We have received a statement from Biogen UK on the recent decision of the Scottish Medicines Consortium. We are posting it below.
Biogen statement to SMA community re: SMC routine funding recommendation
Today, the Scottish Medicines Consortium (SMC) announced the recommendation of the routine funding of nusinersen® for the treatment of symptomatic type 1 (infantile onset) 5q spinal muscular atrophy (SMA).
The acceptance comes after the SMC reviewed the evidence from clinical trials, as well as heard testimonials from patients, patient groups and clinicians at a recent committee meeting. These testimonials provided information relating to the impact of SMA on patients, their quality of life, and the wider community, as well as current disease intervention options and the potential benefits of nusinersen. The recommendation means that anyone diagnosed with type 1 SMA can now be considered for treatment with nusinersen via NHS Scotland. The recommendation of routine funding does not currently include later onset SMA (types 2 and 3).
The SMC took just four months to review the evidence and make their decision, which involved examining the clinical trial evidence of nusinersen including data that demonstrate significant improvement in the disease across types 1, 2 and 3 SMA,¹ with motor function improvements from the start of treatment such as independent sitting, standing and walking, as well as improvements in breathing and survival among those most severely affected.1
Biogen is extremely proud to bring nusinersen to type 1 patients in Scotland and very pleased that the SMC has acted quickly. This will make a momentous difference to the lives of those who previously had no other options. However, our job is not done and we continue to actively collaborate with patients, clinicians and government bodies to find a way to make this important treatment available to all who may benefit.
What happens now – type 1 SMA?
Patients with type 1 SMA are already receiving treatment via the Biogen expanded access programme (EAP), which provides infantile onset SMA patients (consistent with type 1) with nusinersen, free of charge. Now that the SMC has recommended nusinersen for these patients, the EAP will close in Scotland and they will transition to treatment provided by NHS Scotland.
What happens now – later onset SMA (types 2 and 3)?
Biogen is in active discussions with the SMC to resubmit nusinersen for later onset SMA (types 2 and 3) and urges the initiation of a reformed appraisal process for ultra-orphan medicines as soon as possible to allow a greater degree of flexibility for the assessment of such treatment options. These reforms were proposed to the Scottish Government in 2016 following a review of the system and the government confirmed that they would take forward the recommendations. Biogen hopes this new, more appropriate process will lead to a positive outcome and we remain steadfast in our commitment to making nusinersen available to all who may benefit.
In the meantime, applications for patients with later onset SMA to receive nusinersen are being made on a case-by-case basis by treating hospitals in Scotland.
What is the current status across the rest of the UK?
Biogen has provided all eligible (type 1) children in the UK with nusinersen free of charge via the EAP. Appraisals of nusinersen remain in progress in England, Wales and Northern Ireland, with decisions expected later in 2018, and Biogen is working closely with the relevant authorities to expedite the timelines where possible to bring the treatment to patients without delay.
¹ Nusinersen Summary of Product Characteristics (SmPC). November 2017.
