Blog Archives - PANTHERx Rare

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

Phillip Mulkins — 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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Seeing the Zebra Can Change Everything

Phillip Mulkins — 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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Specialty Pharmacy Continuum: As Rare Disease Drug Approvals Accelerate, Survey Highlights Gaps in Care Coordination

Phillip Mulkins — Mon, 02 Mar 2026 20:30:03 +0000

As the pipeline for rare disease therapies continues to expand, new survey data suggest that the healthcare system is not keeping pace with the coordination demands required to deliver those treatments effectively.

A national survey commissioned by PANTHERx Rare and conducted by Morning Consult found that patients, caregivers, and healthcare professionals perceive wide gaps in rare disease care delivery, whereas payors view care coordination far more favorably.

Among patients and caregivers, 76% rated the current rare disease care system as average, below average, or poor, with 44% describing it as poor. Healthcare professionals were even more critical: 86% rated the system as average or worse, and 50% described it as poor. In contrast, a majority of payors (58%) rated the system as good, revealing what the report describes as a stakeholder disconnect.

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Celebrating Rare Disease Day at PANTHERx: A Day of Purpose, Connection, and Community

Phillip Mulkins — Mon, 02 Mar 2026 14:23:54 +0000

Rare Disease Day is more than a date on the calendar at PANTHERx. It’s a moment to pause, reflect, and reconnect with the mission that drives our work every day. Although Rare Disease Day falls on a Saturday this year, associates across our Pittsburgh and Collierville locations came together on Wednesday, February 25 for a companywide celebration honoring the strength, resilience, and stories of the rare disease community.

A Day Built Around Purpose

From the moment the day began, I felt the energy across all sites reflecting our PANTHERx Pride. Associates wore their PANTHERx gear, gathered for a catered lunch, and stopped by the photo booth to capture team moments. Throughout the day, employees also participated in “drop-in” testimonial recordings , sharing, in their own words, what Rare Disease Day means to them. The celebration continued with a watch party of Matter of Time, a documentary highlighting the lived experience of the rare disease, epidermolysis bullosa (EB).

Welcoming a Special Guest: Leah Bender

The heart of this year’s celebration was our Patient & Caregiver Panel featuring 14-year-old Leah B. and her parents. Leah lives with PraderWilli syndrome and is on a therapy to treat hyperphagia. Her family graciously joined us for an intimate conversation about their journey and their experience working with PANTHERx.

Associates heard firsthand about the daily realities of living with a rare disease, the importance of coordinated care, and the difference compassionate support can make. The Bender family’s openness and honesty reminded me, and all of us why our work matters, deeply and personally.

To help Leah feel welcome, we set up a cardmaking station where associates wrote notes of encouragement and appreciation. We also surprised her with a gift of PANTHERx swag!

Carrying the Spirit Forward

Rare Disease Day is a reminder that every role at PANTHERx contributes to something bigger. Whether you work directly with patients or support the teams who do, our work helps deliver hope, clarity, and care to those living with rare conditions.

Thank you to every associate who participated, volunteered, or simply showed up with pride and purpose. And a special thankyou to the Leah and her family for trusting us with their story and inspiring us with their strength.

At PANTHERx, every day is Rare Disease Day — and this year’s celebration made that clearer than ever.

Angelica Duria, Senior Manager, Communications and Engagement

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Patient-centric care needs to be at the epicenter of CGT expansion

Phillip Mulkins — Wed, 18 Feb 2026 19:10:33 +0000

Dr. Jastrab speaks on a panel at Advanced Therapies Week in February 2026.

By Abigail Jastrab, PharmD, BCPS

Director, Patient Engagement and Clinical Innovation

As the Director of Patient Engagement and Clinical Innovation at PANTHERx® Rare, my work sits at the intersection of clinical excellence and the lived patient experience. Every day, I see how transformative these therapies can be, but I also see the very real barriers that stand between patients and the care they need. My role is to help design clinical models based on the complexity of these journeys while creating interactions that feel personal, accessible, and supportive.

