Post by Deanne Jenkyns on Jun 26, 2007 19:03:49 GMT 1
Most of us know about the Drug herceptin and how it was eventually approved by NICE. There are many more approved drugs being denied to patients some of them are below.
BREAST cancer care in the UK was transformed when the NHS caved in to pressure to prescribe wonderdrug Herceptin.
The climbdown last year gave hope to thousands of women with the aggressive HER2 form of the disease.
And with technology that wouldn’t look out of place on the Starship Enterprise, another miracle treatment launched today could prove an even bigger hitter in the war on cancer.
It is called Avastin and, like Herceptin, it is one of the new wave of treatments known as targeted therapies or “magic bullets”.
They work a bit like the microscopic “nano-probes” used by the evil Borg in Star Trek to hijack the body of Seven-of-Nine played by Jeri Ryan.
But where nano-probes work against the body on TV, the real-life targeted therapies — genetically engineered antibodies — work in our favour.
One of the keys to their success is that they battle cancer directly and leave healthy cells untouched, unlike chemotherapy.
For example, Herceptin works like a guided missile, seeking out and destroying tumours by targeting the protein HER2, which is found on the surface of some cancer cells.
This protein is just one of hundreds, perhaps thousands, of potential targets — and there are already other drugs such as Glivec, Sutent and Tarceva available for specific cancers.
What makes Avastin potentially very important is that it targets a growth factor called VEGF, which is involved in a large number of cancers.
When they form, tumours draw oxygen and nutrients from surrounding tissue.
But as they grow — and their demands increase — cancers have to create their own blood supply to continue feeding the tumour. Avastin starves tumours by blocking VEGF and cutting off their blood supply.
Top cancer doctor Professor Ian Smith, of the Royal Marsden Hospital in London, says: “When I was a young doctor studying in the Seventies I heard people talking about blocking VEGF. It was the holy grail of treatment — something that could switch off the cancer.”
Today, Avastin is licensed for use in advanced breast cancer. And it is already being used to combat bowel tumours.
Trials are under way testing its effectiveness against more than 20 other types of tumour, including often lethal forms such as kidney, ovarian and pancreatic cancer.
But it is not all good news.
Like Herceptin, Avastin does not come cheap — it costs around £17,000 a year to treat a bowel cancer patient.
And as with any new treatment, it is offered first to terminal patients with no other hope.
Only when it has proven successful will it be tested on patients with early signs of the disease to see if it can stop the cancer before it takes hold.
Then there is the question of how long to take these new treatments.
With Herceptin, health watchdog NICE agreed NHS funding for 12 months, based on studies. But a smaller Finnish study suggested Herceptin may be just as effective when taken for nine weeks.
That is now being repeated on a much bigger scale and the results could mean huge savings for primary care trusts here.
However, Professor Smith says that other targeted therapies may need to be taken for life.
He adds: “It’s a bottomless pit when it comes to demand for these new treatments. They cost so much that I don’t think any state-funded organisation can be expected to pay for them.”
The dilemma is that if a patient offers to pay for it they have to become a private patient and then pay for everything.
“Suddenly, the cost of the drug becomes just a small part of the total bill — the hospital charges are enormous.”
PASSING THE
QALY-TY TEST
NHS bosses decide whether or not to fund new treatments by working out their cost per QALY, or Quality Adjusted Life Year.
One QALY equals 12 months of perfect health, and factors including pain reduction, savings on other treatments, mobility and a patient’s ability to look after themselves are used to come up with a quality of life score.
For instance, if a new drug costs £10,000 and provides four extra years of perfect health, it has a cost per QALY of £2,500.
But if the quality of those four years is rated at only 75 per cent, the drug would have a cost per QALY of £3,333 (£10,000 divided by four, divided by 0.75).
It means that drugs that appear expensive can be deemed better value than cheaper medicines.
