Plea For Access To A Leukaemia Drug
28 June 2002
New Zealanders Plea For Access To A
Leukaemia Drug That Will Save Their
One doctor has said funding Glivec only for late stage CML is like a fireman refusing to turn his hose on until the house has nearly burnt down.
Rudolph Wenk has embarked on a gamble for life.
The 62-year-old is paying $6000 a month for a drug to combat his blood cancer in the hope that he will at least achieve a remission in 12 months. After that his money will run out.
"Hopefully I'll perhaps buy myself another 10 years, if not ...." his voice trails off. "I'm able to do this because of a small legacy received recently by my wife. Otherwise we have very little."
Mr Wenk of Wellington is one of about 30 New Zealanders taking the drug Glivec which is proving effective against chronic myeloid leukaemia (CML), a form of blood cancer diagnosed in about 40 - 50 new cases in New Zealand each year. CML is usually fatal in about three to five years.
Like so many others taking Glivec, Mr Wenk has had a remarkable response. He's had CML for seven years and says he was an invalid before he started taking Glivec. In less than three months his blood count is near normal and he's hoping to get back to work.
"I never thought I'd feel so well again."
However, he is the only one paying for Glivec. The others receive their daily dose, taken in pill form, free. It is supplied by the makers, the international drug company Novartis, under what it calls an "expanded access programme," a compassionate use programme, while it negotiates for payment with the Government drug buying agency, Pharmac.
But Pharmac is only considering funding Glivec for patients in the most advanced "accelerated phase" and "blast crisis" stages of CML, whereas all medical case evidence points to it being most effective in the so-called "chronic" early stage of the disease.
Pharmac's argument is cost although it says it will continue to review chronic stage administration of Glivec and has called for submissions from "interested parties".
"I'm one of those," says Mr Wenk. "I've also contacted my MP, Peter Dunne, and am trying to speak to [Minister of Health] Annette King. It would help me a lot if they'd just relent and let the cost of my standard drug [interferon] which has failed me, to be put towards the cost of Glivec. That would about halve my monthly bill."
The chairman of the board of trustees of the Leukaemia and Blood Foundation, Douglas Strong, says that for Pharmac to suggest that only patients in the most advanced stages get Glivec, was to deny access for those in whom the drug was most effective. He says Pharmac's own specialists strongly recommended administration of Glivec to patients in the early stages of CML.
Glivec is sometimes called a miracle drug, but Novartis emphasises it is not a cure for CML. However, the most recent results, announced last month, show that when Glivec is given as initial therapy to newly diagnosed patients in the early or chronic phase of CML, 96 percent achieve normal blood counts. Even more astoundingly, 84 percent of patients achieve a major cytogenetic response where there is a significant disappearance of cancer cells. This makes it three times more effective than the standard treatment used at present, interferon.
Wanganui Hospital radiographer Helen Pickering has been declared "in remission" after battling CML for more than seven years. She started taking Glivec in October last year and got the glad news from her haematologist three months ago.
"As far as I'm aware I'm the first to be declared in remission so quickly, within three months of starting on Glivec," says Mrs Pickering (47) who has four teenage children.
"This was a milestone for CML because before Glivec you either were cured with a successful bone marrow transplant or stabilised with the standard therapy. Remission was unheard of."
"All I can do is hope I continue to receive Glivec because there's no way we could afford to pay for it ... I can only hope that Pharmac and Novartis can arrive at an agreement and the drug is provided to all who can benefit from it."
Paul Goodwin is another successful Glivec patient. The Gore farmer and father of six children, aged three to 16 years, started on Glivec in February and after three months he has gone from chronic ill health to working full days back on his farm. His most recent bone marrow test this month showed no evidence of any abnormal cancer cells in his bone marrow.
Ray Braddock, of Taupo, was in a wheelchair at the beginning of November last year, residing in a rest home because his wife could no longer cope. Over three and a half years, he'd progressed to the advanced blast crisis stage of CML and his doctor thought he'd be dead by Christmas. As a last resort he was prescribed Glivec.
Today he's back home, out of his wheelchair and rated as chronic.
"That may sound bad to a layman but the result is a miracle ... But there's no way I could afford to pay for Glivec. I'm just lucky to be getting it free from Novartis. What worries me are the 80 or more CML sufferers out there who're suffering so greatly because they can't afford to pay. It's so unfair ... and we've made submissions to Pharmac along these lines."
Doug Strong of the Leukaemia and Blood Foundation has also been lobbying Pharmac, policymakers and politicians in Wellington on behalf of patients over the past few months.
"It's beyond me that [Pharmac] continues to ignore the advice of independent specialists on the effectiveness of Glivec," says Mr Strong. "Under Pharmac's proposals only about 8 or 9 patients would receive Glivec free whereas it should be made available to between 80 and 90 CML patients in the chronic stage when it is most effective...
"It just doesn't make sense in terms of economics. Consider how much better is it to have CML patients take Glivec and be capable of looking after themselves and work than to administer the ineffectual [interferon] free to people who continue to be sick, on social welfare and hospitalised at a huge cost."
While Glivec is not promoted as a cure for CML, it's hoped that once control has been achieved in patients and blood counts returned to normal the dose may be reduced and the estimated cost of $60,000 for chronic phase patients lowered.
At the same time, interferon may cost more than $30,000 a year but the mean was more like $20,000 a year simply because most patients cannot tolerate the drug at the highest recommended level.
"I would say that due to the fact that they [Pharmac] are pleading budget for limiting the supply of Glivec to only the most serious, they are being extremely cynical. However, Pharmac is required to do the best for the taxpayer but not by the abrogation of their responsibility to provide the best drugs available at the best price obtainable. Glivec is the best treatment yet for CML by far and without it New Zealanders will die - unnecessarily."
The New Zealand trials with Glivec were led by Professor Peter Browett of the Auckland School of Medicine.
He says the results of the Glivec trial are different from those of many other drugs in that often the early promise of a new therapy became less marked over time when compared with the standard treatment.
"Quite the opposite is happening with Glivec and that's very exciting," says Dr Browett. "The excellent results seen in the trial are not only being maintained in the majority of patients, but the responses are continuing to get better and better with time."
Certainly, that's the case with Hugo Geluk, of Paekakariki. For the past eight months, 62-year-old Mr Geluk has received Glivec which has dramatically returned his white blood cell count to normal. That was after three years when his interferon treatment failed.
Like other patients, he says he can't afford to pay for Glivec and, in effect, his life is in the hands of Pharmac's administrators. And if Pharmac does fund patients with advanced disease, he will miss out because he's not sick enough.
"I suppose it's a question of am I worth $60,000 a year," says Mr Geluk. "My family and friends think I am, my grandkids do. But I can't afford $60,000 a year for Glivec and that'd be beyond the ability of most people.
"I can see why Pharmac is kicking against the price but then again, it costs more in a year to have me in hospital. Then it costs even more to have a person in jail. I don't know where the justice is in all this. If they cut many of us off from Glivec it really will be a death sentence ..."
Doug Strong - 025 673 0787 Chairman Leukaemia and Blood Foundation