90 lives could be lost after delays to wonder drug
CYSTIC fibrosis sufferers have been left devastated and fearful after approval for a wonder drug was delayed by nine months.
Pharmaceutical giant Vertex claimed it needed more time to “collect, analyse and submit clinical and economic evidence”.
The Daily Express, which has been fighting for NHS patients to receive the miracle treatment, fears the postponement will cost 90 people their lives.
The delay comes 15 months after Health Secretary Matt Hancock ordered the NHS’s process to obtain the drug, Trikafta, to be accelerated by a year.
US-based Vertex agreed to submit its Trikafta data by April this year so NHS drugs appraiser NICE could start its six-month probe into the benefits.
Dr Keith Brownlee, a director at the British charity Cystic Fibrosis Trust, said yesterday: “This delay is deeply frustrating.
“Trikafta is a vital treatment that could benefit up to 90 per cent of people with CF.
“We are asking Vertex for a full explanation and to see the steps they’re taking to ensure speedy access to the drug across the UK once licensed.”
Trials have shown Trikafta to be vastly superior to the currently available Vertex treatments Kalydeco, Orkambi and Symkevi, all of which help around 50 per cent of sufferers.
Trikafta helps up to nine in 10 patients, with one UK sufferer revealing that it had increased his lung function from 30 to 90 per cent.
The Daily Express, which first fought for access to Symkevi and Orkambi, has been demanding a swift deal for Trikafta – hailed by Vertex as almost a “cure” for CF.
We have also successfully fought for numerous patients in intensive care to access Trikafta through Vertex’s compassionate-use scheme. The first of these, Nicole Adams, 28, of Belfast, is dancing again, while Darren Waite, 41, can cycle 10 miles, and Michelle Bamber, 37, of Farningham, Kent, is exercising in her garden.
Around 10,500 people in the UK have CF, a genetic illness that clogs the lungs with mucus and affects the pancreas, liver, kidneys and bowel. Only half of sufferers live to the age of 32. Initially a seven-month NHS appraisal had been due to start in January, with hopes of a deal in 2022 at the earliest.
But Mr Hancock accelerated the process by ordering NICE (the National Institute for Health and Care Excellence) to start appraising the drug immediately.
It scheduled Vertex to supply all of its data by last month – but the company now says it will do it in January 2021.
Vertex said: “We want to make our submission to NICE with the most robust data. This timeline will enable us to collect, analyse and submit clinical evidence from trials still ongoing.
“We are confident that we will find an innovative solution for access to Trikafta.”
Sharon Cranfield, 54, from Tadworth, Surrey, whose daughter Jessica, 19, has cystic fibrosis, said: “She is now in hospital for two weeks of intensive treatment due to having recurrent lung infections. This isn’t fair when Trikafta exists and could have prevented this.”
A Department Of Health and Social Care spokesman said: “We are disappointed Vertex have decided to delay engaging with the NICE process, which is vital to getting these medicines to NHS patients.
“We urge the company to play its part in the NICE assessment urgently, so patients will be able to access this treatment at the earliest opportunity.”