“One should judge a society by how it looks after the sick and vulnerable” – part tahi
NZ, Wellington, 27/28 February – This blogger was invited to attend a forum held by NZORD (New Zealand Organisation for Rare Disorders). The Forum addressed issues and problems surrounding PHARMAC funding (or lack, thereof) for “orphan drugs” (see: Wikipedia – Orphan Drugs), for rare medical conditions such such as Pompe Disease.
Until now, many of the folk involved (John Forman, Allyson Locke, and others) were just names and media stories to me.
But on the evening of 27 February, when I met Allyson at Wellington Airport, and subsequently met John at Rydges Hotel in downtown Wellington – they became very real, engaging people. I was finally able to put people-to-names.
And then I heard and learnt their own stories.
What I found humbled me and made me realise that their stories and their problems with PHARMAC could be any New Zealander impacted by government policies.
These were our stories as well as theirs.
About 20 people met in the Rydge’s Function Room. It was a bright sunny day outside – and the hotel’s air-conditioning was on the blink. The heat was stifling; comments were made about hot-house conditions suitable for growing tomatoes; fans were brought in; and Management expressed their apology by shouting us five free bottles of wine later, during our buffet dinner. (Since I was driving, it was water for me.)
The Forum kicked off with John Forman, Executive Director of NZORD, welcoming attendees,
John introduced himself and explained his own situation, referring to two family member with a rare disorder. This has given him first-hand, intimate empathy with people living with rare diseases and disorders. He works in the dual roles of Executive Director of New Zealand Organisation for Rare Disorders and Chairperson for the Lysosomal Diseases support group. He has worked in this field, at first part-time and now full-time, for fifteen years.
John then invited those present to give a brief introduction to their own situation.
Daniel explained his rare disease – PNH – and that he is currently fighting to gain access for medication that is known to be a life-saving treatment for people with his condition. His group, The PNH Support Association of NZ, launched a media campaign this year to highlight their fight to access treatment for their condition.
He said that the Pharmac process had been ongoing for twelve to fourteen months. His group are pushing for release of documents from Pharmac relating to their application for drug-funding.
Daniel is a founding member of the The PNH Support Association of NZ which is currently running an online Petition to Government to Make Soliris available to New Zealanders. [Blogger’s note: please take a moment to sign the petition. It will take only a couple of minutes, and will be the best thing you can do to help Daniel and his fellow PNH sufferers.]
Daniel said he lives with a disease that effects his life on a daily basis, and was excited about prospects to help his, and fellow sufferer’s, situation.
Paul spoke on behalf of his young son, “Ja”, advising the group of the rare disease, Hunter Syndrome (MPSII) that affects him. His son had undergone a bone-marrow transplant in October last year, following twelve weeks of Enzyme Replacement Therapy (ERT). Paul’s son was one of the few cases where Pharmac has funded ERT, and they appeared on “Campbell Live”. (See: Campbell Live – $500,000 drug treatment for boy)
Paul said that his son is doing “really well” at present, but will be going back to Starship Hospital for further, ongoing treatment.
Shannon also spoke on behalf of her three year-old young boy, “Je”, who has a life-threatening, genetic disorder. She rattled of the lengthy, tongue-twisting, medical name for the disorder with an ease that suggested how frighteningly and intimately familiar she was with her son’s condition. (I doubt most doctors could repeat the name with such ease.)
Shannon explained that whilst her little boy was presently stable, there was currently no cure for the condition. “Je” has plasma infusions every week – not much fun for a three year old lad who should be out playing with his mates and doing all the things that other three year olds do.
Shannon said that at some stage he will need “soliris” – “the sooner the better for a quality of life”. She hopes for funding in the near future, and in the meantime has been privately fund-raising. Shannon is asking ten thousand people to donate $1 a week, to pay for upcoming treatment with ‘Soliris’. She said they were a quarter of the way to her target with two and a half thousand people making $1 weekly donations, plus extra donations coming in from other fund-raising activities, events, and auctions.
