Waikato joins campaign for rare treatment

FIGHTING ON: Matamata's Natalie Butcher has a rare and potentially lethal blood disease and wants Pharmac to fund a drug that could save her.
FIGHTING ON: Matamata's Natalie Butcher has a rare and potentially lethal blood disease and wants Pharmac to fund a drug that could save her.

A Waikato doctor is going to war with government drug agency Pharmac for refusing to fund a treatment that could save Kiwis battling a rare blood disease.

Waikato District Health Board haematologist Humphrey Pullon has joined a campaign, launched today, to lobby for funding for Soliris - a drug that has proven effective treating Paroxysmal Nocturnal Haemoglobinuria.

There are currently 19 Kiwis with PNH, which develops without warning and can strike at any time.

One of those is Matamata's Natalie Butcher, 32, who was diagnosedin 2007 after suffering years of unbearable abdominal pain.

PNH can trigger a number of conditions, including anemia and fatigue, abdominal pain or stroke.

Some 35 per cent of sufferers die within five years of diagnosis from blood clots or kidney failure.

So far Pharmac has refused to fund the drug despite 40 other countries having done so, among them Australia, England and the United States.

Funding was declined in August, but medical director Peter Moodie yesterday told the Times a final decision had not yet been made.

"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," Dr Moodie said.

But Dr Pullon says it is "critical" the drug get even partial funding to prevent Kiwi deaths.

"If Pharmac opt not to fund it, there's no doubt about it that those patients severely affected from PNH in New Zealand will die."

While it was an expensive drug when broken down by individual patient, it was not a massive number given how few people were affected in New Zealand, Dr Pullon said.

"My plea [to Pharmac] is please don't run away from it, please look at the plight of these patients. This is a highly effective treatment."

Ms Butcher said PNH had forced her to rely on her family for support, as her constant fatigue compromised her quality of life.

"Mum is really the only person who truly understands the exhaustion and excruciating pain that I experience with PNH," she said.

"There are some nights when I just can't sleep because I'm in so much pain."

The symptoms had worsened in the past 12 months. While she was not at the stage of needing Soliris, she knew others who were and urged Pharmac to look at funding options.

"I don't want to be on death's door before finally accessing the treatment. People like me should not be discriminated against because we have an ultra-rare disease, as opposed to a more common disease.

"If money is the only reason that's preventing Pharmac from funding this treatment, then I simply don't understand how they can put a figure on someone's life."

Waikato Times