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‘Yeah, it kind of just dissolved’, said the 49-year-old woman with metastatic melanoma for which all conventional therapies had previously failed.

She received an experimental combination of two novel cancer drugs (Ipilimumab and Nivolumab) at the Memorial Sloan Kettering Cancer Centre (New York). Oncologists watched her bulky tumour mass disappear within three weeks after a single dose.

The year was 2015, almost a decade after the Nobel prize-winning discovery that a specific antibody could block the pathway which prevented immune cells in the body from killing tumour cells. This so-called ‘immune checkpoint inhibition’ (ICI) antibody fostered a new class of anti-cancer drugs.

The director of scientific affairs at the Cancer Research Centre (USA) declared in 2017, ‘… With the power of the immune system at our disposal, no cancer is beyond reach.’

Meanwhile, an oncologist explained to Danielle T., a young woman with a rare neuroendocrine carcinoma sitting before her. ‘The great thing about these ICI drugs is that they should work for all cancers, unlike your regular chemotherapies each of which is usually specific to one, or a few, tumour types.

‘But there is a catch’, she continued. ‘Except for a few commoner cancers, the ICI drugs aren’t available on the Pharmaceutical Benefits Scheme (PBS). Certainly not for rare and less common cancers. You will have to pay the full price demanded by the pharmaceutical company. And they aren’t cheap.’

Pharmaceutical companies maintain they must charge a high price to recoup drug development costs and to foot the bill for expensive Phase-III clinical trials. Not to mention the outlay of making costly submissions to the government’s pharmaceutical benefits advisory committee (PBAC). The government maintains that it cannot subsidise a drug for a particular indication without rock-solid efficacy data derived from hundreds or even thousands of patients. Those numbers for rare and less common (RLC) cancers cannot be achieved. And no, different types of RLCs cannot be lumped together for a trial; the rules didn’t allow for that. Furthermore, the government stresses it has a limited budget to be distributed equably across all aspects of health care for all Australians.

Log-jam. And it is those with RLC cancers that suffer.

There are hundreds of RLC cancers which together account for almost 40% of all cancers. Twenty-five thousand people die each year from RLC cancer in Australia.

Fast forward to 2022 (a decade since the Therapeutic Goods Administration approved Ipilimumab), there are about ten approved ICI drugs for around 50 (mainly common) cancer types.

Rock up to the oncology outpatient clinic with your RLC cancer to receive the ICI drug prescribed by your physician, and you will have to fork out almost a thousand times more than a patient with a PBS-subsidised common cancer pays for the same drug.

In her role as an advocate for Rare Cancers Australia, Danielle T. remarked, ‘I have yet to meet anyone in the medical profession or at any level of government who could look me in the eye and say that is fair.’

To rub salt into the wound, the Federal Minister for Health pronounced, ‘…legislation does not permit exceptions to be made for individual patients, even (in those) where the drug has been shown to be useful on clinical grounds’ (

The usual course of six injections may cost you $40-80 thousand, and your physician may have in mind three courses. Yes, there are ‘special access’ and ‘buy now pay later’ schemes to alleviate the pain of the expense. And some pharmaceutical companies make ‘compassionate access’ available (or, more often, not) upon individual application.

However, it transpires that ICI drugs is have not lived up to their early expectations. While durable remissions may be obtained in some patients, often survival times are only a few months longer than that achieved by conventional therapies. Furthermore, outcomes can vary between tumour types and among patients with the same tumour type. Indeed, quality of life may be worse than that afforded by palliative care. The autoimmune side effects due to turbo-charged immune cells see to that.

How does the managing physician relate all this to an RLC cancer patient with terminal disease sitting before her? She knows the personal circumstances of the patient and the patient’s family. She knows the sequelae for many families forking out such huge sums to pay for a treatment for a loved one, which may well land a family into financial circumstances from which it will never recover. Homes get re-mortgaged, businesses sold, retirement savings spent, superannuation cashed in, all to pay for the ICI drug. There will even be a double financial hit if a combination of two ICI drugs is indicated. And the drug(s) might not even work for that particular patient.

A family has one preoccupation; a last-ditch attempt to save the life of a loved one, no matter how costly and how slim the chances. Some RLC sufferers struggle with guilt over the financial and emotional turmoil their disease has landed the family.

Whether to impose further turmoil upon a family on top of what they are already shouldering is a quandary the managing physician must navigate.

As Danielle T. remarked to her parents; ‘How much is my life really worth’?

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