“One should judge a society by how it looks after the sick and vulnerable” – part rua
John opened the NZORD seminar with a welcome to attendees and members of the media. He was sporting a newly designed purple ribbon, denoting support for people with rare conditions, and the every day struggles they face in their lives.
Paul began by telling those present about his son, “Ja”, and his rare disease, Hunter Syndrome (MPSII). “Ja” is only one of two children in the country with this rare disease.
Paul’s concern, he told listener’s, was the problem of fairness in getting access to specialised medicines for rare diseases. Paul said that current enzyme replacement therapy, available world-wide, was proven to have an effect on this disease, by slowing its progress.
Paul told the seminar that in June 2010, Pharmac’s Dr Peter Moodie appeared on TVNZ’s “Close-Up” (now replaced by ‘Seven Sharp’) agreed that the drug ‘Elaprase’ (an enzyme replacement therapy) was an effective drug for “Ja’s” disorder. The drug was approved for the second child suffering Hunter Syndrome – but bizarrely, six months later, Pharmac declined access for “Ja”.
Paul’s son’s doctors immediately placed a second application for Pharmac to get funding for this treatment for his son.
Paul said they got results when they went on nationwide TV (See: Campbell Live – “$500,000 drug treatment for boy”). He was not the first case where media attention had both accelerated the decision making process, but also had a positive treatment result. As he said,
“When you talk about fairness and equity, this is the perfect example of two cases, of two kids, with exactly the same disease, wanting exactly the same medicine. One was allowed access, one wasn’t. And we had to go on TV and beg for the drug. Parents of kids with rare diseases shouldn’t have to do that.”
Paul’s address to the seminar was short, lasting only a couple of minutes. But the power of his soft-spoken words, describing his family’s experience in their dealings with Pharmac, was sufficient to get his message through, with chilling clarity.
Jo wanted to share her perspective of how a rare disease affects an ordinary family. In fact, Jo’s experiences and struggles were anything but “ordinary”. Jo described herself as the “proud mother of two wonderful boys”, “J”, 9, and “C”, 11.
“C” was diagnosed two years ago with a rare brain disease for which there is no cure; is degenerative; and life expectancy is mid-to-late teens.
At this point, I wondered at the inner strength of a parent who could talk about her child’s impending fate, in a room full of strangers. There was an underlying tension in Jo’s voice – unsurprising considering the subject matter she was talking about. As if to underline this, Jo said,
“No one said life was going to be easy, but we were not ready for this.”
Jo told the seminar that a drug called “genistein” – whilst not a cure – might help ease the symptoms. The drug is not available in New Zealand and they source it from a pharmacy in New York, USA. She said the US pharmacy will not permit the medicine to be sold to a local pharmacy here in New Zealand. They will only on-sell to patients and their families.
Jo outlined the process by which they purchased “genistein” from overseas. She had the support of her pediatrician in Dunedin; a metabolic specialist from Auckland’s Starship Hospital; and advice on dosage from another specialist, a Professor, in Poland. They then “crossed their fingers” that the exchange rate was favourable when they made the purchase.
She then told the seminar that soon after the medicaton arrived into the country, they were phoned by NZ Customs, demanding payment of GST and Duty, on the medication.
Jo said they no longer have to pay some of the Duty on the “genistein”, as they were exempt. She added, “I struck the nicest guy [at Customs] who had a heart“.
Jo questioned why her family should have to pay GST for a drug for her terminally ill son – a drug not otherwise available here in New Zealand. She asked,
“Why should the government benefit from my son having an illness that will take his life. It is not a lot of money, but it is to us.”
Jo has written a book called “It’s OK”, about a child with a disability, starting a new schiool, and another child recognising that it’s ok to have a disability. She said it was the book they had been searching for, in relation to their son’s condition. It did not exist, so she wrote it herself.
Jo said that her local community in Otago held regular bingo nights to raise money to purchase the “genistein“ for “C”, and was hugely appreciative of their effiorts to support “C”.
Does the “genistein” work? Jo said they don’t know, and with a hint of resignation in her voice said,
“There’s nothing else. That’s it.”
Jo then told listeners that their son, “C”, will become a donor. Jo said that she and her husband were trying to think of other parents, and hoping that this decision would help them,
“It will be ‘C’s’ gift to them.”
[Blogger’s note: At a time when Jo and her family are faced with the unimaginable, they still have the courage and forethought to think of others. This is people in their darkest moments, rising and being the best they can. I cannot put into words what I feel as I listen to Jo’s voice on my Recorder and write them here, for others to read. To continue Jo’s story…]
Jo said it was impossible to try to explain, so others understood, how such a rare disease affected their family. She said, “as a mum, there are days I cannot breathe“.
As Jo tried to describe the heart-wrenching feeling , as the disease took more and more “everyday things” from ‘C’, her voice began to break. She said,
“There is not a day that I walk past ‘C’ without touching him as one day I will not be able to.”
