The astonishing story of how the NHS is spending £100 million on the WRONG DRUG
. . . egged on by a bunch of headline-grabbing, shameless politicians. The victims? Grace and thousands of cystic fibrosis sufferers like her
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 bargainbucket 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.
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. 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 lH 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 t hen 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: ‘ I n 2015, when i t 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 presi dent 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 t his, i n 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.
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 t ake t he 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 t he deal is t hat 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.’