How Reimbursement Uncertainty Delays Access to Rare Disease Therapies

In the final part of Pharmaceutical Commerce’s conversation with Will Shrank, MD, co-founder and CEO of Aradigm Health, live at Asembia AXS26, the discussion focuses on the operational and policy challenges that continue to affect access to specialty and rare disease therapies.

Shrank points to provider reimbursement uncertainty as one of the most significant barriers delaying patient access. He explains that providers often must purchase therapies upfront through buy-and-bill models without knowing exactly what reimbursement they will receive through single case agreements, creating cash flow and sustainability challenges. According to Shrank, this unpredictability can make providers more hesitant to invest in the infrastructure and operational capabilities needed to consistently deliver advanced therapies, ultimately contributing to uneven patient access.

Looking ahead, Shrank says FDA policies supporting rare disease and cell and gene therapy development could have one of the biggest long-term impacts on market access. He highlights the agency’s efforts to create regulatory flexibility around development and manufacturing platforms, stating policies supporting more efficient manufacturing could help lower costs and improve the sustainable delivery of these therapies to patients over time.

Watch the previous installments of Shrank’s interview with PC:

1. Asembia AXS26: How the Next 3-5 Years Will Define Specialty Pharma
2. Asembia AXS26: How Drug Innovation Could Strain Access Systems