Beyond the Odds:
The Rare Disease Winning Formula

A candid conversation about the history, current state, and future of rare disease — and what it means for executives planning their portfolios and launches in this space. Watch the full recording, explore the underlying research, and connect with our team.

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Recorded May 6, 2026
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~60 min
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watch the recording
Key Takeaways

Four Ideas Worth Your Time

01
The market is bigger, and more crowded, than the headlines suggest
Over 6,500 orphan designations have been granted, but only ~14% of rare disease assets reach blockbuster status. Strategic differentiation, not just designation, drives commercial outcomes.
01
Launch readiness starts years before approval
Winning teams build evidence, access, and patient-finding infrastructure in parallel with development — not as a Phase 3 afterthought.
01
Pricing and access are converging with policy
IRA, value frameworks, and global reference pricing are reshaping the orphan economics. Portfolio decisions made today need to model the world of 2028, not 2018
01
Patient communities are commercial infrastructure
Advocacy partnerships, real-world data networks, and HCP education are now table stakes — and they take years to build authentically.
Companion Research

The Rare Disease White Paper Series

The data, frameworks, and case studies that anchored the conversation.
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