Still Fighting for ERT in NZ & Still Need Your Help!

 

Allyson needs ERT to help her fight Pompe disease, an email from you could help make that happen.

On July 28, 2010, I posted an entry (“10 Minutes”) asking everyone to write letters to New Zealand’s Minister of Health, Hon Tony Ryall on behalf of Allyson Lock. Several of us received standard letters of response from Ryall’s office. Yesterday, my best friend received a “personal response” from Ryall – I have copied the text of the letter below. (Thank you V!)

In the letter, Ryall encourages us submit information directly to PHARMAC, which is the NZ government agency that decides which medicines used in the community are publicly-funded.

“PHARMAC is currently consulting on potential changes to its EC policy. You, your friend Monique or Ms Lock may like to make a submission on the discussion document. It can be found on PHARMAC’s website (www.pharmac.govt.nz). Select ‘Patients & Consumers’, then ‘Have Your Say’ and “EC Review’.”

CLICK HERE TO SUBMIT NOW

Hurry! The deadline to submit is September 3, 2010! I have already sent my letter, now it is your turn.
 

Ryall’s letter…

Dear Ms. Shannen

Thank you for your email of 29 July 2010 about enzyme replacement therapy (ERT) for the treatment of Pompe Disease.

I am advised that Myozyme (alglucosidase alfa) was registered by Medsafe for use in New Zealand in April 2009. However, this medication is not currently funded by PHARMAC, the government agency that decides which medicines used in the community are publicly-funded. Ms Lock’s GP, if they have not already done so, can make an application to PHARMAC to fund the medication for her under the Community Exceptional Circumstances (CEC). PHARMAC advises that CEC funding is only available in situations where the disease/condition is rare, or the response to treatment is unusual, or there is some other unusual combination of clinical circumstances. PHARMAC defines ’rare’ and ’unusual’ as less than ten cases nationally.

Where one of the above criteria is met, CEC applications are then further examined taking into account the following considerations:

  • the suitability of the pharmaceutical for which funding is sought;
  • the clinical benefit;
  • the cost-effectiveness of the treatment; and
  • The patient’s ability to pay for the treatment.

Where the cost of a treatment being applied for exceeds $15,000 for the total course of treatment, PHARMAC’s 9 Decision Criteria are also considered.

PHARMAC received two applications last year to fund Myozyme under CEC. The Exceptional Circumstances Panel sought further information on these applications and will consider any other applications it receives for new patients applying for ERT for Pompe Disease.

PHARMAC is currently consulting on potential changes to its EC policy. You, your friend Monique or Ms Lock may like to make a submission on the discussion document. It can be found on PHARMAC’s website (www.pharmacy.gov.nz). Select ‘Patients & Consumers’, then ‘Have Your Say’ and “EC Review’.

The Government has provided an extra $40 million in 2009/10 and $20million in 2010/11 for funding medicines used in the community, but it is not possible to subsidise every medicine available in New Zealand.

Yours Sincerely

Hon Tony Ryall

Minister of Health

Saves Lives!

Let your voice be heard! Patents in New Zealand don’t have to be dying from Pompe, they could be receiving enzyme replacement therapy instead and be FIGHTING POMPE!

Please take 10 minutes to make a submission to the discussion document. Thank you!

32 comments

  1. This looks like an important opportunity to be heard. Do you think there’s anything in your friend’s letter that might have provoked a personal response? That would be good for us to know. Sometimes it’s just the way something is phrased.

    1. Brad:

      Here is a copy of her original email. My best friend rocks!

      Dear Hon Tony Ryall,

      My closest friend Monique has informed me about her friend and fellow Pompe Disease patient Allyson Lock. She lives in Masterton, New Zealand and has been denied enzyme replacement therapy. She was told there simply wasn’t funding for it. With the help of her GP, who is currently trying to generate the much needed funding, she is asking her politicians to help allow more funding for her disease. I know from my friend Monique that Pompe Disease can be treatable.

      Please help people like Allyson!

