Would Nicole be in such desperate peril if she had been given the drug that’s already saving lives in the US?
patients. UK doctors may not be able to start prescribing it until 2021, as financial watchdogs who assess drugs are ‘very busy’ at the moment, according to sources.
The drug, called Trikafta, has, however, been available in America since October, after US authorities gave it the green light following ‘stunning’ clinical trial results. In the next few months it will also be available in the Republic of Ireland – just a short train ride away from Nicole but under an entirely separate healthcare system.
Trikafta has been dubbed the ‘Holy Grail’ by cystic fibrosis sufferers and campaigners.
Although it has not yet been passed by the European drug safety body, the company which makes it, Vertex, could bypass the system and supply it directly to Nicole’s medical team. So-called ‘compassionate use’ of new drugs is legal, if sanctioned by doctors in a bid to save the sickest patients.
Nicole’s devoted boyfriend Ciaran McVarnock has also launched an appeal to raise the estimated £20,000 it would cost to buy a one-month supply of Trikafta from America. By yesterday they had almost reached their target.
Yet, while the fund may buy Nicole time, what she and other patients in similarly bleak situations need urgently is a longer-term solution, say her supporters.
Speaking last week to The Mail on Sunday from Nicole’s bedside at Belfast City Hospital, Irish boxing champion Ciaran, 27, said: ‘They’re calling Trikafta the wonder drug. It’s saved lives, and we need to get it for Nicole as soon as possible.
‘I know it’s a complicated situation, and the care she has received is incredible, but this is life and death for her – it makes me so angry and frustrated that the drug is out there and that she can’t get it.
‘We’ve been overwhelmed by the positivity from around the world – we’ve even had cystic fibrosis patients in America offer to send Nicole their Trikafta tablets, saying it’s made them feel so much better they could do without it for a bit, but of course we can’t accept.
‘She needs her doctors to give it to her, and she ticks all the boxes to receive it on compassionate grounds. Nicole is a fighter, she is so strong, but I’m worried about how much fight she has left in her. She is trying to stay positive. We all are. But she needs Trikafta now.’
A BRITISH DEAL… BUT FOR ‘OBSOLETE’ DRUGS
AT THE end of October, the Government announced a landmark deal for cystic fibrosis medicines
– ending a four-year row with the drug manufacturer, Vertex, over costs. The contract to supply the tablets Orkambi and Symkevi was said to be the largest financial deal of its kind in NHS history – potentially amounting to £100million over two years.
But the talks dragged on for so long the drugs are now, since the introduction of Trikafta in America, ‘obsolete’, some campaigners have claimed.
Last month, this newspaper revealed how NHS chiefs, under pressure from politicians wanting to break the deadlock, signed the deal for Orkambi and Symkevi – but not Trikafta, because they were unable to agree on a price for the newer drug.
At present, if a medicine is to be approved for routine NHS use, first the European Medicines Agency has to give it a licence. Then UK prescribing watchdog the National Institute for Health and Care Excellence (NICE) assesses its value – a calculation based on the cost of the drug versus how well it works.
If it brings a significant improvement in quality of life and reduces the amount of other treatments a patient needs, it is deemed cost-effective and given the green light.
Finally, NHS chiefs make a financial agreement with the drug’s makers before it goes to patients.
Publicly available documents show that Vertex wanted NHS bosses to pay a single price per patient, per year, that would cover all cystic fibrosis drugs, as well as those being developed – namely Trikafta, which was showing promise in trials from 2017. Although financial details are confidential, The Mail on Sunday has been told the amount Vertex was asking for was close to £50,000 – for Orkambi, Symkevi, an older drug Kalydeco, and the thenforthcoming Trikafta.
NICE and NHS England would pay only up to £12,000 per patient, per year, sources have said. And not without reason: Orkambi and Symkevi bring about only a modest improvement in health. Many clinicians believed the price Vertex was asking was too high. Vertex argued the figure reflected the gamechanging nature of Trikafta.
NHS chiefs seemed unwilling, or unable, to put faith – and finances – into a drug that wasn’t yet launched. Neither side was prepared to budge and in May, despite all parties knowing how important the new drug was, Trikafta was ‘taken off the table’ in talks.
The result of the impasse is that Trikafta was approved for use in America on October 21, and two days later Orkambi and Symkevi – older, less effective drugs – were approved for UK use.
While they do stabilise symptoms, slowing deterioration, Trikafta improves lung function – and is four times as effective.
And while Orkambi and Symkevi work for less than 45 per cent of cystic fibrosis patients, Trikafta is effective in 90 per cent of cases. Experts anticipate almost all