Skip to content

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, NHS England is normally expected to fund the treatment within 90 days.

Our Advocacy and Access Diary:


We started the year writing jointly with MDUK to representatives at the Committee for Medicinal Products for Human Use (CHMP), the European Medicines Agency’s committee responsible for recommending the licencing of human medicines. We requested assistance in ensuring nusinersen was made available at the earliest possible time for the treatment of people who have SMA in the UK and Europe for as broad a label as possible and asked the SMA community to join us in this request.

Anticipating that the treatment could be licensed for use in Europe, NICE undertook their ‘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 highly unlikely to meet the STA cost/benefit (ICER) price requirements. In the light of this and the clinical trial results to date, they were at this stage therefore seeking access only for those diagnosed with SMA Type 1 with a view to a later application for other groups. Their view was this path would be the one that would have the best chance and be the quickest way to achieve treatment for those most severely affected and that access for others would then follow more easily.

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

1st June:

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: NICE announced the appraisal would be an STA. This would consider access for all those diagnosed with SMA Type 1, 2 or 3 as NICE’s view at the time was that nusinersen could be a potential suitable treatment for all. This meant that the potential population was above the ‘rule of thumb’ NICE operated at the time for defining a rare condition and an HST route.

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

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

14th August: 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 that Scotland (see this page) 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 nusinersen had been assessed via an HST process 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 to Biogen, NICE and NHS England about the decision, the long delays, the unsuitable nature of the appraisal process and the cost of the treatment in letters to Biogen, NICE and NHS England.

5th September: 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.

23rd October: we joined other patient and clinical experts at the 2nd committee meeting and between us again strongly advocated for access.

25th February: 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: 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.

6th March: 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 and the results of our previous survey helped to inform discussions. TreatSMA organised a protest outside the NICE offices.

30th April: 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 their MP could write 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.

8th May: NICE’s fourth committee meeting was held in private.

15th May: 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) and would be then funded by the NHS after their full guidance publication date of 26th June.
  • 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 which would be included in the published guidance and services being ready and able to cope with the patient load.
  • Treatment would 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: NICE released:

  • Its Final Appraisal Determination (FAD) which would form the guidance to the NHS;
  • The draft Managed Access Agreement (MAA);
  • The 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: 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: 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.

Read the letter here >

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.

Read the letter here >

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 all the relevant Ministry leads for Wales and Northern Ireland, urging 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.

3rd July: 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 treatment
  • The MAA committed 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 had been treated overseas was clarified
  • 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: 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.’

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 did not mean the end. Going forward:

  • Biogen committed to do all they possibly could to collate clinical evidence globally as rapidly as possible and present this to the MAA Oversight Committee. We made it clear we would hold them to this.
  • We, along with representatives from the other PAGs and treating clinicians would be part of the Managed Access Agreement. This would enable us to input and participate in the process. It would also help us be better prepared as a patient group when nusinersen 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 throughout 2017 with the NHS England working party 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 argued all along that the STA route for nusinersen was 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 suggested that 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 was nothing in between that route and the STA route. This aspect of the NICE appraisal system was not, we suggested fit for purpose. This was being argued at many levels by clinicians, pharmaceutical companies and charities but had always been refuted by the Ministry of Health, NHS England and NICE officials in their public response to letters about it.

We were very concerned that the NICE system and NHS funding levels were leading us to a place where England would be unable to benefit from the very exciting innovations and breakthroughs in drug treatment that were happening all the time.

Nusinersen highlighted significant problems due to:

  • The length of time this process took (NICE was 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 was unacceptable. Every day matters for someone with a progressive condition.)
  • The binary STA / HST route

The community was also left devastated by the limits the MAA had placed on access for some groups of people. The work that followed is summarised in the other advocacy for access sections.

Access to nusinersen was one telling story used many times by consultees in the subsequent review of NICE methods and processes. It helped inform some of the changes there have since been. For more on this, see Our Advocacy for Regulatory Change.