woods (01:34): Chloe, you actually said to me when we started planning this episode, you almost gave me a warning. You said, "Rae, you know that our new research isn't about how the industry, how society will pay for these drugs. And clearly, that means you know that this is something that's keeping me up at night." I know you tried to give me a warning, but then I actually got excited because you said that we need to talk about how we actually deliver these therapies and maybe leave the payment conversation off to the side. Why is that the right problem for us to discuss today? Gina Lohr (02:09): I asked the same question when the team came to me and said- Rae Woods (02:13): Really? Gina Lohr (02:13): "We want to focus on delivery instead of payment." And I said, "Wait, that's not the problem we're talking about here." But they also convinced me that delivery actually is both, A, as much of a blocker, potentially, to patient access as the payment challenges are. And B, potentially, more solvable at this point. Which was really exciting because we are seeing enough variation across the industry to know that there are practices that can be shared, and there are opportunities for organizations to get better at this so that it becomes less of an access barrier for patients. And that's exciting. Rae Woods (02:52): That is exciting. And let's just be clear, that's the purpose of Advisory Board. That's the purpose of Radio Advisory. We want to share guidance. I always like to end these episodes with actionability. And so I'm excited that we're going to have actual things to say when it comes to cell and gene therapies, if we focus on the delivery space. And in particular, because I want to remind folks, cell and gene therapy isn't brand new. The first one came out in what, 2017? Is that right? Chloe Bakst (03:18): Yeah. Yeah. It's been about seven years, and now we have about 22 drugs, cell and gene therapies, approved right now. Rae Woods (03:25): That are being deployed by someone. Chloe Bakst (03:27): Yeah, they're being delivered. They're approved by the FDA. Someone's delivering them. And what I think is exciting about where we are now, like Gina was saying, each drug that comes out is new, but the processes that it takes to deliver them might not necessarily be new. So we're at this interesting pivot point. We're seven years into these drugs even existing, and we're starting to see, I wouldn't call them best practices yet because it's still pretty early, but emerging standards perhaps, or interesting directions or places where people have seen success and we can get some shared learnings happening. And that was what made our conversations with leaders in this space really exciting and interesting. Rae Woods (04:10): Do some level setting for me. What do we need to know about the changing pipeline of these high-cost and ultra-high cost drugs? Chloe Bakst (04:17): So 73% of the 22 drugs that are currently approved are for hematology or oncology conditions. So that's the CAR Ts that have been approved, sickle cell hemophilia drugs. The vast majority of the products that we have available today are for drugs that fall into those two camps. Where we're starting to see the pipeline shift is that about 80% of the 650 plus drugs that are currently in phase two or phase three clinical trials- Rae Woods (04:46): 650? Chloe Bakst (04:48): 650, it's a lot, are for non-oncology conditions. So things like ALS or cardiology, things that fall into just different camps. Rae Woods (05:01): And this is why we keep saying that this class of drugs is going to be revolutionary for patients, and frankly, that it's also going to fundamentally change and challenge the way that the healthcare industry operates. But I'm struck by the fact that, again, these are not brand new. There are organizations that have been on the forefront. Do I dare and say, they may even have a mature understanding of how to deliver this kind of care? I know that this is far from every organization. What kinds of hospitals, health systems are we actually seeing being leaders in this space? Gina Lohr (05:37): Yeah. So where we see organizations leaning in here are the same types of organizations that may have been involved in the testing and the clinical trials. And so, they've probably actually been involved with these drugs even more than the seven years that they've been on the market. And these are really the academic medical centers and children's hospitals who are providing really that cutting edge care to their patients and who have been able to build on that existing infrastructure to make delivering these drugs a little bit less daunting. Rae Woods (06:10): What do you mean by existing infrastructure? What are they building off of? Gina Lohr (06:13): Often, it is coming out of the cancer program and especially, the bone marrow transplant program. So if you look at the evolution, it started with bone marrow transplant, which evolved into the ability to deliver CAR T therapies. And then that chassis, that infrastructure then provided support for leaning into gene therapy programs as well. So it's been more of an evolution than starting a new program there. Rae Woods (06:42): So we know who has been successful here thus far, and it makes sense to me that it would be the AMCs and the children's hospitals, like you said, Gina, the ones that have the existing infrastructure. But with such a huge pipeline coming, 650 drugs, and going into new areas, it's not just blood disorders and oncology, are we saying that not only is delivering these drugs the central problem that we need to solve, but we need to solve it so that all providers are able to actually deliver cell and gene therapy? Gina Lohr (07:17): The answer there is, probably not. Even though there are a ton of drugs, it's not going to be the same level of volumes as a traditional cancer drug for breast cancer where you have a huge patient population that is ready to receive that drug, so it's still going to be lower volumes. And you still really need a lot of infrastructure to deliver them. It probably will expand beyond where it is right now, but when we look and say, "Where might it go?" Certainly the most obvious next level are other organizations that haven't been