“I basically fund-raise non-stop… I’d like to basically give him a relatively normal life ,” she told the Forum.
Despite Shannon’s soft-voice and demeanour, she held a steely determination to do the very best she could for her son. She is obviously not going to give up on him.
People wanting to help Jethro can do so through the following;
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Told the Forum that her eleven year old son, “Co”, was diagnosed with a rare disorder two years ago. She said, “there is no cure, and there is no treatment”.
Jo said that her family imports a drug from the United States called “genistein“, but it’s use is “purely a trial”. She said it may help to ease the symptoms.
Jo said that “Co” is still walking – but his life expectancy was mid-to-late teens only.
At one point Jo found it difficult to explain why she was attending the Forum, but said that it was good to be with other people who had “been through the same journey, and probably understands”. She referred to the “huge” implications of a late-diagnosed disease and ongoing battles relating to her son’s condition.
Marianne is from Dunedin and both her adult sons are affected with the same rare disorder, MPS1. She said that whilst both her sons are “living relatively normal day-to-day with the condition, as much as they can”, there was no enzyme-replacement therapy that would be effective for their particular situation.
Marianne said that they were living their lives in the “present day like kids would their age” but that the disease might not manifest problems until they were much older. She said she was attending the Forum to be kept up to date “in the loop”, and appreciated that others who were present, had a “hard journey to make”.
Brenda’s family in the UK had been devastated by a rare disease and told the Forum that “it’s now working it’s way through family here”.
Brenda’s granddaughters have not been tested yet, and said that was “a shock yet to come”. She says her daughter, “D”, has Fabry diagnosis – hence a 50/50 chance that her two granddaughters may also have the disease.
Brenda said she had been “a bit naive all my life”, thinking that “when you got sick, you got treated”. But she said she was horrified to learn that even living in a developed country that was not always the case.
Brenda said she wanted to help, “I want to do something, I don’t know what….just to do something, to kick arse!”, which prompted laughter from the Forum attendees.
Jane told the forum that her condition was often just a chronic disease but in her case was more serious. She’d recently sought an opinion from our nearest centre of excellence for the disease in Melbourne where she was told that despite having ‘a performance level of 0’ and being ‘extremely well’ she is now in the ‘high risk’ category. The specialist described her as being “a really good ice skater but that the longer she skated the thinner the ice”, and she could “crash through” at any moment.
Quite liking being alive, Jane’s uses her skills and resources to challenge and achieve opportunities. She expressed her determination “to get what I need“, and referred to her “terrier” nature; getting her teeth in; and not letting go, but it takes huge effort.
Jane said “the rules of engagement are not explicit” and it is hard to work them out. They [the system] expected people in her situation to follow a certain “role”. She said if you altered that, it can make a big difference, but not everyone could do that and many would just walk away.
A treatment is available in many other countries for people categorised as high risk however, it’s not available here. She has decided to self fund even if it means becoming “houseless” in the process. However she expressed real concern and called it “dreadful” that someone else with similar health status but without her resources would just have to accept that only supportive care would be available for them.
Susan, Jane’s Friend
Susan and Jane have been friends for the last 20 years and as she put it, “it’s a helluva journey to be a support person“, and immediately expressed her feelings at what she had heard from others at the Forum.
Susan launched into a well-reasoned, articulate description of the system. She also said that whenever they go to Capital Coast Health [Blogger’s note: Wellington Public Hospital as it was once called, in simpler times], it was as if they were teaching them that they were people first and patient second.
Susan referred to the “ease with which judgements are made” with regards to Jane’s situation and that often it was necessary to challenge what was behind the judgements. She said not knowing how the system worked that made being proactive in support of Jane more complex. For example under what criteria would a person qualify for special assistance and does a successful application come with additional funding.
Dr Greg Coyle
Dr Greg Coyle explained that he is a principal advisor to the Salvation Army who has written a thesis on exploring the boundary between what was provided to an individual, compared to what was provided to society as a whole. His thesis explored how fairness came into the issue. He said it was a policy analysis, and looked at how the system worked and did not work.
