Evaluation of the Medical Research Future Fund Clinical Trials Activity Final Report


The Health and Medical Research Office’s (HMRO) Performance and Evaluation Section within the Department of Health and Aged Care (the Department) recently asked Rare Voices Australia (RVA) to share their final report on the evaluation of the Medical Research Future Fund Clinical Trials Activity (MRFF-CTA) grant opportunity (Evaluation Report) with the rare disease sector. The report was prepared by the Institute for Evidence-Based Healthcare, Bond University Australia.

As the national peak body for Australians living with a rare disease, RVA’s work is non-disease specific and is based on the commonalities of rare disease. Our advocacy focuses on rare disease policy, processes and systems. RVA advocates for all rare disease communities.

RVA commends the Department for commissioning this work and encourages any evaluation of the use of health and medical research funding in Australia. The Evaluation Report highlights several important strengths and gaps in how MRFF-funded clinical trials are conducted and how they perform against national and international comparators. However, important gaps in clinical trials for rare disease were not identified in the Evaluation Report. Both RVA and the HMRO recognise that rare disease research is not being funded to the extent anticipated. These gaps are discussed in detail below.

Download the Evaluation of the MRFF-CTA Final Report at the Department Health and Aged Care’s website.

Rare Voices Australia’s Response to the Evaluation Report

RVA wrote a letter to the Department to encourage any future evaluation of funding for rare disease research to include broader stakeholder consultation, including consultation with RVA as the national peak body for Australians living with a rare disease and RVA’s Scientific and Medical Advisory Committee. RVA and the HMRO recognise that few rare disease clinical trials have been selected for funding under the MRFF-CTA grant opportunity to date. RVA is in ongoing discussions with the HMRO to identify ways to facilitate greater investment in rare disease research, including clinical trials.

The MRFF’s commitment to priority-driven research has been transformative for rare disease. The explicit and intended focus of rare disease in particular grant rounds are a welcome change for rare disease, where knowledge is limited, and treatments are scarce. As acknowledged in the Australian Government’s National Strategic Action Plan for Rare Diseases (the Action Plan), for many people living with a rare disease, participation in a clinical trial may be the only way to access treatment. This reality emphasises the considerable need for greater investment in clinical trials to contribute towards achieving the Action Plan’s vision: the best possible health and wellbeing outcomes for Australians living with a rare disease.1

While RVA acknowledges the intentions to fund rare disease research, and the explicit mention of rare disease in grant names, it is clear—from RVA’s experience, grants selected for funding and now evidenced in the Evaluation Report—there remain few rare disease research studies selected for funding through the MRFF-CTA grant scheme. This claim is evidenced by the following:

  • Clinical trials addressing rare disease were not mentioned as part of the collective conditions studied under MRFF-funded clinical trials. This observation excludes rare cancers, which were noted as having the most trials funded under the MRFF-CTA grant scheme in the Evaluation Report.
  • There was no mention in the Evaluation Report about the need to encourage unique trial designs for rare disease. For example, n of 1 clinical trials and decentralised trials, which are vital to enabling access to life saving treatments where sample sizes are inherently few and geographically spread and patient populations are heterogenous. Whilst larger cohorts and conventional trial designs are important for statistical significance, they are not fit-for-purpose for rare disease due to small patient numbers. Trial designs that enable longer recruitment times could increase patient numbers; however, as mentioned in the results of the Evaluation Report, the limited duration of funding was a significant barrier to clinical translation and impact reporting for MRFF-funded trials.

The barriers and enablers to clinical trials identified in the Evaluation Report are relevant to rare diseases, including the duration of grant funding, workforce limitations, site governance and approval processes and patient recruitment. However, there are several barriers to clinical trials that are compounded in rare disease, including expectations around more conventional trial designs due to inherently small and geographically spread patient cohorts, and the expectation that verified Standardised Outcome Sets for rare diseases exist.

The Australian Government’s Action Plan addresses the importance of enabling clinical trials for rare disease in Australia.

Note: this article was updated on 31 August 2023.


  1. Commonwealth of Australia. Department of Health. National Strategic Action Plan for Rare Diseases. Canberra; 2020. 63 p. Available from: https://www.health.gov.au/sites/default/files/documents/2020/03/national-strategic-action-plan-for-rare-diseases.pdf