      Thanks,
      Vanessa Shannen

      I have enclosed an article about the treatment that is beign done in the US:

      http://news.health.ufl.edu/2010/11843/colleges/college-of-medicine/uf-doctors-among-first-to-administer-therapy-for-rare-disease/

    2. Also – here is what I wrote to the EC Review Board along with what I got back…

      Upon the advisement of Hon Tony Ryall, Minister of Health I am writing to you today regarding the use of Myozyme (alglucosidase alfa) for Pompe patients. The drug is administered through IV infusion every two weeks – enzyme replacement therapy (ERT).

      I am an American Pompe patient who has been receiving this treatment for only 5 months and I have already experienced a great improvement in my condition. In America both Myozyme and Lumizyme are available to children and adults diagnosed with Pompe, and yes it is an expensive drug, but it is a life-saving drug. I understand you consider the cost-effectiveness of the treatment. I will say it again: It is life-saving. How do you put a price on someone’s life?

      I have become a Pompe patient advocate of sorts here in the small but supportive Pompe community. You can even read about my journey with Pompe on my blog: https://purplelv93.wordpress.com . I have spoken with many patients both in America and elsewhere who are so grateful for this life-saving treatment. Without this treatment infants die usually before reaching their first birthday. In older patients the progression is slower, but it does progress with the end result being death usually from respiratory failure.

      Can you imagine having a life threatening condition and know there was a treatment out there that could save your life but the government will not allow you to have it? Again, yes it is a big expense, but there are very few cases of Pompe in New Zealand so you would only have to cover a few patients.

      When patients were diagnosed with Pompe before ERT became available it was a death sentence. It doesn’t have to be that way anymore. We can fight Pompe, but we need this drug in order to do that. Otherwise we are just dying, waiting for our lungs to give out. That hardly seems fair when modern medicine has produced a synthetic enzyme that will allow us to live. If you do not have Pompe your GAA enzyme works just fine and you are blessed enough not to need this treatment. All I am asking for is that you grant that same opportunity to have a “working enzyme” to those handful of New Zealander’s who desperately need it. This treatment really does work, it really does make a difference. I am living, breathing, fighting proof!

      Thank you for your time and consideration.

      Sincerely,
      Monique Griffin

      Dear Monique

      Thank you for your feedback to our review of medicines funding in “exceptional” cases. We will consider your comments as we progress with developing options for an improved Policy. Your comments are particularly interesting given your perspective as an American not in New Zealand.

      We will also add you to our review database to ensure you are informed of the second phase of the review later this year where you will have the opportunity to provide feedback on the options we develop.

      Thanks again

      Bryce Wigodsky

      PHARMAC Communications

      EC Review Policy Team

  2. Awesome Monique, quick reply! I will write right away. I hope a response form yet another country will help.

    Please everyone, don’t be shy, be heard!

    Brad

      1. My email to the EC Review, and their reply…

        I am writing to share my experience in hope that it helps in your review process. I encourage that your Exceptional Circumstances criteria allow for funding of Myozyme for Pompe patients. Myozyme is a life-saving enzyme replacement therapy that is administered bi-weekly by infusion.

        I am a Canadian with adult-onset Pompe disease. I was diagnosed in 2006. Since that time, I have suffered typical loss of muscle strength associated with that disease. I used to be very active but have had to give up almost all of the activities that I had. The most significant change for me is weakened breathing muscles. I was told earlier this year that I would be on mechanical ventilation in two years. You can imagine how difficult that was to hear, but I knew that it was true.

        The prognosis for adults with Pompe disease is progressive muscle weakness, leading to mechanical ventilation and eventual death by respiratory failure. This is a serious, cruel, disease for which there is no cure. I have watched my health worsen and know very well that I will die from this. I live with this every day, as do my family and friends.

        But, something has changed my outlook. Three months ago, our Pharmacare approved my funding for Myozyme. I just had my 7th infusion. I already see measurable signs of improvement. I feel like my life has been given back to me after being snatched away.