The dilemma of deciding which medicines should be available on the NHS is highlighted by the row over funding of Alzheimer drugs.
Health rationing body NICE found that, although the dementia medicines appear inexpensive, the quality of life improvement they produce can be low.
One dementia drug had a cost per QALY of £139,000 compared with £18,000 for Herceptin.
One possible solution are the “top-up” insurance policies suggested by cancer expert professor Karol Sikora.
The first of these has just been launched by health insurer WPA.
It has introduced a new stand-alone policy, called mycancerdrugs, which will charge a one-off annual fee based on your age. So a 40-year-old will pay £40 for a lifetime benefit of £50,000 towards cancer drugs.
Doctors, politicians, NHS bosses and taxpayers will have to make increasingly tough calls when it comes to new cancer treatments and we will have to decide how much we are prepared to pay to save a life.
BOWEL CANCER
BARRY CROMPTON’S only hope of a longer life is Avastin, a drug the NHS refuses to fund.
The dad of five was diagnosed with advanced bowel cancer in 2004 and given just months to live.
On visiting relatives in Australia for one last farewell, they persuaded him to see local experts, who gave him Avastin as part of a trial.
His health turned around within months.
But when the 44-year-old sales director returned to the UK he was denied treatment.
And when he said he would pay for it, the NHS withdrew all other treatment — including the chemotherapy.
The NHS rule is that if you pay for one element of your treatment you become a private patient and have to pay for everything — whether it’s the chemotherapy you’ve previously been given for free or a cup of tea you have while in hospital.
Barry, from Southampton, now has to pay £5,500 in medical costs every three weeks and is horrified at the NHS’s policy.
He says: “It’s outrageous that people in the UK are being denied such a life-saving treatment.
“My tumour count went from 188 to just 22 in just three months after starting Avastin.
“It has meant I can carry on with a relatively normal life.”
For more information on bowel cancer visit bowelcanceruk.org.uk or call 08708 50 60 50.
LUNG CANCER
NON-SMOKER Susan Allen was given just ten months to live after being diagnosed with lung cancer in 2005.
The 43-year-old mum of one could hardly breathe and was house-bound. X-rays revealed snow-white lungs — they were a mass of tumours.
When her oncologist gave her a prescription for Tarceva last October Susan did not hold out much hope, though it was one of the first targeted treatments against lung cancer.
It targets the EGFR enzyme, a protein that is over-produced in up to 80 per cent of people with lung cancer tumours. EGFR sends out signals to replicate the cancer cells. Tarceva blocks these signals.
In one study 31 per cent of the patients taking Tarceva were still alive after a year compared with 22 per cent of those taking a placebo.
It costs £7,000 for a course of treatment.
Susan, from Rushton Spencer, Staffs, says: “I was going into a hospice and no one expected me to come out alive.”
But three days after taking Tarceva she was walking and talking normally again.
Susan is married to Phil, who insisted she give Tarceva a go. She adds: “A month later I was out of the hospice and down the pub scoffing chips.”
NICE rejected Tarceva in March, saying it was not an effective use of NHS resources.
But research shows giving Tarceva to lung cancer patients works out at an additional cost to the NHS of just £1.7million a year — a drop in the ocean compared with the £186million spent on private management consultants for the NHS last year.
Susan says: “Denying the drug is condeming people to death.”
For more information on lung cancer, contact the Roy Castle Lung Cancer Foundation at roycastle.org or call 0871 220 5426.
KIDNEY CANCER
MARK FRANKLIN was so ill with kidney cancer he could barely walk up the stairs — but after getting on to a clinical trial for the wonderdrug Sutent he is now climbing mountains.
The 39-year-old builder, a bachelor from Chesham in Bucks, says: “I was diagnosed in 2005 and after it spread to my liver, lungs and back, I was given just six months to live.
“I was in such pain I had to give up my job and move in with my parents so they could look after me.”
