Neurology Central

NICE issues draft guidance against Spinraza® for spinal muscular atrophy

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In a draft guidance issued this week, NICE (London, UK) has recommended against the routine NHS use of Biogen’s (MA, USA) spinal muscular atrophy (SMA) therapy, Spinraza® (nusinersen), due to uncertainties over its long-term effectiveness and its extremely high cost.

Individuals with the most severe forms of SMA usually die before the age of 2. There are currently no active treatments targeting the underlying cause of SMA and thus, the condition is managed through supportive care that aims to minimize the impact of disability, address complications and improve quality of life.

The committee concluded that, based on its list price, the cost–effectiveness estimates for nusinersen ranged between £400,000 and £600,000 per year of quality adjusted life gained. NICE have stated that even with a proposed confidential price reduction, the cost of nusinersen is too high for it to be considered a cost-effective use of NHS resources.

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