I had this great letter from Ayumi Sugimoto in Auckland.  Please read it, and if you are able to get along to see this and offer support that would be wonderful :)


My name is Ayumi SUGIMOTO – Originaly from Japan and have been living in Auckland since 2002. I am organaising Japanese Dance group called “Southern Stars” (www.yosakoinz.org.nz) in Auckland.

I would like to introduce one of my friends called Ayame YOSHIDA who aged 12, living in Japan and has a Pompe disease (Also he has a autism). When Ayame was 2 years old his doctor said he has a muscle disease then when he was 5 years old the doctor found that he has the Pompe disease. Then in 2007 when he was 7 years old he started get the Myozyme treatment.

Because of Ayame was a little boy he didn’t understand what happned in his body also why he need to have the Myozyme treatment so that he was crying while his treatment and didn't want to do it. Then his mum (Kasumi) wrote a story book (~A Warm and Fuzzy Heart~) for him to understand why he needs the treatment. Amazingly since he had read the book he understand how important the treatment, no more crying and go to a treatment room by himself.

While Kasumi (Mum)was waiting his treatment she read the book to children and parents who were in the hospital. All the people were moved the story and asked her to make the book for them. Because of the book was handmade even she wanted to give it to many people it wasn't so easy. But last year after lots of support, the book was able to published (You can read it from our web-site www.yosakoinz.org.nz . We helped translation so it has English too). Kasumi(Mum) would like to return something to people, supporters, and organizations so that she has donated the profit of the sales to the Make a Wish and Pompe patients association etc..

Why I am writing this email to you because New Zealand is Ayame’s special place.
Why New Zealand is Ayame's special place? Yes! he has visited New Zealand three times and is going to visit on 23rd November 2011 again.

In 2007 when he started the Myozyme treatment his parents weren’t sure what will happened to him so that they wanted travel together somewhere in spacial. Then they choose to came to New Zealand. At that time Ayame was smiling which Kasumi had never seen such a brightly face all the time and talk to people which Ayame usualy didn't in Japan. Ayame enjoyed staying in New Zealand and wanted back to New Zealand but it wasn't so easy. Then parents asked to the Make a Wish to support his dream to come to New Zealand again in 2008.

I mentioned earlier in this email that I am organising a Japanese dance group in Auckland. Ayame has joined us since 2008 when our group went to Japan to participate at a Festival. Then in 2009 he came to New Zealand to participate with us at the Farmers Santa Parade (We've been participated every year).

Actually the doctor was surprised that he was able to perform our dance which has tricky and quick movement also he wasn't able to do it before but Ayame did it.

This year the Farmers Santa Parade will held on 27th Nov 2011 in Auckland so they are coming to perform with us.
I know you are not living in Auckland but if you are able to visit Auckland please come to see/meet them.

I read through your web-site and understand that you are not able to receive any funding from country yet. But I hope you will receive funding soon.

Thank you for your time to read this email.

I look foward to hering from you soon.

Warm regards,

Ayumi SUGIMOTO
[email protected]
www.yosakoinz.org.nz (Southern Stars: Japanese dance group in Auckland)

Yesterday (7th June 2011) we met in Wellington to launch a campaign to get medicines for rare diseases funded in NZ.

"We" includes our 4 New Zealand Pompe patients Laurie, Freda, Dean and myself, John Forman from NZORD, Jenny Noble from LDNZ, Chris Higgins and Marty Davis from MDA and some parents of children/adults who have been, and still are being treated with ERT therapies.

Peter Moodie of PHARMAC is still adamant there is not enough long term data to prove Myozyme works, even though they said it does work in my "decline letter"!  Stalling tactics of course, they just don't want to fund us.  But they will, we'll make sure of it.  I will post links on the "current news" page so you can see what we were up to yesterday.

Knock Knock Peter - let us in!  Just how wide open is that door??

