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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.

For an example of a MAA involving another drug in another rare disorder, please click here.

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.

Why will guidance take so long as it is to be published on 26th June?

We are baffled, too, as our belief was that the treatment programme will start within a few weeks of positive recommendation. However, NICE are within their rights to set such a distant date. Actually, it is not unusual for NICE guidance to take months to be developed, vetted and published.

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 will be able to receive Spinraza treatment within the next few weeks 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, which will likely take place in late June or July.

We expect that it will take several months, up to a year, 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 days. We have requested to 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 criteria will be announced by NICE in the next weeks. Although we do not know the final version, they have been developed by clinicians with good knowledge of SMA and we expect them to be relatively patient-friendly.

My child is ventilated, will they still get access?

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. Any use of mechanical ventilation is not and will not be considered an exclusion criterion in accessing Spinraza. We have now received conflicting information, please bear with us while we clarify this.

Children who have been treated under Expanded Access Programme will certainly continue to do so.

Will adults receive Spinraza?

Yes. The NICE recommendation does not propose age restrictions on treatment. Adults will also be treated.

Will Spinraza be offered to those with SMA type 4?

Yes. NICE uses the traditional classification in which people who are sometimes called “type 4” are counted as type 3b.

As we have now received conflicting information on adult-onset SMA, please bear with us as we clarify this issue.

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

Even though NICE recommendations are binding on the NHS in England and Wales, Northern Ireland largely follows NICE recommendations. Please bear with us as we are clarifying the issue.

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. However, please keep in mind that newer molecules like AVXS-101 and risdiplam are widely expected to have higher efficacy and a better safety profile than the relatively old molecule nusinersen (Spinraza). 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 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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