        Because it’s so rare, there is limited information available about Myozyme’s effectiveness for adults. That is a reality for treatments for rare diseases and it is understandable. Without a doubt though, this is a life-saving treatment. There are patients all over the world that are benefiting from it. I have spoken with many of them and this is the consistent message that I get.

        Pompe disease is very rare but there are a small number of people in New Zealand that are fighting it. Please help them. I want so much for them to have the same opportunity at life that I’ve been given. Let them be proud that they live in a country that values their lives. Don’t let them suffer when something can be done to help them.

        Thank you very much for your time. I would be happy to discuss this matter further at any time.

        Sincerely,
        Brad Crittenden

        Dear Brad

        Thank you for your feedback to our discussion document, PHARMAC’s Review
        of Exceptional Circumstances: Seeking your views. We will consider your
        submission as we progress with developing options for an improved EC
        Policy.

        We will also add you to our review database to ensure you are informed
        of the second phase of the review later this year where you will have
        the opportunity to provide feedback on the options we develop.

        Thanks again

        Bryce Wigodsky
        PHARMAC Communications
        EC Review Policy Team

      2. This is my second email to the EC Review and their reply. I received this less that 24 hours after sending it. I’m so impressed with the respect that they show by replying so quickly…

        I’m writing briefly to add important information to my previous email. I
        have just had a pulmonary function test after receiving Enzyme
        Replacement Therapy (Myozyme) for three months to treat Pompe disease.
        I’m ecstatic that all of my pulmonary function values are up
        significantly. I hope that this additional information will help in your
        review of Exceptional Circumstances, especially in how it supports this
        life-saving treatment for Pompe patients.

        [a graph showing my decline and then improvement with ERT was attached]

        Sincerely,
        Brad Crittenden

        Dear Brad

        Thank you for the further information you have provided for us to
        consider during our review of Exceptional Circumstances funding. I will
        be sure to include this update in our consideration of the policy as we
        progress.

        Sincerely

        Bryce Wigodsky

  3. Well anything that will help you and others to get the treatment you need — I will do. I still don’t know why my brief email with spelling errors and bad grammar even would draw a response. But I’m glad it is helping with awareness and hopefully Allyson will get the treatment she needs 🙂

  4. I have also written!! Had to chew it over what to write, so I hope this is ok!

    Dear Bryce Wigodsky

    Upon the advisement of Hon Tony Ryall, Minister of Health I am writing to you today regarding the use of Myozyme (alglucosidase alfa) for Pompe patients. The drug is administered through IV infusion every two weeks – enzyme replacement therapy (ERT).

    My sister-in-law Allyson Lock from Masterton had Pompe Disease, With help the myozyme drug she would have the chance to continue to be a Wife and Mother and a much loved family member to her wider family.

    If the progression of this can be halted or slowed down, Allyson and her Husband will have the chance to carry on with a “normal” life, that they have worked very hard to attain.

    If this drug is not funded 2 young boys will watch their Mother die, Stephen her husband will loose his wife and the mother of his children.

    How hard is that knowing that there is a drug out there to save your life and to keep the quality of life you already have, but you cannot have it.

    Please consider the funding of Myozyme to keep our Loved one alive and mobile with a quality of life she with not have without this drug and for the other sufferers of this very rare but nasty disease.

    Regards Julie Wells

    1. Awesome Julie – very heartfelt. Thank you!

      Please encourage everyone you know to send emails too and let me know if you get a response and I’ll post it here.

      Monique

      1. Wow A reply within 12 hours…………
        Dear Julie

        Thank you for your feedback to our discussion document, PHARMAC’s Review
        of Exceptional Circumstances: Seeking your views. We will consider your
        submission as we progress with developing options for an improved EC
        Policy.

        We will also add you to our review database to ensure you are informed
        of the second phase of the review later this year where you will have
        the opportunity to provide feedback on the options we develop.

