Chapter 3Treatment and clinical trials
You don't have to look far to see that the healthcare system discriminates against rare cancer patients … We are often not candidates for many clinical trials or the PBS listed drugs which could prolong life … This inequity in access to genetic testing and PBS listed medicines and trials is not just unfair but also grossly unethical, in my mind.
Introduction
3.1Inequitable access to medicines and clinical trials are two of the major barriers to treatment for people with rare and less common cancers, including neuroendocrine cancers, in Australia. This chapter examines Australia’s current and emerging cancer treatments, Australia’s policies on subsidising medicines, and patient access to clinical trials.
3.2Firstly, this chapter examines Australia’s shifting landscape from a traditional, one-size-fits-all approach to cancer treatment, to personalised forms of medicines and treatment that are guided by genomic screening and precision oncology.
3.3The chapter then explores Australia’s Health Technology Assessment (HTA) processes, which regulate what medicines are subsidised under the Pharmaceutical Benefits Scheme (PBS). It also outlines the current and past reviews of HTA processes to improve patient access to treatments and considers issues relating to indication coverage of medicines and challenges with reimbursement for specific cohorts.
3.4Finally, the chapter examines clinical trials in Australia and discusses the barriers that patients often encounter when attempting to receive treatment through these trials. It then explores existing initiatives that are designed to improve patient access to clinical trials.
3.5The chapter concludes with the committee’s view and recommendations to improve treatment outcomes for patients with rare and less common cancer.
Treatment
3.6Queensland Health explained that typically, there are three main types of cancer treatment that are available to patients in Australia. These treatments can be used in conjunction with one another to treat a patient’s cancer:
Surgery:
Removes the tumour or repairs part of the body impacted by cancer.
Radiation therapy/radiotherapy:
A localised, controlled dose of radiation that that kills or damages cancer cells so that they cannot grow, spread or multiply.
Chemotherapy:
Uses cytotoxic (toxic to cells) drugs to stop or slow cancer cells that are dividing rapidly. Chemotherapy can also impact non-cancer cells, causing serious side effects.
3.7In their submission, Queensland Health also indicated that there are other drug therapies available to patients, including targeted therapy, hormone therapy and immunotherapy. However, it noted that these treatments may not work for every person who has been diagnosed with cancer.
3.8Many submitters indicated that cancer care is usually delivered in metropolitan centres, which can force patients who live outside these areas to travel to receive treatment. The geographic barriers that patients experience and existing schemes that are designed to assist patients to receive care are also discussed further in Chapter 5 of this report.
3.9Omico, a national not-for-profit organisation focussed on delivering precision oncology to Australian cancer patients, and Roche, a pharmaceutical and biotechnology company, submitted that the typical treatments (surgery, radiotherapy and chemotherapy) are usually used as a one-size-fits-all approach. Roche elaborated that cancer medicines were historically developed to treat large patient populations, and as a result, most treatments have been developed to treat a cancer type that has developed within a specific organ or tissue, for example breast cancer.
3.10Relatedly, Omico explained patients with the same type and stage of cancer are receiving similar treatments, and as a result, patients ‘can experience debilitating side effects that can limit the treatment journey.’
3.11Inquiry participants also told the committee about the negative side effects that they, or their loved ones, have experienced from traditional cancer treatments. For instance, Michelle told the committee of her life partner’s, Mark, battle with stage 4 pancreatic cancer, and the severe chemotherapy side effects he suffered:
We were only offered standard of care (one of 2 available chemotherapy regimes) – both of which were completely brutal and apparently unsustainable. It soon became obvious that if the cancer didn’t kill him, the chemotherapy would. The landslide of side effects that come with such intense therapy, also come with a barrage of medications.
3.12Similarly, Emily, who was diagnosed with a rare cancer known as NUT Midline Carcinoma at age 27, detailed her own experiences receiving traditional cancer treatments. Emily originally received chemotherapy and radiotherapy, with concurrent immunotherapy, then switched to targeted treatments. Emily told the committee that the chemotherapy and radiotherapy resulted in:
… permanent vision loss, permanent damage to my pituitary gland (resulting in ongoing hormone imbalances), infertility, post traumatic stress disorder, as well as a medically induced opioid addiction. It could be argued that, if access to targeted treatment was allowed at an earlier point in my treatment, that my condition would not have deteriorated as far as it has. In addition, a number of the side effects previously mentioned, as well as those experienced during chemotherapy and radiotherapy, could well have been avoided.
A shifting treatment landscape
3.13Pharmaceutical company Bayer Australia identified a shifting cancer treatment paradigm, in which treatment is moving away from the one-size-fits-all approach, towards precision oncology.
3.14Omico submitted that precision oncology and therapies are sometimes called ‘next-generation treatments’ and commented that ‘cancer patients can now be treated based on the genetics of their disease, rather than where in the body the cancer has occurred.’
3.15Chief Executive Officer of Rare Cancers Australia, Ms Christine Cockburn, echoed similar sentiments when she explained the concept and advantages of precision oncology:
Lung cancer, at some point, was regarded as a single cancer or as a single disease … Now we’re starting to lean toward a more precise understanding, which is where the word ‘precision’ comes from, and now we understand lung cancer is 30 different diseases … what we can understand is what the fingerprint of each of the cancers is, and we end up with a target, or a biomarker, that we can target to treat the cancer. So what we’re looking at is each individual’s fingerprint of their cancer and what really will effectively treat that cancer. That concept is precision, or personalised, medicine.
Accessibility of genomic testing and precision oncology
3.16Roche explained that genomic testing is an essential component of precision oncology, as it identifies the specific molecular abnormalities that drive a patient’s tumour. Once abnormalities are identified, precision medicines can be matched to the patient to specifically inhibit what is causing the tumour to grow.
