UK Rare Disease Drug Review Reform Urged As Spinraza Finally Gets NICE OK
It has taken 17 months of negotiations but soon 600-1,200 people in the UK may be able to get Biogen's spinal muscular atrophy therapy now that an access agreement has been inked.
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NICE will review new data in deciding whether to OK the use of Spinraza in non-ambulant SMA type III patients.
With the emphasis on getting lower prices even for cost-effective drugs, England’s proposed commercial framework may be sending a negative signal to global companies.
The NHS in England says it has made a “revised and improved” offer to Vertex to make Orkambi available, but the company is not impressed and says its attitude to the negotiations has been mischaracterized.