type-1-diabetes

Getting Medical Interventions Approved By Government

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Having played for a few years at the nexus of academia, big pharma, and government, it is interesting to watch how these three bodies orbit each other and interact (for those of you familiar with the Three-Body Problem you know such a system is often unstable, but I digress). It is often frustrating, being a person with type 1 diabetes, knowing there are, for example, medications out there with overwhelming evidence behind them but which are not funded by government national health programs. GLP-1s are a great example. These are well established to provide benefit to people with type 1 diabetes but are still, certainly in Australia, not covered and financially out of reach for those who would benefit.

So, what does it take to get something approved for government subsidy? I see four major factors which are necessary, and I thought I would write this article to describe them. For context, I will use the subsidy of continuous glucose monitors (CGMs) for people with type 1 diabetes (T1Ds) in Australia as a case example.

The ‘Ask’

The first step is a clearly articulated ask: “We would like the government to fund/subsidise <x>”. In the case of CGMs for T1Ds, the ask is “We would like the government to fund/subsidise CGMs for T1Ds”. In reality, the ask for was for a specific subset, and the program was expanded to all T1Ds over time (for reasons explained later), but I will keep things simple to illustrate the point.

It is, at this stage, where we can answer why GLP-1s are not available for T1Ds in Australia. The fact is, in the case of Ozempic, Novo Nordisk has not asked because they have no need to. Demand is outstripping supply such that Novo can set the price as they wish and run their factories at full speed. Asking will create a new audience which they cannot serve and force a negotiation on price. There is no motivation to do this until general demand abates or supply can be scaled up.

The Medical Benefit

The ‘Ask’ needs to be justified by a tangible benefit which can encompass quality of life measures and overall health measures. In the case of CGMs for T1D, subsidy would allow more people to access the technology and significantly reduce the need for finger pricking. Near-real-time tracking of glucose levels would reduce the risk of serious hypoglycaemia and hospitalisation. All good things and a great improvement for the lives of people living with type 1 diabetes.

The Medical Benefit provides the “So What?” element to the request and can be very emotive. A piece of wisdom often used in sales is people make decisions on how they feel about the purchase and justify it with logic after the fact. The Medical Benefit is the door to stirring the heart of government to act and, in turn, makes for an emotive tale for voters for justify the spend and show the government cares.

The Evidence

A good tale still needs evidence. Aristotle’s ‘Art of Rhetoric’ speaks of three key elements necessary to persuade:

  • Ethos: The argument should be from a credible body
  • Pathos: The argument should stir the emotions of the receiver
  • Logos: The argument should be based in reason

I have taken it as given that, if a request is being made, for it to get the ear of the government, it will need to be from a credible source (Ethos). The Medical Benefit is the key to providing Pathos and clinical/world evidence is the key to Logos. Without evidence, it is a stirring tale but an unjustified one. A government needs to back decisions on reason otherwise the opposition will exploit the weakness.

In the case of CGMs for T1Ds, there was a wealth of clinical evidence showing improved health outcomes with the use of CGMs. I gave an example of this back in 2021 in the context of PWD pregnancy and the use of CGMs.

The Economic Benefit

Governments are required to be fiscally responsible because of scarce resources. In short, if the government is to spend a dollar, it needs to do so on whatever yields the most benefit. For medical interventions, economic justification is usually measured in cost per QALY (Quality-Adjusted Life Years). Basically, a threshold needs to be met where the cost generates a minimum level of benefit which, in this case is a longer and better quality of life for the individual.

The threshold varies from country to country and, while there is no official limit in Australia, a general rule of thumb is government will consider a medical intervention which costs less than $50,000 per quality adjusted life year.

In the case of CGMs for T1D, the economic numbers came out at around $30-35k per QALY and the request was approved.

Case Example: Automatic Insulin Delivery for T1Ds

Let us consider a couple of items being discussed in the diabetes community at this time. The first is equitable access to insulin pumps and, by extension, looping (Automatic Insulin Delivery aka AID) for all Australians. In Australia, while CGMs are subsidised by the government for T1Ds, pumps are not and need to either be purchased directly or obtained through private health insurance. Given a pump costs literally thousands of dollars this puts it out of the reach of many Australians and I recently helped co-author a petition to help get this changed which I encourage you to sign (QR code below).

So, based on the four elements mentioned above, how would we expect the request to fare if raised by a credible body to government as part of, say, the Inquiry Into Diabetes?

The ‘Ask’: This is relatively straightforward i.e. “AID systems should be subsidized for T1Ds”

The Benefit: Lots of qualitative and quantitative benefits to AID systems such as better overnight control, improved HbA1c and Time in Range, and less manual intervention by the T1D.