3.17Cancer Council Australia highlighted that a comprehensive profile of a patient’s tumour at the DNA level can result in greater diagnostic accuracy, targeted treatment options, and improved patient outcomes.
3.18However, Australian Genomics raised that a barrier preceding the selection of appropriate precision treatment is getting a molecular test in the first place, because if a person with cancer can get a test, ‘they have the chance to be matched to an existing or novel therapy’.
3.19Relatedly, inquiry participants have detailed that genetic testing and screening can be costly, thus acting as a barrier to accessible treatment. For example, Jon raised that genetic screening is not covered by the PBS and costs more than $5000. He also told the committee that genetic screening is often required more than once as his type of lung cancer, ALK+, frequently mutates. Jon raised concerns that ‘this is existing technology which has significant life extending benefits and may be out of reach of many patients.’
3.20Judi, who has been diagnosed with pancreatic cancer, expressed similar concerns when she told the committee that she could not afford a $6000 fee to have a genetic test that would have told her:
… what drugs would have killed that cancer and what drugs I could have taken in lesser quantities that wouldn't then have ravished my body, ravished my brain, ravished what's left of the organs that they didn't remove.
3.21Associate Professor Christopher Steer, President of Private Cancer Physicians of Australia reinforced concerns around the lack of government funding to provide wider access to genomics for more Australians:
It’s an unfair irony that the arrival of genomics or precision medicine is the first real opportunity to close the gap of patient disadvantage. However, without government funding or regulatory reform, genomics will also remain out of reach for those who need it most.
Existing genomic screening programs
3.22The Department of Health and Aged Care (the department) drew the committee’s attention to two major genetic screening programs that the government has provided funding to: the Childhood Cancer National Precision Medicine Program (ZERO Childhood Cancer program or the ZERO program), and Omico’s Precision Oncology Screening Platform Enabling Clinical Trials program (PrOSPeCT).
ZERO Childhood Cancer program
3.23The Children’s Cancer Institute and the Kids Cancer Centre at Sydney Children's Hospital Randwick developed the ZERO program – the largest research initiative for children’s cancer in Australia and one of the most advanced precision medicine programs globally.
3.24The Children’s Cancer Institute told the committee that they have delivered their program to children with aggressive, high-risk, relapsed or rare cancers since 2017, by providing them with genomic testing and recommended personalised treatment options.
3.25Associate Professor Vanessa Tyrrell, Program Director of ZERO at the Children’s Cancer Institute, explained that of the first 250 children that participated in the program, the molecular basis of cancer in more than 90 per cent of cases was identified, and a precision guided treatment recommendation was made 70 per cent of the time.
3.26Associate Professor Antoinette Anazodo, a paediatric and adolescent oncologist, provided the committee with an example of the impact that the ZERO program has had on children with cancer and their families:
An example that I am able to share, with the family’s permission, is a child with a liver tumour that I have been looking after. Liver tumours are very rare anyway, but this liver tumour had some features of an adult tumour and some features of a paediatric tumour. It’s been sequenced on ZERO and we know it’s a more aggressive tumour. We would never have had that new data earlier. But now, last week, we had a conference with the surgeons. Rather than having an upfront resection, this child will have a liver transplant. That's important because we know, based on the data, that this patient has a more aggressive tumour and has no molecular targets, so we know that the chance of cure is going to be based on the surgery that can be done upfront. It's information like this, upfront, that is really having a significant impact on what we as clinicians can offer.
3.27While the ZERO program received $61.3 million in funding via the Medical Research Future Fund (MRFF), the Children’s Cancer Institute drew to the committee’s attention that funding is only guaranteed until June 2025.
Precision Oncology Screening Platform Enabling Clinical Trials (PrOSPeCT) program
3.28Omico launched the PrOSPeCT initiative with the intention of using genomics to save or extend the lives of Australians who have incurable or advanced cancers, including rare cancers.
3.29PrOSPeCT builds on Omico’s Molecular Screen and Therapeutics (MoST) program – which focusses on specific populations with rare and uncommon cancers, or unmet need. The MoST program provides patients with genomic screening and then depending on eligibility, matches them with a clinical trial or other targeted therapy.
3.30Omico told the committee that it will provide 23 000 Australian patients with free access to genomic profiling and a clinical assessment of their results. Omico will also match patients to advanced precision treatments, including through linking patients to clinical trials.
3.31In its submission, Omico detailed that it received funding from both private and public funds, with the government providing $61.2 million in funding. The organisation anticipates that the PrOSPeCT program will lead to a $525 million investment in local clinical trials and $135 million in savings to the health system by providing access to innovative therapies via clinical trials.
3.32Many submitters called for an increase in genomic testing for cancer patients due to the positive treatment outcomes that it can generate.
Access to medicines
3.33The Minderoo Foundation told the committee that whilst exploring targeted therapies based on genomic abnormalities is key, it is equally important to create pathways that make it easier to access drugs and repurpose drugs, to ensure patients can receive the treatments for their unique genetic makeup.
3.34Similarly, speaking on precision oncology and childhood cancers, Professor Matthew Dun, Founder and Director of RUN DIPG and Professor of Paediatric Haematology/Oncology Research at the University of Newcastle, commented that:
drug access remains the largest challenge in the realisation of the full potential of precision medicine, and it is here that we see the largest inequities affecting the health outcomes of our Australian children with this disease.
Australia’s Health Technology Assessment (HTA) processes
3.35Many submitters highlighted that Australia’s current Health Technology Assessment (HTA) processes, which inform whether a medicine will be listed on the PBS, are a major barrier to ensuring that patients can access subsidised treatments in a timely manner.
