Medicines

PBAC blocks access to new myeloma drugs: Prof Andrew Spencer

Thursday, 13 May 2021


Professor Andrew Spencer

Australian blood cancer patients are being denied access to life-extending drugs and treatments because of long delays and excessive regulation by the Pharmaceutical Benefits Advisory Committee (PBAC), a parliamentary inquiry has been told.

Leading haematologist Professor Andrew Spencer said the registry of myeloma cases established some eight years ago showed “very poor” outcomes for myeloma patients in Australia and New Zealand compared with other developed countries.

In myeloma patients over 70, the median survival rate in Australia was 44 months, almost half the median of 60 to 70 months in other countries, he said, giving evidence at the inquiry into approval processes for new drugs and novel medical technologies in Australia.

“That gap is getting bigger and bigger and bigger. That is not because we’re not good clinicians and good nurses and good hospitals but boils down purely to access to therapeutics. I could send someone to Greece three years ago to get daratumumab that I couldn’t give to my patient in Melbourne … We consider ourselves to be a First World nation, but we have Second World nation access to drugs,” he said.

Professor Spencer said it was notable that daratumumab was the first new drug  approved for myeloma in Australia in more than a decade.

“For the past 14 years, there were only two classes of drugs available to treat myeloma. Not only were they only two classes of drugs available but the PBAC would not allow us to combine those drugs—they could be used in isolation—which beggars belief scientifically, but that’s how it was.

“In that time frame, half a dozen other drugs have been approved in other First World countries with different mechanisms of action to treat myeloma but not in Australia.”

The new drug, indicated for relapsed myeloma in combination with another drug already in use, took five years after its initial presentation to be approved by PBAC.  However, the permitted combination was “the cheapest combination, the most toxic combination”, he said.

“It seems to me that this approach from PBAC is being driven by accountants; it’s not being driven by people who are actually looking at the evidence and saying, ‘Well, what is the best option for these patients?’ Because that clearly is not what happens in Australia. The process … is adversarial and it’s unrealistic.”

Australia’s small market size and PBAC’s approval delays and cost thresholds deterred pharmaceutical companies from even trying to get a drug reimbursed in Australia, he said.

“The process is failing on so many levels and, unless we change it, we are going to go further and further behind in drug delivery for cancer. We used to be held up as an example with the UK and Canada, where there were systems in place to look at the cost effectiveness and such,” he added.

“We now are so far behind those jurisdictions in drug access and it’s widening and widening because the process here does not work. There is a high level of arrogance at a level of the PBAC towards clinicians and pharma, and as for the modelling they use, in my experience, I don’t know where they get their information from – quite odd.”

Professor Spencer said Australian myeloma patients risked missing out on other new classes of drugs, such the monoclonal antibody elotuzumab.

“It has an incredibly favourable toxicity profile, so you can deliver this agent with minimal toxicity, which is very important in a disease like myeloma, because a lot of these people are in their 70s; they’re still productive people.”

However, the pharma manufacturer Bristol Meyer Squibb “didn’t even bother trying in Australia”.

Another example was the Novartis oral medication panobinostat, which he said had been proven effective in people with very advanced myeloma.

“So there are two agents which clearly have got utility in subsets of patients, and we’ve used extensively in clinical trials, so we know that they work. But there’s been no attempt to progress either of those drugs through the processes.

Professor Spencer was giving evidence to the ongoing inquiry conducted by the House of Representatives Standing Committee on Health Aged Care and Sport 

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