UK Access Summary:
From Autumn 2016 - July 2019

Last updated 27th September 2019

We have continued to advocate for access for all since July 2019  - for more on this, please go here.

1. The Global Expanded Access Programme for those with SMA Type 1

2. NICE’s appraisal of the treatment for all with 5q SMA

3. Access in Scotland

4. Europe and the rest of the world

5. Nusinersen's International Prizes

1. The Global Expanded Access Programme for those with SMA Type 1

In Autumn 2016, the ENDEAR clinical trial results of nusinersen for children with SMA Type 1 were sufficiently positive for Biogen to end the trial early and for all children in the trial to be transferred to the treatment. Biogen opened their global Expanded Access Programme (EAP) for children with SMA Type 1 providing the drug free of charge while countries assessed the drug and arranged for ‘reimbursement’ of the cost of the drug to Biogen to come from their own health care system. This was quite unprecedented.

On 23rd December 2016, the US Food and Drink Administration (FDA) approved nusinersen's use for both children and adults with SMA Types 1, 2 and 3 SMA.

By January 2017 the EAP had opened in many countries but was only beginning in the UK (at GOSH) as the NHS Trusts didn’t have the funds / capacity to cover the administration costs of delivering the treatment. Families had started to travel to Europe to access the treatment via the EAP at considerable cost to themselves and with all the stress and risks of travelling with a very sick child.

There was a huge amount of advocacy work from clinicians and patient groups with support from MPs (All Party Parliamentary Group 18th July) to open the EAP in the UK. It soon opened in Northern Ireland and Scotland but took until late July 2017 for NHS England to agree to fund the treatment and for the EAP to roll out to other Centres.

The programme closed in Scotland in May 2018 following the decision there for the Scottish NHS to fund treatment for infants with SMA Type 1.

Biogen closed the programme to new infants in England, Northern Ireland and Wales on 1st November 2018 despite the fact that NICE (England's National Institute for Health and Care Excellence) was still engaged in appraising the treatment and had not made a final decision whether it would recommend it for funding by the NHS. We strongly urged Biogen not to take this step and following this, continued to advocate for an extension of the programme or a ‘bridging solution’ until such time as NICE made its final decision. You can read more about this lobbying, here.

More than 80 children remained on the programme. Biogen advised this was the most for any country in the world and that they had reached the limit of their willingness to continue to support the NHS – due largely to their frustrations with the NICE process (see below).

Following the NICE access decision on 15th May 2019 (see below), Biogen commited to reopen the EAP for infants newly diagnosed with SMA Type 1 in England, Wales and Northern Ireland until the new guidance was finally published with NHS England then starting to take over funding completely from July 2019. 

You can read more about which children were treated via the EAP and the huge amount of advocacy by SMA UK, MDUK, TreatSMA, clinicians, the SMA community and politicians here.

2. NICE’s appraisal of the treatment for all with 5q SMA

NICE (The National Institute for Health and Care Excellence) is the regulatory body for England which reviews evidence from clinical trials, and the costs of providing the treatment. They then make evidence -based recommendations / issue guidelines. If NICE recommends the drug for use through the NHS, then NHS England is normally expected to fund the treatment within 90 days. 

January 2017

Anticipating that the treatment could be licensed for use in Europe (which at this time was the point at which the Secretary of State / NICE could start to consider a treatment for provision by the NHS), NICE undertook what is called a scoping exercise to gather information about the condition and the treatment and, whether if there were to be an appraisal, this should be as a:

  • Single Technology Appraisal (STA) used for common diseases or
  • Highly Specialised Treatment (HST) used for rare conditions – but with strict criteria limiting this possibility.

An STA has a much lower price threshold for approving a treatment than an HST.

It was known that nusinersen had a high price, which Biogen argued was in line with similar products and reflected the research costs etc. and that it would be unlikely to meet the STA cost/benefit (ICER) price requirements.

We made a written submission at this scoping stage and, with representatives of the other Patient Advocacy Groups (PAGs: MDUK, Genetic Alliance, TreatSMA), attended the scoping meeting. SMA UK argued that though neither route was a 'good fit' for SMA, the HST route was most suitable for this appraisal.

