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“Please don’t let us die”: three cystic fibrosis patients beg the NHS to fund “miracle medicine”

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 get hold of 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, in which more than 200 people died of the cruel condition.

However, the National Institute of Health and Care Excellence (NICE) has not been able to reach an agreement with the manufacturer.

The government-run 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 down to Downing Street 10 to share their frustrations.

Mrs. May responded last night by telling the companies ‘to do everything’ to break the deadlock.

And MailOnline has spoken with three cystic fibrosis patients, who have begged the parties to make progress.

Hannah Chew born Lindley, 23, Shiloh Howells, nine, and Lorcán Maguire, two, are desperately looking for the chance to try Orkambi.

Three people with cystic fibrosis have begged the NHS to fund a “panacea” to combat the paralyzing condition. Lorcán Maguire, from Downpatrick, Northern Ireland, is two

Shiloh Howells, nine, has lungs so vulnerable that she is expected to undergo a risky double lung transplant by the age of 12. Her mother, Nichola Howells, said it was heartbreaking knowing that Orkambi could prevent surgery

Shiloh Howells, nine, has lungs so vulnerable that she is expected to undergo a risky double lung transplant by the age of 12. Her mother, Nichola Howells, said it was heartbreaking knowing that Orkambi could prevent surgery

Shiloh Howells, nine, has lungs so vulnerable that she is expected to undergo a risky double lung transplant by the age of 12. Her mother, Nichola Howells, said it was heartbreaking knowing that Orkambi could prevent surgery

Hannah Chew born Lindley, 23, from Leeds, said CF stole her joy, childhood, and her best friend - she had to tragically see her sister, Iona Kesteven, die of the disease. Shown on her wedding day in August 2018

Hannah Chew born Lindley, 23, from Leeds, said CF stole her joy, childhood, and her best friend - she had to tragically see her sister, Iona Kesteven, die of the disease. Shown on her wedding day in August 2018

Hannah Chew born Lindley, 23, from Leeds, said CF stole her joy, childhood, and her best friend – she had to tragically see her sister, Iona Kesteven, die of the disease. Shown on her wedding day in August 2018

WHAT IS ORKAMBI AND WHO WOULD BE WITH IT?

Orkambi can delay the decline of lung function – the leading cause of death among 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 traps bacteria.

It causes persistent lung infections and limits the ability to breathe over time, limiting life expectancy to mid 40.

There are nearly 3,000 people in Britain who have two copies of the F508del mutation – about half of the patients – who could benefit from Orkambi, according to the UK Cystic Fibrosis Registry Report 2014.

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 offer from NHS England for a five-year delivery, the largest offer it has ever made, and calls have been completely cut off.

The drug is only ever prescribed on rare compassionate grounds, which many charities say it’s too late.

Ms. May said she wanted a “quick fix” in the ongoing negotiations so that CF patients could gain access to Orkambi and Symkevi – another drug that treats the cause of the condition but is also unavailable on the NHS.

Since then there have been no resolutions.

Vertex reached an agreement with Scotland for access to Orkambi and Symkevi in ​​December, but the battle for the medicine continues in England, Wales and Northern Ireland.

Yesterday, the Cystic Fibrosis Trust handed over a letter to Theresa May on number 10 Downing Street.

Zoey Jones, the distressed mother of one-year-old Eva, who has cystic fibrosis, was also there to give 65 roses – a term used by children to pronounce the name of the condition.

David Ramsden, chief executive of the Cystic Fibrosis Trust, said: “This tragic situation cannot continue. Every day the health of thousands of people in this country is deteriorating – and this is damage to their lungs that cannot be undone. “

“We ask Prime Minister Theresa May to attend the Westminster Hall debate on Monday, June 10, and to inform the house of the next steps.”

“My sister died while waiting for that medicine”

Hannah Chew born Lindley, 23, said CF stole her joy, childhood and her best friend – she had to tragically see her sister, Iona Kesteven, disappear from the condition.

