Opposition parties work together on “orphan drugs” (part wha)

Continued from: Opposition parties work together on “orphan drugs” (part toru)


NZORD - seminar - 1 August 2013 - Wellington - pompe disease - manual cover


NZ, Wellington, 1 August 2013 – A seminar in Wellington was held NZORD, the New Zealand Organisation for Rare Disorders , to discuss the problem of lack of funding for “orphan drugs”. People with rare diseases are missing out of medication – a life-threatening situation.

After a break for lunch, Wallace introduced the four members of Parliaments;


L-R: Barbara Stewart (NZ First), Kevin Hague (Green Party), Annette King (Labour) and Paul Hutchison (National) – Wallace Chapman (standing)


Associate Minister for Health, Paul Hutchson, took the podium first;

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Minister Hutchison began by acknowledging his Parliamentary colleagues, Wallace Chapman, and John Forman.

Of John, Hutchison said this,

“May I acknowledge John Forman and the Organisation for Rare Diseases for all the the work that you do, John. Absolutely committed,  enthusuiastic, and assiduous. So please may I express that appreciation…”

The Minister continued by saying that he was sorry he was not present earlier in the day to hear the previous speakers. He then launched into his speech,

“… The principle of Universality does not mean that the public should pay for every test, treatment, or medicine, that improves health no matter the price of how effective it is. You may be aware that a new concept has crept in called proportionate universality, universalism, which in other words, is targetting. And it’s something that appears to be, ah, almost superceding the principle of universalism.

In his press release relating to the ombudsman, John Forman says the Ombudsman noted the contestable legal argument about whether PHARMAC appropriately excludes social and ethical factors from their consideration, though he said it was not his role to make a definitive decision on that legal dispute’.”

Minister Hutchison “forgot” to mention also that the same Ombudsman,  David McGee, had been highly critical of   PHARMAC’s  policy that “supported the position that high and low cost medicines should  be examined by the same decision-making criteria, and found that whilst it was lawful, was not reasonable“.

If Minister Hutchison had attended the earlier speaker’s addresses, he might have remembered to add that salient point. He continued,

“And John Forman also said that it is time for PHARMAC to acknowledge that a strict economic focus without a moral compass is abandoning patients at the margins. We hope that this opinion will cause PHARMAC, government ministers,  the health select committee, and other officials to respond with serious scrutiny and review of PHARMAC’s policies regarding socialised medicine for rare diseases. I don’t consider for one moment that PHARMAC acts without a moral compass… but  nothing should be for granted.

And I do note that PHARMAC’s key objective is  to  secure for eligible people in need of pharmaceuticals the best health  outcome that are reasonably achieved from pharmaceutical treatment and from within the amount of  funding  provided. I also highly respect the members of the Pharmaceutical Advisory Therapeutic Committee who are all dedicated clinicians who have committed their expertise to  attempt fairness and equity guided by a scientific evidence basis.”

I doubt if this next bit went down well with the audience,

“I must say I well remember Sir William [Bill] Birch telling me some years ago that from every nook and cranny, town and hamlet in New Zealand, comes a perfectly legitimate reason to spend money. The whole skill is how to prioritise it.”

If Minister Hutchison was invoking the ghost of Bill Birch, known for his extremist monetarist views, then he had come to the wrong place. This was not a Chamber of Commerce or NZ Initiative (formerly the NZ Business Roundtable ) business lunch. He was addressing desperate people who were seeking answers and solutions to life-threatening diseases – not hearing that the purse-strings were being closed by an acolyte of a past Finance Minister.

The Minister continued,

“And I guess that’s the blance and the tension that we have. Where do you achieve equity and fairness in comparison to the resources that we have available. New Zealand does indeed now-a-days spend amongst the top of  OECD countries in terms of it’s overall health budget. Some of you may say  that the pharmaceutical budget in comparison to the whole $14.7 billion is less than it should be although of course that is arguable.

