The parents of a seven-year-old boy are going to the High Court on Thursday 25 May 2017 to challenge NHS England’s refusal to fund life-changing treatment for him.
The boy, who is subject to an anonymity order and is known as “A”, has a genetic and rare metabolic condition phenylketonuria (“PKU”), which means he is unable to metabolise phenylalanine, a substance contained in most protein-rich foods. Untreated, phenylalanine will accumulate in blood in dangerous concentrations and can cause serious cognitive impairment and/or permanent brain injury.
PKU is generally managed through dietary restrictions limiting proteins to restrict intake of phenylalanine supplemented with protein replacements to ensure development and growth is maintained.
Though most patients with PKU are able to manage their diet to avoid protein rich food, A also has autism. His autism is at the severe end of the spectrum, and he is currently non-verbal. Helping A to control his diet to ensure he ingests enough of his protein replacement has been extremely difficult. Due to the sensory needs inherent to his autism, A often rejects supplements and will only accept foods with a high protein content, rejecting substitutes leading to heightened levels of phenylalanine (a protein) in his blood representing a significant risk of serious injury to A.
In the last 12 months, about 60% of A’s blood tests indicated his phenylalanine was outside the target range – despite huge efforts to help A to manage his condition by his parents and school.
The NHS Consultant with responsibility for treating A’s PKU (Dr Santra from Birmingham Children’s Hospital) advised that he should be prescribed a drug known as ‘Kuvan’, which would help A to metabolise phenylalanine, reducing the danger to him and enabling him to lead a more normal life.
Kuvan is a new and expensive treatment which is not routinely commissioned for PKU except for women with PKU in pregnancy. The Consultant applied to NHS England by an “Individual funding request” for funding for A on the grounds his case was “exceptional”. The request was refused as it was not accepted A was exceptional.
A further application, supplemented with further supporting evidence was also refused. NHS England maintained that despite having both PKU and severe autism, A was not sufficiently exceptional to warrant funding.
A’s family then consulted Peter Todd of Hodge Jones & Allen, one of the UK’s leading lawyers in challenging medical funding decisions, who secured funding from the Legal Aid Agency to enable a legal challenge to be brought against NHS England.
As NHS England refused to change their mind, on 11 October 2016, court proceedings were commenced against NHS England seeking permission to seek a judicial review of the refusal of funding. The challenge was brought on the basis that the exceptionality test, used to determine whether a drug not offered under the NHS should be funded on an individual basis, had been mis-applied. The challenge also included allegations of breaches of A’s human rights. NHS England are contesting the claim.
Her Honour Judge Karen Walden-Smith, sitting in the High Court on 23 November 2016, granted A permission for a full judicial review hearing of that decision
An NHS England panel then met again on to review their decision in light of the impending judicial review and this time accepted that A was an exceptional case but refused the funding on the grounds the clinical effectiveness and cost effectiveness of Kuvan had not been established.
A’s treating Consultant believes the clinical effectiveness of Kuvan is well established. NHS England maintain the drug is not cost effective. The new reasons for refusal means the family have had to amend the grounds of their challenge and their application for permission to amend the claim and to rely on new grounds of challenge is listed for hearing by the High Court on 25th May 2017.
Peter Todd of Hodge Jones & Allen says: “A’s family are very disappointed with NHS England. Up until their meeting in December last year they had vigorously denied that A was an exceptional case and refused funding on that basis. However, at their latest meeting they recognised that A is an exceptional case yet refused on new grounds that were never raised before. My clients do not want to take this action, but need A to have the treatment he has been recommended by his NHS Consultant.
A’s father added: “No family should have to fight with the NHS for over 18 months to ensure that their child is given proper treatment. We, as a family, have found the whole process extremely frustrating, unfair and inhumane. Our treatment has been exacerbated by NHS England’s approach. For 18 months’ they have refused treatment based on this not being an exceptional case yet have now admitted it is an exceptional case but, have introduced a raft of new reasons to justify their refusal. It is clear to me they do not want to fund this life-changing drug and are prepared to put my family through a court case. I can only hope that the High Court can do what is right for my son and our family.”
The case is being supported by the patient group “Phedup” which is campaigning for the NHS to fund Kuvan for people with PKU. A representative of the group who is also a parent of a child with PKU said “Children with the rare disease PKU have to eat an extremely restrictive diet virtually devoid of natural protein to avoid permanent damage to the brain. This is an enormous strain on children and their families. The drug treatment Kuvan helps many PKU patients manage their condition and is routinely available for PKU patients throughout Europe. It is completely unacceptable that the NHS have forced a family to bring this legal challenge for a treatment which is available to PKU patients across the EU from France to Romania.”
Notes for Editors
Hodge Jones and Allen
- Hodge Jones and Allen is one of the UK’s most progressive law firms, renowned for doing things differently and fighting injustice.
- For almost 40 years’ the firm has been at the centre of many of the UK’s landmark legal cases that have changed the lives and rights of many people.
- The firm’s team of specialists have been operating across: Personal Injury, Medical Negligence, Industrial Disease, Civil Liberties, Criminal Defence, Court of Protection, Dispute Resolution, Employment, Family Law, Military Claims, Serious Fraud, Social Housing, Wills & Probate and Property Disputes.
- Co-founder Patrick Allen is still at the helm of the firm he co-founded in 1977.
- In 2016 the firm launched Hearing their voices – a campaign to raise awareness and build conversations around the issues and the injustices we might all face.