She said she enjoyed days when she watched her boys play. “Life,” Jo said, “was not a dress rehearsal“, and she emphasised making the most of every day we had. She ended her address by saying,
“Families dealing with a rare disease should be able to enjoy the time they have, not have to spend their time fighting for what they need.”
Shannon began by explaining that in 2010 her eight month old infant son, “Je” was diagnosed with kidney failure. At the time, she says, she believed “that was all she was dealing with“. Later, it was discovered, “Je” was suffering from a rare disease with a long, complicated name (but which Shannon can roll off her tongue with extraordinary ease), with the initials aHUS.
The condition is a genetic mutation resulting in chronic kidney failure, high blood pressure, neurological damage, risks of heart attacks and strokes and “the list is pretty endless“.
Though her young son was in bad shape at the time, doctors managed to restore kidney functions assisted with dialysis and stabilise him with plasma infusions. Later still, Shannon and her family learned that their best hope for “Je” was a drug called “soliris” – an expensive medication. Though “Je” was undergoing weekly plasma infusions – not much fun for a three year old little boy – that was not a long-term solution. When infusions are no longer effective, “Je’s” only other option will be “soliris”, Shannon said.
The medication is not funded by Pharmac.Two applications have been made to Pharmac – both rejected by the Agency.
Shannon and her family are busy fund-raising (see previous blogpost: ), knowing that every dollar they manage to raise will be a dollar that goes towards saving “Je’s” life. Shannon describes her activities as “a mad fund-raising mission”.
She says that “Je’s” health is currently stable, but that 60% of aHUS sufferers do not survive their first year of diagnosis., “so we’re very lucky there”.
Shannon concluded by saying, “we have a pretty intense journey ahead“.
Blogger’s note: “Je’s” life will depend upon the generosity of ten thousand strangers, each contributing $1 per week to save him – an awful dichotomy between the life of a child and $1 per week. It is amazing how Nature’s cruelty can be offset by human beings’ love and compassion toward each other.
Allyson (assisted by Jenny)
Allyson has Pompe Disease – a condition shared by only six other people in this country.
With Jenny’s help, Allyson read from a pre-prepared power-point presentation on her lap-top.She called it “Innocent people dying under John Key’s Watch”.
Quoting Treasury’s website, Allyson stated that $7.9 million was spent annually on Ministerial cars and chauffeurs. The figures covered Members of the Executive (government Ministers); leader of the Opposition; former Governor Generals; former Prime Ministers; their wives’ judiciary; Distinguished Visitors (see: Govt paid $6000 limo tab for Warner Bros in Hobbit talks); as well as self-drive vehicles for Members of the Executive.
Allyson suggested that $8 million could pay for life-giving medication for everyone in the room.
Allyson pointed out other examples, such as drunk-drivers who killed someone being elegible for ACC, if they also happened to injure themselves. Even incarcerated criminals, she said, received more funding from government than sufferers of rare diseases did.
By comparison, she said, rare genetic diseases like theirs “got zero”.
Allyson’s application for enzyme replacement therapy treatment (ERT) has already been declined twice by Pharmac. “But I’ll go for three“, she said with a quiet chuckle. She said that the medication would slow down, or even stop, the progression of her disease and offer a modest improvement, which is a “huge thing for a degenerative disease”. But Pharmac still rejected her application.
Allyson said she’s currently on a trial programme, for which she has to fly overseas – initially to Florida, and subsequently to Australia – and had to fund-raise by selling raffle tickets outside a super-market.
“Selling raffle tickets to save my own life. That’s pretty bad, isn’t it? For New Zealand. Or any other country.”
Allyson said there were sixty countries that funded ERT, but New Zealand and Australia were not one of them. “So we’re really in the Third World.”
Allyson explained that currently she was part of a drug trial that involved her travelling to Brisbane every two weeks (see previous blogpost for details). She was obviously worried that if the pharmaceutical company stopped the trial at any time, she would have to go “cap in hand” to Pharmac. There was an edge to her voice – Allyson must have realised the likelihood of Pharmac granting consent to her application would most likely be nil.
Blogger’s note: Allyson’s life is currently in the hands of a pharmaceutical corporation that owes very little to her. She is assisting their drug trial in a purely commercial transaction. By contrast, our own government does nothing for her, or others, suffering from rare diseases.
Allyson considers herself lucky to be part of this drug trial, and says,
“The alternative to not getting treatment isn’t worth thinking about.”
Olivia began her talk by explaining the nature of Hereditary Angioedema (or HAE).
HAE is a potentially fatal, genetic condition that involves parts of the body swelling, and affects one in 50,000 people worldwide. There are 49 known sufferers here in New Zealand.
