Elevating Rare Disease Care

Lesley Pink: Hello and welcome to the Strategic Dose, Driving Your Pharmacy Mission. I’m Lesley Pink, your host. Today, we’ll be talking about VytlOne’s specialty pharmacy clinical programs and experience in rare disease with two members of the VytlOne team. They are Maria Roberts, Director of Specialty Clinical Programs, and Alicia Morris, Director of Pharmacy and Clinical Operations. Maria has been with VytlOne since 2023 and is responsible for development and maintenance of clinical programs. Alicia has been with the company for 16 years and is responsible for clinical program operations and data reporting. Maria and Alicia, welcome to the podcast.

Maria Roberts: Thank you, Lesley.

Alicia Morris: Thank you. Good to be here.

Lesley Pink: To begin, would you explain how VytlOne Specialty Pharmacy works? How are the company’s clinical programs unique?

Alicia Morris: VytlOne Specialty Pharmacy is dedicated to serving patients with rare and orphan diseases. And to define that a little bit more for you, this is when a patient has a medical condition that affects a small percent of the population. Because of that, we have a high-touch model that is built around total patient care, and that support really goes beyond just dispensing their medication. A vital piece of our process and one that we place a lot of value in is the close coordination among our pharmacists with clinicians and care teams to make sure that everyone involved in that patient’s care is working together. And another thing that I think is unique with VytlOne is that we have dedicated teams with decades of combined experience in navigating rare and orphan diseases. Our teams really are and do have the expertise to help our patients navigate these complex and nuanced challenges that they face when having a rare disease.

Lesley Pink: And you had mentioned information about rare diseases. I just want to give people context for what a rare disease is. A rare disease is defined as a condition or disease that affects less than 200,000 people in the U.S. And about 30 million Americans are affected by a rare disease, which comes to about one out of every 10 people. So, Maria, Alicia, why is a customized approach so important in the management of rare diseases?

Maria Roberts: Something that’s really important to consider is knowing that there are so few people with these conditions. We need to recognize that each patient who has a rare disease has a very unique experience, even if they share the same diagnosis. Not all rare diseases are the same, and so what we need to do is make sure that we don’t treat these patients the same. Disease presentation, progression, and their response to therapy can really vary significantly from patient to patient.

In addition to all of that, there is nuance and really complex patient management needs. There is going to be dosing, monitoring, and support needed for all of their medication care. And also we need to make sure that we’re developing a customized approach that allows our care teams to address the clinical needs, of course, but also any additional needs that the patient may have. This could include adherence, education, and even quality of life considerations for these patients.

Lesley Pink: Can you walk us through how you would design a clinical program for a rare disease patient?

Maria Roberts: When we’re developing a program, we really need to have a very good understanding of the rare disease itself. So as we begin to develop the program, it’s a thorough review of the disease, including a typical path to diagnosis. It’s important that our team has a really deep understanding of delays to treatment as well as any misdiagnoses that the patient may be experiencing on their way to this treatment. We’ll also review current treatment landscapes and really understand what’s currently available and then any emergent therapies, getting a really good understanding of the treatment guidelines, any consensus statements or clinical recommendations, and we use all of that to develop an evidence-based care program. We also work really closely with SMEs to get a really good understanding and then generate all these insights needed to make a program that’s really going to support the patients.

Lesley Pink: A lot of factors go into consideration when doing this. Are there additional factors as well that you can think of?

Maria Roberts: Yeah, when we work with these patients, we really want to focus on making sure that we’re gathering the right information upfront so we can really understand that whole patient, not just the diagnosis. This would include things like treatment history, any risks or expectations and barriers that they may be facing. We also want to make sure we’re tailoring all of our support that sets that patient up for long-term success. Again, realizing that this is a unique patient and unique experience.

Lesley Pink: And how has your staff been educated on rare disease?

Alicia Morris: Like Maria said, our training foundation really starts with collaborating with subject matter experts. And then we utilize that evidence-based data to develop training on each condition. Our teams are trained not only on the condition and the treatment options, but also the clinical challenges that are unique to that rare disease. Our training is very in-depth Let’s be honest. Drug names are not always easy to pronounce, so we even have training on that.

And then something that I do feel is very valuable… our staff also receives training in empathetic and compassionate care, which really helps support our rare disease patients, not just clinically.

Lesley Pink: That’s really interesting. And we’re talking somewhat in generalities now. Recently though, you supported the launch of a new therapy for a rare lung disease. How did you approach designing a clinical program for a brand-new product launch, especially when there may be limited real-world experience?

Maria Roberts: That’s a great question. In this case, we really started with the foundation that we use for all of our rare disease programs, which is that deep understanding of the condition, the patients who have this condition, and any available clinical evidence that’s out there.  I’ll start with new products that really have a lot of limited real-world data. We have to rely on clinical trial data, any existing treatment guidelines, and then also a close collaboration with our pharma partners and any experts in the field to really get an understanding of what this upcoming therapy is going to look like. It is really critical as we build this program that we are flexible. There could be changes. We may need to adjust the program, adapt it, and evolve it as we learn more from our patients and our providers. We do place a strong emphasis on early patient education and proactive outreach to ensure that our patients are onboarded and ready to start this therapy as soon as it launches.

Lesley Pink: Speaking of that, were there any early lessons you had once patients did start therapy?

