A group of New Zealanders battling an ultra-rare blood disease are pleading for Pharmac to fund the world's most expensive drug to save their lives.
There are only about 20 cases of paroxysmal nocturnal haemoglobinuria (PNH) in New Zealand - a disease that causes the immune system to destroy red blood cells.
It can also cause anaemia, fatigue, abdominal pain, stroke and fatal blood clots, chronic kidney disease and vital organ damage.
Without treatment, one in three patients dies within five years of diagnosis.
Eight sufferers are asking Pharmac to fund the drug eculizumab (brand name Soliris), which is the only known treatment to slow the rate of blood cell destruction.
New Zealand is the only OECD country that doesn't fund the drug - the world's most expensive, at between $US440,000-$US500,000 ($NZ522,500-$NZ593,700) per person per year, according to reports by Forbes and the Financial Times.
That price has risen from $US409,500 in 2010.
However, Hamilton haematologist Dr Humphrey Pullon says Pharmac's failure to fund the drug is "a public scandal".
"It's critical that Pharmac considers the plight of those living with PNH by reaching an agreement with the company that supplies Soliris, to fund the medication for those whose lives desperately depend upon it."
PNH usually affects people in their mid-30s, but 10 percent start developing symptoms before age 21.
The cause of the disease is unknown and its diagnosis often takes years, Dr Pullon said.
Soliris' manufacturer Alexion Pharmaceuticals has applied for Pharmac to fund the drug for PNH.
Pharmac's primary clinical advisory committee recommended last February that the application be declined, saying a major issue was its cost.
Pharmac's haematology subcommittee is also considering the application and will report back to the primary clinical advisory committee next month.
In a statement, Pharmac says its decision-making process involves an assessment of clinical evidence, cost-utility analysis and analysis of the long-term affordability of funding decisions.
"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."