Anguished parents of perilously ill children threatened last night to take to the streets as health chiefs were accused of fudging negotiations and doing a bargain-bucket deal for lifesaving cystic fibrosis drugs.
After a four-year row over costs, one medication was finally approved in October – in what was claimed to be the largest-ever financial agreement of its kind in NHS history.
But talks dragged on for so long the tablets are already ‘obsolete’, say campaigners.
Britain’s 10,000 cystic fibrosis sufferers now face the prospect of years more delay in receiving the therapy they desperately need – while a new drug, dubbed the Holy Grail by researchers, is transforming the lives of patients in the US.
Insiders claim that NHS chiefs, under pressure from politicians hoping to score points in the looming General Election, agreed to a deal that excluded the latest treatment, a Mail on Sunday investigation can reveal. Drug makers Vertex will not even submit the breakthrough tablet, called Trikafta, for UK approval until January 2021, according to documents seen by this newspaper.
Cost assessments are likely to delay the process even further. To add to frustrations, patients in Ireland and Denmark are likely to get the medication by May. Both countries have already done deals with Vertex for Trikafta.
17-year-old Grace Miller, who also has cystic fibrosis, said: ‘One of my best friends got Orkambi. She’s going through a rough patch at the moment, so I’m really happy for her. But that drug won’t work for me, so I’m also a bit jealous. It would be nice to have something too’
Furious campaigner Sharon Cranfield, whose 18-year-old daughter Jessica is living with cystic fibrosis, said: ‘We can’t wait that long [until 2021].
‘Hundreds of people could die in that time – my daughter could be one them. If that happens, we will start to count the numbers and we will hold everyone accountable. We shouldn’t have to, but we will take to the streets and fight so our children get the medicines they need.’
A source close to talks on the fallout from the deal said: ‘They [the NHS] bought the wrong drug. It’s simply an astonishing mistake.’
THE ‘COVER-UP’ DEAL THAT FAILS HALF OF PATIENTS
Just over five weeks ago, the NHS announced a landmark deal to provide a different cystic fibrosis drug called Orkambi to thousands of British patients – and it was, on the face of it, tremendous news.
Campaigners – patients with cystic fibrosis and their families and supporters – fought hard for access to the medication, which helps slow the decline caused by the genetic disease.
And senior politicians wasted no time in letting the public know their part in breaking the deadlock. ‘The proudest moment of my career and a joyous day for patients with cystic fibrosis,’ proclaimed Health Secretary Matt Hancock in a tweet.
In a video clip, also shared on social media, he hugs a woman in a hospital corridor as she fights back tears.
‘I can’t thank you enough. Matt, you’ve completely changed my life,’ says the woman – we learn her name is Carlie Pleasant, and that she is a 29-year-old cystic fibrosis patient and mother-of-one from Kent. Hancock later tweets a still from the video, adding: ‘After a long, busy week, this’ll keep me smiling all weekend.’
A day later, Labour leader Jeremy Corbyn appeared on daytime chat show This Morning with nine-year-old cystic fibrosis sufferer Luis Walker and his mother Christina – appearing to boast that, in fact, it was he who saved the day, after former Prime Minister Theresa May had failed take action. The Labour leader claimed when Luis wrote to him in August asking for help, he ‘took the case up’ and lobbied the Government.
‘And you know what, it seems to have worked,’ concluded host Richard Madeley. ‘[Orkambi] is going to made available to patient in the next 30 days. Thank you Mr Corbyn… it’s a big achievement.’
But, amid the political points-scoring and camera opportunities, 17-year-old Grace Miller, who also has cystic fibrosis, has a different recollection of the day.
‘It was like the worst case of FOMO ever,’ says the A-level student from London, using the slang term for ‘fear of missing out’. She adds: ‘One of my best friends got Orkambi. She’s going through a rough patch at the moment, so I’m really happy for her. But that drug won’t work for me, so I’m also a bit jealous. It would be nice to have something too.’
Earlier this year she was forced to put on hold her plans to study medicine. Illness, hospital stays and missed school meant she had to drop an A-level.
Grace is currently in hospital receiving treatment.
FINALLY IT FEELS LIKE I CAN TAKE A DEEP BREATH
Life-changing: US student Claire Tinsley, who is on Trikafta
Student Claire Tinsley, who has cystic fibrosis, has a simple, yet eloquent way of describing her wait for drugs that would help her.