Alicia Morris: Once a drug becomes available in the market, that’s really where that real-world experience begins. And it’s essential to have partners who actively share feedback and collaboration with all of the stakeholders. And that’s exactly what we did. Through patient feedback, we were able to understand some common questions and concerns that the patients were having. And that led to being able to provide additional patient education, like Maria mentioned, and in turn we saw this contribute to lower discontinuation rates and better adherence patterns.

Lesley Pink: And again, as we’re talking about dropping therapy, are there specific monitoring protocols or interventions? Are there adherence issues you see and how do you address those?

Alicia Morris: Sure, so I think any time you have a new to market therapy, it can create some uncertainty for patients. Again, education is always a major focus for our clinicians, and we provide detailed patient education. We engage with the patients proactively to monitor their therapy and then provide interventions if it’s needed. I think when we see adherence issues, especially early in therapy, it often happens when patients aren’t informed or don’t know what to expect. Again, the more education we can provide and address questions to help ease those concerns and assist them in feeling more confident in their treatment, the better off we see adherence.

Lesley Pink: Can you talk more about a recent case study you completed on a rare disease population?

Maria Roberts: We actually collaborated with one of our partners and identified some potential adherence concern in some of our patients. As a result, we adjusted our call cadence for some more intentional and timely patient engagement. Alicia, did you want to talk a little bit more about that?

Alicia Morris: Sure. That enhanced early outreach really did provide us with the opportunity to address patient concerns quickly. And through that proactive monitoring, our clinicians were really able to have meaningful conversations with our patients. And that ultimately led to improvement in their refill behaviors. And the patients were just overall more confident in their plan of care.

Maria Roberts: And I think this really highlights how we work really closely with all of our partners and just make sure that we’re providing the best care possible for our entire rare disease population.

Lesley Pink: And we’ve talked specifically about programs for two lung diseases. How would you refine a clinical program for another type of rare disease?

Maria Roberts: I think another important piece to consider here is making sure we continue to recognize any distinct characteristics and any considerations we have for care for that rare disease. It’s important to really understand the differences in the condition and then how each condition uniquely impacts these patients. We want to also continue to work really closely with our partners to ensure that we align our shared goals.

And then as we build the program, we want to make sure we’re designing a support that really promotes smooth onboarding for the patient. They’ve already gone through a lot to get to this diagnosis as well as ongoing management. For us, patient experience is really the center and provides the most meaningful outcomes.

Lesley Pink: And we’ve talked about how you work with patients. How do you work with manufacturers of these specialty drugs?

Maria Roberts: Absolutely, it’s really important that we have a strong partnership with the manufacturers. We really want to have a shared commitment to ensure that we’re delivering the best possible care for all of our patients living with rare conditions. Our focus is really making sure we align on patients’ needs, helping them meet the clinical goals, and having overall treatment success. As Alicia already alluded to, we really strive to maintain open communication and make sure that all of the programs that we’re developing are thoughtfully designed and that they are adaptable to any real-world experience. We want to make sure we’re working with our partners for smooth access to therapy for their patients as well as providing clinical support and then also providing post-launch insights.

Lesley Pink: And how does your team measure success both on the clinical side and on the operations side?

Alicia Morris: So clinically, we measure success through patient-reported outcomes, safety data, adherence, and persistence to therapy, and also reviewing insights from our clinical interventions and outcomes. And then operationally, we measure success through access to therapy, turnaround times, our patient and provider experience surveys, and also cost management and resource utilization.

Lesley Pink: What kind of tools and innovations are you using in your work?

Alicia Morris: Technology supports our work, but our human connection really remains at the core. And so, if you think about it, there’s lots of ways today to communicate with patients. And it’s essential that we engage with the patient in a way that works best for them. Our technology-enabled workflow tools help us with this. We also use dashboards, queue management systems, patient engagement platforms.

From a patient engagement perspective, we do utilize dynamic capabilities that help us to drive appropriate patient communication. And we also rely heavily on data analytics tools. That’s both within our organization and with our external partners. And I would be remiss if I didn’t talk about AI. And so, we’ve worked to integrate AI, and we continue to develop and optimize that.

Lesley Pink: And how do you see specialty pharmacy evolving in rare disease over the next few years?

Maria Roberts: I’ll start with this. One of the things that I’ll call out is that the rare disease pipeline continues to be very robust. There are more therapies coming to the market and for a wide range of conditions that currently have unmet needs. In addition, new innovations, new treatments are on the horizon that are going to force specialty pharmacy to continue to evolve and ensure that they’re meeting the needs of our patients. Also, a need for deeper clinical expertise, very strong patient engagement, and continuing strong collaborative relationships with our manufacturer partners.

Lesley Pink: Alicia, do you have anything? What do you see?

Alicia Morris: AI is a hot topic right now in a lot of industries and specialty pharmacy is no different.  I really think that the rare disease space is uniquely situated and suited for AI capabilities. If you think about it, rare disease, we have small patient populations, the data is often fragmented. And so, what that gives you is sort of just enough data and a lot of complexities. This is where AI tends to shine. I think that we’ll see specialty pharmacy industry really evolve around AI over the next several years.

Lesley Pink: This was a really good conversation. Thank you, Maria and Alicia, for joining us for the April episode of the Strategic Dose. Please join us for the next podcast in May. Remember that you can find us on Apple podcasts, Spotify, and YouTube.