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Last updated 11th May 2018.

Access in England, Wales and Northern Ireland – pushing for progress

On 18th January 2018, the National Institute for Health and Care Excellence (NICE) started its appraisal of Spinraza for the treatment of all types of SMA via the Single Technology Appraisal (STA) route. SMA Support UK, MDUK, the SMA Trust and TreatSMA were all invited to make Patient Group submissions by 15th March 2018. Thank you to those of you who responded to our surveys that informed our submissions. You can read the survey results here and a copy of our submission here.

Patient Experts were nominated by the patient support and advocacy groups. From these, NICE selects representatives who will take part in their first committee meeting on June 27th 2018

NICE advise they will report on their findings in November 2018. 

The STA route is designed to assess drugs for both clinical- and cost-effectiveness and is usually used for more common conditions. We are concerned that this route is not set up to assess rare disease drugs, like nusinersen. However, there is some promising news. Biogen, has announced that they are in discussions with NICE and NHS England to put an interim access scheme in place. This scheme, known as a Managed Access Agreement (MAA), could give others with SMA access to the treatment while further data and evidence are gathered over several years.

Time is key and it is crucial nusinersen is not subjected to any delays during the NICE assessment process. We are working with MDUK, the SMA Trust and TreatSMA to push for the MAA to be implemented as soon as possible. Find out how you can help by writing to your MP here.

We would expect guidance produced by NICE to be followed in Wales and Northern Ireland but they could follow Scotland.

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.

The SMA Type 1 Expanded Access Programme (EAP) - voicing our concerns

There are now some 66 children receiving treatment via the UK EAP.

After a considerable amount of advocacy work by clinicians and patient groups, including SMA Support UK, NHS England announced an initial interim policy in August 2017 which, following further advocacy, led by Professor Francesco Muntoni, was updated in March 2018. This programme can now be accessed by any new children diagnosed with SMA Type 1 at 7 months of age and under. You can read more about this here 

We are very aware that it is not uncommon for a family to experience delays in diagnosis, how distressing this is and how unfair this age limit on access would be for any family in this position. MDUK has a training programme on SMA for GPs. We also try to raise awareness of SMA within the health community. We need to push for more to be done so that children are diagnosed in a timely way. 

Biogen has said it will continue to review the future of the SMA Type 1 EAP and that its decision will depend on what progress there has been with NICE's appraisal of the treatment. We will continue to urge Biogen to keep the programme open for new children until such time as treatment of children with SMA Type 1 can be fully funded by the NHS. We hope the progress with NICE’s appraisal, will ensure they do. 

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.