In Australia, it is estimated that more than 40,000 Australians are diagnosed with a rare or less common form of cancer and for many, support options are minimal with a lack of evidence-based information to inform treatment options and support networks1.
Rare diseases are life-threatening or chronically debilitating disorders/conditions uncommon in the general population. Rare diseases typically exhibit a high level of symptom complexity leading to diagnostic delays and require frequent, ongoing multidisciplinary care and treatment2. To date, more than 10,000 rare diseases have been identified and approximately 8 per cent of Australians live with a rare disease3.
Unmet medical need arises where members of the Australian community are living with a serious (debilitating or life-threatening) health condition where there are no satisfactory options for prevention, diagnosis, early detection or treatment of the condition that would support improved health outcomes. In addition, an unmet medical need could arise where there has been little or no progress in the development of tools or therapies to address the condition.
The objectives and intended outcome of this grant opportunity are aligned with the following Australian Medical Research and Innovation Priorities 2020-2022:
- Clinician Researcher Capability
- Comparative Effectiveness Research
Consistent with the Medical Research Future Fund Act 2015, the objective of this grant opportunity is to provide grants of financial assistance to support Australian medical research and medical innovation projects that:
- Stream 1: conduct a single clinical trial in women of potential new treatments and therapies for rare cancers of the reproductive system
- Stream 2: conduct a single clinical trial of potential new drugs for treatment refractory, low survival breast cancer in women who have been identified through molecular testing
- Stream 3: conduct a single clinical trial of potential new treatments and therapies for rare diseases caused by genetic mutations
- Stream 4: conduct a single clinical trial of non-drug treatments and therapies (for example surgery, radiation, rehabilitation) for rare cancers, rare diseases and conditions with unmet medical need
- Stream 5: conduct a single clinical trial assessing the comparative effectiveness of health interventions in improving outcomes for individuals with rare cancers, rare diseases and conditions with unmet medical need
- Stream 6: conduct a single clinical trial of new treatments and therapies for individuals with low survival cancers and low survival rare diseases
- Stream 7: conduct a single clinical trial of treatments and therapies for conditions with unmet medical need.
Please note that Stream 2 has a dedicated application form in Sapphire and earlier Minimum Data and Closing dates.
The Clinical Trials Activity - Rare Cancers, Rare Diseases and Unmet Need (RCRDUN) Initiative aims to enable or support:
- New opportunities for clinical trial participation and associated benefits from accessing the latest research by members of the Australian community living with a medical condition for which there are limited effective treatment options.
- Attention given to under-researched health priorities and conditions, such as rare cancers and rare diseases.
- Deployment of innovative trial designs and recruitment strategies to provide high-quality evidence of the efficacy of new health treatments, drugs and devices.
- Purposeful health service engagement to improve the translation of research into practice and improve outcomes for patients.
- Implementation of new health treatments, drugs and devices to improve health and wellbeing.
- Reinforcement of Australia’s position as a preferred destination for clinical trials.
Further information on the rationale of the Initiative is available on the Department of Health website.
Further information on the Medical Research Future Fund (MRFF) is available from the
Department of Health’s MRFF website.
2 Anderson M, Elliott EJ, Zurynski YA. Australian families living with rare disease: Experiences of diagnosis, health services use and needs for psychosocial support. Orphanet J Rare Dis. 2013;8:22
3 Elliott EJ and Zurynski YA. Rare diseases are a ‘common’ problem for clinicians. AFP. 2015;44:9
4 Comparative Effectiveness Research (CER) is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The purpose of CER is to assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve health care at both the individual and population levels.