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NICE Announces Appraisal Route for Nusinersen

19 January 2018

On 18th January 2018, the National Institute for Health and Care Excellence (NICE) started its appraisal of nusinersen for the treatment of all types of SMA.

NICE is now looking at the treatment via the Single Technology Appraisal (STA) route. SMA Support UK, Musculary Dystrophy UK, the SMA Trust and TreatSMA have been invited to make Patient Group submissions by 15th March 2018.

The STA route is designed to assess drugs for both clinical and cost-effectiveness and is usually used for more common conditions. As such, we are concerned that this route is not set up to assess rare disease drugs, like nusinersen. You can read about the NICE process here and why there is concern, here.      

However, there is some promising news. The pharmaceutical company, 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 more people 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 have worked with Muscular Dystrophy UK and the other charities to prepare a template letter that you can use to write to your MP. You can download the letter here and find your MP’s address here. Please, do send us a copy if you do write.

We would expect guidance produced by NICE to be followed in Wales and Northern Ireland.