Accelerating innovation in rare diseases

Millions of people living with rare diseases are urgently waiting for diagnosis, care, and life-changing treatment options. Alexion’s Soraya Bekkali considers how to get new solutions to them faster.

Rare diseases are a global health concern. They affect an estimated 400 million people worldwide and are often severe, progressive, and life-limiting. For patients living with rare diseases, getting a diagnosis – then accessing treatment if it even exists – is a challenging journey, which, on average, takes over five years. And, despite significant R&D progress in some areas, there are still no meaningful treatment options for 90% of the estimated 10,000 rare diseases.

People living with rare diseases desperately need innovation in diagnosis, treatment, and care – and they need it fast. So, how can innovation in rare diseases be accelerated?

Expertise and innovation: Tackling the challenges of rare disease R&D

The complex biological nature of rare diseases and their infrequency in the general population pose a myriad of obstacles to R&D that can slow – or even stall – innovation.

• Read the full article in pharmaphorum's Deep Dive digital magazine