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WHAT WE ARE DOING TO PROGRESS ACCESS TO THE TREATMENT IN THE UK

Last updated 18th October 2017

The SMA Type 1 Expanded Access Programme (EAP)

There are now some 60 children receiving treatment via the UK EAP.

The interim NHS England policy (4th August 2017) will fund the hospital costs of administering this programme until NICE makes a decision on NHS funding. However it has limited which children with SMA Type 1 may have access, to those diagnosed by 7 months of age and with two SMN2 copies. We joined with clinicians and the other charities and advocacy groups to appeal these limits and with them have written to NHS England and submitted a Preliminary Policy Proposition (see NHSE letter)  about expanding the SMA Type 1 EAP criteria . 

We are concerned that Biogen has said it will review the future of the SMA Type 1 EAP at the end of this calendar year and that its decision will depend on what progress there has been with NICE's appraisal of the treatment. We continue to do all we can to find out why this is so delayed and will urge Biogen to keep the programme open.

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 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. We have asked families to send us their personal stories of delayed diagnosis with permission to forward them to NHS England as a first step and will be working further on this in the next few months.

Long term access for those with SMA Types 1, 2 or 3

We were advised that the likely NICE appraisal path is as a Highly Specialised Technology (HST). We are concerned that the new HST criteria introduced on 1st April 2017 threaten the possibility of nusinersen eventually being funded by the NHS in England and the long-term potential of this new treatment being available for those affected by SMA (for more information, see ‘Drug Funding and Approvals Process - changes to HST criteria’).

We:

  • continue to try to find out why there is no announcement from NICE - we had expected to hear in July
  • have asked MDUK as part of their Fasttrack campaign to highlight nusinersen in national media as a strong example of what is wrong with the system. 
  • 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
  • are urging Biogento keep the SMA Type 1 EAP open until there is a final decisison about long term provision by the NHS
  • are in close contact with TreatSMA - working in our different ways to maintain pressure and give voice to the SMA Community.