Report released for Rare Disease Day shows that while these diseases are rare, the barriers that patients face are unfortunately all too common

PITTSBURGH, Feb. 25, 2026 /PRNewswire/ -- As the world prepares to mark Rare Disease Day on February 28, the rare disease community faces a moment of both reflection and optimism, with a record number of therapies advancing toward FDA approval. According to IQVIA, 40 to 45 rare disease therapies are expected to launch in the U.S. over the next 18 months, continuing a multi-year trend in which orphan drugs account for more than half of new active substance approvals.

To help illuminate the impact of this rapid expansion, PANTHERx released new findings capturing the real-world experience across rare disease patients, prescribers, and payers supported by a dozen specialty pharmacies.

The research comes at a pivotal time as the rise in highly specialized rare therapies underscores the need for stronger, more reliable support systems to handle increasing complexity without placing additional burdens on patients and caregivers.

Key Findings
The survey results reveal a rare disease care system where communication gaps, preventable delays, and limited support place significant responsibility on patients and caregivers to manage each individual complex care journey.

Despite living in an increasingly connected world, 90% of patients and caregivers report delays due to communication-related issues and information gaps:

• 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.
• 90% of patients and caregivers say they have to advocate for individualized care tailored to their needs, and more than half feel responsible for determining the next steps on their own.

Even as therapies advance, 72% of patients are still asking for the fundamentals of support and services:

• Less than 10% of patients and caregivers reported facing no barriers during diagnosis (3%) or when trying to start treatment (9%), and challenges persist into sustaining care; 47% say support feels generic rather than personalized to their situation.
• Two of the most valued specialty pharmacy functions emphasize ongoing relationships: 72% of patients and caregivers would find dedicated coordinators who stay involved over time very helpful, and an equal share (72%) stated that seamless coordination without patient intervention would be very helpful.

Stakeholders converge on a clear priority: a single, reliable point of coordination is the top-ranked solution across stakeholders as the most effective way to reduce delays and ease the burden on families:

• Nearly three-quarters of patients and caregivers (73%) and healthcare professionals (73%) agree that it's not always clear who is responsible for coordinating care for rare disease patients.
• Nearly half of patients (49%) say their ideal specialty pharmacy is one primary partner who knows their situation and manages logistics across doctors, insurance, and pharmacy.

"Insights like these from a range of rare disease stakeholders make clear that therapy innovations alone aren't enough," said Dr. Richard Faris, Chief Commercial and Clinical Officer at PANTHERx Rare. "Each rare disease journey is complex and deeply personal. Coordinated navigation, clearer communication, and stronger clinical partnerships are essential to ensure today's therapeutic advances translate into meaningful, durable improvements across a patient's entire care journey."

PANTHERx Rare: A Purpose-Built Model Designed for Rare Diseases
As therapies become more specialized and care journeys more complex, the research underscores the need for approaches that reduce fragmentation, anticipate barriers, and relieve patients and caregivers of the burden of managing their own care. PANTHERx applies these principles through its RxARECARE® approach that delivers hyper-personalized care through long-term coordination, patient advocacy, and collaboration across providers, payers, and pharmaceutical partners. By listening closely to the rare disease community, PANTHERx Rare remains focused on identifying and implementing improvements to make complex rare journeys less overwhelming.

To learn more, download the research summary report here.

About the Research
The survey was fielded January 27-February 11, 2026, among 226 rare disease stakeholders, including 126 patients and caregivers, 75 specialist healthcare professionals, and 25 payer decision makers who make or influence coverage decisions for rare and high-cost therapies. The research was conducted by Morning Consult in partnership with PANTHERx Rare Pharmacy to increase awareness of patient challenges for Rare Disease Day (February 28, 2026). 

About PANTHERx Rare 
PANTHERx Rare makes rare disease care more hyper-personalized and less overwhelming by focusing relentlessly on each patient and each therapy. PANTHERx experts develop deep personal relationships with patients, prescribers, and pharmaceutical partners, serving as trusted advocates to ensure seamless collaboration and exceptional care. Since its founding in a garage in Pittsburgh, PA in 2011, PANTHERx has grown into the largest independent rare pharmacy in the U.S., leveraging established-company resources while maintaining small-company responsiveness, innovation, and attention to detail.

PANTHERx is licensed in all 50 states and U.S. territories and was the first national pharmacy to achieve dual accreditations in rare disease from the Accreditation Commission for Health Care (ACHC) and Utilization Review Accreditation Commission (URAC). PANTHERx is also the eight-time winner of the prestigious MMIT Patient Choice Award for patient satisfaction, including the 2025 honor.

For more information, please email TheRareSP@pantherxrare.com or visit www.pantherxrare.com.  

References:
1. Stefan Lutzmayer, et al, "From Orphan to Opportunity: Mastering Rare Disease Launch Excellence," IQVIA, April 30, 2024.
2. "Rare Disease Care Landscape Survey," Morning Consult, 2026. Custom survey commissioned by PANTHERx Rare Pharmacy and conducted by Morning Consult from January 27 to February 11, 2026, among 226 rare disease stakeholders, including 126 patients and caregivers, 75 specialist healthcare professionals, and 25 payer decision makers involved in coverage decisions for rare and high-cost therapies. Data are unweighted.

Media Contact:
PANTHERx Rare
Jennifer May, Senior Manager, Marketing Communications 
jmay@pantherxrare.com 

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SOURCE PANTHERx Rare Pharmacy