NICE / NHS England announce changes to the nusinersen Managed Access Agreement

03 July 2019

You can read NICE’s announcement here.

What are the changes?

The new MAA for nusinersen (SpinrazaTM) can be read in full here (see the list on the left-hand side; MAA link is dated 3rd July). It confirms access for those with SMA Types 1, 2 and 3 or who are pre-symptomatic and who meet the revised entry criteria. There is still no upper limit of age.

Key changes are:

  • Paediatric patients who have recently (in the previous 12 months) lost the ability to walk independently (MAA pg. 6 bullet point 3) are now included. These patients will need to have regained independent ambulation within 12 months of the treatment having been initiated in order to continue treatment (MAA pg. 12, table 1, line 3, final box)
  • The MAA commits 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 (MAA pg. 4 point 4.2) This keeps 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’, has been amended to clearly state that this judgement rests with the treating clinician (pg. 6 bullet point 10).
  • To assist with this, NHS England will set up a multidisciplinary clinical panel which will include physiotherapists. The panel will be able to give expert advice to centres providing treatment on the feasibility of intrathecal administration of Spinraza. This will ensure that access to treatment will not be affected by geographical/postcode variations in the ability of clinicians to administer it. (MAA pg. 6 bullet point 2)
  • The entry criteria that excluded ‘those with severe contractures that prohibited measurement of milestones’, has been amended to clearly state that this judgement rests with the treating clinician (MAA pg. 6 bullet point 4)
  • The position of patients who have been treated overseas is clarified (MAA pg. 22):
    • Where a patient/family has been residing abroad to receive treatment, they can join the MAA so long as they meet the MAA eligibility criteria and their treating centre confirms that they are eligible for NHS treatment
    • Patients who have been receiving treatment privately can join the MAA as long as they meet the MAA criteria, are eligible for NHS treatment and their NHS clinician is content that the proposed ongoing NHS treatment is clinically appropriate in light of the privately funded treatment that the patient has received
    • In both of these above scenarios, it is for the patient/family to ensure that there is 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 may 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 is ready to treat other similar NHS patients.
  • All patients will be treated / continue to be treated in line with the published 2017 International Standards of Care for SMA. Patients / families will be expected to cooperate with treating centres so that the patient receives 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.

What happens next - who will have access first?

The MAA states that where possible, ‘the newly diagnosed paediatric population should be prioritised’ (MAA pg. 9 point 5.8). We expect there to be further information from NHS England in the next few weeks about which paediatric centres will offer treatment and plans for the development of the programme for adults. We also expect answers to outstanding questions we collated on behalf of the Community and submitted in writing on June 12th, working with MDUK and TreatSMA. We will be doing all we can to support the successful implementation of the programme and its further development.

What role have SMA UK and you played in achieving these changes?

Following the release of NICE’s draft documents on 2nd June, Biogen made a proposal to consider further clinical evidence. This was by Darras et al. published in Neurology 21st May 2019. This evidence was outlined in the joint clinician and patient advocacy group letter sent on June 14th.

As a result, Biogen and NHS England agreed to extend the terms of the MAA to include the paediatric patients who have recently (in the previous 12 months) lost the ability to walk independently. The MAA was also refined in the light of a number of requests for clarification from stakeholders. This included those made verbally at a confidential meeting called by NICE / NHS England on June 11th with all the Patient Groups. We advocated for all the changes you now see in the new MAA along with access for all with SMA Type 3. SMA UK followed these requests up further in writing. We were also in regular contact with Biogen advocating in the same way. Again these discussions were confidential.

As these discussions were already taking place and a new agreement was already underway, the appeal made on June 17th against the original draft MAA is now not relevant and will not be considered by NICE. We are fortunate that this was the case. Had it been followed through and been deemed to have required further exploration, it would have triggered a new committee meeting which would have taken place some two months further down the line creating further critical delays. The committee may then have arrived at the same decision we have now, but more importantly, a new review by the committee could well have resulted in losing some of what we had all fought so hard to achieve. Knowing these grave risks, we chose not to make an appeal but to trust the process and the advice and consultations we had been part of.