3.36Rare Cancers Australia explained that Australia, like many other countries, uses HTA processes to inform decisions about what health technologies can be sold in Australia, and which will be subsidised by the government. It elaborated that:
HTA provides a pathway to achieving subsidised access to a new medical technology, but it is a time-consuming process. For cancer treatments, the time between submission to the [Therapeutic Goods Administration] for regulatory approval, which makes a drug available to people in Australia, and PBS listing, which makes it subsidised by the public healthcare system, may be long, and some medicines may take several attempts to achieve a successful listing.
3.37Medicines Australia, a representative body for research-based pharmaceutical companies, drew the committee’s attention to the timeframe between regulatory approval and subsidisation of innovative medicines in comparative countries. In Australia, the delay is 466 days across all medicines, compared to Japan where it takes 102 days, 136 days in Germany, and 156 days in the United Kingdom. However, Medicines Australia also pointed out that for medicines that treat rare diseases, including rare cancers, this timeframe is two to four years longer in Australia than in other comparable countries.
3.38Drawing on the findings of the House of Representatives Standing Committee on Health, Aged Care and Sport’s ‘The New Frontier Report’, Ipsen explained that Australia’s HTA processes are designed primarily for more common diseases.
3.39Bristol Myers Squibb Australia considered that the lack of evidence for rare cancer treatments, and the challenges of demonstrating cost-effectiveness of those treatments, makes the HTA evaluation processes very challenging.
3.40Consequently, Bristol Myers Squibb Australia called for the HTA processes to accommodate the limitations and challenges associated with rare cancer treatments when considering whether to list a treatment on the PBS.
3.41Relatedly, Medicines Australia concluded:
In some cases, Australian patients miss out entirely on treatments where other countries have established HTA processes tailored to evaluate treatments for rare diseases, including rare cancers.
3.42More specifically highlighting these issues, Dr Minmin Li, Fellow of the Royal Australasian College of Physicians, and Associate Professor Kathy Tucker AO, highlighted the impact of Australia’s HTA evidentiary barriers on patients with Von Hippel‑Lindau disease (VHL). They explained that VHL is a rare syndrome caused by a germline heterozygous mutation in the VHL gene and is associated with a high lifetime risk of multisystem rare tumours. They informed the committee that a treatment exists for VHL patients known as belzutifan, an oral therapy which ‘has shown to be highly efficacious’. Belzutifan has been available to patients in the United States since 2021, however, access in Australia is severely limited since it is TGA approved but not listed on the PBS.
3.43NeuroEndocrine Cancer Australia reported that Australian VHL patients are ‘forced into a cost share model of $12 000 AUD per month’ and noted that this is a cost that very few Australians can afford. Lana, a submitter who detailed her experiences after being diagnosed with VHL, echoed these sentiments:
It [belzutifan] is TGA-approved and it is HERE in Australia. But because it is beyond unaffordable, it is therefore inaccessible. Belzutifan shrinks VHL tumours, simple as that. It is the only drug treatment available to those suffering from VHL.
3.44Lana also noted the delays in PBS funding for belzutifan:
… the process is agonisingly slow. This is due to the number of affected individuals in the trial being lower (thanks to their disease rarity) than other trials which are conducted against a placebo (proving that the drug works when compared to this). As such, the results from the Belzutifan trial, which would be used to make an application for it to go on the Pharmaceutical Benefits Scheme (PBS), are limited … inappropriate evidence standards are applied to drugs for rare conditions when they shouldn’t be.
Findings of the House of Representatives Standing Committee on Health, Aged Care and Sport’s ‘The New Frontier – Delivery better health for all Australians’ report
3.45When considering approaches to reforming HTA processes, many submitters echoed the recommendations made in the ‘New Frontier Report’. For instance, Bristol Myers Squibb Australia reinforced the need for HTA evidentiary requirements for rare and less common cancers to be developed, which would include a greater acceptance of real-world evidence. Additionally, pharmaceutical companies AstraZeneca, Bayer, Telix and Roche highlighted the recommendation of establishing a Centre for Precision Medicine and Rare Diseases within the department, with the view of ensuring that the department’s capacity is enhanced to provide Australians with timely access to new medicines and technologies.
Managed Access Program
3.46Some submitters also highlighted the specific findings in the ‘New Frontier Report’ regarding Australia’s Managed Access Program (MAP). For context, Bristol Myers Squibb Australia explained that the MAP is a mechanism that enables products to be listed on the PBS where there is a special circumstance of high unmet clinical need, or to allow for the resolution of otherwise unacceptable clinical or economic uncertainty.
3.47However, IQVIA Australia pointed to the findings within the ‘New Frontier Report’ that the MAP is underutilised. MSD Australia corroborated this finding, and raised that from 2010 to 2017, the MAP was used for only 11 drugs and resulted in the implementation of only three drugs.
3.48Consequently, some submitters recommended increasing the use of the MAP to improve access to treatments. NeuroEndocrine Cancer Australia specifically stated that this avenue could assist patients with VHL to access belzutifan.
3.49IQVIA Australia suggested that the MAP could be guided by England’s Cancer Drugs Fund (CDF) as an example model. The CDF provisionally funds cancer therapies for two years, which allows the sponsor to gather evidence to manage uncertainties and support the routine use of the drug. An example is the funding of a therapy called Yescarta via the CDF:
Yescarta is one of those therapies that in the UK have been funded through that program. It meant that, from the regulatory submission to patient access, there was only 15 months. In Australia is has been 32 months, so there is a huge difference, and I think that it highlights that there is a huge need for that type of program in Australia.
3.50AstraZeneca was also supportive of Australia introducing a model similar to the CDF.
HTA Policy and Methods Review
3.51Submitters drew the committee’s attention to the department’s HTA Policy and Methods Review (HTA Review), which was due to report on 4 May 2024 but at the time of writing has not yet been published. For instance, Rare Cancers Australia argued that:
Through the HTA Review, currently underway, we need to seize the opportunity to adapt our HTA system to consider different levels of evidence, as well as novel trial designs, such as basket trials or umbrella trials, that consider multiple targeted therapies across multiple cancers.There is an increasing number of precision oncology therapies that are coming to market with phase II data, and as such, a greater reliance on real world evidence for ongoing evaluation is needed.
