Lead clinicians, centre service managers and SMA Support UK attended a meeting on 6th June on the expanded access programme (EAP) for nusinersen. Due to the election ‘purdah’ and the NHS England’s (NHSE) communications approval process, it has not been possible to report on the outcome of this meeting until the release of the meeting notes, which are now available.
A lot of issues, which families are all too familiar with, were raised and actions were planned. These 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, 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.
The work relating to the establishment of clinical prioritisation criteria for access to the programme is on-going. This is still in draft form and is 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 continental Europe who will need access in the UK as soon as this option closes.
SMA Support UK is also trying to collect information about all children waiting to access the UK EAP, so that the national picture can be discussed with clinicians and NHS funding bodies. If you are in this position, you will find more details and a form to complete here (by Friday 14th July).
SMA Support UK, Muscular Dystrophy UK, the SMA Trust and Treat SMA UK continue to work to bring together NHSE, NHS Trusts and Biogen to progress access. Let’s keep the pressure up.