delivering these therapies yet, but that do have a large cancer program. They probably are closer to having the infrastructure they would need to deliver that. And then I think there's interesting questions about partnership, whether different organizations will collaborate together around how to make these therapies more accessible to patients in their area. Rae Woods (08:14): Okay. My head is spinning because these are all really good practices that our listeners can adopt even if they're not going to be the ones building from scratch the next cell and gene therapy infusion center. You're having conversations with health leaders all across the country right now as part of this research. In your conversations with the ones who are the fast followers, maybe they still fit into that AMC or children's category, maybe they're a more traditional hospital or health system. What is it that they're prioritizing to not just build a program but build one that is ultimately sustainable? This has to be expensive. Chloe Bakst (08:54): One interesting approach that I've heard, is similar to a hub-and-spoke model. So you have a health system that has an AMC that operates as that hub expert, taking on the more clinical trials, taking on the more innovative drugs, the ones that are just getting approved. And then to expand access to patients who may have difficulty traveling to the AMC site or just maybe looking for care closer to home, the AMC is collaborating with community hospitals in their network to onboard CAR Ts that have already been approved for a number of years, so they have a clear understanding of what the risk profiles are and what the safety concerns are and what the delivery processes need to be. They're not asking the community sites to take on every new gene therapy that the AMC may be onboarding, but they can take specific drugs that they feel more comfortable with and train [inaudible 00:09:51] providers who are part of this health system, so they have resources to rely on and expertise to rely on. That maybe they're starting a net new CAR T program, but they're not starting from scratch. Rae Woods (10:04): So I like this because you ran right at sustainability in terms that I didn't necessarily expect. You're saying that not only can not every health system deliver cell and gene therapy in the same way that the AMCs and children's have over the last, let's say, decade. You're saying that this partnership, like Gina just mentioned, is a way that they can be a little bit more specific and more scoped in the kinds of patients they work with, the kinds of therapies that they provide, which ultimately means a more sustainable model. They don't need to be doing 650 different cell and gene therapies. Or even 22. Chloe Bakst (10:42): Yeah, there are very few programs that we talked with that are looking to onboard every single new cell and gene therapy. I think a big part of being strategic in this space is taking the time to understand your patient population, understand your physician wants and needs. And this is, like you said, it's really expensive to do this. So if you're onboarding a new drug, there are a lot of considerations to weigh and value. And I don't want to get too into the weeds of that now, but I know that Gina will be talking about this in a lot more depth at our clinical innovation summit this June. And we have an entire session on this topic specifically of how do you build sustainable cell and gene therapy program. So maybe I'll just drop that teaser now instead of getting too- Rae Woods (11:29): Register in the show notes. Good job, Chloe. Yes, yes. But again, I appreciate this. A more scoped program is a key factor in the path to sustainability. If we're going to talk though, about sustainability, if we're going to talk about finances, I want to get ahead of the question that might be on our listeners' minds, which is, okay, this is expensive, but is there also a revenue opportunity here or is that not the right way that we should be thinking about this? Gina Lohr (11:59): What we heard from most, if not all of the organizations we spoke with is that these are not big revenue drivers. The goal for most organizations is, can we break even? Can we be made whole for delivering these drugs? And not everybody's hitting that right now. Rae Woods (12:16): Yeah. And to be clear, bone marrow transplant also not a revenue driver. Gina Lohr (12:22): Right. When you have such expensive therapies, it's hard to also then ask for reimbursement for all of the other wraparound pieces that really need to come together to care for a patient. Chloe Bakst (12:37): But there are other ways. The revenue piece is obviously very important, but there are other value adds that this program can bring to a health system beyond direct revenue. I think reputational excellence, there are many cancer centers who need to be, in their area, the premier cancer center, that's what they hang their hat on. And part of that is offering the latest innovative therapies. There are destination healthcare sites that need to offer every novel therapy that they can, every rare disease that they can, because that's where patients go to seek care when they are a rare disease patient. (13:13): And I think maybe a part of this that is less talked about but is particularly important is physician recruitment. Rae Woods (13:18): Yes. Chloe Bakst (13:19): I mean, there are providers who want to offer the most exciting or potentially effective product for their patient who, maybe this is a rare disease patient who has never been able to access a treatment for their condition before, and this represents a whole new opportunity for care. And so being the site of care where physicians can know that they will have access to this type of therapy is definitely a way to keep physicians engaged and excited about your health system. Rae Woods (13:51): Everything that the two of you are saying is reinforcing precisely why AMCs and Children's have been at the forefront of this space. And I hear you loud and clear that not everyone should be crafting cell and gene therapy delivery models. Sustainability is a key question here, which means being more focused, it means being more scoped. Let's take a moment and talk about the