Rare diseases impact about 25 million people in the United States. The vast majority of rare diseases have no effective treatment, creating an urgent need for more research and development in this space. While there is a growing number of therapies in development for rare and ultra-rare conditions, manufacturers developing these products face unique challenges throughout the patient and product journey.
AmerisourceBergen, a global healthcare company, has provided commercialization services for more than 100 rare disease products.
Rebecca Willumson, Publisher of Fierce Biotech, spoke with Teri Burnell, SVP of Innovative Field Solutions at Xcenda, and Susan Weidner, SVP of IntrinsiQ Analytics, both a part of AmerisourceBergen, about market access and commercialization strategies manufacturers need to consider in order to drive product success and patient access.
Rebecca Willumson: Hi there, I'm Rebecca Willumson. I'm the publisher of FiercePharma. And I'm joined today by Susan Weidner, Senior Vice President of IntrinsiQ Analytics, as well as Teri Burnell, Senior Vice President of Innovative Field Solutions at Xcenda. Both companies are a part of AmerisourceBergen. Today, we'll be discussing commercializing orphan and rare disease products. Susan and Teri, thank you both for joining me.
Teri Burnell: Thank you for having us.
Susan Weidner: Thank you for having us.
Rebecca Willumson: So before we dive in, can each of you provide a bit background on yourselves and your companies. Susan, we can start with you.
Susan Weidner: Sure. I'm Susan Weidner, I'm the Senior Vice President of IntrinsiQ Analytics. We're very focused on the analytic aspects of the specialty therapies divisions of AmerisourceBergen. We also provide support around our medical affairs activities in precision medicine and provide our research. I come from a deep background in pharmaceutical industry with over 30 years of experience. Most of that focused on helping to demonstrate the value of the therapies being delivered, both for acute chronic and specialty care.
Rebecca Willumson: And Teri.
Teri Burnell: Well, good morning. My name is Teri Burnell. I'm the Senior Vice President of Innovative Field Solutions as Rebecca mentioned. At Xcenda, we are a global pharmaceutical consultancy company that is focused on the application of health outcomes and reimbursement. The area I have responsibility for are experts that are provider-facing. They really help providers navigate, access and reimbursement challenges with the ultimate goal of helping patients get access to therapy through educating offices around those payer requirements, access and reimbursement processes. I am a pharmacist by background, so I truly believe in the value of pharmaceuticals. I am certainly very excited about this opportunity. Just on a personal note, this rare disease space is intriguing to me, because of a rare disease that impacts my family as well as the community. so really excited to be here and to contribute to this discussion.
Rebecca Willumson: We're happy to have you. So Teri, can start with you with our first question. We know there is significant demand for new products to treat rare diseases. So what are some of the market access and commercialization challenges that manufacturers face when trying to develop these products and bring them to market?
Teri Burnell: Well, that's a great question, Rebecca. And I'll just start with some of the information about this patient population, because I think it really grounds us from some of the challenges. First of all, there's about 7,000 rare diseases. And then when we think about the actual number of people that are impacted in the United States, there's about 25 million. Here is the piece that is concerning. About 95% of rare diseases have no effective treatment. So there's certainly a urgent need for more research and development in this space. And because rare diseases are defined here in the United States as less than 200,000 patients associated with that rare disease, it really just gives you context to the small patient population. Now fortunately, the Orphan Drug Act of 1983 really has given manufacturers an opportunity to have incentives to bring more of these products to market.
Teri Burnell: So we do see that there is a growing number of therapies in development for rare and ultra-rare conditions. And really, that gives us promise for future treatment landscape for these patient population. One of the things though that we have to keep in mind is that manufacturers that are developing these products, they truly do face some of the challenges throughout a patient and the product journey. Some of those challenges include things like costs. Cost of development, cost to commercializing a product, out-of-pocket cost to the patient, cost impact to the provider. They also require specific patient and provider education needs, because of the complexity of some of these diseases as well as the payer requirement education needs.
Teri Burnell: And we do know that some of these products, especially when you think about gene therapy as an example, will have some special storage requirements and they are going to require some high touch from a patient support perspective. So when you kind of look at that holistically, the manufacturers of these products will have a challenge in front of... And certainly as with other products that come to market, really being able to prove the value of these products become really important when you talk about a successful commercialization strategy.
Teri Burnell: Orphan drugs often have far more complex evidence hurdles, as well as education hurdles, do impact to the limited knowledge and awareness of these conditions. So keeping all of those things in mind, we do know that manufacturers... And they tell us is, they are looking for partners who can provide support throughout the entire development launch and commercialization process. And when we think about that end to end kind of partner solution, one of the things we can say here at AmerisourceBergen, is that we've been really fortunate to help provide commercialization services for more than a hundred rare diseases. And our support for those are manufacturers that are providing those products to market, expands all stages of the product life cycle. From clinical trial logistics, through provider education, to market access, strategies and patient support services that help remove barrier to access.
