At the age of 29, Toral Shah was warned she faced losing both her breasts to avoid death from an aggressive cancer. The disease had affected both her mother and her aunt. Devastated by the news, she asked for time to come to terms with the prospect of surgery that could disfigure her for life — and chemo or radiotherapy that could prevent her from ever having children. In a matter of weeks, the tumour had grown to the size of a mango. It was on the verge of invading other tissues — and the prognosis was bad. The surgeons removed a substantial amount of tissue with the tumour and she was given a synthetic implant. In common with thousands of women, Toral faced a stark choice: have both breasts removed, and endure the harrowing ordeal of chemo or radiotherapy, or somehow find an alternative. Toral comes from a medical background and she herself came across the alternative — Tamoxifen, a drug that had been mired in controversy, considered an expensive and unproven cure, offered to only a handful of NHS patients. On the advice of Macmillan Cancer Support, she asked to be given the drug. She was told by her doctors that her tumour was a localised variety. Such tumours grow in the presence of the female hormone oestrogen, which can be blocked by Tamoxifen. She was determined to get it.

At the time, the drug was only available to those in certain areas — the “postcode lottery”. The controversy was based not just on expense but potential side-effects: it can start an artificial form of the menopause. The effects in young women such as Toral were little-documented, but she felt she should be allowed to choose for herself. Her determination paid off; she got the drug.

She is still being regularly monitored to check the cancerous cells are not reappearing. But so far, things are looking positive. And since she began the treatment, the questions about Tamoxifen’s safety and expense have been answered, and it’s now prescribed across the country. Toral believes her fight with the system has been worthwhile.

Tens of thousands of people in the UK also owe their lives to drugs that not so long ago were deemed expensive and risky. But there are still more for whom the fight has only just begun. It was in the spring of last year that Clive Stone, a 59-year-old retired bank manager, found that he was tiring easily. He had always been pretty fit — the gym two or three times a week, swimming once a week — so he did not worry too much. When he became sore across his chest he initially thought that he must have pulled a muscle. But the soreness persisted and eventually he went to his doctor, starting an increasingly alarming cycle of medical tests that ended in the nightmare diagnosis he had most dreaded — he had a cancerous tumour on his kidney. Within days he’d had an operation to have the kidney removed, but he was still not in the clear: the cancer had metastasised and got into his bloodstream. The spots on his lungs that had been revealed in an initial scan were growing. Another scan discovered a cyst on his testes, which would require a second operation.

By then he realised he was dying. Kidney cancer is a killer, spreading quickly round the body; around 2,500 people in the UK die from the disease every year. “My oncologist was frank. He said, ‘There is nothing I can do for you, all I can do is make you as comfortable as I can.’ It’s horrible to have this thing growing inside you. You can’t sleep, and when you do, you have terrible dreams. I’m sorry… I get very emotional about this…” His eyes fill with tears as he struggles to contain himself. This happens several times as we talk at his home in an Oxfordshire village outside Witney. On the table in front of us are two bulging files filled with correspondence and research that document his determined fight for a few extra months of life. There are posters in the windows on either side of his front door: “Kidney Patients Deserve the Right to Life”.

Stone believes that right is vested in Sutent, a new drug he claims could extend his life by as much as two years. He was convinced the National Institute for Health and Clinical Excellence (Nice) would approve the drug, with three others, for NHS patients this year. But in August, Nice announced its interim decision not to recommend Sutent, Nexavar, Torisel and Avastin on the grounds they were not cost-effective. “My world fell apart,” says Stone. “Sutent is the first effective treatment for kidney cancer for 30 years. I just can’t understand it… why the NHS can’t afford to give kidney-cancer patients a drug that is proven to be effective and that’s available worldwide — you can even get it in Romania, for heaven’s sake.

“Nice says the drug will only give you an extra five or six months. But there is new information all the time. The American Society of Clinical Oncology talks about an extra 14 months. There are people in Canada who have been on the drug for three and four years. If you had the choice of dying tomorrow or in a year’s time, which would you choose? I’d like to take my wife away to Italy next year, but I don’t know if I’ll be alive… ” His voice breaks up again. “I’ve got this horrible thing growing inside me. It’s as if no one is listening. As if Nice has signed our death warrants.”

