Families await news on drug to treat muscle-wasting disease
Parents desperately waiting to discover if a new drug to treat children with a muscle-wasting disease will be offered by the NHS in England are hoping a decision will be made next week.
The National Institute of Health and Care Excellence (NICE) will meet on Wednesday, May 8, to consider whether to recommend funding for the only-known treatment for spinal muscular atrophy (SMA).
Among those waiting for news is Katie Prescott and her 11-year-old daughter Heidi from Wolverhampton who had hoped a decision would have been made by now.
The schoolgirl's condition has deteriorated in recent months and she now needs spinal surgery - something they believe wouldn't have happened if she had been given access to the drug.
SMA is a genetic condition that makes muscles weaker and causes problems with movement.
Although there is no cure families say Spinraza can help slow down the effects and help maintain independence.
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.
Speaking on her Facebook page, which she uses to raise awareness, Katie said: "This is just an absolute joke now. They do not care about our SMA community. They are not looking out for us like other countries are for theirs.
"So much damage done already. So much deterioration over the years amongst us all. But still no urgency? Makes me sick to my stomach."
Wolverhampton South West MP Eleanor Smith, who has been supporting the family, said: "Delays on making a decision to make Spinraza available in England have a real cost. Children and adults with spinal muscular atrophy keep irreversibly losing muscle function every single day.
"The parents of my 11-year-old constituent Heidi Prescott are very scared for their daughter. She has gone from an independent walker able to dress herself, to constantly being in a wheelchair, having to be dressed and undressed, showered, toileted. The deterioration in her condition has happened far faster than they expected. She needs urgent surgery."
NICE had previously ruled out recommending funding for Spinraza,also called Nusinersen, saying it wasn't 'a cost-effective use of NHS resources'.
But since then further meetings, including one in March, have taken place with Biogen which makes the drug and the outcomes will be discussed at next week's meetings.
A statement from NICE reads: "Following the meeting on 6 March 2019, the appraisal committee asked NHS England and Biogen to enter into commercial discussions to see whether a managed access arrangement could be agreed for Nusinersen consistent with NICE’s framework for determining cost effectiveness.
"The outcome of these discussions and the consequences for guidance development for this topic will be considered by the committee on Wednesday 8 May. Given the nature of the discussion, this will be held in private.
