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Access to Nusinersen Update & How You Can Help

24 August 2018

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Dear SMA Community,

You will no doubt have heard the devastating news in NICE’s consultation paper that at this stage, they are not recommending nusinersen for funding by the NHS (more on this below). Added to this was the further distressing announcement this week from Biogen that on 1st November 2018, it will be closing the Expanded Access Programme (EAP) for eligible infants newly diagnosed with SMA Type 1. Read more about this here.

A 'Managed Access Agreement'

The glimmer of hope is that NICE’s consultation paper encourages the possibility of a ‘Managed Access Agreement (MAA)’. This would be a 3 to 5-year scheme agreed between Biogen, NICE and NHS England that would enable access to treatment and the opportunity to collect further evidence of the treatments, clinical benefits, and impact on quality of life. These talks have already started, with Biogen committed to the aim of finalising a Managed Access Agreement (MAA) ahead of November 2018. They, and all the charities and campaign groups, are determined that this will offer wider access than the current EAP and be a step towards long-term sustainable access to all who wish to receive, and could potentially benefit, from treatment.

How You Can Help

Media Involvement

Our thanks to the many families who have been prepared to tell their stories to the media and increase public awareness of what is happening, and to Muscular Dystrophy UK and TreatSMA who have been organising this. If this is something you would be comfortable to do, please get in touch with either organisation directly or contact us.

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Tell NICE your views:

The other way that everyone can help us is by responding to NICE’s consultation paper with your views by 5th September. You can read the full consultation paper here.

NICE is interested in comments on the following:

  • Has all the relevant evidence been taken into account?
  • Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?
  • Are the provisional recommendations a sound and suitable basis for guidance to the NHS?
  • NICE also wants to know if the preliminary recommendations may need changing because they could have an adverse impact on people with a particular disability or disabilities

You may want to comment on all of these, but we have highlighted ones where you might (again) want to remind NICE of:

If you haven’t had access to nusinersen:

A. The evidence of your need:

  • The Type of SMA you / your child has and your / your child’s age?
  • The impact SMA has on your family lives / the lives of all who provide care and support. For example: sleep, hospital admissions, appointments, equipment, housing adaptations, work, finances, social opportunities, getting around, mental well-being.
  • How many people are involved in caring responsibilities (grandparents and others)

B. The adverse impact NICE’s decision not to recommend nusinersen for funding by the NHS will have on your / your child’s life – as someone with a particular disability (SMA):

  • Based on all you know from your medical team and others in the SMA community, how your / your child’s SMA will progress without treatment
  • The impact this will have on you / your child / family and all those who care for and support you / your child. For example: sleep, hospital admissions, appointments, equipment, housing adaptations, work, finances, social opportunities, getting around, mental well-being

If your child has had access to nusinersen:

A. The evidence of your need:

  • The Type of SMA your child has and your child’s age – when they started treatment and now
  • Based on what you know / were experiencing, what would have been the impact of your child’s SMA if not treated on your family lives / the lives of all who provide care and support. For example: sleep, hospital admissions, appointments, equipment, housing adaptations, work, finances, social opportunities, getting around, mental well-being
  • How many people are involved in caring responsibilities (grandparents and others)

B: The effectiveness of the treatment

  • How long your child has been receiving nusinersen and how many doses. The impact it has had on your child and all those who care for and support them – physical, mental, emotional, financial -  physical milestones, respiratory impact, sleep, hospital admissions & appointments, equipment, housing adaptations, work, finances, social opportunities, getting around

To send NICE your comments (you will need to either sign in to / create a NICE account first) please go here.

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Tell your MP about the importance of access to nusinersen
MP drop-in event on Tuesday 11 September

MDUK has been working closely with SMA Support UK, The SMA Trust and TreatSMA, and wants to give families the opportunity to talk to their MPs about the impact of SMA and the importance and urgency of having access to nusinersen (Spinraza).

They have organised an ‘MP drop-in event on access to Spinraza’ in Parliament to enable you to speak to your MP. They have booked Committee Room 17 in the House of Commons from 10.30am to 12pm on Tuesday 11 September, with the event hosted by Julian Sturdy MP and supported by the All Party Parliamentary Group for Muscular Dystrophy, chaired by Mary Glindon MP.

We know that many of you have already been in touch with your MP in recent weeks and months, and this is clearly having an impact as they are asking about how they can provide support.

Contacting your MP

Space is limited at the event so it is essential you book your place. To book, and for more information and help about contacting your MP, please get in touch with Jonathan Kingsley at MDUK: 
j.kingsley@musculardystrophyuk.org / 020 7803 4839.

MDUK will be inviting MPs to the ‘drop-in event’ on 11th September, but we need you to contact your MP to ask them to:

  • meet you at the event if you are attending, or
  • to come along on your behalf to find out more about access to Spinraza.

You can find your MP’s contact details on the Parliament website.

Following the event, and having heard from the families, we are hoping that MPs will be taking the opportunity to support their constituents by asking questions in Parliament, writing letters, and supporting the All Party Parliamentary Group for Muscular Dystrophy.

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National Screening Consultation

The National Screening Committee (NSC) has invited us to give comments and feedback on its June 2018 evidence review. Their current recommendation is ‘systematic population screening programme not recommended’. When we submit our response, we would like to represent views from the SMA Community about screening newborns for SMA. Let us know your thoughts in our survey by 27th August.

 

What We Are Doing

We are doing all we can to advocate for access to nusinersen for all and will continue to do so. What we do is not always visible, but be assured it is a huge focus for us every day. In addition, our day-to-day support of families and adults with a new diagnosis, or who face issues (such as battling for equipment or a care and support package) remains an absolute priority. You can find out about all our other information and support services, here.

Please do phone or email if you think we may be able to help you:
- Email: supportservices@smasupportuk.org.uk 
- Phone: 01789 267520