Archive

Archive for 4 March 2013

“One should judge a society by how it looks after the sick and vulnerable” – part toru

.

Frank Macskasy Frankly Speaking blog fmacskasy.wordpress.com 27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

.

Continued from: “One should judge a society by how it looks after the sick and vulnerable” – part rua

.

Chris Higgins – Muscular Dystrophy Association

Chris introduced himself as Chief Executive of Muscular Dystrophy Association. He was present to give the MDA’s perpective on high-cost medicines. Chris told the seminar that the MDA covers over forty  neuro-muscular conditions, including Pompe Disease. All forty conditions are rare, he said.

Chris acknowledged Pompe Disease patients, Freda and Allyson, who were sitting in the audience. He thanked both women for supporting him, at the seminar.

Chris opened his talk by saying that Freda and Allyson “should be given a fair go”. They deserved the opportunity for a longer, and higher quality of life, so they can achieve their full potential. He said that going by what Freda and Allyson had been saying at the Forum, that family/whanau were really important to both women.

He added that family members of people suffering neuro-muscular diseases were important. Chris said that Freda’s grandchildren should not be deprived of growing up and getting to know their nana.

He said Allyson’s husband shouldn’t be deprived of the opportunity of having a long marriage with his wife. And Allyson’s children shouldn’t have to watch their mother slowly get worse, and then eventually be deprived of her altogether.

Chris said,

“And believe me, if you’ve ever been through the experience of having your mother die, it is a truly devastating experience. It just never leaves you.”

Chris then outlined his Association’s concerns. He spoke of being worried that the health system seemed unable to respond to the  needs of people living with rare conditions such as Pompe Disease. He asked if other people with neuro-muscular diseases have to go through the same processes as Freda and Allyson have had to face.

Chris said that as new advances are made with drugs to treat neuro-muscular conditions such as Pompe Disease, and other rare diseases,  that Freda and Allyson’s experiences will be repeated over and over again, as the relatively small  numbers of sufferers result in high-priced treatments.

Chris then questioned whether Pharmac’s NPPA Scheme   system was actually becoming a mechanism to justify their decisions to denying treatments, rather than providing access to much-needed medicines for rare diseases. He added,

“We’re concerned that Pharmac seems to have the ability to ration resources using cost-benefit criteria which do not seem to be applied anywhere else within the New Zealand health system. So it seems  to be one rule for some, and another rule for others…  because of that, MDA members, and I’m sure it applies to a lot of people in this room, as well, are starting to feel really discrimnated against by the New Zealand health system. Seems if you have a rare condition, you don’t get the same consideration from the health system as other people.”

Chris said that discrimination is inconsistent with medicines strategies as well as community values and ethics.

He said there seemed no political will to require Pharmac to relax its cost-benefit criteria, so access becomes a reality rather than a vain hope.

Chris then dropped a quiet ‘bombshell’,

“We’re coming to the point where [the] MDA has lost confidence that  Pharmac will ever grant it’s members access to high cost medicines.”

Chris told the seminar that his group, in conjunction with others, will continue to support their members who are suffering rare diseases.

But Chris expressed a view that he did not believe that it would be a long term solution for the future. Instead, he offered a solution to this problem, by advocating for a separate agency, outside of Pharmac, of an “orphan drugs access programme”,

“We see that  operating within the Ministry of Health but outside of Pharmac. And we see it as having access criteria which are a lot more relaxed than Pharmac’s, and [which] acknowledge  the particular requirements of people living with rare conditions… the thresholds need to be different… and people need to be given a fair go.”

He added that a different mechanism will better address the medicines requirements of rare disease sufferers,  and which are more equitable and reflective of community values.

Chris offered an ‘olive branch’ to the Health system and stated that the MDA is willing to collaborate with both the Ministry of Health and NZORD (New Zealand Organisation for Rare Diseases) in designing an affordable and mutually satisfactory alternative programme.

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

.

Jenny

Jenny works for the support group, Lysosomal Diseases New Zealand. She gave a brief outline of events over the last few years – some of which has been covered by others at the seminar. She referred to one application, for treatment for Gaucher’s disease patients, that had “sat” on Pharmac’s desk for two years before being prompted to make a decision. Funding for that application was finally granted.

