The NHS in England is to offer the first gene therapy for children with a rare genetic neurodegenerative disorder, it was announced today.
It will be the most expensive drug ever evaluated by the National Institute for Health and Care Excellence – with a list price approaching £3 million.
The public body has today published highly specialised technologies draft guidance that recommends atidarsagene autotemcel for youngsters with late infantile or early juvenile forms of metachromatic leukodystrophy (MLD).
The one-off treatment costs about £2.8 million at its list price, but following consultation on NICE’s earlier draft guidance, which did not recommend atidarsagene autotemcel, manufacturer Orchard Therapeutics has increased the confidential discount to the price of the drug.
Clinical evidence has suggested that atidarsagene autotemcel, which is the first treatment for MLD, improves motor and cognitive function in the short term and could correct the enzyme deficiency caused by the disease.
Although cost-effectiveness estimates show it provides substantial extra health and quality-of-life benefits, the extent of these benefits is uncertain and it varies for the different types of the disease.
However, the estimates are now within the range NICE normally considers an effective use of NHS resources. It is thought there are about five children born each year in England with MLD.
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.”
Professor Simon Jones, consultant in Paediatric Inherited Metabolic Disease at Manchester University NHS Foundation Trust, welcomed the news.
He said: “This is enormously welcome news for all families affected by MLD, a progressive, life-limiting condition for which there were previously no approved treatment options. While there are various sub-types of the condition, in its later stages, all forms largely result in children losing their ability to move and speak.
“It is extremely pleasing that this treatment will now be offered to all eligible patients, which is testament to the rigorous clinical trials that have paved the way for this decision, and the world-leading research and innovation capability within the NHS.
“As a Trust, we are delighted to be selected as one of five European centres to begin offering this new service as part of NHS standard of care, furthering our work as a European centre of excellence for metabolic conditions.”
Atidarsagene autotemcel is a single-dose intravenous infusion and is designed to correct the genetic cause of MLD by inserting functional copies of the faulty gene into the patient’s own haematopoietic stem cells taken from their bone marrow or peripheral blood.
The disorder is caused by a deficiency of the enzyme Arylsulfatase-A, which results in the nerves in the brain and the peripheral nerves ceasing to function properly. This causes several symptoms including peripheral neuropathy, muscle weakness, sight and hearing loss, difficulty walking, loss of speech, cognitive decline and seizures.
Children with the late infantile type of MLD, the most common and most rapidly progressing type, are usually diagnosed before 30 months. Their life expectancy is between five and eight years.
Children with early juvenile MLD, which usually starts between 30 months and six years, have a life expectancy between 10 and 20 years after onset.
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