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NICE finally starts review of Biogen’s Spinraza for SMA

Will evaluate the treatment based on its Single Technology Appraisal process

Biogen

The National Institute for Health and Care Excellence (NICE) has started its appraisal of Biogen’s Spinraza drug for spinal muscular atrophy (SMA), ending a “frustrating delay”, according to the SMA Trust.

Spinraza (nusinersen) became the first drug to be approved for the often-fatal genetic disease, which affects muscle strength and movement, last May but its review by NICE has been delayed by deliberations over the best way to move forward with the pricey drug, which has a UK list price of more than £80,000 per dose.

Rather than gauging the cost-effectiveness of Spinraza via the Highly Specialised Technology (HST) route typically used for high cost therapies aimed at small patient populations, NICE is instead looking at the treatment via its Single Technology Appraisal (STA) route.

This is designed to assess drugs for both clinical and cost-effectiveness usually used for more common conditions and according to the Trust, the process has the advantage of broadening the number of patients able to receive the drug as the HST route would mean that only patients with type 1 (infantile-onset) SMA would be eligible.

However, it adds: “there is concern that this route is not set up to assess rare disease drugs, like Spinraza.”

In trials, Spinraza has been show to improve survival in infants with SMA and reduce the chances that they go on to need mechanical ventilation.

In a statement, Biogen points out that “on its own this choice of route could have signalled a setback for access, the openness demonstrated by NICE and NHS England to commence development of a Managed Access Agreement straight away has reassured the company that there is still a viable, long-term reimbursement path for this significant therapeutic advancement in England”.

The charity says this swift move towards MAA negotiations is ‘promising’, and has called on supporters to lobby MPs to push for an agreement on access as soon as possible.

Biogen notes that Spinraza is reimbursed in Italy and Sweden, with ongoing negotiations taking place across Europe including Scotland where it is under active appraisal by the Scottish Medicines Consortium (SMC).

At the moment eligible children in the UK have been treated with the drug free of charge under a deal between the pharma company and the NHS, but the SMA Trust says that while this programme will continue for patients already on the drug it may be closed to newly-diagnosed children.

Biogen intends to submit an MAA proposal to NICE in March, and the agency is due to make initial recommendations in July, at which time a public consultation period will get underway with a definitive judgment expected around November.

Article by
Phil Taylor

22nd January 2018

From: Regulatory

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