Hannah Chew born Lindley, 23 said that her and her sister Iona, who died, are best friends

Hannah Chew born Lindley, 23 said that her and her sister Iona, who died, are best friends

Hannah Chew born Lindley, 23 said that her and her sister Iona, who died, are best friends

When Hannah looks back on her childhood, she usually remembers Iona, who died at the age of 19, sick. Pictured on vacation

When Hannah looks back on her childhood, she usually remembers Iona, who died at the age of 19, sick. Pictured on vacation

When Hannah looks back on her childhood, she usually remembers Iona, who died at the age of 19, sick. Pictured on vacation

Around the age of eight, Hannah remembers that Iona's disease got a better hold and they were placed in separate bedrooms. The date in the hospital is unclear

Around the age of eight, Hannah remembers that Iona's disease got a better hold and they were placed in separate bedrooms. The date in the hospital is unclear

Around the age of eight, Hannah remembers that Iona’s disease got a better hold and they were placed in separate bedrooms. The date in the hospital is unclear

Hannah, from Leeds, is not afraid to admit that her life with CF, apart from some happy memories, was “horrible.”

She said: “Many people mask it, which annoys me because it has to be taken seriously.

‘It has stolen my joy, health and mobility. It stole my childhood and my best friend – my sister. I had to watch her die.

“On some days you feel better and on other days you wonder why you are on this planet.”

When Hannah looks back on her childhood, she usually remembers Iona, who died at the age of 19, sick.

She said: “We lived life permanently in a touch and go situation. I remember my sister getting sick all the time and not knowing if she would live or die and live constantly with the fear of – is this the time she dies? “

In November 2015, Iona's lung collapsed due to multiple lung infections and she was rushed back to the hospital. She died a week later. Pictured in advance on vacation

In November 2015, Iona's lung collapsed due to multiple lung infections and she was rushed back to the hospital. She died a week later. Pictured in advance on vacation

In November 2015, Iona’s lung collapsed due to multiple lung infections and she was rushed back to the hospital. She died a week later. Pictured in advance on vacation

Hannah’s parents tried to make their lives as normal as possible by taking the girls to ballet, piano lessons and playing groups.

She said: “We even went to the theater to perform from the hospital. We made ourselves up at the hospital. We did not want to be a number of children in the ward. We have minimized as much as possible. “

But around the age of eight, Hannah remembers that Iona’s disease got a better grip and they were placed in separate bedrooms.

People with CF grow insects in their lungs that are usually harmless to people who do not have the condition, but who can easily be transferred from one person with CF to another, including siblings, and who are very harmful.

Hannah said, “I felt guilty a lot because I wasn’t as poor as she was.”

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, was “horrible”

Iona was known to her consultants as a complex patient, but Hannah believes it was in May 2015, when Iona contracted a stomach virus during a flight home during a vacation that went downhill heavily.

In November 2015, her lung collapsed due to multiple lung infections and she was rushed back to the hospital. She died a week later.

Hannah, who married Nathan Chew, 23 in August 2018, said: “One of the consultants was in shock because they believed she would have survived longer.

“She could still be here with Orkambi. My sister died awaiting that medicine. It was approved on compassionate grounds two weeks after her death, but it was too late.

“The healthier you are, the more likely you are, so they don’t have to wait until they reach a certain point.”

Hannah, who completes exams to become a music teacher, won an Outstanding Young Person prize at the Cystic Fibrosis Holiday Fund awards, sponsored by CLC World Resorts and Hotels. She also received more than 23,000 signatures on a petition that has since been closed.

She said she is frustrated that CF patients with other mutations receive medication that they can treat – despite being happy with it.

She said, “It’s disgusting. It’s like we say we treat breast cancer, but not lung cancer. Or type 1 diabetes but not type 2. ”

“Orkambi can help me prevent a double lung transplant at the age of 12”

Shiloh Howells, nine, has lungs that are so fragile that she is expected to have a risky double lung transplant by the age of 12.

Doctors say the extremity of her condition is much worse than other CF patients of her age who have undergone a lobectomy – an operation to remove part of her damaged right lung in October 2017.

Her mother, Nichola Howells, 40, said knowing just that Orkambi exists is heartbreaking, since it could save what lung function Shiloh still has.