He said he looked at how disadvanged people have huge barriers put in front of them for the normal and simple things in life.
Dr Coyle advised the Forum that he would explain his thesis, in more depth, tomorrow.
Freda, of Ngapuhi, greeted the Forum in Te Reo.
Freda told the Forum that twentyone years ago, she had been diagnosed with “acid maltose glycogen deficiency” – aka Pompe Disease. In fact, Freda was the first person in New Zealand ever to be diagnosed with the condition.
At first, Freda was happy with the diagnosis, as she had been sick for a long time without knowing the cause. That happiness rapidly disappeared when she was told; there was no cure. Her reaction was simple,
“What the ‘heck’?!”
At this point in her life, Freda had a young, three year old son, and her oldest son was getting reading to go to Boarding School. And now she’d been diagnosed with a condition for which there was no known cure.
She asked the specialists, “Well, what do I do?“.
One specialist replied, “Nothing. Go home.You probably won’t live till 40.”
Freda did live to see her 40th birthday, and in her own words, celebrated the event with a “hua of a party – two days, we partied!”. And every birthday party since then has been an unbridled celebration of another year lived, and the disease kept at bay.
Freda says that whilst it is her pure determination that has kept her going, that it is getting harder for her to walk and get around.
Freda then shared with the Forum the chilling fact that she had applied three times with Pharmac for treatment – and had her application denied all three times. She said she had been given a “host of reasons“.
When asked by this blogger if she would apply again, Freda said she was considering a fourth application. She said she was thinking about it. Having just become a grandmother, and seeing another granddaughter come into the world in three weeks time, Freda said she wanted to take them to Kapa Haka; she wanted to take them to ballet. “Because trust me,” she said, “both my grand-daughters, they’re going to do ballet“.
Freda said she wanted to sing to them, as she had done for her sons.
She then wondered if it was worth putting in a fourth application, and suffer the emotional let-down? She said, “stuff it, I don’t want to be let down again“.
She said her condition was worsening and even a common cold affected her harshly, as she had very little immune system. If she fell, Freda said, her bones would not mend,
“I’m getting tired. I’m so tired… It’s a struggle. I think all of us are in the same boat. Be it your daughter. Be it your son. Be it yourself, we’ve all been there“.
Freda brightened when she referred to Allyson, who was sitting beside her, saying,
“Allyson, she and I may not be bioligically comnnected. [But] She’s my sister. For she’s the only other woman in this country who has the same disease as me.”
It was something that this Blogger noticed; Freda and Allyson seemed to have a special bond. Closer to being sisters than friends. I wondered if their mutual support for each other gave them both added strength – to keep going despite constant rejections for medical treatment, and a looming future that was bleak.
Allyson began by confirming her status as a Pompe Disease sufferer. She said, with a warm smile at Freda,
“We, that’s going to be a hard act to follow…”
” Well, I would break out, with a waiata, but...”
They both laughed at that and Allyson continued,
“…All of us are struggling. None of us should have to struggle this hard to get treatment. Just having the disease is bad enough. Why should you have to grovel to Pharmac and have them say ‘no, you’re not worth spending the money on’.
I don’t quite know what to do about it. We’ve tried and tried. We have to keep trying. Eventually they’ll have to do something.”
Allyson then pointed to the National Government over-turning Pharmac’s decision on Herceptin and another medication,
“They’ve set a precedent for being able to do that [over-turning a Pharmac decision].”
Allyson also pointed to Pharmac using high costs of drugs to garner public sympathy for their refusals to fund treatment,
“It’s actually a lie and they say that to try and gather sympathy from the public and people say , ‘Oh well, yeah why should we spend $500,000 on you when they could help fifty other people with diabetes?'”
But we’re going to die if we don’t get help, she said.