Well, it was an interesting day. Pharmac gave us 2 hours of their time. There were many things discussed. At the end of the meeting I had a chance to talk to Peter Moodie (head of Pharmac) to discuss my own application for ERT which was of course declined. He asked me what I thought about it. I said I thought it was lazy, ignorant, poorly researched, full of errors and that they hadn't even followed their own criteria in reaching their decision. I told him that they should have me working for them because at least I would do a good job. I said when they are deciding on whether someone lives or dies, they should at least give us the courtesy of getting their facts correct. Several times they tried putting words in our mouths, tried turning it around on us saying that if we get this medication so many others would be disadvantaged...Anyway, at the end of it all, we all came out of there feeling like we had served ourselves well. We are not neccessarily confident that we will get ERT funded, but we did not let them beat us into a pulp and make us feel like a bunch of fools. I think we stood up to them and i'm very proud of our small group of pompe peeps, and also we had wonderful support from MDA, LDNZ and NZORD. Thank you everyone for your kind thoughts :) xo

Well, today I found out that our dumbass government thinks i'm not worth a crap.  Here is the email I have sent to our local Minister of Parliament this evening.  I will post his reply when i get one.  I would appreciate anyone writing their thoughts for me to pass onto our idiots in charge.

Dear Mr Hayes   I have had official word today that Pharmac have refused to fund treatment for the rare disease that I unfortunately have.  Pharmac say it is too expensive.  I know that Pharmac only receive 3 million dollars for Exceptional Circumstances medications per year.  They have estimated the cost of my medication at $575k per year, every year for the rest of my life.    It seems clear that the government does not supply Pharmac with enough money to cover all of the exceptional circumstances applications which are applied for each year.  Please bear in mind that there are four of us here in New Zealand with this disease.  Some of us will cost more than me, some of us less.  The medication dose depends on the weight of the individual.  One of the members of the New Zealand Pompe Network is 6'9" tall and he is 110 kilos in weight.  A man that tall is never going to weigh a small amount!  What do we need to do to get your government to HELP US?  I cannot believe that you cannot save our lives!  What about the losers that kill their friends in drunken road smashes?  Ambulances turn up to save them without a moments hesitation!  But we, who have some horrible deadly disease which is no fault of our own, are left languishing alone and forgotten down the bottom of the heap where no one gives a toss and our government says we're not worth a damn.  I'm disgusted!!!  I'm begging you, do something to save the 4 of us here in New Zealand!!!   I've got the opportunity to go overseas to try to save my life since our government is so totally useless.  Is this government willing to help subsidise this venture?  Are you willing to help us get to the United States to help us save our own lives?  Are you?   I very much look forward to your reply.  We have much to discuss.  This has been many many months in the works, and finally, the answer I knew I would be giving you all along.   Sincerely Allyson Lock

In a period of TOTAL annoyance (even moreso than usual) at the absolute disregard for Pompe patients in New Zealand, I decided to write to PHARMAC to see where my application for Myozyme was standing.  Please read this letter below, and also note the people to whom it was CCd.  Also included are replies from our local Minister of Parliament, plus an email just received from Pharmac themselves.

My email:
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7 February 2011

　
Dear PHARMAC Exceptional Circumstances Panel

• Dr Howard Wilson – Chair, GP, PTAC (Deputy Chair), Pharmacologist

• Dr Paul Tomlinson – Paediatrician, Southland DHB

• Dr David Waite –Physician, Capital and Coast DHB

• Dr Sharon Kletchko – GM, Planning and Funding, Nelson Marlborough DHB

• Dr Andrew Herbert - Consultant Gastroenterologist, MidCentral DHB

• Dr George Laking - Oncologist, PTAC, Auckland DHB

　
APPLICATION FOR EXCEPTIONAL CIRCUMSTANCES FUNDING

I have an application in with you at PHARMAC for the drug Myozyme. The application has been with you for several months. I am writing to enquire as to what stage my application is at now. I quote from the PHARMAC website;

"How often does the Exceptional Circumstances Panel meet?