Despite his vulnerable emotional state, Stone is not going down without a fight. Patients once had little alternative but to meekly accept the diagnosis of medical experts: the internet has changed all that. As soon as he learnt he had cancer, Stone began trawling the net to find out about the disease and what treatments were available. He quickly unearthed the intractable problem within the NHS — the postcode lottery, which results from individual primary care trusts (PCTs) having the authority to decide which drugs it will make available to patients in its area. Nice only issues “guidance” to the 152 PCTs in England and Wales. Stone found out that less than one third fund Sutent. If he lived in Birmingham he could get Sutent on the NHS. In Oxfordshire, forget it.

Primary care trusts are responsible for spending more than 80% of the total NHS budget. It is possible, but difficult, to challenge a PCT decision on drug funding — the patient has to demonstrate that he or she is an “exceptional case”, but the criteria for “exceptionality” varies from PCT to PCT: some approve all requests, some approve none.

“The exceptional-cases process is bizarre and demeaning,” says Penny Wilson-Webb, chief executive of the Rarer Cancers Forum. “In the last 20 months, we know that at least 5,000 cancer patients have been forced to plead for their lives by trying to prove to their PCT that they are an exceptional case. Around 1,300 had their requests rejected and are thus denied treatment.

“We got these figures as a result of a Freedom of Information application, but around a third of the PCTs chose not to respond. Why? Was it because they were too lazy, or inefficient or ashamed? Or because they had something to hide?”

Patient power is increasingly putting PCTs under siege. Kate Spall, a feisty 36-year-old mother of two from Chester, and her sister, Emma, stormed a board meeting of the Conway and Denbighshire NHS Trust last year to get treatment for their mother, who was dying of kidney cancer. “After my 10th e-mail had gone unanswered, I decided direct action was the only answer,” says Kate. The chairman asked them to leave, but Kate persisted with her speech to the board: “Who gave you the right to be judge and jury, and condemn my mum to a premature death?” Her mum got the drugs the next day, but by then, says Kate, “it was too late. She was in a hospice and she died four-and-three-quarter months later at 59. She would have lived longer had she got the drugs sooner but she definitely improved in that time, physically and psychologically. She was very upset the PCT did not think she was worthy of being given the drugs. She’d say, ‘Aren’t I good enough to be treated?’ It was heartbreaking”.

Kate has set up the Pamela Northcott Fund in memory of her mother and now actively campaigns to assist patients to overturn PCT decisions. So far she has helped more than 80 people in the UK to get treatment they were previously denied, and she intends to continue her work. “Mum would have wanted me to do it,” she says.

Similar distress has been suffered by critically ill patients who have had their NHS funding withdrawn because they chose to supplement their treatment by paying for “top-up” drugs not available on the NHS. In December, Colette Mills, 58, a breast-cancer sufferer from north Yorkshire who had been taking the NHS-prescribed medication Taxol, kickstarted The Sunday Times’s Right to Pay campaign when she was refused Avastin, a drug not routinely available on the NHS. In trials, when combined with Taxol, Avastin doubled the chance of preventing the disease spreading compared to taking Taxol on its own. Avastin is one of a number of drugs that, depending on where you live, PCTs either fund or allow patients to pay for privately — called “topping up” — while still keeping their NHS care, or withdraw all NHS care if they top up. Mills’s trust did not allow topping up privately, so she launched a legal battle to force the NHS to allow her to pay for Avastin and not forfeit her NHS care, which amounts to about £10,000 a month. She was told by doctors that in the four months since asking for the drug, the disease had spread and the cancer would no longer respond to the treatment. This case and others like it provoked a storm of criticism and forced a review by the Department of Health.

Word is spreading fast that patients need not succumb to PCT rulings. Colin Ross, 55, a former engineer, had been told earlier this year he had only months to live. He took West Sussex PCT to the High Court in a desperate attempt to get a drug, Revlimid, that could prolong his life for another three years, but which has not yet been approved by Nice. Justice Simon Grenfell overturned the PCT’s decision that the drug was not cost-effective, ruling that it was one “no reasonable authority could have made on the application before it”, and issued an emergency injunction for him to begin treatment the following day.

Colin, who is suffering from multiple myeloma, an incurable cancer of the blood cells, was too ill to attend the hearing in September, but says he is now much improved. “I just hope it’s not too late,” he said at his home in Horsham. “I was told in March that I only had two or three months to live without this drug. Since then hope has kept me alive, but part of me is still fretting I have started the treatment too late.” Diagnosed with cancer in May 2004, Colin has been in and out of hospitals ever since, enduring innumerable treatments. The disease caused him a lot of pain and made his bones brittle; at one point he was so weak he needed two hands to hold a toothbrush. When his oncologist at the Royal Marsden Hospital told him there was nothing further they could do for him, and only Revlimid could prolong his life, he applied to West Sussex PCT for treatment as an “exceptional case”, backed by the full support of his medical team at the Royal Marsden.

