NICE Appraisals: Rare Diseases Treatments

Emma Lewell-Buck Excerpts

Thursday 21st March 2019 Commons Chamber

[Mrs Emma Lewell-Buck (South Shields) (Lab)]

I would like to thank my hon. Friends and neighbours from the glorious north-east, the Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon), for securing a debate on this important topic, and the Backbench Business Committee for granting it.

I want to focus my comments on phenylketonuria, which is classed as a rare disorder and has stark impacts for sufferers. If left untreated, PKU can result in brain and nerve damage, behavioural difficulties, vomiting, tremors and epilepsy, yet some of the impacts can be mitigated to a degree, if only the NHS would allow the drug Kuvan to be readily available.

PKU sufferers cannot metabolise protein and, as such, are kept on a strict diet and take a large number of supplements to ensure that they do not become deficient in anything that we would normally get from the foods they are unable to eat. Many sufferers also need to take an unbelievable number of tablets each day—tablets that are large and difficult to swallow. My constituent Harvey Parker has PKU. He is 15 years old and takes a staggering 65 of these tablets every single day. Although it is a distant memory for some of us, I am sure we all remember how difficult it is being a teenager, with that want to fit in and not stand out. Harvey told me that Kuvan would help him lead a more “normal” life.

Eating out and sharing food with others is an important part of socialising and something that most of us do without thinking, but many places do not cater for Harvey’s needs, and he ends up feeling “embarrassed”, “isolated”, “angry” and a “burden”. He said that when he has been out with his mates on a weekend, he tells his mam and dad that he has not eaten because he was not hungry, when really it was because he could not find anything that he could eat. Harvey told me:

“I don’t really talk to any of my friends about PKU as I get embarrassed and when I’m with my friends with no tablets to take, blood tests to endure or bland, unpalatable foods then I am just Harvey, one of the lads and not that boy with the rare invisible condition that no one has ever heard of.”

It is clear to see how access to Kuvan and the more relaxed diet that would follow could improve Harvey’s life. It has improved the lives of others who have taken it, so why is it being denied to so many sufferers?

Kuvan is used by more than 2,000 patients worldwide and is commonly used in 23 other European countries, but not England. Some sufferers have had access to the drug on the NHS after costly and lengthy court battles, but most are not that lucky. At an estimated annual cost of up to £50,000, the drug remains out of reach for so many sufferers. An adult with PKU’s protein-restricted diet costs the NHS £12,000 per year. Many sufferers get prescriptions for their food, which has a high price tag: a packet of pasta is £7.20, and a loaf of bread is £7.48. Our NHS was created on the principle that good-quality healthcare and medication should be accessible to all and free at the point of need, regardless of income, but time and again under this Government we are seeing the destruction of our NHS by stealth, with creeping privatisation and a rolling back of that principle.

Constant wrangling led to a situation where, four years ago, NHS guidance said that there was not enough evidence to prescribe Kuvan, and it then referred the issue to NICE to see whether Kuvan could be made readily available on the NHS. As we have heard, NICE has two methods of appraising medicine: the highly specialised technology appraisal for rare diseases, and the single technology appraisal route for common diseases. NICE decided to assess Kuvan using the same route as for common diseases, despite the fact that only one in 10,000 people has this illness—hardly “common”. Thanks to campaigning by the National Society for Phenylketonuria, NICE then suspended the appraisal and decided to reconsider, but the new process has yet to be started. However, as other Members have already noted, the HST route is not ideal for rare diseases, and a whole new different approach is needed.

The NHS also decided to prescribe Kuvan, then changed its mind due to costs. In short, it decided that it was too expensive. That is what happens when privatisation and outsourcing are prioritised over patients’ care and wellbeing. Harvey’s mam, Diane, said to me, “How can you put a price on my son’s health?” I sincerely hope the Minister will let Diane and Harvey know.