Our campaign - 2017

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.

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