Home > Social Issues, The Body Politic > Opposition parties work together on “orphan drugs” (part rua)

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


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


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.

Following on from Kris Gledhill, host Wallace Chapman – of Prime TV’s ‘Backbenchers‘ fame, introduced the next speaker; Matthew Smith;




Matthew is a barrister at Wellington’s Thorndon Chambers, and practices in the area of civil and commercial litigation. His focus is on public laws and judicial reviews – something of particular relevance to NZORD’s members and supporters.

Matthew presented an overview of the Pharmaceutical Management Agency (PHARMAC) with explanations as to it’s guiding legislation; policies; and obligations.  His primary speech notes can be read here, and are worthwhile  accessing, if only to gain a deeper understanding how the Agency works (the speech notes are brief, only 5 pages, double-spaced typing).

He began with a “starting point” of  public law and the consideration for the judicial oath of office where anyone who becomes a judge of a Court “must do right for all manner of people“. He said this was relevant because that it was part of the decision-making process, and would be relevant in terms of international human rights obligations , domestic human rights obligations, and at an individual level for any funding decisions that PHARMAC makes.

General principles of law were also relevant to all  public-sector decision-making, “and that applies as much to PHARMAC as it does to any other  body“.

Matthew wanted to draw attention to  three main principles;

Firstly, that decisions have to be individual-specific and case-specific; that PHARMAC has to be consistent in decision-making, treating similar patients similarly, as well as recognising that there are points of difference that meant the dis-similar should be treated dis-similarly. Which was relevant, he said, to cost assessments in the context of PHARMAC’s funding decisions.

The third point was the human rights consideration and Matthew referred to Kris’s in-depth analysis of this point. He  confirmed Kris’s comments by stating,

“The relevance of human rights considerations is in least two dimensions in PHARMAC’s decision making. One is in the development of  any general policy  which applies and governs or informs the decision making process. And two, is in the individual level, the application of individual applications by individual applicants for funding, and their relevant human rights and how those rights impact upon the  decision that PHARMAC has to make.”

Matthew said that PHARMAC’s functions are set out in section 48 of the NZ Public Health and Disability Act, which firstly tasks the Agency with maintaining a Pharmaceutical Schedule, and the second to focus on the circumstances in which PHARMAC will make individual exceptions to the Schedule  with additional funding and grants.

PHARMAC’s policy to determine individual applications is governed by  their  Named Patient Pharmaceutical Assessment (NPPA) Policy. There are nine criteria by which they make their decisions. Whilst none of the nine criteria specifically referred to human rights considerations, Matthew pointed out that the ninth criteria referred to “other considerations“.

Matthew wasn’t certain if Pharmac considered human rights factors as part of  “other considerations“, but he said that they should under “human rights obligations“. He said that the general statute under which PHARMAC operated does mandate consideration for human rights factors.

Interestingly, Matthew pointed out that PHARMAC’s,

“…budget is notional, because PHARMAC itself does not pay the subsidies for pharmaceuticals. They are paid by the Ministry of Health, on behalf on the DHBs.”

Matthew repeated that general and broader law required PHARMAC’s decision-making for individual’s making applications,  to consider an  individual’s circumstances, and of the patients who would be beneficiaries of the drugs to be funded. He added that a practical consequence of that criteria was that PHARMAC could not use the price of a medicine as a reason to decline an application.

Matthew also pointed out that in analysing the cost of a particular drug, that cost had to be offset against any other costs otherwise spent by the health system for providing a service that otherwise would not be provided by PHARMAC. Costs, he said, had to be considered in a fairer, broader, more holistic way, taking into account offsetting costs, indirect as well as direct.

In terms of consistency, Matthew said that general consistency of treatment was identified as a principle of law and treating “like with like” flowed from principles of equity and equitable considerations. He added that often it was over-looked that consistency also meant identifying those who were in a different situation  and treating them differently. He used an example of  a population group with disimilarities to the rest of  the population, and that those disimilarities should be taken into account.