The Evidence: Lots of clinical and real-world evidence to support this from companies such as Medtronic.

The Economic Benefit: While it would be great to subsidise AID systems for all T1Ds, to get the appropriate cost per QALY, it may be necessary to pick a sub-group e.g. children (who consistently have higher HbA1cs than their older counterparts in trials), T1Ds who are pregnant and so on. Generally, the cohort selection will be driven by The Evidence as this informs the economic modelling. In other words, if there are no studies of AID systems helping pregnant T1Ds, it will be harder to economically justify their inclusion.

Case Example: CGMs for People with Type 2 Diabetes (T2Ds)

On the back of the benefits seen in T1Ds with the use of CGMs it makes sense to expand the program to T2Ds.

The ‘Ask’: Again, this is relatively straightforward: “CGMs should be subsidised for T2Ds”

The Benefit: T2Ds being able to see how food affects them will inform eating habits and improve health outcomes.

The Evidence: There is some evidence of benefit for insulin dependent T2Ds but the body of evidence for general benefit is still being gathered with limited long-term studies to justify The ‘Ask’. To quote this last paper, literally written this year, “…few studies reported on important clinical outcomes, such as adverse events, emergency department use, or hospitalization. Longer term studies are needed to determine if the short-term improvements in glucose control leads to improvements in clinically important outcomes”

The Economic Benefit: Without evidence to act as a foundation for the modelling, it is harder (not impossible) to determine a cost per QALY. The best hope would be to exclusively focus on where there is evidence (insulin-dependent T2Ds) with the hope that, as more evidence is acquired, the program can be expanded to the wider T2D population.

Conclusions

It is easy for us to be frustrated with the glacial movement of governments to get behind the latest advances in technology and medicine when it is clear, for those of us at the coalface, there are benefits to many, many people. However, as we can see, with competing priorities, it is important government spending is used as effectively as possible.

The inclusion of medical interventions in national health programs requires collaboration of all three bodies (big pharma to provide the intervention, academia to show it works, and government to provide the funding) and the will by all of them to drive it. Without this willingness and the elements mentioned above it is hard, if not impossible, to achieve success.

Where to Find Australian Clinical Trials

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Ever since being diagnosed seven years ago, I have been keen to participate in trials of new treatments. Unfortunately, there are not a lot of trials for LADAs (at least, not in Australia). When I became insulin dependent, a couple of years ago, I thought things would change but either my age (I am 50) or my HbA1c excludes me (studies generally like higher HbA1cs and mine is too low). I do check for new studies every month or so and thought putting a list together of where people can go may be useful.

Diabetes Australia’s Research Opportunities

Current research opportunities | Diabetes Australia has a great list of research studies for all types across all of Australia

Disclosure: I am on the Diabetes Australia Community Advisory Council

JDRF’s Clinical Trials Archive

Clinical Trials Archive – JDRF lists trials specifically for type 1 diabetes all across Australia

Australia/New Zealand Clinical Trials Registry

ANZCTR search results | Australian Clinical Trials is an Australian Federal Government registry for many health conditions with filters to show only ones relating to diabetes. Despite the title it seemed to only show trials in Australia at the time of writing.

Baker Heart and Diabetes Institute Clinical Research Trials

Clinical research trials (baker.edu.au) covers all types of diabetes (or, at least, types 1 and 2). The Baker is based in Melbourne but my understanding is some of their trials do recruit outside of Melbourne.

Disclosure: I am a voluntary clinical affiliate with The Baker (basically I am on the books if they need Lived Experience input on a trial) and an Associate Investigator on their FAME-1 Eye trial.

Australasian Type 1 Diabetes Immunotherapy Collective (ATIC) Clinical Trials

Clinical Trials | ATIC (svi.edu.au) are studies focused on immune system mediation which means they generally relate to type 1 diabetes. Arguably this is the frontier of type 1 diabetes treatment and has recently seen exciting results with medications such as Teplizumab for delaying the onset of symptomatic type 1 diabetes. Recruitment is across Australia and, at the time of writing, there was even a study recruiting in New Zealand.

Disclosure: I am on the ATIC Community Engagement Panel.

Living Without Air

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Imagine a bomb which kills by removing air with its blast. Armor would be useless against it and civilians would have no defense. The only way to survive would be to have a personal supply of oxygen to breathe until air returns to the area. Such a bomb does exist; it is called a thermobaric bomb and it has been used in warfare. Thankfully, it is seen as something exclusively for the battlefield and, while not banned, its use on civilian populations is seen as possibly contravening the Protocol on Incendiary Weapons.

