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Published on 19 January 2018

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Optimism for rapid and broad patient access for SMA treatment following NICE update

The National Institute for Health and Care Excellence (NICE) has formally invited Biogen to submit Spinraza (nusinersen), the first and only disease-modifying treatment for 5q spinal muscular atrophy (SMA), for assessment via the Single Technology Appraisal (STA) route.

 

The National Institute for Health and Care Excellence (NICE) has formally invited Biogen to submit Spinraza (nusinersen), the first and only disease-modifying treatment for 5q spinal muscular atrophy (SMA), for assessment via the Single Technology Appraisal (STA) route.

 

Whilst 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.

Terry O’Regan, Vice President and Managing Director of Biogen UK and Ireland, comments, “We now have an opportunity for SMA patients in England & Wales who deserve access to nusinersen, a medicine which has the potential to address unmet medical need in a devastating disease. Whilst we have been disappointed by the delays in the NICE process so far, we have been encouraged by a recent meeting with NICE and NHS England, where the need to respond to specific STA and access challenges in appraising nusinersen was expressly acknowledged, and an immediate plan for a Managed Access Agreement that can work alongside the STA process was agreed. Now is the time for all stakeholders to work together to ensure as many patients as possible get rapid access to this life saving medicine.”

Nusinersen received European marketing authorisation following accelerated assessment in May 2017, and since that time NICE has been reviewing the best route for the appraisal. So far, nusinersen is reimbursed in Italy and Sweden with ongoing negotiations taking place across Europe.

 

Due to the significant clinical improvements shown with nusinersen in clinical trials and the urgent need for a treatment where before there was none, Biogen opened one of the largest global expanded access programmes in rare disease (since Autumn 2016) as an interim measure for those with infantile-onset SMA (consistent with type 1). To date, all eligible children in the UK have been treated with nusinersen following a Biogen / NHS collaboration in which it was agreed that Biogen would provide the medicine free of charge, and the NHS would devise an urgent policy to administer the treatment.
What happens now?
Biogen will submit to NICE in March 2018 (in line with the submission request date), and the NICE appraisal committee will outline their initial recommendation in June 2018 – at this point, feedback will be invited from relevant stakeholders including clinicians and patient representatives. NICE then provides its recommendation on whether routine funding can be made available to patients in England via the NHS, currently scheduled for November 2018. The STA will consider nusinersen for all appropriate patients who may benefit, which extends beyond those affected by infantile-onset SMA (consistent with type 1).

 

The Biogen expanded access programme remains open to all eligible children at the current time, however cannot be a replacement for health services, and urgent funding is needed in order to make nusinersen available long-term to the whole patient population who may benefit.



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