Last week I attended the Cambridge Rare Disease Network (CRDN) 2016 Summit. CRDN is a newly established charity working to build a community of people in Cambridge to address the unmet needs in rare disease research and treatment. As last year, there was a great set of speakers, from patient groups, academia, pharma/biotech and vendors.
There has been a step-change in awareness within the pharma industry in the last decade, with an increasing interest and investment in tackling rare diseases. I blogged last year about an interview with Patrick Vallance on this same topic. He gave several reasons why GSK are interested in rare disease, and these three reasons all were echoed by the CRDN speakers.
The topmost of these reasons was given most clearly by speakers from patient groups, such as Daniel Lewi from the Cure & Action for Tay-Sachs (CATS) Foundation, Karen Harrison from ALD Life, and Emily Kramer-Kolingoff, from Emily’s Entourage. They spoke of the huge impact that rare disease has on individuals and families, and the urgent need for research into new or repurposed treatments for the 1 in 17 people affected by a rare disease.