Chapter 6Research
We've got amazing researchers in Australia; we're world leading. Some people with rare and less common cancers have exceptional outcomes—world's best. Other people … pay with their lives
6.1This chapter explores Australia’s research landscape and examines ways to improve research and clinical trial activity in Australia, in aim of improving outcomes for people with rare and less common cancers, including neuroendocrine cancer.
6.2Firstly, this chapter outlines the Australian landscape of research and clinical trials by exploring the existing levels of funding, as well as funding gaps based on tumour types.
6.3It then considers options to address research gaps for rare and less common cancers, including:
financially supporting researchers;
funding biobanks;
encouraging genomic research; and
attracting pharmaceutical company investment in Australia.
6.4Finally, it examines how encouraging collaborative trials and adjusting evidence standards could generate increased clinical trial activity in Australia.
6.5The chapter concludes with the committee’s view and recommendations on providing better outcomes for rare and less common cancer patients through research and clinical trials.
Existing funding environment for Australian research and clinical trials
6.6The Department of Health and Aged Care (the department) outlined that the Medical Research Future Fund (MRFF) has invested $2.78 billion across 1145 health and medical research grants from 2015 to 2023, which includes $37.9 million in 25 research grants that focus on rare cancers.
6.7Rare Cancers Australia drew the committee’s attention to the findings of the 2023 National Audit of Cancer Research Funding in Australia, which identified 419 cancer trials with a total of $315 million in funding from 2012 to 2020. Rare Cancers Australia also highlighted that the Australian Government provided $147 million in funding for these trials, and that the state and territory governments provided $77 million.
6.8Subsequently, on 2 May 2024, the Australian Government announced its new ‘Health Research for a Future Made in Australia’ package, which includes a new 10-Year Low Survival Cancers Mission, focused on improving outcomes for people with a cancer where the 5-year survival rate is less than percent.
6.9The National Health and Medical Research Council (NHMRC) identified itself as the single largest funder of health and medical research in Australia. In its submission, the NHMRC explained that:
Most NHMRC funding is awarded in response to investigator-initiated applications in which the research is proposed by the researchers in the application to NHMRC, based on the expertise and research interests of the applicants.
6.10The NHMRC advised that a smaller proportion of funding is allocated to specific areas of unmet need, which can be through Targeted Calls for Researchand international collaborative schemes.
6.11For example, the department highlighted the MRFF 2021 Rare Cancers, Rare Diseases and Unmet Need grant opportunity, which awarded $2.3 million to Flinders University for the ‘Implementing a Nurse-Enabled, Shared-Care Model to Address Unmet Needs of People with Neuroendocrine Tumours: the AUS‑NET Trial.’ The department elaborated that this project intends to improve the quality of life and reduce the unmet needs of people with neuroendocrine tumours.
6.12However, the department acknowledged that proportional funding for research and clinical trials for many cancers ‘is low compared with the burden on the Australian population.’
6.13Rare Cancers Australia also noted that Australia is a leader in cancer research and that funding for rare and less common cancers has increased in recent years. Yet, it raised that the total funding required to close the gap between funding and the burden of disease and mortality caused by rare and less common cancers compared to common cancers ‘remains significant.’
6.14Professor David Chan, Chair of the Neuroendocrine Tumours Group, also raised concerns about the framework used by the NHMRC. Dr Chan contended the framework structurally disadvantages rare and less common cancer projects:
… one of the newest things I've noticed in the NHMRC framework is an instruction to the referees to evaluate feasibility, and that's defined as 'the ability to have enough patients for what you propose'. And the statistics are the same, whether you do a common or less common cancer. Unfortunately, that means less common cancers, rare cancers, are, by definition, almost structurally disadvantaged when it comes to the NHMRC framework.
