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What We Are Doing To Progress Access To The Treatment In The UK

Last updated 6th September 2018.

Access in England, Wales and Northern Ireland 

On 5th September we submitted our response to NICE’s consultation paper which had been released on 14th August with the devastating news that they had not recommended nusinersen for funding by the NHS. You can read our response in which we put forward further evidence to challenge this decision and in support of access to nusinersen for all, here. We know many of you will also have responded.

The paper encourages, and we have supported, the interim possibility of a Managed Access Agreement (MAA). This would be a 3 – 5-year scheme agreed between Biogen, NICE and NHS England that would enable access to treatment and the opportunity to collect further evidence of nusinersen’s clinical benefits and impact on quality of life. In our response we have urged that this should enable access for all with SMA Types 1, 2 or 3 where there is agreement between the family/adult and clinician that it is a clinically safe option. We have also urged that there is a seamless transition from Biogen’s Expanded Access Programme (EAP) for those with SMA Type 1 that closes on 1st November to a new agreement so that there is no delay in treatment of any infant.

We will continue to push for these outcomes when we take part in any further discussions between NICE, NHS England, Biogen clinicians and patient groups over the next weeks.

NICE will hold a second committee meeting on October 23rd when it will review all the consultation responses. We will be present at this meeting. We continue to push for an announcement of final decisions by the originally announced date of 21st November 2018.

You can read what we have done previously to advocate for access for all and how the NICE appraisal process works here and about the EAP and our advocacy work for this here

Access in Scotland - our submission

The Scottish Medicines Consortium (SMC) has made its decision. You can find out more about it and what we are now doing here.

Access to new treatments in general – working to change the system

We are supporting MDUK’s efforts to highlight nusinersen in national media as a strong example of what is wrong with the system. See their Fasttrack campaign.‚Äč

We have committed to support Genetic Alliance UK, which has been challenged by the All Party Parliamentary Group (APPG) on Rare, Genetic and Undiagnosed Conditions, to work with a range of stakeholder groups to propose a method of making decisions about rare disease medicines that is ambitious, effective, transparent and fair. Read more here.