How NHS drug chiefs' diktat that terminal cancer is only 'moderately severe' has left thousands at risk of missing vital treatment By Ethan Ennals Published: 01:58 BST, 11 August 2024 | Updated: 01:58 BST, 11 August 2024 e-mail View comments A row has broken out between doctors, drug firms and the NHS spending watchdog over its refusal to give the green light to a lifeline drug for women with advanced breast cancer . Medics hailed Enhertu after trials showed it could extend the lives of thousands with one of the hardest to treat forms of the disease, buying them an extra year or more of life. There was a standing ovation when its trial results were announced two years ago at a cancer conference, while campaigners and patient groups welcomed a breakthrough that offers women precious time with loved ones.
But hopes were dashed last week when the National Institute for Health and Care Excellence (NICE) prevented the drug from receiving NHS funding – a decision the charity Breast Cancer Now called 'a dark day for women with incurable breast cancer'. It accused the NHS of 'failing' patients by 'blocking a vital treatment'. Patients and experts were equally horrified, branding the ruling 'cruel' and 'a huge blow'.
Campaigners in Westminster paint their breasts to send a message that the cancer drug Enhertu should be made available on the NHS Kathryn Hulland, 46, who was diagnosed with breast cancer in 2020, two years after the birth of her daughter, Grace, told The Mail on Sunday: 'Enhertu would give me more time to spend with my daughter. 'I try to make as many memories as possible. We go to the zoo, the beach and to musicals.
But sometimes I'm filled with grief because it hits me that I won't get to see her grow up.' Following the outcry, in an unusual move – as drug negotiations are usually shrouded in secrecy – NICE hit out at AstraZeneca, the British firm behind Enhertu, accusing it of being 'unwilling to offer a fair price'. The firm pointed out that Enhertu – which is believed to cost about £120,000 a year per patient – was available in 18 other European countries, including Scotland.
It also claimed the failure to come to a deal was due to a new NICE policy that brands all terminal cancers as a 'medium severity' disease. However, just days later, the watchdog announced it would allow funding for a new gene- editing drug to tackle the rare blood disorder beta thalassaemia – at a cost of £1.65 million a dose.
So what's going on? Experts say the main problem is a significant change made to the way NICE decides whether new treatments are cost effective. The purpose of NICE is to make sure the Health Service spends its £19 billion drug budget on treatments that can provide the maximum benefit to patients. It does this by comparing the health gains expected from a new treatment to its cost.
This might mean patients taking the drug live longer, but it can also mean patients feel better – or both. A cheap drug that has a significant health benefit is likely to be approved. An expensive drug that provides only limited health benefits will struggle to get funding.
Analysts say that for a drug to get NICE approval it can cost up to £36,000 for every extra year of improved quality of life it provides. However, around a decade ago it became clear this system left cancer patients at a disadvantage. Newer drugs were effective but, according to research into them, extended the life of patients by only one or two years, meaning they were not getting approved.
In 2009 the issue came to a head when NICE came under fire after rejecting a life-extending kidney cancer drug called sunitinib. After political pressure it amended its evaluation system to include an 'end-of-life modifier' that gave more weight – and more money – to drugs that extended the time patients had left. This meant a drug would be approved if it cost up to £50,000 for every extra year of life it provided patients.
But this system also led to complaints because, it was argued, it disadvantaged patients who did not have cancer. Chris Skedgel, a director at the Office For Health Economics research group, said: 'Cancer is unique because, unlike most diseases, doctors can say with some confidence how much time patients have before they die. So patients with severe diseases which left them with a poor quality of life, but were not about to die, were not benefiting from this end-of-life modifier.
There is only so much money available to spend on new drugs, and people felt that cancer was getting an unequal amount.' Nigel Spencer was prescribed IsaPD before the rules changed, so he can still keep taking it to treat his myeloma This issue became pressing in 2020 with a new breed of drugs for cystic fibrosis, a genetic condition affecting 11,000 in the UK that impairs lung function and raises the risk of life-threatening infections. At the time, few patients lived into middle age.
New drugs, such as Kaftrio, improved lung function by up to 90 per cent – with doctors hailing it as 'close to cure'. But it cost about £100,000 per patient per year, meaning it could cost the NHS more than £1 billion annually. Since most cystic fibrosis patients are diagnosed in infancy and are expected to live at least 20 years, they did not benefit from the end-of-life modifier.
There were also worries about how NICE would justify the cost of new gene-editing drugs. These one-time treatments are designed to combat rare inherited diseases by fixing faults in the patient's DNA. Many of these treatments involve a single jab but cost £2million or more.