1st June 2017, the European Commission released its report (prepared by the European Medicine Agency (EMA)) approving nusinersen for marketing as a treatment for all with 5q SMA.

Biogen, clinicians and patient advocacy groups waited and waited for NICE to announce that it would be starting its appraisal. We had anticipated hearing this by September 2017 but heard nothing until:

18th January 2018 NICE announced the appraisal would be an STA, despite SMA not being a common condition. 

Clinicians, patient groups and Biogen responded to calls for evidence submissions. You can read the results of our community survey about the impact of SMA and people's views of access to nusinersen here and a copy of our submission that drew on these here.

27th June 2018 we joined other patient and clinical experts to look at all the evidence at NICE's first committee meeting  

14th August 2018 NICE released its consultation paper  containing the devastating news that they had not recommended nusinersen for funding by the NHS. This was on the grounds that:

  • Its clinical effectiveness was not yet proven – the treatment hadn’t been trialled for long enough. NICE indicated that this though would not be a block to possible access via a 'Managed Access Agreement' (MAA). This would be a 3 – 5-year scheme agreed between Biogen, NICE and NHS England (NHSE) that would enable access to treatment and the opportunity to collect further evidence of its clinical benefits and impact on quality of life. 
  • The price was too high. Biogen argued that the package/price they were offering NHS England was the best offer any country in Europe had had and that countries with a lower GDPR than the UK and Scotland (see below) had managed to afford it. Also, the budget /price threshold that Biogen was expected to meet was the one set by the STA process which was much lower than if had been assessed via an HST process (as we and Biogen believe should have been used) so, unless NICE and NHSE budged on this, price was going to continue to block access. 

We joined with clinicians to express our grave concerns about the decision, the long delays, the unsuitable nature of the appraisal process and the cost of the treatment in letters as follows:

5th September 2018 CAR-T therapy Kymriah got “fast-track” recommendation from NICE/NHS England through the Cancer Drugs Fund. It took only 10 days. On the same day we submitted our response to the consultation paper - 462 days after EMA’s marketing approval. You can read this here 

23rd October 2018, we joined other patient and clinical experts at the 2nd committee meeting and between us again strongly advocated for access. A final decision was due by the originally announced date of 21st November 2018 but this date was later taken down from NICE’s website.

25th February 2019, SMA Community representatives spoke about their lives to MPs at the Access to Nusinersen parliamentary 'drop-in' session and gave out this ‘Time is Running Out’ handout. The session was organised by MDUK and SMA UK and facilitated by Mary Glindon MP.

During these months, families wrote to MPs, NICE and NHS England officials and appeared in both local and national media highlighting the impact of SMA and what was happening with access to nusinersen. All the PAGs were involved in supporting these activities.

4th March 2019, the Guardian published an open letter signed by thirty clinicians involved in the care of both children and adults who have SMA. It had our support and called for NICE to approve Spinraza and for a change to the appraisal system. Read the letter here.

6th March 2019 was NICE’s third committee meeting. We joined the patient and clinical representatives at the meeting to speak up for access. The results of the Dec 2018/Jan 2019 survey on the costs of caregiving that we had undertaken with Wickenstones (you can read the results hereand the results of our previous survey helped to inform discussions. TreatSMA organised a protest outside the NICE offices.

30th April 2019 NICE announced that the outcome of their discussions, following the meeting on 6th March, and the consequences for access to nusinersen, would be considered by a private committee meeting on 8th May.

Following this, SMA UK and MDUK asked the Community to send our letter, accompanied by a flyer, to their MP so that they could write three letters – to NICE, to Biogen and to NHS England - calling on them all to work strenuously so that on 8th May, the NICE committee could endorse an agreement for access to nusinersen for all with SMA Types 1, 2 and 3. Read more here. SMA UK and MDUK also sent their own letters to the three organisations, which you can read here.

8th May 2019 NICE's fourth committee meeting was held in private.