Teri Burnell: Ultimately for manufacturers as well as for us at AmerisourceBergen, our goal is to really help patient access these life-changing, lifesaving medication.
Rebecca Willumson: Oh, good. So Susan, staying in the pre-launch phase, how can data and clinical information help manufacturers successfully plan and develop a therapy?
Susan Weidner: In the early stages of drug development, manufacturers can leverage data and clinical information to address key questions ranging from market potential and clinical trial design, the value proposition and care coordination. A deep understanding of specific disease and the patient journey is critical, as is early engagement with physicians. With a comprehensive understanding of the rare disease they seek to treat, manufacturers can identify where the patient is most likely to present for intervention, the specialist involved in patient care, and any potential challenges or opportunities along the patient journey, factors that may affect the market potential. This is especially true for clinical trials recruitment. Through our AdvanceIQ Network, especially provider research network, we can identify which physicians currently manage these patients and whether or not the specialists are research investigators or work at a recognized clinical trial site. In fact, more than a quarter of rare disease trials were stopped between 2016 and 2020, due to low accrual rates. A more directed approach such as the AdvanceIQ Network is needed if these new therapy options are going to be made available to patients.
Rebecca Willumson: Susan, in general, how does the AdvanceIQ Network support clinical research efforts?
Susan Weidner: The AdvanceIQ Network's been designed specifically to try to open access to community-based practices. We represent more than 5,000 specialty providers across the US. As a part of that, our goal is to be able to identify where those patients reside, that support the clinical trial process, as well as the clinical research activities post approval. Through that process, our network is really intended to provide access to clinical trial candidates faster and more efficiently via a data-driven feasibility assessment, to be able to identify the sites that are appropriate for this particular trial, eliminating potential barriers for contracting and patient recruitment. And more importantly, expanding patient access to the most advanced therapies and treatment strategies that are available within marketplace for their specific disease. They don't have many options. And by being able to make these practices have access as well as manufacturers to know that they need it, is a critical component of our intended success.
Rebecca Willumson: So Teri, moving back to you. As manufacturers move into the development stage, what do they need to consider when developing a payer strategy?
Teri Burnell: Well, when the manufacturers are developing a payer strategy, the first thing I think is important for them to understand is that, there is an heightened demand among payers for disease information, as well as product value information. And they want that earlier in the process. And really why they want that early in the process is really to help inform their clinical review from a schedule perspective, as well as from the information they want to gather from a clinical perspective. Their formulary planning to understand what the impact to formulary would be and what are the decision points around the formulary positioning as well as to allow them to forecast their budget. Understanding the per member per month impact, and other analysis that they need to do internally to plan for a new product and the impact on their business.
Teri Burnell: And one of the things I'll highlight here is that we gather information from our managed care network, which is a network of a payer consultant that we work with. And they have told us that payers really prefer to see study results, the pricing information as well as patient use projection, somewhere along 7 to 12 months prior to the anticipated approval date.
Teri Burnell: We talked about the growth of these therapies and seeing many more of these coming to market. And with that explosive growth for rare disease, it has led to the payers paying more attention to these products. And given that it is critical for manufacturers and their partners to really connect with payers early on in the development process, to educate them on the product, its value, the patient population, as well as the unmet needs. And important to highlight the unmet need, because certainly some of these patients with rare diseases may present more from a hospitalization standpoint and incur other costs. And here manufacturers are providing a solution that's really going to allow for the treatment of the disease which has not been there before.
Teri Burnell: The one thing it is important for us to highlight as well is that payer engagement does not stop at the development stage. Payers will influence patient access and provider reimbursement. And because of that, manufacturers need to maintain connectivity with payers throughout the product life cycle, because things may change. Payers are collecting data on their side. And having that ongoing communication with payers help to understand where they are, what their thinking is and what manufacturers will need to do to support the payers.
Teri Burnell: Also, at Xcenda we know there continues to be a gap in the information payers are seeking and what they're actually receiving. And to really help facilitate more robust communication between the manufacturer and the payer, including pre-approval information exchange which is called PIE, we acquired the FormularyDecisions platform about two years ago that really helps engage manufacturers and payers in a bidirectional way. Through this secure online platform of FormularyDecisions, really will help facilitate that exchange of credible product value and draw a pipeline information between manufacturers and more than 2,400 registered formulary decision-makers. So not only are manufacturer sharing information, but they can get back information around what payers are doing with that information, what they're looking at, and how they're engaging with the information.
Rebecca Willumson: Teri, how can manufacturers use Xcenda's FormularyDecisions platform to drive awareness of their product? Have you seen a demand among payers for information related to rare disease products?
Teri Burnell: Yeah, that's a great question. And certainly because there's so much demand and communication with payers, really in a complex and competitive landscape, and we're talking about rare diseases in general from that perspective, FormularyDecisions can help manufacturers reach a large network of payers, increase brand awareness and really maximize that brand visibility. Through this platform, manufacturers can share product information at all product development stages. That means from pre-approval to postlaunch. And by doing so, they can also gain access to payer activity metrics, like I mentioned before, and obtain feedback. Including the perception of the payer related to the client's product value and also compared to competitor benchmarks, if any. All of which can inform a manufacturer's market access strategy.
