Biogen has informed us that the Nusinersen Expanded Access Programme will close for new patients on 1 November 2018, even as those currently in the programme will continue without changes.
As you are aware, the Expanded Access Programme (EAP) to nusinersen was set up by Biogen globally in 2016 to offer free drug to those with type 1 SMA, as clinicians requested access to this life-saving treatment while it was going through regulatory procedure. The programme’s start in the UK, however, was repeatedly delayed by the NHS. TreatSMA and its international partners arranged for UK kids to be able to access the treatment in Paris, and many families availed of this opportunity. In 2017, we joined hands with clinicians and other charities and managed to get the programme kickstarted in the UK.
Currently there are approximately 70 children in England and Wales being treated via this route with 14 hospitals taking part in the programme.
Those children who are already receiving Spinraza via this route will continue to do so. Nothing will change for them and we would like to take this opportunity to reassure their parents should they have any concerns: your child will continue to receive treatment whether or not NICE recommend or NHS agree to provide Spinraza as a treatment.
Unfortunately, Biogen have decided to close new recruitment to the EAP for Type 1’s from 1 November 2018. This is devastating news for us as the SMA community, for those newly diagnosed babies and families. This lifeline, the only current real hope for families which could see their loved ones condition potentially reversed and many lives changed will be a route closed off for them. We all remember upon diagnosis we were told – terminal – EAP changed that for many.
Children will now be left to die and terminal will again be once more.
Although the news to close new intake EAP at present is still more than two months away from happening, it is one which is very realistic, very near and very serious. TreatSMA have reached out to NHS England with a request to hold a meeting with key decision makers to discuss the matter further.
Therefore we must continue to stand together and to push NHS England, NICE and Biogen to ensure continued treatment for those who need it now.
Biogen’s statement
As many within the spinal muscular atrophy (SMA) community are aware, Biogen opened the nusinersen global expanded access programme (EAP) in Autumn 2016. Biogen designed the EAP to provide an interim solution for patients with infantile onset SMA (consistent with type 1) between the end of the clinical trials and EMA approval, which was granted in May 2017. In the UK, it was extended further to support continued access for these patients until NICE had completed its appraisal. Two years on, we are extremely proud to have provided over 80 eligible children in the UK with nusinersen free of charge.
It has come to our attention that there has been some discussion within the SMA community in regard to the future of the EAP in the UK, and Biogen would like to take this opportunity to provide some background and clarity. As per the original planned timeframes provided by NICE – completion of the appraisal process and therefore final appraisal document (FAD) was scheduled for November 2018. Biogen agreed to extend the EAP in England, Wales and Northern Ireland in line with this timeframe, and the programme will be closed to new patients as of 1 November 2018.
While the EAP goes beyond what many companies offer, it was designed as a short-term solution, and we are aware that it has not met the needs of all patients with SMA, particularly type 2 and 3 patients where the clinical trial evidence indicates that there is potential benefit. It is therefore critical for the SMA community that a long-term and sustainable plan for access to nusinersen is established in the UK for all who may benefit. This can only be provided by the NHS (particularly NHS England), and the EAP should not be seen as a replacement for role of the health service.
Biogen continues to remain focussed on working with NICE and NHS England to agree the terms of a managed access agreement ahead of 1 November. Biogen has committed to ensuring that patients already enrolled on the EAP by 1 November continue to receive access to nusinersen.
During the past weeks, we have informed relevant members of the clinical community (as well as patient advocacy groups) of this decision to allow clinicians to ensure that all eligible patients are enrolled onto the EAP ahead of 1 November. The programme in England, Wales and Northern Ireland will be the final EAP in Europe still accepting new patients ahead of November 2018.
Other countries have either stopped enrolment or completed transition to access via the local health service, for example in Scotland.
Biogen is committed to working towards a sustainable solution, and we continue to collaborate with stakeholders from NICE and NHS England, as well as SMA Support UK, The SMA Trust, Muscular Dystrophy UK and TreatSMA, with the aim of finalising a managed access agreement ahead of November 2018. We urge that the terms be agreed by NICE and NHS England without further delay to ensure access for patients who may benefit from nusinersen, recognising that 20 countries across Europe have already provided access to nusinersen via their healthcare systems, and bringing an end to the uncertainty faced by patients in England, Wales and Northern Ireland.