6.15In a joint submission,Cancer Council Australia, the Cancer Nurses Society of Australia, the Clinical Oncology Society of Australia, Private Cancer Physicians of Australia, the Medical Oncology Group of Australia and the Lung Foundation Australia flagged that lower levels of funding for rare and less common cancers impacts on diagnosis:
the lack of investment to better understand rare and less common cancers limit the development of specific diagnostic criteria and appropriate diagnostic tools to identify those cancers that are already classified as rare and less common as well as cancers that are not sufficiently understood and remain ‘undiscovered’ yet still numerically rare and less common.
6.16In addition to improving diagnosis, Dr Chloe Georgiou, Chair of the Victorian Rare Cancer Alliance, Clinical Trial Alliance, emphasised the importance of research and trials for improving a patient’s quality of life:
We need more research and trials in rare cancers to provide information to assist patients and funders to make value based decisions on management options, and to know what treatments improve survival and quality of life, also avoiding unnecessary expenditure of money and time on treatments that do not have benefit …
Gaps in research funding by tumour type
6.17The Australian Rare Cancer Portal contended that current funding models favour cancers where breakthroughs have already been made, and further argued that ‘success begets success, with more challenging areas being left behind … Yet those rare cancer types require more research, not less.’
6.18The Daffodil Centre and the University of Sydney Cancer Research Network similarly told the committee:
Disparities in cancer research funding by tumour type are complex and multifaceted. A history of investigator-driven research and a tendency to build on success has led to underfunding of complex, rare and low-survival cancers. In addition, cancer research funding generated by large, tumour‑specific nongovernment organisations further skews the investment towards comparatively well‑served cancer types often with large survivor populations …
6.19In addition to gaps in research funding for rare cancers overall, inquiry participants also drew the committee’s attention to specific gaps by tumour type. For example, the Ovarian Cancer Research Foundation identified a ‘shortfall’ that ‘has persisted for two decades’ for funding allocated to ovarian cancer medical research and clinical trials. It reported that from 2016–2022, 0.7 per cent of all Commonwealth research funding was directed to ovarian cancer research.
6.20NeuroEndocrine Cancer Australia similarly stated that neuroendocrine tumours (NETs) have been traditionally underrepresented in research and stressed that targeted funding is needed through the MRFF for NETs research.
6.21The Pancare Foundation also pointed to low levels of funding for upper gastrointestinal cancers. They provided research funding figures from 2018 to 2020, which they stressed are ‘significantly less’ than ‘other low‑survival or high-survival rate cancers:’
Pancreas – $17 million
Liver – $11.4 million
Stomach – $7.2 million
Oesophagus – $2.8 million
Biliary/gallbladder – $0.
6.22Relatedly, Emma, whose mother passed away from pancreatic cancer, stressed the importance of even funding across all tumour types:
Why can there not be an overall even distribution of government funding to ALL cancers. What makes one cancer more important than the other? It’s like telling a family with a member with pancreatic cancer, ‘you’re not as important’ as another family whose member has breast cancer. How do you compare 92% survival rate for breast cancer to 12% for pancreatic? Money=research. Research = cure. Cure = having my mum with me still.
6.23Grace, Angie and Tamer, Beau and Terry, and Alan and Truc also identified gaps in Diffuse Intrinsic Pontine Glioma (DIPG) research. They submitted that:
DIPG has been plagued by a lack of progress in treatments and cures since the 1960s, leaving families and researchers grappling with frustration and heartbreak. Regrettably, DIPG stands out as the deadliest childhood cancer, inflicting immense suffering on young patients and their loved ones. What exacerbates the distress is the profound absence of answers concerning the disease's origin, prevention, and cure, leaving families grappling with the haunting uncertainties surrounding DIPG.
Improving levels of rare and less common cancer research
6.24In addition to increasing funding for underfunded tumour types, submitters also highlighted various mechanisms and options to address gaps in rare and less common cancer research. These are explored below.
Supporting researchers
6.25The Ovarian Cancer Research Foundation stressed that the research sector needs to be able to attract long-term funds and resources, since research projects are not short-term endeavours.