“I was told it would be routine and it was just a matter of time before I could begin treatment. The PCT should have responded within two weeks. It took them 11 weeks, and then only after a lot of chasing up by the Royal Marsden. They turned down my application on two grounds: that the drug was not sufficiently proven and that it was too expensive, at £5,200 a course. The people at the Royal Marsden were astonished.

“It was a very distressing time for me. I was really scared and worried. I was already prepared to die, but the thought that I could have a few more years to see my two daughters and my grandchildren spurred me on and I decided to go legal. I had nothing to lose. The judge made it abundantly clear my application should never have been turned down and awarded me costs, so the PCT faces legal fees of around £100,000 — more than three times what it would cost to give me the drug. It makes no sense, does it?

“The latest research indicates this drug could give patients an improved quality of life for three years. Who knows what they could come up with during that time? Now I have some hope — before I had none.”

Clive Stone’s PCT, Oxfordshire, defines an “exceptional-status” patient as one liable to “gain significantly more benefit from the intervention than might be expected from the average patient with the condition”. This does not apply to him, but it hasn’t stopped him badgering them, particularly when he found the trust had made a £5.8m surplus last year and is on track to make a further £3m surplus this year. It’s more than enough, he says, to treat every kidney-cancer patient in its area with Sutent, which costs £24,000 per year per patient. As it is only a palliative and not a cure, it is not a long-term financial commitment.

When a PCT representative said on local radio that it would cost “millions” to treat patients with Sutent and might require a hospital to close, Stone wrote to Andrea Young, the £160,000-a-year chief executive of Oxfordshire PCT, demanding an apology. She eventually agreed to meet a delegation of eight people, including Stone and his wife, Jan. It did not go well. Stone later turned up at the AGM to demand a review of the decision not to fund Sutent. The chairman refused. “In the mean time, then,” Stone persisted, “you won’t mind if some of us die an early death?” The chairman swiftly moved on to the next question.

Andrea Young refused to be interviewed but she gave a statement confirming her PCT’s policy of not providing Sutent to kidney-cancer patients had been in place since January. “We recognise this is a deeply emotive issue,” it noted, “and eagerly await the final guidance from Nice, expected in January 2009.” Currently, Oxfordshire PCT funds only two patients as “exceptional cases”; it has turned down 23 applications. Four patients in this group have since died.

By the time the Nice interim decision was announced in August, Stone was chair of a support group, Friends of Renal Oncology (Frog). “On the day we heard the news my phone went mad. People were saying, ‘Our last hope is gone.’ I said, ‘Why don’t we go to the Nice offices and deliver our protest letters?’ ”

About three weeks after the announcement, a group of 100 kidney-cancer patients and supporters led by Clive Stone gathered outside the London headquarters of Nice in T-shirts with the slogan “Kidney Cancer Patients Deserve the Right to Life”. Andrew Dillon, the chief executive, agreed to meet three protesters in the reception. Stone made a speech to Dillon that pulled no punches. “Can you look me in the eye? You have committed me to an early death. I, and a lot of people here, don’t know how you sleep at night.” He was too distressed to continue. One of the other delegation members asked if they could sit down. Dillon refused, apparently on the basis that it would “not be fair” to other patient groups.

Stone wrote to Dillon to complain about the “hostile and indifferent” reception they had received, and asking for the group to be consulted before Nice makes its final decision on kidney-cancer drugs in January, and also to be given access to the algorithms and models underpinning Nice’s decision. He received no reply.

A month later, at a public meeting in Plymouth, more angry kidney-cancer patients confronted the Nice board. Jane Thompson, 49, travelled from her home in Birmingham to tell the meeting she had been given six weeks to live when she was diagnosed with cancer in March, and the reason she was still alive was she had been prescribed Sutent before the Nice decision. “Sutent doesn’t work for everyone, but every patient deserves the right to it. To be told to go away and die quietly is utterly callous.”

Nice — an ironic acronym for an organisation that attracts so much opprobrium — is a new Labour invention, set up in 1999 to evaluate the cost and clinical effectiveness of new drugs and treatments on behalf of the NHS in England and Wales (there is a separate body for Scotland). It tries to balance the cost of treatment against the benefit the patient gets from it, and the anticipated time the benefit will last. It uses a system of “quality-adjusted life years” — or QALY — under which the cost of treatment is divided by the number of years gained. But how much is a life worth? According to Nice, the figure is £30,000. It does not consider any treatment that costs more than £30,000 to provide an extra year of life. The figure has not changed since Nice was set up.

