Children in England with a rare and fatal genetic condition will soon be able to get the world’s most expensive drug after NHS bosses negotiated a substantial cut in its £2.8m price.
NHS England hailed the deal that will make the “life-saving” therapy, called Libmeldy, available to the small number of babies and young children who have metachromatic leukodystrophy (MLD). Those who receive it will be able to enjoy a normal life, it said.
The inherited neurodegenerative disorder severely damages a child’s nervous system and organs. Sufferers experience progressive disability similar to that caused by multiple sclerosis. Symptoms include spasticity, seizures and difficulty talking, swallowing, hearing and seeing.
The condition is fatal and children diagnosed with MLD usually only live for five to eight years. NHS England said it will make Libmeldy available from this spring after talks with its manufacturer, the drug company Orchard, led to it securing a “significant confidential discount”.
Around four children a year are born in England with the condition. Those who receive the therapy will do so at the Centre for Genomic Medicine at Saint Mary’s hospital in Manchester.
“This revolutionary drug is a life-saver for the babies and young children who suffer from this devastating hereditary disorder and will spare their families untold heartache and grief”, said Amanda Pritchard, NHS England’s chief executive.
“The deal we have struck is just the latest example of NHS England using its commercial capabilities to make good on the NHS Long term Plan commitment to provide patients with cutting-edge treatments and therapies at a price that is fair to taxpayers.”
NHS England said that clinical trials “have shown this treatment will deliver substantial health benefits, drastically improving the patient’s quality of life and life expectancy, giving them the prospect of a normal life”.
Babies and young children who have not yet shown clinical signs or symptoms of MLD will be able to receive Libmeldy, as will those showing early signs, provided that they can still walk independently and show no signs of cognitive decline.
Nicola Elson from Cumbria’s daughter Connie and son Joe were both found to have MLD. They were referred to a clinical trial of Libmeldy taking place in Milan. Connie did not meet the entry criteria and deteriorated. But her brother – now 11 – was treated during a five-month stay in Italy and now attends school and is both a physical and academic match for his peers, his mother said.
“When I found out the treatment was being offered on the NHS I was so emotional and cried happy tears. I’m overjoyed and, having witnessed the suffering my daughter has endured, I don’t want another family to go through this,” she said.
However, Prof Andrew Hill, a drugs expert at Liverpool University, warned that NHS England had struck the deal despite the evidence for Libmeldy’s effectiveness being questionable.
“This is a very high price, but MLD has a very poor prognosis”.
He added: “The main clinical trials used for approval [of Libmeldy] are not randomised. There are many examples of misleading results from other non-randomised trials. So it is not clear if the results are reliable.
“Approving drugs at high prices without properly randomised trials sets a dangerous precedent. Long-term follow up will be required to ensure that this drug is truly cost-effective at this high price.”
