By: Antonia Hoyle
- Cancer Drugs Fund was set up to allow patients to buy pricey medicines
- CDF could’ve been a last-chance saloon for patients desperate for Zykadia
- The drug works by blocking a cancer-causing protein in rare lung cancers
- But NICE said the drug wasn’t a ‘cost-efficient’ use of NHS resources
I don’t know Demi Pestell, but after a few hours together I feel as if we’re friends. Within a few minutes of meeting, she confides hilarious – and unprintable – secrets about a dreadful ex-boyfriend. Cue much laughter.
Given the fact that I know she has a rare terminal lung cancer, it feels like a terrible cliche to say she is ‘so full of life’. I can’t help but think it though. She’s turned up in a stunning body-con dress and heels while I’m in Saturday jeans and ill-fitting shirt – and I feel as if I’m the one who looks unwell.
Yet three years ago, the 36-year-old former diving instructor from Northampton was effectively handed a death sentence when doctors warned she had just weeks to live. Tumours had spread from her lungs to her liver, bones and brain, and her only hope of survival was taking part in a clinical trial for a new drug.
That drug, called Zykadia, transformed her. She had been wheelchair-bound and suffering daily seizures. Today, although doctors can find ‘no trace’ of cancer in her body, she still, apparently, has it – and it could return at any moment. But she is an outwardly healthy-looking young woman leading a normal life.
It seems like a miracle, but experts have hailed Zykadia as consistently performing such works of magic in similarly bleak cases.
Yet this is where the good news begins to end. The lifeline handed to Demi by Zykadia – proven in trials to prolong life by an average of 16 months – is being denied to other patients in the same position.
In January this year medicines watchdog the National Institute for Health and Care Excellence (NICE) rejected the £165-a-day drug for the NHS because of cost.
Nice has since revealed it is looking again at this decision, saying an error was made in the evidence they received. However, the drug is still not currently available on the NHS.
To make matters worse, Britain’s flagship fund to pay for life-enhancing cancer drugs in England is up for review and the approval of new treatments is suspended.
The Cancer Drugs Fund (CDF) was set up five years ago to allow patients to access new and expensive medicines before they were approved for widespread NHS use.
The CDF could have been a last-chance saloon for patients desperate for Zykadia.
The drug works by blocking a cancer-causing protein found in a rare form of lung cancer that typically affects younger people who, like Demi, are non-smokers.
Despite acknowledging its ability to ‘prolong life and delay disease progression’, NICE said the drug, which costs just under £5,000 for a 30-day supply, wasn’t a ‘cost-efficient’ use of NHS resources.
Yet Zykadia was approved for use in Scotland in December by NICE equivalent the Scottish Medicines Consortium. It was also licensed for use by the USA’s Food and Drug Administration in 2014 and by the European Medicines Agency in May last year.
While Demi and others currently on trials for Zykadia will be allowed to continue taking the drug for as long as it is effective, NICE’s current position means others won’t be so fortunate. Asked about this, Demi is unequivocal: ‘People in boardrooms are handing out death sentences by not approving this drug. They are putting a price on people’s lives, and effectively saying those lives are worthless. I’m proof Zykadia works, and don’t understand how cost comes into it – who wouldn’t give everything they have for a few more months with their family?’
Demi was diagnosed with stage four cancer in December 2012 after developing a persistent cough, back pain and coughing up blood. An MRI scan revealed cancer in her liver, bones and brain.
Doctors decided it had originated in her lungs as non-small-cell lung cancer. Of the 39,000 people diagnosed with non-small-cell lung cancer in the UK every year, about five per cent have a type caused by a defect in a gene called ALK, or anaplastic lymphoma kinase.
This produces a faulty protein that causes cancer cells to grow and spread. Sufferers of ALK+ non-small-cell lung cancer tend to be under 50 and, like Demi, non-smokers. Her consultant told her that her cancer was incurable and that without chemotherapy she had just days to live.
‘I was devastated,’ Demi recalls. ‘All I could think about were the things I had left to do, like falling in love, having children and travelling the world. I was only 33. I wasn’t ready for my life to be over.’
