Glivec -- putting a pricetag on life

By MARTIN JOHNSTON

For Fran O'Keefe, life has become a battle against tiredness. Diagnosed with leukaemia in January, she is denied access to the best drug available - by the Government's health-rationing policies and her bank balance.

Her disease could be treated with Glivec, an expensive but highly effective drug hailed by doctors here and abroad as a breakthrough in cancer therapy.

But in New Zealand, it is only for the well-off, or the lucky 30 or so who receive it free from its maker, Novartis, mainly through clinical trials.

Instead of Glivec capsules, O'Keefe has injections most days of interferon alpha, which can have debilitating side-effects.

"I have a constant feeling of being sick and generally unwell all the time," says the 53-year-old health and safety co-ordinator from Green Bay in Auckland. "I have things like backache. Every day I'm just totally lethargic. I spend all my spare time sleeping."

O'Keefe, whose husband died from a brain tumour about five years ago, has chronic myeloid leukaemia.

She is one of 90 people for whom the Government has so far refused to buy Glivec, to the anger of the Leukaemia and Blood Foundation.

This month it launched a "Save the 90" campaign with election-style posters of the patients' faces, aimed at pressuring the Government.

The reason patients such as O'Keefe are not getting state subsidies for Glivec is that, since last year, the Government drug-buying agency, Pharmac, has been haggling with Novartis, trying to force down the price.

Pharmac's starting bid was $10,000 a year per patient. Chief executive Wayne McNee says Novartis has offered reductions on its earlier price of $60,000 to $100,000.

But even at the new price, which is a commercial secret, he says the drug is still not cost-effective compared with other treatments.

"It's just too expensive."

On Monday, Pharmac announced that it had deferred a final decision on paying for the drug. The agency says it was swayed by the many submissions criticising its proposal to buy Glivec for the handful of patients in the most-advanced stages of the disease and withhold it from those in the first "chronic" phase.

The reasons for delay may also lie in the workings of bureaucracy. Pharmac has asked the Ministry of Health for money for Glivec and expects to find out in a few weeks if it will be granted.

For leukaemia sufferers, the latest decision offers hope, but also frustration. Pharmac says that until the negotiations with Novartis are finished, it will not pay for the drug at all.

Chronic myeloid leukaemia (CML) is a cancer of the blood and bone marrow. It leads to a proliferation of abnormal white cells and, in some patients, enlargement of the spleen. About 70 new cases are diagnosed in New Zealand every year. It is usually fatal within five years if left untreated.

Because of Glivec's potential benefits, the ministry gave it fast-track approval last year, registering it in six months rather than the usual 12 for a new drug.

Novartis then applied to Pharmac to subsidise Glivec. The agency set up a cancer treatment subcommittee, which in February recommended buying the drug for patients in all three stages of the disease.

But Pharmac proposed paying for Glivec only for those in the accelerated or final blast crisis phases, and only under strict criteria.

The Leukaemia and Blood Foundation was dumbfounded at the idea of leaving out those in the chronic phase - the majority of patients and those most likely to benefit.

Leukaemia specialists said six to eight of the 90 patients were in the advanced stages. Under Pharmac's suggested criteria, only one or two would have been able to stay on Glivec after six months.

McNee says Governments around the world are finding Glivec too expensive.

"I'm not aware of it having public funding for chronic CML anywhere at this stage."

But Novartis and Auckland Medical School leukaemia specialist Professor Peter Browett say he is wrong.

Novartis New Zealand managing director Andrew Moore says the list of countries whose Governments buy Glivec for patients includes Scotland, Japan, Canada and a number of countries in Europe and Latin America.

In England, Wales and Australia, the drug is state-financed for patients in the advanced stages, but without the strict criteria proposed in New Zealand, he says.

Despite the tension between Pharmac and Novartis, both parties are keen to make a deal that would give the drug to patients in all stages.

Moore sees Pharmac's latest decision as a positive step.

"I think it's great, because they are now recognising Glivec is beneficial in the earlier stages."

He says Pharmac's single-minded pursuit of a lower price means it is taking too little notice of Glivec's benefits.

It has produced "phenomenal results", he says, bringing the counts of abnormal white blood cells - the best indicator of the presence of cancer - back to normal in up to 95 per cent of patients in the chronic phase.

"In addition, 84 per cent have a major reduction in their leukaemia cells, which means the cancer is literally disappearing from their body. A staggering 69 per cent go into complete remission."

But he maintains that Glivec's cost-effectiveness has been artificially weakened by Pharmac, which has compared it with hydroxyurea, a drug "so old it costs nothing".

Moore says the problem is that Glivec is registered for use only as a second-line therapy, for use only after interferon, a first-line therapy, has failed.

Novartis applied to register Glivec as a first-line therapy 11 days ago. The ministry says a decision will take until November at the earliest.

This application to extend the use of Glivec follows trial results, announced in May, showing the drug is nearly three times more effective than combination therapy using interferon and chemotherapy.

The Leukaemia and Blood Foundation's executive director, Jim Hamilton, is amazed that Pharmac is still concerned about the cost-effectiveness of Glivec.

"Pharmac knows very well that this argument is a nonsense. Glivec is three times more effective than interferon, and interferon puts one in three patients back into hospital.

"Glivec gets CML patients out of bed and working, saving the taxpayer thousands of dollars in associated loss of earning, hospital and medical charges."

But he says patients have been put in a terrible position, tantalised by the prospect of receiving the drug.

"In some ways it would be better that people didn't know there was a drug able to manage their condition."

Hamilton says withholding money for Glivec shows that New Zealand is heading for a Third World health system.

Terrence Aschoff, general manager of the Researched Medicines Industry group, says the Glivec saga shows that New Zealanders' state-funded access to new and highly effective drugs for cancer and other diseases that will come on to the international market will be increasingly restricted.

"And if the Government do provide reimbursement, it's targeted to specific categories of patients. Some will be in, some will be out. If it's not funded at all, access will be based on the ability of the person to pay."

The challenge is for the Government and pharmaceutical companies to reach commercially, financially and socially acceptable deals, he says.

O'Keefe considers it "very mean" of the Government to withhold money for Glivec.

She is in the accelerated phase of CML, but prefers not to ask her doctors how long she may live.

"I could get run over by a bus and it doesn't help me by having a number put on it."

She does know that with Glivec she could live to a "riper old age". Without it, she says, she could soon be joining her husband.

'Smart drugs' like Glivec offer targeted approach

nzherald.co.nz/health