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Children First

Medicine Balls

Phil Hammond

Monday, 29 April 2019

AdobeStock_229330224_children_first_blog.jpgThe Children’s Act 1989 was supposed to make the welfare of our children paramount, trumping all other considerations. So why is child health in the UK still so poor, and getting poorer? By not putting our children first, we are fouling our nests on a grand scale and destroying our futures. 

A care system with limitations

No health service can afford the highest quality care for all its citizens, even if it claims to be universal. This truth was first observed by Nye Bevan in 1948. “We shall never have all we need. Expectations will always exceed capacity.” In 1966, Enoch Powell expanded on this theme as health minister: “The vulgar assumption is that there is a definable amount of medical care ‘needed’, and if that ‘need’ was met, no more would be demanded. This is absurd. Every advance in medical science creates new needs that did not exist until the means of meeting them came into existence.” In 1972, the epidemiologist Archie Cochrane argued for a thorough cost/benefit assessment of all treatments to ensure they were both clinically and cost effective. Nevertheless, rationing remained a dirty word in an NHS born out of war, derided as immoral and repugnant in the press, denied by politicians and yet carried out in secret. In 1999, Labour grasped the nettle and introduced the National Institute of Clinical Excellence, with the remit to ration new drugs and technologies in England fairly and openly. As its former chair, Prof Michael Rawlins observed: “There always has been, and will always have to be, rationing alongside rational healthcare.”

Fighting for a piece of the cake

20 years on, and the task of carving up the NHS cake is as tough as ever. New cancer drugs are particularly expensive and periodically rejected by NICE. In exasperation at the press outcry, prime minister David Cameron set up a cancer drugs fund to pay for drugs that had either been rejected by NICE or were awaiting assessment. The fund paid out £1.27 billion between 2010 and 2016. In 2017, researchers at the London School of Hygiene and Tropical Medicine published an analysis of this spending. Of the 47 drugs funded, only 18 (38%) improved survival, and then only by an average of three months. The remaining 29 had no benefit and caused significant side effects. The only certainty is that we’re all going to die. The cancer fund cash could have been more wisely spent funding every hospice in the UK for a year-and-a-half.

Aside from the relative ineffectiveness of many new cancer drugs, it is absurd that cancer patients are given an unfair advantage over patients with other seriously debilitating diseases (e.g. arthritis, genetic conditions) because of the Cancer Drugs Fund, which gives some of them “access to promising new treatments while further evidence is collected to address clinical uncertainty”, and “interim funding for all newly recommended cancer drugs, giving patients access to these treatments many months earlier than before.” It makes no moral sense to trade off one disease against another and is clearly unjust.

Children are entitled to special consideration

NICE faces a bigger challenge when new drugs are shown to work, particularly when they are for children, but are still considered too expensive for the NHS. Children should be a special case, at least according to the 1989 Children Act, which states “the welfare of the Child is Paramount" as a legally enforceable principle. The right of children to be protected from avoidable harm should override all other considerations, not just in situations of potential abuse but in access to care, education, nutritious food, sufficient sleep, mental health support and treatments that have been proven to work.

And yet in 2016 NICE rejected the cystic fibrosis (CF) drug Orkambi for general prescription, ruling the price was "unsustainable". Orkambi treats a mutation which around 50% of people with CF in the UK have, and up to 5,000 people in the UK could benefit from it. 96-week data shows that Orkambi can slow decline in lung function - the main cause of death among people with cystic fibrosis - by 42% . But it costs £207,000 a year in the US, and you need gold-plated insurance. In the UK, the manufacturer Vertex has set a price of £105,000 a year, which NHS England says is still unaffordable. Vertex turned down NHS England’s offer in July of £500m over five years for access to Orkambi and its other cystic fibrosis drugs. It has also withdrawn the newer and potentially more effective drug Symkevi from the approval process, which means it will not be available to any NHS patients, who can currently only apply for Orkambi from Vertex on compassionate grounds. The company earned $2.5 billion from Orkambi sales in 2017, and when the drug patent expires, the likely generic cost will be £5,000 a year. In Canada it costs £146,000 a year, and patients have launched a class action against the government to access it.

