People with rare diseases are missing out on new treatments because of lack of co-ordination on pricing and reimbursement in Europe, according to a leading campaigner.
On Rare Disease Day we relate the personal 'diary' experience of a 28-year-old woman with systemic scleroderma, an uncommon condition in which the immune system attacks the connective tissue un
NICE says it can approve BioMarin's ultra-rare drug treatment Vimizim, but has proposed real-world data collection and a price cap to overcome doubts about the drug's value, reports Andrew McCo