Attending Advanced Therapies Week and participating in the “Patient-centric by design: Creating therapies with the patient in mind” panel was insightful and meaningful, because it brought together voices across the ecosystem who share a commitment to ensuring that the promise of cell and gene therapy is reflected in the support structures patients deserve.

The timing is also truly relevant as the cell and gene therapy (CGT) field is expanding at an unprecedented pace. Today, 37 CGTs are FDA approved, and nearly 2,000 programs are progressing through clinical development.¹ Many of these therapies target rare diseases and oncology populations with already complex, emotionally demanding, and resource-intensive care journeys.

During the panel, I was glad to hear similar perspectives from my colleagues who also recognize that as this pipeline grows, one reality is becoming increasingly clear: Education and transparent communication is paramount, especially as these therapies expand into community-based practices. Patient-centric design must evolve if patients are to start, complete, and safely navigate highly complex therapies.

Having consensus across my fellow panelists was certainly positive, but careful consideration needs to be made as to how we turn this insight into an actionable plan.

A few themes emerged during the panel that can help us action and create a CGT model that reflects the realities of patients’ lives.

Reach: Expanding Real World Access

During the panel, we examined what “access” truly means in a patient’s daily life. Beyond geography and cost, hidden barriers—like the inability to travel, taking time off work, or managing complex logistics—often disrupt CGT readiness. The group emphasized the need for stronger provider and payer education and care pathways that reflect real-world patient burdens.

Right Patient, Right Product

Matching patients to the appropriate therapy includes developing standardized measures of immune fitness to guide eligibility, timing, and stratification. Healthy cell samples must be collected before pre‑treatments, because those therapies weaken the immune system and may leave patients too fragile to continue safely. Early collection may ensure healthier, more viable cells instead of waiting until the patient is too sick for optimal harvesting.

Learn and Sustain: Building Trust and Long-Term Engagement

CGT is one-time dose, long term care. Patients require sustained support, clear expectations, and trusted partners to navigate multisite journeys. The conversation highlighted the importance of long-term monitoring systems that capture early safety signals and support durability in a patient-centric way.

To me, this was the heart of our panel discussion, and what excites me the most, as it directly aligns with what we are building at PANTHERx through our hyper-personalized RxARECONCIERGE model delivering individualized, proactive engagement across the entire patient journey to support long-term monitoring.

This work belongs to all of us: therapeutic developers, payers, providers, specialty pharmacies, advocates, and patients themselves. When we collaborate, we can deliver on the full promise of advanced therapies.

I am proud to have a voice in building the infrastructure that brings that vision to life.

1. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products

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PANTHERx Rare Opens New Pharmacy Fulfillment Center in Collierville, TN

PANTHERx Rare — Tue, 30 Sep 2025 12:00:16 +0000

PANTHERx associates celebrate Collierville’s first dispense in August 2025.

PANTHERx Rare Pharmacy is proud to announce the opening of our new state-of-the-art pharmacy fulfillment center in Collierville, Tennessee. This expansion marks an important milestone in our mission to provide exceptional care for people living with rare diseases and to support our partners with scalable, best-in-class pharmacy solutions.

The Collierville facility has already reached its first 1,000 dispenses since its doors opened in early August, underscoring the speed with which the team has brought this operation online. In addition, this location provides additional capacity and redundancy to our Pittsburgh location, ensuring that PANTHERx can continue to scale while maintaining the highest standards of safety, reliability, and service.

Our new pharmacy is designed to meet the growing needs of rare disease patients today and into the future. This investment demonstrates PANTHERx’s unwavering focus on excellence, innovation, and patient-centered care. By adding capacity and ensuring operational continuity across multiple sites, we’re better positioned than ever to deliver life-changing therapies to the rare disease community.

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New Article from PANTHERx Explores Best-in-Class Approach to Improve Services for Patients Living with Rare Disease

PANTHERx Rare — Tue, 07 Jun 2022 16:29:05 +0000

As a five-time MMIT Patient Choice Award Winner with a portfolio of 25+ rare programs, PANTHERx attributes much of our success to our ability to hire and cultivate talent while simultaneously investing in state-of-the-art proprietary technology systems. With the expected surge of FDA rare medication approvals over the next decade, talent and technology will become increasingly crucial in order to continue to serve this growing patient population. To successfully complete, pharmacies will be required to adapt and scale their operations, expanding their knowledge and experience while keeping patient care front of mind.

