Sisters who may not live to teens given 'death sentence' after NHS rejects drug

Mum Gail Rich today slams health officials for imposing a “death sentence” on her daughters by refusing to fund vital drugs.Gail and husband Matthew face watching their girls’ slow decline at the hands of terminal Batten disease. Nicole, seven, and Jessica, three, could be left blind, unable to walk and hit by dementia then dying before they reach their teens.NHS England could not agree a price for a drug given to the girls in a clinical trial and there were questions over long-term effectiveness.But the drug has stabilised Nicole and prevented any symptoms emerging in Jessica.And in Wales, another child WILL get it because of funding rules there.
Gail, 41, says: “We have to fight. If this treatment is ripped away our girls will die. They will lose their abilities one by one and they will die.“That is something we’re trying not to comprehend. Everything a parent dreams of could go because of a decision that will tear our family apart and destroy our happiness.“It is a death sentence. That’s what it is if the treatment is taken away.“If there wasn’t a treatment or anything they could do you would be devastated, but accept it. You’d make the most of every second. But the fact there is a treatment, it works, and they can see it is working, is just cruel.
"It’s frustrating, incomprehensible. These decisions are made on economic models and financing. That’s not the way it should be governed. Where is the compassion, the human element?”Full-time mum Gail, from Throckley, near Newcastle upon Tyne, has seen Nicole suffer the unforgiving effects of the disease. She is unable to walk unaided or talk – but the plucky girl is happy and is given every opportunity to live as normal a life as possible.The fact NHS Wales has funded the drug adds to the torment. Gail goes on: “It’s not just a postcode lottery, it’s a country lottery. Wales is just down the road! Are we a ‘United’ Kingdom? It doesn’t feel like we’re united in this.“Kids in England are denied funding because they don’t live in the right country. Yet, just over three hours away in Wales, treatment is granted.”Nicole and Jessica have been given enzyme infusion therapy as part of a trial run by a US drug provider.

Gail and 31-year-old Matthew, a production operative at a paint factory, say the results are amazing.However, the National Institute for Health and Care Excellence (NICE), has recommended the therapy should not be made available on the NHS.NICE expressed concerns about the long-term effectiveness of cerliponase alfa and said the NHS could not reach a commercial deal with the provider.Gail said Jessica’s trial, funded by American pharmaceutical firm BioMarin, ends in May 2020. Nicole is on a “compassionate use” programme at the goodwill of the company. But it could now end any day.Eleven UK children get the therapy – four on trial, seven on the compassionate or sibling programme.Four newly diagnosed children have so far been denied the treatment. It costs around £500,000 per patient, per year and is the first and only drug aimed at all 14 forms of Batten disease. Some 50 children in the UK have the illness.