organizations that are not, cannot, should not be building these programs. I'm guessing you're going to tell me that that doesn't mean that they get to do nothing when it comes to cell and gene therapy. Gina Lohr (14:29): So what we know about these patients who are often on their way to needing cell and gene therapy, is that they may have had quite a diagnostic odyssey to figure out what was wrong with them and to realize even if they were eligible for these treatments. So there's a need for referring organizations to understand what is out there and what is available, but sometimes I think they don't realize what the possibilities are there, and it may even be the job of the organizations that are administering these therapies to help referring providers in their region understand what makes a good candidate. We heard one example of an organization that wasn't getting referrals from providers in their region because the providers thought that CAR T really just wasn't going to be a good option for their patients because of travel burdens, et cetera. And so there's a real need there for partnerships and for education from the AMCs to the referring providers about what is possible for their patients. Rae Woods (15:34): Yeah, this is a really important point because we don't want, well-intentioned clinicians in the market making assumptions that because their organization hasn't invested in a cell and gene therapy program or because the next closest AMC is four hours away or because it's expensive and they're dealing with a Medicaid patient, we don't want them to assume that that means that these options are off the table for their patients. Which means, we need the rest of the industry, the rest of those partners. I'm thinking about the hubs maybe in that hub and spoke that you just mentioned, Chloe, are out there educating and making sure that there are the right simple referral pathways so the patients can get this kind of revolutionary care that we're talking about. (17:19): Forgive me for being a little bit too black and white here, but it seems to me like there is a group of providers that has already been doing this or is very well positioned to be a leader in cell and gene therapy. On the other end of the spectrum, there are folks that cannot, should not necessarily do this, that are really going to need to focus on partnership. They're going to need to focus on education, they're going to need to focus on referrals. I want to come back to a group that's somewhere in the middle, that really needs to figure out how do we answer the question of creating a sustainable financial model for cell and gene therapies. Gina, Chloe teed you up perfectly to say that this is a big part of what you're going to be presenting at this year's clinical innovation summit. What are you finding as the essential points that you want to teach the leaders in the room and the listeners on the line when it comes to making sure that cell and gene therapy programs are sustainable? Gina Lohr (18:14): Yeah, I mean, this is the key question because for so many of these organizations, they feel like delivering cell and gene therapies is part of their mission for how they care for patients. But is that a mission that they can continue to achieve? Rae Woods (18:28): Or is that enough? My mission isn't enough. We have to be able to answer these questions. Gina Lohr (18:32): Yeah, for sure. One of the areas where we see care changing is thinking really thoughtfully about when to deploy these therapies in the inpatient setting versus the outpatient setting. And we're seeing a lot of the more advanced cell and gene therapy programs really take a nuanced view to this. They're pushing, for example, nearly all of their CAR T patients to the outpatient setting so that they can use buy and bill for the drug and save on inpatient costs. Doesn't mean that they won't have to have those patients inpatient sometimes, but really by being thoughtful about that outpatient inpatient divide, it's a more cost-effective way to deliver the care. Chloe Bakst (19:13): I think that that is a great place to start. Like Gina said, the advanced programs have been running at outpatient delivery for this for quite some time, they figured out how that works for them. A lot of times they have a 24/7 nurse hotline where folks can reach out to them or they have inpatient bed always at the ready in case a patient needs to get admitted who got their treatment outpatient. But there are a lot of programs who aren't comfortable delivering that kind of care yet, and there are a lot of drugs that have a safety risk profile that doesn't allow them to be delivered in outpatient care. So it's a piece of the puzzle, it's definitely not the key to financial sustainability, but it's one path forward to get closer to that goal. Rae Woods (19:57): I have to say, you all have convinced me that this was the right problem to address in your research. We started off by saying, the reason why it was the right problem is because we actually have some answers, but it's clear to me throughout this conversation, that there are still a lot of questions that need to be addressed when it comes to figuring out how do we deliver these programs? How do we do it in a way that's financially sustainable? How do we balance inpatient versus outpatient versus what the community looks like? What kinds of drugs you're going to prioritize, how you're going to create partnerships. What other challenges do we need to name so that we can ultimately address them when it comes to ensuring access to these kinds of drugs to the patients who need them? Chloe Bakst (20:42): I think one of the biggest challenges that we've heard is how difficult it is to treat the Medicaid population in this space. Medicaid coverage of cell and gene therapies is incredibly variable across states, across drugs, and the bureaucratic load of dealing with that can be really difficult for a program. And it's especially true if that patient has to travel from out of state, so they're covered by one Medicaid program, but in order to receive care, they have to go to a health system that's not within that state. Rae Woods (21:17): You're getting dangerously close to talking about the payment problem, Chloe. Chloe