NHS England funds additional costs of SMA drug

Published Date
Peter Sutton

NHS England has announced today that it will fund the hospital costs to administer Spinraza for patients with spinal muscular atrophy Type 1 on Biogen’s Expanded Access Programme (EAP).

The drug company, Biogen, made the drug available for free for SMA Type 1 patients through the EAP. However, due to lack of capacity and a funding pathway at some hospitals a number of eligible patients remain untreated.

The news today is a significant step forward – and means that previously untreated infants should have the remaining treatment costs funded at their hospital.

The announcement comes after pressure from families, MDUK and fellow patient groups.  MPs and Peers have also helped keep the issue in the spotlight – including at a recent meeting of the All Party Parliamentary Group for Muscular Dystrophy, where NHS England’s Director of Specialised Commissioning, James Palmer, was pressed on the need for NHS England to provide support to SMA Type 1 patients needing access to the EAP.

The policy will remain in place until either Biogen closes the EAP to new patients – or a final decision is taken by NICE on the drug’s availability on the NHS.

NICE is yet to announce whether it will carry out an evaluation of the drug – which has been licensed by the European Medicines Agency for SMA Types 1, 2 and 3. An announcement on this is expected shortly.

You can read NHS England’s policy on the Type 1 EAP here – which includes more details on starting and stopping criteria.

Kerry Storr, whose two-year-old son William has SMA Type 1, said:

We’re absolutely delighted that this has been taken seriously and that such great progress is now being made. Thanks to the support of Muscular Dystrophy UK and fellow charities, William and other SMA Type 1 babies in Sheffield are now due to receive their first dose of Spinraza this month, which is what we have been hoping for throughout this campaign.

Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy Uk said: 

Muscular Dystrophy UK is delighted with the news today from NHS England that it will fund hospital costs to administer Spinraza for patients with spinal muscular atrophy Type 1 on Biogen’s Expanded Access Programme (EAP). This means that infants with Type 1 SMA – who had faced an agonising wait for treatment – should now be able to access the drug.

SMA Type 1 is the most severe form of SMA and approximately 50% of children diagnosed with childhood onset SMA have SMA Type 1. Although it is not possible to predict accurately life expectancy for babies diagnosed at a very early age, without intervention this is usually less than two years, so today’s news is extremely welcome.

The decision is a tribute to the families who have campaigned so hard to get to this stage. But future treatment depends on NICE and the other authorities in the devolved nations recommending that the NHS fund the drug in England, Scotland, Wales and Northern Ireland.

For more information, please contact Peter Sutton on p.sutton@musculardystrophyuk.org or call 020 7803 4838.

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