‘It felt like I was holding on to a rope, and hoping eventually that someone would pull me up.’
That moment came in October, a week before Claire’s 26th birthday, when she became one of the first patients in America to receive Trikafta. She said: ‘For the most part of my childhood I was pretty healthy.
‘Having cystic fibrosis was just another fun fact about me.’
But from the age of 16, her health took a turn for the worse.
‘When it’s that bad, it feels like I can’t take a full breath. You’re winded by doing very basic activities, like walking or even doing housework.
‘It’s scary, especially as I know people with cystic fibrosis who have died in their 20s.’
Within four days of taking Trikafta, Claire noticed a difference. Tests show that her lung function has already improved by as astonishing 20 per cent.
‘It’s been life-changing. I feel like I can breathe again.
‘So it’s hard to know there are people out there who aren’t going to benefit right away.
‘It’s three pills, every day.
‘Part of me wishes I could share my stash.
‘I just hope, once people see how much of a difference it makes, everyone will get it.’
Her mother, Emma, 50, says: ‘I was already heartbroken when I realised Grace wouldn’t benefit from Orkambi. It only works for people with certain types of cystic fibrosis – fewer than half of patients – and, unfortunately, she isn’t one of the lucky ones. Now we’re seeing stories on Facebook from people on the new drug, Trikafta.
‘People who are really sick are able to do normal things they haven’t been able to in years – like walk up stairs, take a deep breath, or sleep on their back. It seems to happen in days for some.
‘I just feels like there is something historic going on across the Atlantic.
‘It’s so devastating for Grace, because the drug would help her, but she could be years away from getting it.’
WONDER DRUG’S STUNNING EFFECT
Trikafta was approved for use in America on October 21 – just three days before the NHS gave Orkambi the green light.
The new drug had produced ‘stunning’ results in trials and in a record time of three months, US health watchdogs the Federal Drug Administration had assessed the data and given doctors the go-ahead to start prescribing. While Orkambi simply holds the disease at bay, Trikafta brings about an astonishing reversal.
It dramatically improves lung function – four times as much as Orkambi – and overall health in patients. And while Orkambi works for fewer than 45 per cent of cystic fibrosis patients, Trikafta is effective in 90 per cent of cases. Experts anticipate almost all patients on Orkambi will switch to the newer drug, once they can get it.
But the news went largely unreported in the UK. A source who has been close to the negotiations said: ‘It’s hard not to question the timing. The way the two stories broke so close to each other, patients might have assumed Trikafta was part of the British agreement, but it isn’t.
‘The deal is a fudge, because it excluded an option for Trikafta. And politicians didn’t make that clear at all. It all feels like a cover-up – or deliberately misleading at best.’
Sharon Cranfield, 54, agreed: ‘Matt Hancock gave people false hope. On social media, he gave the impression that the deal they’d done for Orkambi paved the way for Trikafta. He said the deal was great value for the NHS, and that it meant he was getting medicines to those who need it.
But, in reality, they’ve done a bargain bucket deal for an old drug. And the only reason they’ve been able to do that is that there is a new, much better one already out there.
‘This is just politicians playing games with our children’s lives.’
IT’S THE TREATMENT WE HAVE BEEN WAITING FOR
FOR a new drug to be offered to UK patients, it must first be given a licence by the European Medicines Agency. It can then be assessed by The National Institute for Health and Care Excellence (NICE), which makes a recommendation on cost-effectiveness based on a number of factors, including trial data showing how well the treatment works.
Finally, acting on this advice, NHS England then makes a financial agreement with the firm that makes the drug. UK health chiefs and Vertex began talks on a deal for Orkambi in 2015, when the drug was first licensed.
Publicly available documents show that, all along, the drug company were proposing something new: a pipeline or portfolio deal.
This means the NHS would agree to paying a single price per patient, per year that would cover all existing medicines including Orkambi, a newer version called Symkevi, as well as those being developed – namely Trikafta, simply referred to as ‘triple therapy’, as it contains three active ingredients.
It has been reported that Vertex was demanding £100,000 per patient, per year. The Mail on Sunday has been told the amount was, in fact, closer to £50,000 – for Orkambi and the then forthcoming triple therapy, too. NICE and NHS England would agree to no more than £12,000 per patient, per year, sources have said. Neither side were prepared to budge. And by July last year, talks had stalled – somewhat acrimoniously. Many clinicians were similarly circumspect about the value of Orkambi.
