Feature|Videos|July 7, 2026
Launch is Just the Beginning: Rare Disease Care Requires Durability
Author(s)Bansi Nagji, CEO, PANTHERx Rare
Key Takeaways
- Understand why scientific innovation targets rare and orphan disease treatments
- Learn what distinguishes a rare-only pharmacy model from traditional specialty pharmacy approaches
- Recognize why launch is only the starting point for long-term therapy success
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Bansi Nagji discusses the shifting science behind rare and orphan drug development, including what it takes for therapies to succeed well beyond launch
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Bansi Nagji, CEO of PANTHERx Rare, explores how innovation is increasingly directed toward rare and orphan diseases, driven by targeted incentives, tax breaks, and a more streamlined FDA approval pathway. Nagji covers the operational realities that set rare disease therapies apart, including cold chain logistics, weight-based titration regimens, and the intensive patient interactions required before a single prescription is even filled. Learn why launch marks the beginning of a therapy’s journey rather than its conclusion, and why ongoing work is necessary for payer access and long-term adherence.
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