The Australian Government’s National Strategic Action Plan for Rare Diseases (the Action Plan), was launched in 2020. The Action Plan is the first nationally co-ordinated effort to address rare disease in Australia.13
RVA led the collaborative development of the Action Plan, which outlines the priorities, actions, and implementation steps required to achieve the best possible health and wellbeing outcomes for Australians living with a rare disease.
This Toolkit aligns with the Action Plan, which is centred around three interrelated pillars, as shown in Figure 2, below. More information about the Action Plan can be found at RVA’s website.
The Action Plan highlights the importance and value of involving and engaging many different people and organisations (stakeholders) in actions and activities to improve health outcomes and experiences of Australians living with rare disease. Rare disease stakeholders include:
- People living with a rare disease, including those with an undiagnosed rare disease, families and carers
- Consumer-led organisations, peak bodies, and charities
- Health professionals
- Academics, researchers, and research funders
- Governments and policy makers
- Biotechnology and pharmaceutical companies.
These different key stakeholders make up the rare disease sector (Figure 3). More details about these groups can be found in the Toolkit companion document Who’s who: the people and organisations involved in the Australian rare disease sector
What the Toolkit is for
This Toolkit demonstrates how the Australian rare disease sector has worked effectively together to advocate (speak up) for systemic change benefitting the rare disease community. Systemic changes are those that result in changes to the systems involved in providing care and support to people living with a rare disease.
The Toolkit describes key approaches that people and organisations have taken that resulted in effective systemic advocacy and positive change for Australians living with a rare disease.
These changes have improved the health and wellbeing of Australians living with rare disease by enhancing:
- awareness and education,
- care and support, and
- research and data.
Effective approaches are ones that gain and maintain the trust of all stakeholders, and lead to a common desired outcome. Trust among all members of the rare disease sector is important, and gaining and maintaining the trust of the rare disease community is crucial to effective systemic advocacy and positive change.
The Toolkit is designed to help people:
• Learn about approaches that can help to bring about systemic change
• Plan their own approaches and next steps.
Although the rare disease sector extends beyond health to the disability, social and welfare sectors, education, employment, housing and other areas, this Toolkit primarily focuses on the health system, including health policy at state and federal levels, medical and health research, clinical trials, access to therapies and clinical care, and health service provision.
Who the Toolkit is for
To help address the challenges faced by people living with a rare disease, the Toolkit provides inspiration, resources, practical tools and links to training and education to help the entire rare disease sector advocate more effectively to achieve the best possible health and wellbeing outcomes for people living with a rare disease.
How to use this Toolkit
Section 2 includes case studies provided by rare disease organisations and rare disease advocates which are designed to showcase how different stakeholders have worked together to bring about systemic change.
These case studies highlight four key approaches commonly used by stakeholders to achieve systemic change in rare disease (Table 1). The intention is that you can read these case studies to gain inspiration and insight into approaches that have been effective.
Then, in Section 3, each approach is described in more detail. Accompanying tools are provided that could be helpful for individuals and organisations considering how to apply these approaches to their own rare disease advocacy.
Table 1. The key approaches for effective systemic advocacy described in this Toolkit
 | Networking and collaboration | How to find your allies and experts |
 | Communication and storytelling | How to create clear messaging and share personal stories safely |
 | Co-design | How to apply co-design to ensure the needs and priorities of all rare disease stakeholders are included |
 | Patience, persistence, and flexibility | How to keep going despite the uncertainty and challenges – rare disease challenges are complex and often require complex solutions |
The icons below are used throughout the Toolkit:
 | Rare disease-specific tools |
 | Links to more information and practical tools |
 | Quotes from the rare disease community |
 | Key messages |
How the Toolkit was developed
This Toolkit was developed as part of the Australian Government-funded Rare Disease Awareness, Education, Support and Training (RArEST) Project. The RArEST Project is a partnership between RVA, the University of New South Wales (UNSW Sydney), Macquarie University, and the University of Western Australia.
The Toolkit was co-developed with the rare disease sector, including people living with a rare disease, leaders of rare disease organisations, health professionals, and RVA as the national peak body for Australians living with a rare disease. More information about the development process and the frameworks that guided the Toolkit development can be found in the Toolkit companion document How the Toolkit was developed.
