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.

Complex reasons exist. Many people live longer with chronic conditions like high blood pressure or kidney disease. They need to be controlled by daily pills.

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.

They can be a source of hope, however they often have astronomical prices.

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.

Healthy people have a gene called SMN1 that produces a protein to protect the spinal nerves which control their muscles. These nerves can die without this protective layer. This causes severe and sometimes fatal weakness in the muscles. Children often die before the age of 2 because they are unable to breath for themselves.

Zolgensma is a single-injection gene therapy that corrects this problem, and allows children to walk, sit and breath as usual.

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

Useful for treating: Spinal muscular Atrophy. This genetic disorder affects approximately 1,300 people in Britain. It robs their muscles of power, often leading to death before the age of 2.

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

Myalepta

This is used to treat Berardinelli–Seip Syndrome, which affects around 1 in 2 million people. It causes significant weight loss and a prominently pronounced muscle dilution.

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

Kymriah

It is used to treat patients with rare forms of non-Hodgkin Lymphoma (a type of blood cancer). It is one of the newer cancer treatments that uses the immune system to fight off disease.

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

Orkambi

It is used to treat Cystic Fibrosis. This life-threatening illness affects 1 in 2,500 British babies. This drug was the first to address the problem.

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’. A year of good health is one QALY.

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.

Zolgensma is approved by the NHS despite its high price. It’s for rare diseases like those that only one in fifty people has.

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 problem led to the establishment of The Cancer Drugs Fund in 2011, a distinct pot of NHS cash to fund 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. The patient would not have survived and it would have been expensive.

‘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.

Nic received the all clear. However, in 2015 she noticed a small lump in her jaw. Tests revealed that it was cancerous.

‘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 had to undergo a nine hour operation to remove her tumour, all lymph nodes and part of her salivary gland. This was just below her 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.’

According to surgeons, they discovered more cancer cells that were expected. There was also a chance for the disease to return at 50%.

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.

Three out of the four cancer-free patients who were treated with this drug in previous trials were left unaffected after four years.

The treatment lasted a year and wasn’t without side-effects — including sore joints. The cancer hasn’t returned five years later.

‘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.’

NICE, however, ruled in 2020 against widespread NHS use of Nivolumab for advanced melanoma. They said there was not enough data to support the expense.

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

Not every patient with cancer who has received expensive, cutting-edge immunotherapy is as fortunate as Nic.

Nicki Hopkins, 46 years old, of Scunthorpe lost her 46-year-old husband Dave to glioblastoma multifere in November.

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. A few weeks later, he was diagnosed as having the most advanced form of brain tumor.

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

Nicki was desperate to have more options after Dave started radiotherapy.

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.

Dave was able to raise tens and thousands of pounds through crowdfunding. However, the cancer grew again within months.

In the UK, physicians suggested Keytruda as an immunotherapy drug. The generic name was pembrolizumab. The immune system is alerted to any malignant cells, just like nivolumab.

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.

Although initial hope was high for the autumn, scans confirmed that the cancer had grown again. Dave passed away in early November.

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. While some people hope to find a cure for their cancer, at worst the drugs will only last a few more 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.’

The U.S. healthcare system which is almost entirely private drives up drug costs.

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.’

According to the Association of the British Pharmaceutical Industry, which represents pharmaceutical companies, a voluntary agreement between the UK government and commercial businesses would limit the NHS’s ability to spend new treatment money and reduce the annual rise in new drug spending by 2%.

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

Nic and other grateful patients find it hard to value their care.

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