Bakst (21:19): I know. Well, this is sort of, we're dancing around and I told you we'd hint at it. But this is one of those areas where maybe the mission is not enough. And so health systems are in this really precarious position of having to figure out what the compromises they're going to make within their program to make sure that they can treat as many patients as possible. And maybe that means for every Medicaid patient they have to treat a certain number of commercial or Medicare patients to offset the costs. Maybe it means they don't treat Medicaid patients for cell and gene therapies because the loss is just too substantial for the program to take. And that's a real access issue. Rae Woods (21:59): And this is why I'm so excited to eventually hear how the conversation goes, Gina, when you bring a group of leaders together in person, because even though we do have guidance, we do have practices to share. We're also in this moment where learning from each other is so critical, and I can imagine that having the conversation about what you don't do is going to be really, really eyeopening. Gina Lohr (22:23): Yeah. Another area where we hear health systems wrestling with, where does the line go? Who can we treat and who can we not treat, is around patient caregivers. If you think about a patient who might need to be away from home for a month or more receiving care, especially if they're receiving care in the outpatient setting, often that means they need a caregiver who can be with them 24/7. And so, we're hearing health systems really wrestle with, do we screen out patients and exclude patients from access to these treatments if they don't have a caregiver who can travel with them and be with them while they're receiving this care, or is there something else that we can do as an organization perhaps pulling in the foundation or others to provide some of that backstop support for these patients? Chloe Bakst (23:10): Yeah. I just want to chime in here and say the caregiver role is one that I did not know a lot about before I started this research and came up on every single call as a challenge that health systems are dealing with. Caregivers are a requirement for pretty much anybody looking to receive a cell or gene therapy. Often, I mean, if you're a CAR T patient, they often require that you stay within 30 minutes of a health system. Rae Woods (23:36): And for a long time. It takes a long time to go through this process. Chloe Bakst (23:38): For at least two weeks, usually a month, and you're usually going in for appointments once or often, twice a day, and caregivers are expected to provide 24/7 care to their patients for a long time in a place that might not be home. And I mean, I've even heard of one health system who spoke with who they have the primary caregivers required, who's there on site with the patient, and then they need to have a secondary caregiver on call in case something happens to that first primary caregiver before they would consider letting a patient get a therapy. And a lot of that is because what caregivers do is very important. They monitor for patients' health, and it's a risk issue too. If you're delivering this care outpatient, you need to make sure that patients are being tended to. Rae Woods (24:24): I want to take a moment and speak beyond the providers though. We know that ultimately, ensuring access to cell and gene therapies is an ecosystem problem that's going to require cooperation and partnership from everyone, from everyone who's listening to this episode. So what do you want to see outside of the provider space, outside of the care delivery space so that we can actually make progress here? Chloe Bakst (24:46): A couple of times throughout this conversation is that now is a great time for providers to learn from each other to get news and best practices, but it's also a great time for other stakeholders to learn from providers. Payers need to know and understand how these drugs operate in the field. What are the care delivery challenges? What are the wraparound services that do need to get reimbursed when you deliver this kind of drug? And they don't really want to hear from consultants or from pharma. They want to hear from providers. (25:16): So the more providers that can do this work and can get those kind of key lessons and then communicate it to the payers they work with, it can create a ripple effect where payers get more comfortable with covering these drugs so more providers feel comfortable with growing their programs so that they can get more key learnings, so they can talk more to payers, and all of a sudden, we have a more robust cell and gene therapy delivery system. But like Gina said, somebody has to be that first step and it looks like it's providers. Rae Woods (25:48): And this is why I am so excited to hear what you learn when you gather that group of cross industry stakeholders together to have these hard but necessary conversations. So thank you for coming on Radio Advisory, and I can't wait to see what you do next. Chloe Bakst (26:03): Thanks, Rae. Gina Lohr (26:05): Thanks so much, Rae Rae Woods (26:12): Gina and Chloe spent a lot of time in this episode talking about some of the first movers, but there's also something really important that I heard, and I want to make sure that you hear it too. It's just as important for the first followers, the people who are going to be learning from the examples of their peers, of the exemplars, and who are going to be essential to making sure that other stakeholders in healthcare start to jump in and start to solve their side of the problem. This is complicated, but remember, as always, we are here to help. (27:08): New episodes drop every Tuesday. If you like Radio Advisory, please share it with your networks. Subscribe wherever you get your podcasts and leave a rating and a review. Radio Advisory is a production of Advisory Board. This episode was produced by me, Rae Woods, as well as Abby Burns, Chloe Bakst, Kristin Myers, and Atticus Raasch. The episode was edited by Katy Anderson with technical support provided by Dan Tayag, Chris Phelps, and Joe Shrum. Additional support was provided by Carson Sisk, Leanne Elston, and Erin Collins. (27:41): We'll see you next week.