The Panel meets every two weeks by teleconference to consider all Community Exceptional Circumstances applications. Urgent CEC applications may be considered via email or teleconference as required".

If the panel meets every two weeks, why is my application taking such a long time? I’m dying, surely this must be URGENT?

As my health deteriorates more and more on a daily basis, I do not have the luxury of time on my side. I know that along with my application was the mention of overseas drug trials being available if you were not going to fund Myozyme for those of us in NZ who need it. At the time you said that you had not yet decided to decline applications for Myozyme. I need to know if you are going to decline my application, or whether you are going to fund Myozyme for me. The fact that you are continuing to make me wait when I have a degenerative disease is a callous disregard for my life.

I may have the option now of taking part in an overseas drug trial. This is a new drug for Pompe Disease. If you are going to decline my application for Myozyme here in New Zealand I need to know. Then perhaps you would like to fund my trip to the United States in my desperate attempt to prolong not only my life, but my quality of life. Let’s face it, without treatment it is only a matter of a few short years, if that, until I am permanently wheelchair bound and reliant on mechanical ventilation to keep me alive. This is something that I would dearly love to avoid as I am only 46 years old! I imagine if you were in my situation, you would be eager to avoid that as well!

This situation IS avoidable if only you would either fund Myozyme, or, help us get overseas for the upcoming trial. If you say that it is "not cost effective", we all know that you really mean that my life isn’t worth it. You cannot say that the drug doesn’t work, it has been proven to work.

The following are figures obtained by The Press under the Official Information Act.

North Island-based child, aged between five and nine, costing $966,000 to treat, was the most costly patient in New Zealand last year.

The nine most costly patients required "tracheostomy or ventilation", meaning they were being supported to breathe and most likely in an intensive care unit.

A South Island man, aged between 45 and 49, was the second most costly patient, at $650,000. A child, aged up to four, cost $540,000, while three people, aged over 65, cost $1.5m in total.

Intensive care unit (ICU) beds cost about $3000 a day".

The Press


Will you pay $3000 per day for my intensive care bed in the hospital for the rest of my life? Let’s face it; I could go for years taking up a bed, just a useless blob taking up space. I assume you will pay someone to take full time care of my children while I am in this state? My husband will still have to work to pay the mortgage. He cannot get my life insurance until I am dead you see.

All I want is to live my life as a functioning member of society. Are you going to help me, or do I have to go to a different country for their help? What has become of this country when you cannot see fit to help your own citizens? This is NOT a disease brought on by myself. This is a hereditary disease! I did not wrap myself around a pole in a drunken night time speeding frenzy and then expect you to pay for fixing me up. I do not smoke and then ask for you to fund my quitting. This is NOT MY FAULT!

I look forward to your urgent response.

Sincerely

Allyson Lock

Cc

Prime Minister John Key
Marty Davis MDA
Miriam Rodrigues MDA
Tony Ryall Minister of Health
Tariana Turia Associate Minister of Health
John Hayes MP for Wairarapa
PHARMAC Board
Jenny Noble LDNZ
John Forman, NZORD Executive Director
New Zealand Pompe Network
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Reply from John Hayes MP

Dear Allyson

Thanks for your email. I don't have access to your file tonight but I'll review it tomorrow. That said I can see there has been quite a bit of correspondence.

I have done some trawling with the help of Mr Google.

A couple of questions. Do you have the late onset version of the disorder??

If you do then why are you chasing Myozyme which appears to be the drug used to treat the child and infant version of the disorder?

The internet suggests a drug called Lumizyme for the later onset disease?

No doubt you will have all this sorted but I'm just trying to get my head around these issues before I try to rattle the tree to get you an answer one way or the other.

Best

John Hayes
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Dear Mr Hayes

Thank you for your reply. I have the adult onset version of the disease. If I had the infant form, and lived in NZ, I would be dead a long time ago. As it currently stands, I will more than likely be dead within the next 10 years without help.

