The rare disease trial landscape stands at a pivotal tipping point. While unprecedented advances in trial design, digital tools, and regulatory flexibility are accelerating innovation like never before, the reality for patients remains stark: over 90% of rare diseases still lack an FDA-approved treatment. For many, clinical trials represent the only path to care, yet access remains uneven. Geographic isolation, diagnostic delays, and socioeconomic barriers continue to exclude many patients, especially from underserved communities, from participating in research.