Batten launch legal challenge over drug treatment

The parents of two girls with a rare degenerative disease have launched a legal bid to force health chiefs to fund a drug which could slow progression of the illness.

Nicole and Jessica Rich, of Newcastle, have the incurable Batten disease.

It causes seizures, visual impairment and a loss of mobility.

The National Institute for Health and Care Excellence (NICE) previously said there was not enough long-term data about the drug, cerliponase alfa.

Lawyers representing the family have applied to the High Court for a judicial review of NICE's decision not to fund it on the NHS in England, saying it is "unlawful on several grounds and is a breach of human rights laws".

A NICE spokeswoman said the organisation was unable to comment on the legal proceedings.

Nicole, seven, and Jessica, three, currently receive the drug through a scheme funded by an American pharmaceutical company.

Their parents, Gail and Matthew, said the treatment had appeared to halt the effect of the disease.

"All we want is to be able to give them the best chance in life," Mr Rich said.

"We are trying to remain positive, but it is difficult knowing that if their treatment stops it will effectively hand them a death sentence.

"What makes this even harder to try and come to terms with is the fact 19 other countries fund the treatment. If it can be available there, why not in England?"

The enzyme replacement treatment costs about £500,000 per person per year.

The average life expectancy of a child with the condition is 10.

In February, NICE said it could not make it available in England as a lack of data meant it could not be certain it would provide value for money.

However, last month it said it had agreed "to undertake further work" and added it "will not issue draft final guidance on this topic while these discussions are ongoing".