It has been praised as a panacea with the potential to extend the lives of thousands of people with cystic fibrosis.
But patients in the UK still can't reach Orkambi – even though it was a year ago that Theresa May promised to take action.
The drug, which slows down the deterioration of the lungs, received its European license three and a half years ago, at the time when more than 200 people died from the cruel condition.
However, the National Institute of Health and Care Excellence (NICE) could not reach an agreement with its manufacturer.
The government body and NHS England are at an impasse with the American company Vertex about the drug, which costs an estimated £ 104,000 per patient per year.
On the birthday of the Prime Minister's promise on May 16, furious campaigners marched to 10 Downing Street to share their frustrations.
Mrs. May responded last night by telling the companies & # 39; everything they could do & # 39; to break the deadlock.
And MailOnline has spoken with three cystic fibrosis patients, who begged the parties to make progress.
Hannah Chew née Lindley, 23, Shiloh Howells, nine, and Lorcán Maguire, two, are desperately looking for the chance to try Orkambi.
Three cystic fibrosis patients have begged the NHS for a & # 39; miracle drug & # 39; to fund the crippling situation. Lorcán Maguire, or Downpatrick, Northern Ireland, is two
Shiloh Howells, nine, has lungs so vulnerable that she is expected to have a risky double lung transplant at the age of 12. Her mother, Nichola Howells, said it was heartbreaking to know that Orkambi could prevent surgery
Hannah Chew née Lindley, 23, from Leeds, said CF stole her joy, childhood, and her best friend – she had to watch tragically how her sister, Iona Kesteven, lost the disease. Shown on her wedding day in August 2018
WHAT IS ORKAMBI AND WHO CAN BENEFIT?
Orkambi can delay the decline of lung function – the leading cause of death in people with cystic fibrosis – by 42 percent.
CF is a progressive genetic disease that causes the mucus to become thick and sticky in various organs and to trap bacteria.
It causes persistent lung infections and limits the ability to breathe over time, reducing life expectancy to mid 40.
There are nearly 3,000 people in Britain who have two copies of the F508del mutation – about half the patients – who can benefit from Orkambi, according to the 2014 UK Cystic Fibrosis Registry Report.
There are 2,834 people in England, 243 people in Scotland, 118 people in Wales and 101 people in Northern Ireland.
Vertex refused a £ 500 million NHS England offer for a five-year delivery, the largest offer it has ever made and the talks have failed completely.
The drug is only ever prescribed on rare, compassionate grounds, which according to many charities is too late.
Mrs. May said she was a & # 39; quick fix & # 39; wanted in the ongoing negotiations so that CF patients had access to Orkambi and Symkevi – another drug that treats the cause of the condition but is also unavailable on the NHS.
There have been no resolutions since then.
Vertex reached an agreement with Scotland for access to Orkambi and Symkevi in December, but the battle for the drug is still raging in England, Wales and Northern Ireland.
Yesterday, the Cystic Fibrosis Trust handed over a letter to Theresa May at number 10 Downing Street.
Zoey Jones, the ailing mother of a one-year-old Eva, who has cystic fibrosis, was also there to give 65 roses – a term used by children to help them pronounce the name of the condition.
David Ramsden, chief executive of the Cystic Fibrosis Trust, said: “This tragic situation cannot be continued. Every day the health of thousands of people in this country is deteriorating – and this is damage to their lungs that cannot be undone. & # 39;
& # 39; We ask Prime Minister Theresa May to attend the Westminster Hall debate on Monday, June 10, and to update the house on the next steps. & # 39;
& # 39; My sister died waiting for that medicine & # 39;
Hannah Chew née Lindley, 23, said CF stole her joy, childhood and her best friend – she had to watch tragically enough as her sister, Iona Kesteven, died of the condition.
Hannah Chew née Lindley, 23 said that her and her sister Iona, who are deceased, are best friends
When Hannah looks back on her childhood, she mainly remembers that Iona, who died at the age of 19, was sick. Pictured on vacation
Around the age of eight, Hannah remembers getting hold of the Iona disease and they were put in separate bedrooms. The date is unclear in the hospital
Hannah, from Leeds, is not afraid to admit that her life with CF apart from some happy memories, & # 39; horrible & # 39; has been.
