Opportunities in Rare Disease Research: Embracing Unique Considerations

Rare disease clinical trials are gaining momentum—in 2022, more than half of the FDA’s novel drug approvals were treatments for rare or “orphan” diseases. 

Researchers are working hard to develop safe and effective treatments for thousands of rare diseases. To do so, sponsors and CROs must navigate the unique concerns within rare disease research and design trials that accelerate ethical, groundbreaking research.

So, how is rare disease research different from other forms of clinical research? And how can researchers design trials that consider these unique aspects?

Read this eBook that shares insights from leading experts and learn:

• Strategies for engaging patient communities and retaining participants 
• Tailored approaches and study designs for rare disease clinical trials
• Techniques to manage and optimize a lean supply chain