We have been unable to speak publicly about any of this until now as we have all been bound by confidentiality agreements. At times this has been very frustrating.

It has been up to each group to make its own decision about what actions to take during what has been a very fraught and difficult time. We have taken care to respect these decisions.

Over the last many months, we have worked in particular with MDUK. We linked families willing to speak to the media with their Press Office and joined with them in writing media releases. Together we organised letter writing campaigns and MP drop-in sessions. We were pleased to attend All Party Parliamentary Groups facilitated by MDUK aware that, at times, they were also meeting in confidence to discuss progress.  In the last few weeks, MDUK has ensured that MPs and Ministers were kept as up to date as possible with developments so that they could ask more questions at PMQs, to Ministers and to NICE and NHSE representatives about the progress of the agreement.  

Most recently SMA UK chose to focus our energies on preparing the clinicians / patient advocacy group letter that was led by Professor Francesco Muntoni. We joined the SMA REACH clinicians network to discuss and help draft this. Many clinicians also made independent representations to NICE. On June 14th we also wrote the letter to NICE / NHS England expressing our concerns at the impact their communications and the proposed MAA was having on the SMA Community. This was co-signed by MDUK and TreatSMA. We took part in the confidential discussions we have outlined above.

We have been greatly saddened to hear of the divisions that have arisen in some parts of the community over the last few weeks and some of the social media directed at people who have chosen one path or another. At times, we have also been under fire but have been unable to say anything more than we have to date.

We are sorry that we have not achieved all we hoped to with this MAA, but there’s no doubt we have squeezed all we can out of ‘the system’ at this moment. This does not mean the end. Biogen have 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.

Being part of the Managed Access Agreement will enable us to input and participate in the process which will help us be better prepared as a patient group when Spinraza is reviewed by NICE at the end of the 5-year managed access period.

We, the Support Services Team, have, with the support of our Managing Director, put as much time and energy into this advocacy work as possible. This has spanned from the time of our confidential and extensive work with the NHS England working party through 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 are a very small team which, during this time, as well as working to keep you accurately up to date with all the developments that were not confidential via our website, E-news and social media, and work on access in Scotland, have also endeavoured to:

  • maintain and further develop our information and outreach services across the UK (families needing support now are our top priority – this has included the additional work created by the many enquiries about access to nusinersen that we have had from both within and outside the UK)
  • work on the Guide to the 2017 International Standards of Care
  • deliver the Information Weekend in April 2018 and shared experience events at the end of the year
  • further develop other shared experience networks
  • speak up for SMA and, for example, the need for emotional and psychological support and employment opportunities to name just two other significant pieces of work.

We are now getting ready to make a submission to NICE on the potential provision of Zolgensma (please do let us know your view via our survey) and working hard to launch our new website area ‘Living with SMA’ by the end of this month. This has, and still often does, take far more hours than we are paid for which we have not begrudged as this is much more than a job to us and we always push to do as much as we can.

Though the merger last year of The SMA Trust and SMA Support UK to form SMA UK will, we believe, be of great benefit to the Community in the long run, it did not at the time bring with it any additional staff. Commitments to ongoing research projects had to be honoured and to the events that have been part of the regular SMA Trust calendar. This required recruitment of contract Fundraising staff. There was no capacity to add further to Support Services resources. The timing of the merger was difficult but unavoidable for a number of reasons  beyond our control. We endeavoured at all times not to let it get in the way of our priority work.

We would like to thank all those in the Community who have worked both individually and alongside us to push for access, including: contacting MPs; raising awareness, such as in media campaigns and on social media; sharing personal experiences and stories; responding to surveys; and more. Only by working together have we been able to achieve what we have so far, and only by continuing to work together can we progress this MAA and further ‘help for today, hope for tomorrow’ for all.