Making sure treatments are available for rare diseases

The NHS was founded to treat people according to need, and it would be hard to find greater need than children with rare, horrifically debilitating and terminal conditions such as Batten Disease. Because such diseases are rare, there is less financial incentive for drug companies to want to develop effective treatments, especially given that most will fail and so risks tend to massively outweigh rewards. For companies that do succeed, the cost is again likely to be far greater than what the NHS considers reasonable value. In February 2019, NICE decided that the drug cerliponase alfa (Brineura) would not be made available for the treatment of Batten Disease.

There are only 30-50 children living with this disease in the UK, and their average life expectancy is 10. Symptoms begin from around the age of two and can then progress rapidly with the onset of seizures, decline in speech, loss of mobility, involuntary muscle spasms, progressive dementia and visual impairment leading to blindness. In NICE’s own words “substantial benefits with cerliponase alfa in the short term have been shown, and there are likely to be important longer-term effects, although these are more uncertain”. It is the only treatment available for Batten disease and would cost NHS England £20 million per year from a budget of £127 billion. However, the manufacturer Biomarin has refused to budge from its price of £500,000 a year per patient and NICE will not recommend it without more evidence of long-term effectiveness. A compromise would be for the NHS to pay up until the time the drug has been shown to be effective, and the drug company should pay beyond that – as part of a longer-term trial –until evidence of longer-term effectiveness is determined.

Failing to look after children puts future adult lives at risk

The best argument against expensive niche drugs for children is that paediatric care has a randomly capped budget and so funding such drugs for a few would deprive many more of less sexy services such as physiotherapy, speech therapy, occupational therapy, psychology, play therapy, community nursing, school nursing, child protection teams, community child health and CAHMS staffing. The depressing reality is that the Children Act offers children very little welfare protection outside abuse situations. Child poverty in the UK has been increasing continuously since 2011. The number of children living in absolute poverty increased by 200,000 in 2017-18. 30% of all children (4.1 million), were living in relative poverty (after housing costs) in 2017-18, and 70% of children living in poverty were in working families on dismal wages. The four-year freeze on children’s benefits due to end next year will likely see the poverty figures continue to worsen. Clearly, the welfare of children in the UK is far from paramount, despite clear evidence that the future risks to adults (and cost to the state) is clearly defined by the number of adverse childhood experiences they have to endure. These put you at much higher future risk of physical and mental illness, poverty, social exclusion and alienation, suicide, violence (victim and perpetrator), and drug and alcohol abuse. Clearly, we need to move upstream, and fast.

UK child mortality is not only higher than many other countries but is on the increase, both for the neonatal period and for children under five. An excellent review by paediatrician Neena Modi paints a depressing picture. About a third of 10-year-old children are overweight or obese; a quarter of five-year-olds have tooth decay, self-harm among girls aged 13–16 has risen by two-thirds in the last three years and compared with 2015–2016, there has been a decrease in 2016–2017 in coverage of four of the six routine vaccinations at age one and two years, and coverage for Measles, Mumps and Rubella decreased for the fourth year in a row, following previous annual increases over nine years. Over 80% of obese children will remain obese as they grow older, leading to them losing about 15–20 healthy-life years as adults. Teenagers, even if only at the upper end of normal body weight, have a substantially increased risk of premature death in adulthood. Air pollution experienced in foetal life, infancy and early childhood scars lungs for life, increasing the likelihood of chronic respiratory conditions in old age. As Modi puts it: “These are classic examples of societies fouling their own nests, by failing to see the destructive consequences for everyone of not safeguarding child health.” It’s time to put children first.

Vested Interest – Phil works in a Department of Paediatrics. He is also doing two shows at the Edinburgh Fringe www.drphilhammond.com

Author's Image

Phil Hammond

Phil Hammond is an NHS doctor, journalist, author, broadcaster, speaker and comedian. He qualified in 1987 and worked part time in general practice for over 20 years. For the past seven years he has worked in a specialist NHS team for young people with chronic fatigue. He presented five series of Trust Me, I’m a Doctor on BBC2, encouraging patients to be more involved, assertive and questioning. Phil is Private Eye’s medical correspondent; in 2012, he was shortlisted with Andrew Bousfield for the Martha Gellhorn Prize for Journalism for ‘Shoot the Messenger’, an investigation into the shocking treatment of NHS whistleblowers. In 2013 and 2014, he was judged to be one of the top 100 clinical leaders in the NHS by the Health Service Journal. As a comedian, Phil was half of the award-winning double-act Struck Off and Die, with Tony Gardner. He has done five solo UK tours, appeared on several TV shows, and has written five books.
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