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How does the NHS decide who they pay to treat – and who misses out? 

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The numbers are mind-blowing. In England alone, the NHS spends an average of £52 million on prescription medicines every day. 

Over the past decade, the annual drugs bill has soared from £13 billion to £19 billion.

The reasons are complex; people are of course living longer with chronic conditions, ranging from high blood pressure to kidney disease, that need daily tablets to control them.

But our soaring drugs bill also owes much to the development of ground-breaking new medicines for some of the world’s most deadly diseases.

These can offer hope where there was none, but often come with breathtaking price tags.

Take for example Zolgensma, the most expensive drug in the world, costing almost £1.8 million for a single dose.

As Arthur Morgan’s father Reece, a plasterer from South London, said when the family heard their baby would receive the drug: ‘This is the best possible treatment and will give him the best life he can have — we are so grateful the NHS is here for him.’

As Arthur Morgan’s father Reece, a plasterer from South London, said when the family heard their baby would receive the drug: ‘This is the best possible treatment and will give him the best life he can have — we are so grateful the NHS is here for him.’

In June, doctors at the Evelina Children’s Hospital in London became the first in the UK to use it — treating then five-month-old Arthur Morgan, who has spinal muscular atrophy (SMA), a genetic disease that affects the muscles.

In a healthy person, a gene called SMN1 produces a protein which forms a protective layer around the nerves in the spine that control the muscles. Without this layer, these nerves die, which causes debilitating and often fatal muscle weakness. Many children do not survive beyond the age of two as they lose the ability to breathe for themselves.

But Zolgensma, a gene therapy given as a one-off injection, corrects the fault, enabling children to sit, walk and breathe as normal.

As Arthur’s father Reece, a plasterer from South London, said when the family heard their baby would receive the drug: ‘This is the best possible treatment and will give him the best life he can have — we are so grateful the NHS is here for him.’

The NHS expects around 80 babies a year to be treated with Zolgensma and has negotiated a deal with the maker, Novartis Gene Therapies, to ensure ‘a price that is fair to UK taxpayers’, without disclosing it.

The difficult question is which of these kinds of costly ‘wonder drugs’ the health service can afford to fund.

‘What drugs to pay for is the fundamental economic question the NHS faces,’ says John Appleby, chief economist at The Nuffield Trust, an independent health think tank.

Nic Sanger, 38, from Westgate, Kent, has watched her young children — now aged ten and seven — growing up and returned to work as a midwife thanks to one such medicine, called nivolumab

Nic Sanger, 38, from Westgate, Kent, has watched her young children — now aged ten and seven — growing up and returned to work as a midwife thanks to one such medicine, called nivolumab

UK’s Dearest Medicines

Some of the costliest medicines* available in the UK include:

Zolgensma

Used to treat: Spinal muscular atrophy, a genetic disease that affects around 1,300 people in the UK, robbing their muscles of power and often leading to death by the age of two.

Cost: £1.79 million per single, one-off injection.

Myalepta

Used to treat: Berardinelli-Seip syndrome, a genetic disorder affecting around one in a million people, causing significant loss of body fat and extremely pronounced muscles.

Cost: Up to £800,000 a year per patient (a daily DIY injection).

Kymriah

Used to treat: Patients with a rare type of non-Hodgkin lymphoma, a form of blood cancer. The drug is one of a new generation of cancer therapies that harness the immune system.

Cost: £282,000 per single infusion (although the NHS has since negotiated a confidential discount).

Orkambi

Used to treat: Cystic fibrosis, a life-shortening illness which affects one in 2,500 babies in the UK. The drug is one of the first to target the cause.

Cost: £100,000 per patient per year (although the NHS has agreed a discount — it’s taken as a powder, twice a day).

* List price

 

‘Drug spending has more than doubled in the past 15 to 20 years and the NHS must decide what’s worth the money while all the time drug companies keep inventing costly new medicines.

‘On the face of it, £1.8 million for a single treatment is a lot of money. But if it’s only being used in small numbers of patients and it reduces the amount of long-term care a patient needs, is it any dearer than, say, a much cheaper drug that could benefit tens of millions of people?’