As Dr Ian Balfour-Lynn, consultant in paediatric respiratory medicine at Royal Brompton Hospital, said: ‘There’s a lot of hype about Orkambi. For some patients it makes a difference, but it’s not a wonder drug. Vertex was probably asking too much, considering the modest benefits.’
The hype was no doubt bolstered by UK charity The CF Trust’s Orkambi Now campaign, and launched in 2017. Some in the medical community called it ‘a mistake’ to push the drug when by then there were the newer, more effective medicines on the horizon. The triple therapy, then at trial stage, was already creating huge excitement.
An insider in cystic fibrosis treatment and research told this newspaper: ‘In 2015, when it was launched, Orkambi was the best there was. But the triple produced stunning results. It improves lung function dramatically, reduces illness and is well tolerated. It’s the one we’ve all been waiting for.’
PRESSURE MOUNTS TO DO A DEAL… ANY DEAL
In March this year, amid growing anger from campaign groups, health chiefs and Vertex president Dr Jeffrey Leiden were hauled before frustrated MPs to explain themselves.
The NICE process, complained Dr Leiden – who, it was revealed, pocketed roughly £13 million in pay last year – wasn’t coming up with the right figure, considering the promise its forthcoming drugs were showing.
Following this, in May, both Vertex and NHS England wrote to Health Select Committee chairman Dr Sarah Wollaston.
In key letters, seen by The Mail on Sunday, both said an agreement would soon be made – but, in an astonishingly short-sighted move, that the triple therapy had been ‘taken off the table’ in talks.
NICE, which until this point had been key to the process, was not present at three of five key meetings prior to this decision.
Then something even more unexpected happened.
False hope: Matt Hancock’s tweet, with patient Carlie Pleasant
In September, Jeremy Corbyn launched Labour’s Medicines For The Many policy to create cheap, generic copies of expensive medicines, such as Orkambi.
He proposed using a legal loophole, known as a Crown use licence, to break patents held by pharmaceutical giants, to ‘save the NHS money, and save lives’.
The move was widely condemned by NHS mandarins, manufacturers, industry bodies and patient groups alike – as it would, undoubtedly, drive drug developers away from Britain.
But, as one political source who had been involved in the Orkambi talks, explains: ‘Corbyn’s move was pivotal, and put the issue on the radar of senior Government officials. Orkambi had become highly political and there was a pending Election.
‘I think by this time, too, everyone was exhausted. They made the only deal they could.’
The price ultimately agreed between NHS England and Vertex in October this year is confidential – and may never be made public, as it is considered commercially sensitive. But it is rumoured to be a two-year contract, paying in excess of £10,000 per patient, per year.
More than 5,000 patients are expected to take the drugs, amounting to around £100million over two years. But the deal specifically excluded any financial agreement on Trikafta.
In that statement, NHS England claimed ‘a binding condition of the deal is that Vertex will submit its full portfolio – including in due course its new triple therapy – to NICE for comprehensive appraisal.’
But in published transcripts of conversations between Vertex executives and investors, the company says it doesn’t intend to hand over the data on Trikafta until January 2021.
This assessment could take months and only then will the NHS be able to start negotiating again with Vertex, a process that could – if history repeats itself – take a long time.
TIME: A LUXURY MANY JUST CANNOT AFFORD
For many of those with cystic fibrosis, further delay is an unaffordable luxury. Experts claim that, during the wait for Orkambi, more than 270 patients died who might have lived had they been able to access the drug.
That is more than just a number to Grace Miller and her mother Emma. ‘For the last few years, I’ve stopped looking forward to things because I just assume I’ll be ill or in hospital,’ says Grace. ‘Sometimes, it just feels so hopeless. And I’m one of the lucky ones, because I’m relatively healthy. I know people with cystic fibrosis who can’t wait. It’s unfair some people are getting Trikafta and some aren’t.’ Emma adds: ‘Grace has had to give up a lot because of cystic fibrosis, but she still moves forward. If the system worked properly, she would be able to go to university, and get on with her life. If she doesn’t get the drug, I don’t know what will happen.’
Sharon Cranfield’s daughter Jessica was given special early access to Orkambi after she fell gravely ill two years ago.
She is hoping Vertex will agree to a similar ‘try before you buy’ scheme with Trikafta.
