Living under "a blanket of fatigue" and facing the prospect of a shortened life, Dion Bonner is seeking access to the treatment he desperately needs.
The Christchurch man is among eight Kiwis with a rare blood disease appealing to Pharmac to subsidise the medicine that could save their lives.
Bonner suffers from paroxysmal nocturnal haemoglobinuria (PNH), which destroys red blood cells and can lead to blood clots, high blood pressure and organ failure.
Soliris, a treatment that reduces the rate of blood-cell destruction, is funded in more than 40 countries, including Australia, England, Canada and the United States.
Pharmac does not currently fund the drug, which was approved for use in New Zealand by regulator Medsafe in September 2011.
Soliris costs about A$400,000 (NZ$0.5 million) a year for each patient.
Pharmac medical director Peter Moodie said the application, made by Alexion, was being assessed through the usual processes.
The Pharmacology and Therapeutics Advisory Committee last year declined the application for Soliris, also called eculizumab, as it was not impressed with the quality of clinical evidence. The application was referred to the haematology subcommittee, and the advisory committee is due to hear back from the subcommittee next month.
These committees provide recommendations to Pharmac, which then makes the final decision.
Bonner, a 28-year-old data analyst, was diagnosed with the disease in 2006 after a bout of fatigue led him to visit his GP.
"Walking to the bus stop or even getting out of bed, I'd be tired, and I also had a very pale appearance."
He has to have blood transfusions every six to eight weeks to build up his red blood cells, and he takes anti-coagulants to reduce the risk of a fatal blood clot.
"I'm constantly living under a blanket of fatigue. I just don't have energy a lot of the time." Bonner said the Government should fund the treatment to save patients, who had to deal with the likelihood of a significantly shortened life. "I understand there are cost limitations in the health sector and that they have to make the numbers work, but people's lives are more important than getting to a number," he said.
Pharmac medical director Peter Moodie said the organisation had received a funding application for Soliris, with no decisions made.
He said the decision-making process was based on an assessment of clinical evidence, weighing the cost of the drug against how long it would extend the lives of the patients, and the medicine's "long-term affordability".
The country's limited pharmaceutical budget meant it had to "carefully prioritise" which medicines it bought, he said.
- The Press
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