Melyssa Nocar: Hello, and welcome to our #ADVIExperts discussion, as we think about the evolving healthcare landscape. Today, we will be covering several issues that we help our clients with in cell and gene through data-informed commercialization, market access, and policy expertise. My name is Melyssa Nocar, head of marketing at ADVI Health. I am joined by two of my colleagues, Alicia Silver, director, policy and reimbursement and Anqi Liu, director, value, access, and pricing. Let’s jump in, it’s already proving to be a dynamic year in cell and gene, an area of tremendous potential that can bring a great deal of value to patients in medical need. As we think about innovation and commercialization in this space. What are some of the big issues, whether in manufacturing, standardization, efficiencies, and launch strategies, etc.? Anqi, let’s start with you and with what you’re seeing and experiencing, whether on the cell or gene side of things.

Anqi Lu: Thanks, Melyssa. A topic we have been hearing quite a bit about recently is the high cost of goods associated with manufacturing, cell and gene therapies. As you know, many of these therapies are personalized to an individual, using either their own cells or donor cells – and then altered, using advanced gene editing and other manufacturing techniques. This is much more complex than traditional pharmaceutical manufacturing for small molecules, and therefore also much more costly. ADVI recently conducted an assessment looking at cost of goods for a number of cell therapies, and found a really broad range of costs depending on the specific characteristics of the product itself. Through our assessment, however, we did find a common theme is that increasing automation in the manufacturing process, ends up improving the overall yield and reduces the overall costs. This is also an area where we’ve seen the White House pay some attention to recently. On March 24, 2023, the office of Science and Technology Policy (OSTP) issued a report on advancing biotechnology and bio-manufacturing in the US. Which included a really lofty policy goal of decreasing the manufacturing costs for cell therapies by tenfold in the next 20 years.

Melyssa Nocar: A lot to keep an eye on. Alicia, what are some other big issues that we haven’t touched on, from your perspective?

Alicia Silver: Thanks, Melyssa. One of the things I want to talk about is needs that teams have as they are launching both in the US and in the EU markets. Many of our clients, especially in cell and gene therapy, have relatively small commercial teams that are responsible for launching in both the EU and US markets. That’s challenging because manufacturers have to come up with a way to develop a strategy for the US market that is likely very different from what’s needed in EU approvals and launch. We at ADVI are helping our clients think through overarching market access strategies, and to the extent that they can leverage clinical evidence, health economics studies, and payer perspectives from the US, informing how they’re going to execute their EU market access strategy.

Melyssa Nocar: Absolutely, switching gears here a little bit. There are several opportunities for modernized approaches in the US and across the EU to overcome potential patient access barriers to life-changing, or life-saving treatment. What are some top-of-mind access issues that we’re helping clients with? Alicia, let’s start with you on this one.

Alicia Silver: I come relatively recently from industry. And so I look at this also from the provider lens, which I think is critical. From a provider perspective, hospitals are overwhelmed with the number of cell and gene therapies that are near commercialization, recently launched, and how they’re planning onboarding strategies. Every manufacturer works individually with their initial launching hospitals to set up the clinical administration, which is key. But there are other administrative challenges that hospitals are addressing with all, and chain therapies. For example, hospital legal teams are spending months and months reviewing and negotiating complex trade policies for each product, setting up operating protocols for how to receive high cost, personalized products in their cell labs. And really, I think, an important thing we’re not talking enough about is how to operationalize manufacture IT portals for ordering. Every manufacturer has their own IT portal that you have to get available on each hospital computer for ordering, and that can really slow down the entire process for the administrative team who’s also responsible for securing a complex prior authorization to ensure coverage and reimbursement from all payers. The good thing is, there are many groups who are starting to think about this exact problem and ways to standardize and simplify the highly burdensome process which would benefit all stakeholders, but especially patients who are waiting for their treatment.

Melyssa Nocar: All great points with a significant impact as we look to the future. Anqi, I know you have some valuable insights to share here, as well.

Anqi Lu: From the policy perspective, ADVI has been keeping really close tabs on potential reimbursement changes that could be coming up in the next couple of years. As we see new non-CAR T-cell immunotherapies get approved out of FDA, we could see some really interesting changes in Medicare and patient DRGs, and how they decide to perhaps revise those. For example, one of the questions I think that could pop up is will CMS treat cell therapies for solid tumors differently than CAR T? What about stem cell transplant replacements? How are those going to be treated relative to existing stem cell, transplant, or CAR T? And I think we’ll have a much better understanding of the direction that CMS is headed, and sort of what they’re thinking in the next few years of rule-making. Additionally, as we saw earlier this year, the CMS center for innovation is also interested in exploring alternative payment models for cell and gene therapies, and they’ve proposed a voluntary demonstration project that would allow the Federal government to negotiate and administer outcomes-based payment arrangements on behalf of State Medicaid programs. Our roster of Medicaid experts often talk about how the administrative burden of running these outcomes-based agreements and negotiating these agreements is a major barrier to their implementation. And so with this demonstration project, if it is launched, and if folks decide to participate in it – Will we see this sort of centralized administration negotiation process lessen the burden on individual states and hopefully improve access to these new therapies? We’ll stay tuned.

Melyssa Nocar: Indeed. With cell and gene is a critical focus area for ADVI’s team, we are supporting a wide range of commercialization, market access, and policy issues. Anqi, what are you most excited about in this space? And are there other noteworthy challenges to highlight that we’re helping clients solve, whether in the US, or globally?

Anqi Lu: One of the things that I’m most drawn to in the cell and gene therapy space is really improving access and helping our clients understand the reimbursement landscape in order for them to ensure that patients can get access to these life-changing and oftentimes life-saving therapies. From the payer perspective, they need to know that paying for these therapies will improve patient outcomes, and then perhaps even provide cost savings elsewhere in the system. One of the great things about ADVI, and one of our particular strengths is not only our ability to provide the high-level, strategic insight and policy overview, but also really get down to the granular details and tactics to help our clients figure out where they need to go, in order to make sure that patients can get access to these innovative therapies.

Melyssa Nocar: 100 percent, Alicia, what comes to mind for you?

Alicia Silver: I think the biggest thing is that, having worked in cell and gene therapy for the past decade, I’ve just seen a huge transformation in the available treatments for patients with life-threatening conditions not only like leukemia, but also sickle cell disease. Patients have treatment options today that were only dreams 10 years ago. I’ve been really fortunate to work closely with patients and families and caregivers, and I’ve seen first-hand how the work we do can change lives forever. At ADVI, our role is relatively small. We’re here to enable patient access to the most exciting areas of medicine, through policy, advocacy, payer, engagement, and launch readiness. But we’re really excited to continue working with clients and cell and gene therapy to ensure every patient has access to novel treatments.

Melyssa Nocar: Well said. I’m thrilled to be part of a team that cares so deeply about improving patient access and patient outcomes, especially in cell and gene. Alright, let’s wrap up. We look forward to discussing how these issues are relevant to your business needs. To learn more about how ADVI is solving complicated problems in healthcare, through data-informed commercialization, market access, and policy expertise, please get in touch directly, or visit us at advi.com. Thank you.