Only drug to treat debilitating childhood disease will no longer be funded by NHS for new cases
Families of children with Batten Disease hit out.
The family of two children who have a life-limiting disease are hitting out at the NHS for stopping the only treatment.
There's no cure for Batten disease, a rare genetic disorder, which progresses rapidly, causing seizures and dementia, and gradual loss of speech, mobility and vision, but an enzyme therapy being used on select child patients at Great Ormond Street hospital, has slowed the progression down, particularly for 15-year-old Ollie Carroll and his 12-year-old sister Amelia, from Poynton in Cheshire.
Both took part in a trial and have been receiving infusions for the last nine-years, but it's now been decided by health body NICE that no new cases will be offered the treatment. Those currently on the Cerliponase Alfa programme can continue until they and their NHS healthcare professional consider it appropriate to stop.
"It's wrong and it's disgraceful"
Ollie and Amelia's dad Mike said, "I can't put into words how shocked and appalled I am by this decision."
"When Ollie and Amelia first started this treatment the seizures stopped, we lived a normal life. We could go on holiday, we could go on planes and make precious memories with our children. So the treatment made a huge, huge difference."
"You can't put into words how it feels to be told your children have a disease with no cure, in a hospital room, basically go home and wait for your children to die. Families in the future will now be told there is a treatment there that is slowing down the progression of the disease but you can't get that treatment because the NHS and NICE has decided it costs too much money. It's wrong!"
Why the committee made these recommendations
On the NICE website the decision states:
Cerliponase alfa has been available through the MAA but is not routinely available in the NHS. Standard care without cerliponase alfa is supportive, aiming to relieve symptoms, and maintain function and quality of life.
The new evidence includes data from clinical trials and from children having treatment in the NHS in England. The clinical trial evidence shows that cerliponase alfa slows progression of CLN2. Patient experts and clinical experts have also explained that cerliponase alfa is a transformative treatment. But there is not much evidence about how well it works long term.
The committee took into account the condition's rarity, severity and the effect of cerliponase alfa on quality and length of life. But using the proposed price of the medicine, the most likely cost-effectiveness estimate is not within what NICE considers an acceptable use of NHS resources for highly specialised technologies. So, cerliponase alfa is not recommended.
Mike and the Batten Disease community in the UK say they will fight this decision.
"It's wrong and it's disgraceful. There is a huge worry here, not just for Batten's disease but for all rare diseases because if they say no to this drug the chances are they'll say no to all rare disease drugs.''
A NICE spokesperson said: “We know today’s news will be very upsetting for people with Batten disease and their families. NICE and NHS England have used all available flexibilities to reach a positive solution: collecting four years’ of real-world clinical data on the treatment’s efficacy; using the Highly Specialised Technology process for its assessment which allows a cost-effectiveness threshold ten times higher than usual; and discussing a potential commercial agreement with the company for the last 9 months. We are, therefore, deeply disappointed that the company was unwilling to agree a reduction to the £522,000 per patient per year list price that would have allowed newly diagnosed children to benefit from this treatment.”