So what’s PHARMACs position? As you know, PHARMAC pointed out there have been several reviews of the question of New Zealand providing subsidised access to high cost medicines.  Firstly in 2006, and then of course the McCormick report in 2009. They explicitly recommended against a separate high cost medicines funding [board?] approach for New Zealand. The reason they gave for this were that the main rationale for such a fund is to improve health outcomes rather than because of the particular charachteristics of the medicines themselves are a fundamental importance. The Panel noted that the PHARMAC model is already based on the objective of improving health outcomes. The panel was not convinced that the approach used by other countries such as Australia was superior to the status quo.

Government responded to a number of that reports recommendations and that led of course to the establishment of the  Named Patient Pharmaceutical Assessment scheme, which  we’re now  currently running with.

I also note  that most of PHARMAC’s funding is already committed to high cost medicines.  The PHARMAC annual review shows that the top 20% of patients account for … 86% of expenditure. That’s 20% of patients accounting for 86% of expenditure. Which means a smaller patient group is obtaining a greater share of pharmaceutical expenditure than the majority.”

“That’s 20% of patients accounting for 86% of expenditure.’ – is an interesting statistic. Is it code for implying that that a small group receive a disparate amount of tax-payer funded support?

How does that statistic compare  to the 10% of top income-earning families earning 30% of the income?  (see: Household Economic Survey 2010) Or the wealthiest 10% of New Zealand families controlling/owning  approximately  50-60% of  New Zealand’s wealth?  (see:  New Zealand Institute’s The Wealth of a Nation 2004)

Minister Hutchison concluded his speech,

“…I think it’s also important to point out that since the NPPA has come into being, that we’ve gone from where there was the previous regime which was $2.1 million and now  to $8 million. Clearly it’s not enough.There will always be pressure on it.

The last thing I just wanted to mention was that there is going to be  future reviews and right now PHARMAC is keen to look at new ways of serving  New Zealanders. That’s why  they are currently conducting their  significant review on operating policies and procedures. First thing under review includes the criteria by which PHARMAC makes it’s decisions. This  is an important opportunity to define what best… health outcomes means in terms of it’s  legislative objective,  and in doing so to change the mix of treatments that are ultimately funded  within the budget that is made available.

As you’re aware PHARMAC is  meeting communities around New Zealand in a series of  eleven forums and here is a superb opportunity for everyone here. I would put in a submission, attend the forums, and express your views.”

Wallace asked the Minister for his views on  creating a separate Rare Diseases Funding Agency, with a budget of around $25 million. Wallace explained that many people in the room were “falling through the gap” and a RDFA could plug that gap.

The Minister’s response was less than helpful, and defaulted to a predictable excuse not to consider the option. He said,

“…The issue is always once you get separate funding streams, you get extra bureacracy, you get an extra pressure on that funding stream as well as the main Schedule. So that it may be that you find you  have to take away from the main Schedule and vice versa. It’s a very difficult dilmemma. I think that this latest round of opportunities to relook at  how  PHARMAC  is setting it’s basic criteria of improving health outcomes is an opportunity to explore it.”

It is unclear as to why the Minister actually turned up to the seminar. His speech offered nothing new except, perhaps, to announce the  upcoming PHARMAC reviews.

If National is going to spring a herceptin-style change in policy toward sufferers of rare disease, the Minister was less than clear in his speech.  To use the Minister’s own words, he had expressed the status quo as policy and nothing more.

The real surprise was to come from the next speaker, Labour MP for Rongotai, Annette King;




Ms King has served as Minister of Health in the previous Clark-led government and had over-seen the re-building of the health sector after the disastrous cuts to services and budgets in the late 1990s. Ms King put an end to user-pays within the public health system, implemented by the previous National government.

Ms King firstly acknowledged those with rare disorders who displayed “advocacy, tenacity, longevity, and your committment to fairness and equity in health.” Ms King added that, “I particularly want to thank John Forman, who has dedicated years to NZORD and if knighthoods actually went to the people who really deserved them, then John certainly would get one.

That suggestion  was received with  a loud round of applause.

Ms King continued,

“…I think the problem has really  reached a critical point because we have, as you heard from Dr Hutchison, there have been many reviews into this issue, going back to the 2007 New Zealand Medicines Strategy; the 2010 report on high cost, highly specialised medicines;  and as you know from that we still haven’t had this issue resolved for those who have very rare conditions. PHARMAC now, as we’ve already heard, have established what they call a new special pathway, their Named Patient Pharmaceutical Assessment, the NPPA, which follows the review of the exceptional circumstances. But by my reading of it, is that this policy runs counter to their current policy settings, because when you read it, they must take account of things like if a dollar is spent in one area, it is not available in another.