6.26Professor Matthew Dun, Founder and Director of RUN DIPG and Professor of Paediatric Haematology/Oncology Research at the University of Newcastle, echoed this sentiment, yet he noted that most research teams operate on research grants, which he referred to as ‘soft funding’. The Sydney Children’s Hospitals Network similarly noted that soft funding tends to be awarded to children’s cancer services via short term mechanisms, like philanthropic donations or grants.
6.27Professor Dun contended that soft funding fosters a ‘lack of job security’ which ‘promotes the loss of talented research minds to overseas positions or alternate industries.’ Relatedly, Dr Peter Wejbora, Director of Research Development and Partnerships at the Children’s Cancer Institute, told the committee that soft funding:
… introduces a great deal of uncertainty not only for our staff funded through those sources but, most importantly, for the kind of innovation and research based clinical services we can provide patients access to …
6.28Sandra, who was diagnosed with a midgut neuroendocrine tumour in 2016, emphasised that:
There is no personal income security for our researchers and no guarantee that funding for ongoing work will be forthcoming. Many research projects are abandoned as money streams dry up. Frustration levels escalate for researchers, doctors, patients and advocates as years pass with little improvement in treatment options and life expectancy … Who would want to spend their career chasing money and writing proposals for research which has virtually no chance of success in becoming realized? … How can we be “the clever country” with lacklustre support for our best and brightest?
6.29Ovarian Cancer Australia stressed that ‘we must provide researchers with ongoing funding to see the breakthroughs we have in other cancer types, as opposed to one off funding injections’ and that ‘it cannot continue to be so hard for [researchers] to persist in their work.’ Soft funding is further discussed in Chapter 5 of the report when examining broader health workforce challenges.
The role and funding of Australian biobanks
6.30The Victorian Cancer Biobank explained that biobanks ethically procure, manage and distribute human biomaterials, which are ‘critical for translation of research into new treatment, detection and prevention approaches, leading to improved cancer outcomes.’
6.31Professor Matthew Dun also highlighted the importance of collecting and assessing rare and less common cancer biospecimens, as ‘we cannot understand the biological drivers of diseases or find/develop therapies that address such [diseases] without material for study.’
6.32The Ovarian Cancer Research Foundation also identified biobanks as a component of a sustainable research sector, submitting that enhanced biobanking systems would allow sufficient clinical tissue access for researchers, who can then achieve ‘relevant, translatable research’.
6.33The Victorian Cancer Biobank considered that while all cancer research can benefit from biobanking, long term support towards collecting specimens for rare and less common cancers is specifically required, as the ‘accrual of a significant cohort size that is meaningful for research may take years due to the rarity’.
6.34Dr Wayne Ng, General Manager of the Victorian Cancer Biobank, also raised the importance of coordination between biobanks to achieve sufficient cohort sizes for research:
The whole industry is starting to work quite closely in coordinating how biobanks could work with each other. Currently, it’s quite an uncoordinated way, especially interstate because there are often different jurisdictions and so on … In our case, we’re doing it at Victoria [sic] level, but hopefully this can expand. I think this is very valuable, especially in rare and less common cancers, because it's very hard to capture a sufficient cohort to even do something meaningful for research and training.
6.35However, Professor Dun claimed that biobanks are ‘critically underfunded’, which he argued impacts recruitment, participation, ease of access, as well as the quality and availability of samples. He further stressed that:
Add to this the sheer number of samples that need to be collected to provide adequate sample sizes for many studies (the low incidence of rare/less common cancers a confounding factor in this regard), the support of active and functional biobanks should be prioritised in our efforts to enhance equity of diagnosis and treatment availability.
6.36The Victorian Cancer Biobank also submitted that ‘investment in biobanking research infrastructure remains as a gap necessary to successful research of rare and less common cancers’ and called for the lack of ongoing funding for the collection of rare and less common cancers to be addressed. Further, it submitted that funding should consider leveraging existing biobanking infrastructure, which already have established patient access pathways.
Genomic research
6.37As detailed in Chapters 2 and 3 of this report, submitters highlighted the potential of genomics to improve patient outcomes.
