Urgent call to NHS decision makers for access to life-saving treatment for babies with SMA

We continue to work with Muscular Dystrophy UK, SMA Support UK and TreatSMA to call on NHS decision makers to unblock the problems that are preventing access to the first and only treatment for patients with spinal muscular atrophy (SMA).

Spinraza (formerly known as nusinersen), could help babies with the most severe cases of SMA – SMA Type 1 – live beyond their two-year life expectancy.

The drug is currently available to eligible children for free via an Expanded Access Programme by the drug manufacturer Biogen.

But an estimated 70 children in the UK with SMA Type 1 are still waiting for access to Spinraza as their local NHS Trusts have not agreed to help with additional costs and arrangements needed to make the drug available to them.

Postcode lottery

Spinraza is already available in Northern Ireland, France and much of Europe for children with the most severe cases of SMA. A third of eligible children in England are receiving the treatment through individual NHS Trusts including Great Ormond Street Hospital.

However, two thirds of children are still waiting for access as their local NHS Trusts and NHS England fail to address the funding and capacity issues, continuing to prevent the administration of the drug, which requires a lumbar puncture injection into the spinal cord. It is a procedure that is not dissimilar to those already routinely performed by the NHS.

So far rollout of the Expanded Access Programme has been so slow that many parents of children with SMA Type 1 have felt forced to take their children to Paris to access treatment at considerable personal and financial cost.

“It is unbelievably frustrating that a drug that has shown such exciting results in clinical trials is being given for compassionate use in other countries and yet is not widely available for UK babies,” said our Chief Executive Joanna Mitchell. “NHS England needs to sort out the current postcode lottery so that all babies have access to this potentially life-saving treatment as soon as possible.”

An urgent call for change

NHS delays are risking the lives of 70 babies and there is no time to wait. We’re calling on the NHS Trusts responsible for delivering treatment, NHS England, Biogen and patient advocacy groups to meet and reach an agreement urgently so that all eligible infants with SMA Type 1 who are not already receiving Spinraza can access it.

This meeting will involve agreeing on costs, treatment centres and sharing of funding responsibilities. Any finally agreed provision of treatment should be delivered in a way that will ensure even-handed access based on clinical evidence, not on a postcode lottery.

Recently, NHS England stated that it isn’t able to consider any requests for support until Biogen has its Phase 3 clinical trial results published in a peer reviewed journal.

We’re calling on NHS England to urgently review its criteria on peer review publication and ensure its policy is consistent with major regulators such as the European Medicines Agency (EMA), National Institute for Health and Care Excellence (NICE) and US Food and Drug Agency (FDA).

However, given the immediate need to secure NHS England support, we’re calling for Biogen to publish the clinical trial data in a peer reviewed publication.

We’re committed to continuing to work with Muscular Dystrophy UK, SMA Support UK and TreatSMA to push for access to nusinersen.

We’re also pushing for a broad approval from regulators in the UK, including NICE and the Scottish Medicines Consortium, which would see NHS funding provided for the drug for SMA Types 1, 2 and 3.

How you can help

Take action on this life-threatening issue by writing to NHS England Chief Executive Simon Stevens to ask NHS England to reach an agreement with Biogen and NHS Trusts without delay using our template letter. You can adapt the letter to include your own personal stories and experience of SMA.

If you live in Scotland, you can write to your MSP using our template letter.

If you live in Wales, you can write to your AM using our template letter.

If you live in Northern Ireland, you can write to your MLA using our template letter.

You can also help us continue to advocate for access to Spinraza for those living with SMA and support our research work searching for a cure and treatments by making a donation.

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