Sufferers plead for life-saving drug

20:46, Jan 23 2013
FIGHTING: Natalie Butcher is fighting with Pharmac for funding for her medical treatment.

Kiwis living with an ultra-rare and deadly blood disease are pleading with Pharmac to approve funding for an expensive drug that could save their lives.

"We needed this drug yesterday," said Palmerston North-born Natalie Butcher, among a group of eight Kiwis suffering from life-threatening illness Paroxysmal Nocturnal Haemoglobinuria (PNH).

They are campaigning for Pharmac to subsidise Soliris, a drug containing Eculizumab currently available in about 30 countries around the world, including Australia.

A distributor has made a bid to bring it to New Zealand. Pharmac is in the process of making a decision on whether to fund Soliris.

Former Palmerston North man Daniel Webby, 32, is fronting the PNH Support Association of New Zealand's campaign for access to treatment for the rare disease attacking his red blood cells.

PNH typically surfaces in adulthood and little is known about its cause. Mr Webby described a debilitating condition, causing severe abdominal and back pain, as well as fatigue. Facing a future of blood clots, heart attacks, stroke and kidney disease if his PNH is left untreated, he wished to be treated with Soliris so he could watch his 6-year-old son grow up.


"I don't feel I have a future that is certain, I feel angst when I think about whether I will see my son grow up, not being sure about what legacy I can offer him . . . it's hard to know that there's a treatment that could give me my life."

Pharmac has estimated the cost of subsiding Soliris would be in the realm of half a million dollars per patient a year for the rest of their lives. Cost was among the key factors underpinning a recommendation in February last year, that the application to bring the drug to New Zealand be declined.

Mr Webby held hopes money would not stand in the way of saving lives.

"For me it's about health equity, it's about fairness of access. The cost of medicine is a small fraction of the healthcare system . . . we are still funding medicine for people with health problems linked to their lifestyles - who are overweight, who smoke - I feel that we have the same right to healthcare as any New Zealander," he said.

Peter Moodie, Pharmac medical director, said the application to introduce Soliris was now facing further review under the Crown entity's normal process.

"Pharmac is always interested in reviewing new medicines and prioritises funding for those medicines with good evidence of benefit and which are cost-effective. The pharmaceutical budget is limited, so funding needs to be carefully prioritised to ensure it is spent on medicines with the greatest benefit for New Zealand patients," he said.

There are three New Zealand PNH patients receiving compassionate supply of Soliris from the manufacturer.

Mr Webby had spoken to people receiving the treatment and yearned for the quality of life they had regained. "It's quite exciting, to be honest, the thought of what's just sitting on the shelf," he said.

Similar battles for funding for Soliris had already been won in Australia, Canada and the United Kingdom. Ms Butcher said they had been bolstered by the success of Kiwi breast cancer sufferers' three-year battle to get funding for Herceptin, which was eventually approved in 2008.

"They had a lot more people to fight their fight, this disease is extremely unusual, there's so few of us fighting for it our voice may be forgotten."

A petition to get treatment for New Zealand PNH sufferers can be found here.

Manawatu Standard