Olivia said that an attack can occur in the stomach, internal organs, and sometimes fatally, in the air-tract. Death by asphyxiation can occur in under twenty minutes, if not treated appropriately. She said there is one treatment only, which is available only in certain major hospitals, for in-patients.
This current situation affected families of HAE sufferers who had to familiarise themselves with the locations and means of access to specific hospitals that carry suitable treatment. Olivia said that because frontline staff are not always familiar with the condition, there can be critical delays in treatment. Olivia pointed out that a sufferer in Spain died during an attack, when hosital staff failed to recognise the condition correctly – even when presented with information regarding HAE.
As a consequence, Olivia said that,
“International physicians are recommending that patients have the ability to carry their own medication for treatment at a hospital...”
Olivia added that there is a new medication available to 37 other countries, including Iceland and Russia.Whilst the drug is not available here in New Zealand, if it were, it could be self-administered by the patient in his/her own home.
Olivia outlined what was required for HAE sufferers,
“We need equal and fair access for all patients across all of New Zealand. Even if they live in a smaller town or city. We need timely access to treatments, either through a patient carrying their own supply, or through the ability to self-administer at home which is available in other countries as well. Also access to the best treatments that are available in other developed nations. These allow early treatments at the first sign of attack, meaning less hospital admissions; less severe attacks; and the ability for patients to carry on with their every day lives. “
Olivia criticised the current system which forced people to spend their limited time and resources on fighting for access to treatments, rather than “looking after the health that we do have“.
“And we don’t think that’s fair.”
Daniel is a 32 year old father of one. He suffers from Paroxysmal nocturnal hemoglobinuria (PNH). He started by explaining his “journey”, and which started with experiencing odd symptoms that his then- partner suggested that he check up on. His GP insisted on blood tests which led to an MRI; then to a urologist; and then haematologist… and finally after nine months, a diagnosis of PNH was arrived at.
Daniel’s current treatment is referred to as a “supportive treatment”. This does not address the underlying causes of his condition, and only manages his blood levels, which are constantly fluctuating.
For deeper treatment, Daniel requires “soliris” – which Shannon’s son also needs for his condition. The drug prevents the body’s immune system from attacking red blood cells.
Daniel explained that many of the new drug treatments “are at the leading edge of science and health technology” and by funding these innovative new treatments, they will eventually have greater applications and benefits. He said that by not funding these new treatments, the government is putting out a message that it is not interested in seeing science and technology develop in such a way as to treat other diseases and benefit more people.
As a treatment, Daniel was offered a bone marrow transplant – which has considerable difficulties and risks for complications involved. With no sibling as a close blood-type match, Daniel would have had to seek assistance from a non-relation who matched him as closely as possible. He said a bone-marrow transplant offered a 50/50 chance of success – or coming out at the end in a worse state, “or potentially dead“.
An alternative was presented to Daniel when a UK specialist shared his research-findings in April last year (2012), regarding the use of “soliris” to treat PNH sufferers. What Daniel heard was like a beacon of hope being switched on,
“With the treatment option [ “soliris”], patients were living for the same period of time as the general population, so mortality rates had dropped away. Without treatment, the expected survival is around ten years, so the median is about ten years, post-diagnosis. But with treatment, patients were living long and full lives. And the important thing for me was hearing some of the quality of life strories which he shared…living with PNH, one of the major symptoms is this constant and sometimes quite severe fatigue burden… to live day to day.“
Daniel said it wasn’t just physical but the psychological impacts of living with a chronic disease, and the awareness that it’s “not going to go away”.
Daniel described “soliris” as an “amazing treatment” that is “now proven to give people back quality of life” as well as extending their life span and which has been part of the British public health system since 2008. In Australia it has been available since 2011.
Daniel asked the UK specialist,
“What would you do in my situation?”
The reply from the visiting doctor was unequivocal, according to Daniel,
“There’s no question. If you were in the UK you’d be treated with “soliris”. You don’t go down the bone-marrow transplant [option], there’s too much risk involved. Internationally doctors are moving away from bone marrow transplantation. Come to the UK.”
Whilst he was tempted to make use of other country’s health systems, Daniel recognised that there were many others in New Zealand – many of whom were young people – who desperately needed leading -edge drugs such as “soliris”, and he couldn’t “run away from these problems”. He said he and others were entitled to receive treatments that he had been paying taxes for a number of years.
Instead, Daniel did what Kiwis are famous for – he decided to take a stand for something which he recognised was grossly unfair. He and others set up a patient association [The PNH Support Association of NZ] and have taken to lobbying government and Pharmac for funding for “soliris”.
Daniel made the point that PNH “rogue cells” are common in approximately 10% or 20% of the population. Occassionally, these rogue cells over-whelm healthy cells in the bone marrow and the disease becomes manifest. However, the visiting UK doctor informed Daniel that with life-spans extended by soliris, sufferers were often experiencing “spontaneous remission” as the body had more time to adapt and experience a kind of “cure”.