Yet the £1.8 million cost of Zolgensma would cover the cost of treating 52,000 people a year with type 2 diabetes with the cheaper but hugely effective drug metformin, for example.

Zolgensma is just one of the new medicines to have emerged in the past decade that benefit relatively few people — roughly 1,300 people in the UK have SMA — and come at a massive cost.

Others include cancer drugs that harness the body’s own immune system to attack and destroy tumours once thought to be incurable.

Tisagenlecleucel — a type of cancer therapy known as CAR T-cell — got the go-ahead from NHS England in 2018 to treat children with B-cell acute lymphoblastic leukaemia, a blood cancer.

CAR T-cell treatment involves collecting the patient’s immune cells from blood samples to make a ‘personalised’ medicine that is injected back into the body over several weeks.

At £282,000 per patient, it’s one of the more expensive drugs on the NHS and about 30 patients a year will benefit. But trial results have been remarkable — with 93 per cent of patients going into complete remission in one study.

The decision about which drugs to fund on the NHS falls to the National Institute for Health and Care Excellence (NICE) — which uses a complex formula called quality-adjusted life year (QALY) to decide if new drugs are worth the price tag.

Simply, it measures how many extra years of good-quality life a drug will ‘buy’. One QALY represents a year of perfect health.

For example, a £50,000 drug that leads to six months of good health works out at £100,000 per QALY. But if the same drug at the same price led to two years of healthy living, it would be around £25,000 per QALY.

NICE rejects most drugs that exceed £30,000 per QALY, although it does give some more expensive ones the green light provided firms can come up with more evidence that their drugs are effective.

Treatments such as Zolgensma, despite being hugely expensive, win NHS approval because there is no alternative available and they are for diseases that affect fewer than one in 50,000 people.

For highly-expensive cancer drugs that often buy seriously ill patients a few extra months of life, the NICE formula poses a problem — almost all of them bust the budget.

This dilemma led to the creation in 2011 of the Cancer Drugs Fund, a separate pot of NHS money to pay for cutting-edge treatments.

It was meant to be a temporary arrangement but ten years later it’s bigger than ever.

Funding of £50 million in 2011 has soared to £300 million and at least 100,000 people are thought to have accessed drugs through the fund.

But it has faced criticism from, among others, the House of Commons Public Accounts Committee, which in 2016 issued a report saying it was not demonstrating with research that the money it spends actually improves patients’ lives.

‘Cancer treatment has become a growth area and some of these medicines can look quite expensive,’ says John Appleby.

‘But there has to be a degree of flexibility in the cost of new medicines or procedures so that innovation is introduced in the NHS.

‘If you think back to the first-ever heart transplant in 1967, there is no way NICE — if it had been around — would have approved it. It would have cost a fortune and the patient only lived for 18 days.

‘But here we are 40 years on, and heart transplants are routine because surgical techniques have improved considerably — we even do them on babies.’

There’s no doubt that breakthrough drugs can be life-savers.

Nic Sanger, 38, from Westgate, Kent, has watched her young children — now aged ten and seven — growing up and returned to work as a midwife thanks to one such medicine, called nivolumab.

It’s a type of immunotherapy that blocks a molecule called PD-1, which stops immune cells detecting and destroying skin cancer — it costs around £125,000 for a year’s treatment.

Nic was diagnosed with a malignant melanoma — the most dangerous type of skin cancer — at the age of 23, after noticing an irregularly shaped dark mole on the right side of her head.

It was removed and Nic was given the all-clear. But in 2015, she developed a pea-sized lump in her jaw and tests determined that her cancer had returned.

‘I was devastated,’ says Nic, who is married to postman Jon, 44.

‘I was still breastfeeding my daughter and had only just gone back to work after my maternity leave. ‘My biggest fear was that the kids would grow up without me and not remember anything about me. That thought kept me awake.’

Nic underwent a nine-hour operation to remove the tumour and all her lymph nodes as well as part of her salivary gland, just under the jaw.

‘They basically had to peel open my face and it left me quite disfigured for a long time,’ says Nic. ‘The pain afterwards was horrendous.’

Surgeons told her they had found more cancer cells than expected and there was a 50 per cent chance the disease would return.