The appraisal process is lengthy, bureaucratic and cumbersome, often taking two or three years. Patients are meant to be integral to it, but there are many signs that their views are more or less ignored. Bill Savage, 62, a kidney-cancer patient and retired management consultant from Amersham, was invited to a Nice assessment meeting in Manchester as a “patient expert”. He returned disillusioned. “The whole consultation process is a sham. The meeting lasted four hours and the three ‘patient experts’ spoke for a total of less than five minutes. The first two hours was taken up by a presentation by health economists that was incomprehensible. We felt patronised, intimidated, bullied and marginalised.”

Ailsa Bosworth, chief executive of the National Rheumatoid Arthritis Society (NRAS) and a rheumatoid-arthritis sufferer herself, says the involvement of patients and patient groups in Nice decisions is a token gesture. “I’ve attended at least six Nice appraisal meetings as a ‘patient expert’. Only once has anyone apart from the chairman asked me a question. They seemed uninterested in quality-of-life issues that matter to real people.” NRAS reacted angrily when, earlier this year, Nice reduced the range of drugs available to the 60,000 rheumatoid-arthritis sufferers in England and Wales. “Nice completely ignored the views of patients and clinicians, along with the effectiveness of the drugs,” says Bosworth.

The fuss over this decision was as nothing compared to the outcry following Nice’s announcement in August not to recommend the kidney-cancer drugs. The National Kidney Federation (NKF) called the decision “dreadful, barbaric, devoid of patient concern”, and launched an appeal. Twenty-six top cancer specialists signed a letter to The Sunday Times asserting Nice’s system for assessing new drugs was not working, Nice had not “got its sums right”, and it was a “tragedy” Britain’s leading role in cancer research was not being translated into treatment for cancer patients. Among the signatories was Jonathan Waxman, professor of oncology at London’s Imperial College and a prominent, persistent critic of Nice. He wrote to The Times: “Our government has been a fabulous custodian of healthcare, but there are areas… where things have gone badly wrong, where wrong meets bad, becomes worse, and then spirals to appalling, and these areas are approached through the bloodstained portals of the National Institute for Health and Clinical Excellence.” Professor Waxman believes that Nice, which costs the taxpayer around £30m every year, is a waste of money and should be abolished. Doctors and specialists, he says, should be making decisions on how and where NHS money is spent. “The Nice decision with regard to kidney cancer is absurd and arrogant. This is a wonderful new treatment that can double life-expectancy, is widely available in Europe and America, yet is condemned as junk by Nice and denied to patients here largely on the basis of advice from health economists manipulating the truth to justify their decisions. As PCTs have no legal obligation to follow Nice guidance, why do we need Nice?” He makes no apologies for his comments: “There are people all over the country who are blind, demented or dead because of delays caused by Nice,” he says.

It took years for the Royal National Institute for the Blind (RNIB) to force Nice to rescind its recommendation not to treat people suffering from wet AMD (aged-related macular degeneration), until they had gone blind in one eye. Wet AMD affects 26,000 people every year. It is the main cause of sight loss in Britain, and strikes rapidly: permanent blindness can occur only three months after diagnosis. “Many thousands have gone blind unnecessarily because of the delay,” says RNIB spokesman Bill Alker. “The cost of treating a patient who has gone blind is much greater than the cost of treating wet AMD with the drug Lucentis. It was an accounting exercise, not a clinical one protecting patients’ interests.”

The charity pulled out all the stops to overturn the Nice ruling, mounting a massive campaign with financial support from the pharmaceutical industry, and encouraging patients to protest to their PCTs. Nice eventually received more than 13,000 complaints — the biggest response to any issue — and agreed last year to undertake a review. In July, three pensioners suffering from wet AMD, backed by the RNIB, won a test case against Warwickshire PCT; the court ruled they should be allowed the drug, despite its cost of £10,700 per eye. The next month, Nice announced its U-turn. But Tom Bremridge, chief executive of The Macular Disease Society, is bitter it has taken so long to get help for his members: “Nice should be aware that during the two-year review process individual PCTs have had the power to decide whether to let patients go blind or save their sight. The stress and suffering has been cruel and un-necessary and hundreds of patients have been subjected to an appalling two years, during which many of them have lost their remaining sight.”

