Hundreds of cystic fibrosis patients become eligible for ‘life-changing' therapy
CF is caused by a faulty gene that affects the production of a protein called CFTR.
Modulator drugs work by helping to make the CFTR protein work effectively.
NHS officials said the treatments can 'transform' life expectancy and quality of life for patients.
There are a number of modulator therapies available but some patients with the rarest forms of the disease have been unable to access them until now.
Nice approved Alyftrek, which is known as a triple therapy, for patients aged six and over for the most common form of CF as an alternative to another treatment called Kaftrio.
And NHS England announced that the treatment would also be made available for children and adults with rare forms of cystic fibrosis, who have not previously been eligible for modulator therapy.
NHS England said the move allows patients with the rarest form of the disease to access the latest treatments, including Alyftrek and Kaftrio, for the first time.
It said that 95% of people with cystic fibrosis in England will now be eligible for modulator therapy.
The new drug, also known as vanza triple, has been found to be as effective as current drug Kaftrio in clinical trials in improving and maintaining lung function in people with CF.
The Medicines and Healthcare products Regulatory Agency (MHRA) approved the triple combination medicine – which is also known as deutivacaftor/tezacaftor/vanzacaftor – for use in the UK, for certain patients with CF over the age of six, in March.
Cystic fibrosis is a genetic condition that causes thick mucus to build up in the lungs and digestive system which leads to breathing difficulties and serious infections.
John Stewart, NHS England's director for specialised commissioning, said: 'This is a major leap forward for hundreds of patients living with the rarest forms of cystic fibrosis, offering fresh hope of a better quality of life.
'Access to a once-daily treatment at home can make an enormous difference to patients and their families – reducing the burden of hospital appointments and allowing children and young people to live more freely and independently.
'For those living with the rarest forms of the condition, this represents the very first time they will be able to access this new standard of care that has been so transformative for many since 2019.
'The rollout of this life-changing therapy demonstrates how the NHS continues to embrace innovation to deliver significant improvements in care for patients across the country at a fair price for the taxpayer.'
Helen Knight, director of medicines evaluation at Nice, said: 'CFTR modulators are already revolutionising the way cystic fibrosis is treated so we're pleased to be able to recommend Alyftrek, the latest of this type of treatment that has been shown to be effective, with significant benefits for people with the condition.'
David Ramsden, chief executive of the Cystic Fibrosis Trust, said: 'Today's announcements are another positive step in the journey to better treatments for everyone with cystic fibrosis – a lifelong, life-limiting condition without a cure.
'It's thanks to the incredible support and many years of campaigning of the CF community, and the work of all our partners, that modulator drugs are now a treatment option for thousands of people.'
Ludovic Fenaux, senior vice president at Vertex International, said the move 'represents a significant milestone in our journey to serially innovate and further improve the lives of people living with this disease'.
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
The Independent
26 minutes ago
- The Independent
Medics issue patient safety warning over union's approach to strikes
Top medics have urged the British Medical Association (BMA) to suspend its guidance advising doctors not to disclose strike plans to employers. The Academy of Medical Royal Colleges said that withholding this information risks patient safety as it makes it extremely difficult for health service leaders to maintain adequate patient care. Resident doctors are set to begin a five-day strike on Friday after pay talks with the government broke down, with the BMA seeking a 29 per cent pay rise. Hospital leaders anticipate having to cancel some operations and appointments, despite NHS England 's mandate to continue routine care, with one A&E in Cheltenham considering reducing services. The health secretary, Wes Streeting, described the strike action as 'completely unjustified', while the BMA argues the government's pay offer is insufficient.
Telegraph
27 minutes ago
- Telegraph
NHS investigation ‘bent the rules' in favour of trans doctor
The NHS investigation into a nurse who complained about a transgender doctor using a female hospital changing room repeatedly 'bent' impartiality rules, an employment tribunal has heard. Naomi Cunningham KC, barrister for nurse Sandie Peggie, said there was a 'pattern' of 'rules being bent and the usual boundaries transgressed' to favour trans medic Dr Beth Upton. She challenged Dr Kate Searle, an emergency medicine consultant at the Victoria Hospital in Kirkcaldy, Fife, that the 'usual rules don't apply when dealing with Dr Upton, do they?'. Dr Searle disagreed but Ms Cunningham pointed to an email she sent to Dr Upton, passing on a message of support from Jamie Doyle, the hospital's head of nursing. The barrister said Mr Doyle was involved in the investigation and 'shouldn't it have been obvious to both you and Jamie that that was a wholly improper message from him.' Dr Searle replied: 'In hindsight, yes.' Ms Cunningham highlighted that Dr Searle was allowed to be present during Dr Upton's interview for the investigation, despite also being a witness in the case. She read an email from Angela Glancey, a clinical nurse manager, in which it was stated that Dr Searle would be interviewed first as 'that way there's no conflict once we speak to Dr Upton'. Dr Searle replied that she did not know the rules but Ms Cunningham said: 'Once again, this is a situation where we see rules being bent and the usual boundaries transgressed for Dr Upton. That's what you want and Angela indulges you.' She disagreed but then admitted she had committed a 'flagrant breach' of an instruction not to discuss the case with others, by speaking with another witness. The tribunal had previously heard how Dr Searle had emailed 19 of the hospital's consultants shortly after the