6.38When speaking about genomic research, The Menzies Institute for Medical Research told the committee about the work they do to translate ‘genetic/genomics testing’ and in ‘accelerating the implantation of health innovation in Tasmania.’ It noted the success of their research in achieving outcomes for one patient in particular:
Our team recently identified a rare inherited clinically actionable mutation in a participant diagnosed with a high-grade prostate cancer. This participant will now be offered clinical genetics services through our research program and would qualify for access to a new targeted therapy. In the absence of our research program, this person would not have qualified for genetic testing at this time.
6.39The Leukaemia Foundation also highlighted that ‘raising our genomics capability in turn provides a powerful tool to drive further research,’ and noted that the Department of Industry, Science and Resources identified genomics as a ‘critical technology’.
Attracting and incentivising further investment from pharmaceutical companies
6.40Submitters also noted the importance of attracting pharmaceuticals to conduct research and clinical trials in Australia. Cancer Trials Australia identified Australia’s ‘research and development’ (R&D) tax incentive as a ‘key driver’ of international biotech companies choosing to conduct their clinical activities in Australia.
6.41However, Melanoma Patients Australia submitted that ‘there a number of issues’ regarding funding for rare cancers, including a ‘lack of financial incentives’ for companies to develop and bring to market new treatments or innovative technologies.
6.42Pharmaceutical company UCB Australia similarly contended that ‘there are currently no specific incentives for pharmaceutical manufacturers to prioritise Australia over other countries for new and novel therapies.’
6.43Melanoma Patients Australia signalled its support for the government to continue to provide tax incentives, as well as other financial incentives, to encourage research and the development of new technologies in Australia.
6.44As discussed in Chapter 3, the Cancer Council Australia also stressed that anecdotally, rare and less common cancer clinical trials are not attractive to research sponsors. Consequently, it reinforced the need for greater investment and incentives to undertake research and clinical trials to improve access and outcomes for people with rare and less common cancers.
6.45Professor David Thomas, Chief Executive Officer of Omico, also stressed the need for the government to work with the private sector to make Australia an attractive destination for the development of new treatments:
I would advocate, in essence, that we need an infrastructure that enables government and the private sector to work together collectively to make Australia a preferred destination for this era of drug development.
6.46Pharmaceutical companies AstraZeneca, IQVIA and Bristol-Myers Squibb Australia drew the committee’s attention to the United Kingdom’s Cancer Drugs Fund, which as discussed in Chapter 3, funds cancer therapies to assist sponsors in gathering evidence. Mr Owen Smith, Bristol-Myers Squibb Australia’s Managing Director, noted that the Cancer Drugs Fund:
… is not a perfect system, but it is a system that has … afforded a greater degree of risk sharing between the developers of the medicines and the payers in the healthcare system, and that’s a positive.
Importance of further investment in clinical trial research
6.47Whilst clinical trials were discussed in Chapter 3 in terms of access to treatment for patients, these sections will examine clinical trials in the context of future research.
6.48Cancer Australia explained that clinical trials are ‘fundamental to establishing whether new cancer treatments or new ways of using existing therapies, diagnostic tests, preventative or supportive interventions are effective.’
6.49Further, both Cancer Trials Australia and TrialHub contended that clinical trials are a critical step in the research and development of new drugs, vaccines, medical devices and diagnostics. They added:
Patients in clinical trials get early access to potentially life-saving treatments or medical interventions, while at the same time advancing medical knowledge. More broadly, clinical trial activity contributes to the development of a thriving research culture and promotes Australia's international research profile.
6.50However, Cancer Australia reported that conventional, randomised clinical trials require large patient populations to ‘detect significant effects on health outcomes.’ It stated that there are smaller patient populations to participate in trials for rare and less common cancers, which raises ‘unit costs per patient due to longer recruitment times’ and increases ‘difficulty in achieving statistically significant outcomes.’ Cancer Australia also noted that industry‑led trials tend to prioritise cancers with larger population, which leads to ‘reduced volumes of cancer-specific research available in rare and less common cancers.’
