Action for Access for All
(Aug 2019 – May 2020)

Last updated 4th May 2021

You can read about all the advocacy work by patient groups and clinicians between Autumn 2016 and July 2019, here.

All the patient groups and clinicians have representatives on the Managed Access Agreement Oversight Committee which monitors the progress of this Agreement in England. It meets twice a year.

Adults and children who have Type 3, had lost their walking ability and were ineligible for access in England

In January – February 2020, patient groups conducted a survey of those who have SMA Type 3 who were ineligible for treatment and the impact of this exclusion on them. The results were collated by SMA UK, presented to the Managed Access Agreement Oversight Committee and forwarded to Biogen. We heard directly from individuals / from parents of 37 children / adults who have no access to treatment. You can read the results here

As members of SMA Europe we were able to talk with other member countries where those who have SMA Type 3 and have lost their walking ability do have access and gather testimonies about the impact of treatment for them. We forwarded this evidence to NHS England/ NICE and Biogen. You can read where access was up to in these countries in June 2020 and these testimonies, here.

In October 2020, NICE announced their promised MAA review of this eligibility rule. You can read about the process that was followed, here. SMA UK made a submission to Biogen to be included in the evidence review. During the review period, interim arrangements were set up for children with SMA Type 3 who lost ambulation in the 12 months prior to 28th July 2019 who had been receiving nusinersen.

SMA UK took part in this review as a member of the MAA Oversight Committee. On 4th May 2021 NICE announced a positive decision to change this rule

Children who have Type 3 in England who have access but may have faced stopping criteria

In February 2020, SMA UK conducted a first survey via the SMA REACH clinical network  for parent(s) / guardian(s) of any child / young person age 19 years and under who has SMA Type 3 and had been accepted for nusinersen treatment. We wanted to give parents and children the opportunity to report on outcomes they were seeing with treatment and find out what impact the stopping criteria that applied to children who lost their walking ability after 28th July 2018 was having on parents and children. We conducted follow-up surveys with respondents every 3 months, collated results and made these available to these children’s clinicians, NICE, NHS England and Biogen. We continued to do this and to advocate for the removal of the stopping criteria. We were able to submit the reports from two families affected by the stopping criteria as part of our submission to the NICE evidence review.

Access for adults

  • In general, across the UK

To help with planning the roll-out of the nusinersen treatment programme across the UK, between Feb and March 2020, patient groups conducted a survey for all adults who have 5q SMA Type 1, 2 or 3  to find out who does / doesn’t want access to nusinersen treatment. SMA UK collated the results. 145 people replied. We sent an overall summary to the adult services network, NICE, and NHS England. Due to Covid-19, programme roll out was significantly delayed at most Centres, but we were able to liaise with many about who was waiting and get back to respondents with any updates and advice about what they could expect next.

  • Who have Type 3 and had lost their walking ability since 28th July 2019

These adults would have been eligible for treatment on this date. SMA UK has taken up individual cases and the case in general to argue that in view of the delays in setting up adult services and the impact of Covid -19 that has further increased delays, any adult in this position should still be eligible for treatment

Access for Children across the UK

Between Nov and Dec 2019, patient groups conducted a survey about what progress there had been with children’s access to treatment. SMA UK collated the results and worked with families, NHS England and Centres where any family had told us they were having problems with access. Since then, children’s services have rolled out the treatment programme across the country and all treatment Centres are now assessing and treating children.

We continue to work to achieve access for all.