Andrea Murphy says her life has been saved from a killer blood disease by an expensive "miracle" drug and is pleading for Pharmac to approve state funding of it for her and seven others - at a cost of $4 million.
The 52-year-old, who has the extremely rare blood and immune system disorder PNH, and two other patients have been given Soliris at no charge by supplier Alexion under a compassionate access scheme. Pharmac, the Crown's pharmaceuticals management agency, is deciding whether to support state funding.
Up to 20 people are thought to have PNH; blood specialists consider eight are candidates for Soliris.
The drug costs around $500,000 a year per patient or $4 million in total, which is 0.5 per cent of the Pharmac budget of about $750 million, based on eight patients.
The only other drugs Pharmac has approved at such a high price are enzyme-replacement therapies for three patients - one ongoing and two in the lead-up to a bone-marrow transplant.
But similar patients, for whom other drugs are available, have been denied state funding.
Blood diseases specialist Humphrey Pullon said Soliris was extremely effective as it transformed patients' lives. It prevented or reduced complications and prolonged survival.
Ms Murphy, from Dunedin, said she began suffering PNH some years ago. It laid her low with regular chest spasms and severe stomach pains.
After she suffered a blood clot, brain bleed, seizure and left-side paralysis two years ago, a Dunedin Hospital doctor managed to get her onto infusions of Soliris. "I haven't had a stomach ache since being on it. I would be dead without it. I've basically resumed a normal life with Soliris. I consider the treatment a miracle.
"I think it's a crime that there are other people at risk of dying and that they cannot get on this medicine."
"How can Pharmac deny terminally ill people this treatment?"
Last February, a Pharmac committee opposed Alexion's application for funding of Soliris. It was concerned by the high price and questioned some of the evidence of its benefits.
But the committee also suggested seeking an opinion from the agency's haematology group. Its minutes - still under wraps - will go to the committee's meeting next month.
A support group for patients with PNH said Pharmac was taking too long to decide, has begun a lobbying campaign, whose costs are met in part by Alexion.
The group's founder, Auckland artist Daniel Webby, 32 - who almost died from PNH complications - said the funding process did not recognise the rights of rare-disease sufferers.
"They have got a figure they say a life is worth and if it's more than that figure then they deny access.
"They need to recognise that for rare diseases, [drug] development costs are higher per patient. They need to put that into their budget and make sure people get ... life-saving treatments when they are available."
* Extremely rare blood and immune system disorder
* Called PNH (paroxysmal nocturnal haemoglobinuria)
* Sufferers lack a protective protein on red blood cells
* Red blood cell breakdown happens at a greater rate than normal
* Leads to anaemia, severe stomach pain and potentially fatal blood clots, stroke, organ failure.
* Soliris, also called eculizumab
* Costs around $500,000 a year
* Needed life-long.