She said: & # 39; Many people mask it that bothers me very much because it has to take it seriously.
& # 39; It has stolen my joy, health and mobility. It stole my childhood and my best friend – my sister. I had to watch her die.
& # 39; There are days when you feel better and there are others who wonder why you are on this planet. & # 39;
When Hannah looks back on her childhood, she mainly remembers that Iona, who died at the age of 19, was sick.
She said: & # 39; We were permanently living in a touch-and-go situation. I remember my sister getting sick all the time and not knowing if she was going to live or die and would live constantly with the fear of – is this the time she dies? & # 39;
In November 2015, Iona's lung collapsed due to multiple lung infections and was quickly returned to the hospital. She died a week later. Pictured on vacation before
Hannah & # 39; s parents tried to make their lives as normal as possible by taking the girls to ballet, piano lessons and theater groups.
She said: & # 39; We even went to the theater to perform from the hospital. We have done our make-up at the hospital. We didn't want to be another number of children in the ward. We minimized that we stayed inside as much as possible. & # 39;
But around the age of eight, Hannah remembers getting hold of the Iona disease and they were placed in separate bedrooms.
People with CF grow insects in their lungs, which are usually harmless to people who do not have the condition, but can easily be transmitted from one person with CF to another, including siblings, and are very harmful.
Hannah said: & # 39; I felt a lot of guilt because I wasn't as bad as she was. & # 39;
Hannah, who married Nathan Chew, 23 (photo) in August 2018, is not afraid to admit that her life with CF, apart from some happy memories, & # 39; horrible & # 39; has been
Iona was known to her advisers as a complicated patient, but Hannah believes it was May 2015, when Iona took a stomach flu on the flight home from the vacation, that things went downhill.
In November 2015, her lung collapsed due to multiple lung infections and she was quickly returned to the hospital. She died a week later.
Hannah, who married Nathan Chew in August 2018, said: & One of the consultants was in shock because they believed she would have survived longer.
& # 39; She can still be here with Orkambi. My sister died waiting for that medicine. It was approved on compassionate grounds two weeks after her death but it was too late.
& # 39; The healthier you are, the more likely you are, so don't wait until they reach a certain point. & # 39;
Hannah, who completes exams to become a music teacher, has won an award for Outstanding Young Person at the Cystic Fibrosis Holiday Fund awards, sponsored by CLC World Resorts and Hotels. She has also collected more than 23,000 signatures on a petition that has now been closed.
She said she is frustrated that CF patients with other mutations receive medication that they can treat – despite being happy for them.
She said: & # 39; It's disgusting. It is the same as saying that we will treat breast cancer, but not lung cancer. Or type 1 diabetes, but not type 2. & # 39;
& # 39; Orkambi can help me avoid a double lung transplant at the age of 12 & # 39;
Shiloh Howells, nine, has lungs that are so fragile that a high-risk double lung transplant is expected to occur at the age of 12.
Doctors say the extremity of her condition is much more severe than that of other CF patients her age after having had a lobectomy – an operation to remove part of her damaged right lung in October 2017.
Her mother, Nichola Howells, 40, said that just knowing that Orkambi exists is heartbreaking, since it could retain what lung function Shiloh has left.
Shiloh, pictured with her mother, Mrs. Howells, and grandmother, has very fragile lungs with a baseline lung function of around 60 percent
Shiloh's lungs are so fragile that it is expected that they will have a risky double lung transplant at the age of 12. Pictured in the hospital where she goes for routine treatment every two months
Mrs. Howells said: & It would mean anything to get Orkambi within that time. With a lung transplant there is no guarantee that you will survive. It is still life threatening.
& # 39; Her condition is not stable and it is frightening. If she had that medicine, within a year, who knows what an improvement it would make.