That they must work to obtain the greatest  benefit. That the best place  to invest  the next dollar, to achieve the best access to health. So these are the things that they have to take  account of, even in the NPPA policy. But at this point I do want to stress as John Forman has on a number of occassions, that I do support PHARMAC in their   role of getting the best possible deal for medicines of the bulk of  New Zealanders. I think they have done a fantastic  job over many years. In fact I think they’ve been a stand out organisation.

And the bottom line for Labour in  terms of  the Trans Pacific Partnership negotiations, the latest round of free trade aggreements,  is that PHARMAC continues to have the right to purchase our  pharmaceuticals  to get the best health outcomes from effective budget management.”

Then came the ‘crunch’ moment,

“…But I think there has to be, and we have to acknowledge, that what we have done in the past,  as you saw from the question that John just asked, there has to be a change in the way in the way we deal with orphan drugs.”

Ms King said that it was clear that the NPPA scheme was not working for people with rare disorders. PHARMAC was able to over-rule any recommendations to purchase drugs for patients with rare  disorders. Ms King then stated,

“It is time for us to separate the two issues…”

“In 2014 our election policy will have two main parts to it. First of all the establishment of an orphan drugs policy.That policy will include international information sharing and monitoring  of orphan drugs and sharing that information as others countries do, about the clinical viability and acceptability of those drugs.
The second, I believe, is very important, and that is the establishment  of a fund with it’s own Board. Now I don’t believe this has to be [as] highly bureacratric as Paul mentioned. I believe that you can set up a separate Agency within, for example the Ministry of Health,  to give it’s freedom, but it has it’s own Board. And it has it’s own fund to administer.”

“So one of the things that would need to happen soon after an election would be the establishment of on implementation working group, which could be made up of clinicians; of patients; of community representations, and others,  to put in place the details and work on the criteria for access. I do believe that in separating the funding and operation of the orphan drugs policy from PHARMAC. It will let them get on with doing what they do really well, and I think in some ways it will free them to get the best they can for the most of us who don’t need special medicines. But it will mean that for those who have rare disorders, that there will be a fund around that.’

Ms King said,

“We’d be looking at a fund between $20 to $25 million.”

Which is approximately what National spent on the Rugby World Cup in 2010 – $26 million of taxpayer’s money, on funding the tournament’s deficit. [Update: And on 8 August it was announced hat National would be giving a $30 million taxpayer’s subsidy to Tiwai Aluminium Smelter.)

“…That would be the way that we’d go in New Zealand, in line with other countries, including our closest neighbour Australia, who have managed a separate orphan drugs policy, for many, many years. And the advantage I suppose  from here is that we can learn from the mistakes from others, look at ways we can get the best value from such an agency.”

Ms King concluded that she believed this was a policy that other parties from the Opposition would support this new policy.

The audience responded enthusiastically to Annette’s announcement.

Wallace welcomed the Labour Party policy, and referring to  a Labour-Green-NZ first coalition, asked Barbara Stewart, “actually, which way will Winston go, Barbara?”

She smiled coyley, responding “we’ll just have to wait and see“.

That elicited  a mix of laughter and “awwwww” from the audience.

Next up, Wallace introduced Kevin Hague, from the Green Party,




Kevin began with,

“Congratulations to Jenny [Jenny Noble – one of the seminar organisers] and to  “Sir John”… [laughter]

… And acknowledging my Parliamentary colleagues. Could I give a special acknowledgement to Paul Hutchison who’s gone now of course. National wasn’t going to have someone here. But Paul decided that that wasn’t ok, so he came along at short notice. So I didn’t agree with anything he said, but it was really great to have Paul here.”

Kevin expressed his regret at not attending the morning part of the seminar,

“Can I give you an apology for having missed this morning’s programme, as I thought it was a really exciting-looking programme. I intended to be here  for the entire time but I had to sit on the Select  Committee for the Pike River  Implementation Bill…”

Kevin continued,

“…My starting point actually is the right to life. Because that basic human right, it’s pretty universally acknowledged, seems to have embedded in it, the right to health.”