Shiloh, pictured with her mother, Mrs. Howells and grandmother, has very fragile lungs with a basic lung function of around 60 percent

Shiloh, pictured with her mother, Mrs. Howells and grandmother, has very fragile lungs with a basic lung function of around 60 percent

Shiloh, pictured with her mother, Mrs. Howells and grandmother, has very fragile lungs with a basic lung function of around 60 percent

Shiloh's lungs are so fragile that she is expected to have a risky double lung transplant by the age of 12. Pictured in the hospital where she performs routine treatment every two months

Shiloh's lungs are so fragile that she is expected to have a risky double lung transplant by the age of 12. Pictured in the hospital where she performs routine treatment every two months

Shiloh’s lungs are so fragile that she is expected to have a risky double lung transplant by the age of 12. Pictured in the hospital where she performs 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. It is still life threatening.

“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.

“It is soul that destroys us. It is money and finances during a lifetime. We are angry and frustrated. “

In March 2018, former Health Secretary Jeremy Hunt visited Great Ormond Street Hospital in London and was welcomed by Shiloh who gave him a teddy bear, the hospital’s mascot.

But according to Howells, as soon as he was told that Shiloh was being treated for CF, he quickly moved on in the midst of the outrage over the price war.

Mrs. Howells said, “While she was talking, he got up and walked away. Maybe he was afraid that we would bombard him with questions. We were all taken back. “

From the age of six weeks, Shiloh has hospitalization every three months to receive treatment to prevent infections. Pictured, with medication

From the age of six weeks, Shiloh has hospitalization every three months to receive treatment to prevent infections. Pictured, with medication

From the age of six weeks, Shiloh has hospitalization every three months to receive treatment to prevent infections. Pictured, with medication

In March 2018, former Health Minister Jeremy Hunt visited the 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.

In March 2018, former Health Minister Jeremy Hunt visited the 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.

In March 2018, former Health Minister Jeremy Hunt visited the 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 has hospitalization every three months to receive treatment to prevent infections.

WHAT IS CYSTIC FIBROSIS?

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)
  • squeak
  • Breathlessness
  • Exercise intolerance
  • Repeated lung infections
  • Inflamed nasal passages or stuffy nose

Although healthy people naturally cough, that does not happen for people with CF.

Ultimately, lung function gets so far that patients need a double lung transplant to survive.

Source: Cystic Fibrosis Foundation

“But she didn’t have three months to get sick,” Mrs. Howell said. “So she now goes to the Great Ormond Street Hospital every two months for antibiotic and IV treatment, between two weeks and three months.”

During Shiloh’s longest stay, eight years old, she stayed for three months when her lung function dropped to 36 percent.

Her lung function at the start is 60 percent, but in the past she was around 75 percent.

However, Shiloh is always positive and smiling and has recently been able to go to school for two months – the longest period in two years.

Mrs. Howells, who has an older daughter named Essence, 20, said: “She is on the brink, but luckily she picks herself up. But it’s getting harder and harder.

“Her lungs are already very delicate. To prevent other stories such as hers, patients should receive Orkambi as early as possible.

‘CF is all about prevention. We throw medicines to the patients forever to make them as comfortable as possible. But Orkambi tackles the genetic side. “

“It’s a lot for a little man, and he’s still dying”

Lorcán Maguire from Downpatrick, Northern Ireland, has just turned two and still does not know how different his life is from that of other children.

Every day he has 18 tablets, two nebulizers for antibiotics, another nebulizer for a medicine to dilute the mucus, two fifteen minutes of physiotherapy and vitamins because he cannot absorb them properly from food.

His mother, Jen Maguire, 34, said: “It’s a lot for a little man, and he’s still dying.

“The estimate is now in the mid 40s. But Orkambi has shown to add 20 years to the current life expectancy.

“It’s such a terrible situation. I wish he had the same life expectancy as his sister.

Jen Maguire, 34, said her family, including 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

Jen Maguire, 34, said her family, including her husband Dermot, 45, 11-month-old daughter, Saorla (left), and son Lorcán, who has CF, are about to move to the Republic of Ireland to live in Orkambi to get

Jen Maguire, 34, said her family, including her husband Dermot, 45, 11-month-old daughter, Saorla (left), and son Lorcán, who has CF, are about to move to the Republic of Ireland to live in Orkambi to get

“He only thinks his life is like that of others. I hope we get Orkambi before he is aware of the situation.