A subsequent application, for enzyme replacement therapy (ERT) for another rare disease was declined for a number of reasons, including, she said, that the applicants were “too sick” and “not sick enough“, etc..Some of those patients are now on clinical trials (Allyson) and some are untreated (Freda).

Jenny said that the 2008 election gave her group hope. The extension of Herceptin treatment from 9 weeks to 12 months was a campaign pledge from John Key in 2008 (see:  12-month Herceptin treatment now available)  –  and was enacted within a month of National being elected into office.

Jenny said,

“The Minister of Health funded [an extension to] herceptin, and we thought this is actually a really good step. And we might get some of our other Enzymes [Replacement Therapies – ERT] across the threshold.

The Minister pledged to improve access to specialised medicines…… we took families to Wellington so they could meet the politicians, and tell their stories.”

Jenny’s group thought they were making progress in 2009 when a little boy, with Hunter’s Syndrome, was quickly given Pharmac funding to  receive enzyme replacement therapy (ERT). Adult patients with similar conditions, though, were untreated and without funding for ERT.

Despite a Ministerial review to investigate how adults could gain access to treatment, there was no change, Jenny told the seminar.

Furthermore, in 2010, Allyson’s application for myozome (ERT) was declined by Pharmac, even though the Agency acknowledged there would be stabilisation and improvement to Allyson’s health through treatment.

An appeal to Pharmac’s decision was also rejected.

By 2011, Jenny said,

“We really ramped it up. We put a call out to the community and we asked our families to go and talk to their own local politicians; brief them about what we were doing;  what we needed; about the  issues around access to medicines. And John [Forman] and Chris Higgins got a meeting with Tony Ryall, who said to them that the ‘EC’ (exceptional circumstances) review would solve the problem.

It didn’t.”

Jenny’s group made submissions to Pharmac about the (revised EC scheme), the NPPA Scheme, stating that they believed that people with rare disorders would not make it past the threshold and “would be cut out”.

In September, three more applications were made to Pharmac for treatment for Pompe Disease. They were all declined said Jenny.

In desperation, the group brought a patient with Pompe Disease from the United States to New Zealand, so she could “tell her story” to Pharmac, politicians, and to the media,

“But still that didn’t make a change.”

Jenny then told the seminar about their experience in trying to seek a meeting with Prime Minister John Key,

“We went to Parliament to  request a meeting with the Prime Minister. We got turned out. They got the  security [guards] and we got bailed out of Parliament.”

Jenny read out a statement from Labour’s deputy leader, who called the decision to forcibly evict Jenny’s group, “cowardly and disrespecful”. (For more in-depth information how rare-disease sufferers have been treated by this government, see: Lysosomal Diseases New Zealand News & Issues  . See Party responses here:  Political party commitments on Myozyme and orphan drugs )

The National government had stated that the ‘new’ NPPA (Named Patient Pharmaceutical Assessment ) Scheme would solve the problem of funding for rare diseases,

“The High Costs Highly Specialised Medicines Review – a National promise – recommended PHARMAC review its Exceptional Circumstances process. PHARMAC did that and the result is a new scheme
to replace the old one called “Named Patient Pharmaceutical Assessment”. It starts next March and its budget is expected to go up from $4 million to $8 million in the first year.” – Jackie Blue

Source

Jenny said that in August last year, her group tested the new NPPA process by submitting an application for treatment for Freda (see previous blogpost). Like Allyson, Freda has Pompe Disease – but is receiving no treatment whatsover.

We thought we’ve got nothing to lose, let’s see if it works,” said Jenny.

Well guess what,” she asked, “It didn’t work. She got declined.”

Freda “was not eligible” because, amongst other reasons,

“Pharmac had not provided a pathway forward for their new scheme for rare diseases. So in October we went to the Health Select Committee to ask for help. They sent us to the Minister. Then to the Ministry. Back to the Minister. And he said go and talk to Pharmac.”

They had nowhere else to go, except on a bureacratic roundabout.

Health Minister, Tony Ryall, simply refused to address their problems in any meaningful way.