        Thanks again

        Bryce Wigodsky
        PHARMAC Communications
        EC Review Policy Team

        I found it very hard to put my feelings into words to somebody that does not know Allyson, as she is my Husbands twin sister and we have been through thick and thin over the last 30 years. I did not want to let my emotions run away. As we are starting the process to the world of Pompe. Thank goodness for the internet and for the information you are all passing on to us, The love and support you are giving to Allyson is wonderful, Thank-you so much as I can tell you she is well worth fighting for.

      2. Julie, you bring up an important point. We tend to think about the patient, but this is about many more people; family, friends and people that we don’t even know. We’re used to things being fixable but this isn’t one of those things and that’s frustrating.

        It’s hard to see someone that you love battling something like this when there’s not much you can do to help. You know what? There’s a lot you can do, and you already are. Write letters, and ask the people that care about you to write too. They will and they will be happy that you asked them. Support Allyson and encourage her to join groups like Muscular Dystrophy. There are really good people there and they offer services that she may or may not need. Help her with fundraising and raising awareness. Learn about the disease, and the treatment. Do a happy-dance because there actually is a treatment! It’s all part of taking back some of the control that has been taken away, and that’s important.

        Allyson isn’t the only one that has been thrust into the wonderful world of Pompe, you and your family have been too. You’re right, the internet is great, it makes the world smaller. Ask people like us lots of questions. Just make some of them simple because we love it when we actually know the answer, lol!

        Brad

  5. Thanks you guys. You’re all wonderful! Now i just gotta mop up my tears!

    My husband and i both wrote seperately to PHARMAC, and we got exactly the same reply as everyone else. We have also sent these to our local MP and he has contacted me to sign a privacy waiver so he can look into this. I was happy to do that! At least finally someone might actually REALLY look into it rather than passing the buck and/or giving a standard reply. Will keep you posted!

    Much love
    Allyson

  6. Allyson, when I went through this, there were a lot of letters written too. They all got the same response. Don’t read anything into that. They have to acknowledge the letter and, really, they have pretty much the same thing to say to everyone, right? At this point they have to be noncommittal. They also have to be quite careful in how they word things. But, every letter represents at least 7 votes!

    The only political difference between you and me is that Canada’s healthcare is administered provincially, instead of federally. I signed the same kind of waiver that you did. That’s great that you were asked to. Because of privacy, they can’t do much without it. I’m sure they will do everything they can to help. Your MP has joined the A-Team!

    Brad

    1. Brad is right – as usual. 🙂 We are on their radar and that is what matters. The letter I got back was a tad different, as he noted that I was an American – so I really feel they are getting read.

      During my infusion today, I got to talking with my nurse about your situation and she asked about this blog. I gave her the link and she said she would write a letter too. Couldn’t hurt to have a letter in the mix from a nurse who has a Pompe patient right?

      Keep reaching out for more emails – swamp them with emails. We only have until 3 September. And then, I assume we wait.

      Monique

      1. I think they are reading the letters too. These responses are very quick. Mine took weeks, sometimes many. It’s rare to be able to be involved in this part of the process, very cool. Allyson, I read that there is also an opportunity for you to talk to them in person. You already wrote this time, but maybe in the next stage you might consider that? There’s value in putting a face on this. You’re going to learn pretty quick (I did!) that any privacy that you thought you had went poof!

        Good idea Monique! That’s great that your nurse is writing. You’re right, I think you were the only one that got a slightly different response.

        Just wanted to say one thing. Sept 3 is their date, not ours. Unless they approve this by that time, nothing has changed, right? We might not be able to contact the EC Review until the next stage, but there is still the Health Minister, your MP and anyone else we can think of. We can’t let up. This is important stuff. As Monique says, swamp them with emails!

  7. I had a letter from my local Member of Parliament yesterday saying he has written to our Health Minister Tony Ryall on my behalf.

    You guys all warm my heart. I’m totally lucky to have you all on this journey with me. Thank you, thank you, thank you!

    Allyson

  8. This week, Peter Moodie who is the head of PHARMAC held a meeting for medical people here in town. Being that i’m not a medical person i could not attend but my nurse did. Here is what she got from the meeting. Does not look very promising for those of us here in New Zealand.

