lab technician

NHS roll out most expensive drug in the world for babies with rare disease

The gene therapy treatment, which is used to treat metachromatic leukodystrophy (MLD), is thought to be the most expensive drug with a list price of over £2.8 million.

MLD most commonly forms in babies under the age of 30 months and results in a life expectancy of between five and eight years.

The drug works by removing the patient’s stem cells and replacing the faulty gene that causes MLD. The treated cells are then re-injected into the patient.

NHS Chief Executive, Amanda Pritchard, said: “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.

“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.

“It also shows that while rolling out the world-leading NHS COVID vaccination programme, and caring for people with the virus, the health service is also doing its very best to care for millions of patients with other conditions.”

The National Institute for Health and Care Excellence (NICE) have recommended the drug, which is the most expensive drug it has ever evaluated, for use in MLD patients in its draft guidance.

Known as Atidarsagene autotemcel, the new gene treatment is given to patients in a single-dose intravenous infusion

Helen Knight, programme director in the Centre for Health Technology Evaluation at NICE, said: “The independent committee recognised that MLD is a life-limiting, relentless, disabling and isolating condition, affecting all aspects of patients’ and caregivers’ lives. It also recognised that treatment options for MLD are limited to managing symptoms, and that there is a significant unmet need for disease-modifying therapies for MLD.

“The clinical evidence presented showed that for those children in whom atidarsagene autotemcel was effective, it had a substantial clinical benefit in terms of enabling children to maintain their motor and cognitive functions. We’re therefore pleased that the company has agreed an improved discount to the price of atidarsagene autotemcel which, in better reflecting the uncertainty in the evidence, has enabled the committee to recommend this potentially transformative treatment for children with this condition.”

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