3.52Additionally, IQVIA raised that HTA review provides an opportunity to streamline access for rare cancer therapeutics. Similarly, Ms Penny George, Director of Corporate Affairs for AstraZeneca, called for the HTA review to reduce wait times between TGA approval of a treatment and PBS listing to three months.
3.53Ipsen echoed this sentiment and considered that the committee’s inquiry was timely as it coincides with the HTA Review. It expressed hope that the inquiry would lead to a meaningful reduction in wait times.
3.54MSD Australia was also of the view that the HTA Review provided an opportunity to address delays in the system.
Indication coverage
3.55Rare Cancers Australia told the committee that once a treatment is subsidised on the PBS, people with rare and less common cancers continue to face challenges in accessing affordable medicines. In Australia, medicines are TGA registered and PBS listed based on specific medical conditions, known as indications. Consequently, a new TGA registration and PBS listing is required for each indication where a cancer medication could be used.
3.56Rare Cancers Australia also informed the committee that doctors can prescribe medicines for another indication once it has been approved for an original indication. However, Rare Cancers Australia flagged that this can lead to inequitable access, as only a small proportion of people can afford the non‑subsidised medicines, which can cost many thousands of dollars.
3.57In a joint submission, Cancer Council Australia raised that it has previously reported the confusion and frustration that people with cancer experience when they discover that their medication is approved for reimbursement for one purpose, but not for their own condition.
3.58For example, The Sydney Children’s Hospital Network explained that crizontinib, an anaplastic lymphoma kinase (ALK) inhibitor, is TGA registered and subsidised by the PBS to treat high stage ALK-and/or reactive oxygen species-rearranged lung cancer. However, despite its approval for the treatment of other cancers in the United States, the Sydney Children’s Hospital Network reported that:
… in Australia, there is no mechanism for crizotinib to be funded for cancers other than lung cancer, unless the sponsoring pharmaceutical company is willing to reapply to the TGA to extend the registered treatment indications of crizotinib to include other rare cancers.
3.59Telix Pharmaceuticals and the Children’s Cancer Institute suggested that indication coverage could be improved by adjusting regulatory and reimbursement processes for treatments to be registered by molecular indication, as well as by disease indication. This recommendation was also made in the ‘New Frontier Report,’ supported by evidence in the United States where a treatment was approved for a molecular indication, where the treatment was not listed for a specific type of cancer, such as breast cancer, but instead was listed for every cancer where an NTRK fusion was found.
3.60Cancer Trials Australia told the committee that clinical trials are the ‘only way to improve the evidence base’ to help ensure the availability of new, potentially life-prolonging treatments that may ‘have an excellent rationale, but not yet be reimbursed for rare cancer indications.’
3.61TrialHub echoed this sentiment when it told the committee that it remains difficult to achieve PBS reimbursement for rare cancer novel targeted therapies without Phase 3 clinical trials (explored below), which are commonly reimbursed for multiple common cancer indications, as there is a lack of evidence that is ‘too difficult’ to generate for many rare cancers. It stressed that this needs to change for the ‘third of Australians that are dying of a RC [rare cancer]’.
Reimbursement challenges
3.62Submitters drew the committee’s attention to other reimbursement challenges that rare and less common cancer patients can encounter on their treatment journey.
Cover for paediatric inpatients
3.63The Sydney Children's Hospital Network pointed to gaps in patient coverage under the Medicare Benefits Schedule (MBS) and PBS. It explained that the state governments fund inpatient care in public hospitals and that the federal government covers outpatient health care costs via the MBS, and outpatient medications via the PBS.
3.64The Sydney Children’s Hospital Network explained that as a result, patients are not eligible for MBS funding when they are inpatients at public hospitals. Consequently, any tests for inpatients in public hospitals must be paid by the hospital itself, the oncology department, or the patient and their family. It stressed to the committee that almost every child with cancer has their ‘diagnostic work-up, including tumour biopsies and treatment’ performed whilst they are inpatients at a public hospital.
3.65Further, the Sydney Children’s Hospital Network argued that:
Whilst the intent of Australian health care funding is to provide equitable access to appropriate tests for all eligible patients, in practice admitted inpatients in public hospitals are disadvantaged …
3.66Dr Richard Mitchell, the Director of the Kids Cancer Centre, Sydney Children’s Hospital Randwick, further stressed that:
When it comes to paying for treatments, all of the expensive cancer therapies do not fall under the PBS when you're a child stuck in a hospital.
Support for patients who require dental and maxillofacial rehabilitation
3.67Head and Neck Cancer Australia flagged their concerns with the committee regarding reimbursement for oral and maxillofacial rehabilitation.
3.68It told the committee that patients with head and neck cancers are offered ‘the most complex and involved surgery covered by Medicare’ but highlighted there is no affordable route for patients to access dental and maxillofacial rehabilitation afterwards. Head and Neck Cancer Australia also noted that this includes dental implants and facial prosthetics to replace parts of the mouth or face, including the eye, ear, nose or cheek.
3.69At a public hearing of the inquiry, Ms Nadia Rosin, Chief Executive Officer of Head and Neck Cancer Australia, explained that dental implants could cost anywhere between $20 000 and $30 000, and that a facial prosthetic can cost up to $6000, which also needs to be replaced every few years.
3.70Relatedly, Michael who was diagnosed with throat cancer and is a member of the support group ‘the Larykins,’ told the committee of his experience with throat cancer and the resulting dental costs:
The other downside is that I've now got eight cracked teeth, a crook tendon in the jaw and a broken muscle in the jaw thanks to radiation. So, if you think the first quote to get my 2½ teeth fixed was $13,600, you don't want to know what it is for that amount of work. I'm like, 'What do you do?'—and there's no dental cover.
