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Update from NHS England’s June 6th meeting about the SMA Type 1 EAP

05 July 2017

Lead clinicians and centre service managers were called together. SMA Support UK was also present. Due to the election ‘purdah’ and the NHS England (NHSE) communications approval process, no one at the meeting has been able to discuss what went on until now with the release of the meeting notes

You will see that a lot of issues with which families are all too familiar were raised and actions were planned. These actions are now underway.

On 25th April, NHSE had not supported a proposal that they fund the additional costs of administering the EAP as they were not persuaded by the published evidence presented. This evidence only covered the interim results of the clinical trials. They stated then that they required peer review publication of results before they would re-consider their position.

Importantly, you will see that it was reported at the meeting that James Palmer (Specialised Services Medical Director) had indicated that when the European Public Assessment Report (EPR) became available, a further submission for funding the additional costs would be made to the NHSE Clinical Panel. This suggests the peer review requirement may no longer be necessary. The EPR was released on 26th June. You can read this here.

You will also see that work is going on to establish clinical prioritisation criteria for access to the programme. These are still in draft and are being circulated around the clinical group for further comment. They are not yet public but will be based on criteria that have been developed and are being used in other countries.

We continue to make every effort to work with clinicians and NHSE to progress access to the EAP. We continue to support any family trying to access the EAP in the UK by writing on their behalf to the CEOs of NHS Trusts and NHSE. This includes any families currently travelling to Europe who will need access in the UK as soon as this option closes.

We are also trying to collect information about all children waiting to access the UK EAP, so that we can discuss the national picture with clinicians and NHS funding bodies. If you are in this position, you will find more details and a form to complete hereIf you wish to complete this form, we need to hear from you by Friday 14th July.

If you need any support, please do contact us on 01789 267520 or email

SMA Support UK, Muscular Dystrophy UK, the SMA Trust and TreatSMA continue to work to bring together NHSE, NHS Trusts and Biogen to progress access. Let’s keep the pressure up. See how you can help here.