Asembia 2025: FDA Uncertainty Sparks Shift Toward Outsourced Pharma Support

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: From your perspective, how is the current regulatory landscape either supporting or complicating rare disease access through specialty pharmacy?

Will Pih: That’s a tough question. There’s a lot of turmoil right now, and I think the main challenge is the uncertainty. Take the FDA, for example—there are many moving parts, and that uncertainty creates a kind of paralysis. People feel insecure and unsure of what to do next. They typically have a playbook they want to follow, but now that playbook is being paused or even rewritten. As a result, we’re seeing slower uptake in clinical trials and delays in hiring, as companies question what’s needed. Interestingly, this has actually benefited specialty pharma and pharma services overall—when companies delay hiring but still need to make progress, they often turn to outsourced services to keep things moving.

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.