6.51The mechanisms that submitters raised to address this issue around small patient populations in clinical trials are explored below.
Encouraging collaborative trials
6.52Numerous submitters identified a need for collaborative research and clinical trials to improve both future research and patient outcomes.The NHMRC called for collaborative research between national and international parties to ‘increase the critical mass needed for research progression.’
6.53Cancer Australia also noted that patient numbers in clinical trials for rare and less common cancers can be improved via national and international collaboration, thereby improving the cost-benefit ratio for trials, as well as statistical power.
6.54Melanoma Patients Australia similarly noted that Australia’s small population base, which contributes to low clinical trial activity, can be overcome by international clinical trial collaboration.
6.55This was echoed by Ms Delaine Smith, Chief Executive Officer of the Australasian Leukaemia and Lymphoma Group (ALLG), who also outline the importance of international collaboration for clinical trials:
We have welcomed through our ALLG collaborations with major global drug companies and smaller biotech companies instances where there might be only five or six cases a year diagnosed with a particular type of cancer, a rare form of blood cancer. We can operate the sites. We can work with our member network to find those patients and give them the opportunity to be a part of those trials. This is a challenge for us generally because our numbers in Australia are so small population-wise … We need to work with international partners on those clinical trials, bringing them to Australia for the benefit of patients to get those opportunities in Australia.
6.56However, Ms Smith also considered that ‘we are not supported in building those international collaborations as well as we possibly could be’ when working to bring clinical trials to the region.
Adjusting evidence standards
6.57Bristol Myers Squibb Australia submitted that studies for rare and less common cancers are usually small, single-arm or non-comparative due to the small patient populations. Yet, it noted that phase 3 clinical trials are seen as ‘the gold standard of clinical evidence’ for the Pharmaceutical Benefits Advisory Committee (PBAC).
6.58Pharmaceutical company MSD Australia similarly raised that:
Due to the challenges associated with conducting Phase III randomised control trials (RCTs) for rare cancers, often the best available evidence often comes from early phase, single arm trials. The key challenge with single arm trials is that there is less evidence upon which the PBAC can base their assessment of comparative health gains and comparative cost‑effectiveness.
6.59Noting the inherent limitations in rare and less common cancer clinical trial evidence, Bristol Myers Squibb Australia called for a ‘more pragmatic approach’ to Australia’s Health Technology Assessment (HTA) processes, which ‘places equivalent weight and value of all available evidence used to treat rare and less common cancers.’
6.60Several submitters pointed to the United Kingdom’s National Institute for Health and Care Excellence (NICE) ‘Real-world evidence framework’ (NICE framework) as a relevant example of such an approach. For example, the Leukaemia Foundation submitted that the NICE framework:
accepts that "real-world data can improve our understanding of health and social care delivery, patient health and experiences, and the effects of interventions on patient and system outcomes in routine settings." In acknowledgment of the challenges with generating evidence for some treatments – particularly those regarding children, rare diseases and innovative treatments - NICE's committees will now be able to better manage and consider uncertainty.
6.61Relatedly, AbbVie told the committee that the United Kingdom, France and Germany have adjusted their regulatory approaches to rare disease therapies to allow for greater uncertainty in clinical evidence and broader acceptance of real-world evidence.
6.62Rare Cancers Australia argued that as more therapies ‘come to market’ with phase 2 data, there will be the need for a greater reliance on real world evidence:
We need to act and ensure systems are put in place to make this the norm, not the exception. The health system must develop and adapt so that evidence-based knowledge can be translated quickly and seamlessly into the real-world clinical setting …
Committee view
6.63The committee received compelling evidence regarding the importance of boosting research and clinical trial activity to improve outcomes for people with rare and less common cancers, including neuroendocrine cancer.
Addressing gaps in funding for research and clinical trials
6.64Evidence shows that whilst funding for research into rare and less common cancers has increased in recent years, it remains insufficient.Throughout the inquiry, the committee heard that the lack of investment in research contributes to lower survival rates for people with rare and less common cancers. The committee understands that some of the criteria to grant research funding are currently disadvantaging research applications pertaining to rare and less common cancers. The committee believes that it is paramount to address these disparities in research funding with the view to close the gap between funding and the burden of disease and mortality caused by rare and less common cancers.
