NICE Turns Down Spinal Muscular Atrophy Drug Spinraza

Nicky Broyd

August 14, 2018

The National Institute for Health and Care Excellence (NICE) has rejected for NHS funding in England a new drug for spinal muscular atrophy (SMA).

New draft guidance found there were "substantial benefits" of treatment with nusinersen (Spinraza, Biogen) but there were also "significant uncertainties" around its long-term use. It also said its cost to the NHS was too high to be considered cost-effective.

Motor Neurone Degeneration

Current SMA treatments do not address the underlying cause of the rare genetic disorder. Nusinersen is the first treatment that helps to tackle motor neurone degeneration in SMA.

For those receiving the treatment, drug injections directly into the cerebral spinal fluid would be carried out for life. After the first injection, follow up infusions are given after 14, 28 and 63 days, then with a maintenance dose every 4 months.

NICE says there are estimated to be between 1200 and 2500 adults and children and adults in the UK with SMA.

Cost estimates ranged from £400,000 and £600,000 per quality adjusted life year (QALY) gained. Even with an undisclosed NHS discount, the NICE assessment committee did not find the drug to be cost-effective.

Paucity of Evidence

Meindert Boysen, director of the centre for health technology evaluation at NICE, said: "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.

"Nusinersen is a promising treatment that has been shown to improve a range of outcomes important to patients. 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."

This could result in a managed access arrangement (MAA) under which patients could access the treatment as data is collected on its effectiveness. However, the NICE committee said that "the details of the company’s proposed MAA were vague and currently insufficient for it to assess whether it could be an option".

The Scottish Medicines Consortium (SMC), recommended the drug for restricted use in symptomatic type 1 SMA (infantile onset) patients and it is due for reconsideration under Scotland's 'ultra-orphan treatment' scheme.


Commenting on the NICE draft guidance, Muscular Dystrophy UK Chief Executive, Robert Meadowcroft, said: "Spinal muscular atrophy can be devastating and today’s news will be heartbreaking 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. The one glimmer of hope is for a temporary scheme that ensures access. 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."

The decision by NICE is not definite and its recommendations may change. It is continuing to consult and take comments on its draft guidance until 5th September. It will then review any feedback before making a final decision. 


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