The reason I am "chasing" Myozyme, is because the drug is called Myozyme here in New Zealand. It is only called Lumizyme in the United States.

If you would like to visit this website, you will see the reason for this.

http://quest.mda.org/news/lumizyme-horizon

This is a quote from this website;

"Coming to terms: Myozyme and Lumizyme

In 2006, the FDA gave approval to Genzyme to market Myozyme. The drug was the first-ever treatment approved for Pompe disease in the United States, and has since been approved for use in Europe and more than 40 countries worldwide.

After FDA approval, growing demand for Myozyme led Genzyme to change its production processes. The company moved the bulk of its production from Allston, Mass., where the enzyme was made in 160-liter batches, to Geel, Belgium, where the drug could be produced in 4,000-liter tanks instead.

With the change in production came a new name for the familiar Myozyme: Lumizyme. The drug will continue to be called Myozyme outside the United States.

The name change was based on an FDA determination that the enzyme produced on the larger scale possessed slightly different biochemical characteristics than the original Myozyme, and should therefore be classified as a different drug.

I ask for your urgent help in this matter. This has dragged on for a very long time now as you are aware. I would also like to visit you in person to discuss this.

Sincerely

Allyson Lock

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Dear Allyson

Im in Wellington till Thursday evening late then to Waipawa Friday and booked all weekend. Let me try to lean on Pharmac for some answers then we can meet when I am next in Masterton.

Ill be in touch.

J

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Thank you Mr Hayes

I look forward to hearing from you.

Sincerely

Allyson Lock

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Morning Allyson

I've now had a chance to review your file. I assume your GP has submitted an application to Pharmac as suggested by Tony Ryall. Can you please advise the name of the GP and the date of the application. It would be helpful if you could leave a copy of the letter at my Masterton office so that we can keep abreast of developments. Look forward to hearing from you.

Kind regards

John Hayes

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Dear Mr Hayes  

Yes my GP along with the Pompe specialist sent in an application for Myozyme in November 2010.   Please find attached the letter to PHARMAC for my application for Myozyme.  Also a letter from PHARMAC to the specialist who did my application.  PHARMAC prefer a specialist to do the applications rather than a GP.  The name of the specialist is Callum Wilson, the name of my GP is Dr Tony Becker from Masterton Medical.   I look forward to hearing from you on this very urgent matter.    

Sincerely

Allyson Lock

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Seems to me Allyson that Pharmac were pretty speedy. They responded to Dr Wilson on 1 December, ie within 5 days of receiving Dr Wilson's request. Do you know if Dr Wilson supplied the summary statement of how he expected the drug to benefit you?  Do you have a copy of any statement?  I've tired to speak to Greg Wilson of Pharmac but he was not in so I'll try again later in the afternoon. 

J
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John, Pharmac are NOT being speedy at all.  They have NOT made a decision on my application.  This is what my email yesterday is all about.  I'm going to die, there is a medication which can help me, 44 other countries around the world fund it!  An application was made to Pharmac on the 25th of November, it is now the 8th of February, and i have yet to have a decision.  Yes Dr Wilson supplied the statement of how he expected the drug to help me and i included this with my previous email to you.  Did you not receive the attachments?   Excuse me if i seem a little perplexed by your response, I get the feeling that you are not quite understanding my plight.  You know i'm going to die a painful horrible death at a young age?  You know i have young children still at home?  Age 7 and 9?  You know that within a short space of time i will be in a wheelchair?  You know i will be on mechanical ventilation to keep me alive?   Have you seen those?  The hose shoved through a hole cut into my throat?    Should I HAVE to fight this government to keep myself out of a wheelchair and off mechanical ventilation?  I'm really not sure what else i can tell you that will enable you to help me?  But i would like you to consider my future.  If i was your daughter would you want this for her?  Would you want her to have to fight Pharmac to save her own life?  Would you expect her to have to repeat herself over and over and over again for months and months and months while those much more fortunate than herself decided her fate?  And not only that, TOOK THEIR TIME deciding her fate?   Are you able to help me with this?  If not, please say so.  

