We need faster approval of new drugs

12 minute read

No-one wants a system where treatments that could have changed the course of patients’ lives aren’t accessible to anyone but the wealthy.

For patients with cancer, the right test and the right treatment at the right time are critical to optimal patient outcomes. 

Consider the following hypothetical scenario: 

A particular new and expensive drug very substantially prolongs patients’ lives when administered immediately following surgical resection of a specific type of cancer. It’s not currently subsidised through the PBS (Pharmaceutical Benefits Scheme). It’s been approved by the US FDA (Food and Drug Administration) and is considered standard of care in the recently updated Guidelines of the American Society of Clinical Oncology (ASCO) for the management of patients with this particular cancer.

A patient at high risk of tumour recurrence and otherwise a candidate for the drug in question presents to their local oncologist shortly after recovering from their surgery for this specific tumour, but is told that although there is a treatment available in some parts of the world, it’s not currently on the PBS. To expand on this hypothetical scenario, the PBAC (Pharmaceutical Benefits Advisory Committee) had met 2 weeks earlier, rejecting the pharmaceutical company’s application for a PBS listing, instead recommending the pharmaceutical sponsor provide additional evidence and/or reduce the price of the drug to support its future listing, given the uncertainty of the available evidence and the high cost of the drug in question.

This scenario is typical of situations frequently faced by oncologists and patients as a result of current HTA processes. The PBAC must reach a decision based on the sometimes limited evidence available. In such circumstances, there may be considerable uncertainty regarding cost-effectiveness, particularly for drugs targeting rare and less common cancers or biomarker-driven subgroups of more common cancers for which large scale definitive clinical trials are either not feasible or arguably not ethical (given the magnitude of the benefit seen in earlier, smaller studies). It’s worth noting though, that the sponsor may simply not have any additional clinical data to support their application.

In any case, the patient in this hypothetical example is unable to pay for the drug in question and instead receives the best available standard of care (but not the new drug).

Unfortunately for this patient, the tumour recurs two years later. Now, even if the new drug were generally available on the PBS at this point, that’s not relevant for this patient as they are no longer immediately post-surgery – the circumstance in which the new drug in question has been shown to be clearly beneficial. Any potential benefit of this life-prolonging treatment is lost forever for this particular patient and their family. 

Far from being unusual, this scenario is typical of what happens, day after day, to patients with cancer around the country. Many new drugs subject to HTA (Health Technology Assessment) processes are just not available at the time the patient actually needs them. And because neither the cancer nor the appropriate treatment can be put on hold while the current ‘business as usual’ HTA processes play out over lengthy periods – on average around 2-3 years (depending how you measure these time frames) – a tragic reality of present day-to-day practice is that these patients often miss out on the potential opportunities offered by new drugs altogether.

Clearly, not every new cancer drug has the potential to substantially prolong/improve patients’ lives, but given the extraordinary wave of new cancer drugs, cellular therapies, devices, diagnostic tests and procedures currently under development, this is fast becoming the norm.

HTA processes are meant to be patient-centric, but no priority seems to be given to existing patients – patients requiring treatment today and tomorrow and the day after. There are over 600 people who walk into clinics across the country every single working day and walk out with a new diagnosis of cancer. That was more than 160,000 Australians in 2022. 

And of the 600 people diagnosed today, over 180 are destined to die prematurely of their cancer. Almost 50,000 people in 2022.

Potentially, one of the ever-increasing numbers of novel diagnostic tests, devices or innovative treatments available in other developed countries could prolong some of those lives, if only they were available at the time they were actually needed.  

The context is important here. Like virtually all drugs subject to PBAC consideration, the hypothetical drug referred to above will have already been shown to be safe and effective in the specific clinical scenario by the TGA, because that’s invariably a prerequisite for any drug to be considered by the PBAC. And in this example, it’s been approved by the FDA, which is also required by law to consider the safety and effectiveness of any new drug or device or test intended for the US market.

But until it’s approved by the PBAC (on average 2-3 years), and subsequently listed on the PBS (which often takes an additional 6 months once actually recommended for funding), the drug is essentially not available to most people because of the high out-of-pocket costs involved.

It seems as if current HTA processes are primarily concerned with how to best spend taxpayer funds in order to provide cost-effective health care for future generations of patients, rather than the patients of today.

Until HTA processes prioritise the needs of Australians who are currently unwell rather than those diagnosed with cancer sometime in the future, we are limited to a model that treats the patients of today as second-class citizens. A person who is sick today and who needs a specific new test or treatment or novel diagnostic procedure which is still subject to ongoing ‘negotiations’ (otherwise called the HTA assessment process) will miss out. They are just not going to receive the right test and the right treatment at the right time.

Unless these priorities change, existing patients – fathers/ mothers/ brothers/ sisters/ children/ grandparents – must endure a system that takes little cognizance of the fact that they too were once taxpayers (and indeed may still be), who could potentially lead better and longer lives if they could access new tests or treatments at the time they were most needed. 

Looking after the future patient is an important imperative, but to do so at the expense of the 600 patients diagnosed with cancer on the day this correspondence is being read, and the 600 diagnosed tomorrow and the next day and each day after that, is a cruel and inherently inequitable system. Inequitable because as soon as drugs become available on the market anywhere in the world – in this hypothetical example, immediately after FDA approval – wealthy Australians can and do access these treatments from international markets. These privileged few achieve the right test and the right treatment at the right time – something the vast majority of Australians can only dream about. 