Taking differences into consideration maintained consistency. That had to be reflected in processes, as well as in end-decisions.

In answer to a question from the audience, which asked why applications from rare disease patients were still being turned down, Matthew replied, that the Act allowed for cost as one of three considerations.  He accepted that cost was relevant to PHARMAC.  Two other considerations were clinical needs and determinations, and health needs – the latter not defined in the Health Act.

He suggested that too much focus was currently being placed on cost, cost-basis,  and economic analysis, and that we had lost sight of the fact that we are dealing with people and individuals first and foremost. Mathew said,

“…As people born into the Human Family, so to speak, which is the starting point of all human rights obligations… those are being lost sight of, and those aren’t being given sufficient weight in the context of individual decisions,  and circumstances where, as I understand it, PHARMAC has decided that there is no specific earmarking in terms of the last year the $770 odd-million that was allocated for funding. There’s no specific earmarking for exceptional circumstances or rare diseases…”

Without specific “earmarking” of funds, it seems that those with rare disorders were destined to be sidelined by PHARMAC.

But, there was to be a glimmer of hope later.

Wallace then introduced Andrew Moore, associate professor of Philosophy at Otago University. His field and interest was in  ethics as they related to public policy. He has advised four Health ministers, from Labour’s Annette King to National’s Tony Ryall. He was a founding member and chairperson of the National Ethics Advisory Committee.

His advice has contributed to the national health policy, resource allocation, prioritisations, as well as contracting to PHARMAC;




Andrew started with paying tribute to NZORD and it’s director, for an “ongoing resilient committment”  and respect for  the democratic process, to work toward their goals.

Andrew then defined his concept of ethics – which he tied to the seminar issues,

“I have a fairly basic idea about ethics. Ethics, according to me, is just to do with what matters, and how to live in the light of that.”

Andrew added, “that  the topic here is what matters in the allocation of public funds for medicines, devices, and the like“.

He said that what matters in this context is the people who need these treatments and who can benefit from them. Andrew acknowledged PHARMAC’s statutory role was  to deliver best health outcomes for available  funding. The process was driven by the idea of maximising the public health benefit, and because of  budgetary constraints, there was priority for the greatest  health benefit per dollar.

That meant others missing out and not gaining any public funds, or opting for private support such as friends; reliant on corporate largesse;  missing out altogether,

“Or whatever survival means are available.”

Andrew suggested “pushing at the margins” to achieve ends. He suggested pushing for the ideas of “severity of condition” or “severity of circumstances“; or lifetime disadvantage.

He said the the current “maximised benefits” idea was simply not enough to deliver outcomes for those who needed it. He preferred promotiong the idea of “need” and “severity of condition” as a means to focus on.  Andrew suggested keeping things as simple as possible. For some in PHARMAC, ethics was “too complicated”.

He referred to the UK’s citizen  jury process to arrive at good outcomes and ideas.

Any solution had to be “need weighted” benefit, especially for those in dire danger from disorders.

Despite some fearing the possibility of getting into an “ethical view” on this issue, Andrew reminded the audience that even the current system was built on an ethical view, even though it was a somewhat narrow view. It was impossible to avoid ethics in favour of the status quo, said Andrew,

“You can only choose some views over others. There was no hiding place in the status quo”.

Following on from Andrew Moore, Wallace introduced Dr Andrew Veale, a Respiratory and Sleep specialist and Clinical Director for a private Lung Function and Sleep laboratory at the NZ Respiratory and Sleep Institute in Auckland. Dr Veale is also a sleep specialist at Middlemore Hospital, and has diagnosed and treated Acid Maltase deficient (Pompe Disease) patients. He is deeply interested in clinical trials and physiological measurement;




Dr Veale began,

“I’m involved because I fortuitously diagnosed a few patients with Acid Maltase deficiency, or Pompe Disease, and  they’ve allowed me to walk through their lives. In Freda’s case, for twenty years, as they cope with this disorder, which has had no cure. So it’s been an education for me and I hope of some benefit to them. But most of the time we just talk shop. Social things.”