Jenny outlined the stress on families whose loved ones were either not receiving medication because Pharmac refuse to fund treatment, or, were forced to fly to Australia every two weeks to be part of clinical drug trials,

“That’s a huge stress on their families. We shouldn’t have to do this when there are treatments available in New Zealand. And our patients say ‘it’s like genocide – they pay their taxes, they’ve contributed to society, why can’t we have a fair go?’.

Jenny then asked, “Where to from here? I guess we’ll have to have a Plan B for what [comes] next.”

Blogger’s note: With each passing story that I write up for this blogpost, I am more and more impressed by the sheer heroism of these good people.

See previous related blogpost: Health Minister circumvents law to fulfill 2008 election bribe?

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

Jenny

.

John Forman introduced the next speaker, saying  that the seminar had heard from patients and from support groups, but there were very few people who could contribute to this issue from an academic viewpoint. He said that  few people with an academic or professional background were willing to challenge or criticise Pharmac and that many tended to have a sympathy for the core role that Pharmac has.

John explained that Dr Greg Coyle had written his University Ph.D thesis on the role of Pharmac and how it manages exceptional circumstances.

“It is exactly the topic that we’re interested in, and it’s exactly the kind of academic rigour, independent of a direct patient interest or family interest in this issue.”

Dr Greg Coyle

– Health Researcher

– Principal Advisor to the Salvation Army

Dr Coyle voiced his appreciation for meeting the members of the Forum the previous night (27 February),

“I want to thank the people I met last night…It was important to me for me to meet some of the people that I thought about; researched; and wrote about. And I’m the better for that.”

Following Dr Coyle’s very personal remarks that were directed to those present at the seminar, he launched into his pre-prepared speech and power-point present.

This blogger will re-print some  of his main statements and the rest of the text of his speech can be found here: How does the operation of PHARMAC’s ‘Community Exceptional Circumstances’ policy align with the distributive justice principles of fairness and equity?

“Thank you for inviting me to talk about my research. The subject was fairness and how we might know if agencies are being fair when we ration health care, particularly pharmaceuticals, in New Zealand.”

“…Today I would like to spend a few minutes talking about my study into PHARMAC’s operation of the exceptional circumstances policy (now renamed the Named Patient Pharmaceutical Assessment scheme). This policy of PHARMAC’s was interesting to me because it is located at the very boundary where the needs of the whole society and the needs of an individual meet.

Examples of how we ration healthcare in New Zealand in an explicit manner are very rare. I do not wish to suggest that health rationing is rare –it most certainly is not –but it is mainly done in an implicit manner and patients and families often will not be aware that it is even happening. PHARMAC is one agency which is explicit about limiting care. Many of you know this well.”

“…So the questions I suspect that you want answered is how much cost for the treatment of a person with a rare diseases is too much? As a country do we have processes to decide this? Are the processes rational and fair? Would providing more money for public health mean that other sectors like education, police, welfare, public housing or recovering from natural disasters would be given less? ”

“…John Rawls, an American philosopher, developed a theory of ‘justice as fairness’the chief purpose of which was to ensure that utilitarian and consequentialist thinking, in meeting the greater good, would not sacrifice the position of individuals.   He presented the principle that each person is to have an equal right to the most extensive liberty compatible with a similar liberty of others. However when social and economic inequalities are to be arranged, they should be arranged so that they are both to the greatest benefit of the least advantaged and attached to positions and offices which are open to all under the conditions of fairness and equality.”

“…Amartya Sen, a contemporary welfare economist, philosopher and winner of a Nobel Prize for economic science in 1998, developed a contemporary widely held view of distributive justice…

…  He clarified perceptions of distributive justice in terms of a person’s capability to experience justice.  Sen’s notions of justice are ultimately linked with the capability of people and how their lives are lived in the world.”

“… Sen held that the reach of health equity is immense and health care must be of primary importance in any discussion on social equity and justice;

“…health equity cannot but be a central feature of social arrangements in general”.”

“… Consequently, using these two philosophers, I posed the question of PHARMAC:

How does the operation of PHARMAC’s Community Exceptional Circumstance’policy align with the distributive justice principles of fairness and equity as described by John Rawls and Amartya Sen?”

