PANTHERx Rare

The Voice of the Patient: Lessons from a Wilson Disease Roundtable

PANTHERx Rare — Wed, 27 May 2026 20:30:49 +0000

By Robert Fetty, Account Manager, PANTHERx Rare

Earlier this spring, I attended the Wilson Disease Association for its 2026 Spring Support Symposium in Ann Arbor, Michigan. It was an inspiring day centered on connection, education, and shared experience. During the event, we were honored to host a roundtable with our manufacturing and patient advocacy group partners focused on shaping the future of Wilson disease care.

What made this event truly special was its focus on everyday life with Wilson disease—support, adherence, recovery, and resilience. Hearing patient perspectives on treatment journeys, clinical trial participation, and rebuilding quality of life was a powerful reminder of why we do what we do at PANTHERx^® Rare.

About Wilson disease
Wilson disease is a rare, inherited genetic disorder that causes excessive copper to accumulate in the liver, brain, and eyes because the body cannot properly eliminate it. If left untreated, this toxic buildup causes severe organ damage, but it can be manageable with lifelong medical treatment, typically involving chelating agents or zinc. Symptoms often appear between ages 3 and 40. (Source: Mayo Clinic website).

Patients are people first
My work often involves prescription data and patient information.

But sitting across from the people behind those records—hearing their voices, their struggles, and their resilience—was a moving experience I won’t soon forget. Their stories carried both honesty and strength.

A relationship built on trust
One of the most consistent themes throughout the discussion was gratitude, not just for the medications patients receive, but for the relationships they’ve built with our team. Several patients spoke about knowing their PANTHERx care coordinators by name, and what that personal connection means to them.

When you’re managing a lifelong rare condition like Wilson disease, knowing there’s a real person on the other end of the phone who knows your situation and genuinely cares about your wellbeing is not a small thing. Familiar relationships create space for questions, concerns, and confidence to surface naturally.

The work behind the scenes
One moment from the roundtable particularly stood out to me. A patient shared that, until recently, they had no idea how much administrative work went into keeping their therapy covered and accessible. They had been focused, understandably, on managing their health and had simply assumed that things “just worked” on the pharmacy side. When they learned about the behind-the-scenes efforts—prior authorizations (PAs), appeals, and the coordination required to get a rare disease therapy covered and into their hands—they were genuinely surprised and deeply thankful.

Keeping physicians and care teams in the loop
I also had the chance to connect with several physicians at the event. Their feedback reinforced something we’ve always believed at PANTHERx: regular communication with prescribers is essential. Staying informed about access progress and challenges helps them support patients consistently and respond quickly as needs evolve.

The physicians were complimentary of our approach to patient care, and several emphasized how important it is to stay connected throughout the PA and appeals processes. They want to be partners in the journey with a shared focus on what matters most: patient experience and outcomes.

Inspired by the people we serve
I’ll be honest—I walked into this roundtable not knowing exactly what to expect, and I walked out inspired. Meeting patients face-to-face and hearing their stories has a way of cutting through the day-to-day hurdles and reminding you of the real stakes involved in what we do. These are people navigating a complex, lifelong condition, and they’re trusting PANTHERx to be in their corner every day.

That trust is something I’m proud to have earned, and it’s a responsibility our entire team takes seriously. We never take it for granted. Events like this roundtable are a vital reminder that behind every rare disease prescription is a person with a unique rare story, and that person deserves our very best.

Thank you to the Wilson Disease Association for creating a space where real stories, real questions, and real support took center stage. We’re proud to stand alongside this community and remain committed to supporting patients and families every step of the way.

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How Rare Disease Therapy Launches Will Adapt | Pharmaceutical Commerce

PANTHERx Rare — Fri, 08 May 2026 15:34:42 +0000

Author(s) Stephen Appezzato
Live from Asembia AXS26, Austin Russian examines how specialty therapy launches are evolving.

In part two of Pharmaceutical Commerce’s conversation with Austin Russian, SVP of Program Excellence at PANTHERx Rare, conducted live at Asembia AXS26, he discusses the advantages and challenges of manufacturers taking a more active role in the patient journey, as well as how rare disease and cell and gene therapy launches are evolving.

Continue reading at pharmaceuticalcommerce.com

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How Fragmentation Delays Rare Disease Therapy Access | Pharmaceutical Commerce

PANTHERx Rare — Tue, 05 May 2026 21:42:11 +0000

Author(s) Stephen Appezzato
Live from Asembia AXS26, Austin Russian explains how access to rare disease treatments can be delayed.

