Three-Year-Old unable to access Life-Saving Treatment in NZ

Three-Year-Old Girl unable to access Life-Saving Treatment in New Zealand

A little Kiwi girl currently living in Texas with her Kiwi parents is unable to live in New Zealand because the medicine she needs to survive is not available to her here.

Supporters of three-year-old Molly have written to the Prime Minister, Jacinda Ardern, in an effort to get the treatment made available in this country.

Molly has Pompe Disease which falls under the Muscular Dystrophy group of diseases.

According to the President of the New Zealand Pompe Network, Allyson Lock, the disease has no cure, but a treatment called Myozyme is available to help stop the progression of the disease. This medicine has proven to be life-saving in nearly eighty other countries. "The problem is," Mrs Lock said, "Pharmac refuses to fund it".

Pompe Disease is an extremely rare disease, with only eleven people diagnosed with it in New Zealand. It is treated with a fortnightly infusion of an artificial enzyme replacement that the body uses to break down a complex sugar called Glycogen. Without the enzyme the sugar builds up in muscle cells causing them to deteriorate. Most people with Pompe disease, left untreated, will die of respiratory failure as the diaphragm (the breathing muscle) is destroyed.

Molly is currently receiving the life-saving infusions in Texas. Molly's mother Amanda has seen great improvements. According to Amanda, "Since starting treatment, Molly’s endurance has improved, she began meeting milestones and has been really working hard at tackling stairs. Before treatment she could only crawl up the stairs now she tries hard to do them by herself."

Pharmac funds Infantile Onset Pompe Disease (IOPD) patients if they are diagnosed before the age of two. If a patient is diagnosed after the age of two they will be declined treatment. There are zero New Zealanders diagnosed with Infantile Onset Pompe Disease, but eleven unfunded patients with Late Onset Pompe Disease (LOPD).

Unfortunately, Molly was diagnosed after her second birthday.

Mrs Lock says, “Pharmac have turned down numerous applications for Myozyme to be funded since 2006. This treatment is available in almost eighty countries. There is a proven and documented 59% lower mortality rate in patients on Myozyme.

“We realise that Pharmac has a budget, but there seems to be a never ending open budget for welfare. New Zealand surely isn’t so broke that we have to pick and choose who we let live and who we let die. But that is currently where we find ourselves”.

"It is heartbreaking that essentially Molly and her parents are in medical exile. It is shameful that our socially progressive country allows this situation. We are hoping the Prime Minister shows some of the family values and compassion she is known for and ensures people with these fatal diseases are treated".

Every letter we have written over the years to beg for our lives goes on a merry-go-round. The Prime Minister says it falls under the portfolio of the Health Minister. The Health Minister says they can’t interfere with Pharmac. Pharmac decline to fund citing “not enough evidence the drug works”, “it hasn’t had long enough studies done”, “it’s not a cure”, and many other excuses.

Pharmac fund hundreds of drugs that aren’t a cure. Myozyme has been an approved drug since 2006 (13 years!). Pharmac fund many drugs which have been around a far shorter time. There are many studies proving the efficacy of Myozyme and we have provided Pharmac with letters and peer reviewed articles from world experts telling of the importance and positive impact Myozyme has. All of this has been ignored.

In New Zealand you have a price on your head, and in many cases the government stands by and does nothing while Pharmac (which is a government entity) leaves us to die.

We are not saying to Ms Adern “please interfere with Pharmac”. We are asking the Prime Minister of our country to make sure citizens aren’t left to die when people in other countries are deemed worthy of treatment.

www.nzpompe.network

End of release.

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