& # 39; It is a soul that destroys for us. It is money and money for a life. We are angry and frustrated. & # 39;
In March 2018, former Health Secretary Jeremy Hunt visited Great Ormond Street Hospital in London and was welcomed by Silo, who gave him a teddy bear, the hospital's mascot.
But according to Mrs. Howells, as soon as he was told that Shiloh was being treated for CF, he quickly pulled himself into the middle of the price war.
Mrs. Howells said: & while she was in the middle of conversation, he got up and walked away. Maybe he was afraid that we would bombard him with questions. We were all taken back. & # 39;
From the age of six weeks, Shiloh receives routine hospital admissions every three months to undergo treatment to prevent infections. Pictured, with medication
In March 2018, former Secretary Jeremy Hunt visited Great Ormond Street Hospital and was welcomed by Shiloh. But Mrs. Howells claims that he was contemptuous as soon as he heard she had CF
From the age of six weeks, Shiloh receives routine hospital admissions every three months to undergo treatment to prevent infections.
WHAT IS CYSTIC FIBROSE?
Cystic fibrosis is an incurable genetic disease that affects around 70,000 people worldwide.
A defective gene causes an accumulation of mucus in the airways, making it increasingly difficult to breathe over time.
Mucus also blocks the natural release of digestive enzymes, which means that the body does not break down food as it should.
Signs and symptoms:
- A persistent cough that produces thick mucus (sputum)
- Exercise intolerance
- Repeated lung infections
- Inflamed nasal passages or stuffy nose
While healthy people naturally cough, that does not happen for people with CF.
Ultimately, lung function decreases to the point that patients need a double lung transplant to survive.
Source: Cystic Fibrosis Foundation
& # 39; But she wasn't busy with the three-month mark before she got sick, & # 39; said Mrs. Howell. & # 39; So she now goes to the Great Ormond Street Hospital every two months for antibiotics and intravenous treatment, between two weeks and three months. & # 39;
During Shiloh's longest stay, at the age of eight, she stayed for three months while her lung function dropped to 36 percent.
Her baseline lung function is 60 percent, but he used to be around 75 percent.
However, always positive and smiling, Shiloh managed to go to school for a solid two months ago – the longest period in two years.
Mrs. Howells, who has an older daughter, Essence, 20, said: “She is on the edge of the ground but gratefully she picks herself up. But it's getting harder and harder.
& # 39; Her lungs are already very sensitive. To prevent other stories such as hers, patients should receive Orkambi as early as possible.
& # 39; CF is all about prevention. We always throw medication at the patients to make them as comfortable as possible. But Orkambi tackles the genetic side. & # 39;
& # 39; It's a lot for a little man, and he's still dying & # 39;
After just turning two, Lorcán Maguire, from Downpatrick, Northern Ireland, he still doesn't know how different his life is for other children.
Every day he has 18 tablets, two atomizers for antibiotics, another nebulizer for a medicine to dilute the mucus, two quarters of an hour of physiotherapy and vitamins because he is unable to absorb them properly from food.
His mother, Jen Maguire, 34, said: “It's a lot for a little man, and he will still die.
& # 39; The estimate is now in the mid-40s. But Orkambi has shown to add 20 years to current life expectancy.
& # 39; It's such a terrible situation. I wish he had the same life expectancy as his sister.
Jen Maguire, 34, said her family, including her husband Dermot, 45, 11-month daughter, Saorla (left), and son Lorcán, who has CF, are about to move to the Republic of Ireland to Orkambi to get
& # 39; He just thinks his life is like everyone else's. I hope we get Orkambi before he is aware of the situation.
& # 39; It must be so difficult for elderly people with CF who know the government just doesn't care. & # 39;
Mrs Maguire, who also has an 11-month-old daughter, Saorla, said that the situation has ruined Lorcán's childhood – and that is just the beginning.
She said: “He can play with other children, but we constantly use bacterial gel and keep eyes open and ears open.
& # 39; As soon as you hear a cough or smell a cigarette, we throw it away immediately. & # 39;
When Lorcán was diagnosed with CF two weeks after birth, Mrs. Maguire and her husband, Dermot Maguire, 45, a social worker, had barely heard of the condition that would change the lives of the entire family.
