​National stay silent on rare disease fundingTuesday, 19 September 2017, 9:33 am
Press Release: New Zealand Pompe NetworkNational stay silent on rare disease funding
With less than a week before the elections, I still feel that one of the most important issues in this election is not being fully addressed; health!
We are told by the National Party that we have a world class health system, and yet when we speak to the doctors and nurses who are overworked, to the people on waiting lists, and to the very sick who are waiting for access to proven treatments to be approved and funded, we hear a completely different story.
I am one of those sick people. I suffer from a rare muscular condition called Pompe disease. A debilitating illness that without treatment will cause me to waste away prematurely. There is a medicine which halts the progression of this disease. This medicine is called Myozyme, and is funded in 80 countries. It is a proven enzyme replacement therapy that is not funded in New Zealand. The Government repeatedly says the cost outweighs the benefits and that there is not enough proof this medicine works.
Myozyme is a proven enzyme replacement therapy, with numerous clinical trials attesting to its effectiveness. It has shown to represent a 59% reduction in mortality. It slows down the devastating decline of muscles in the body and gives people more time with their families. Time to lead a life not restricted by mobility issues or mechanical breathing assistance.
I, and other members of the New Zealand Pompe Network, have written to the Prime Minister, and to the Health Minister asking them to look into the funding of Myozyme. I even presented a petition to Parliament, and went before a Health Select Committee in June this year, where I presented my case as to why this drug should be funded and why Pharmac needs better resources to fund rare disease treatments. In response, the Prime Minister referred us to the Health Minister and the Health Minister says that he cannot interfere in matters of Pharmac. The Health Select Committee have said they hope to have an answer to my petition by the end of the year. But this isn’t good enough. This is a fatal disease and people are dying while the government wastes time, precious time!
Labour, New Zealand First and other parties have responded positively to our communications. We have a commitment that they will help us. We have heard nothing from National, either positive or negative, just stone cold silence.
Why do we still hear nothing from National on funding of Myozyme or even on the funding of rare diseases in general? We are a minority population group in New Zealand, but we are New Zealanders and while we are small in number we deserve to be heard. People will DIE without intervention from our government!
Samantha Lenik
Vice President 
New Zealand Pompe Network

Thursday, 7 September 2017, 11:34 am
Press Release: New Zealand Pompe NetworkHealth Announcements Slammed by Pompe Disease Patients
The announcements made by Prime Minister Bill English in Wairarapa this week have been slammed by patients suffering with Pompe Disease.
Pompe Disease is an extremely rare inherited and fatal disease for which there is no cure.
Only eleven people in New Zealand are currently known to be sufferers.
The only treatment available is an enzyme replacement therapy (ERT) that the Government refuses to fund.
Mrs Allyson Lock who was diagnosed with the disease in 2010 has been refused treatment despite several applications and an appeal to Pharmac, the Government body that funds medicines.
Mrs Lock criticised the PM's claims about access to new drugs.
“Mr English told the audience that we now have access to new drugs which for quite a while New Zealanders couldn't access”, Mrs Lock said.
“Myozyme is the only treatment available for Pompe disease patients like me, yet Pharmac refuses to fund it. It is funded for Infantile Onset Pompe Disease patients here, but we don’t have any! The 11 patients still surviving have Adult Onset Pompe Disease, and we have all been refused funding”.
Mrs Lock said that Pharmac's standard response has been that the benefit to the patient is not worth the cost to the Government, and that there is not enough proof the medicine works. This is despite nearly 80 countries around the world making Myozyme available to their patients with Pompe. Mr English claims that he is committed to world class health services. Countries like Algeria pay for the treatment yet New Zealand won't “.
Mrs Lock has been given charitable access to the treatment by the manufacturers, Sanofi Genzyme, but she wants to see the Government do the right thing by other New Zealanders also suffering with Pompe Disease. “New Zealand is a first world country and the government tells us that we have a big surplus of money. But people are still dying of Pompe disease. How is this allowed to happen”? she said.