Daniel then shared his experiences with Pharmac, after he and others had launched a media campaign, earlier this year, highlighting their difficulties at getting funding for treatment. He said,
“I felt it was fairly non-agressive, fairly straight-forward… ‘We’re a group of patients; we’re sick; we want access to a particular drug. We didn’t hide away from the fact that it was a high cost treatment…”
He said, “we didn’t really attack Pharmac, we simply told the story of ‘this is a treatment which has been under consideration for over 14 months’. We made it clear that there had been funds provided by the drug company to support our media relations company, [but] none have come directly to the Association, but they have been paying for us to have access to this group. And we told our story.
And immediately on the same day, we began to see what John has described as the ‘clobbering machine’. The misinformation machine… essentially Pharmac is a hugely resourced government agency. It doesn’t take these stories lying down. It fights.”
According to Daniel, Pharmac fought back in the media with a variety of strategies, including denunciation by recognised leaders in various medical-related fields and painting the PNH Association as a proxy, attempting to speak on behalf of pharmaceuticals. Pharmac was also derisory about PNH sufferers, dismissing it as a “fashionable disease“.
(Blogger’s note: if, reading this, you are experiencing a sense of unease that taxpayer-funded state agencies are able to employ taxpayer-funded ‘spin doctors’ to counter public concerns surrounding Pharmac funding decisions – that is a normal reaction. It signals that, in an Age of Madness, you are sane.)
Daniel said that many of Pharmac’s public statements did not make sense and it appeared that there was “a mechanism” within this public agency that worked to actively counter any argument it deemed critical of it’s performance. He said that whilst Pharmac was entitled to make its position clear, that it seemed that the agency was speaking through other groups and ‘proxies’ to put their message out to the public. Daniel said “to me, this is an abuse of power“.
He said that the system is broken if people, such as the ones who had spoken at the seminar, were having to beg for treatment whilst being very sick.
Daniel said that he felt that the disease had not only taken over his health, but had taken over his life and that he was having to counter Pharmac and fight for what he believed to be a simple request; access to healthcare.
He said his concerns were two-fold.
Firstly Daniel believed that the system was “broken” and that decision-making for funding treatments for rare diseases be taken away from Pharmac, and given to a separate decision-making body. Such a new body, he said, would recognise a “different equation” where, by default, rare diseases would have to be treated differently.
Secondly, Daniel said that “we need to be looking at Pharmac and asking some questions about how it operates”. He said that as a government agency it is accountable to the citizens of the country, who it is supposedly serving, and has no mandate to attempt to “squash a small patient group”. He said there were problems around dissemination of mis-information, poor presentation of decision making processes, and a lack of transparency.
He repeated his assertion that, to him, this was an abuse of power by a governmental agency, and he hoped for better outcomes than this.
(Blogger’s note: And it should be said that when a government agency – of any description – uses media streategists and spin doctors to counter public concerns and criticisms – they are using our own taxes against us.)
Jane began by telling the seminar how shocked she was to hear other peoples’ stories on the previous evening of the Forum (27 February). She said she considered herself “lucky” in that she has resources, plus resources of her close friend, Susan, who Jane describes as “incredibly astute and on-the-ball” and very supportive.
“So together, we quite a formidible team.”
Jane said that she’s managed to cope with her situation, but expressed concern for those less fortunate and with less resourcing and support.
“What about all those other people when someone looks them in the eye and says, ‘We’re terribly sorry but this is a disease that we just have to watch and wait‘?”
Jane detailed her own situation from initial diagnosis; the search for a stem-cell donor (no match anywhere); flying to Melbourne to consult a specialist; and finally getting approval to commence a specialised drug, “azacitidene”.
“Azacitidine” is not available in New Zealand. She said therefore that you either fund it yourself – or you don’t have it at all. Jane said if she doesn’t have it, “she’s going to drop through the ice” (See previous blogpost: “One should judge a society by how it looks after the sick and vulnerable” – part tahi )
Though not funded by Pharmac, “azacitidine” has won approval for it to be administered. Her treatment starts in a couple of weeks.
Jane then posed the question to the seminar, “so why am I here?”.
She answered that she had the skills; the resources; the “team”; to work her way through the system; to research and network and therefore give herself a chance.
“What about the person who can’t do that? What about the person who hasn’t got the resources? … They’re going to have no chance.”
Jane concluded by saying,
“I just have this real concern that this is not fair. Health systems are not fair and equitable. It really relies on the individual, and relies on luck around the individual…This is an organisation that … by bringing people together, can make a difference.”
Where this blogger reveals the author of the quote, “One should judge a society by how it looks after the sick and vulnerable” .
And where concrete proposals are made to reform Pharmac with a bold plan for a “Baby 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
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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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