Sincerely

Allyson Lock 
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Of course I am trying to help Allyson.  I have not received a copy of a letter Dr Wilson presumably sent in December in response to Greg william's letter of 1 December. Anyhow I am not out to defend Pharmac simply trying to get my head around all issues so I can best argue your case. I have talked to Pharmac this afternoon. The first thing I discovered is that from Pharmac's viewpoint, it is your specialist and not you that is the applicant. As I understand an email or letter went to him on 23 Dec saying it would take 3 months for Pharmac to make up its mind. Because the cost of the drug is greater than the level delegated for decision taking by Pharmac staff,  the decision on whether or not to provide you with  Myozyme  has to be taken by the Pharmac Board. The research by Pharmac has gone faster than expected and a paper with a recommendation is being put before the board at a meeting on 25 February. That is the earliest the meeting can happen. The Pharmac Board decision will be sent to your specialist soon after that date. Because new information about the drug has become known Pharmac have started a separate process to consider whether the drug should be made available to all sufferers of the condition.. This process is going to take about a year and is unrelated to your application which relates to giving the drug to you alone. Can you please let me know what happens at  the 25 Feb meeting because you are likely to hear before me. 

Best of luck. Will be thinking of you.

John Hayes  MP
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 John, i have not received a copy of the letter that Dr Wilson sent either.  I forwarded you the statement in which he said how Myozyme would benefit me.   I appreciate you trying to help.  But i'm sure you must be able to understand my complete and utter frustration.  This has been a long drawn out process, during which my health declines on a daily basis.    All 4 sufferers, yes there are only 4 of us, should not have to wait for a year while Pharmac goes through the process of deciding our fate.  The literature about the benefits of Myozyme is very clear.  I can understand it, and i'm not a medical person.  That it will take a full year for Pharmac to decide on this seem absolutely ludicrous.  Particularly due to the fact that this is a degenerative disease.  New Zealanders deserve a better deal.   As soon as i hear the result regarding my application i will let you know.   I thank you for your interest in this matter.  

Sincerely Allyson Lock

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And from PHARMAC

8th February 2011

Community Exceptional Circumstances
Panel Co-ordinator
PHARMAC
PO Box 10-254
Wellington 6143
Phone (04) 916 7553
Fax (09) 523 6870 (redirects to Wgtn)
Email [email protected]

Dear Allyson

In response to your email, which we received this morning, I would like to let you know the current staus of the application made by Dr Callum Wilson for Exceptional Circumstances funding of alglucosidase alfa (Myozyme) for you.

We received the application from Dr Wilson on Thursday the 9th of December 2010. The next meeting of the Exceptional Circumstances (EC) Panel was on the following Tuesday, December 14th. After that meeting, at which the EC Panel noted that the cost of treatment was greater than the level at which it could approve funding, the application was sent for further analysis and the Panel noted that a decision would be required by the PHARMAC Board.

We wrote to Dr Wilson on December 23rd to inform of the expected timeline noting, that as the holiday season was starting, that we expected the analysis and Board decision to take up to 2-3 months.

PHARMAC staff yesterday completed the further analysis which was then sent to the EC Panel in preparation for the Panel meeting tonight. The Panel will be asked to make a
recommendation on the provision of funding.

The PHARMAC Board will then make a decision on the application at its next meeting on Friday February 25th. As is the standard practice for all EC decisions, the applying clinician will be notified of the decision in the days following the Board meeting.

Our usual process is to inform the applying clinician of the status and expected timelines of any EC applications. You should therefore contact Dr Wilson to discuss the outcome of his application in early March.

Yours sincerely

Jayne Watkins

Team Leader, Medical

Cc: Dr Callum Wilson

We really appreciate the support that we are getting from all around the world in our fight to get treatment for Pompe here in New Zealand.  Thank you everyone, it's great to know we are not alone!