Signs & Symptoms of Pompe Disease
The signs and symptoms of Pompe disease vary widely and may first emerge at any age from infancy to late adulthood. These sample patient experiences illustrate how the disease can affect different people differently:
• A 3-month-old baby is admitted to the hospital with breathing problems brought on by a bad cold. An x-ray reveals a greatly enlarged heart.
• A 2-year-old child who has just started walking has an odd walk that is a cross between a waddle and a limp. As an infant, he had trouble turning over and holding up his head.
• A 20-year-old woman becomes winded and her muscles ache when she climbs a flight of stairs.
• A 37-year-old man starts falling asleep during the day and has trouble breathing when he is lying down.
In all cases, Pompe disease is caused by the same underlying problem: an enzyme deficiency that causes build up of excess glycogen within muscle cells. However, the resulting muscle weakness can affect many different parts of the body and cause a variety of health problems. In some cases, the initial impact may be very serious and progress (worsen) rapidly, while in others it will be less extreme and progress more gradually.

Most Common Effects of Pompe Disease
Although the effects of Pompe disease vary from patient to patient, some broad generalizations can be made. Most patients experience muscle weakness in the arms and legs, usually most prominently in the legs, making walking or climbing stairs difficult. Muscles used for breathing are also often affected, making it difficult to breathe, especially when lying down. In infants, the heart is usually affected, resulting in greatly enlarged heart and other heart problems.
• Infants typically have extreme muscle weakness and a “floppy” appearance. X-rays usually reveal a greatly enlarged heart. Other symptoms include breathing difficulties, trouble feeding, and a failure to meet developmental milestones such as rolling over and sitting up.
• Children and adults tend to have greater variety in their symptoms, often including weakness of the leg and hip muscles, leading to difficulties with mobility, as well as breathing difficulties. Older patients rarely have the heart problems typical in infants.

Describing the Disease
Today, Pompe disease is usually described as a single disease that manifests as a spectrum of symptoms and rates of progression across patients of different ages. However, sometimes terms are used such as “infantile-onset” and “late-onset,” which were historically used to describe the disease in terms of “sub-types” based on patient age at onset of symptoms.

Ongoing Disease Progression
Pompe disease is always progressive, meaning that its symptoms worsen over time. In general, the earlier in life the symptoms appear, the faster the rate of progression. Most infants affected by the disease experience very rapid progression, and they rarely survive past the age of 1 year.
When symptoms first appear later in life (children or adults), the rate of progression is generally slower than in infants, although there is great variability across different people. In addition, an abrupt and rapid decline can happen at any time, so careful monitoring of the disease’s progression is very important. Whether the disease progresses quickly or slowly, movement and breathing difficulties worsen over time.
If you require more information on Pompe Disease, please visit our website; http://nzpompe.network

ENDS

I am writing to you again regarding the Pompe disease patients in New Zealand who are being denied treatment by our government. 
 
There are 11 adult patients in NZ, there were 12, but one passed away from Pompe disease late last year at the age of 54.  There is a medicine which halts the progression of this fatal disease.  Myozyme has been FDA approved since 2006 and is available in nearly 80 countries around the world.  NZ is one of just three OECD countries which deny their citizens access to this life saving treatment.  The other countries are Austria and Iceland. 
 
In a recent round of extra funding for a “Rare Disease Pilot” Infantile Onset Pompe Disease (IOPD) was given funding for Myozyme.  Yes, this was great, well it would be if we actually had any infantile patients.  We have zero.  But as mentioned, we do have 11 Late Onset Pompe Disease (LOPD) patients who were left abandoned yet again.
 
We have a family with three sisters who have Adult Onset Pompe Disease.  How disgusting is it that they are being abandoned by the government? 
 
The government continues to say that they cannot interfere with PHARMAC decisions.  PHARMAC is a government entity.  The government is responsible for whether or not PHARMAC is able to fund medicines.  Yet time and time again the government washes their hands of responsibility by saying it is up to PHARMAC to make the decisions.
 
What we need to know from you is;
 

1. If you are part of the government from September 23 2017, will you commit yourself to ensuring that Myozyme is funded for all ages of patients in New Zealand with Pompe disease? 

 

1. Will you do this urgently before any more of our patients die?

 
 
Yours Sincerely
 
Allyson Lock
President
New Zealand Pompe Network
 

 
Dear Ms Lock
 
On behalf of Hon Dr Jonathan Coleman, Minister of Health, thank you for your email of 6 August 2017 with a letter from the New Zealand Pompe Network.
 
