'You are not worth saving'

03:33, Jun 27 2013
Watene Waenga
DESPERATE MEASURES: Hamilton man Watene Waenga says he will have to shift to Australia to get access to a life-saving drug.

Imagine being told you have caught a rare disease that is going to kill you in about 10 years time.

Then imagine being told there is a drug that can prevent this, but you have to move overseas to get it because the Government refuses to fund it.

That is the scenario being faced by people living with the rare and fatal blood disease Paroxysmal Nocturnal Haemoglobinuria (PNH).

Watene Waenga
BATTLING RARE BLOOD DISORDER: Local rugby player Watene Waenga with his children. From left: Kaeya, Jah-vis and Kiahn

Among them are Hamilton man Watene Waenga and Matamata woman Natalie Butcher, who face enormous challenges staying healthy, including having to take regular doses of steroids and the anti-blood clotting drug warfarin.

PNH can trigger several conditions, including anemia and fatigue, abdominal pain or stroke.

About 35 per cent of sufferers die within five years of diagnosis, from blood clots or kidney failure.


Natalie Butcher
PNH SUFFERER: Matamata woman Natalie Butcher says the disease is a curse.

A drug that has proved very effective in keeping PNH in check - Soliris, also known as eculizumab - is available, however Pharmac, the Government's medicines funding agency, is proposing not to fund it because it is too expensive. Forty other countries, including Australia, England and the United States, are funding it.

Waenga, Butcher and the 18 other PNH sufferers in this country are pleading for Pharmac to change their policy - and now could be their best chance to do so, with Pharmac giving the public an opportunity to have a say on the decision-making criteria it uses to stock the national drug cabinet.

Butcher, who describes herself as a "good old Kiwi girl who likes to get out and about on the farm", said PNH was a curse.

"It's really nailed me down. It has just given me a bout of haemolysis, which is when the blood cells burst and break down. It was a real bad patch and I had to go on steroids to get out of it."

Butcher was diagnosed with PNH in 2007, after she began to get stomach pains which were revealed to be small blood clots associated with the disease.

She said it was "totally unfair" Pharmac was not funding the disease.

"We might be a minority, but it is pretty scary for us."

Waenga, a former Waikato rugby player, was diagnosed in 2009 and faces having to permanently relocate to Australia to get Soliris.

"I was always bleeding, always tired . . . my PNH levels are always going up."

PNH support group spokesman Daniel Webby is frustrated by Pharmac's refusal to fund the drug.

"Pharmac has attempted to present patients with an extremely rare disease as an unwanted burden to the health system. Unfortunately in the case of PNH sufferers, rejecting funding of the Soliris treatment is a death sentence."

PNH sufferers caught the disease through no fault of their own, he said. The average age of diagnosis is mid-30s, and the median survival is just 10 years.

"Soliris could prevent this and yet Pharmac are proposing not to fund it, at an estimated cost for eight patients of $4 million . . . People are struggling with a devastating disease and are essentially being told by Pharmac: ‘Your lives are not worth saving'."

Pharmac wanted to hear views on the availability and suitability of medicines, therapeutic medical devices and related products through public meetings. It is also seeking feedback through its website, pharmac.health.nz.

People can sign a petition calling for Soliris to be funded here.


Pharmac chief executive Steffan Crausaz says while he sympathises with the plight of people with PNH, it is not cost-effective to fund it.

"Pharmac is currently consulting on a proposal to decline funding . . . No decision has been made at the moment," he said, in response to inquiries.

"We recognise that a 'decline' proposal is not what patients with PNH are hoping for . . . [However] the price appears to be extreme and out of reach.

"At the current price the drug is not cost-effective and would be likely to be at the back of the queue."

While there was evidence of some clinical benefits of Soliris, even if it restored every patient to full health it would be almost 20 times less cost effective than the average medicine funded by Pharmac during the last financial year.

"Funding this treatment would probably mean around 40,000 patients missing out on health gains from other medicines - a reality of a capped budget where decisions have to be made to secure the best health outcomes."