A BRAVE young girl from Essex who is at risk of brain damage as a result of a rare condition has lost a High Court bid for a drug to be made available on the NHS.

Cait Cotter, from Chelmsford, suffers from the inherited condition PKU, which inhibits the body's ability to metabolise protein and could lead to "profound and irreversible brain damage" unless she adheres to a strict diet.

The 11-year-old, who is treated by a consultant at Great Ormond Street Hospital in London, successfully underwent a trial of the drug Kuvan, which allows her to increase her food intake and reduces her risk of brain damage.

However, she has not been able to continue with the treatment because it is not made available by the NHS.

Through her mother, Natasha Cotter, she challenged the decision of the National Institute for Health and Care Excellence (Nice), the body that recommends which treatments should be provided by the NHS, to assess the drug on its standard basis, rather than using a more specialised process.

Her lawyers argued at a hearing in January Nice's decision to appraise Kuvan through its standard technology assessment, which has an upper cost-effectiveness limit of £30,000, rather than its highly specialised technology (HST) process, with an upper limit of £100,000, was unlawful.

But in a ruling on Friday, Mr Justice Cavanagh dismissed her case, saying Nice was entitled to reach its decision in the way that it did and it was "not irrational".

Justice Cavanagh said: "In an ideal world, there would be no restrictions on the availability on the NHS of very useful drugs such as Kuvan, but we do not live in an ideal world, and it is Nice's responsibility to decide which drugs to recommend for use in NHS England, against the background of a finite budget.

"In exercising that responsibility, Nice must have regard to the broad balance between the benefits and costs of the provision of health services in England, and must exercise its functions effectively, efficiently and economically.

"This means, unavoidably, that Nice must make difficult decisions which will disappoint people who have every good reason to hope that a drug or treatment will be recommended for use in the NHS in England."

Cait's lawyers previously told the High Court the distinction between the two processes was "crucial" in her case, as the standard process has a much lower threshold for cost-effectiveness.

Lawyers for Nice said the treatment may have been made available through the standard route but that it was unable to complete the assessment because the manufacturer of the drug withdrew from the process.

Cait's barrister Ian Wise QC said her condition prevents the breakdown of an amino acid known as Phe, which is extremely toxic to the brain.

Standard treatment for the condition is a very restricted diet, which according to one professor leaves patients unable to eat 80% of all normal food - including meat, fish and eggs.

Mr Wise said in court documents: "Because Cait is on this rigid diet, she struggles with persistent hunger and is clinically underweight.

"Despite her strict diet, tests show that Cait's Phe levels are usually at a level which is toxic to her brain.

"Additionally, the burden of managing this diet and her fears of becoming brain damaged causes Cait to suffer from anxiety."

He said the consequence of the decision, made in April 2019, was that the drug - which has been approved for use in Europe since 2008 - would not be approved for Cait and others like her who would benefit from it.

Daniel Stilitz QC, for Nice, argued the decision was lawful and that the issue was not one of cost, as the drug may have been recommended following the standard assessment if the manufacturer BioMarin had not pulled out of the process.

He said BioMarin had claimed the assessment was a "waste of time and money" and that it was not possible for a treatment to be assessed without the manufacturer's co-operation.

Mr Stilitz said at the January hearing: "Had BioMarin continued to co-operate with the process, it is entirely possible that (the drug) would be recommended for use in the NHS.

"Rather, it is BioMarin's unilateral withdrawal from the appraisal process which has precluded the possibility of Nice taking any decision on the drug."

The barrister added: "Nice is of course sympathetic to the claimant, a young person with a serious illness.

"However, it must be stressed that no part of the decision under challenge related to the claimant's personal circumstances in any way, nor could it."