NHS green light for life-saving wonder drug
PARENTS of children suffering a rare fatal illness were celebrating last night after winning a battle for life-prolonging treatment following a Daily Express campaign.
Families of youngsters with genetic late infantile Batten disease – who have a life expectancy of 10 and are slowly robbed of sight, speech and mobility – hailed the decision as “a victory for children” after NHS England agreed to fund a drug which halts its devastating symptoms.
They thanked the Daily Express for highlighting the crisis after a row over the treatment’s cost – funded by health services in 23 other countries, including Wales.
Realistic
Two children died while bureaucrats wrangled over funding Brineura enzyme replacement therapy with US pharmaceutical firm BioMarin.
But yesterday NHS England announced an agreement had been struck.
Simon Stevens, NHS Chief Executive, said: “This is another concrete step towards ensuring NHS patients with rare conditions get access to important new treatments.
“The deal reached today is a reminder that companies must be flexible and realistic, because the NHS in England cannot write blank cheques at taxpayers’ expense.”
The decision comes just days before two sets of parents of children with the condition were due to take their battle to the High Court.
Lucy Carroll, 34, and Gail Rich, 41, put their case at a meeting in Manchester last month with the National Institute for Health and Care Excellence which rules on NHS drug use in England.
Yesterday the mothers wept with joy. Gail said: “Now we can just focus on being a family and enjoying every single second.”
She and husband Matthew, from Newcastle, have Nicole, seven, and three-year-old Jessica with the condition. Lucy, of Poynton, Cheshire, said: “It shows the power of parents and the public.”
Her son Ollie, eight, touched the heart of Prince Harry at a children’s charity awards and the prince later privately visited him at Great Ormond Street Hospital.
Sister Amelia, six, also has the condition.
Families visited Health Secretary Matt Hancock last May begging for him to intervene.
Yesterday he said: “I’m absolutely delighted that this treatment will be funded by the NHS.” NHS England said Brineura will be offered to sufferers not currently receiving treatment by Christmas.
Samantha Barber of the Batten Disease Family Association, said: “Brineura offers real hope, but the human cost of this delay cannot be underestimated.”
NICE said the treatment showed “great promise” in slowing down the condition.