The Alzheimer’s Society is also engaged in a long-running legal battle with Nice. In 2005 Nice was expected to approve Aricept, a drug that greatly inhibits the symptoms of dementia; instead it decided the drug should no longer be available, despite its finding that it was clinically effective. “All the experts were baffled,” says Neil Hunt, chief executive of the Alzheimer’s Society. “Aricept is the only drug available, there is no other option, and it only costs £2.50 a day. It was a total shock when Nice issued a blanket ban.”

As a result of the furore that followed the decision, Nice relented — in part. In 2006 it ruled that patients who were already on the treatment could continue, but in future it would only be available to those with the moderate stage of the disease. “This means 100,000 people every year must wait until their condition deteriorates before being offered treatment,” says Hunt. “Research suggests patients who begin the treatment at a later stage never catch up with those who began earlier.”

Despite nearly 8,000 responses, numerous appeals, a protest march, the condemnation of the Royal College of Psychiatrists and an action in the High Court, Nice has stood by its decision. The Alzheimer’s Society argues it has not been given access to the “model” on which Nice based its decision, and the treatment’s wider benefits to carers and families might not have been considered. The drug companies Pfizer and Eisai took up the issue, and in May the Court of Appeal ruled against Nice, ordering the model to be made public. Last month Nice was refused permission from the House of Lords to contest the ruling.

The Society’s campaign is being supported by Cliff Richard, whose mother died of Alzheimer’s last year; Judi Dench, whose portrayal of Iris Murdoch in the movie Iris brought home the tragic reality of dementia; and the author Terry Pratchett, 60, who was recently diagnosed with an early-onset form of the disease. Speaking at the Tory conference in September, Pratchett likened the disease to a “slow-motion car crash… with always, at the back of your mind, the thought that sooner or later you will go through the windscreen”. He said it was “unthinkable” that early stage sufferers were being denied the treatment.

Controversy over new treatments is often followed by acceptance once the price goes down. Tamoxifen was discovered in the 1960s, but its initial cost, with fears about possible side-effects, meant it took more than 30 years to be widely used. But now many of its users, such as Toral Shah, can look to the future with confidence: “Today the drug is the gold-standard treatment, and the belief is that people like me should take it for up to 10 years, stop if they want to get pregnant, then start again,” she says.

Another breast-cancer drug, Herceptin, was the cause of some embarrassment to Nice in 2005 when Patricia Hewitt, then the health secretary, announced that all women diagnosed with early-stage breast cancer would be tested for suitability for treatment with Herceptin — before Nice had completed its appraisal of it. Hewitt’s intervention seemed to make a nonsense of Nice’s much-vaunted independence from the government. There are also rumours the current health secretary, Alan Johnson, might attempt to influence Nice’s January decision on kidney-cancer drugs.

“I think political realities will force Johnson to intervene,” says Professor Karol Sikora, former head of the World Health Organisation cancer programme. “They will disguise it in some way but I think by Christmas those drugs will be available. Otherwise it could cost too many votes.”

Nice’s chief executive, Andrew Dillon, firmly denies it. “Nobody can interfere with the independent-advisory committee. I can’t. Alan Johnson can’t. The only influence anyone can have is a reasoned argument over something wrong or flawed in the committee’s draft recommendations.” Dillon is passionate about his work, anxious to dispel the idea of Nice as a ruthless, death-dealing machine. “If you advise healthcare systems on how to use their resources where there is a clear and fixed amount of money available, decisions will be made that individuals or groups find hard to accept — understandably so,” he says. “But most of our guidance does not leave an aggrieved group somewhere, and most of what we produce is received positively by NHS professionals and patient organisations.”

A career health-service manager for 34 years, Dillon says he was not surprised by the furore following Nice’s interim decision on kidney-cancer drugs, though he admits the protest letter to The Sunday Times was unexpected. “I can understand why a small group of oncologists might say they feel it was wrong, but their responsibilities are to their individual patients; they don’t have a broader responsibility to make sure the money we set aside as a nation for healthcare is applied as fairly and efficiently as possible.” He is proud that Nice has gone from being unknown to having, he claims, significant credibility in the health service. Its reports, he says, “may not be perfect, but they are the very best interpretation of evidence that exists in the world”. If he’s seen as a bogeyman by some, he does not lose sleep over it. “This,” he says, “is the best job I’ve ever had.”

Additional reporting by Lois Rogers

and Charlotte Hunt-Grubbe

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