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Brief Report from the All Party Parliamentary Group that focused on nusinersen

21 July 2017

Held on 18th July, representatives from NHS England, Biogen, Great Ormond Street Hospital and leading neuromuscular consultants shared their views in a Q&A discussion. This covered the current provision of nusinersen for infants with SMA Type 1 through the Expanded Access Programme (EAP) and what progress there has been towards the possibility of wider access.

The APPG is a group of MPs and Peers in the Houses of Parliament who raise awareness of all types of muscle-wasting conditions and push for political reforms. The group is chaired by Mary Glindon, MP and meets throughout the year in order to encourage parliament, charities, scientists, health professionals and decision-makers to work together. Muscular Dystrophy UK provides the group with organisational support and makes sure that individuals and families affected by muscle-wasting conditions are also involved and steering the group in the right direction.

SMA Support UK, The SMA Trust and Treat SMA were all at this meeting to support individuals and families affected by SMA to share their testimonies. We are very grateful to the people who travelled long distances and were prepared to tell the group about the impact SMA has had on them or their child and family and what access to treatment would mean for them. They made a powerful impression on MPs, Peers and everyone there

The main news from the Q&As included:

  • NHS England is now considering the clinical evidence from the European Medicines Agency (EMA) Assessment Report  and funding implications to see if there is any possibility of their supporting the EAP for eligible children with SMA Type 1 until such time as it is closed by Biogen
  • Biogen has given the green light to any centre that has applied to them to be part of the EAP
  • There are now some 25 children receiving treatment, with another 20 who wish to take part waiting to hear what their position is
  • A referral from the Health Minister for NICE to begin to assess nusinersen’s longer term / wider availability is expected in the next few weeks

SMA Support UK passed on information from families who are waiting to access the EAP to NHS England and clinicians.

We continue to write letters and ask questions in support of an individual child’s access when asked to do so. As soon as we hear from NICE, we will work with individuals and families affected by 5q SMA (SMA Types 1, 2 and 3) who want access to the treatment to ensure their voices are heard. We continue to work with the other charities and TreatSMA.

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