They are about to move to the Republic of Ireland, where Orkambi has been given to eligible patients since 2017.
Mrs Maguire said that the situation has ruined Lorcán's childhood – and that is just the beginning. He is currently trying to fight an infection called pseudomonas aeruginosa, which has always been difficult to treat since November 2018. Depicts the two together as they pick out one of Lorcán's medicines
Mrs. Maguire said that she wished Lorcán had the same life expectancy as his sister, pictured
Since January, Orkambi has had a license – but not paid – for two-year-olds in the UK.
WHAT COULD THERESA SAY?
On May 16, Theresa May told MPs in the Lower House that they had a & # 39; quick solution & # 39; had searched for the Orkambi prize row.
She said: “Cystic fibrosis is clearly a terrible, life-limiting condition, and it's good that patients have access to cost-effective, innovative drugs and technologies.
& # 39; The problem has been picked up by members from all over the country and, like the hon. Lady mentioned, there is an ongoing dialogue between NHS England and Vertex, but I am keen to see a quick solution for the negotiations. I understand that several members have asked me to inform me about the problem and I am happy that this will happen. & # 39;
Mrs. Maguire, who has put her PhD on hold because she is too stressed to study, said: & # 39; If nothing happened during the summer vacation, we are considering renting out our house and moving there.
& # 39; We hope that something will happen sooner because it is not ideal. It's scary to know how quickly things get worse with CF. & # 39;
Lorcán is currently trying to fight an infection called pseudomonas aeruginosa, which has always been difficult to treat since November 2018.
He has just returned from a ten-day work placement in a hospital, isolated in a room to prevent him from getting more infections from other CF patients.
Drugs were pumped through an infusion line after antibiotics had not treated the infection for three months. The doctors are still not sure if it went.
Mrs. Maguire said: & # 39; Can you imagine a two-year-old sitting in a room? He screamed and screamed as they put the infusion lines in it and he shook every time.
& # 39; It is worrying because the infection becomes more resistant to drugs, but it causes a lot of damage.
& # 39; Orkambi has been shown to reduce bacteria and hospital admissions by 60 percent. It treats the cause by making the mucus less sticky.
& # 39; It is not good for children to be pumped with these drugs that only treat the symptoms if you could stop something in the first place. & # 39;
MailOnline contacted NICE and Vertex for comments, both of whom said the talks are ongoing.
Vertex said in a statement: & # 39; Vertex is in talks with NHS England and NICE. We cannot say anything further at this time. & # 39;
NICE said: & # 39; We would welcome re-engagement from Vertex. Vertex, NICE and NHS England met in March and agreed to continue the discussions. & # 39;
For more information about cystic fibrosis and the fight for Orkambi visit the Cystic Fibrosis Trust.
WHAT IS ORKAMBI?
Orkambi is a drug approved for use by people in the UK with cystic fibrosis, but it is not offered on the NHS except in extreme conditions.
The drug is targeted against the F508del gene mutation, which affects approximately 50 percent of all people with cystic fibrosis.
It is made from a combination of medicines – lumacaftor and ivacaftor – that work together to maintain a healthy balance of salt and water in the lungs.
There may be more than 3,000 people with life-limiting cystic fibrosis in the UK who may benefit from the drug.
But the National Institute for Health and Care Excellence (Nice) said in 2016 it might not be profitable enough, at £ 104,000 per patient per year, to be offered at the NHS.
More than 117,000 people have signed a petition calling on the government to make Orkambi available on the NHS, and Parliament discussed the petition in March this year.
Nice is expected to revise its position on the drug in July 2019.
Daniel Bodio, an engineer from Swindon in the mid-1930s, was prescribed the drug on compassion.
He said it improved his lung function from 25 percent – when faced with a transplant – to 39 percent within three weeks.
He told MailOnline last year: & # 39; The drug should be available to everyone – it brings normality to an otherwise difficult life. & # 39;
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