Sharon says: ‘Without Orkambi, I’m convinced Jessica would be dead. But we still live with the fear that the next cold will be the one she can’t get rid of.
‘Orkambi and Symkevi are now obsolete. It’s a matter of life and death that she, and everyone else gets Trikafta.’
I fear this fiasco is the shape of things to come
By Barney Calman, Health Editor for the Mail on Sunday
Barney Calman, Health Editor for the Mail on Sunday
If Matt Hancock is ever looking for a career after politics – and who knows, he may well soon be – he’d make a great used car salesman. Orkambi is a four-year-old drug. It was effectively replaced a few years back by a newer version, Symkevi, which is less likely to mean patients are hospitalised due to side effects. But both pills were polished up and presented as if they were the latest, greatest thing by Mr Hancock and NHS England.
Only a few miles on the clock, one careful owner. Just don’t look under the bonnet.
The announcements were classic political sleight of hand – designed to distract attention from the real action that was going on across the Atlantic.
But as patients in America share, via social media, the miraculous effects of the newest drug, Trikafta, this tactic won’t last long. Those who already realise what’s happened are, justifiably, furious. And many more will soon be waking up to the reality that the NHS has bought the ‘wrong’ drug.
Mr Hancock knew full well just how important Trikafta was.
At a Health Select Committee grilling in July, as chairman Dr Sarah Wollaston demanded he give them a deadline for talks over cystic fibrosis drugs, he said: ‘It’s not just about Orkambi, it’s also about the triple… the next therapy.’
So why did they agree to ‘take it off the table’? Was the sudden cave-in, after standing firm for years, simply to score political points? It seems likely, although the fine detail of these deals are kept a commercial secret.
In this context, the tweets showing Mr Hancock hugging cystic fibrosis patients seem particularly disingenuous.
And there is a bigger issue at stake. Senior figures in NHS policy have spoken to me about concerns that this saga signals the shape of things to come.
Britain pumped a record £34.8 billion into drug research in 2017, which included Government investment and charity funding. This might seem a lot, until you discover that Vertex alone spent more than £8 billion developing medicines over the past two decades, and is still £3 billion in the red because of it.
We invest in science, but not enough. And so private drug firms, which employ some of the best and brightest minds, fill the void. New players, such as Vertex, behave more like Silicon Valley tech disruptors than traditional pharma giants.
They want to innovate, to create game-changing treatments – but they also exist to make a lot of money. And they are, by all accounts, ruthless negotiators.
The breakthrough in targeted therapies for cystic fibrosis is just the start. We could soon see similar breakthroughs for haemophilia and the deadly brain tumour neuroblastoma.
These conditions are rare, but still affect thousands.
The drugs may need to be taken long-term and will undoubtedly be expensive.
Orkambi wasn’t worth what Vertex was demanding, most experts agree. But NICE came up with the figure of roughly £10,000 per patient per year for every drug it had to offer, including ‘the triple’ which everyone agreed was showing huge promise.
Pulmozyme, a much older drug for cystic fibrosis, which helps with some of the lung symptoms, costs a similar amount, apparently. No wonder Vertex wouldn’t agree to this kind of figure.
And it knew what it had in Trikafta. NICE’s inflexibility and claims of a ‘global reputation’ seemed just as arrogant.
During talks, Vertex was repeatedly and publicly accused by our politicians and health chiefs of ‘poor practice’ for not ‘complying’ to the NICE process, and told it ‘ought to know better’. And then Dr Wollaston – who really should know better – Jeremy Corbyn and others kept bringing up the threat of stealing the recipe for Orkambi so we could make it ourselves.
It was not the best negotiating tactic, as Vertex has a monopoly on new cystic fibrosis medicines so hold all the aces.
As we speak, the entire NICE ‘mechanism’ for evaluating new drugs is being reviewed, in a process expect to be completed in January 2021.
Funnily enough, this is when Vertex plan to submit Trikafta for appraisal.
And what if, once again, it asks for £50,000 per patient per year, as it has in the past?
Our team has been outplayed, and out-negotiated at every turn so far. New approaches are needed if we are to stay astride of the latest healthcare innovations. Because it’s not dodgy used cars that these cystic fibrosis patients have been lumbered with. It’s outdated drugs for a condition that kills them before middle age.
Sadly for some, whatever happens next, it will all be too late.