Kidney cancer is a disease that affects 6,600 people in the UK every year. More than half of those will die. Mark adds: “I should be dead by now.
“My tumours measured 58cms in total and they said there was nothing more in the world that could help me.”
But after getting on to a clinical trial for Sutent, his tumours have now shrunk to 7½cms.
After just two weeks the tumour in his back had halved.
Although the drug is approved for use in the UK, the NHS has failed to issue guidance that Sutent should be funded by primary care trusts. It costs £2,500 a month.
Studies show it shrinks kidney tumours in 31 per cent of patients in the late stages of the disease, compared with just six per cent with standard treatment.
Sutent prevents growth of the tumour by interfering with VEGF and PDGF (platelet-derived growth factor) pathways, depriving the tumour cells of blood and nutrients.
Mark adds: “Before the trial I could barely climb stairs.
“Now I go to the gym regularly and am taking part in the Three Peaks Challenge, climbing Ben Nevis, Mount Snowdon and Scafell Pike to help raise money for three cancer charities.”
For more information, call Kidney Cancer UK on 01889 565801.
LEUKAEMIA
WHEN ten-year-old Max Horwood was diagnosed with an aggressive form of leukaemia in 2003 his parents Simon and Parmjit thought it was the end of the road.
Max, now 14, had chronic myelogenous leukaemia, which attacks the bone marrow, and he needed a transplant — but he was in for a long wait. Dad Simon, 44, says: “The outlook was very bleak and our only hope was to keep Max healthy until we found a match.”
Luckily, Max became one of the first children to be treated with Glivec, which was already used successfully in adults. The drug targets protein Bcr-Abl tyrosine kinase — a by-product of a gene abnormality.
Simon, a lawyer from Fleet in Hampshire, says: “Within a couple of months we seemed to be getting our son back. The drug had targeted and destroyed cancer cells in his spleen and he was getting stronger daily.”
When a donor was found two years later, Max was healthy enough for the op.
Delighted Simon adds: “He is now thriving.”
Glivec costs £19,000 a year. It has been approved by NICE but it cannot force primary care trusts to prescribe it.
BREAST cancer care in the UK was transformed when the NHS caved in to pressure to prescribe wonderdrug Herceptin.
The climbdown last year gave hope to thousands of women with the aggressive HER2 form of the disease.
And with technology that wouldn’t look out of place on the Starship Enterprise, another miracle treatment launched today could prove an even bigger hitter in the war on cancer.
It is called Avastin and, like Herceptin, it is one of the new wave of treatments known as targeted therapies or “magic bullets”.
They work a bit like the microscopic “nano-probes” used by the evil Borg in Star Trek to hijack the body of Seven-of-Nine played by Jeri Ryan.
But where nano-probes work against the body on TV, the real-life targeted therapies — genetically engineered antibodies — work in our favour.
One of the keys to their success is that they battle cancer directly and leave healthy cells untouched, unlike chemotherapy.
For example, Herceptin works like a guided missile, seeking out and destroying tumours by targeting the protein HER2, which is found on the surface of some cancer cells.
This protein is just one of hundreds, perhaps thousands, of potential targets — and there are already other drugs such as Glivec, Sutent and Tarceva available for specific cancers.
What makes Avastin potentially very important is that it targets a growth factor called VEGF, which is involved in a large number of cancers.
When they form, tumours draw oxygen and nutrients from surrounding tissue.
But as they grow — and their demands increase — cancers have to create their own blood supply to continue feeding the tumour. Avastin starves tumours by blocking VEGF and cutting off their blood supply.
Top cancer doctor Professor Ian Smith, of the Royal Marsden Hospital in London, says: “When I was a young doctor studying in the Seventies I heard people talking about blocking VEGF. It was the holy grail of treatment — something that could switch off the cancer.”
Today, Avastin is licensed for use in advanced breast cancer. And it is already being used to combat bowel tumours.