15th May 2019 At last, the SMA Community had the answer it had been asking for since NICE started its appraisal almost 17 months ago. Nusinersen was to be made available by Biogen immediately to the youngest and most severely-affected infants (SMA Type 1). The NHS were then, after their full guidance publication date of 26th June,  to begin to provide nusinersen to those with SMA Type 1. All patients already on the EAP would continue to have access to treatment. Those with SMA Types 2 and 3 would start to get access including anyone who had shown symptoms of SMA before the age of 18. This would be subject to: the Managed Access Agreement (MAA) starting criteria (including, that patients must not be permanently ventilated and there must be full consultation between the family and the treating clinician) which would be included in the published guidance;  services being ready and able to cope with the patient load. The MAA would also define any stopping criteria. Treatment would therefore be available to adults who met the starting criteria. Nusinersen would also be available to pre-symptomatic siblings who, based on their genetics, were very likely to develop SMA. The SMA community was elated, many people in tears with emotion and relief.

3rd June 2019 NICE released: its Final Appraisal Determination (FAD) which would form the guidance to the NHS; the draft Managed Access Agreement (MAA); and committee papers that summarised the information it considered in the process of getting to this stage. We noted that the MAA was only a draft and as such still required some further clarity and development. Jointly with MDUK and TreatSMA we immediately raised concerns that as it was currently written, it appeared to exclude some people from access for whom the treatment would be potentially beneficial, and clinically safe, in particular, it excluded those with SMA Type 3 who had lost walking ability (lost ambulation) and there was also ambiguity around whether spinal fusion and contractures might prevent access. We did not consider this to be in line with the announcement NICE made in May that, "This is one of the most comprehensive deals in the world, meaning all relevant SMA patients (SMA 1, 2, 3a and 3b), including adults and siblings who are yet to show symptoms, will be able to benefit from this treatment". 

NICE and NHS England committed to respond to our concerns at the earliest possible time and we continued to work with MDUK, TreatSMA and clinicians to establish the next most constructive steps at what was now a most delicate stage in this long, difficult and distressing process. We had until June 17th to appeal against NICE's recommendations.

11th June 2019  NICE / NHS England called a confidential meeting with all the Patient Groups. We all advocated for all the changes finally published in the new MAA  (see below) including access for all with SMA Type 3. SMA UK followed these requests up in writing. We were also in regular contact with Biogen advocating in the same way. Again these discussions were confidential.

14th June 2019 We announced that we had co-signed two letters to address the concerns that were being widely expressed about the MAA:

Letter from SMA REACH Clinicians and Patient Advocacy Groups (PAGs): led by Professor Francesco Muntoni with input from clinicians and PAGs.  In summary it asked NICE and NHS England to consider the following:

  • Based on the strong clinical evidence available, amend the MAA criteria now to include access for paediatric patients with SMA Type 3 who have lost ambulation. 
  • As part of the MAA, introduce regular reviews of new evidence for groups of patients currently excluded from the MAA and a commitment to amend the MAA if there is clear evidence of the clinical benefit. 
  • Establish an SMA REACH steering committee of clinicians and academics to ensure a uniform interpretation of the guidance and to discuss complex cases. 


Letter from the Patient Advocacy Groups (PAGs): to NICE and NHS England raised our concerns about the way decisions had been communicated and the emotional distress this had caused the SMA Community. It asked for this to be taken into consideration when NICE and NHSE were making their next decisions and communicating them. We made it clear that we wanted to be able to work with them positively and collaboratively on the next steps of the MAA. 
Deciding whether to appeal 

We had been advised that if an appeal was lodged and was deemed to require further exploration, it would halt the final publication of NICE/NHSE guidance and therefore the progress of the roll out of the programme for all. This would add many months of delay to the possibility of anyone other than infants with SMA Type 1 receiving the treatment. It would trigger a new committee meeting which would take place some two months further down the line creating further critical delays. This committee could then arrive at the same decision, but more importantly, a new review by a new committee could result in losing some of what we had all fought so hard to achieve. Knowing these grave risks, SMA UK, MDUK and clinicians decided not to appeal but to trust the process and the advice and consultations we had been part of. TreatSMA decided to appeal. It was up to each group to make its own decision about what actions to take during what was a very fraught and difficult time. We took care to respect these decisions.

We were unable to speak publicly about any of this at the time as we were all bound by confidentiality agreements. At times this was very frustrating. We were greatly saddened to hear of the divisions that were arising in some parts of the community and about some of the social media directed at people who chose one path or another. At times, we were also under fire but were unable to say anything.