“It must be so difficult for older people with CF who know the government just doesn’t care.”

Mrs. Maguire, who also has an 11-month-old daughter, Saorla, said the disorder has ruined Lorcán’s childhood – and it’s just the beginning.

She said: ‘He can play with other children, but we constantly use bacterial gel and keep our eyes open and ears open.

“As soon as you hear a cough or smell a cigarette, we’ll knock it away immediately.”

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 situation that would change the lives of the entire family.

They are about to move to Ireland, where Orkambi has been given to eligible patients since 2017.

Mrs Maguire said that the situation has ruined Lorcán's childhood - and it is just the beginning. He is currently trying to fight an infection called pseudomonas aeruginosa, which has been persistently difficult to treat since November 2018. On the photo, the two together while picking out a Lorcán medicine

Mrs Maguire said that the situation has ruined Lorcán's childhood - and it is just the beginning. He is currently trying to fight an infection called pseudomonas aeruginosa, which has been persistently difficult to treat since November 2018. On the photo, the two together while picking out a Lorcán medicine

Mrs Maguire said that the situation has ruined Lorcán’s childhood – and it is just the beginning. He is currently trying to fight an infection called pseudomonas aeruginosa, which has been persistently difficult to treat since November 2018. On the photo, the two together while picking out a Lorcán medicine

Mrs. Maguire said she wanted Lorcán to have the same life expectancy as his sister, pictured

Mrs. Maguire said she wanted Lorcán to have the same life expectancy as his sister, pictured

Mrs. Maguire said she wanted Lorcán to have the same life expectancy as his sister, pictured

Since January, Orkambi has had a license – but not funded – for two-year-olds in the UK.

WHAT CAN THERESA SAY?

On May 16, Theresa May told MPs in the Lower House that she had been looking for a “quick fix” for the Orkambi prize line.

She said: “Cystic fibrosis is clearly a terrible, life-limiting condition and it is right that patients should have access to cost-effective, innovative drugs and technologies.

“The issue has been raised by Members from across Parliament and, like the hon. Dame said, there is an ongoing dialogue between NHS England and Vertex, but I would like a quick solution for the negotiations. I understand that several members have asked to see me about the issue and I am happy that this is happening. “

Mrs Maguire, who stopped her doctoral research in education because she is too stressed to study, said: “If nothing happened during the summer holidays, we are thinking of renting our house and moving there.

“We hope that something will happen sooner because it is not ideal. It’s scary to know how quickly things get worse with CF. “

Lorcán is currently trying to combat an infection called pseudomonas aeruginosa, which has been persistently difficult to treat since November 2018.

He has just returned from a ten-day stay in the hospital, isolated in a room to prevent him from getting more infections from other CF patients.

Medications were pumped through an IV line after antibiotics could not treat the infection for three months. The doctors still don’t know for sure if it’s gone.

Mrs. Maguire said: “Can you imagine a two-year-old sitting in a room? He screamed and screamed as they put the IV lines in and he shook every time.

‘It is worrying because the infection is becoming more resistant to medicines, but it causes a lot of damage.

“Orkambi has been shown to reduce bacteria and hospitalization by 60 percent. It treats the cause by making the mucus less sticky.

“It is not good to pump children with these drugs that only treat symptoms if you could stop anything at all.”

MailOnline contacted NICE and Vertex for comments, both of whom said conversations were going on.

Vertex said in a statement: “Vertex is currently conducting discussions with NHS England and NICE. We are currently unable to comment further. “

NICE said: ‘We would like to be involved with Vertex again. Vertex, NICE and NHS England met in March and agreed to continue the discussions. ”

For more information about cystic fibrosis and the Orkambi battle, visit 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 targets 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 4,000 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 it may not be cost-effective enough in 2016, with £ 104,000 per patient per year, to be offered at the NHS.

More than 117,000 people signed a petition calling on the government to make Orkambi available on the NHS and Parliament discussed the petition in March 2019.

After four years of negotiation, it finally became available on October 24 at the NHS in England.

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