We’re continuing our SMA Awareness month campaign “Every Piece of the Puzzle” with a look into the journey of securing access to Spinraza®.
The introduction of the first disease-modifying treatment for spinal muscular atrophy (SMA) marked a turning point for the SMA community, but access did not come easily.
From 2016 onwards, patient organisations worked together, alongside families, clinicians and Biogen to secure access to nusinersen (Spinraza®) through the Expanded Access Programme (EAP), ensuring that the voices of people affected by SMA were heard throughout the process.
Fighting for access through the Expanded Access Programme (EAP)
In 2016, regular discussions with Biogen began, to advocate for UK access to nusinersen. As the EAP was developed, Great Ormond Street Hospital (GOSH) and Newcastle were identified as the first potential treatment centres due to their experience delivering nusinersen clinical trials.
From the beginning, it was clear that demand would be urgent. Families of babies diagnosed with SMA Type 1 were facing a race against time, and clinicians and patient organisations worked together to establish fair and ethical approaches to prioritising access. Families who shared their personal stories had a huge impact on this work.
Through the beginning of 2017, work continued with clinicians, NHS England, Biogen and other advocacy groups, including the newly formed Treat SMA, to address the challenges preventing children from accessing treatment. With the Expanded Access Programme running successfully across Europe, families living with SMA were travelling to France to access treatment for their children, an unsustainable solution that was impossible for many. Although Biogen approved additional hospitals to participate in the EAP, each centre needed local NHS support, trained staff and resources to deliver treatment safely. Limited capacity at specialist hospitals meant that many families faced delays.
SMA UK and Treat SMA supported families seeking access, helped gather information about children waiting for treatment and advocated for a sustainable national approach, including discussions about specialist “hub” centres to expand capacity.
Challenging barriers and securing progress
In spring 2017, patient organisations joined clinicians in urgent discussions with NHS England about the barriers facing the EAP. While NHS England recognised the urgency for families, funding arrangements for the additional costs of delivering treatment had not yet been resolved.
The SMA community highlighted that the needs of children with SMA Type 1 required urgent action, particularly as nusinersen had already been approved by major regulators including the European Medicines Agency and the US Food and Drug Administration.
Throughout 2017, SMA organisations continued to advocate for change, supporting individual families, engaging with decision-makers and calling for fair access. In August 2017, NHS England announced an interim policy providing funding support for hospital costs associated with administering nusinersen to some children with SMA Type 1.
However, the eligibility restrictions raised concerns among clinicians and families, particularly for children who fell outside the criteria but who could potentially benefit from treatment. Further advocacy continued to push for a fairer approach.
Continuing the campaign during uncertainty
In 2018, campaigning continued to protect access for children with SMA.
Following Scotland’s decision to fund access to nusinersen, the EAP closed there. Later that year, Biogen announced the closure of the programme to new infants in England, Wales and Northern Ireland before NICE had completed its final funding decision.
Strongly advocating for a bridging solution, SMA organisations highlighted the devastating impact that delays could have on families of newly diagnosed babies. More than 80 children in the UK remained on the EAP at that time, but newly diagnosed infants faced uncertainty about when they would be able to access treatment.
Securing long-term NHS access
In May 2019, following NICE’s recommendation for NHS funding of nusinersen, Biogen committed to reopening the EAP for newly diagnosed infants with SMA Type 1 in England, Wales and Northern Ireland while NHS funding arrangements were finalised.
By July 2019, NHS England began taking over responsibility for funding treatment, bringing an end to a long campaign for sustainable access.
A legacy of advocacy
The journey to secure access to Spinraza® was one of the most significant advocacy efforts in SMA’s history.
It showed the power of collaboration between families, clinicians, patient organisations, NHS decision-makers and industry. Through persistent campaigning, evidence sharing and direct support for families, the SMA community helped overcome significant barriers and paved the way for access to the first life-changing treatment for people living with SMA in the UK.
The introduction of nusinersen transformed what was possible for many people with SMA and remains a powerful example of what can be achieved when a community comes together to demand change.
Community Story
“His fine motor skills have enabled him to be able to fully access the school curriculum – using his iPad to write the most wonderfully imaginative stories. I have many other anecdotes of how seemingly small, clinically unmeasured gains have improved Ezra’s health and wellbeing, and the wellbeing of the whole family in a huge way. Spinraza has not only kept Ezra alive, but it has also enabled him to thrive.“
