Batten Disease families get green light for High Court challenge over decision not to provide treatment

Four children with the neurodegenerative condition Batten disease have been given permission to bring a judicial review challenge over a decision to not make a potentially life-saving treatment available through the NHS in England.

The children, from Cheshire and Newcastle, have been given access to a specialist treatment known as cerliponase alfa as part of programmes run by pharmaceutical company BioMarin.

The claimants’ law firm Irwin Mitchell said: “The drug has appeared to halt the degenerative effect of the disease in all of them and it is estimated that it could even boost life expectancy from a maximum of 13 years to potentially 60 years.

“However, with BioMarin unable to fund access to the treatment indefinitely, the affected families were stunned to learn that regulatory body The National Institute for Health and Care Excellence (NICE) concluded in February that it could not recommend the drug for use by the NHS due to it being too expensive. The drug is known to be funded by health services in 23 other countries, including Wales.”

The claimants’ legal team believe the decision is unlawful on several grounds and is also a breach of human rights legislation, Irwin Mitchell said.

A full hearing in the High Court is now scheduled to be held on 16 and 17 October. The families have been granted legal aid.

NICE estimates that between 30 and 50 children in the UK have Batten disease.

Saoirse De Bont, solicitor at Irwin Mitchell, said: “We are delighted that the judge reviewing the application agreed that this case should proceed and that there is now a date on which this life-critical challenge will be heard.

“This treatment has made a huge difference to the lives of all four children involved in the case and it continues to be offered in an increasing number of countries worldwide. Our clients consider that it is heart-breaking that the NHS considered that it could put a price on their children’s lives.

“There clearly needs to be a reconsideration of this issue and we are determined to fight hard to ensure that our clients’ voices are heard on this incredibly important matter.”