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Government breaks promises to Pompe disease sufferers

5 July 2019

The New Zealand Pompe Network (NZPN) recently presented a petition at Parliament asking that the House of Representatives urge PHARMAC to fund Myozyme for Late Onset Pompe Disease (LOPD), and also urge the Minister of Health to provide additional funding to Pharmac to enable the drug to be subsidised.

Mrs Allyson Lock, President of NZPN said, “A submission to provide the Health Select Committee with background information about the matters outlined in our petition has also been provided. We will also present an oral submission in front of the Health Select Committee at Parliament on the 7th of August.

Our petition asks that the drug Myozyme be funded by PHARMAC and that the Minister of Health and the Prime Minister, increase the PHARMAC budget so that this drug can be purchased. More people will die without it. Maybe our government is ok with sacrificing a few for the greater good, but New Zealand can do better than that. If this government won't change things, who will? How many more have to die?

Mrs Lock also said “We are not asking politicians to make decisions on the efficacy of medicines. We are asking that politicians ensure that PHARMAC is funded adequately to make sure no person is left behind to die, when if they lived in other countries they would be given best class medicines”.

“I was diagnosed with Pompe disease in 2010 and have been declined by Pharmac on several occasions for the life saving drug, Myozyme. My pleas for treatment bounced from PHARMAC to the Prime Minister, to the Health Minister to PHARMAC and kept going around in circles with no one wanting to help me, or the other 11 people in New Zealand with this fatal disease.

While everyone sat on their hands refusing to help, one of our patients died. Laurie Hill was 54 years old. He was a Dad, a teacher and a counsellor. His death was preventable. But no one cared. Well, lots of us cared - his family, his friends. But we didn't have the power to grant Laurie access to treatment. Our government did, but it failed him.

Fortunately, after the numerous declines from PHARMAC, the company which makes Myozyme gave us compassionate access under their International Compassionate Access Program. This program is geared toward countries where the governments are poor or have third world health systems. Kiwis are now part of this program. I'm lucky that someone cared enough to help, but it wasn't anyone in New Zealand. It wasn't our government, or PHARMAC, which as we all know is a government entity.

My cousin has Pompe disease, she lives in Australia, she got treatment without having to beg her government for her life. That's how it should be".

What is Pompe Disease?

Pompe disease is a rare, inherited and often fatal disorder that disables the heart and muscles, including the diaphragm muscle which drives breathing. 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.

“Pompe disease is a terrible disease that robs us of our lives, our dignity and the ability to perform everyday tasks that most people take for granted. Without treatment, most will die early of respiratory failure, preceded by years spent in a wheelchair, or bedridden; being fed via a stomach tube due to the lost ability to swallow; breathing through a tube inserted into the front of our throat due to the loss of function of the diaphragm which drives our ability to breathe naturally. As there is no funding available (and most of us couldn’t afford to pay for it), there is no option for people on mechanical ventilation to stay in their own home. We must live in a hospital facility for the duration of our lives, even someone who might be in their 30s with young children”.

PHARMAC and our “hands off PHARMAC” government, refuse to fund the only medicine available to treat Pompe disease. A medicine that is available in 75 other countries. It is funded in all OECD countries except New Zealand, Austria, Iceland and Turkey. Don't we always pride ourselves on being the best in the world? Or is that only true of rugby?

Recent years have seen many wonderful new targeted drugs and immunotherapy medicines that are now international best practice treatments. The United States Food and Drug Administration (FDA) has approved these drugs following stringent peer reviewed studies, but PHARMAC refuses to approve many of them. It insists its own experts need more evidence. The idea that a small panel of people at the bottom of the world (and has no expertise on Pompe disease) is a higher authority than the FDA is farcical, and extremely dangerous.

PHARMAC is proud of the money it saves. It is proud that our expenditure on medicines is bottom in the OECD. PHARMAC, and our government would have the public believe that their low expenditure still gets New Zealand good medicines that the rest of the world gets, but at a much lower cost. Yes, it does get medicines at a much lower cost, but they are not funding many many better medicines that other countries do fund. Meanwhile, ordinary people are being forced into poverty to buy drugs that will give them longer and better-quality lives.

