Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy UK:
“SMA is a life-limiting rare condition and patients desperately need access to Spinraza – the first and only treatment for this devastating condition. Without it, babies with the most severe form of SMA are likely to die, while others will irreversibly lose the ability to walk, crawl, breathe and swallow.
Spinraza is an award-winning drug, yet NICE is denying patients access. It’s crucial that the drug manufacturers, Biogen, and NHS England negotiate on the price of the treatment. NICE also needs to look at its appraisal processes, which are clearly not fit for purpose when it comes to assessing treatments for rare conditions. Failure to come to an agreement will impose a death sentence on many infants.