Kevin referred to the UN human rights treaties discussed earlier in the day. He said that for the right to life to be meaningful, it had to include the right to health. He acknowledged the high cost of medical treatments and the need to ration  those dollars. He said he “unashamedly” used the word “ration”.

“Governments decide whose needs will be met and whose will not be met.”

Kevin referred to “utilitarianism; the need to stretch health dollars for the greatest gain for the greatest number. It is only PHARMAC that tries to do that – the rest of the [public] health sector does not use this system.

While Kevin did not disagree with the concept of utilitarianism, he said that those whose health needs are furthest away, from the right to health,  will tend to be  those whose health needs are not met.

“And I don’t believe that that can be an acceptable consequence,” he added.

“So for that reason , we believe that the New Zealand health system needs to be able to have a second approach… Our approach is very congruent indeed with that you just heard outlined by Labour. I think  it’s very exciting indeed that Labour and ourselves have that same approach…”

Kevin said that whilst he believed that some of PHARMAC’s criteria for cost-benefits could be amended to take other criteria into consideration – such as participating in the workforce –  that he did not believe that the Agency should be bound by the “right to life” argument. Kevin preferred keeping PHARMAC’s “structures” as simple as possible, and keeping it’s cost-utility as straight forward as possible.

He would not “load” PHARMAC with the responsibility of resolving the orphan drugs and rare disorders  issues.

Kevin spoke to the PHARMAC representative in the audience and said,

“I would say just keep doing what you’re doing now, Stefan.”

Kevin then added,

“But. We are going to create another fund, which is specifically to be used on this right-to-health basis. I have no problem with  the cost effectiveness being one of the  criteria that is  used on the fund, but it’s  only one of a range of criteria. And I have no problem with PHARMAC’s people doing the analysis, but it can’t be PHARMAC that makes the decisions and I favour an independent Board very much as Annette outlined under Labour’s policy.”

Kevin said that even under two  systems there would still be inequities as there would always be a mis-match between dollars available and the need it has to try to cover. He said no system could be perfect in this regard.

“But using the two approaches actually reduces the size of that inequity, and that has to be a good thing.”

Kevin said “a great injustice has been committed” and the Greens would work to end that injustice.

Again, the audience responded with enthusiasm, obviously welcoming the Green position on the issue.

Wallace then introduced the last political speaker, New Zealand First’s spokesperson on Health, Barbara Stewart;




Barbara began with a greeting and an apology for not being present for the first part of the seminar. She explained that the House was sitting under Urgency and extended hours. She congratulated John Forman for the “wonderful job he has been doing over the many years”, and thanked him for continuing to keep NZ First appraised of the issues surrounding rare disorders and orphan drugs. Without further preamble, Barbara launched into her policy speech. She got straight to the point;

“In NZ First, we believe that as a First World country, we should be able to afford access for orphan drugs. There is an underlying right to health  care. We are very aware that sufferers of rare diseases deserve fair treatment when it comes to access to orphan drugs. So we’ve been very pleased to hear what  Annette has announced. And I know that  Kevin thankfully  supports it, and I know that  we would in New Zealand First as well.

The last thing that want  to see is  people keeping on falling throught the gaps. The status quo needs changing. There is nothing surer than that. Particularly for orphan drugs. New Zealand was once thought of as one of  the highest  for the quality of  healthcare in the OECD. And it’s interesting to note that this ranking is slowly dropping away.”

Barbara said that many other countries ahead us on the OECD scale did indeed supply medicines for rare diseases. She said that NZ First has looked at the Australian model and “it appears to be successful“.

Barbara said,

“Here we would support Annette King with her model that she is proposing.” She added, “we don’t want to see New Zealand behind the rest of the world”.

Barbara acknowledged that PHARMAC has done a good job over the years, but that it was time “for a review”.

“We’re disappointed to see that the government, through PHARMAC , seems to be taking a relatively hard-line approach on medical funding and we know that this is putting people’s lives at risk… This is an issue that does need to be resolved.”

“So, we believe that equity and fairness is essential and whatever we can do to ensure that sufferers of rare diseases… can have access to the best treatment, we will do.”

