NICE issues draft guidance against Spinraza® for spinal muscular atrophy

Written by Sharon Salt, Editor

In a draft guidance issued this week, NICE (London, UK) has recommended against the routine NHS use of Biogen’s (MA, USA) spinal muscular atrophy (SMA) therapy, Spinraza®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.

However, NICE are hopeful that further negotiations can be held between themselves, the company and NHS England, and welcome further discussions about access to the drug.

Nusinersen gained European approval last year for the treatment of 5q SMA, making it the first therapy authorized in the region for the condition. Additionally, the drug was previously cleared in the USA, where Biogen set the list price at USD$750,000 for the first year of treatment and USD$375,000 for each additional year of therapy.

Meindert Boysen, Director of the Center for Health Technology Evaluation at NICE, commented: “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 nusinersen meant it could not recommend the drug as a cost-effective use of NHS resources.”

“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,” concluded Boysen.

The draft guidance is open for public consultation until 11 September.

TreatSMA (Royal Tunbridge Wells, UK) have issued a statement mentioning that NICE has left open a possibility for the NHS to provide nusinersen under a separate agreement with the manufacturer – a so-called Managed Access Agreement. They expect Biogen to propose a Managed Access Agreement to the NHS within the next weeks.


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