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Biogen Announces November Closure of the Expanded Access Programme for Infants Newly Diagnosed with SMA Type 1

23 August 2018

You can read about Biogen’s distressing decision in their community update.

The Expanded Access Programme (EAP) was set up by Biogen and was designed as a short-term solution for access to nusinersen for eligible children with SMA Type 1. The programme will stay open in England, Wales and Northern Ireland until 1st November 2018 for any child with SMA Type 1 where both the child’s medical team and the child’s parents / guardians have agreed that it could be of potential benefit and that the child is eligible for the treatment.

Children with SMA Type 1 in Scotland are now able to access NHS funded treatment there. They are not affected by this decision.

Eligible children

All children diagnosed by age 7 months with SMA Type 1 are eligible for access via the EAP if the family and clinical team agree they may potentially benefit. Children with SMA Type 1 who were diagnosed before 4th August 2017, are older than this and are not already on the EAP, remain eligible for treatment up until 1st November if their medical team agrees it could potentially be beneficial. 

Anyone enrolled in the programme by 1st November will be assured of continued treatment unless the family and their clinician consider it appropriate to stop.

Continued Advocacy

We will continue to focus our advocacy for access to treatment on both:

  • the urgent need for NICE, NHS England and Biogen to agree the terms of a Managed Access Agreement (MAA) to widen and replace this EAP ahead of 1st November, and
     
  • the need for a long-term sustainable plan for wider access.

How you can help

You can add your voice by responding to NICE’s consultation paper either directly by registering here or via one of the charities / campaign groups. Please help spread the word.

If anyone would like to speak to someone for support during this difficult time, please do contact our Support Services Team.