'It’s just such an awful situation': Young Heidi heartbroken as new drug is refused
A schoolgirl with a rare muscle-wasting disease is devastated after being told she can’t receive a new treatment recently approved for use on the NHS.
Heidi Prescott-Booth, who is just 11, has spinal muscular atrophy (SMA) – a genetic condition that makes muscles weaker and causes problems with walking and movement.
Last month Heidi and her family, who live in Wolverhampton, were celebrating after the National Institute for Health and Care Excellence (NICE) agreed to recommend Spinraza, the trade name of the drug nusinersen, which can help slow down the effects and help maintain independence.
But they’ve been left heartbroken after being told Heidi, who has type 3 SMA, wouldn’t qualify for the only-known treatment because she can no longer walk the required five steps.
It’s also not being recommended for anyone who has had or will be having spinal fusion surgery which Heidi is waiting to receive to urgently correct a curve in her spine.
Heidi’s mother Katie said they were shocked and saddened by the restrictions but vowed to continue to fight for the treatment.
“After announcing Spinraza had been approved for all types, the criteria excludes 15 per cent of people with SMA, Heidi being one of them.
“They have stated that those who could once walk but now can’t take five steps are not eligible.
“They have stated that any appeal will halt the distribution of Spinraza for anyone, which really is disgusting.
“As you can imagine we are devastated. Heidi has put so much into campaigning and fighting for Spinraza for all, for herself and everyone else, this has come as such a big blow, especially as she is amongst those excluded.
“I feel NICE and NHS England have a lot to answer for. They can’t announce Spinraza for all, and then go back and exclude people knowing full well we have told our children. It’s disgusting.
“I am heartbroken. Heidi is heartbroken. And it’s just such an awful situation after everything,” she added.
'Feels like cruel rationing'
Wolverhampton South West MP Eleanor Smith, who has been supporting the family, said: “Heidi Prescott and her mum Katie have been amazingly brave campaigners for getting fair treatment for everyone with Spinal Muscular Atrophy.
"Their campaign was about 'Spinraza for all' so this development where Heidi is unlikely to be given the drug is dreadful. It feels like shocking and cruel rationing.
“Heidi and her family have again been let down. This has been heart-breaking news for all those with SMA excluded from Spinraza treatment.
“The fight will go on for fairness and treatment for all. I have written to the National Institute for Health and Care Excellence asking them to make changes and act on the guidance from clinicians and patient organisations regarding the criteria for accessing Spinraza.”
Campaign group TreatSMA has responded to NICE and NHS England urging them to reconsider the proposals on who would be qualify for Spinraza.
It has provided detailed feedback on the potential impact of the proposals on some groups of patients, and also offered recommendations.
The group has also lodged a formal appeal but says it will withdraw it as soon as the views of patients and clinicians are taken into consideration.
NICE had previously not recommended it due to concerns over its long-term effectiveness and its high cost, but lengthy talks had been taking place with its makers, Biogen.
Now last month an agreement was made between NHS England and Biogen which will see the NHS fund the treatment, already available in Scotland, for a time-limited period, allowing further data to be collected on its effectiveness.
There are different types of SMA ranging from very severe type 1 through to type 4 – Heidi has type 3 – and one in 40 people carry the gene.
It is estimated there are there are between 600 and 1200 children and adults in the UK living with SMA.