Asembia 2025: How Specialty Pharmacies Are Evolving to Support Rare Conditions

In an interview with Pharmaceutical Commerce, Will Pih, co-founder, Two Labs, shared insights from his recent panel discussion on “Economics and Specialty Pharmacy for Rare Diseases: Challenges and Opportunities.” The conversation explored the growing complexities of delivering care in the rare disease space, particularly as specialty pharmacies and pharmaceutical manufacturers navigate high drug costs, small patient populations, and an evolving regulatory landscape. Pih discussed the need for innovation, patient-centric service models, and policy reform to ensure sustainability and access without compromising the quality of care.

PC: You were a part of the ‘Economics of Specialty Pharmacy for Rare Diseases: Challenges & Opportunities’ panel. Can you provide a brief synopsis of what the session entailed?

Pih: The session centered around the current economic challenges in specialty pharmacy, especially in the context of rare diseases. Both manufacturers and specialty pharmacies are facing increasing pressure. With small patient populations and high-cost therapies, the financial dynamics become even more complex. The goal was to highlight these difficulties from both the pharmaceutical and provider perspectives.

PC: High costs are often unavoidable in rare disease therapies—what innovative strategies are you seeing to balance affordability and sustainability without compromising patient care?

Pih: There are several promising approaches. From the pharma side, particularly in rare diseases, there's a growing emphasis on enhanced pharmacy and patient services. Hubs are playing a more critical role—they're either developed internally, with proprietary technology and staff, or outsourced to external partners offering advanced platforms. Some companies are adopting hybrid models that combine both.

On the specialty pharmacy side, there’s impressive innovation around patient support. I’ve visited a few specialty pharmacies and seen firsthand the rare disease-specific training they provide. They’re continuously working on quality improvements in clinical care and call center interactions, becoming increasingly patient-centric to meet the complex needs of this population.

Full Interview Summary: The panel on "Economics and Specialty Pharmacy for Rare Diseases: Challenges and Opportunities" focused on the pressing financial and operational challenges within the rare disease specialty pharmacy landscape, particularly from both the pharmaceutical and patient/provider perspectives. With small patient populations and extremely high drug costs, rare diseases present unique difficulties for maintaining sustainable yet affordable care models.

Key innovations discussed included the evolution of patient services through advanced hub models. Pharmaceutical manufacturers are increasingly leveraging internal or third-party hubs that incorporate sophisticated technology and personalized support to enhance patient engagement. Specialty pharmacies, on their end, are refining rare disease training and clinical services, with a growing emphasis on patient-centricity, quality improvement, and tailored communication strategies.

Regulatory uncertainty was highlighted as a significant concern in 2025, with fluctuating guidance from agencies like the FDA creating hesitation in hiring and investment. This paralysis has led some organizations to turn to outsourced service providers, which may benefit specialty pharmacies and support vendors in the short term.

A central recommendation from the panel was to recognize specialty pharmacies serving rare disease populations as a distinct class of trade. The panelists argued that the level of service required for rare disease patients—often involving higher-touch care and more complex coordination—justifies differentiated reimbursement and policy consideration.

Looking forward, panelists expressed optimism about the pipeline for rare disease therapies, particularly with the growth of cell and gene therapies. As these treatments evolve from individualized products to off-the-shelf options, specialty pharmacies must prepare to support more complex therapies and new conditions they’ve never encountered before—marking a transformative phase in patient access and outcomes.