“…I decided to distill their theories into four questions which decision-makers could ask of themselves to discover (or simply begin to debate) whether their decisions are fair and just.

Here are my 4 questions. I use the term ‘distribution’to mean decision, policy, funding or delivery of a service:

1. Would the most advantaged in society accept this distribution if they, at an instant, found themselves to be the least advantaged in society and requiring such distribution for themselves?

2. Is this distribution arranged so that it is attached to positions and offices which are open and accountable to all?

3. Is this distribution based on the efficiency of substantive opportunities and on procedural fairness in defining efficiency?

4. Is this distribution based on information available to decision makers about the capability of this person to do things he/she has good reason to value?”

“… We see the two purposes for PHARMAC in the governing legislation. One is to approve subsidy on an adequate range of quality pharmaceuticals for the general community. The second purpose is to approve subsidy for citizens whose needs are not met by the general schedule and considered exceptional.”

“… The evidence showed that in PHARMAC’s first purpose, general allocative policies have been highly successful in procuring an adequate range of quality medicines at internationally low prices.

PHARMAC has saved the New Zealand health system approximately $1.17 billion in 14 years. This has been achieved by methods of utilitarian efficiency analyses relying heavily on the Quality Adjusted Life Years calculation. PHARMAC has also expertly utilised subsidising and purchasing decisions based on evidence of clinical effectiveness.”

“… However, in PHARMAC’s second purpose,  its success has been achieved, in part, by managing the claims of individuals in exceptional circumstances in a way that has not closely aligned the Rawls’and Sen’s principles of fairness, equity, openness and consistency.

The research shows that using the functions required of it by governing legislation, PHARMAC well achieves its statutory purpose. However, in doing so, PHARMAC must deal with the tension between justice as fairness to individuals whose needs are exceptional, and fairness to the needs of wider society.”

“… There are three structural problems here. The first is that PHARMAC has no stated philosophical principal on which to base its decision-making other than limiting expenditure to achieve its budget, which is not a principle but an outcome.

The Minister, parliament, the courts, the Ombudsman, DHBs, the pharmaceutical industry, doctors or patients have no way of knowing if the decisions being made by PHARMAC are fair to both the community and the individual. ”

“… The second problem is that PHARMAC and its committees claim to use the 9 decision making criteria to guide decision making. However, my research showed that the priority, or weighting, of the criteria are never explained. So one cannot know which criteria were met or not met when PHARMAC comes to a rationing decision. ”

“… The third problem is that under these conditions PHARMAC will always feel embattled and defensive because it cannot serve both purposes and adequately protect the Minister from criticism. My research showed that PHARMAC Community Exceptional Circumstances Committees don’t record the reasons for their decisions because they would most certainly be challenged. If I was a panelist I am not sure I would record the reasons for my decisions either because of the hazard of doing so.”

“… The pharmaceutical industry has worked out how to squeeze PHARMAC in the media over funding for pharmaceuticals for rare diseases particularly if these drugs are available overseas or the drug company has made a new drug available for a short period provided to individuals on compassionate grounds. Pressure groups, such as your own, highlight individual cases in the media appealing to the ‘Rule of Rescue’to raise public sympathy and put pressure on PHARMAC.”

“… PHARMAC’s claim that it simply does not have the budget for $500,000 a year for drugs for a single person is quite true. But the government does have budgets for other things for example $30 million for a sports stadium in Christchurch, $8m a year on ministerial limousines, assisting a private boarding school in Wanganui for $3.8m,  a greater than $350m tax subsidy for the Lord of the Rings and Hobbit  pictures or the government considering recapitalizing the state coal company for $400m… I could go on. ”

“… and one million for the penguin!” [Not in pre-prepared speech notes.]

“… In this first purpose they are world beaters and we should celebrate the relatively large savings PHARMAC has made…

… However in this system the needs of the individuals with rare diseases will always take second place. To achieve budgetary control over the community pharmaceutical schedule and provide for people in exceptional circumstances are contradictory purposes. The first purpose is explicit meso-level rationing and the second purpose is explicit micro-level rationing. They are not the same and cannot be treated the same way.”

“… Well what system for analysing pharmaceuticals for rare diseases would I propose?

