[NAME] MP
House of Commons
London
SW1A 0AA

Dear [NAME]

Re: Access to spinal muscular atrophy treatment called Spinraza

I am writing to you regarding access to the new spinal muscular atrophy (SMA) treatment called Spinraza.

Spinraza is the first and only treatment for patients with the rare inherited muscle-wasting condition spinal muscular atrophy (SMA). There are up to 1,300 children and adults living with SMA in the UK. For the most severe cases, life expectancy is only two years without access to the drug.

[You could include some more personal details here, for example the impact of spinal muscular atrophy on you or a family member, and the effect of any delays on you and your family]

Thus far in the UK, Spinraza has only been made available through a ‘compassionate use’ Expanded Access Programme (EAP) funded by Biogen and NHS England for those affected by SMA Type 1. This EAP is now restricted to infants under age 7 months. Biogen has also advised that although they will continue to support children currently receiving the treatment, they may close the programme to those newly diagnosed.

On 1st June 2017, Spinraza was granted a licence by the European Medicines Agency for treatment of those with 5q SMA, which includes those with SMA Types 1, 2 and 3.

We have now heard the news that Spinraza will be assessed through what is known as NICE’s Single Technology Appraisal (STA) route, which is a process designed for assessing treatments for more common conditions. Treatments such as Spinraza, that prolong the survival of people with severely debilitating diseases, face challenges demonstrating cost-effectiveness via the STA route. This is due to the ongoing costs of additional supportive care and resources, which increases costs. The pricing caps (cost of the drug per person) for an STA treatment are too low for Spinraza to have any hope of being recommended for NHS funding.

We are aware that it will take until November 2018 for NICE to conclude its STA appraisal of nusinersen and that it is almost 100% likely that we will then hear that it cannot be recommended.

However, there is some promising news. Biogen have announced that they are in discussions with NICE and NHS England to put an interim access scheme in place. Known as a Managed Access Agreement (MAA), the scheme could see more people with SMA able to access the life-changing treatment while further data and evidence are gathered over several years.

I would therefore greatly appreciate your support in writing to NICE and NHS England on the following key issues.

For NICE:

  • Urging the STA process to be conducted without any delays

For NHS England:

  • Urging them to conclude a Managed Access Agreement with Biogen as soon as possible to make Spinraza more widely available than to only those with SMA Type 1

For more information and any queries about contacting NICE and NHS England, please contact Clare Lucas at Muscular Dystrophy UK at or call 020 7803 4838.

Thank you for your support and I look forward to hearing from you shortly.

Yours sincerely,

[YOUR NAME]