There has been further activity over the last week to push for access to the first drug for people with Spinal Muscular Atrophy (SMA); Spinraza. Below is a summary of the latest news in the fight for access to this life-changing treatment.
This week, Wednesday 6 March, we were in Manchester along with SMA UK, TreatSMA, clinicians and families for the third NICE committee meeting on Spinraza. We are unable to share the details of the discussions that took place at this stage but assure you that the patient voice was well represented by those present. We expect to hear the outcome of the meeting in the next few weeks and will share any news as soon as we are able to.
In more positive news, it was announced last month that Spinraza is due to be made available on the NHS in Scotland for children and adults with SMA Types 2 and 3. Subject to final agreement, people will be able to receive treatment from April 2019, news which has been welcomed by families in Scotland. It was approved through the new ultra-orphan pathway assessment process for the rarest conditions, which was set up in October 2018 by the Scottish Government and follows the approval for people with SMA Type 1 to access treatment on the NHS in Scotland from May 2018.
Prof Alison Strath, Principal Pharmaceutical Officer at the Scottish Government, provided an update at the Cross Party Group in the Scottish Parliament on 27 February on the plans for making Spinraza available. We look forward to hearing more news in due course.
Alongside Mary Glindon MP and leading clinicians Prof Muntoni and Dr Manzur, we met with Health Minister Baroness Blackwood on 5 March to discuss access to Spinraza and reforming NICE’s appraisal processes for future rare disease treatments. The Minister committed to working closely with Muscular Dystrophy UK when NICE’s appraisal routes are reviewed to learn from the charity’s current and past experiences.
The campaign for access to Spinraza has been featured in national, broadcast and regional media. This wouldn’t have been possible without the support of families across the UK, who have shared their personal stories with us.
Some of the key coverage is listed below:
- An open letter was published by The Guardian from 30 clinicians involved in the care of children and adults with SMA. The letter highlighted clinicians’ frustration at an effective treatment being out of reach. The signatories – including consultants, physiotherapists and nurses – also joined Muscular Dystrophy UK and SMA UK in asking NICE to show flexibility.
- BBC News and ITV News covered the story online, sharing UK families’ stories and a quote from the charity. ITV News also covered the story on its national lunchtime, evening and 10pm bulletins.
- The Mirror Online spoke to families for a series of pieces highlighting the campaign for access. This included interviews with Paul Mercer, whose daughter, Kennedy, has SMA Type 1 and receives Spinraza; Shakil Malji, who launched a petition after his daughter was diagnosed with SMA Type 1; and Katie Prescott, whose daughter, Heidi, has SMA Type 3.
- STV News and The Scotsman covered the announcement that Scotland is due to approve Spinraza for use on the NHS for people with SMA Types 2 and 3.
- Metro Online ran a first-person opinion piece from mum Rachel Jamieson, whose daughter, Matilda, has SMA Type 3, to coincide with Rare Disease Day.
- The Sun and Mail Online featured Shakeel Khan’s plea for the treatment for his six-month-old son, Haris. Haris was diagnosed with SMA Type 1 last month – after Biogen closed its compassionate scheme.
- ITV London, BBC East Midlands Today, BBC Look North, ITV Calendar, ITV Granada, BBC Radio York and BBC Leicester have also interviewed families and Muscular Dystrophy UK spokespeople.
We would like to thank all the individuals and families who have been campaigning so tirelessly for access to Spinraza; writing letters, attending meetings and generally making your voices heard. We will keep fighting alongside you to make this life-changing treatment available.
Have you got a story to share?
If you would like to share your story, please email Laura Burge at: firstname.lastname@example.org