In the first part of his interview with Pharmaceutical Commerce, live from Asembia AXS26 in Las Vegas, Austin Russian, SVP of Program Excellence at PANTHERx Rare, focuses on the operational factors that can delay patient access to rare disease therapies. As more orphan drugs enter the market, stakeholders are paying closer attention to how access is managed across an increasingly complex care journey…

Continue reading at pharmaceuticalcommerce.com

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From Hope to Access: Three Lessons From the Front Lines of Rare Disease Launches

PANTHERx Rare — Thu, 30 Apr 2026 15:09:38 +0000

By Katie DiLorenzo, PharmD, SVP, Patient and Pharmacy Services and Austin Russian, PharmD, SVP, Program Excellence

Every day, our work is guided by one question: how do we help people with rare diseases get the treatments they need so they can focus on living their lives, not navigating systems? At the same time, we have learned from nearly 50 rare disease launches that they are never one‑size‑fits‑all. Every program must be built differently and centered around the patient from the very start. That perspective shaped our conversation at Asembia’s ASX26 conference, where hope took center stage—not as a slogan, but as something that depends on how well we coordinate once innovation meets the real world.

Across perspectives from advocacy, manufacturing, specialty pharmacy, and access, the discussion consistently returned to three priorities that define successful rare disease launches today.

Key Takeaway #1: Hope reaches patients fastest when launch strategies are designed as living programs, not static milestones

In rare disease, launches are often treated as a moment. Patients experience them as a journey.

Panelists emphasized that approval alone does not ensure access. True launch excellence depends on thoughtful planning and operational readiness that can evolve in the real world.

As one panelist put it:

“We’re not just launching a product; we’re often launching a market. If our program looks the same on day one as it does six months later, we’re not doing something right, because we’re not learning.”

First‑in‑class therapies frequently require building disease awareness and educating prescribers and payers simultaneously.

Key Takeaway #2: Coordination isn’t fewer voices, it’s aligned voices

Rare disease care is inherently multi‑stakeholder and no one stakeholder can succeed in isolation.

One observation that resonated strongly:

“When coordination breaks down, patients become their own advocates, connecting dots the system never connects for them.”

We see on a daily basis that without clear ownership and real‑time communication, responsibility shifts to patients and caregivers, who are already carrying the weight of diagnosis, uncertainty, and long delays. Fragmentation doesn’t eliminate work; it redistributes it to the people least positioned to manage it.

Panelists repeatedly stressed that coordination works best when partnerships are transparent, accountable, and designed to act as a single team on behalf of patients.

Key Takeaway #3: Patient‑centered support models must be built from lived experience

Hope becomes meaningful only when patients feel understood, supported, and confident in what comes next. Patient advocacy organizations play a unique role, bringing lived experience that no dataset can replicate. As one panelist noted,

“Including advocacy gives you more insight than any form of market research ever will. ”

The panel underscored how critical it is to truly understand the patient journey—especially in rare disease, where moments that look ordinary on the surface can carry very different meaning for families. One point resonated throughout the discussion:

“If you treat every patient the same, you’re not hitting the mark.”

One anecdote shared by our advocacy partner really drove home the point that empathy and trust must be built into programs from the start, informed by real context—such as understanding what a family is carrying into a conversation. They shared that for some genetic conditions, birthdays can remind parents of the stress and anxiety of realizing their child was born with a rare disease. What many consider a happy milestone may be far more complicated than it appears.

At PANTHERx^®, this isn’t theoretical for us. It’s personal. Every day, we meet patients and families who have already carried too much on their own. That understanding shapes how we show up: how we build support programs and how our RxARECARE® care teams communicate and educate. It’s why our teams get to know our patients as people and why we take the time to understand what a patient may be feeling in that exact moment—fear, relief, exhaustion, and yes, hope.

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The Top 15 Specialty Pharmacies of 2025 (PANTHERx #6)

PANTHERx Rare — Tue, 21 Apr 2026 13:03:00 +0000

Drug Channels Institute’s (DCI’s) latest analysis reveals that PBM-affiliated specialty pharmacies continue to dominate the dispensing of specialty drugs.

For 2025, DCI has identified more than 1,900 dispensing locations with specialty pharmacy accreditation from one or both of the two major independent accreditation organizations. The overall number of accredited locations grew by only 3% in 2025, but is more than five times larger than the 2015 figure.