By now wheelchair-bound and too sick to work, she endured four gruelling rounds of chemotherapy, but in March 2013 Demi’s oncologist told her that chemo was putting her body under so much stress that one more round could cause heart failure. She was then put on a drug called Xalkori, which works by blocking the faulty ALK protein. Within weeks, the four-a-day pills were having a remarkable effect, with scans showing the tumours on her liver and lungs were shrinking.
‘I started to stand up, and then hobble, and by the summer I was out of my wheelchair,’ says Demi. ‘I could go for walks in the countryside again, and even went to Paris. I started to enjoy life again.’
But Demi’s progress was short-lived as, by spring 2014, the cancer in her brain started to cause seizures that left her unable to walk, write or see properly. ‘I lost consciousness for 90 minutes after one particularly bad seizure, and the doctors told my mum I had two weeks to live,’ she says.
DEMI was handed a lifeline when she was offered a place to take part in a clinical trial of Zykadia at Leicester Royal Infirmary. Crucially, unlike Xalkori, the drug is able to pass through the brain membrane to target cancerous cells there. Two single-arm studies into Zykadia of patients with
One study involved 163 patients with cancer that had spread and who had previously had both chemotherapy and Xalkori. Zykadia was shown to shrink or slow the growth of tumours in 55 per cent of cases.
Outlining its decision to approve Zykadia, the Scottish Medicines Consortium described it as a ‘potent’ drug and ‘strongly’ recommended it. Yet despite describing Zykadia in its initial decision as ‘innovative’ and acknowledging its ability to ‘prolong life and delay disease progression’, NICE said the extent of treatment benefit was ‘uncertain’ and that the data provided by Zykadia manufacturer Novartis had not yet been examined ‘robustly’ enough.
Dr Rohit Lal, a consultant oncologist at Guy’s and St Thomas’ NHS Foundation Trust specialising in the treatment of lung cancer, is ‘deeply disappointed’ by NICE’s initial decision to reject Zykadia.
He claims the gap has widened between England and the rest of the world as far as availability of cancer drugs is concerned. ‘This type of cancer tends to happen to younger people – and in particular those who don’t smoke,’ he says. ‘Many have small children and are still the cornerstone of their family. In those circumstances the extra months of life make a real difference. Zykadia is the only treatment available for patients after Xalkori and the only drug available here that can effectively treat this type of lung cancer once it has entered the brain.’
Demi has been taking Zykadia four times a day for nearly two years. It has been so successful at shrinking her tumours that, at a scan in November, doctors were unable to detect any sign of the disease.
‘My hair has grown back, I’m looking for part-time work and I’ve fallen in love,’ beams Demi. ‘People think lung cancer is a disease that only happens to old smokers – I’m proof this is not the case.’
Swiss drugs giant Novartis claims NICE initially rejected Zykadia because it reviewed it prematurely.
At that stage Zykadia had shown promising phase I and phase II trials but, Novartis believe, the NICE appraisal process is not designed to appraise drugs that haven’t released the results of their phase III trials, and these are due later this year.
Others, meanwhile, say the financially stretched NHS simply can’t afford the new generation of ‘designer’ cancer drugs that Zykadia belongs to and that pharmaceutical companies have a responsibility to sell at lower prices, which vary between countries.
In the US for example, Zykadia costs £4,000 more than here, at £9,000 a month. Drug companies are prepared to negotiate on price – indeed NICE says Novartis offered a discount on Zykadia to the Scottish Medicines Consortium that was not submitted as part of the initial decision review with NICE – but insist costs need to remain high to justify the millions spent on research and development.
NICE also points out that it is told which drugs to review by the Department of Health. It adds that consultations between Novartis, healthcare professionals and the public are ongoing. But to Demi, cost doesn’t come into it.
‘I should be dead by now but I am fit, healthy and alive,’ she says. ‘I am intent on enjoying the present rather than what the future holds. I love my life and have so much to give. How can anyone put a price on that?’
Barbara Jacoby is an award winning blogger that has contributed her writings to multiple online publications that have touched readers worldwide.