Parents beg NHS for wonder drug to save their dying girl age 4
A DEVASTATED family has pleaded for a groundbreaking new drug to be approved on the NHS – and give their terminally ill girl a chance to live.
Saffa Shehzan suffers from CLN2, a type of Batten disease affecting just six children a year in the UK.
The average life expectancy for the neurodegenerative condition is 10. Children typically deteriorate as they near five, and four-year-old Saffa has already lost the ability to walk or eat.
But in the US a new drug called Brineura slowed the progress of the incurable disease for 20 of 23 children in a recent 48-week clinical trial.
It will be discussed by the National Institute for Health and Care Excellence – which decides what drugs to fund on the NHS – on April 25.
NICE has been reluctant to rubberstamp Brineura without “long-term evidence” of its benefits. A year’s treatment reportedly costs £500,000.
Yesterday parents Saima and Majid, both 35, of St Annes, Lancs, pleaded with NICE to approve the drug quickly to give their daughter some hope.
Dad Majid said: “Twelve months ago, she was normal, but the speed of the regression is incredible.”
Saima added: “Timing is of the essence. Usually five is a huge milestone for a young child but I dread her birthday because I know things are getting closer.”
Local Tory MP Mark Menzies has urged Health Secretary Jeremy Hunt to fund the drug. “Given the small number of children it affects, I see no reason why this treatment cannot be made available,” he said.
A petition by the Batten Disease Family Association, signed by 90,000 people, is also urging funding.
It said: “Children are able to access treatment in the US and other European countries. The UK lags behind.”