The Minister has noted your comments and advises that there is no further update on this matter his last correspondence of 30 May 2017.
 
Thank you for writing.
 
Kind regards,
 
Melissa Buckle
Private Secretary (Admin) I Office of the Hon Dr Jonathan Coleman
Minister of Health, Minister for Sport and Recreation

Dear Ms Buckle
 
Thank you for the reply.  But it clearly didn’t answer the questions I asked. 
As Mr Coleman is an MP, he works for the taxpayers of New Zealand and is answerable to us.
Please advise Mr Coleman that we are seeking answers to these questions as we are wanting to use his replies in our campaign leading up to the general election.
 
What we need to know from you is;
 

1. If you are part of the government from September 23 2017, will you commit yourself to ensuring that Myozyme is funded for all ages of patients in New Zealand with Pompe disease? 

 Yes or No?

1. Will you do this urgently before any more of our patients die?

Yes or No?
 
Obviously, if he refuses to answer, it is pretty telling about his contempt for Pompe disease patients.
 
 
Yours sincerely
 
Allyson Lock (Mrs)
 
 
 

The New Zealand government and its pharmaceutical buying agency, PHARMAC, are happy to sacrifice a few for the greater good.  While this government forks out money for sporting events, flags and a host of other uneccessary goodies, Pompe patients are left to die.  Is this a good thing in New Zealand?  We don't think so! 

We embarking on a new campaign leading up to the General Election on the 23rd September 2017.  To start off with, we have emailed all of the MPs and Candidates for the upcoming General Election.  We have a lot more planned!

This is our first email -


 NEW ZEALAND POMPE NETWORK
                                        Charities Registration Number CC54486
 



181c Willow Park Drive
RD11
Masterton
New Zealand 5871
 
Email: NZ.Pompe@gmail.com
 
8 July 2017
 
Dear Sir/Madam

I am writing to you on behalf of the New Zealand Pompe Network.  As you are a candidate in the upcoming General Election, I would like to know what your position is on the funding of medicines for rare diseases.  Specifically, I want to know your position on the funding of Myozyme for Pompe Disease patients.

Pompe Disease is usually fatal and affects about 1 in 40,000 people.  There is a treatment available that significantly increases the quality and length of life of sufferers who are treated[i].  This treatment, called Myozyme, is available to patients in almost 80 countries world wide.   Myozyme is not available in New Zealand to adults because PHARMAC has consistently refused to fund it.  11 New Zealand sufferers are dying while PHARMAC waits for a bigger price reduction, while using the excuse there isn’t enough science to prove its benefit.  This is despite research and proof to the contrary[ii].  We have already lost one patient to Pompe disease in recent months.  Is this acceptable?  Why aren’t Pompe patients worth it?  There are other medicines which PHARMAC have funded “despite the uncertainty over the clinical benefits for these patients”[iii].
What we would like to know is:

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?

If you would like to know more, please refer to – New Zealand Pompe Network
I look forward to your reply stating the position you will take regarding these questions I have raised.
 
Yours sincerely
Allyson Lock
President
New Zealand Pompe Network
 

[i] Güngör et al 2013 showed in a pivotal survival study, with a median six years of follow-up: 35.4% of Late Onset Pompe Disease patients in the untreated cohort died; as compared to only 8.8% of patients who received Myozyme.
 
[ii] https://www.ncbi.nlm.nih.gov/pubmed/23531252
 
[iii] Galsulfase (Naglazyme)
https://www.pharmac.govt.nz/medicines/how-medicines-are-funded/medicines-for-rare-disorders/

Listed from 1 May 2016, galsulfase (Naglazyme) is a treatment for people with the rare enzyme deficiency condition Maroteaux-Lamy Syndrome, or mucopolysaccharidosis (MPS) VI. It’s for a very small but high need patient group – we think about 4-5 people in New Zealand.

Because of the very low numbers of people with MPS VI, there’s uncertainty of benefits in the clinical evidence. But there’s a high unmet need for some people with the disease, and the potential for people diagnosed early to be severely affected.

So we’re pleased to have reached a commercial agreement with BioMarin that makes funding possible and manages cost and risk. The agreement makes it possible to offer funding to all existing patients, regardless of severity, and despite the uncertainty over the clinical benefits for these patients.