Editor’s Note: Patient Worthy is honored to share this submission from Katie DiLorenzo, SVP, Patient and Pharmacy Services, PANTHERx® Rare.

If you’re living with a rare disease or caring for someone who is, you don’t need anyone to define “care coordination” for you. You live it every day. It’s every appointment, every insurance call, every unexplained delay. It’s the quiet work of managing a condition few people understand, while trying to figure out who is supposed to do what next.

For many patients, the hardest part of rare disease isn’t just the diagnosis. It’s navigating everything that comes after: appointments, insurance decisions, paperwork, follow-ups, and the constant question of who is responsible for the next step.

A February 2026 national research survey on the state of rare disease patient care conducted by PANTHERx and Morning Consult, across patient experiences with 16 specialty pharmacies, confirms this reality. Nearly nine in ten patients and caregivers reported experiencing delays because critical information didn’t move between stakeholders. Not due to lack of effort—but because no one was accountable for the whole picture.

The survey showed information gaps remain a major barrier, with patients most often missing clarity on insurance or coverage status and who is responsible for moving the process forward. 68% of respondents reported waiting for updates or answers from doctors, pharmacies, or insurance.

When Patients Become the Coordinators

One of the clearest findings from the research is how much of the coordination burden falls directly on patients and families:

• 90% said they personally feel responsible for advocating for individualized care
• More than half said they are often the ones determining what happens next in their care journey.

Patients described waiting for updates that never come, repeating the same information to different people, and trying to understand insurance decisions without clear explanations.

That’s not what coordination should feel like, especially not in rare disease, where the stakes are high and the pathways are anything but predictable.

What Care Coordination Should Mean

At its core, coordination should reduce the burden on families, not add to it. Patients repeatedly told us they want:

• One person or team who knows their situation
• Proactive communication—updates before they have to ask
• Clear roles and responsibilities among providers, payers, and support organizations
• Someone looking across the entire journey, not just one transaction
• Support that remains personal, not generic, and doesn’t disappear after treatment starts

Patients don’t expect the system to be perfect. They just don’t want to feel alone in it. Yet today, rare disease care remains highly fragmented. Each stakeholder may be focused on their piece, but no structure consistently ensures someone is looking across the entire experience.

Across patients, healthcare professionals, and even payers, there was strong agreement on one point: having a single, reliable point of coordination is the most effective way to reduce delays and confusion in rare disease care.

Why Coordination Often Runs Through a Rare Pharmacy

If you’re living with a rare disease, there’s a good chance a specialty pharmacy is already part of your care. Many rare therapies are distributed this way because they’re complex, costly, and/or require ongoing monitoring.

But for patients, that experience can vary widely.

Traditional specialty pharmacies are built for scale: high volumes, established protocols, and therapies with predictable workflows. Rare disease is the opposite:

• Therapies are often first-in-class, complex, and highly individualized
• There are few, sometimes no, established clinical protocols
• Patient journeys are nonlinear and full of uncertainty
• Clinical, financial, and emotional burdens overlap in ways typical models don’t accommodate

These journeys require support tailored at the individual level, not to a segment or condition category. This is why care structures that work in broader specialty pharmacy simply do not translate to rare disease. Rare patients need more than episodic check‑ins or transactional dispensing. They need a model built for uncertainty.

Survey respondents shared that two of the most valued specialty pharmacy functions emphasize ongoing relationships:

• 72% of patients and caregivers coordinators who stay involved over time very helpful,
• An equal share (72%) stated that seamless coordination without patient intervention would be very helpful.

That gap is part of why PANTHERx built a pharmacy model focused exclusively on rare disease. From the beginning, our goal wasn’t just to deliver medication, it was to help navigate complexity so patients wouldn’t have to.

What we’ve learned is that coordination only works when it’s intentional and personal.

For patients, that means having a consistent team who knows the journey you are on, understands the disease, stays involved beyond the first fill, and communicates proactively, especially during moments when things tend to stall. It means fewer handoffs, fewer surprises, and fewer times where you’re left wondering who is working on your behalf.

A Better Future for Rare Disease Care

Rare disease care is never simple, but it shouldn’t feel isolating. Rare disease coordination cannot be episodic, reactive, or optional. It must be foundational, structurally built for complexity, and deeply human.

We still have work to do as a healthcare system. But listening to patients has made one thing clear: When care is coordinated the right way, patients are informed, supported, and connected rather than having to hold everything together, and that’s what care coordination should actually feel like.

A Place to Share—and Learn From—Each Other’s Stories

One thing we’ve learned by listening to patients is that no one understands rare disease better than the people living it.

That’s why we created Rare Stories, a space dedicated to sharing real experiences from patients, caregivers, and advocates across the rare disease community. These stories highlight the moments that don’t always show up in medical charts or policy discussions, the waiting, the resilience, the questions, and the small wins that matter.

Explore Rare Stories: Rare Stories – PANTHERx Rare

About the Author: Katie DiLorenzo, PharmD, serves as Senior Vice President of Patient and Pharmacy Services at PANTHERx, driven by a deep commitment to improving the experiences and outcomes of patients with rare and complex conditions. She leads a team of more than 500 dedicated team members who help support patients and families.