Trials are under way testing its effectiveness against more than 20 other types of tumour, including often lethal forms such as kidney, ovarian and pancreatic cancer.
But it is not all good news.
Like Herceptin, Avastin does not come cheap — it costs around £17,000 a year to treat a bowel cancer patient.
And as with any new treatment, it is offered first to terminal patients with no other hope.
Only when it has proven successful will it be tested on patients with early signs of the disease to see if it can stop the cancer before it takes hold.
Then there is the question of how long to take these new treatments.
With Herceptin, health watchdog NICE agreed NHS funding for 12 months, based on studies. But a smaller Finnish study suggested Herceptin may be just as effective when taken for nine weeks.
That is now being repeated on a much bigger scale and the results could mean huge savings for primary care trusts here.
However, Professor Smith says that other targeted therapies may need to be taken for life.
He adds: “It’s a bottomless pit when it comes to demand for these new treatments. They cost so much that I don’t think any state-funded organisation can be expected to pay for them.”
The dilemma is that if a patient offers to pay for it they have to become a private patient and then pay for everything.
“Suddenly, the cost of the drug becomes just a small part of the total bill — the hospital charges are enormous.”
PASSING THE
QALY-TY TEST
NHS bosses decide whether or not to fund new treatments by working out their cost per QALY, or Quality Adjusted Life Year.
One QALY equals 12 months of perfect health, and factors including pain reduction, savings on other treatments, mobility and a patient’s ability to look after themselves are used to come up with a quality of life score.
For instance, if a new drug costs £10,000 and provides four extra years of perfect health, it has a cost per QALY of £2,500.
But if the quality of those four years is rated at only 75 per cent, the drug would have a cost per QALY of £3,333 (£10,000 divided by four, divided by 0.75).
It means that drugs that appear expensive can be deemed better value than cheaper medicines.
The dilemma of deciding which medicines should be available on the NHS is highlighted by the row over funding of Alzheimer drugs.
Health rationing body NICE found that, although the dementia medicines appear inexpensive, the quality of life improvement they produce can be low.
One dementia drug had a cost per QALY of £139,000 compared with £18,000 for Herceptin.
One possible solution are the “top-up” insurance policies suggested by cancer expert professor Karol Sikora.
The first of these has just been launched by health insurer WPA.
It has introduced a new stand-alone policy, called mycancerdrugs, which will charge a one-off annual fee based on your age. So a 40-year-old will pay £40 for a lifetime benefit of £50,000 towards cancer drugs.
Doctors, politicians, NHS bosses and taxpayers will have to make increasingly tough calls when it comes to new cancer treatments and we will have to decide how much we are prepared to pay to save a life.
BOWEL CANCER
BARRY CROMPTON’S only hope of a longer life is Avastin, a drug the NHS refuses to fund.
The dad of five was diagnosed with advanced bowel cancer in 2004 and given just months to live.
On visiting relatives in Australia for one last farewell, they persuaded him to see local experts, who gave him Avastin as part of a trial.
His health turned around within months.
But when the 44-year-old sales director returned to the UK he was denied treatment.
And when he said he would pay for it, the NHS withdrew all other treatment — including the chemotherapy.
The NHS rule is that if you pay for one element of your treatment you become a private patient and have to pay for everything — whether it’s the chemotherapy you’ve previously been given for free or a cup of tea you have while in hospital.
Barry, from Southampton, now has to pay £5,500 in medical costs every three weeks and is horrified at the NHS’s policy.
He says: “It’s outrageous that people in the UK are being denied such a life-saving treatment.
“My tumour count went from 188 to just 22 in just three months after starting Avastin.
“It has meant I can carry on with a relatively normal life.”
For more information on bowel cancer visit bowelcanceruk.org.uk or call 08708 50 60 50.
LUNG CANCER
NON-SMOKER Susan Allen was given just ten months to live after being diagnosed with lung cancer in 2005.
