Introduction to Patient Registries

Rare diseases pose great challenges for research, primarily because each condition affects so few people. Patients are dispersed around countries, around the world, and very little is known about how the condition progresses and affects individual people. Patient...

Navigating NICE and drug evaluations

The number of treatments (orphan drugs) being developed for rare diseases is on the rise. In the USA, FDA orphan designations have steadily increased year on year since 2000 (EvaluatePharma – Orphan Drug Report 2014). While this is great news, these orphan drugs come...