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Rare Disease Day 2020

New Zealand Pompe Network

Allyson Lock, MNZM

An open letter to New Zealand Prime Minister, Jacinda Ardern

“Know us by our deeds”. This was the headline next to your picture on the cover of Time Magazine, for all the world to see.

I saw it. I’m a Kiwi living in New Zealand. I feel like I should have been proud to see our PM on display with her concerned and sympathetic look, portraying to the citizens of the world what a caring and empathetic person she is...

I have a very rare disease called Pompe disease. There are just 12 of us in NZ.

Pompe disease is a rare, inherited and fatal disorder that disables the heart and muscles. It is caused by mutations in a gene that makes an enzyme called alpha-glucosidase (GAA). Normally, the body uses GAA to break down glycogen, a stored form of sugar used for energy. But in Pompe disease, mutations in the GAA gene reduce or completely eliminate this essential enzyme. Excessive amounts of glycogen accumulate everywhere in the body, but the cells of the heart and skeletal muscles are the most seriously affected. Researchers have identified over 300 different mutations in the GAA gene that cause the symptoms of Pompe disease, which can vary widely in terms of age of onset and severity.

Fortunately there is a treatment for Pompe disease available in 76 countries, including Australia. Unfortunately, New Zealand isn’t one of those countries. PHARMAC has continuously declined funding of Myozyme for adults with Pompe disease since it was approved for use back in 2006. PHARMAC has agreed to fund Myozyme for use for Infantile Onset Pompe Disease, but NZ doesn’t have any patients who fit the criteria. NZ is also reluctant to initiate newborn screening for Pompe disease, so an infant is unlikely to be diagnosed, meaning that baby will die within the first year of life. Meanwhile, other countries are rolling out newborn screening for Pompe.

Leading up to the last election, we asked each party these 3 questions:

  1. If elected in this upcoming General Election, will you support Pompe Disease patients by pressing for PHARMAC to fund the treatment?
  2. Will you apply your voice and vote in Parliament to ensure sufficient funds are available for PHARMAC to approve the funding of Myozyme?
  3. Do you think, in a first world country like New Zealand, medicines for fatal diseases should be withheld from sufferers?

These are the stances of the current coalition party members:

Labour Party - David Clark. “Labour's policy of establishing a rare diseases fund would include Pompe disease. We acknowledge that the Pharmac model does not provide for those with rare diseases. This fund would have consumer representatives on the committee so the need for medications to treat Pompe disease would be clearly outlined”.

NZ First - “We have committed to two things, we understand the system is not working and hence we have called for a review of Pharmac’s funding policy. Secondly, I quote from Ria Bonds (Health spokespersons NZF) “New Zealand First will set up a separate pharmaceutical fund for sufferers of rare diseases to be administered separate to PHARMAC.”

As the last answer, we need both a review of Pharmac funding policy and a separate fund for such diseases.

No, definitely not - medicines should not be withheld for fatal diseases”.

Greens - “We have deep concern for any patient who can’t access treatment.

We do support a separate fund for people with rare disorders, to be allocated according to need (rather than Pharmac's current criteria). This fund should make use of Pharmac's negotiating and purchasing leverage but decisions should be made independently.

Overall funding for pharmaceutical access is inadequate to meet the cost of new medicines with potential benefits. National have a history of doing just enough without solving the bigger problem. This includes the latest injection of money in Budget 2017. We would fund the pharmaceutical budget at a higher level to help clear the backlog of recommended medicines”.

In October 2019, Deputy Prime Minister Winston Peters said on “The Project” TV program, “Right now…we know we are not spending nearly enough on pharmaceuticals and first world drugs…the reality is that PHARMAC’s budget is not big enough and we have to face that as a country and make some accommodations to give people the treatment and first world drugs that they deserve. We have a $7.5 billion surplus, so let’s take a look at PHARMAC’s funding to ensure that people get the treatment they need, at the time that they need it.”

New Zealand spends considerably less on health than other OECD countries. Numerous people have written to you, Prime Minister, regarding the underfunding of PHARMAC. We get fobbed off to the Minister of Health, David Clark. Then he fobs us off to PHARMAC who in turn sends us back to David Clark. It’s a nightmare merry-go-round that never ends - and believe me, there is nothing merry about it.

People with rare diseases have been abandoned by successive governments, I was hoping you would be better than that. I voted for you. When will you abide by your own party’s pre-election promise/policy? “Labour's policy of establishing a rare diseases fund would include Pompe disease. We acknowledge that the Pharmac model does not provide for those with rare diseases. This fund would have consumer representatives on the committee so the need for medications to treat Pompe disease would be clearly outlined”.

As Rare Disease Day rolls around yet again, it serves as a reminder that the people who have the power to do something for those suffering with a rare disease, continue to ignore what is happening in our own country. If the Health Minister believes that pulling a weed from the concrete in Dunedin signifies the beginning of a new hospital then it would be fantastic if he would stick a spade into PHARMAC and create new life from the compost heap it has become. Contrary to popular belief, PHARMAC is not the envy of the world. If it was, why don’t other countries emulate it?

Prime Minister Ardern, I “know you by your deeds”, and I am ashamed of you, because as our Prime Minister you choose to ignore people who desperately need life saving medicines. I am NOT asking you to decide which medicines PHARMAC funds, I am asking you to make sure PHARMAC has adequate funding so that PHARMAC doesn’t have to make a decision on who lives and who dies. You have the power to change that!

This Saturday, the 29th of February 2020 is Rare Disease Day.

Rare Disease Day takes place on the last day of February each year. The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients' lives.

The campaign targets primarily the general public and also seeks to raise awareness amongst policy makers, public authorities, industry representatives, researchers, health professionals and anyone who has a genuine interest in rare diseases.

Building awareness of rare diseases is so important because 1 in 20 people will live with a rare disease at some point in their life. Despite this, there is no cure for the majority of rare diseases and many go undiagnosed. Rare Disease Day improves knowledge amongst the general public of rare diseases while encouraging researchers and decision makers to address the needs of those living with rare diseases.

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