We wrote jointly with MDUK to Health Minister, Vaughan Gething, in Wales and received this reply. In the absence of a Northern Ireland Executive, we wrote to the NI Department of Health Permanent Secretary, Richard Pengelly, and Secretary of State for NI, Karen Bradleyurging them to be ready for NICE guidance on nusinersen being issued on 26th June so that there wouldn't be delay and any difference in access between countries. We received a response from Karen Bradley which you can read here.

3rd July 2019 NICE /NHS England finally announced the new MAA which confirmed access for those with SMA Types 1, 2 and 3 or who were pre-symptomatic and who met the revised entry criteria. There was still no upper limit of age. The key changes were: 
  • Paediatric patients who had recently (in the previous 12 months) lost the ability to walk independently were now included. These patients would need to have regained independent ambulation within 12 months of the treatment having been initiated in order to continue treatmen
  • The MAA commited to considering any significant evidence being made available by Biogen in relation to all with SMA Type 3 who are non-ambulant that may impact on the eligibility criteria of the MAA.This kept the door open for there to be a potential change to the above stopping criteria for children and for other children and adults to become eligible at a later date.
  • The entry criteria that excluded those with spinal fusion surgery that prohibits safe administration of nusinersen’, was amended to clearly state that this judgement rested with the treating clinician.
  • To assist with this, NHS England would set up a multidisciplinary clinical panel which would include physiotherapists. The panel would be able to give expert advice to centres providing treatment on the feasibility of intrathecal administration of Spinraza. This would ensure that access to treatment would not be affected by geographical/postcode variations in the ability of clinicians to administer it. 
  • The entry criteria that excluded ‘those with severe contractures that prohibited measurement of milestones’, was amended to clearly state that this judgement rested with the treating clinician 
  • The position of patients who have been treated overseas was clarified:
    • Where a patient/family had been residing abroad to receive treatment, they could join the MAA so long as they met the MAA eligibility criteria and their treating centre confirmed that they were eligible for NHS treatment
    • Patients who had been receiving treatment privately could join the MAA as long as they met the MAA criteria, were eligible for NHS treatment and their NHS clinician was content that the proposed ongoing NHS treatment was clinically appropriate in light of the privately funded treatment that the patient had received
    • In both of these above scenarios, it was for the patient/family to ensure that there was an appropriate handover between the organisation currently treating the patient and the NHS treating centre in England
    • In light of the need to stagger the start of treatment beginning with ‘those patients in whom motor neuron loss can be minimised’, it might be necessary for patients receiving nusinersen under other funding mechanisms (such as those above) to continue to access treatment via these mechanisms until the system was ready to treat other similar NHS patients.
  • All patients would be treated / continue to be treated in line with the published 2017 International Standards of Care for SMA. Patients / families would be expected to cooperate with treating centres so that the patient received the appropriate standard of care for them.

What was considered but not changed:

  • The starting criteria 'excluding those on permanent ventilation / tracheostomy at baseline in the absence of acute and reversible infection' was not changed. This was in view of there being no published evidence that treatment would reverse the need for permanent ventilation.
  • The exclusion of further groups of patients such as those with SMA Type 0 or SMA Type 4 was not changed. This was in view of there currently being no evidence base to support their inclusion.

The MAA also stated that where possible, 'the newly diagnosed population should be prioritised'.

10th July 2019 NICE responded to the PAGs joint letter of 14th June acknowledging saying, 'we recognise that we could have been clearer and more sensitive in our communications about the outcome of this appraisal. Our senior team will reflect on your comments to identify how we can improve our communications for future appraisals to ensure that we provide as much clarity as possible.' You can read the letter in full here

In conclusion.

We were sorry that we didn't achieve all we hoped to with this MAA, but there’s no doubt we squeezed all we could out of ‘the system’ at the time. This does not mean the end. Going forward:

  • Biogen committed to do all they possibly can to collate clinical evidence globally as rapidly as possible and present this to the MAA Oversight Committee. We will hold them to this.
  • We, along with representatives from the other PAGs and treating clinicians will be part of the Managed Access Agreement. This will enable us to input and participate in the process. This will help us be better prepared as a patient group when nuinersen is reviewed by NICE at the end of the 5-year managed access period.