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The Rare Journey Needs a Special Pharmacy Partner

PANTHERx Rare — Fri, 27 Mar 2026 13:27:10 +0000

PANTHERx Rare’s CEO, Bansi Nagji, authored an insightful guest post for Drug Channels Institute titled, “The Rare Journey Needs a Special Pharmacy Partner.” He makes the case that rare pharmacy is not simply a subset of specialty pharmacy—it requires an entirely different ecosystem. In the post, Bansi also details several key differences between rare pharmacy and traditional specialty, and why these differences enable the depth required to deliver hyper-personalized care to those living with rare conditions.

Download PDF | Read post at Drug Channels >

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From Insight to Intention: What the Rare Disease Community Is Asking of Itself

PANTHERx Rare — Tue, 24 Mar 2026 15:16:59 +0000

I recently had the privilege to serve as a Co-Chair of this year’s Access USA Rare Disease Summit, which means I had the privilege of hearing so many engaging and rich discussions, led by passionate leaders in the rare disease industry. One of the most powerful aspects of the experience was what emerged when conversations began to echo with one another… in a positive way.

Across conversations spanning development, access, commercialization, and patient experience, a shared set of questions helped anchor discussions in real-world realities:

Are we designing how care is delivered?

Are patient and caregiver voices shaping decisions early enough?

Are we clear about what evidence can and cannot tell us today?

Several themes stood out, not as theoretical ideas, but as practical signals for where rare disease strategy must continue to evolve.

Clinical Trial Design Shapes Access Long After Approval

Clinical trial design is an access decision. Early choices, endpoints, eligibility criteria, and diagnostics often become the basis for payer coverage long after approval. From my role at PANTHERx, I see how those upstream decisions show up downstream for patients, shaping who can start therapy and how long it takes to get there. That’s why access cannot be treated as a post-approval problem to solve later. Designing trials with real-world use in mind is essential to ensuring evidence supports not just approval, but meaningful, timely access for patients.

One Size Doesn’t Fit All in Rare Disease

Throughout the Summit, one phrase kept resurfacing: “When you’ve seen one patient, you’ve seen one patient.” It captured a shared reality across discussions, that even among patients with the same diagnosis, experiences can differ dramatically.

Speakers and patient advocates reinforced that rare disease burden extends far beyond what patient data captures. Emotional realities like isolation, frustration, and loss of independence often shape whether patients can stay engaged in care, even when the therapy itself is effective.

In my role at PANTHERx, we see how these non-clinical factors directly influence adherence and trust. That’s why hyper-personalized support is not a ‘nice-to-have,’ it’s foundational. Designing care around the whole person, not just the diagnosis or the trial protocol, is what makes rare disease care work in the real world.

Central Role of the Patient Voice

A clear takeaway from the Summit was that patient advocacy and patient voice are not nice to have; they are central to how rare disease care works. Advocacy partners shape trial design, support patients emotionally, build community, and translate lived experience for regulators, payers, and providers.

As a pharmacist by training, I’ve always seen care as something that happens in the community, not in isolation. Medications don’t succeed on their own; people do, supported by advocates, caregivers, and care teams who understand the realities patients face every day. When advocacy and patient voice are embedded early and authentically, they strengthen evidence, improve program design, and create care models patients can trust and sustain over time.

Looking Ahead

The questions that framed the Summit still matter most: are we designing real-world care, listening early enough to patients, and being honest about what evidence can—and can’t—tell us? In practice, progress only holds when those questions are answered in ways that reflect how patients live with and access care.

A heartfelt thank you to Informa and Access USA for their work to create an engaging conference. I’m deeply thankful to the speakers, advocates, and everyone who participated so openly. Your willingness to share experience, challenge assumptions, and listen to one another is what makes this community special, and what turns good conversations into better outcomes for patients and families.

By Austin Russian, PharmD
Senior Vice President of Program Excellence and Co-Chair, Access USA Rare Disease Summit

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The Top 15 U.S. Pharmacies of 2025 (PANTHERx® Rare #11)

PANTHERx Rare — Tue, 17 Mar 2026 21:07:15 +0000

Next week, the Drug Channels Institute (DCI) will release our 2026 Economic Report on U.S. Pharmacies and Pharmacy Benefit Managers. This 17th edition provides our most comprehensive and up-to-date examination of the U.S. drug pricing, reimbursement, and dispensing system.

For 2025, DCI estimates that total prescription dispensing revenues at retail, mail, long-term care, and specialty pharmacies reached $751 billion in 2025, up 10% from the 2024 figure.