3.71Ms Rosin also told the committee that not having access to facial prosthetics after receiving treatment can severely lessen a patient’s quality of life:
We've been told stories of people not leaving the house, avoiding their grandchildren or using a tissue with tape to cover their face when they go out. For some people, wearing a mask during COVID gave a sense of freedom they hadn't experienced since surgery.
Clinical trials
3.72The committee received overwhelming evidence from submitters on the important role that clinical trials play in providing treatment to people with rare and less common cancers.
3.73Queensland Health explained that clinical trials are conducted to investigate the safety and efficacy of new cancer treatments and drug combinations, with results being used to identify new treatment options or complement existing treatments.
3.74Emphasising the importance of clinical trials, Rare Cancers Australia informed the committee that clinical trials have ‘become the new standard of care’ for many people with rare and less common cancers where they have exhausted other options, or where there are no other effective treatments available.
3.75Cancer Trials Australia echoed that clinical trials are frequently the only ‘viable treatment option’ for people with rare and less common cancers but indicated that they are often difficult to initiate and conduct, and to access for patients.
Clinical trial activity in Australia and overseas
3.76TrialHub informed the committee of the various phases of clinical trials that a treatment must undergo in Australia for it to become standard and widely accessible for use:
Phase 1: establishes the safety of the treatment;
Phase 2: evaluates dosing administration, including frequency and some signal of efficacy; and
Phase 3: involves a larger study which compares the new treatment against the existing treatment standard.
3.77Pharmaceutical company MSD Australia submitted that compared to more common cancer clinical trials, there are fewer clinical trials for rare and less common cancer treatments due to the difficulty in recruiting sufficient patient numbers to conduct Phase 3, randomised controlled trials. Evidentiary standards and clinical trials are further discussed in Chapter 6.
3.78TrialHub provided statistics in their submission that compared open clinical trials for all cancers with open rare cancer trials:
Table 3.1 Clinical trial activity in Australia
| |
| | | |
Phase I/II | 72 | Phase I/II | 29 |
Phase II | 100 | Phase II | 32 |
Phase II/III | 11 | Phase II/III | 3 |
Phase III | 118 | Phase III | 32 |
Phase IV | 6 | Phase IIIb/IV | 2 |
Other | 96 | Other | 33 |
Total: | 504 | Total: | 170 |
Source: TrialHub, Submission 37, p. 2.
3.79Some inquiry participants flagged that pharmaceutical companies do not view Australia as an economically attractive destination to conduct research or clinical trials due to Australia’s relatively small population. Cancer Council Australia drew on anecdotal evidence to explain that these types of clinical trials ‘are not attractive to research sites/sponsors, as they are less financially viable than trials for more common cancers, with larger patient pools to draw from.’
3.80TrialHub and Cancer Trials Australia pointed out that consequently, rare cancer clinical trials can be difficult to initiate, as funding tends to be based on per‑patient enrolments. They explained that for low incidence diseases, it may be insufficient to prioritise trial unit resource allocations over common cancer trials. TrialHub and Cancer Trials Australia advised that this results in higher running costs to the participating unit with lower reimbursement.
3.81Relatedly, Elsa spoke of her sister, Kristen’s, battle with low-grade serous ovarian cancer, and their experience relating to clinical trials:
We came to understand that clinical trials are typically not funded or resourced for rare-cancers. This is economically understandable, however it felt incredibly unfair that Kristen potentially could have benefited from these promising new drugs or new drug combinations being tested in cancers that have similar profiles to hers.
3.82Dr Kurt Lackovic, Chief Executive Officer of Cancer Trials Australia did raise that Australia ranks third in the world, behind the United States and China, for the initiation of phase 1 clinical trials. Dr Lackovic also highlighted that ‘later‑stage trials are more likely to include Australia if the phase 1 trials are undertaken here.’
3.83The department advised that the Australian Government has committed $750 million from 2022–23 under the MRFF’s Clinical Trials Activity Initiative to increase clinical trial activity in Australia.
Access to international clinical trials
3.84The committee received evidence from inquiry participants who have had to consider travelling overseas to access clinical trials for themselves or their loved ones. For example, Megan, whose daughter Kira is now in a surveillance phase after being declared NED (No Evidence of Disease) following her neuroblastoma battle, stressed to the committee that:
The only thing scarier for a Neuroblastoma parent than receiving that initial cancer diagnosis, is the threat of relapse. Relapse for an Australian child with Neuroblastoma is almost certainly a death sentence with no access to curative therapies on home soil. Families are forced to fundraise and/or self‑fund travel to access treatment overseas, where clinical trials are more readily available …
3.85Megan also submitted that there is ‘very little hope for neuroblastoma families’ that ‘meaningful research and clinical trials on home soil will be forthcoming’ which ‘provides a very bleak view of what the future might hold for our child should she relapse.’
3.86Additionally, Elnaz, who was diagnosed with a rare type of lung cancer called ALK Positive in her late 30s, told the committee of her own experience relating to treatment and the possibility of overseas travel to access clinical trials:
Advanced ALK Positive lung cancer usually gets treated with targeted therapy using oral medication. Unfortunately, I have progressed on the two medications available for treatment and have to wait for my oncologist to advise if any clinical trials are available for me. Unfortunately, not so many clinical trials are recruiting in Australia, and I may have to travel overseas to save my life.
3.87The department highlighted that the Australian Government funds the Medical Treatment Overseas Program (MTOP) to ‘assist eligible Australians with a life‑threatening medical condition to access life-saving medical treatment overseas where treatment is not available in Australia.’ However, some submitters informed the committee that the MTOP excludes travel for clinical trials.