    *******************************

    There are 3 schemes
    1. CEA – community exceptional circumstances
    2. CaEC – cancer exceptional circumstances
    3. HEC – Hospital Exceptional circumstances

    Number 1 was the area most talked about – there are 500 plus
    applications per year. The total funding stream is 2.5 million per
    year(comes from a national budget) that must be divided between any of
    the applications that get in.
    There is a two week turn around from the time the application lodged.
    These are for rare and unusual circumstances. Usually less than ten
    people will get the funding.

    Who administers it?
    Administers
    Rachel Pratt and Katie Appleby
    And a panel of 8 – mix of GP, Specialists.

    A review is going on now and submissions being made re the best way
    forward with the Community Exceptional circumstances – funding. They
    have been going around the different health boards collating information
    from doctors on “how would you want exceptional circumstances to work?”
    The information sounds like is in – the first draft will be in November.

    Peter Moodie when into more detail of a few conditions that had been
    declined over the years (Pompe wasn’t mentioned)One of the conditions
    was going to cost the same amount as the treatment for pompe- it was
    declined.
    It was also for life.

    There is an appeals process
    This can go to court – for a judicial review
    No one up to this point has taken it that far when they have been
    declined.

    1. Are any of these sessions a matter of pubic record? Is there a place where the meeting notes can be viewed or is it filmed?

      If it needs to go to appeal – so be it! We are a stubborn bunch and won’t give up! If that ends up being the course, then that is really the time to invite the media to our battle!

      I hope it doesn’t come to that and instead these folks come to their senses and fund ERT.

      Monique

    2. There are 500 applications and only 10 get funding?! What do the other 490 people do?

      I’m wondering what the actual purpose of this meeting was? Is this part of the EC Review? If he was discussing what has been happening then this must be the current system that he’s discussing, not the proposed changes that may come out of the EC Review. The playing field could change a lot. That doesn’t mean that we should sit back and wait for it to happen, but we do need to recognize that and make sure we are able to participate in those changes as much as possible.

      That panel-type of review system is very similar to what happens here. My doctor made the application and it went through the govt to the review committee. That committee makes recommendations to the govt on funding. The govt takes that recommendation into account when making their decision. Your red tape might be slightly different, but pretty much the same I bet.

      Essentially the request gets reviewed by a group of ‘experts’. It is unlikely that we have any access at all to them. The only pressure that we can apply is political.

      We do need to be somewhat realistic, in that it has not been very long since the request was made and you don’t actually have an answer yet. That said though, he says it only takes 2 weeks for a decision and it’s been longer than that. What’s going on?

      Allyson, have you been interviewed by anyone? Do you have any media contacts, or know anyone that does? Rather than you contacting them, maybe a friend could suggest to the paper, etc., that they should talk to you. Another approach would be for you to write a heartfelt letter that could be printed.

      I know some time ago you spoke with a couple of the groups there, like Muscular Dystrophy, right? Maybe touch base with them again and see if they know of any changes. Also ask them if there is any advocacy help available if this ends up being appealed or even goes to court.

      Don’t get hung up on that $2.5M number. Budgets change and there are various sources of funds if they try hard enough. To put it in perspective, let me give a $$ example. Ticket sales at one Vancouver hockey game are $1.2M, and at one Toronto hockey game they’re $1.9M. Don’t tell me that society doesn’t have the funds to support you. There has to be a way.

      Brad

      1. Yes the meeting discussed what happens with the EC now and the review is to see what they are going to do in the future. They may change nothing, i guess we have to wait and find out. All of us who wrote letters to PHARMAC received letters saying we would be contacted later in the review process. Although i think they will be only telling us very minimal information.

        I know there is enough money in society to pay for ERT, NZ spends way more than 2.5M on foreign aid and keeping murderers and rapists in prison than it does on EC! Clearly if 490 people a year are missing out on EC they are not allowing enough funds for this.