Firstly I believe PHARMAC should continue to do what it does well which is carefully analysing clinical effectiveness and cost utility in the approval of subsidy for medicines for public hospitals and the general community.

(Slide 14) I believe the second purpose of PHARMAC, to approve subsidy for medicines for individuals with rare diseases should be handled by another agency administered by the Ministry of Health. Shall we call it the ‘Rare Diseases Funding Agency’ Its chair and members should be appointed by the Minister of Health.”

“… The Rare Diseases Funding Agency in New Zealand would be similar to PHARMAC in that it would have a fixed budget determined by parliament for the specific purpose of deciding on all claims not just for pharmaceuticals, but also for home support and other necessary assistance for people with rare diseases in the community.  The agency would need to implement the medicine strategy including the objectives of efficient use of resources and fairness and equity considerations. I have no idea what the level of funding should be, but a starting point would be transferring PHARMAC’s budget for ‘Named Patient Pharmaceutical Assessment’scheme, I believe is $8m, to the Agency.”

“… Or, here’s an idea, a reallocation of a proportion of the savings made by PHARMAC could also be made to the Rare Diseases Funding Agency.”

“… Finally, there should be consideration of the fairness of decisions to individuals. Fairness can be a consideration because the Rare Diseases Funding Agency is separated off from the funding of pharmaceuticals of the wider public.

The agency should demonstrate openness and transparency about it decisions. ”

“…A good model of openness and accountability to consider might be the investigation and reporting mechanisms of the Office of the Health and Disability Commissioner which I hold to be exemplary.

Clearly PHARMAC and the Rare Diseases Funding Agency would have boundary issues and it would be in both agencies’interest to have a cooperative relationship.”

“… I believe that there will never be a time when all the pharmaceutical or other health needs of people who suffer rare or common diseases will be able to be met. This day will never come. But such an agency which I am proposing, underpinned by distributive justice principles, will provide an accountable, cost effective and fair system for analysing and managing the limited resources which are available for this purpose.”

“…In summary, (Slide 15) I submit that the current PHARMAC system delivers incredible value on behalf of the general community but it does not deliver justice as fairness to individual claimants who suffer rare diseases. I believe a new Rare Diseases Funding Agency, such as I have described, would.”

(Blogger’s Note: It’s apparent that a general concensus throughout the seminar was that Pharmac’s role to fund treatment for patient’s with rare diseases was not working well, and that a new agency was needed to undertake this responsibility

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

.

Lucy

Lucy has been advising NZORD of legal matters in relation to access to medicines for rare diseases. She has been undertaking this role for a couple of years. She said this has been of limited success, and likened it to trying to put a square peg in a round hole.

She said,

“But it doesn’t necessarily need to be this way. The legal framework feels like it should be capable of making these decisions.”

Lucy said that the Health Minister and Ministry “make all the right noises” when it comes to accessing treatments for rare conditions. She referred to various agencies, laws, and documents which all indicate a need to improve access to highly specialised medicines.

Lucy said that all the documents which refer to specialised medicines and principals of fairness, equity, and distributive justice (prompted with a whisper from Dr Coyle), and affordability,  should allow people to have a “fair go”.  She read from legislation and Statement of Intent relating to Pharmac,

“… including in exceptional circumstances providing for subsidies for pharmaceuticals not in the Schedule.”

She pointed out that rare disease should be the “exceptional circumstances” – “they’re the diseases that are so rare, hardly anyone has them.”

Lucy pointed out that these are the drugs that Pharmac will never fund because they don’t meet their narrow cost-benefit, health-economics basis – which Pharmac uses so well for mass-market medications.

“And why would we fund these things?” she asked. “We would fund them because,  we don’t want to abandon our citizens. We mount  hugely exprensive rescue operations  to rescue people stuck up on mountains even thought they’ve taken themselves up there. “

Lucy said that Pharmac considers that it doesn not need to take into account issues of morality, fairness, equity, and community values, because of the way legislation has been enacted. So they do not take those factors into account.

As a lawyer, Lucy said she would be assisting NZORD to ensure that Pharmac does take those factors into consideration, when making decisions.

