UK watchdog NICE has rejected Biogen’s spinal muscular dystrophy drug Spinraza (nusinersen) over cost concerns, denying patients the only available treatment for the condition.
The news is the latest in a series of roadblocks for the drug. Following its approval last year, review of Spinraza was delayed by the institute until January this year because of contention over how to how to handle its route to market. This time round, NICE took issue with the drug’s price tag. Even after Biogen agreed to offer the therapy at the reduced price of £450,000 for the first year of treatment – down from approximately £586,000 – the institute said “the cost of nusinersen is too high for it to be considered a cost-effective use of NHS resources.”
Besides cost-effectiveness concerns, NICE also noted uncertainty over the drug’s long-term benefits.
Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK, led the calls decrying the decision and its impact on patients: “Spinal muscular atrophy can be devastating and today’s news will be heart-breaking for the families of those living with the condition. Once again we are seeing families suffer due to the appraisal process being too limited to assess costly but life-changing rare disease drugs.
“We call on NICE, NHS England and the drug company, Biogen, to urgently come together and find a solution to provision and pricing so families can get Spinraza before more lives are lost,” he added.
NICE has said that Biogen has offered to develop a proposal for a managed access programme for the drug in cooperation with patient groups and other shareholders. Meindert Boysen, NICE’s Health Technology Evaluation Director., commented: “We are actively engaging with Biogen to discuss how they might address the uncertainties identified by the committee, while demonstrating the potential for nusinersen to be considered cost effective and managing the risk to the NHS of allowing access to this treatment.”