Click here to read our latest article A Best-in-Class Approach from PANTHERx Rare: Harnessing Talent and Technology to Improve Service to Patients Living with Rare Disease as authored by our Trade Relations and Data Analytics Manager, Marissa Puc, Pharm.D.

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New Article from PANTHERx Examines the Evolution of Specialty Pharmacy Channel Strategy

PANTHERx Rare — Thu, 21 Apr 2022 17:38:40 +0000

Change has been a constant in the specialty pharmacy industry since its inception over two decades ago. New medications are routinely launched, more patient populations receive treatment, and public awareness of rare diseases has increased. Additionally, industry players and their specialty pharmacy management strategies have evolved to meet the needs of increasingly complicated treatments and the patients they serve.

Click here to read the latest article from PANTHERx Specialty Pharmacy Channel Strategy Evolves as Rare Disease Drug Launches Become More Prevalent as authored by our Vice President of Rare Markets, Justin Smerker, Pharm.D., MPBA.

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Celebrating Rare Disease Day, Today & Every Day

PANTHERx Rare — Mon, 28 Feb 2022 18:06:38 +0000

The History of Rare Disease Day

On February 29^th, 2008, EURORDIS, a non-profit patient alliance of now nearly one thousand organizations from 73 countries, celebrated the first Rare Disease Day with its Council of National Alliances across Europe. Leap Day was specifically chosen for this special designation as it is a “rare” day that occurs only once every four years. Although advocating for rare diseases is a constant determination, setting aside a special day for combined efforts provides an avenue for patients, specialists, policy makers, activists, and support organizations across the world to amplify the voice of the rare disease community.

Over the last fourteen years, the final day of February has been a day of hope, celebration, and awareness for people and families living with the daily repercussions of rare diseases. This globally coordinated movement has played a critical part in building the rare disease community to the force it is today, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.

Rare Disease Day provides an energy and focal point that enables rare diseases advocacy work to progress on the local, national, and international levels, through people and organizations that are multi-disease and diverse, but united in purpose.

Defining Rare Pharmacy

PANTHERx was founded with the mission to transform lives by delivering medical breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. Since our founding, PANTHERx has grown to dispense 25+ rare and orphan medications while supporting tens of thousands of patients across the country through our proprietary RxARECARE® programs and SWFT® technology systems.

“Rare Disease Day is a chance for us to celebrate the lives improved, the research performed, the medications launched, and the hope created, with our patients, associates, partners, and the rare disease community we serve,” said Rob Snyder, President of PANTHERx. “We are immeasurably proud of the role that PANTHERx and our associates have played in the health journeys of patients and families across the country. We are constantly evolving to meet the needs of new patient populations and expanded indications, while taping into elevated technology, and prioritizing the varying personal goals and preferences of the patients who are at the center of all we do. The passion and purpose of Rare Disease Day helps to fuel the fire for organizations like ours to continue to fervently pursue improved health outcomes for our patients.”

PANTHERx offers dozens of enhanced fully customizable services to our patients and partners ranging from rapid refill capabilities and patient text notifications to custom fulfillment and shipping solutions, and tailored data analytic suites. Our clinical and technical capabilities shine in each of our programs, but it is our personal touch, our ethos of sincere care, that sets PANTHERx apart.

Showing Your Support

Everyone, including individuals, families, caregivers, healthcare professionals, researchers, clinicians, policy makers, industry representatives, and the general public, can participate in raising awareness and taking action today for people living with rare diseases. Visit rarediseaseday.org to find information about the thousands of events happening around the world in celebration of rare diseases. Follow PANTHERx on social media and like, comment on, and repost our Rare Disease Day posts to spread the word about this important day with your family, friends, and connections.

