Understanding the Challenges: Clinical Trials for Rare Diseases

Why is conducting clinical trials for rare diseases difficult? Because studying a rare disease poses unique challenges and requires thinking differently. At Alexion, our approach to rare disease clinical research is guided by the insights and perspectives we gain from listening to and learning from the rare disease communities we aim to serve. We continue to pioneer new approaches to accelerate the discovery and development of transformative medicine where scientific progress has stalled or there are limited or no treatment options – because we know the need is great, and people are counting on us.