PHARMAC's lack of understanding of Pompe disease has CEO Sarah Fitt commenting that Infantile Onset Pompe Disease is not the same condition as Late Onset Pompe Disease. It is the same disease. How can someone in that job be so ill informed and lacking in knowledge, and still be allowed to make decisions on whether we live or die? Sarah Fitt also commented that they sometimes disregard the advice of its expert clinical committees. So why have expert clinical committees?

Sarah Fitt says PHARMAC bases decisions on evidence, and they take their task seriously. Yet they have been given evidence (by NZPN) from experts on Pompe disease from around the world which has been totally ignored by the Rare Disorders sub-committee and PTAC. Somehow PHARMAC don’t seem that serious.

Sarah Fitt says the savings from PHARMAC are used to invest into more new medicines, yet recently $200 million in savings was taken by the Health Minister, David Clark, and given to nurse wage rises.

David Clark also told me “While some medicines may be available in other countries, the funding and reimbursement systems are often not comparable. New Zealand must make its own decisions, carefully assessing the available evidence and thinking about medicines use in the New Zealand health context. I assure you that the Ministry of Health and I are committed to improving health outcomes for all people with rare diseases.”

So that being the case, why does PHARMAC often decline funding of drugs with one of their excuses being “(*Insert country name here*) doesn’t fund it”? If we are not interested in what other countries do, why does New Zealand spend hundreds of thousands of dollars per year sending delegations to countries all over the world?

When the Health Minister assures me that he is committed to improving health outcomes for all people with rare diseases, why doesn’t that include people with Pompe disease? And what is “the New Zealand health context” exactly? Sacrifice a few for the greater good?

When Jacinda Ardern was confronted with the difference between Australia and New Zealand for funding of drugs, Ms Ardern said Pharmac won't purchase the newest products on the market if they don't "add anything that a past product" already has. Really? If you have Pompe disease you can’t even get that “past product”!

The way the government has set up PHARMAC, and the way its budget has been set creates a substantial medicines waiting list which Sarah Fitt says is a tool for getting a cheaper deal for medicines. If a company knows how far up the list they are, they are less likely to drop their price. Meanwhile, people are dying while the tightfisted PHARMAC are waiting for a better deal. Why are PHARMAC tightfisted? There's not enough money in their budget. Who is responsible for that? The Health Minister, and, overall, the Prime Minister. The Health Minister signs off on the budget allocation that PHARMAC is to manage, which decides the fate of a number of drug purchases that will or will not occur, according to the amount available for new purchases. This in turn decides the fate of many people who will die without treatment.

There has been talk of an early access scheme for medicines. How will that help people with Pompe disease? An early access scheme won’t help get a drug which has been around for 13 years.

PHARMAC have several excuses for not funding Myozyme -

- Not enough long-term evidence the drug works. But funds other drugs which have been around for a much shorter time.

- Not enough evidence that it works well enough for the price it costs. Well, it either works, or it doesn't work. In the case of Pompe disease, there are no treatment options other than Myozyme. And experts say that it does work.

- Canada and the UK don’t fund it. Yes, they do.

- Not enough evidence from trials conducted prior to FDA approval of Myozyme. But recently funded a drug that is not yet FDA approved. The name of

Mrs Lock says, “If I drove drunk, crashed my car, killed my friends and disabled myself, you would send an ambulance, scrape me off the road, pick up the tab for my rehabilitation, give me a brand new Mercedes van modified specifically for me (see them at Braiden International, Carterton), pay for home modifications and all of my needs would be taken care of. But I have done nothing wrong, and I am being denied care. Where is the ambulance for Pompe sufferers? When someone is hurt in an accident you make the choice to pick them up because it is the right and humane thing to do. When someone is hurt due to an illness, you toss up whether or not we are worth saving, and so often you make the choice not to help us. Why?

PHARMAC told me that the Rare Disorders Sub-committee is allowed to make decisions about recommending/declining funding a drug taking cost into consideration. Shouldn’t they be there for their clinical experience only?

To get New Zealand treatment options back into the first world, the drug approval function should be removed from Pharmac and given to independent expert clinicians. PHARMAC’s role should be limited to negotiating the best deal on the drugs it is told to buy.

We don’t have a cap on social welfare, prisons, ACC. Why do we have a cap on the medicines budget, which is resulting in the deaths of people who can’t survive without the medicines other countries get?