3.88Kristina and Brett, parents of seven-year-old Oscar who has been diagnosed with neuroblastoma, implored the government to provide further funding to assist families like theirs:
Additionally, we need government support and financial assistance to access overseas clinical trials. Despite what we've been told by our local medical experts, there are cures for relapsed neuroblastoma internationally and there are more treatment options available overseas. So we ask you, why should families be forced to choose between their child's life and selling everything they own to afford treatments abroad? What would you do if it were your child? How can you make a difference today, to help support paediatric cancer patients in the future?
Australian clinical trials - barriers to patient access
3.89Australian Genomics and Omico told the committee that it is currently estimated that only 8 per cent of all cancer patients in Australia access clinical trials.
3.90Submitters outlined various barriers that inhibit patient access to Australian clinical trials, as explored below.
Australia’s fragmented health system
3.91Bristol Myers Squibb Australia informed the committee that there is a disparity in accessing clinical trials in Australia, which is exacerbated for patients with rare and less common cancers. Both Medicines Australia and Bristol Myers Squibb Australia raised that one of the main causes of disparity is Australia’s federated health system:
The current landscape of clinical trials in Australia is marked by fragmentation due to a federated system. There is variability in trial protocols and regulatory processes between each jurisdiction. [Citation omitted] Rare cancer trials suffer from disparities in access to cutting-edge treatments due to these inconsistencies, leaving patients and researchers struggling to navigate a complex and inefficient system.
3.92Dr Chloe Georgiou, Chair of the Victorian Rare Cancer, Clinical Trial Alliance, also told the committee about the difficulties that a fragmented health service system presents in terms of clinical trials:
In Victoria we’ve got a very fragmented health service system in terms of jurisdiction and oversight. New South Wales has got its seven districts, and Queensland has a statewide system. Victoria has 54 health services, I think, and every single of those would need to go through its own governance process to start a trial.
Navigating information systems and websites
3.93Rare Cancers Australia submitted that access to clinical trials is often limited due to the high level of health literacy required by patients to identify trials that are relevant to them. Health literacy is more broadly discussed in Chapter 5 of the report.
3.94Similarly, the VCCC Alliance also identified that clinical trial databases are not well maintained and are often difficult to navigate, and the Pancare Foundation highlighted that there is a limited patient awareness of trials and how to find them, particularly for those who cannot use clinical trial websites.
3.95This sentiment was echoed by Cancer Trials Australia, who submitted that databases are not easily searchable for rare cancers, as they are often included in subgroups or within tumour agnostic trials. Cancer Trials Australia concluded that for both patients and practitioners, this is a barrier to understanding what clinical trials are available.
Rural and regional patients
3.96The committee received evidence that rural and regional patients with rare and less common cancers may encounter additional barriers when attempting to access clinical trials.
3.97The Leukaemia Foundation submitted that many newer treatments or therapies may only be available through clinical trials, ‘which are mostly available at select major metropolitan treatment centres.’
3.98Some inquiry participants were supportive of teletrials when considering how to overcome geographic barriers to clinical trials. Queensland Health explained that the Australian Teletrial Program, funded by the MRFF, intends to create a network of clinical trials which can enable regional, rural, remote, and Aboriginal and Torres Strait Island communities to access trials closer to home.
3.99Dr Wei-Sen Lam, Teletrials Medical Lead at the Western Australian Country Health Service, told the committee that:
You can imagine the patient who is dying from cancer but wants to be close to home or needs to stay in the region because of income and they have to work at home. They often say, 'I can't afford to go to your clinical trial; I can't afford to be away from home.' This program really helps country patients have access closer to home.
3.100Similarly, the Minderoo Foundation submitted that it believes investment to improve tele-trials would ‘support equitable access to clinical trials regardless of geographical location’ and that the Australian Teletrial Program will ‘improve access to, and participation in, clinical trials for rural, regional and remote Australians.’
3.101Issues related to regional and rural workforces and their impacts on diagnosis and treatment for patients with rare and less common cancers, including neuroendocrine cancer, are further discussed in Chapter 5.
Funding of paediatric clinical trials
3.102Some submitters drew the committee’s attention to the particular importance of clinical trials for children with cancer. The Children’s Cancer Institute explained that enrolment in clinical trials is the standard of care for paediatric cancers due to the lack of medicines available.
3.103However, Neuroblastoma Australia raised that due to the small patient populations, drug companies have no incentive to invest in clinical trials for children until a treatment has been proven to be safe and effective in treating adults. Neuroblastoma Australia raised that this is a significant problem, as neuroblastoma ‘almost never affects adults.’
3.104Similarly, the Children’s Cancer Institute raised that this is particularly challenging, as there is ‘a lack of industry support’ to supply drugs or establish clinical trials, meaning that paediatric clinical trials largely rely on philanthropic or government funding. The Sydney Children’s Hospital Network echoed that:
… funding mechanisms for children’s cancer clinical trials units is highly variable across Australia with many cancer clinical trials units relying on philanthropic funding to fund staff positions. Unlike adult oncology services, the majority of clinical trials that are conducted for children with cancer are investigator-initiated trials sponsored by large international academic trials consortia such as the Children’s Oncology Group (COG) and the International Society for Paediatric Oncology (SIOP).
3.105Angie and Tamer, parents of Eve, Beau and Terry, parents of Ryley, and Alan and Truc, parents of Madeline, all told the committee of their children’s battles with Diffuse Intrinsic Pontine Glioma (DIPG). When discussing clinical trials for DIPG in Australia, all three sets of parents highlighted that since 2018:
only three new [DIPG] clinical trials have opened in Australia (which have all only been in the last 12 months). The United States and Europe have a significant number of available clinical trials to give families some hope and yet once again bureaucratic red tape prevents Australian families from applying and accessing these medications and trials. When a clinical trial is available in Australia, positions are limited, and patients may not be able to access the trial. Biopsies are prerequisites for families to access a position on a clinical trial for their child. However, due to the diffuse intrinsic nature of a DIPG tumour, a biopsy is often not feasible or safe for children to partake in thus, forcing families to relinquish positions in a clinical trial.
