Rare Cancers and Rare Diseases - Research Grants

Page last updated: 24 August 2017

PDF Version Rare Cancers and Rare Diseases - Research Grants (PDF 427 KB)


The Australian Government will invest $13 million to stimulate clinical trial and registry activity with priority to be given to under-researched health priorities, such as rare cancers and rare diseases.

Key elements

This competitive grant program will target public good clinical trials and registries that address areas of health burden and unmet need. Novel and innovative trial designs and recruitment strategies and the application of precision medicine will also be encouraged.


  • Clinical trials benefit patients, improve health outcomes, advance medical knowledge and are a significant contributor to the innovation economy.
  • Clinical quality registries are an increasing feature of complex trials – they allow deep data analysis, trend identification, drive improvements in practice, and illuminate new areas for research.
  • Clinical trial public investment is roughly proportionate to the burden of disease; meaning that less common low survival rare cancers and rare diseases can need further investment and investigation.
  • Public good trials activity increases opportunities for patient therapy, strengthens the operational systems that support trials, and grows workforce capacity.
  • The Australian Medical Research Advisory Board acknowledges in their Strategy that the MRFF has a role to play in facilitating non-commercial clinical trials of potential significance.

Desired outcomes

  • New opportunities for those suffering from rare cancers and rare diseases to participate and benefit from the latest research.
  • Attention given to under researched health priorities and conditions.
  • Deployment of innovative trial designs and recruitment strategies.
  • Purposeful health service engagement to improve the translation of research into practice and improve outcomes for patients.
  • New health treatments, drugs and devices to improve health.
  • Reinforcement of Australia’s position as a preferred destination for clinical trials.

Key facts

  • Despite having low incidence compared to the most common cancers, cancers with low survival rates have a high disease burden.
  • Rare cancers with low survival rates together accounted for 47 per cent of all cancer deaths in 2014.
  • While survival rates for high incidence cancers have improved, those for rare cancers have remained relatively static.
  • It is estimated that brain cancer will be the leading cause of death from cancer for people aged 0-24 years and for males aged 30 39 years in 2017.
  • People living with a rare disease face significant challenges including diagnostic delays, lack of available treatments and difficulty in finding the appropriate care.
  • Clinical trials are research investigations that evaluate whether a medical strategy, treatment, drug or device is safe and effective for humans.
  • Clinical trials provide the evidence to inform best practice. They also offer participants opportunities to benefit from the latest medical research and provide legitimate alternative therapy options.
  • For every dollar invested in clinician-driven clinical trials in Australia, an average of $5.80 is returned in health benefit.
  • Innovative clinical trial design methods can overcome barriers to recruitment (including for regional Australians), and enable adaptive platforms with agile treatment protocols.
  • Precision medicine holds the potential to transform medicine – it changes the paradigm to a treatment that is tailored to the individual and the exact nature of their disease.
  • This investment strategically builds on the Government’s 2016 $7 million Encouraging more clinical trials in Australia initiative that will streamline trial operations to improve start-up, navigation and recruitment efforts in each state and territory.


  • The National Health and Medical Research Council (NHMRC) is administering this grant round.
  • Applications consistent with the program guidelines will be assessed using NHMRC peer review processes and procedures.
  • Further details on the opportunity can be found at GrantConnect.

Medical Research Future Fund

The Medical Research Future Fund (MRFF) demonstrates the Government’s strong commitment to research and innovation. The MRFF provides a long term sustainable source of funding for endeavours that aim to improve health outcomes, quality of life and health system sustainability.

The MRFF will double direct Australian Government funding for health and medical research.

The MRFF is a dedicated vehicle for priority investment in health and medical research.

The MRFF, established under the Medical Research Future Fund Act 2015, provides grants of financial assistance to support health and medical research and innovation in improving the health and wellbeing of Australians. It operates as an endowment fund with the capital preserved in perpetuity. At maturity, the MRFF will reach $20 billion.

The first disbursements from the MRFF focus on translating research into real health benefits, breakthrough investments in new technologies and challenges, and enhancing Australia’s reputation for research excellence and leadership.

Over $65 million will be injected into a range of programs that cut across the research pipeline – fuelling new discoveries and the translation and commercialisation of great Australian ideas.

Advisory Board

The Australian Medical Research Advisory Board, established in accordance with the MRFF Act, is responsible for consulting on and developing a five-yearly Strategy that sets out the vision, aims and objectives for the MRFF and an accompanying two-yearly list of Priorities. The Government must consider the Strategy and Priorities in making MRFF investment decisions.

Strategy 2016-2021

Vision: A health system fully informed by quality health and medical research.

Identifies key strategic investment platforms that set to position Australia to meet future healthcare challenges:
  • Strategic and international horizons
  • Data and infrastructure
  • Health services and systems research
  • Capacity and collaboration
  • Trials and translation
  • Commercialisation

Priorities 2016-2018

The first MRFF disbursements address identified Priorities under each strategic platform, are a mix of strategic investments, and use a range of administrative mechanisms to maximise impact. More disbursements will occur in 2017-18.

In this section