Keywords: Decision making, Irrationality
A decision of NHS England to refuse funding to treat a child who suffered from a rare metabolic condition with a drug which would alleviate his condition was irrational.
SB (a 7 year old child) had Phenylketonuria (“PKU”), a rare inherited metabolic condition which inhibited his ability to digest protein. The condition prevented his body from breaking down an amino acid called phenylalanine, which consequently built up in his blood. SB was also severely autistic.
Without treatment, most children with PKU develop profound and irreversible intellectual disability, delayed speech, seizures and behavioural abnormalities. Other adverse outcomes include impaired executive function, reduced processing speed, attention problems, and impaired fine motor skills.
The objective of any treatment is to ensure that blood phenylalanine levels are maintained consistently within a safe range. If levels are consistently above that range, there is a risk of long term cognitive impairment. The link between cognitive impairment, and phenylalaninelevels above the upper limit of the range is well documented.
The standard treatment for PKU is dietary management. This involves restricting the amount of natural protein consumed, often to only 10%-20% of the amount contained in a normal diet, coupled with the taking of a supplement (a protein substitute) to promote normal growth and development. With the exception of fruit and some vegetables, there are few foods that can be eaten without severe limitation.
SB has two siblings who also suffer from PKU, but they are not autistic. In their case, it has been possible to implement a dietary regime that successfully manages the condition. However, the extreme severity of SB’s autism and the way in which it affects his behaviour has made it increasingly difficult to control his consumption of protein and to ensure that he takes his supplements as he should. His parents, teachers and clinicians have tried very hard to manage his diet but despite their efforts his levels of phenylalanine are regularly above the levels that are considered safe.
SB’s father (and litigation friend) sought to challenge on his behalf a series of decisions made by NHS England refusing the application of SB’s consultant for funding to treat him with a drug called Kuvan, which (for some patients) reduces the level of phenylalanine in the blood, reducing the need for dietary restrictions and enabling them to eat more “normal” foods. NHS England had decided not to fund the use of Kuvan as a treatment for children with PKU over the age of 4.
It is well established that individual patients do not have an enforceable right to any particular type of medical treatment from the State and that it is consistent with Article 8 (ECHR) rights for the state to seek to strike a balance between the needs of individuals and the constraints of limited public resources. However, public bodies must act rationally and in accordance with their own policies.
NHS England’s Individual Funding Request (IFR) policy required a clinician to demonstrate that a patient proposed for funding was both:
(1) Significantly different clinically to other patients with the same condition
(2) Likely to gain significantly more clinical benefit (than other patients with the same condition)
In the view of SB’s consultant, if S proved to be responsive to it, as appeared likely, Kuvan would provide a significantly greater clinical benefit for S than for other child patients who were able maintain their phenylalanine levels through dietary control alone, because it would enable the levels of phenylalanine in his blood to be brought consistently within the target range beyond which he ran the risk of suffering long term brain damage.
In response to the funding request, NHS England’s funding panel had agreed that SB was significantly different clinically, due to his inability to implement successful dietary management. They also accepted that, without Kuvan, he would continue to have phenylalanine levels above the target range, which would risk ongoing brain damage. Nevertheless, the panel had refused funding on the grounds of insufficient evidence of Kuvan’s “clinical effectiveness”.
It was held that the panel had both misunderstood some of the evidence presented and had failed to consider the correct question. The panel had evaluated “clinical effectiveness” on the grounds that there was insufficient evidence of long-term improvementin cognition and/or nutritional uptake in response to Kuvan. However, this was a perverse interpretation of “clinical effectiveness” in this context. Instead, the panel should have considered the question of clinical effectiveness on the basis that the target outcome in this case was reduction in blood phenylalanine levels and improved dietary tolerance of phenylalanine. That target was the clear clinical intention of SB’s consultant and there was plentiful evidence of Kuvan’s effectiveness in those respects (as acknowledged in NHS England’s own analysis). Additionally, the panel appeared to have erroneously believed that SB’s current phenylalanine levels were not in themselves hazardous, due to a misinterpretation of the evidence provided by SB’s consultant.
Consequently, Andrews J concluded that the panel’s decision was irrational. The decision to refuse funding was quashed and the matter referred back to the funding panel for reconsideration.
Full transcript available at: https://www.judiciary.gov.uk/wp-content/uploads/2017/08/sb-v-nhs-england-final-80817.pdf