        No one has applied for my EC funding yet as apparently it has to be a specialist who applies. Formerly you needed 3 people apparently, but now you only need the one. I have yet to receive an appointment with the only pompe specialist in NZ. I have a letter to say i am on the waiting list, and i will be seen within 6 months depending on what my status is, urgent, non urgent etc.

        I’m just in the process of joining the muscular dystrophy association and i am told they are very good patient advocates.

        I don’t know anyone in the media but i wouldn’t let that stop me from contacting them if the need arises. No one has interviewed me for anything. I’m going to write to my local member of parliament again. I had a letter from him saying he has written to Tony Ryall, but i suspect he will get the same letter that everyone else got.

        Frustrating!!

  9. I don’t know if any sessions are of public record. The only way i knew about this meeting that took place this week was because my GP sent me an email he received inviting medical people to it. My nurse went along and took notes and emailed me what i copied and pasted in my post above.

    2 and a half million seems like a pathetic amount. Clearly if they receive 500 requests a year and only 10 people are granted anything then they need to put some more money into it! I’m going to write to Peter Moodie tomorrow and also to my local member of parliament again and ask how much they spend on keeping murderers, rapists and thieves in jail each year. I know it’s 90,000 dollars a year for each person, but i wonder how much the total spend is. Bet it’s WAY more than 2.5 million!

    I agree Monique, inviting the media to participate will be high on the agenda should this go to an appeal. I for one would appeal if they turn me down. And i am assuming that i will get turned down for funding.

  10. Allyson, just because they haven’t approved this before in New Zealand doesn’t mean they won’t. I was the first to be approved here. There has to be a first and there’s no reason why that can’t be you. Budget pressures always exist. At some point they have to look at funding like that and see that’s it’s very inadequate. Health Ministers have the power to change things a lot, given enough motivation.

  11. I’m a bit tardy in posting this as I received it a couple weeks ago. It’s just an update but it does say that we won’t get into the next EC Review phase until early next year. I guess they’ve had more input than they had planned – good!

    Brad

    Progress update on PHARMAC’s review of Exceptional Circumstances

    PHARMAC’s review of its Exceptional Circumstances (EC) Policy is progressing well. We received more than 70 submissions in response to our discussion document Review of Exceptional Circumstances: Seeking Your Views. We appreciate the feedback received from stakeholders and thank you for taking the time to provide it to us.

    We are currently developing a proposal for a revised approach to EC, giving serious consideration to feedback to date, for your further comments. Given the number of responses we received, the wide range of views expressed and our wish to provide people with ample opportunity to share their views on our proposals, we are delaying the release of the next consultation document until early in the new year. We will accept feedback on our proposals until the end of March 2011. We hope this provides people with sufficient time to review and comment on the proposal and attend face-to-face meetings to discuss it, if they wish.

    Following further feedback, analysis of those responses and a Board decision on any changes to EC, we expect this review to be completed in the middle of 2011. The timeframe for implementation will depend on the nature of the final EC policy.

    If you have any questions about our review of EC, please contact Bryce Wigodsky at
    ecreview@pharmac.govt.nz or 0800 660 050. I thank you again for your assistance to date and look forward to your further comments.

    Yours sincerely
    Matthew Brougham Chief Executive

    1. Thanks for the update Brad! I received the same letter, but it was in my Spam folder. So, if you are one of my amazing readers who wrote to PHARMAC and haven’t received anything be sure to check your Spam folder.

      Let’s hope they do the right thing and approve ERT!

      Monique

  12. Hi everyone

    My GP and specialist put my application in to PHARMAC for ERT and they wrote back and asked for updated data about the effectiveness of Myozyme. Maybe all is not lost yet!

    Will keep you all posted and thank you all for what you have done 🙂

    Allyson xo

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out / Change )

Twitter picture

You are commenting using your Twitter account. Log Out / Change )

Facebook photo

You are commenting using your Facebook account. Log Out / Change )

Google+ photo

You are commenting using your Google+ account. Log Out / Change )

Connecting to %s