3.106Angie and Tamer also indicated that there is no centralised hub for DIPG families and medical staff, making it difficult for parents to access current and reliable information on developments, treatment options, and clinical trials. They raised that as a result, ‘parents of children with DIPG are placed in the distressing position of having to shoulder the burden of finding relevant clinical trials.’
Existing initiatives to improve access to clinical trials
3.107Submitters drew the committee’s attention to various initiatives that could enhance clinical trial activity in Australia for patients with rare and less common cancer. These are explored below.
Omico’s PrOSPeCT program
3.108In addition to providing Australians with access to genomic profiling, as discussed earlier in this chapter, Omico illuminated that the PrOSPeCT program will also generate an estimated $52 million investment in local clinical trials.
3.109Further, Omico outlined that the program will facilitate the completion of clinical trials for both adults and children with cancer, which they stated is a ‘massive drawcard for new international investment in the Australian medical research sector.’
3.110Some submitters were supportive of the program. For instance, the Children’s Cancer Institute noted:
This initiative intends to attract pharma and biotech companies to our shores to undertake clinical trials, and therefore increase early access to new medicines for cancer patients …
3.111Roche echoed these sentiments and told the committee that it believes programs like PrOSPeCT are vitally important in raising Australia’s profile as an attractive destination for clinical trials. They explained that ensuring Australia has the infrastructure to link genomic analysis to clinical trial therapies ‘increases international confidence that Australia can make significant patient recruitment contributions and deliver on these trials.’
Clinical Trials Activity initiative
3.112As discussed earlier in this chapter, the government has committed $750 million over 10 years from 2022–23 to increase clinical trial activity in Australia under the Clinical Trials Activity initiative. The department submitted that this initiative would improve the evidence base that supports clinical care and help patients access relevant treatment, while also enabling researchers to bring international trials to Australia. The department outlined that the MRFFhas invested $25.53 million in 14 grants with a research focus on rare cancer under the initiative.
3.113Some submitters were supportive of the Clinical Trials Activity initiative. Yet, the Cancer Council Australia raised concerns regarding the broadened scope of initiative. They outlined that the initiative’s original focus was on clinical trials for rare cancers, rare diseases, and unmet needs. However, it explained that the focus has since been expanded to any disease or condition that meets the objective specified in each grant opportunity. It added that:
We are concerned that the Clinical Trial Activity Initiative’s expansion of focus could inevitably result in less priority and funding investment towards rare and less common cancer research.
National One Stop Shop and the National Clinical Trials Front Door
3.114In June 2023, the Australian Government released a consultation report on the National One Stop Shop (NOSS) platform. In October 2023, the government announced that all Australian health ministers agreed to establish the Inter‑Governmental Policy Reform Group, which will provide policy and operational oversight over the NOSS.
3.115Bristol Myers Squibb Australia explained that the NOSS aims to harmonise clinical trial systems in Australia, which will:
… transform the ability of patients to access clinical trials and therapeutic interventions, and make it easier for industry, sponsors, and researchers, to find, invest and conduct research in Australia.
3.116Throughout the consultation process, the government found that stakeholders broadly supported a public facing website, known as the National Clinical Trials Front Door, that would be the central point of access point for a range of groups, including researchers, sponsors, industry and carers.
3.117Medicines Australia argued that a nationally consistent and harmonised clinical trials systems ‘would encourage multinational innovator companies to bring their trials to Australia’ and would provide Australian rare and less common cancer patients with more opportunities to access treatment.
Research processes
3.118Australian Genomics flagged that Australia’s research processes need to be ‘more agile and receive timely approval’ to ensure that Australia is an attractive place to conduct clinical trials. Evidence relating to research and clinical trials is further discussed in Chapter 6 of the report.
Committee view
3.119Evidence to the committee highlighted clear inequities in access to treatments and clinical trials for people with rare and less common cancers, including neuroendocrine cancer.
3.120The committee firmly believes these issues and inequities must be addressed to ensure that patients receive the most appropriate treatment to support them throughout their cancer journey.
Access to genomic screening and precision oncology
3.121The committee received compelling evidence that genomic screening can link rare and less common cancer patients with targeted treatment options, via precision oncology, that may improve treatment outcomes.
3.122The committee is of the view that precision oncology treatments hold the potential to provide cancer patients with improved treatment outcomes, particularly when compared to traditional forms of treatment, such as radiotherapy and chemotherapy. However, the committee holds concerns that precision oncology may remain out of reach for patients if they cannot access genomic screening, particularly due to financial factors.
3.123Therefore, the committee reiterates its call to ensure that people with cancer have access to genomic screening, as recommended in Chapter 2, to enable rare and less common cancer patients to access precision oncology therapies where appropriate.
3.124The committee notes the work of the ZERO Childhood Cancer program. The committee is encouraged that this program can identify the molecular basis of cancer in children and provide their families with precision guided treatment recommendations.
3.125However, the committee is concerned that funding for the ZERO Childhood Cancer Program is only guaranteed until June 2025. The committee considers it vital that this program continues to be funded beyond June 2025.
3.126 The committee recommends that the Australian Government, through the Medical Research Future Fund, extend funding for the ZERO Childhood Cancer Program beyond June 2025.
Health Technology Assessment processes
3.127The committee notes the considerable evidence received in relation to the need to reform Australia’s Health Technology Assessment (HTA) processes to provide rare and less common cancer patients faster access to relevant treatments.
3.128The committee was concerned to learn of the lengthy timeframe between regulatory approval and the subsidisation of novel medicines on the Pharmaceutical Benefits Scheme (PBS) that exist in Australia, especially when comparing these timelines to other countries, such as Japan, Germany, and the United Kingdom. The committee is also mindful that the Managed Access Program is underutilised, therefore further limiting patients from receiving access to novel medicines.