She said,

“I don’t agree with their argument that fairness means the same processing criteria for everyone. Because it can’t be fair to say from the outset that this whole subset of patients will never be funded; ‘just rule them out’. There must be cases within that subset that deserve to be funded.”

Lucy added that in all cases the onus is on Pharmac’s decision makers to make their decisions with humility, empathy, and with respect,

“I think that Pharmac is exceptionally proud of the system that it’s developed and the $1.7 billion of savings that it’s  achieved for the Schedule. Such that, ‘they shall not be questioned’. And I think that;’s really  insulting for the people who don’t fit within that process.”

Lucy agreed with suggestions that a fundamental change to the system was required. She said that in the meantime  “legally we have what we  have”.

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

Lucy

.

At the conclusion of the speakers’  addresses, there was ample time to mingle and ask questions. In a room full of people with serious medical conditions – many life-threatening – there was a strong feeling of cheerful optimism. Never underestimate the human  ability for hope…

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

.

This blogger wishes to thank the fine people who invited me to attend the Forum; to hear their stories; and who trusted me to present those same stories in an appropriate, respectful, and honest way.

The thing that struck me the most was that these were ordinary New Zealanders who most likely have never thumbed their noses at authority; have never engaged in protest activity; and are your typical Middle Class fellow-Kiwis.

They are also people who’ve not been well treated by the system – including politicians who often promise The World – but when held to account – find shabby excuses not to help those who desperately need it.

This country – our government – could help sufferers of rare diseases. The money is there. Perhaps politicians could cut some of the subsidies they give to businesses; build one less motorway; take a bus instead of chauffered limousines (which Green MPs do very well); or any other number of savings.

The money is there.

It’s the will to spend it on medicines, which is lacking.

And to Allyson and Freda – you two are fabulous! My thoughts are with you both (and all the others who I met at the Forum, and those I didn’t).  May the politicians and others in high positions of decision-making hang their heads in shame for how they’ve let you down.

You’re simply the best.

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

.

So who uttered the quote, “One should judge a society by how it looks after the sick and vulnerable”?

Look for yourself,

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

.

Fine words, Mr Prime Minister. Perhaps these people would like to know how deeply you believe them…

.

**Update** **Update** **Update** **Update** **Update** **Update**


NZORD has annnounced that it will be approaching the Office of the Ombudsman.

.

*

.

Addendum

.

Key defends 'small' Longstone payout

Source

.

*

.

Previous related blogposts

Priorities? (19 Oct 2011)

Terminal disease sufferer appeals to John Key (12 Nov 2012)

Terminal disease sufferer appeals to John Key – Update & more questions (28 Nov 2012)

Health Minister circumvents law to fulfill 2008 election bribe? (18 Dec 2012)

Johnny’s Report Card – National Standards Assessment – Compassion (9 Jan 2013)

“There’s always an issue of money but we can find money for the right projects” – John Key (20 Jan 2013)

Media

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)

Call for an Orphan drugs access policy to overcome Pharmac’s systems failure (28 Feb 2013)

Additional Information

Gregory John Coyle: How does the operation of PHARMAC’s ‘Community Exceptional Circumstances’ policy align with the distributive justice principles of fairness and equity?

The PNH Support Association of NZ

PNH Support: Petition to Government to Make Soliris available to New Zealanders!

Support for Jethro Morrow

Facebook: Support for Jethro Morrow Facebook Page

Facebook: NZ Rare Disease Day

Website: NZ Rare Disease Day

Facebook: Treat NZ Pompe Patients Now Facebook Page

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

Copyright (c)  Notice

All images are freely available to be used, with following provisos,

  • Use must be for non-commercial purposes.
  • At all times, images must be used only in context, and not to denigrate individuals.
  • Acknowledgement of source is requested.

.

.

= fs =

Advertisements

“One should judge a society by how it looks after the sick and vulnerable” – part rua

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

.

Continued from: “One should judge a society by how it looks after the sick and vulnerable” – part tahi

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.

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

.

Paul

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.

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

Paul

.

Jo

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.”

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

Jo

.

Shannon

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.

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

Shannon

.

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.”

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

Allyson (L) and Jenny (R)

.

Olivia

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.”

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

Olivia

.

Daniel

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.)

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

Daniel

.

Jane

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.”