Freda (L) and Jenny (R)


So began Dr Veale’s talk.

With the aid of coloured slides, Dr Veale launched into a full scale medical explanation of the causes of Acid Maltase deficiency, or Pompe Disease. He showed cross-sections of the human cell, and explained the simple deficiency that has wreaked so much harm and tragedy in people’s lives.

He began by stating that Enzyme Replacement Therapy (to treat Acid Maltase deficiency) was different from normal administration of drugs, as the Therapy used a much larger molecule. (Thank god for Fifth and Sixth Form General Science and Biology classes.)  Whilst normal drugs permeated through the whole body, he said, enzyme replacement had to be targetted to enter cells.

Dr Veale’s explanation of the complex processes was simplified for ease of comprehension. In fact, it was probably easier to take in than some of the legal matters that had been presented earlier.

After the science lesson preamble, Dr Veale added another complication into the mix; how to test treatments for rare diseases when the numbers of  patients are so small. As he put it,

“Patients with rare diseases will never have a randomised, double blind,  cross-over, trial while facing East…” (laughter) “These  patients are treated with observational treatments… which are not as good.”

He said there was a problem with randomised double-blind trials in that they disguised a sub-group who might benefit from the wider group.  Dr Veale added that Acid Maltase deficiency  involved trials that  lasted over years and reports could not expected any time soon. So there were inherent difficulties with the model of clinical trials.

Another problem, he said was the variability of tests. Dr Veale said test results depended on the precision of  test instruments. Test results, he said, also depended on how  a test subject was feeling on a given day and what kind of activities they had engaged in. He used Freda as an example saying that she might well fine on a Monday – but knackered on a Friday because she had been doing gardening that  morning. These were all factors that affected outcomes.

Dr Veale presented a slide with four curves to illustrate his point. He said the graphic was a “fantastic” example of the point he was getting across;


The  sharp, pointy [red coloured] curve, he said offered a test outcome with a very good precision.  It’s got “tight confidence“; “we can trust it“; “it’s the truth“.

The green curve gaves the same result, but was less precise; “the scatter was wider“. “And it might be the truth“.

Dr Veale then used another chart to show how individual data points, from the same patient, would give differing results. One set of data points showed the patient improving – another set of data points other showed the patient deteriorating. But  it was the same patient and from the same set of data. But overall, it showed a more accurate picture of the state of the patient, “this is some real data”, Dr Veale said.

I was reminded of political polling. Individual polls could give a misleading result for political parties – but an overall picture presented a much more accurate result. (See: Polling Chart on The Dim Post blog)

Dr Veale said that with slow-changing diseases, the temptation was to  test infrequently because not much had changed. As a result, there would be misleading test results from data gained from infrequent  testing. Fewer data points would present an incomplete or misleading picture of the disease’s progression. The infrequency of measurement coupled to an imprecise test would yield poor results. The slower a  disease progresses, he said, the more data points were needed to create a more truthful picture. The same applied to an imprecise test – more measuring was required.

For example, Dr Veale sugggested a year’s worth of testing at one-weekly intervals. And then he would want a further year’s worth of follow-up testing of one-weekly measurements to detect any changes in previous data. That, he said, was using the patient as their own ‘control’, as  the best method of showing a subset of beneficiaries.

Dr Veale presented the final slide in his summation,


Dr Veale made the strong point of having a separate  trial fund for experimental treatments for individuals  patients, with over-sight  by an independent Advisory Board. The purpose was to ensure outcome measures were  important to the individual patient concerned.

He said,

“I think we’ve got to get it right. These are very expensive drugs and it’s all very well to say  ‘well we shouldn’t worry about that’, but there is a [muffled] cost if we don’t do it correctly. I think there is a way forward here. When you make a decision to treat somebody with these sorts of disorders,  you’re not making a decision to spend $100,000 a year, you’re making a decision to spend five million over a life.

And I think there is an obligation on us to show that it’s of use.”

To be continued at:  Opposition parties work together on “orphan drugs” (part toru)




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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