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...

How to sustainably develop your charity

In the rare disease field, patient groups play a crucial role in supporting patients and motivating research into their overlooked conditions. However, research from Global Genes found that half of the thousands of rare diseases do not even have a disease-specific...

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...