Barbara concluded her speech with those words and Wallace thanked her.

NZORD director, John Forman then read out a statrement from the Maori Party. In it, they apologised for not being able to attend. Reading from the paper, he said,

“The Maori Party promotes the idea of a separate policy process for managing New Zealand’s supply of orphan drugs for rare disorders. We have a particular interest in  orphan drugs access policy through our support of people living with Pompe Disease, a  serious muscle wasting disease, that without treatment will result in respiratory and cardiac  failure. We understand the exceptional circumstances approach towards supporting applications for access to specialised and expensive medicines, such as enzyme replacement therapy, has yielded adverse impacts on too many individuals. And we cannot support any policy effect which results in government picking winners and losers.”

The Maori Party statement went on to state that there was an impact on  those suffering rare diseases by the inequitable decisions of this government. “There is a profound injustice at play”  that some families were impacted simply because of the rarity  of certain diseases and the consideration of appropriate treatment. The statement concluded by acknowledging the work done by organisations such as Muscular Dystrophy, the Lysosomal Diseases New Zealand, and New Zealand Organisation for Rare Disorders.

Wallace then opened the floor for questions.

In answer to a question as to when the Parties present would implement a separate Funding Agency, Kevin Hague replied, “in the first hundred days“.

Annette agreed with Kevin that it would be done “as soon as possible“. She gave a “solid committment that this would happen“.

This blogger then asked Barbara Stewart a question relating to her Party’s committment to a separate Funding Agency for orphan drugs. I confirmed that her Party would support a separate Funding model for orphan drugs, and she replied,

“We would support that, yes.”

I asked my follow-up question,

“…Here’s the problem. Is that, it’s  fine for you to sit there, saying you support it… but if your leader decides to go with National, it’s not going to happen, is it?”

To which Barbara replied,

“Oh, we have to wait until after the election before we can actually say anything at this point in time.”

Wallace suggested that Barbara txt-message Winston now to find out. She declined, and added,

“No, we do always say that will wait until the voters say what they’re going to say and then we work it through from there.”

The seminar continued with more questions and answers from the audience, including representatives from pharmaceutical companies and PHARMAC.

A talk was presented by Daniel Webby on his very personal experiences with living with a rare disorder.

John Forman presented his speech on issues and problems surrounding rare disorders and orphan drugs. His slide presentation finished with this image;






A sobering conclusion to John’s speech, I thought.

My own conclusion from listening to the representatives from Labour, The Greens, NZ First, and the Maori Party, is that all profess to support a separate funding agency for orphan drugs.

But only Labour and the Greens can be counted on  to carry out their pledge.

New Zealand First states that it supports a separate Funding Model – but without knowing which way Winston Peters will move post-2014, then his Party’s policies must be viewed with uncertainty.

The Maori Party is in an even more untenable position on this issue.  Traditionally, they have viewed Labour with disdain, and instead chosen to coalesce with National. Unless the Maori Party makes a separate funding model for orphan drugs a bottom-line negotiating point – then their policy-pledge will go nowhere.

New Zealanders living with rare disorders, desperately seeking life-giving treatment, are experiencing stress, anxiety, fear, and an unnecessary interuption to their lives – on top of the effects of their disorders.

Yet, they have come far from their early days when they first approached PHARMAC for assistance, and were constantly knocked back. Those were dark days for people like John, Freda, Allyson, Daniel, Jenny, and many others.

But after this seminar, they found recognition for their efforts; understanding for their plight; and something else to bolster their spirits…

They found renewed hope.




Copyright (c)  Notice

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

* Use must be for non-commercial purposes.
* Where purpose of  use is  commercial, a donation to NZ Organisation for Rare Disorders is requested.
* At all times, images must be used only in context, and not to denigrate individuals or groups.
* Acknowledgement of source is requested.

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)

“One should judge a society by how it looks after the sick and vulnerable” – part tahi (4 March 2013)

“One should judge a society by how it looks after the sick and vulnerable” – part rua (4 March 2013)

“One should judge a society by how it looks after the sick and vulnerable” – part toru (4 March 2013)



UN Special Rapporteur on Health



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