The 43-year-old mum of one could hardly breathe and was house-bound. X-rays revealed snow-white lungs — they were a mass of tumours.
When her oncologist gave her a prescription for Tarceva last October Susan did not hold out much hope, though it was one of the first targeted treatments against lung cancer.
It targets the EGFR enzyme, a protein that is over-produced in up to 80 per cent of people with lung cancer tumours. EGFR sends out signals to replicate the cancer cells. Tarceva blocks these signals.
In one study 31 per cent of the patients taking Tarceva were still alive after a year compared with 22 per cent of those taking a placebo.
It costs £7,000 for a course of treatment.
Susan, from Rushton Spencer, Staffs, says: “I was going into a hospice and no one expected me to come out alive.”
But three days after taking Tarceva she was walking and talking normally again.
Susan is married to Phil, who insisted she give Tarceva a go. She adds: “A month later I was out of the hospice and down the pub scoffing chips.”
NICE rejected Tarceva in March, saying it was not an effective use of NHS resources.
But research shows giving Tarceva to lung cancer patients works out at an additional cost to the NHS of just £1.7million a year — a drop in the ocean compared with the £186million spent on private management consultants for the NHS last year.
Susan says: “Denying the drug is condeming people to death.”
For more information on lung cancer, contact the Roy Castle Lung Cancer Foundation at roycastle.org or call 0871 220 5426.
KIDNEY CANCER
MARK FRANKLIN was so ill with kidney cancer he could barely walk up the stairs — but after getting on to a clinical trial for the wonderdrug Sutent he is now climbing mountains.
The 39-year-old builder, a bachelor from Chesham in Bucks, says: “I was diagnosed in 2005 and after it spread to my liver, lungs and back, I was given just six months to live.
“I was in such pain I had to give up my job and move in with my parents so they could look after me.”
Kidney cancer is a disease that affects 6,600 people in the UK every year. More than half of those will die. Mark adds: “I should be dead by now.
“My tumours measured 58cms in total and they said there was nothing more in the world that could help me.”
But after getting on to a clinical trial for Sutent, his tumours have now shrunk to 7½cms.
After just two weeks the tumour in his back had halved.
Although the drug is approved for use in the UK, the NHS has failed to issue guidance that Sutent should be funded by primary care trusts. It costs £2,500 a month.
Studies show it shrinks kidney tumours in 31 per cent of patients in the late stages of the disease, compared with just six per cent with standard treatment.
Sutent prevents growth of the tumour by interfering with VEGF and PDGF (platelet-derived growth factor) pathways, depriving the tumour cells of blood and nutrients.
Mark adds: “Before the trial I could barely climb stairs.
“Now I go to the gym regularly and am taking part in the Three Peaks Challenge, climbing Ben Nevis, Mount Snowdon and Scafell Pike to help raise money for three cancer charities.”
For more information, call Kidney Cancer UK on 01889 565801.
LEUKAEMIA
WHEN ten-year-old Max Horwood was diagnosed with an aggressive form of leukaemia in 2003 his parents Simon and Parmjit thought it was the end of the road.
Max, now 14, had chronic myelogenous leukaemia, which attacks the bone marrow, and he needed a transplant — but he was in for a long wait. Dad Simon, 44, says: “The outlook was very bleak and our only hope was to keep Max healthy until we found a match.”
Luckily, Max became one of the first children to be treated with Glivec, which was already used successfully in adults. The drug targets protein Bcr-Abl tyrosine kinase — a by-product of a gene abnormality.
Simon, a lawyer from Fleet in Hampshire, says: “Within a couple of months we seemed to be getting our son back. The drug had targeted and destroyed cancer cells in his spleen and he was getting stronger daily.”
When a donor was found two years later, Max was healthy enough for the op.
Delighted Simon adds: “He is now thriving.”
Glivec costs £19,000 a year. It has been approved by NICE but it cannot force primary care trusts to prescribe it.
LC charity Ball