incident to state that Dr Upton 'knows we all support her, and that we condemn the actions of Sandie'. The row centres on an encounter between Ms Peggie and Dr Upton on Dec 24 2023. The nurse experienced a sudden and heavy period and feared that it had bled through to her scrubs. Ms Peggie entered the female hospital changing room to find Dr Upton and challenged the medic's presence. Within hours, a bullying complaint was lodged by Dr Upton and the nurse was then suspended. In May last year Ms Peggie submitted a formal claim to an employment tribunal against NHS Fife and Dr Upton for sexual harassment, belief discrimination and victimisation. The nurse complained of being required to share a single-sex space with someone she believed to be male and being victimised for holding a gender-critical belief that biological sex is immutable. It emerged last week that Ms Peggie had been cleared of gross misconduct by an NHS Fife disciplinary hearing. However, the employment tribunal continues. Dr Searle spoke with Dr Upton on the night of the changing room incident and helped the medic fill out an official report into the matter. 'Flagrant breach' After Dr Searle was questioned about her email to the other consultants, and being present at Dr Upton's interview, Ms Cunningham said: 'Usual boundaries (are) transgressed because it's Doctor Upton, because it's about Doctor Upton. That's right, isn't it? This is becoming a pattern, isn't it?' Dr Searle disagreed but Ms Cunningham highlighted her invitation to an investigation meeting that stated 'you should avoid discussing the case with anyone other than your representative to ensure your confidentiality and that of the other parties involved'. The doctor said she had taken that instruction 'seriously' but she said she had not told Ms Glancey about her email to the other consultants, or passing on good wishes from Mr Doyle to Dr Upton. Ms Cunningham highlighted that Dr Searle had also spoken with the only third-party witness who could substantiate a claim that Ms Peggie had refused to communicate with Dr Upton over a patient. The barrister said the conversation was a 'flagrant breach' of the warning not to discuss the case with others. Dr Searle replied: 'In hindsight now, yes.' The doctor also agreed that a large man in men's clothes should not be allowed to use the female changing room if he self-identified as a woman. But she denied that Dr Upton was 'obviously male', saying she would not have known what sex the medic was 'assigned at birth' unless she had been told. The tribunal continues.
BBC News
an hour ago
- BBC News
NHS consultant spoke to trans row witness despite warnings
An NHS consultant has admitted that she spoke to a potential witness despite being warned not to do so during an investigation into an altercation between a transgender doctor and a nurse.A healthcare assistant was alleged to have witnessed a separate incident involving nurse Sandie Peggie and Dr Beth Upton, days before they were involved in an encounter in a changing room at the Victoria Hospital in Kirkcaldy on Christmas Eve questioning from Ms Peggie's lawyer at a tribunal, Dr Kate Searle admitted that talking to the witness had been a "flagrant breach" of previous warnings not to discuss the she denied claims that she "left confidentiality in ruins" with her actions. Ms Peggie was suspended from work with NHS Fife after she told Dr Upton - a trans woman - not to be in the women's changing rooms. Dr Searle, who was Dr Upton's line manager, became involved with the case on 29 December and helped file a report on the NHS's datex complaints this period Dr Upton also accused Ms Peggie of walking out on treating a patient earlier in December due to the junior doctor's Searle later spoke to a healthcare assistant who was alleged to have witnessed this incident. However, the woman could not remember details of a conversation between Ms Peggie and Dr Upton. Dr Searle said she could not recall whether she had this conversation before or after Angela Glancy - a senior charge nurse involved in the investigation - had spoken to the Peggie's lawyer Naomi Cunningham said if Dr Searle had spoken to the witness before investigators it would be "grossly improper" added: "What possible business did you have making contact with the witness, who was a witness to a live investigation or pending employment tribunal proceedings?"Dr Searle replied "no business".The consultant said she "probably" spoke to the healthcare assistant after Dr Glancy had done so, but could not be sure as "times have blurred into one" during the later admitted that speaking to the witness was a "flagrant breach" of previous warnings not to discuss the case. Dr Searle became emotional after Ms Cunningham named the healthcare worker, saying that the person involved was desperate to avoid any media had to leave the stand at that point, which Ms Cunningham later dubbed "an outburst". The lawyer suggested the emotion was not to do with any concern for the healthcare assistant but out of "a desire to protect yourself."Dr Searle denied this, and NHS Fife's lawyer Jane Russell said Ms Cunningham's questions on the matter "left a little to be desired". Timeline of the Sandie Peggie tribunal Giving evidence for a second day, Dr Searle was again quizzed regarding emails exchanged between senior staff in the aftermath of the Christmas Eve Cunningham focused on one message said to mention avoiding "foot in mouth syndrome" and that not all messages had been provided to the added Dr Searle's search for emails related to the case had either been "surprisingly incompetent or deliberately withholding" the Searle said she rejected the latter Cunningham accused Dr Searle of "turning up the emotional heat" with how she reported the incident in the replied that she had reported the incident as she saw it, with a junior doctor upset about something that had happened at Searle had told the tribunal on Tuesday she considered reporting the matter to Police Scotland as it could be considered a hate Cunningham asked Dr Searle if she would have recognised Dr Upton as trans if she had not been told, and claimed the junior doctor was "obviously male".The lawyer added that the Christmas Eve incident could have been resolved by Dr Upton leaving the changing room, rather than telling Ms Peggie to speak to management about Searle said she disagreed this would have been a suitable way to end the tribunal continues.
