Changes announced to Spinraza MAA

Published Date
Bobby Ancil
Spinraza SMA treatment

Today, NICE and NHS England have announced amendments to the managed access agreement (MAA) for Spinraza (also called nusinersen) for people with SMA Types 1, 2 and 3. These changes follow strong representation from clinicians and the patient community urging NICE, NHS England and Biogen to reconsider the MAA eligibility in line with available clinical evidence.

In light of these changes we have signed up to the MAA alongside SMA UK and Treat SMA. Our role will be to represent the SMA community as part of the MAA oversight committee.

What changes have been made?

NICE, NHS England and Biogen have spent the last fortnight consulting with clinicians and reviewing the available evidence. As a result they have made some changes to eligibility criteria of the MAA. The key changes relate to:

  • Ambulation: paediatric patients who have walked previously but lost ambulation in the last 12 months would now be eligible under the MAA. However, they would need to regain ambulation within 12 months of starting treatment to continue receiving treatment.
  • Scoliosis and contractures: minor changes have been made to the criteria around scoliosis and contractures to make it clear this is a clinical decision linked to feasibility of the intrathecal injection.
  • Clinical oversight panel: a panel of clinical experts will be established who would have oversight of the criteria relating to feasibility of treatment for certain patients. This would ensure consistency in terms of how the eligibility criteria are understood.

You can read the full MAA document here. The revised eligibility criteria have been approved by clinicians.

How has eligibility been decided?

We welcome the flexibility shown by NICE, NHS England and Biogen in response to the clinical evidence presented to them. We also want to thank everyone in the SMA community, clinicians and MP’s for all they have done to campaign for access. However, we know that these new criteria still exclude some people with SMA, particularly non-ambulant adults, and that this will be very disappointing for those affected.

In order for the MAA to be successful it is crucial that the measurements required are manageable for practitioners. The current eligibility criteria has been agreed in consultation with leading clinicians. Decisions about inclusion, exclusion and stopping criteria in the MAA have been based on the way Biogen has measured clinical trial results to date i.e. using motor milestones.

As part of the MAA NICE, NHS England and Biogen have committed to reviewing further clinical evidence for non-ambulant SMA Type 3 patients throughout the duration of the MAA. This evidence would be reviewed by the oversight committee, which patient groups and clinicians would be part of. The committee would then decide if amendments to the MAA were required based on the evidence. The specific process around reviewing new evidence is yet to be developed.

Speaking about the changes to the MAA, Meindert Boysen, director of the Centre for Health Technology Assessment at NICE, said:

“Having considered new evidence about the effectiveness of Spinraza in people who have lost the ability to walk, NHS England and Biogen have been able to find a way forward that all parties are happy with and that will ensure Spinraza is available to as many people with SMA as possible.


“There will still be people with SMA who will not be able to access treatment with Spinraza under the terms of this agreement. But we are not closing the door to these people. Uniquely for this type of arrangement we have made a commitment that, during the 5-year course of the MAA, should evidence become available on the potential benefits of Spinraza for type III SMA patients that are currently not included in the MAA, we will review that evidence to see whether it would support a change in the MAA inclusion criteria.”

You can read the full statement from NICE here. 

What happens next?

The revised MAA has now been published on the NICE website. This replaces the previous MAA. All previous appeals have been set aside and a new appeals period has now begun and will last for a fortnight. We have been told very clearly by NICE and NHS England that any appeal against this new MAA would result in roll out of treatment being halted and could potentially result in substantial changes to the current access agreement, which could lead to a reduction in the number of people able to receive the treatment.

Provided no appeals are received then we would expect final guidance to be published by the end of July 2019. NHS England hope to start commissioning of the treatment as soon as possible.

We know this has been a distressing time for the SMA community who have been desperate for news. These changes would not have been possible without the dedicated commitment of the SMA community, clinicians and MPs in fighting for access and the willingness of NICE, NHS England and Biogen to show flexibility. Our focus now is on ensuring the MAA is implemented quickly and fairly so that people with SMA who are eligible can start accessing treatment as soon as possible.

Access in the rest of the UK

This week the Scottish Medicines Consortium (SMC) confirmed that Spinraza would be prescribed for children and adults with SMA Types 2 and 3 through their new ultra-orphan pathway for a period of up to three years while further evidence on its effectiveness is generated. Some children have already received treatment, having been considered on a case-by-case basis. There is more information about access in Scotland on the SMC website. 

Wales and Northern Ireland usually follow NICE guidance. We have been seeking assurances that they will be doing so in this case. We hope to get greater clarity once NICE have published their final guidance.

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