Expertise: Rare Disease 73 results
Rare Disease
Why Rare Disease Day Matters to Us
An estimated 300 million people globally suffer from a rare disease. Further still, 25 million people in the U.S. alone are living with an undiagnosed rare disease. Limitations in disease understanding and lack of approved treatments can mean countless challenges and diminished quality of life for many battling a rare disease. For all those looking…
Rare Disease
Leslie Wetherell
In a world of ever-increasing clinical trial costs, sponsors can feel overwhelming pressure to stretch their budgets without sacrificing the data they need for regulatory approval. In rare diseases, this pressure is intensified with the many challenges that often accompany these trials, such as small patient populations, limited disease data, and patient engagement challenges. Here…
Rare diseases are a distinctive and complex field within clinical development, characterized by significant hurdles from start to finish. Addressing these complexities requires innovative approaches to achieve a future of adaptability, quality, and efficiency. Here are 5 key steps to bringing rare disease clinical development forward. Predicting the Future of Rare Disease Research As we…