Pharmac says it is very happy to negotiate a better price with a drug company for a treatment which could change the lives of sufferers of a very rare blood disease.
Eight New Zealanders currently have blood disease Paroxysmal Nocturnal Haemoglobinuria (PNH), a condition which claims the lives of one-in-three patients within five years of diagnosis if left untreated.
They want Pharmac to agree to fund a drug which has been shown to stop the symptoms of the disease and prolong sufferers' lives.
"This is devastating disease and anyone with the disease wants all the treamtent they can possibly have and I've great sympathy for them," Pharmac Medical Director Dr Pete Moody told TV ONE's Breakfast.
"At the same time with that sort of price we have to make a careful decision."
The treatment costs $500,000 per patient per year, and Moody said when the costs of a drug are that high careful consideration needs to given to its beneifts.
He said it would take up most of the funds Pharmac has to spend on new pharmecuticals every year.
"Plainly when the company made their first application they put in what they thought was the price, we're always happy to listen to them if they want to offer a better price, but its not just the price," he said.
"It's also the fact there of how much good does this drug do, and how good is the evidence that comes with it?"
Daniel Webby, who has the disease, said the evidence for the drug's effectiveness was as good as it can be for a treatment for a rare disease, citing an eight year study on 70 patients.
Moody said Pharmac's Clinical Advisory Committee had declined the drug on the basis of the information it had, but asked the haemotology sub-committee to make its own assessment.
"Because its a rare drug the evidence can be hard to obtain and we're still asking careful questions about the evidence," he said.
The Clinical Advisory Committee will meet again in February to discuss whether to fund the treatment.
'Living with a timebomb'
Speaking to Breakfast, Webby, the founder of the PNH Support Association of NZ, said complications from the disease could strike at any time.
"Essentially what happens is the bone marrow starts producing cells which are defective and the immune system recognises them as foreign and attacks them destroys them and that process is ongoing constantly."
The process creates a high risk of potentially fatal blood clots.
"It's like living with a timebomb you're certainly aware of the fact that these clots can develop at anytime."
Webby said he wants Pharmac to negotiate a fair price for the treatment, which is availble in 40 other countries around the world.
"We're the last in the advanced OECD to fund this treatment," he said, "but what (Pharmac is) saying to us is they are not going to fund it based on a cost which they yet haven't established."
"As a father this is something that concerns me I want to see my son grow up, but I'm not the only patient in the country."
Haemotoligist Dr Humphrey Pullon said the drug, called Eculizumab or Soliris, is an extremely effective medication for people living with PNH.
"It transforms patients' lives; it improves their sense of wellbeing; prevents and reduces complications of the disease and ultimately prolongs survival," he said.
"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."
Pullon says PNH develops without warning and can strike men and women of all races, backgrounds and ages.
"On average, PNH affects people in their mid-30s. However 10% of all people living with PNH start developing symptoms before age 21. We don't know why people develop PNH.
"The disease often goes unrecognised, with diagnosis sometimes taking more than 10 years," said Pullon.
He says PNH can trigger various health complications including anaemia and fatigue, abdominal pain, stroke and fatal blood clots, chronic kidney disease, and vital organ damage including bone marrow failure.
PNH Support Association of NZ
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