Surely no-one wants a system in which many of the new drugs/devices/tests that could have changed the course of patients’ lives simply aren’t accessible to anyone but the wealthy.

During these protracted negotiations between the PBAC, the relevant section of the Department of Health (DoH) and the sponsor, armies of health economists try and quantify QALYs (quality adjusted life years) or incremental cost-effectiveness ratios (ICERs), with the paramount objective of either reducing or increasing the unit price of the new drug, depending on whether they represent the Government or the product sponsor. Unit price seems to be main game, with a final dollar figure generally requiring multiple resubmissions to the MSAC (Medicare Services Advisory Committee) or the PBAC, or time-consuming out-of-session discussions causing inevitable and prolonged delays.

‘But what about us?’ the anguished 600 hundred patients and their families may shout. Collateral damage is the only way I can describe their predicament.

The agencies involved need to come to grips with the fact that any holdups in the process are at the expense of existing patients. It is perverse that the way we do things currently has become morally acceptable in the name of the economic arguments around the ‘greater good’ – the responsible use of taxpayer funds for future generations of patients. Surely this loss of perspective is not what was intended and urgently needs to be reassessed.

In the end, most drugs with meaningful effectiveness do get approved after a consensus regarding unit price and case volume has been reached through these protracted negotiations between the Department of Health, PBAC, MSAC and the sponsor. But in getting to that point, far too many patients with cancer today will have suffered unnecessarily as a result of the inherent and apparently unavoidable delays in approval for which each party blames the other. 

Is this a patient-centric system? Is this a system that’s fair, equitable and worthy of being part of our world-class healthcare delivery model?

Other countries have managed to address this issue and I am certain we have the capacity to do the same.

Here is one alternative model. When TGA approval is granted, patients with high priority conditions, such as cancer, are given immediate and affordable access to the drug or test in question. (Note that TGA approval is often fast-tracked as a result of recent changes to their processes – a paradigm that has not been significantly taken up by the PBAC, but hopefully will be considered as part of the current HTA Review).

High priority conditions will need to be defined but one such definition might be ‘life-threatening illnesses which are inherently unstable over time (e.g. cancer), that generally worsen progressively and for which treatments that work in one phase of the disease may be much less useful in subsequent phases of an illness’.

When such patients are given immediate access to the innovation in question, the PBAC, MSAC, DoH and the sponsor are then given 2-3 years to resolve whether there is sufficient evidence of cost-effectiveness to allow permanent listing on the PBS. If that can’t be realised, the drug is effectively delisted and is no longer available to future patients. 

This model will require both incentives and disincentives to ensure that both parties negotiate in good faith and in a timely manner, but this should not be beyond the imagination of the various stakeholders.

Under such a model, the fate of the patient of today will not depend on an unpredictable negotiation around price and cost-effectiveness. And the responsible parties (the sponsor and DoH/PBAC, etc) will be accountable for reaching agreement or otherwise in a timely way. Variations of this model work very effectively in several European countries without incurring unmanageable debt. 

The usual objection to such models is that once provided to a patient, drugs can’t be delisted or stopped. However, in clinical practice, no patient actually receiving the drug in question and who is benefitting from treatment would have to stop using it. Instead, where cost-effectiveness and specifically unit price and volume caps can’t be demonstrated or agreed, the drug would be withdrawn from further use for new patients. This seems no more complex than the current situation in which we allow existing patients to be denied what might be effective treatment while cost considerations are explored and resolved. 

Such models would allow the PBAC, MSAC and DoH to both prioritise current patients while at the same time pursue the required objective of maximising Commonwealth investment; a plan that I believe the hard-working, committed members of the PBAC and dedicated public officials would embrace. In addition, I have no doubt such models could be established in a manner that would be acceptable to the pharmaceutical industry, if they were designed in good faith. 

I accept that the model described above may not be perfect, but there are others we could use for inspiration, like the Cancer Drug Fund in the UK. The second iteration of that model has been considered a resounding success by all stakeholders; so much so that similar funds are being established for other serious and life-threatening conditions.

With due respect, I’d urge the Panel not to get caught up in the details of the ‘how’ until there’s an agreement of what tenets need to drive HTA processes in the future in order to avoid the ‘collateral damage’ described herein. 

Putting patients diagnosed today on an equal footing to future potential patients has to be a prerequisite for a truly patient-centric process in a modern, progressive and caring health system. Once we accept the importance of not ignoring what is required for the patient in need today, then as a community – patients and their families, sponsors, clinicians and Government – we can sit down and work out the optimal process.

To move forward we must affirm the key underlying principles defining the a priori importance of our mutual responsibility to today’s patients. Not to do so would be a sad outcome for this ‘once-in a generation’ opportunity to get HTA right for patients, for families, for the taxpayer, for the community of today and tomorrow.

This piece was adapted from a submission made to the HTA review on 29/05/2023. Professor John Zalcberg AO is head of the Cancer Research Program in the School of Public Health and Preventive Medicine at Monash University and a medical oncologist at Alfred Health. He was awarded Officer (AO) in the General Division in 2023 for distinguished service to oncology as a clinician-researcher, and as an advocate for those living with cancer. He is also the Editor of this magazine.

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