The family a four-year-old Sutton girl with a rare genetic condition are campaigning for a drug that could change her life to be made available in the UK.
Elsie Novell has spinal muscular atrophy (SMA), which means she is unable to stand or walk. There is currently no cure for Elsie’s condition, but a new drug called Spinraza could provide hope, as it halts the symptoms of the disease.
Charlotte Novell, Elsie’s mum said: “Unfortunately the drug has not been approved for use in this country which means that Elsie will not have access to this potentially life-saving treatment.
“It’s so disappointing to live in a county that cannot support your child’s medical condition when so many other countries can.”
The drug, which is administered via lumbar puncture and can cost £75,000 per dose, has been denied on the NHS due to the cost.
Charlotte added: “I understand that the cost isn’t sustainable, but if you add up the cost of caring for a child with SMA over a lifetime, it can come to a lot more than the cost of Spinraza.”
“I understand that we live at a time where everything is about money, but it should be about more than that.”
Meindert Boysen, director of NICE’s centre for health technology evaluation, which makes decisions on what drugs will be available on the NHS said: “We are actively engaging with Biogen (The makers of the drug) both on the uncertainties our committee raised about the drug’s long-term effectiveness, and on what they can do to make the drug cost-effective for the NHS.
“NICE believes that our processes do have the flexibility to evaluate drugs for rare conditions and make them available on the NHS. To do this, we also need flexibility on the part of the drug company.”
Charlotte hopes that their decision will be overturned.
She said: “We need to get the drug now, as Elsie’s spine is already starting to curve, so we’re working against gravity.”
“It’s upsetting that a drug has been found that could save a child’s life, only to be told you can’t have it.”
The family of Elsie will hold a fun day to fundraise for the specialist equipment she needs.
The family fun day will be packed with entertainment, such as a bouncy castle, face painting, a children’s entertainer and a Disney princess. The fun day will take place on September 23, 2-4pm at Post Mill Centre, South Normanton, and entry is free