3.129The committee firmly considers that HTA reform is needed to ensure that Australian patients can access affordable treatments. Accordingly, the committee echoes calls from submitters that HTA processes should be adjusted to account for rare and less common diseases, including cancers.
3.130Further, the committee is cognisant of the comprehensive recommendations made in the House of Representatives Standing Committee on Health, Aged Care and Sport’s ‘The New Frontier – Delivering better health for all Australians’ report. The committee notes the specific recommendation on areas for consideration and reform during the HTA Review, such as increasing the use of Managed Access Programs to facilitate earlier access to innovative medicines.
3.131The committee believes that the recommendations of both reviews have the potential to improve patient access to treatment, with a particular consideration on how to increase use of the Managed Access Program.
3.132The committee is also aware of patient frustrations upon discovering that medicines are not listed for their specific indication. The committee sees value in further exploring how to adjust regulatory processes to ensure equitable access to treatments for rare and less common cancer patients.
3.133Specifically, the committee notes the recommendation made in the ‘New Frontier – Delivering better health for all Australians’ report for the Department of Health to reform its regulatory and reimbursement processes to enable therapeutic goods to be registered and reimbursed by molecular indication, in addition to by disease indication.
3.134The committee recommends that the Australian Government utilise the Health Technology Assessment Policy and Methods Review to provide Australian rare and less common cancer patients with timely and affordable access to novel medicines.
3.135The committee recommends that the Australian Government ensure that the Managed Access Program is more widely accessed where appropriate.
3.136The committee recommends that the Australian Government adjust regulatory processes to broaden indication coverage for medicines that treat rare and less common cancers, including neuroendocrine cancer.
Reimbursement gaps
3.137Throughout the inquiry, the committee was made aware of concerning gaps in the reimbursement of therapies for some cohorts. The committee was troubled to learn of the particular impacts that the state and federal funding split for inpatient and outpatient care can have on paediatric cancer inpatients, specifically when families attempt to access reimbursement for testing and treatment.
3.138The committee recommends that the Australian Government work with state and territory governments to ensure the families of paediatric cancer patients are not financially disadvantaged for hospital stays.
3.139The committee is concerned that cancer patients who require oral and maxillofacial rehabilitation, including prosthetics, following treatment for head and neck cancers, must fully pay for these procedures.
3.140The committee is of the view that people who require essential oral and maxillofacial rehabilitation because of their cancer treatment should not be financially penalised.
3.141The committee recommends that the Australian Government work with state and territory governments to identify the barriers faced by cancer patients requiring rehabilitation, prosthetics and implants as a result of their treatment, with a view to ensuring they have financial support for those services.
Access to clinical trials
3.142The committee is concerned by the low number of rare and less common cancer clinical trials in Australia, particularly when considering that clinical trials are often the only treatment option available to rare and less common cancer patients who have exhausted other options.
3.143In particular, the committee notes that patients and families are not covered by the Medical Treatment Overseas Program (MTOP) if they need to travel overseas to access treatment via clinical trials. The committee is of the strong view that Australian patients and their families should not be faced with exorbitant out‑of‑pocket costs to access potentially lifesaving treatment.
Recommendation 15
3.144The committee recommends that the Australian Government review the eligibility criteria of the Medical Treatment Overseas Program, with a particular focus on access to clinical trials and treatments for rare and less common cancer.
3.145The committee notes the concerns raised by submitters that rural and regional cancer patients may be at a disadvantage when attempting to access clinical trials. However, the committee is supportive of the Australian Teletrial Program and believes that continued investment in teletrials will continue to improve access to clinical trials for rural and regional cancer patients. The committee discusses its views and makes its recommendation on teletrials in Chapter 5 of this report.
3.146The committee is also aware of the various barriers that patients face when attempting to access the limited number of rare and less common cancer clinical trials within Australia, including the fragmentation of the health system, the difficulty of navigating complicated websites and databases, the high levels of health literacy required, and reliance on practitioner awareness of existing clinical trials.
3.147However, the committee is encouraged by existing initiatives aimed at improving access to clinical trials and it believes that these stand to greatly benefit rare and less common cancer patients. The committee is optimistic that Omico’s Precision Oncology Screening Program enabling Clinical Trials (PrOSPeCT) program will lead to increased clinical trial activity in Australia for people with rare and less common cancers.
3.148Furthermore, the committee considers that the National One Stop Shop and National Clinical Trials Front Door platform is an initiative that would assist patients, practitioners, and family members to navigate Australia’s complex health system, as well as providing Australians with a simplified website to assist them navigate the clinical trials landscape. As such, the committee believes this platform should be implemented as a matter of priority.
3.149The committee recommends that the Australian Government work with state and territory governments to implement the One Stop Shop and National Clinical Trials Front Door platform as a matter of priority.
3.150The committee notes the concerns raised by inquiry participants that pharmaceutical companies have limited incentives to establish clinical trials for children with rare and less common cancers in Australia.
3.151The committee is concerned that as a result, Australian children with rare and less common cancers may not be receiving the same access to treatments and trials that are available overseas.
3.152The committee considers that there is merit in leveraging the Clinical Trials Activity initiative to have a specific focus on generating clinical trial activity in Australia for children and young people with cancer.
3.153Noting the concerns raised regarding the broadened scope of the Clinical Trials Activity initiative, the committee encourages the Australian Government to ensure that clinical trials and research opportunities for rare and less common cancers in particular are not overlooked under the initiative.
3.154The committee recommends that the Australian Government ensure the Clinical Trials Activity initiative has an appropriate focus on funding clinical trials and research for people with cancer, including children, young people, and patients diagnosed with rare and less common cancers.