Spinraza in the UK ‒ questions and answers

Below we offer answers to the most common questions we received about the Spinraza recommendation.

For information about the drug itself, its history, efficacy, side effects, and dosage, please refer to this page.

What is the MAA?

MAA stands for Managed Access Agreement, which is an agreement between the NHS, NICE, the drug’s manufacturer, and sometimes clinicians to implement and evaluate a new and often expensive treatment or procedure. It allows patients to start being treated while at the same time allowing the NHS and clinicians to better understand how patients being treated respond to treatment over a long time and whether this resembles the way that patients did in clinical trials.

A MAA describes the key scheme elements, such as: who can receive the treatment, how often they should be seen by a clinician, what tests should be performed, what information should be collected, and how decisions on treatment discontinuation will be taken (as well as any appeal process). Usually, pricing arrangements are also linked to MAA performance.

MAA stands in contrast to the standard commissioning process under which NHS procures and delivers common drugs and medical procedures, like antibiotics or X-Ray investigations, that can usually be administered on the NHS to whoever a doctor deems fit, without the requirement of any follow-up.

Click for Spinraza MAA

Which hospitals will provide treatment?

This is yet to be decided. We will inform the community as soon as this decision is taken. However, we expect that all the hospitals that participated in the Expanded Access Programme will offer Spinraza also in the new setting.

When will we get access to Spinraza?

Children with type 1 SMA are already able to receive Spinraza treatment thanks to a “bridging solution” agreed on by the NHS and Biogen upon TreatSMA request. Other children as well as adults will be progressively included in the treatment programme as soon as it formally starts in late July.

We expect that it will take several months, perhaps a year or even longer, before everybody with SMA in the country receives his or her first injection. Hospital capacity is what limits us. NHS trusts and clinicians will discuss triaging and the details of intake procedure in the next weeks. We will be involved in those discussions, so please bear with us while we work with all the stakeholders to provide you with precise information.

Who do we inform we want Spinraza. Will the hospital contact us?

The procedures are not yet established. We will inform the community as soon as more information is available.

What will the start/stop criteria entail?

The eligibility criteria for Spinraza treatment on the NHS are:

  • Patients who do not have SMA type 0 or type 4
  • Patients who are not on permanent ventilation (i.e., more than 16 hours a day for more than 21 consecutive days)
  • Patients with SMA type 3 who are no longer able to make 5 steps unsupported, except children (< 18 years) who have lost such independent ambulation in the last 12 months
  • Patients in whom intrathecal injection is impossible to be carried out safely as determined indivdually by the treating clinician (for example due to scoliosis or spine surgery)
  • Patients in whom it will not be possible to assess milestone achievement due to contractures as determined indivdually by the treating clinician
  • Presymptomatic patients

The stopping criteria are:

  • Deterioration as assessed in two subsequent tests (4 months apart):
    • by more than 2 points on horizontal kick or (more than) 1 point on other scores excluding voluntary grasp – on the HINE scale
    • by more than 4 points on the CHOP-INTEND scale
    • by more than 3 points on the Revised Hammersmith Scale
  • Start of mechanical ventilation for more than 16 hours a day for more than 21 consecutive days
  • In children who stopped walking in the last 12 months: an inability to regain independent ambulation after 12 months of treatment

My child is ventilated, will they still get access?

The currently proposed entry criteria unfortunately exclude those who rely on mechanical ventilation for more than 16 hours a day. Consequently, no ventilated patients can be started on treatment under the Managed Access Programme. However, those who have been receiving treatment under the Expanded Access Programme will continue to do so irrespective of their respiratory status.

The international document on Standard of Care in SMA recommends to use mechanical ventilation (part or full time) as a standard procedure in all children with severe forms of SMA. Parents are strongly advised not to discontinue ventilation.

Will adults receive Spinraza?

The current programme allows treating adults who have either SMA type 2 or SMA type 3 and are able to walk. Adults with SMA type 4 will not be treated. Adults with SMA type 3 who are no longer able to walk might be included at a later date, provided that there will be sufficient scientfic evidence from abroad to prove the treatment’s effectiveness.

Will Spinraza be offered to those with SMA type 4?

The currently proposed entry criteria unfortunately exclude those who have adult-onset SMA.

I live in Northern Ireland. What does this decision mean for me?

NICE recommendations are binding on the NHS in England. Northern Ireland, Wales and Scotland govern their healthcare separately. Nevertheless, both countries usually follow NICE guidance in medicine commissioning.

I live abroad. Can I come to the UK and access treatment?

Access to UK public healthcare for foreign nationals is governed by relevant legislation. For details, please refer to the NHS website.

What if I am on a trial?

If you are participating in a clinical trial of nusinersen (Spinraza), the developments do not affect you. We suggest you continue on the trial, which uses a Biogen-funded drug, so that long-term safety and efficacy data can be collected. You will be able to switch to NHS-funded treatment once the trial is completed.

If you are on a clinical trial of another experimental molecule, you are free to leave the trial and request Spinraza treatment. You will likely lose access to the experimental drug. If in doubt, discuss with the trial coordinator.

Will I be able to switch to another drug in the future?

There are several other drugs for SMA in the development pipeline, some of them are expected to be more effective, safer or more convenient in use than Spinraza. However, none has received marketing authorisation in UK/EU as yet, and there is little in terms of safety and efficacy data of any combination or alternating therapy. Unless you are determined to take part in a clinical trial, we recommend not to delay Spinraza treatment.

What about newborn screening?

The NHS recently rejected the idea of screening all newborns for SMA, citing lack of a path to effective treatment. With Spinraza now becoming a standard treatment in the country, TreatSMA will be campaigning for the NHS to reconsider the decision and add SMA genetic testing to the routine neonatal screening panel.

I have a question not answered above

If your question is not listed above, please contact us.

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