This article is not a treatise on the nastier weapons at the disposal of modern armies but asks the question “Should a civilian population be given what they need to survive in times of war?” If we think of the basics a person needs to survive, we can imagine this includes food, water, and shelter but what about medication? There are diseases, such as type 1 diabetes, where the absence of medication (insulin) literally means death. There is no remission for type 1 diabetes, there is no substitute for insulin. A person with type 1 diabetes needs insulin as much as they need air, food, water, and shelter.

So, do the international conventions go as far as provisioning medication for those who literally cannot live without it?

International Declarations, Covenants, and Conventions

The Universal Declaration of Human Rights thinks so with Articles such as:

  • Article 5: No one shall be subjected to torture or to cruel, inhuman or degrading treatment or punishment.
  • Article 12.1: Everyone has the right to a standard of living adequate for the health and well-being of himself and of his family, including food, clothing, housing and medical care and necessary social services, and the right to security in the event of unemployment, sickness, disability, widowhood, old age or other lack of livelihood in circumstances beyond his control.

The International Covenant on Civil and Political Rights states:

  • Article 6.1: Every human being has the inherent right to life. This right shall be protected by law. No one shall be arbitrarily deprived of his life.
  • Article 7: No one shall be subjected to torture or to cruel, inhuman or degrading treatment or punishment. In particular, no one shall be subjected without his free consent to medical or scientific experimentation.

Geneva Conventions Act 1957 – Schedule 1

  • Article 3.1: Persons taking no active part in the hostilities, including members of armed forces who have laid down their arms and those placed hors de combat by sickness, wounds, detention, or any other cause, shall in all circumstances be treated humanely, without any adverse distinction founded on race, colour, religion or faith, sex, birth or wealth, or any other similar criteria.
  • Article 3.2: The wounded and sick shall be collected and cared for.

Geneva Convention IV 1949

  • Article 56: To the fullest extent of the means available to it, the Occupying Power has the duty of ensuring and maintaining, with the cooperation of national and local authorities, the medical and hospital establishments and services, public health and hygiene in the occupied territory, with particular reference to the adoption and application of the prophylactic and preventive measures necessary to combat the spread of contagious diseases and epidemics. Medical personnel of all categories shall be allowed to carry out their duties.

International Covenant on Economic, Social and Cultural Rights

  • Article 12.1: The States Parties to the present Covenant recognize the right of everyone to the enjoyment of the highest attainable standard of physical and mental health.
  • Article 12.2: The steps to be taken by the States Parties to the present Covenant to achieve the full realization of this right shall include those necessary for…The creation of conditions which would assure to all medical service and medical attention in the event of sickness.

Convention on the Rights of the Child

  • Article 6.1: States Parties recognize that every child has the inherent right to life.
  • Article 6.2: States Parties shall ensure to the maximum extent possible the survival and development of the child.
  • Article 24.1: States Parties recognize the right of the child to the enjoyment of the highest attainable standard of health and to facilities for the treatment of illness and rehabilitation of health. States Parties shall strive to ensure that no child is deprived of his or her right of access to such health care services.
  • Article 24.2: States Parties shall pursue full implementation of this right and, in particular, shall take appropriate measures:
    • (a) To diminish infant and child mortality;
    • (b) To ensure the provision of necessary medical assistance and health care to all children with emphasis on the development of primary health care;
  • Article 27.1: States Parties recognize the right of every child to a standard of living adequate for the child’s physical, mental, spiritual, moral and social development.
  • Article 37: States Parties shall ensure that… No child shall be subjected to torture or other cruel, inhuman or degrading treatment or punishment.

Conclusions and Call to Action

I have included a couple of provisions regarding torture because this is a fitting description for what a person with type 1 diabetes goes through if they do not have insulin. Without insulin, the body breaks down and floods the blood with fuels such as glucose, fatty acids, and ketones. The blood turns acidic and the organs of the body are slowly destroyed. Any ‘state party’ engaged in war and knowingly restricting the movement of insulin is, in my opinion, committing an act of torture. I appreciate others may see it differently.

Obviously, wars make life difficult for those who cannot escape. Inevitably, civilians get caught in the crossfire literally and figuratively. However, if those engaging in war are signatories to the documents above or endorse their principles, it is reasonable to expect them to stand by their commitments. The movement of humanitarian aid and medication should not be weaponized by being withheld, confiscated, or destroyed. To do so is to lose sight of the values often being fought for in the first place.

To this end, myself, and a collection of international diabetes advocates have put together a petition which you can sign urging warring nations to meet their international obligations. While the catalyst for the creation of the petition was the Russia-Ukraine conflict and the Israel-Gaza conflict, this petition is not directed at any one nation and simply demands that if a nation is to be recognized as an international citizen, it must act as one. I urge you to endorse this position so that the international bodies that our group represent, or have ties to, can put pressure on the relevant parties and save the lives of people who will otherwise die.