NICE draft guidance rules against NHS funding of Biogen's spinal muscular atrophy therapy Spinraza

The National Institute for Health and Care Excellence published draft guidance Tuesday recommending against routine NHS use of Biogen's spinal muscular atrophy (SMA) therapy Spinraza (nusinersen) due to uncertainties over its long-term effectiveness and its "extremely high cost." The agency said it "would welcome further discussions about access" to the drug.

Spinraza gained European approval last year for the treatment of 5q SMA, making it the first therapy authorised in the region for the condition. The product was previously cleared in the US, where Biogen set the list price at $750 000 for the first year of treatment and $375 000 for each additional year of therapy.

In the UK, Spinraza's list price is 450 000 pounds ($574 884) for the first year of treatment, and 225 000 pounds ($287 472) for subsequent years. NICE noted that based on the list price, the cost-effectiveness estimates for the therapy ranged between 400 000 pounds ($511 008) and 600 000 pounds ($766 512) per year of quality adjusted life gained. The agency added that even with Biogen's proposed confidential price reduction, the cost of the therapy is too high.

Meindert Boysen, director of the centre for health technology evaluation at NICE, noted "the committee was willing to be flexible because of the nature of the condition and the paucity of the evidence, but the very high cost of [Spinraza] meant it could not recommend the drug as a cost effective use of NHS resources." Boysen added "we are actively engaging with Biogen to discuss how they might address the uncertainties identified by the committee."

According to NICE, while evidence showed that Spinraza provided a "substantial clinical benefit," the appraisal committee felt there were a lot of uncertainties around its long-term benefits. The draft guidance is open for public consultation until September 11.

Biogen, which licensed global rights to Spinraza from Ionis Pharmaceuticals in 2012, recently reported that sales of the product reached $423 million in the second quarter, up 108 percent year-over-year and topping analyst expectations of $393.8 million.

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