Group running for their lives

03:13, Feb 23 2013

A group of Kiwis suffering from an ultra-rare blood disease are running for their lives this weekend, hoping to draw attention to their fight for a drug they need to survive.

Palmerston North-born Natalie Butcher and Daniel Webby will be taking on this weekend's 160km Great Lake Relay in Taupo to draw attention to their campaign to get Pharmac to fund treatment of their rare blood disease, which will otherwise eventually kill them.

The pair will be taking it slowly. They are among a group of eight Kiwis suffering from life-threatening illness paroxysmal nocturnal haemoglobinuria (PNH).

Pharmac is in the process of making a decision on whether to fund Soliris, a drug containing Eculizumab to treat PNH. A distributor has made a bid to bring Soliris to New Zealand.

With a hefty price tag attached to subsidising the drug, PNH sufferers' families are pleading with Pharmac to follow in the footsteps of Australia and 40 other countries around the world that have approved Soliris.

Natalie's parents, who have already lost their son Logan in a tragic quad bike accident, are now fighting to save their daughter's life.


"We have already lost one child, we have been down this track and we don't want to go through it again, it's as simple as that," her dad, Wayne Butcher, said.

"I thought to myself, if you had a child who was dying, would you be talking about bean counting?"

PNH typically surfaces in adulthood and little is known about its cause.

It is a debilitating condition, attacking red blood cells to cause severe abdominal and back pain, as well as fatigue.

Sufferers face a future of blood clots, heart attacks, stroke and kidney disease if the disease is left untreated, and other methods such as surgery and use of blood thinning agents do little to prevent death, which is common within five years of diagnosis.

Ms Butcher said families of PNH would "fight hammer and tong" to get the drug.

"I have seen [Natalie] writhing in pain on the floor at the emergency department in the hospital. It is heartbreaking. Quite literally heartbreaking."

Ms Butcher lives with multiple sclerosis and said it pained her to see a treatment that could help her daughter remain out of reach.

The company that produces Soliris is already providing emergency treatment for the three worst cases in New Zealand.

Pharmac has estimated the cost of subsiding it is in the realm of $500,000 per patient a year for the rest of their lives.

Cost and questions over its efficacy were among the key factors underpinning a recommendation in February last year, that the application to bring the drug to New Zealand be declined.

A petition to get treatment for New Zealand PNH sufferers is at: make-soliris-available-to-new-zealanders/

Manawatu Standard