Designing an Early Access Pathway for a CNS Therapy Using Real-World and Claims Insights
Background: A mid-stage biopharma company was advancing a novel CNS therapy into late Phase II, with early clinical signals suggesting meaningful benefit for patients with progressive neurological decline. While the science was promising, leadership faced a familiar CNS challenge: patients needed treatment sooner than regulatory and reimbursement timelines would allow. Slow disease progression, subjective clinical endpoints, and long follow-up periods meant approval and payer decisions were still years away. Meanwhile, clinicians were encountering patients with limited alternatives, highlighting a growing access gap well before launch. The Challenge: The company needed an early access strategy that balanced urgency with responsibility. Key challenges included: Defining which real-world patients should qualify beyond the trial population Limited visibility into current treatment pathways and unmet needs Payer and regulator concerns around uncontrolled access and fut...