The following is a press release by Grant Robertson while he was in opposition in 2011.

“Government breaks promise to rare disease sufferers

Tuesday, 18 October 2011, 4:03 pm

Press Release: New Zealand Labour Party


Health Spokesperson

18 October

Government breaks promise to rare disease sufferers

The Government’s refusal to explain why it has not delivered on a promise to ensure access to life-saving drugs for Kiwis suffering from rare diseases is cowardly and disrespectful Labour’s Health spokesperson Grant Robertson says.

“A group of people suffering from Pompe disease, along with their representatives, arrived at Parliament today after repeated requests for meetings with John Key and Tony Ryall had been turned down.

“They asked to meet someone – anyone - from the Government and they were turned away. Whatever the difficulty of dealing with this policy issue, the Government should show some respect and meet with these people.

“I met them and the Government needs to do the same. It needs to listen to their concerns, because they are not going to leave this alone,” Grant Robertson said.

“Three years ago the in-coming National Government told these people – who suffer from a rare, progressive disease - that they would fix the problem of getting access to a highly specialised medicine, used to treat 1500 people in more than 45 countries, but not available here.

“That promise has been broken. No one is pretending this is an easy issue to deal with, but the Government set the expectation and it has not delivered.”

“Today’s meeting confirmed that these people understand the value of the Pharmac model to New Zealand in terms of the bulk purchasing of drugs. However their concern is that the policy settings that Pharmac operates under are not appropriate for measuring the benefits of these medicines.”

“I made it clear that while I don’t believe politicians should be choosing what medicines are funded, we do have a moral responsibility to ensure that we set the framework that sees patients treated fairly and humanely.

“The Medicines Strategy sets the framework for how highly specialised medicines might be managed. The Government should do all it can to implement that strategy.

“In the meantime, the least John Key and Tony Ryall can do is show some respect by meeting with these people and discussing their concerns,” Grant Robertson said.


Mrs Lock says “Grant Robertson didn't see fit in this year's Wellbeing Budget to do anything at all for sufferers of Pompe disease, or any other rare disease. Are we surprised? No. Sadly, no”.

In the Wellbeing budget, PHARMAC was allocated an additional NZ$10 million a year over four years. This does not even keep pace with inflation. This means there is no new money in this year's budget for PHARMAC so the situation will continue to worsen as the number of medicines on the waiting list will continue to grow.

1 July 2019 marked the beginning of a new financial year for PHARMAC and the publication of a new Pharmaceutical Schedule. We would not expect to see many, if any, new listings for the next 12 months.

In the lead up to the last election NZPN asked all political parties, and their people vying for a place in the new government, 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 were the stances of Labour and NZ First:

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. And I would be very keen to advocate on your behalf if I were elected to represent you. Please feel free to get in touch again if you would like further clarification”.

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”.

In the recent Queen’s Birthday Honours List, Mrs Lock was honoured with the Member of the New Zealand Order of Merit for services to people with rare health disorders.

Our Prime Minister congratulated the New Zealanders recognised for their contributions to the country and their communities in the 2019 Queen’s Birthday Honours list. She said “Every honours recipient has made a valuable contribution to their community and our country. Their commitment to excellence and service represents the best of New Zealand”. I congratulate all 183 honours recipients and thank them for their leadership and service”.

Mrs Lock says “It is ironic that I can be worthy of such a great Honour for advocacy, but not worthy of receiving the life saving medicine for which I was advocating".

Mrs Lock also asked “if Jacinda Ardern would be willing to swap her Honour for life saving treatment for those patients in New Zealand who need treatment. It's not that I'm unappreciative of the Honour, but it is more important to me that people get the life saving medicines they need. Any good leader like our Prime Minister surely shouldn't ignore the fact that people are dying when they could be treated. Why, when you have the authority and the power, would you not do something? She seems to be idolised all over the world for her compassion and humanity, yet if you have a rare disease in New Zealand we're not getting any of that compassion or humanity. Please Prime Minister, stay home and help those of us in New Zealand who have begged for years for life saving medicines. What say you? Please don't fob me off to the Health Minister yet again, I'd love to hear from you personally”.

You can get me at nz.pompe@gmail.com


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