6.65The committee also notes the gaps in funding for specific tumour types and understands that this leads to inequitable outcomes across diagnosis, treatment, and the survivability of these rare and less common cancers.
6.66Committee members acknowledge the recent government announcement of the 10-Year Low Survival Cancers Mission for additional research funding, however the committee also notes evidence received that gaps remain and recommends that the Australian Government ensure funding continues as a priority for research into rare and less common cancers.
6.67The committee recommends that the Australian Government ensure continued funding for rare and less common cancer projects to reduce existing research and clinical trial disparities.
Funding biobanks
6.68The committee is encouraged to hear that various existing models can be leveraged to increase research into rare and less common cancers.
6.69In particular, the committee is persuaded that research into rare and less common cancers will increase if Australia’s researchers are provided with access to stable funding. The committee discusses funding models for researchers in Chapter 5 of the report.
6.70Based on the evidence received, the committee understands that biobanks could generate sufficient cohort sizes for meaningful rare and less common cancer research and is greatly supportive of collaborative efforts to coordinate the work of biobanks across the nation. However, the committee remains concerned that biobanks are underfunded, thereby impacting the quality and availability of samples, and limiting the potential to improve diagnosis and treatment outcomes into the future.
6.71The committee recommends that the Australian Government fund biobanking initiatives to ensure the availability of quality cancer samples for research.
Genomic research
6.72The committee is encouraged to see that advances in genomic research are leading to more precise diagnoses of cancers, which includes the increased identification of rare sub-types in common cancers and the broadening of treatment options for patients. The committee notes that this is particularly important for rare and less common patients, who often have very limited treatment options available to them.
6.73Increasing funding for genomic research in Australia is paramount so patients continue to have improved and increased treatment options into the future.
Recommendation 36
6.74The committee recommends that the Australian Government explore options to provide incentives to expand genomic research in Australia.
Pharmaceutical companies
6.75The committee also notes the concerns raised by some submitters that Australia does not have enough incentives to sufficiently attract pharmaceutical companies to conduct research, innovation and clinical trials in our country.
6.76To this end, the committee believes it would be valuable for the Australian Government to consider implementing new reforms or financial incentives to attract these international companies to Australia, to stoke research and clinical trial activity in our region.
6.77The committee sees value in exploring mechanisms that have successfully attracted new investment from pharmaceutical companies in comparable jurisdictions, including models such as the United Kingdom’s Cancer Drugs Fund.
6.78The committee recommends that the Australian Government consider new mechanisms to encourage greater private sector investment in rare and less common cancer related research and clinical trial sponsorship in Australia.
Generating clinical trial activity
6.79There is a need to increase rare and less common cancer clinical trial activity in Australia to facilitate not only improved patient outcomes, but also improved research outcomes.
6.80However, the committee is cognisant of the difficulties in obtaining sufficient cohort sizes for rare and less common cancer clinical trials. For this reason, the committee is supportive of the collaboration between international and national bodies to obtain the sample sizes needed for research progression.
6.81Further, noting the inherent challenge of generating large sample sizes of patients with rare and less common cancers, the committee is convinced by the need to explore adjustments to the trial evidence standards that the Pharmaceutical Benefits Advisory Committee currently upholds when it comes to rare cancers in particular.
6.82As such, the committee believes that the Australian Government should consider adjusting its evidentiary standards, where appropriate, to account for real-world evidence and acceptance of greater uncertainty arising from rare and less common cancer clinical trials. The committee notes that comparable jurisdictions, including the United Kingdom, have implemented similar changes to their Health Technology Assessment processes.
Recommendation 38
6.83The committee recommends that the Australian Government review its evidentiary standards specifically for rare and less common cancer clinical trials to consider accepting real-world evidence and greater uncertainty in data where appropriate.