Green Light For Judicial Review Into NICE’s Decision On Drug

Four children with a rare genetic disease have been given the green light to head to the High Court as they battle to overturn the decision to not make a potentially life-saving treatment available through the NHS in England.

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

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.

Following the news, the families instructed Irwin Mitchell’s specialist Public Law and Human Rights team. The children have now been granted permission for a judicial review to be held into legality of the decision not to approve the use of the drug. Their legal team believe the decision is unlawful on several grounds and is also a breach of human rights legislation.

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.

Find out more about Irwin Mitchell's expertise in handling public law cases

Cheshire Family

Oliver Carroll, who is eight, and his sister Amelia, six, from Poynton, both have a diagnosis of Batten disease.

Before commencing treatment Oliver had already lost his ability to walk and talk but the treatment has proved highly beneficial to him. His health is now stable and he is virtually seizure free, whereas he used to experience multiple seizures on a daily basis. 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 started the treatment before symptoms emerged and is able to attend mainstream school and enjoy dance, gymnastics and swimming.

Their parents Lucy and Mike Carroll, aged 34 and 35, instructed Saoirse de Bont at Irwin Mitchell’s Manchester office.

Expert Opinion

“Lucy and Mike are understandably devastated by the decision taken earlier this year and initiated legal proceedings after our analysis concluded that the process used to reach that decision may have been flawed.

“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 client’s 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.” Saoirse De Bont - Senior Associate Solicitor

Lucy set up the Ollie’s Army initiative to support the children.

She said: “Our children mean everything to us. We remain determined to fight so that Ollie and Amelia receive the treatment that they need. It is heart-breaking to think that individuals have made the decision that our children should not have access to the only treatment that could save their lives. 

“We continue to hope that the NHS, NICE and BioMarin will work together to ensure that all children with Batten disease in this country will have access to this life-saving treatment.

“We would also like to thank all those who have supported the Ollie’s Army initiative for their kind and generous donations and support.  Without them we would not be in this position to be able to fight not just for Ollie and Amelia but for all children with Batten Disease in England. We will be forever grateful.”

The Carrolls initiated the legal process in April 2019 and issued proceedings in the High Court in May. The Riches subsequently issued proceedings and the two cases are being heard together in October 2019.

For more information visit the Ollie's Army website

Ollie’s Army Battling Against Battens has been set up to bring the much needed awareness of the ultra-rare Batten Disease. Ollie’s Army fundraises to support research projects and support systems so that other families do not have to endure the same unfortunate experiences as the Carroll family have had in the past. The family share the reality of living with Batten Disease via their Facebook, Twitter and Instagram pages. For more visit the Ollie's Army Battling Against Battens Facebook page

Newcastle Family

Seven-year-old Nicole Rich and her three-year-old sister Jessica have Batten disease and have had access to cerliponase alfa. One of them was granted access via a sibling trial and the other as part of the extended access programme from BioMarin.

Nicole was diagnosed with Batten disease at four. Prior to starting the treatment she was showing symptoms, including seizures, speech problems and unsteadiness on her feet. Since starting treatment she has not shown any additional symptoms and loves to go horse riding.

Following Nicole’s diagnosis Jessica was tested for Batten disease despite showing no symptoms at all. Following the treatment, Nicole has shown no further symptoms and Jessica is yet to develop any signs of the condition.

Jessica is the youngest child in the country currently on treatment and still showing no symptoms. She starts mainstream nursery this September and is proving that with early diagnosis and access to treatment, this drug has the possibility of being her cure.

Their parents Gail, 41, and Matthew, 32, from Throckley, have previously met with Secretary of State for Health and Social Care Matt Hancock to lobby for the treatment to be available for free on the NHS.

The couple instructed Kirsty Stuart at Irwin Mitchell’s Newcastle office.

Expert Opinion

“We have a range of concerns regarding how NICE ultimately made its conclusion regarding this drug – the only treatment currently available for those who suffer with Batten disease.

“Our client considers that the decision that has been made is tantamount to putting a price on the lives of these children and they think that is simply unacceptable. We are hopeful that this judicial review will mean the decision-making process will be thoroughly reviewed and ultimately a better outcome can be reached so that these children can continue to access this hugely beneficial treatment.

“Our clients simply want what any parent wants – the best possible life and future for their children. They would rather not be in this position, but sadly they feel like they have been left with little choice.” Kirsty Stuart - Senior Associate Solicitor

Gail said: “Our children mean the world to us and all we want to do is help them achieve everything they can in life. This treatment has been hugely effective and has given us real hope for the future, so it is difficult to take that it has not been recommended for use here.”

“How can it be available in 23 other countries but not here? Something has to change and we think that NICE need to show some compassion and urgently rethink how they came to this conclusion.”

The Nicole and Jessica Rich Foundation was created by the family to fund research into Batten disease and support the girls as their needs change. For more information visit the Nicole and Jessica Rich Foundation website

Alternatively visit the Nicole and Jessica Rich Foundation Facebook page where Gail and Matthew write about their experience living with their daughters’ condition and they hope it will help others who may be going through similar experiences.