Developing new therapeutics for rare diseases offers a tremendous opportunity, but it also comes with unique complexities. With small patient populations, researchers face significant challenges in trial design, data interpretation, and navigating the regulatory path to approval.
The Top Three Pitfalls in Rare Disease Drug Development
- Limited understanding of disease biology and natural history
- Unclear or incomplete definition of clinically meaningful endpoints
- Difficulty demonstrating clinical benefit and achieving full approval
What You’ll Learn in This White Paper
In this insightful paper, drug development expert Dr. Rajesh Krishna explores the core challenges faced by developers of rare disease therapies and how model-informed drug development (MIDD) approaches can turn these challenges into opportunities.
Key takeaways include:
- A concise overview of the biology and genetics driving rare diseases
- How to leverage existing regulatory frameworks to minimize uncertainty
- The limitations and nuances of the accelerated approval mechanism
- Strategies to streamline development using MIDD principles
Download the white paper to gain a deeper understanding of the opportunities and challenges shaping rare disease drug development, and how innovative modeling approaches can guide smarter, faster, and more successful therapeutic programs.