Rebecca Willumson: Okay. So now I have a question for both of you. Based on your experience working with providers, payers and manufacturers, what guidance would you provide manufacturers in terms of how they should leverage data and engage with stakeholders throughout that product journey? Susan, why don't we start with you on that one?
Susan Weidner: Sure. It's key that they engage with us as early as possible, so that we can help work with them in crafting their data and analytics strategy. And that we can be able to pride them with most customized solution to meet their specific needs. Especially in the small patient population, it's really important to be able to understand when and where they're going to need different types of information to be successful as they commercialize their product.
Susan Weidner: We leverage a longitudinal patient data asset to really identify where and when to engage the providers and target when those new therapeutic options come out. A good example, currently working with one of the manufacturers to identify where in the community setting are those patients on proceeding therapies that are likely to fail, and then bringing that information forward in a more timely manner, so that the manufacturer can have appropriate education in marketing services applied through our GPO partners across AmerisourceBergen. This really allows to be able to help improve the education and awareness of the available therapy.
Rebecca Willumson: Teri, do you want to comment?
Teri Burnell: Yeah. So given some of the access and education challenges that we anticipate that will occur with these products, it's essential for manufacturers to develop and implement engagement strategies to effectively reach physicians and their staff, being respectful of the fact that there are multiple people that may be reaching out to the office. So really that engagement strategy needs to be seamless and coordinated with all the internal stakeholders that the manufacturers may be engaging to support the providers.
Teri Burnell: And ultimately we want to help them overcome any potential access and reimbursement barriers. And we want to do that timely because again, speed to therapy becomes really important for this patient population. And just to give an example, physician offices need to be educated about if it's a buy and bill product acquisition, how is the product going to be coded and billed? What are the payer requirements? What support services and other solutions are available, including patient assistance to really help that patient when they have those challenges that may be barriers to access?
Teri Burnell: And from an innovative feel solution standpoint, we serve as an important extension to the manufacturers team and the support services they provide. We can immediately assist and help address reimbursement and access pain points, so providers can focus on what matters the most, which is really the patient. We will engage with the business operations aspect of the provider office. So the billers, the coders, the office managers, all of those that are really focused on access and reimbursement have the information they need, so they can help that patient move through the process as timely as possible, and work with the payers to get a determination if needed, so that they can know what process they need to follow to, again, ultimately help that patient get access to therapy.
Teri Burnell: And lastly, and ultimately what we're trying to do, is we play a vital role in helping to simplify the provider experience and reduce potential barriers to access. So ultimately, our goal is to facilitate with the provider customer an experience that will be positive as they work with the patients with these rare diseases, to overcome those access barriers. And really provide the right information, so that they can navigate the process as seamless and coordinated as possible.
Rebecca Willumson: So I'll close out with this. In 2020, we saw Cedar approved 31 novel drugs intended to treat rare orphan diseases. So what do you envision this market will look like in three to five years? And again, I'll pose this to the two of you. And I think Susan, we can start with you again if that works.
Susan Weidner: Yes, this is a growing opportunity as more and more patients are appropriately identified and diagnosed with rare diseases. Unfortunately with that comes the complexity of the challenges of additional precision medicine testing, to be able to confirm that they're the correct patient available for that specific therapy. But with it becomes an area of opportunity. An opportunity to target those that are more costly to the healthcare system and to be able to continue to innovate around ways, inform both providers and patients about the options that are available.
Rebecca Willumson: And Teri, do you want to close up?
Teri Burnell: Yes. And I'm going to build off of what Susan just talked about as we expect to continue to see more rare disease treatments come to market. And we'll learn from those that we're currently working with. But also we expect that technology will play a key role in this in terms of identification, but also triggering activity along the patient journey, but also from an outcomes perspective.
Teri Burnell: And I would expect that more and more payers are going to request more information, more analysis, and that creates a great opportunity for all of us within the healthcare arena to really be thoughtful and innovative, to keep all stakeholders in mind, understanding the impact to our manufacturer partners, but also to the payers that have a critical role in terms of coverage and setting reimbursement for these products to the providers that are supporting the patients and making sure that they have all the right tools and solutions and it's available in an efficient way. Because they're managing multiple diseases, multiple products, as well as the patient to get information to them in a way that's meaningful, but understandable. Because as therapy becomes more complex, then demand for more information is needed as well.
Teri Burnell: So overarching, I think it's a unique opportunity to continue with innovation and I believe technology and data will play a significant role as it does today, but even more as we continue to grow in this area.
Rebecca Willumson: Absolutely. Well, I think that is a great place for us to wrap up. I want to thank you both for joining me. I really enjoyed our conversation today.