“But every time you try and stand up to them, to say ‘Hey we really need this thing, ‘cos we’re actually going to die if you don’t, ‘cos this is a fatal disease that we’ve got”… they sort of clobber us down by saying ‘well, you know, if we help you, then Joe over here is going to die along with a hundred mates..”
“We need everyone to be helped.”
Allyson added that she was one of the “lucky few” who was part of a drug-testing trial. Every two weeks she had to travel to Brisbane for treatment.
The travel involved leaving on Wednesday by driving from Masterton to Palmerston North; flying from Palmerston North to Auckland; flying from Auckland to Brisbane; driving next day to a hospital; having treatment; next day flying from Brisbane to Auckland; staying in Auckland overnight; then flying from Auckland to Palmerston North, and then driving from Palmerston North, home to Masterton. In the meantime her husband took time off work to care for their children. (Travel, food, and accomodation costs are met by the drug company.)
This routine takes place every two weeks.
It should be pointed out that Allyson uses an electric mobility-scooter; tires easily; and has difficulty breathing by late afternoon. She can stand, but not walk for more than a few steps.
Imagine the outrage if a government attempted to force a similar wheelchair bound person – suffering from a disease that could be treated easily here in New Zealand – to travel overseas every two weeks.
The stress of this constant travel cannot be helping Allyson’s already weakened state.
[Blogger’s note: by late Wednesday evening, Allyson’s breathing had become a noticeable wheezing. She took laboured breaths at semi-regular moments. I cannot help but wonder at the damage high-altitude flight, in a pressurised cabin, must be causing her. Perhaps Health Minister Tony Ryall and Pharmac Medical Director Dr Peter Moodie, should accompany Allyson next time she makes her arduous journey to Brisbane and back? My money is on a big, fat, ‘No!’ to that.]
However, Allyson expressed her gratitude that she had the option of engaging in the life-giving drug trial. Allyson knew she was one of the lucky ones. She pointed out that Freda is already too frail to make the flight to and back from Australia. Freda has not even been given the choice of participating in the drugs trial. She is receiving no treatment. Freda is simply awaiting her fate.
[Blogger’s pi**ed off note: One wonders if this is good enough for our country? Or is it ok because it’s happening to someone we don’t know?]
Allyson commented that she had an anger towards politicians in the Beehive,
“They spent nearly eight million dollars on chauffeurs. This was, I think, 2010 Vote Ministerial Services. Where I got that was from the Treasury website. So that was verifiable, that’s what they spent. That would help a lot of us in this room. But that was just for one year.”
Greg Coyle added,
“The point you’re making is how does the government prioritise the life of one person?”
Allyson agreed, adding,
“Is providing a limo service for all the dignitaries from overseas and our ones here, at $8 million a year, more important than saving a life? That would help a lot of us in this room, right now.”
Jenny introduced herself as Administrator at Lysosomal Diseases New Zealand head office. It is a field she has worked in for twelve years. Jenny is close to the members of her association, saying,
“I’m right at the coalface and I work quite closely with these guys. We cry together, we laugh together. Obviously it’s very distressing seeing these guys get turned down time and time again [by Pharmac].”
Jenny is also a mother to a son, “Ha” and daughter, “Sa”, who have a very rare disease – in fact the only two people in the country with this particular condition. She said that her role is to fight for good clinical outcomes. Jenny said that managing her children’s rare disease has been difficult, and pointed out that there have been many times when “Ha”, in particular, has been put at risk by the inaction of her local DHB.
Jenny therefore has many roles; organisational administrator, advocate, and a mother.
Chris is Chief Executive for Muscular Dystrophy Association of New Zealand. He said that Freda and Allyson were the reason that he was attending the Forum, as Pompe Disease was also a neuro-muscular condition.
Chris told attendees at the Forum that Muscular Dystrophy Association covered forty different neuro-muscular conditions, including muscular dystropy. All are rare diseases. With the exception of Pompe Disease there was no cure or treatment for any of them.
Chris remarked that he had not seen Allyson for two or three years, and that her disease had taken it’s toll on her. With a wry smile, Allyson said she thought that Chris was amazed she was still alive. Allyson added,
“Damn, I thought you were going to say I looked good.”