Next

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”.

To be Continued at: “One should judge a society by how it looks after the sick and vulnerable” – part toru

.

*

.

Previous related blogposts

Priorities? (19 Oct 2011)

Terminal disease sufferer appeals to John Key (12 Nov 2012)

Terminal disease sufferer appeals to John Key – Update & more questions (28 Nov 2012)

Health Minister circumvents law to fulfill 2008 election bribe? (18 Dec 2012)

Johnny’s Report Card – National Standards Assessment – Compassion (9 Jan 2013)

“There’s always an issue of money but we can find money for the right projects” – John Key (20 Jan 2013)

Media

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)

Call for an Orphan drugs access policy to overcome Pharmac’s systems failure (28 Feb 2013)

Additional Information

Gregory John Coyle: How does the operation of PHARMAC’s ‘Community Exceptional Circumstances’ policy align with the distributive justice principles of fairness and equity?

The PNH Support Association of NZ

PNH Support: Petition to Government to Make Soliris available to New Zealanders!

Support for Jethro Morrow

Facebook: Support for Jethro Morrow Facebook Page

Facebook: NZ Rare Disease Day

Website: NZ Rare Disease Day

Facebook: Treat NZ Pompe Patients Now Facebook Page

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

Copyright (c)  Notice

All images are freely available to be used, with following provisos,

  • Use must be for non-commercial purposes.
  • At all times, images must be used only in context, and not to denigrate individuals.
  • Acknowledgement of source is requested.

.

.

= fs =

“One should judge a society by how it looks after the sick and vulnerable” – part tahi

.

Frank Macskasy Frankly Speaking blog fmacskasy.wordpress.com 27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

.

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.

27 February

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,

.

27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

.

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

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

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

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;

http://supportforjethro.co.nz/

https://www.facebook.com/pages/Support-for-Jethro-Morrow-aka-Jethro-Gantley/251266464918462

http://www.stuff.co.nz/national/health/8262237/500-000-a-year-to-keep-toddler-alive

Remember – just $1 a week!

Jo

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

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

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

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.

See: How does the operation of PHARMAC’s ‘Community Exceptional Circumstances’ policy align with the distributive justice principles of fairness and equity?

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

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.

.

Frank Macskasy   Frankly Speaking  blog  fmacskasy.wordpress.com  27-28 february 2013 - rare diseases disorders - New Zealand Organisation for Rare Disorders -NZORD

Freda (L) and Allyson (R)

.

Allyson

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…”

Freda replied,

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.”

(See: Govt’s VIP limo fleet cost goes up)

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

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 Higgins

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

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

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…

  1. 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.
  2. 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.

Next

Where people’s stories are continued – and begin to touch upon reforming Pharmac…

To be Continued at: “One should judge a society by how it looks after the sick and vulnerable” – part rua

 

.

*

.

Previous related blogposts

Priorities? (19 Oct 2011)

Terminal disease sufferer appeals to John Key (12 Nov 2012)

Terminal disease sufferer appeals to John Key – Update & more questions (28 Nov 2012)

Health Minister circumvents law to fulfill 2008 election bribe? (18 Dec 2012)

Johnny’s Report Card – National Standards Assessment – Compassion (9 Jan 2013)

“There’s always an issue of money but we can find money for the right projects” – John Key (20 Jan 2013)

Media

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)

Call for an Orphan drugs access policy to overcome Pharmac’s systems failure (28 Feb 2013)

Additional Information

Gregory John Coyle: How does the operation of PHARMAC’s ‘Community Exceptional Circumstances’ policy align with the distributive justice principles of fairness and equity?

The PNH Support Association of NZ

PNH Support: Petition to Government to Make Soliris available to New Zealanders!

Support for Jethro Morrow

Facebook: Support for Jethro Morrow Facebook Page

Facebook: NZ Rare Disease Day

Website: NZ Rare Disease Day

Facebook: Treat NZ Pompe Patients Now Facebook Page

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

Copyright (c)  Notice

All images are freely available to be used, with following provisos,

  • Use must be for non-commercial purposes.
  • At all times, images must be used only in context, and not to denigrate individuals.
  • Acknowledgement of source is requested.

.

.

= fs =