To download your own digital and printable media, please visit https://www.rarediseaseday.org/downloads/ We always love to hear from you – tag us in your posts!

About PANTHERx Rare

PANTHERx Rare is one of the largest and fastest growing rare pharmacies in the United States. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. Although the overall incidence of rare diseases is as common as diabetes, less than 7% of the 7,000 known rare and devastating disorders have an FDA-approved therapy. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community.

PANTHERx was recently awarded Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy (NASP) and earned the inaugural Accredited Distinction in Rare Diseases and Orphan Drugs from the Accreditation Commission for Health Care (ACHC). PANTHERx is a four-time winner of the prestigious MMIT Patient Choice Award, including the 2021 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC.

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Celebrating Rare Disease Day

PANTHERx Rare — Thu, 25 Feb 2021 16:07:00 +0000

What is Rare Disease Day?
As scientific breakthroughs have emerged and modern forms of communication have connected patients, specialists, policy makers, activists, and support organizations across the word, the Rare Disease Community has grown and so has its passion to raise awareness in support of rare disease research and patient advocacy. In 2008, EURORDIS, a non-profit patient alliance of now nearly one thousand organizations from 73 countries, wanted to set aside a special day for combined efforts to raise awareness about the prevalence of rare diseases. Leap Day was specifically chosen for this special designation as it is a “rare” day that occurs only once every four years.

The first Rare Disease Day was celebrated on February 29th, 2008 by EURORDIS and its Council of National Alliances across Europe. The tradition quickly spread around the world, amplifying the voice of the rare disease community. Over the last thirteen years, the final day of February has been a day of hope, celebration, and awareness for people and families living with the daily repercussions of rare diseases. It is a chance for advocates, policy makers, and healthcare organizations to celebrate the wins; the research performed, the medications launched, and the patients who have received hope and the chance to live a more healthy life; while recognizing that there is much more research to perform, many more medications to launch, and still many more patients who are living with rare diseases desperate for answers.

PANTHERx Rare: Where Every Day is Rare Disease Day
PANTHERx Rare, now a part of Centene’s Envolve Pharmacy Solutions, is the one of the largest and fastest growing rare pharmacies in the United States. We dispense nearly two dozen rare and orphan medications while supporting tens of thousands of patients across the country through our proprietary RxARECARE® programs and SWFT® technology systems.

“We are constantly evolving, reimagining old programs and creating new ones for the benefit of our patients and their families,” said Rob Snyder, President of PANTHERx Rare. “We have cultivated teams of people who understand operations, business, leadership, and most importantly, rare diseases. Our associates provide every PANTHERx patient with a unique combination of clinical knowledge and personal empathy. We are so invested in the lives of our patients that their wins are our wins—at PANTHERx, every day is Rare Disease Day and we celebrate it.”

PANTHERx offers dozens of enhanced fully customizable services to our patients and partners ranging from rapid refill capabilities and patient text notifications to custom fulfillment and shipping solutions and tailored data analytics suites. Our technical and clinical capabilities shine in each of our programs, but it is our personal touch, our ethos of sincere care, that sets PANTHERx apart.

Rare Disease Day: Participate with PANTHERx
Visit rarediseaseday.org to find information about the thousands of events happening around the world on the last day of February. Follow PANTHERx on social media and like, comment on, and repost our Rare Disease Day posts to spread the word about this important day with your family, friends, and connections.

To download your own digital and printable media, please visit https://www.rarediseaseday.org/downloads/

We always love to hear from you – tag us in your posts!

About PANTHERx Rare
PANTHERx Rare, a Centene Envolve Pharmacy Solutions company, transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to patients afflicted with rare and devastating conditions. Although the incidence is as common as diabetes, less than 7% of the 7,000 known rare and devastating disorders have an approved therapy. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community.

PANTHERx was recently awarded Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy (NASP) and earned the inaugural Accredited Distinction in Rare Diseases and Orphan Drugs from the Accreditation Commission for Health Care (ACHC). PANTHERx is a three-time winner of the prestigious MMIT Patient Choice Award, including the 2020 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC.

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