Legal Challenge Launched Regarding NICE’s ‘Flawed’ Funding Decision On Batten Disease Treatment

Lawyers representing two children with a rare life-limiting disease have launched legal action against NICE, the government health regulatory body, regarding their decision not to recommend that the NHS fund a potentially life-saving specialist treatment.

Irwin Mitchell’s specialist Public Law and Human Rights team acts for Amelia and Oliver Carroll, aged six and eight, who both have a diagnosis of a fatal neurodegenerative condition known as Batten disease.

Batten disease is extremely rare, and NICE estimates that there are currently around 30 to 50 children living with the condition in the UK.

Oliver and Amelia, of Poynton, Cheshire, currently receive access to a specialist treatment known as cerliponase alfa for the condition as part of a compassionate use programme provided by the pharmaceutical company that developed the drug. The treatment has proven to be extremely effective for both Oliver and Amelia, with it appearing to halt the degenerative effect of the disease in both of them.

As a result of the treatment, Amelia and Oliver both enjoy a good quality of life. Unfortunately, before commencing treatment Oliver had already lost his ability to walk and talk but the treatment has had an incredible impact. His health is now stable and he went from experiencing multiple seizures daily to being virtually seizure-free.

He does not suffer from movement disorders and enjoys attending swimming lessons and going to football matches, and loves spending time with his friends and his siblings.

Amelia, who started the treatment before she developed any symptoms of Batten disease, is a happy, healthy little girl, who loves to walk run, jump and climb. She attends a mainstream school, is learning new skills daily, and attends dance, gymnastics and swim classes.

Notably, this is currently the only treatment for children with Batten disease. Without the treatment life expectancy for individuals with Batten disease is around six to 13 years. With the treatment, it is estimated that individuals could live for 60 years.

However, as the pharmaceutical company has stated that it cannot fund the treatment indefinitely, Oliver and Amelia’s mother, Lucy, was stunned to learn that NICE concluded in February that the treatment could not be recommended for use by the NHS.

The decision was apparently made on the basis that it was too expensive yet there was a lack of information about how the calculations were made to determine that the drug is not cost effective.

Cerliponase alfa is known to be funded by health services in 19 other countries, including Wales.

Now, Irwin Mitchell’s public law experts have applied for a judicial review of the decision, on the basis that it is unlawful on several grounds and also amounts to a breach of key human rights legislation.

Expert Opinion

“How can you put a price on a child’s life? NHS treatment should be provided based on the clinical needs of the patient.”

“Our clients are understandably devastated by the decision taken earlier this year and after reviewing all of the facts we have come to the conclusion that the process used to reach that decision may have been flawed.

“This treatment has made a huge difference to the lives of our clients and it is something they could also access in a host of other countries, including France, Germany, the Netherlands and Slovakia.

“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.” Saoirse De Bont - Senior Associate Solicitor

Find out more about Irwin Mitchell's expertise in handling medical treatment dispute cases 

Irwin Mitchell has instructed Vikram Sachdeva QC of 39 Essex Chambers to act for Oliver and Amelia.

Following the decision taken in February, the Batten Disease Family Association sent a letter to appeal the final evaluation. However, this was rejected in the middle of March.

Lucy Carroll, who also set up the Ollie’s Army initiative to support the children, said: “It has been heartbreaking to see what the children have faced and they have shown such courage through their lives.

“It is devastating to think that an effective treatment is not being made accessible on the NHS, particularly considering the impact it has had on them so far. This has to change and we are determined to fight until both Oliver and Amelia are able to access the life-saving treatment that they need.

“The NHS, NICE and the drug company BioMarin need to come to a reasonable compromise and continue to fund the treatment for these very vulnerable patients. ”

For more information, visit the Ollie’s Army website