Nic’s prospects were bleak, until her oncologist referred her to a new trial being run at the Royal Marsden Hospital in London that offered patients with advanced melanoma the chance to try nivolumab to see if it stopped the cancer from progressing.

In previous trials, three out of four patients on the drug were still free of cancer after four years.

The treatment lasted a year and wasn’t without side-effects — including sore joints. But five years on, the cancer has not returned.

‘Six months after finishing treatment I was back at work as a midwife bringing new life into the world, and over the past four years I’ve seen my children start school and celebrate birthdays,’ says Nic. ‘I am so grateful.’

However, in 2020 NICE ruled against the widespread NHS use of nivolumab to treat advanced melanoma, saying there is insufficient data to justify the cost.

Instead, it is only available via the Cancer Drugs Fund — to which doctors must apply on behalf of their patients as a special case.

Certainly not every cancer patient undergoing cutting-edge and expensive immunotherapy has as happy an outcome as Nic.

In November, Nicki Hopkins, 46, from Scunthorpe, lost her husband Dave, who was also 46, to a type of brain tumour called glioblastoma multiforme.

The couple have a daughter, nine-year-old Sydney, and two adult children, Dylan, 24 and Lydia, 21, from Dave’s previous relationship.

In September 2020, Dave started complaining of a ‘flicker’ in one eye and a dull feeling in his head. Weeks later he was diagnosed with the most aggressive form of brain cancer.

‘It was horrific,’ recalls Nicki. ‘We were told that, at best, we could hope for 18 months.’

While Dave began radiotherapy and chemotherapy, Nicki began a desperate search for more options.

She came across a clinic in Germany offering an experimental immunotherapy ‘vaccine’ which unmasked cancer cells so they could be seen and destroyed by the immune system.

With tens of thousands of pounds raised through crowdfunding, Dave underwent treatment sessions in early 2021, but within months the tumour was growing again.

By the summer, doctors in the UK suggested another immunotherapy drug called Keytruda (generic name, pembrolizumab). As with nivolumab, it works by revealing malignant cells to the immune system.

While it is licensed in the UK for other cancers, it was not approved for brain cancer and the couple had to pay £7,000 per injection every three weeks.

But despite initial hopes in the autumn, scans showed the tumour was growing again and in early November, Dave tragically died.

Nicki, who now helps the Brain Tumour Research charity campaign for better treatments, has no regrets but some experts fear patients’ expectations, for new cancer drugs in particular, are often too high. Some hope for a cure, when at best the drugs are likely to buy a few extra weeks.

Karol Sikora, a consultant oncologist and professor of medicine at the University of Buckingham medical school, says: ‘We don’t want to remove all hope but these hopes are being exploited by the pharmaceutical industry, which charges whatever it can get away with.’

‘In the UK, we are effectively paying U.S. prices for many of these cancer drugs because around 75 per cent of all cancer drugs globally are used in the U.S. — even though it only makes up about 5 per cent of the world’s population.’

In other words, the U.S. healthcare system, which is almost exclusively private, helps push up the price of drugs.

The campaign group Missing Medicines, a coalition that lobbies for fairer-priced medicines, says NHS England pays drug firms more than £1 billion a year for three cancer treatments — including Keytruda — which were developed by researchers funded by public money through the Medical Research Council.

‘Drug companies make astronomical profits and always say they need to charge more to recoup their investments,’ says Saoirse Fitzpatrick, a spokeswoman for the group. ‘But the research on these drugs was done in the 1980s and 1990s at UK laboratories funded by public money.

‘That’s the riskiest stage financially — the drug discovery process — and in many cases pharmaceutical companies don’t actually get involved until they know something is already financially viable.’

The Association of the British Pharmaceutical Industry (ABPI), which represents drug firms, says a voluntary scheme between commercial companies and the UK government caps the amount the NHS can spend on new treatments and limits the annual increase in spending on new drugs to 2 per cent.

David Watson, the ABPI patient access director, says: ‘This provides the NHS with absolute certainty of budget.’

Grateful patients such as Nic find it impossible to put a value on their treatment.

She says: ‘The drug saved my life — without it I wouldn’t be here.’

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