Seeing the Zebra Can Change Everything

PANTHERx Rare — Thu, 05 Mar 2026 20:50:29 +0000

By Courtney Nitkiewicz, PharmD, CSP, Clinical Patient Engagement Manager, and Abigail Jastrab, PharmD, BCPS, Director of Patient Engagement and Clinical Innovation, PANTHERx® Clinical Team

On Rare Disease Day, stripes are more than a pattern; they’re a reminder to look closer. What may seem familiar at first glance isn’t always what it appears to be, and in rare disease, that distinction can change a life. This year, we brought that idea to a second and third grade classroom, where curiosity, empathy, and a simple zebra picture helped spark meaningful conversations about why rare diseases are often missed, why awareness matters, and how anyone, at any age can be part of the change.

We began by explaining what Rare Disease Day represents and by breaking down the difference between common and rare diseases. When we shared that there are over 7,000 rare diseases, the room went quiet. The surprise grew when we explained that only 5% of rare diseases currently have approved treatment. Since the students are learning fractions and percentages, they immediately understood just how large the gap is.

One of the most powerful moments came toward the end of the presentation. We showed the students a silhouette and asked them to guess what animal it was. Almost every hand went up confidently with answers like “horse” or “donkey.” When we revealed that it was actually a zebra, there was surprise and recognition.

That’s when we explained why people wear stripes on Rare Disease Day.

In medicine, there’s a saying: “When you hear hoofbeats, think horses, not zebras.” While this makes sense most of the time, rare diseases are the zebras, often overlooked because they look like more common conditions at first glance. Just like the silhouette, many rare diseases can resemble common illnesses, which can lead to years of misdiagnosis or delayed treatment.

We also talked about heroism. We explained that each of them could be someone’s hero simply by being a thoughtful classmate or friend. If a child with a rare disease needs to play a game differently or needs extra understanding, inclusion becomes a form of advocacy.

As we left the classroom, it was clear this experience was impactful not just for the students, but for us as well. Rare disease awareness can start with a question, a silhouette, and a group of curious young minds.

It was a truly rewarding event, and we’re already looking forward to seeing the kids next year, and perhaps one day as future leaders in rare disease! Sometimes, seeing the zebra early can change everything.

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Richard Faris, PharmD, Chief Commercial and Clinical Officer at PANTHERx Rare was a guest on the Pharmacy Podcast Network to discuss new stakeholder feedback data revealing communication gaps in rare disease care.

PANTHERx Rare — Mon, 02 Mar 2026 20:41:44 +0000

On this episode of This Week in Pharmacy, we spotlight two major conversations shaping the future of pharmacy practice in 2026.

First, we welcome Dr. Lisa Faast, PharmD, founder of DiversifyRx, to discuss the Pharmacy Profit Summit 2026 and how independent pharmacies can strengthen margins, diversify revenue streams, and build sustainable growth models in today’s reimbursement environment.

Then, we turn to Rare Disease Day 2026 with Richard Faris, PharmD, Chief Commercial & Clinical Officer at PANTHERx Rare, to discuss specialty pharmacy leadership, pharmacist impact, and new patient feedback data revealing communication gaps in rare disease care.

Segment 1: Pharmacy Profit Summit 2026
Guest: Dr. Lisa Faast, PharmD | Founder, DiversifyRx

Dr. Faast joins us to break down the vision behind Pharmacy Profit Summit 2026 — an event designed to equip pharmacy owners with practical strategies to increase profitability while maintaining clinical excellence.

Key Topics Covered:
• Why independent pharmacies must move beyond traditional dispensing revenue
• Diversification strategies including niche services and high-value offerings
• Real-world financial benchmarks pharmacy owners should track
• How mindset, leadership, and data discipline drive sustainable profitability
• The importance of collaboration and shared intelligence among pharmacy entrepreneurs

The Pharmacy Profit Summit is not just about revenue — it’s about equipping pharmacy owners to compete intelligently in a rapidly consolidating healthcare market.

Segment 2: Rare Disease Day 2026

Guest: Richard Faris, PharmD

PANTHERx Rare Pharmacy

In recognition of Rare Disease Day 2026, we are honored to welcome Richard Ferris, PharmD, to discuss the critical role pharmacists play in specialty pharmacy and rare disease care.

PANTHERx Rare recently released survey findings examining patient and caregiver experiences within rare disease treatment journeys.

The post Richard Faris, PharmD, Chief Commercial and Clinical Officer at PANTHERx Rare was a guest on the Pharmacy Podcast Network to discuss new stakeholder feedback data revealing communication gaps in rare disease care. appeared first on PANTHERx Rare.