Foreseeing this issue, NICE scrapped the end-of-life modifier in 2022. It was replaced with a 'severity modifier', which gave weight to how long a patient would have to live with severe symptoms. The change put cystic fibrosis among the 'most severe' illnesses, and it meant Kaftrio became cost-effective, with the drug being approved in June.
Along with the approval of the £1.65 million-a-dose beta thalassaemia drug last week, in July a gene therapy for the bleeding disorder haemophilia B, with a cost of £2.6 million, also got NICE backing.
However, advanced cancer patients, who may live just a few years, did not benefit as their short life expectancy meant their disease was now, according to the drug firms, deemed 'medium severity'. Experts say that drugs for advanced cancer would have to come in at £36,000 per year of good quality life to get approval – which is not enough for Enhertu. Breast Cancer Now described the rule change as 'a deep injustice'.
Enhertu is a combination of two drugs – trastuzumab and deruxtecan – which target a protein called HER2 found in many breast cancer cells. Rachael Franklin, interim chief executive of Breast Cancer Now, said: 'It is unacceptable this promising treatment will be out of reach for women in England, Wales and Northern Ireland. We're hugely concerned the introduction of the severity modifier will lead to other effective treatments for breast cancer being rejected by NICE.
' The controversial NICE policy has also affected blood cancer patients. In June, it was announced that the myeloma treatment IsaPD would not get NHS funding. The combination of three medicines has been shown in trials to stop blood cancer returning for an extra year compared with other available treatments.
But NICE ruled it was not cost-effective under its new modifier system. Nigel Spencer, 65, from north London, was diagnosed with myeloma in 2018. After other treatments failed, the retired British Library worker was given IsaPD.
He said: 'It's given me more time with my wife and daughter – time I wasn't sure I was going to get.' Read More Why do I get itchy lips on sunny days? DR ELLIE has the answer Like other patients prescribed IsaPD before the rule change, Mr Spencer can keep taking it. But he said it was unfair that newly diagnosed patients won't get it.
Shelagh McKinlay, director of research and advocacy at the charity Myeloma UK, said: 'We should not be in the situation where an effective treatment cannot be approved. The system is not delivering.' NICE's policy is also likely to lead to rejections of other cancer drugs in coming years, experts warn.
A University of York study into the number of cancer drugs approved by NICE in the past concluded the new system would be 'more restrictive and far less generous', meaning 'only a fraction of [cancer] appraisals will qualify'. Richard Torbett, chief executive of the Association of the British Pharmaceutical Industry, which represents drug firms, warned: 'The way in which the severity modifier is being implemented runs the risk of a situation where some cancer patients could miss out on late-stage treatments they would have otherwise benefited from before it was introduced.' So what's the solution? Experts say countries using a similar system, such as the Netherlands, provide more money to drugs for patients at the end of their lives.
NICE has vowed to evaluate its process's effectiveness, and experts hope its new system will force drug firms to reduce the price of their treatments further. But time for patients such as Ms Hulland is running out. She said: 'So many people die of breast cancer.
We need more treatments, not less.' Helen Knight, director of medicines evaluation at NICE, said: 'The only guaranteed way to get medicines like Enhertu to the patients who need them is for companies to offer a fair price. It is the first breast cancer treatment NICE has been unable to recommend for six years.
'If AstraZeneca decides it is willing to drop its price to a level that makes Enhertu good value for money for the taxpayer, NICE will consider it under its rapid review process, with a decision potentially within weeks.' The number of cancer deaths in the UK each year has dropped by about a fifth since the 1970s – in part due to new treatments. The amount the NHS spends on medicines has increased by more than £6 billion over the past decade to reach £19 billion a year.
'It's a lifeline I can't reach' Kathryn Hulland, 46, first noticed a lump on her breast in January 2020. The former marketing professional, who lives in Devon, was found to have breast cancer, and had chemotherapy and surgery to remove the tumour. She responded well, but at Christmas 2022 she found a lump on her neck and was told her cancer had returned and spread.
Cancer sufferer Kathryn Hulland, 46, from Devon, and her daughter Grace. She says the drug Enhertu would give her more time with her seven-year-old daughter It was a hard-to-treat form of breast cancer called HER2-low triple negative. Immunity-boosting drugs gave her seizures, so more chemo is keeping her disease at bay – for now.
She said: 'If my chemo stops working, there won't be many treatments left.' But the drug Enhertu would give her more time with her seven-year-old daughter Grace. She added: 'Six months more with her would mean the world.
It's heartbreaking that patients in Scotland can get it, but I can't. It's a lifeline I can't reach.' NHS AstraZeneca Share or comment on this article: How NHS drug chiefs' diktat that terminal cancer is only 'moderately severe' has left thousands at risk of missing vital treatment e-mail Add comment.
