Leeds MP backs parents’ call for drug access

Clare McVeigh, 41 and Robin Warden, 49, who lost their eight month old, Grace, last year to a rare genetic illness, SMA.
Clare McVeigh, 41 and Robin Warden, 49, who lost their eight month old, Grace, last year to a rare genetic illness, SMA.
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Leeds West MP Rachel Reeves has backed a mum’s call for health chiefs to allow babies with a devastating genetic condition unrestricted access to a new treatment.

The MP has spoken out in support of Clare McVeigh, 41, whose daughter Grace died last year aged just eight months old from the rare illness Spinal Muscular Atrophy (SMA) Type One.

Leeds West Labour MP Rachel Reeves.

Leeds West Labour MP Rachel Reeves.

As reported in Saturday’s Yorkshire Evening Post, the new drug, called Spinraza, arrived too late for baby Grace but now Clare, and partner Robin Warden, 49, are supporting a campaign urging NHS bosses to improve access to the treatment for other babies with Type One, the most serious form of SMA.

The condition causes gradual muscle paralysis resulting in the inability to swallow, eat and eventually breathe, with most babies dying before one.

Current rules mean Spinraza - which has been shown to improve symptoms - can only be given to type one babies under seven months and with two copies of a gene called SMN2, which the drug works on.

Campaigners argue this excludes other babies who could benefit and Ms Reeves has now written to NHS England CEO Simon Stevens and Secretary of State for Health, Jeremy Hunt urging them to reconsider the “discriminatory and unacceptable” restrictions.

Clare said: “We just want to clear any eligibility criteria which might exclude any child who might have a positive response to treatment.”

She added: “Grace was so close. If she had been born six months later, we would have been in the situation where there was potential to access treatment. It shattered my heart that she wasn’t born at the time that it was available but then if we were in the situation of [some of] these parents, I can’t imagine the trauma of seeing your child slowly dying and there being a treatment that they can’t access.”

Ms Reeves said: “I am delighted to be involved in the campaign to provide unrestricted access to this life saving drug. The fundraising, research and continued persistence by Clare and others on this issue is truly inspiring and hope to help them bring about change, so more young lives can be saved.”

Spinraza, also known as Nusinersen, is currently funded by manufacturers Biogen on a temporary basis, through an Expanded Access Programme (EAP), pending the outcome of a formal evaluation from NICE.

Joanna Mitchell, CEO of The SMA Trust, which funds SMA research and campaigns for faster patient access to new treatments, said: “The short-term...programme was intended for all Type I babies and it’s frustrating to see the restrictive criteria being imposed, particularly when clinical trials showed clear evidence of improvement, regardless of [SMN2 gene] number.

“These are very sick babies who stand a high chance of dying without treatment - we are working hard with clinicians, NHS England and other SMA patient advocacy groups to try and get these restrictions lifted as quickly as possible so that all Type I babies can get this effective drug, the first treatment for SMA.”

A spokesperson for NHS England Specialised Commissioning North said: “NHS England has an urgent clinical commissioning policy statement supporting the treatment costs for Biogen Extended Access Program for Nusinersen in SMA Type 1. This was based on the clinical evidence presented to NHS England’s clinical panel earlier this year. It is expected that Nusinersen for SMA will be appraised or evaluated by the National Institute for Health and Care Excellence (NICE).”