YEARS of research into a single molecule have given Karen back her life. Four
years ago, aged 55, her immune system started to attack her joints. The
resulting severe arthritis made her joints seize up one by one. Standard
treatment with aspirin-type drugs and the immunosuppressant methotrexate was
failing. Immobile and in agony, by the spring of 1998 she was going weeks
without sleep, and sank into despair.
Then a press report alerted her to an experimental treatment about to be
tested at Charing Cross Hospital in London. The therapy was a genetically
engineered antibody against the molecule TNF-alpha which plays a pivotal role in
activating the immune system. Its developers hoped that by neutralising the
excess of TNF-alpha in the joints of people with severe arthritis, they could
stop the white blood cells destroying tissue there. This approach had already
shown signs of success in another auto-immune condition, Crohn鈥檚 disease.
Karen began a remarkable recovery that saw her regain most of her mobility
within weeks of the first infusion. With the trial now over, she waits anxiously
to learn if the treatment will be licensed. And the uncertainty doesn鈥檛 stop
there. Even if the antibody is deemed safe and clinically effective, Karen and
other arthritis sufferers may not receive it.
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That鈥檚 because Britain has created an extra hurdle for new drugs and medical
devices: the National Institute for Clinical Excellence (NICE), set up this year
to establish whether new treatments are sufficiently cost effective to justify a
share of the increasingly stretched budget of Britain鈥檚 National Health Service.
If not, they will only be available to patients who can afford to pay for
private healthcare.
High-tech drugs and medical devices may work better than established
treatments: the problem is that they are often phenomenally expensive. That is
proving a burden to healthcare systems throughout the world, which is why
officials elsewhere are keeping a close watch on Britain鈥檚 experiment in
creating an additional economic hurdle for new medicines. 鈥淚t鈥檚 rationing plain
and simple,鈥� says Tony Harrison, a senior policy researcher with the King鈥檚
Fund, an independent health think-tank based in London. 鈥淎nd there鈥檚 got to be
rationing. We can鈥檛 afford everything.鈥�
The government doesn鈥檛 like the 鈥淩鈥� word. Nevertheless, most observers agree
that NICE was set up to make objective decisions to guide rationing of high-tech
treatments that is already being applied in Britain by individual local health
authorities.
Matters have already come to a head with the use of beta-interferon to treat
multiple sclerosis. Some health authorities were happy to pay for the costly
genetically engineered drug. But others said they couldn鈥檛 justify the expense,
causing despair and anger for patients who were denied treatment simply because
of where they live.
The role of NICE is to advise the government on which treatments are
sufficiently cost-effective for them to be available to all patients by
right鈥攖hey will weigh up whether the clinical benefits of a treatment or
device merit its cost. The Multiple Sclerosis Society is expecting the
institute鈥檚 verdict on beta-interferon by July next year. NICE consists of
medical and legal experts plus lay members. Its verdicts are not legally
binding.
However, any decision not to approve a treatment will almost certainly cause
uproar. Patient groups and doctors will be swift to accuse the institute of
callousness if it won鈥檛 approve medicines on grounds of cost. The Association of
the British Pharmaceutical Industry fears the cost effectiveness of drugs might
never be gauged in the first place if NICE blocks them from the market.
NICE has already crossed swords with Britain鈥檚 biggest pharmaceuticals
company. Last month it said that Glaxo Wellcome鈥檚 new flu drug Relenza,
which has been approved as safe and effective by the Medicines Control Agency,
might not be cost effective (快猫短视频, 9 October, p 5). As a
result, the government has decided not to make the drug available on the
NHS.
Relenza costs 鈥渙nly鈥� 拢24 for a course of treatment. So what happens
when NICE is faced with drugs that are tens or hundreds of times more expensive,
such as Karen鈥檚 infusion or high-tech cancer vaccines individually tailored to
encourage a patient鈥檚 immune system to attack cancer cells?
Anne-Toni Rogers, chief spokeswoman for NICE, stresses that the institute
isn鈥檛 there simply to filter expensive treatments. Its remit is to see whether a
drug or device is significantly better than existing technologies, how much it
improves quality of life, and to weigh these benefits against the treatment鈥檚
cost.
鈥淚f something鈥檚 clearly fantastic then we鈥檒l push for it to be available as
quickly as possible,鈥� says Rogers. 鈥淪o it鈥檚 possible we might actually speed
things up. We have a duty to encourage innovation鈥攊f it鈥檚 something that
really makes a difference for the patient.鈥�
Given the severe financial pressure on the NHS, many experts believe NICE
won鈥檛 always often find itself cast in the heroic role that Rogers envisages.
鈥淭he choices are more difficult for Britain because we spend less on health than
our competitors,鈥� says Chris Ham, director of the Health Services Management
Centre at the University of Birmingham.
Indeed, the Cancer Research Campaign has just completed a European survey on
the use of temozolomide, the first major new drug for the treatment of brain
tumours to emerge in three decades. In Britain only three health authorities
have so far used the drug. The drug is used ten times as much in France, Germany
and Italy.
鈥淚 don鈥檛 see how NICE can avoid getting embroiled in some serious rows,鈥� Ham
concludes. 鈥淭hey鈥檝e clearly said they will be looking at cost as well as
别蹿蹿别肠迟颈惫别苍别蝉蝉.鈥�
Richard Smith, editor of the British Medical Journal, worries that
NICE will be a 鈥渟mall group of people making value judgments鈥�. 鈥淲ill they
represent the views of the population? For instance, we know many people place a
higher value on quality of life than length of life,鈥� he says. Echoing this
concern, some patient groups fear that NICE may start to make decisions based
not on cost-effectiveness but on whether it thinks the NHS can actually afford a
new drug or device.
Novel technologies are particularly difficult to subject to a rigorous
economic analysis. The US Food and Drug Administration has just approved a
subcutaneous glucose monitoring system for diabetics. And Japanese engineers
have designed a toilet that analyses urine for glucose, records weight and other
information, and automatically sends a report to the patient鈥檚 doctor. NICE will
have to decide whether or not they are superfluous.
Neurosurgeon Charles Wilson of the University of California at San Francisco
told a joint conference of the British and American Medical Associations last
week these emerging biosensor technologies would have a major impact on health
services within ten years. But he warned: 鈥淭hese advances will be expensive and
in many parts of the world irrelevant.鈥�
Conversely, he鈥檚 confident that in communities with sufficient money to
invest in them, the technologies will pay dividends financially as well as
medically. 鈥淭here will be big savings,鈥� he says. But there鈥檚 a distinct
possibility British patients will miss out if NICE is asked to make quick
decisions on the cost-effectiveness of new drugs and devices, when their long
term economic impact may be almost impossible to calculate.
It鈥檚 possible, for instance, that hand-held biosensors and high-tech beds
embedded with sensors could, one day, save money by replacing bacteriology labs.
Or new arthritis treatments could allow people like Karen to go back to work.
But there鈥檚 no way of calculating this at the moment. 鈥淚t鈥檚 like trying to
speculate what the effect of the railways would be at the start of this
century,鈥� says Harrison.
And even if NICE was equipped with a crystal ball and made the right decision
every time, it can鈥檛 impose its will. A spokeswoman for the Department of Health
told 快猫短视频 that central government cannot force local health
authorities to provide treatments, even if they come with a stamp of approval
from NICE.
For patients like Karen, that means the future is still uncertain. 鈥淚f it
[the arthritis] comes back and I don鈥檛 get the treatment, I don鈥檛 know what I鈥檓
going to do,鈥� she says.
