Breaking barriers in rare disease drug development

Bringing rare disease treatments from pipeline to patient

Rare diseases affect more than 395 million people worldwide, yet only 5% have an FDA-approved treatment. The challenges of small patient populations, regulatory complexities, and limited clinical data have historically slowed progress. But the landscape is changing.

This infographic explores the key barriers in rare disease drug development and the innovations helping to overcome them. From AI-powered research to smarter clinical trial designs and evolving regulatory frameworks, new strategies are accelerating the path from discovery to patient access.

What’s Inside:

• The biggest challenges in rare disease drug development
• How AI, data-sharing, and patient-driven research are transforming drug discovery
• New clinical trial models improving patient recruitment and retention
• The role of regulatory collaboration in streamlining approvals
• Learn how advancements in technology, clinical strategy, and patient advocacy are reshaping the rare disease treatment landscape.

Download the full infographic to explore the future of rare disease drug development.