R&D for Rare Diseases is Growing: How Will Policy Developments Impact the Future Landscape?

The landmark ORPHAN Cures Act has catalyzed investment in rare disease research since its passage last year, bringing hope to millions of patients and their families. Early data demonstrates renewed progress across the rare disease ecosystem—from accelerated drug development timelines to expanded clinical trial networks. However, a dynamic policy environment now threatens to undermine this critical momentum. This session presents new data on rare disease investment impact through perspectives from researchers that are advancing breakthrough therapies, private equity investors, policy leaders, and patients experiencing transformed access to treatments. We'll examine what's at stake and why is it is essential to preserve the policy gains that are driving meaningful progress for rare disease communities.