The SMA UK Support Services Team put as much time and energy into this advocacy work as possible. This spanned from the time of our confidential and extensive work with the NHS England working party throughout 2017 to help to establish the Expanded Access Programme for those with SMA Type 1. From December 2017 onwards, we worked on the community survey about the impact of SMA and views of nusinersen and made submissions to NICE. We were Patient Experts at the three NICE committee meetings and worked with the independent consultants Wickenstones to try to fill the gap about the economic impact of the condition on individuals and carers. It was not only clinical evidence but also agreement over the economic modelling that caused the long delays to NICE coming to its final conclusions.

We have argued all along that the STA route for nusinersen is not fit for a rare and progressive condition like SMA or an innovative treatment like nusinersen which won't have years of clinical trial evidence that include large numbers or patients. We suggest NICE knew  this and that this was why they took so long deciding (a) which route it should follow and (b) reaching this final decision. Nusinersen / SMA didn’t fit all the very strict HST criteria and there is nothing in between that route and the STA route. This aspect of the NICE appraisal system is not, we suggest fit for purpose. This is being argued at many levels by clinicians, pharmaceutical companies and charities but has always been refuted by the Ministry of Health, NHS England and NICE officials in their public response to letters about it.

We are very concerned that the NICE system and NHS funding levels have been leading us to a place where England is unable to benefit from the very exciting innovations and breakthroughs in drug treatment that are happening all the time. Nusinersen has highlighted significant problems due to:
  • the length of time this process takes (NICE is asked to publish guidance within six months of a product receiving its licence from the EMA. In reality the norm for full NICE approval, including possible appeals, takes an absolute minimum of 43 weeks, and can be much longer.  The 110 weeks it took for NICE to publish guidance on nusinersen is unacceptable. Every day matters for someone with a progressive condition.)
  • the binary STA / HST route 
Access to nusinersen is one story - there are many other rare conditions where patient advocacy groups are campaining for access and face the same issues. We are well behind other countries. Clinicians are hugely frustrated that they cannot offer their patients treatments they know are working in the rest of the world. We have heard several talk about leaving the NHS and England because of this. More treatments are on their way – families cannot go through the distress of the long wait they have had for this first treatment again. There are glimmers of the possibility of change with a lot of work going on by Genetic Alliance and MDUK that we are supporting, as well as work that will be feeding into NICE’s ‘methods review’ Find out more here.

3. Access in Scotland

In the meantime, Scotland’s Scottish Medicines Consortium undertook its assessment quickly and efficiently. We made a submission and attended meetings, working with MDUK. You can read about this here.

On 7th May 2018, Scotland agreed to fund the treatment for those with SMA Type 1. 

On 8th October 2018 the Scottish Government announced their new pathway for ultra-orphan treatments which gave some hope for those with SMA Type 2 or 3. To be eligible for this pathway, a company needs to establish that:

  • The condition has a prevalence of 1 in 50,000 or less in Scotland
  • The medicine has an EMA orphan designation for the condition and this is maintained at time of marketing authorisation
  • The condition is chronic and severely disabling, and
  • The condition requires highly specialised management.

If then the Scottish Medicines Consortium (SMC) consider a treatment clinically effective, it will be made available on the NHS in Scotland for at least three years while further information on its effectiveness is gathered. Following this period, the SMC would then review the evidence and make a final decision on its availability on the NHS in Scotland. 

Biogen announced on 13th February 2019 that, subject to successful sign off, nusinersen was expected to be routinely available in Scotland under these terms for those with SMA Types 1, 2 and 3 from April 2019. It was finally confirmed by the SMC in July 2019 that this was going ahead. 

You can read more when you scroll down on this page.

4. Europe and the rest of the world

You can see Biogen’s updates about access here.    

5. Nusinersen's International Prizes

During this time the original scientists who worked on nusinersen won the 2019 Breakthrough prize (the “Oscars for science”). Biogen was also awarded the international Prix Galien Award for Best Biotechnology Product for SPINRAZA (nusinersen), making this its seventh Prix Galien award (country recognitions in the U.S., Germany, Italy, Belgium-Luxembourg, the Netherlands and, ironically, the U.K).