Chris went on to say that he was moved to hear other people’s stories; people who are directly affected by the issues; particularly Freda and Allyson and that it “grounded him back to reality”.
He thanked the Forum for the humbling privilege of being invited to attend.
Olivia is a director of HAE Australasia, which is a patient advocacy and support group for Hereditary Angioedema or HAE. Olivia said there were 40 known patients in New Zealand alone and possibly more undiagnosed. She said HAE is a swelling disorder that can have sudden onset. The condition is life-threatening; extremely painful; and an attack can take place in twenty minutes. There is only one treatment available in thre country. Olivia said there was often difficulty in explaining the condition to EDs at hospitals.
Olivia said that her group is looking for both additional treatments both here in New Zealand and overseas, as well as better access to the one treatment already available.
Lucy is a lawyer who has given assistence to John on various issues. Lucy said she she was also concerned at issues of morality and what sort of society do we want to live in. She said she wanted a society where people who were vulnerable had wrap-around services, rather than being isolated and having to fight the system.
Lucy said there should be abilities for exceptional circumstances; where discretionary decisions can be made.
Lucy said that Pharmac’s process did not provide for exceptional circumstances or funding for treatments outside their usual criteria. But listening to these cases, “is this not the exceptional circumstances they’re talking about“?
She said there will always be people who are not the greatest number and who are at the margins with rare diseases. What kind of equity was there in a system that ignored their needs? Lucy voiced her frustration at a system that ignored people’s needs. She gave an example of ACC providing a better service if you were disabled through an accident than if you were born with it. These are things that reflect on our society.
Lucy closed by saying she would be willing to offer her legal services to help in this area.
At this point one of the attendees quietly asked in a hopeful voice, “Do you do divorce cases?”
There was instant laughter to this.
Lucy declined to take the case.
She did, however, make the point that there was a big difference to where only one drug was available for a rare disease than to situations where Pharmac had to consider a fourth generation drug over a third generation product already funded. In cases of rare diseases, there was no drug treatment being funded by Pharmac whatsoever.
Lucy had no sympathy at all for people in high decision-making roles who wanted to put some difficult issues into the Too Hard Basket, by saying they couldn’t assess morality as part of any decision. Lucy was adamant and said,
“Well tough luck, buddy, you signed up for that decion-making role. You stepped forward to make those tough decisions – so just make them. Don’t say you can’t assess them.”
Meeting and listening to people at this Forum gave me two valuable insights…
- Their afflictions – many of them untreatable and terminal – had not affected their sense of humour. They were still able to crack jokes – often at their own expense.
- The people in the room were ordinary New Zealanders – mostly middle class; ages ranging from early 20s to 60; men and women. These were the “mums and dads” that politicians often refer to when seeking their votes at election time. And they are the ones who will most likely vote.
These folk are not seasoned political activists. They are people that live next to us in our neighbourhoods; who pass us on the footpath; and stand in line with us at the supermarket.
They are the face of New Zealand. And they have come up hard against The System and political intransigence.
Where people’s stories are continued – and begin to touch upon reforming Pharmac…
Previous related blogposts
Priorities? (19 Oct 2011)
Terminal disease sufferer appeals to John Key (12 Nov 2012)
Pharmac: The politics of playing god (16 June 2011)
$500,000 a year to keep toddler alive (5 Feb 2013)
Rare disease sufferers want pricey treatments (1 March 2013)
Rare disease takes awful toll on boy (1 March 2013)
Facebook: Support for Jethro Morrow Facebook Page
Facebook: NZ Rare Disease Day
Website: NZ Rare Disease Day
Website: Pompe Network
Facebook: Lysosomal Diseases New Zealand
Website: Lysosomal Diseases New Zealand
Facebook: NZ Organisation for Rare Disorders
Website: NZ Organisation for Rare Disorders
Pompe Support: Petition to Government Fund Myozyme for Pompe Patients
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