Advocacy in rare disease

Why is advocacy important in rare disease?

Change benefitting Australians living with a rare disease is often driven by effective advocacy undertaken in response to issues, needs or gaps. There is great complexity and unmet need in rare disease, and Australia’s health and other systems are often not fit for purpose for rare disease.  

Even though our organisation is small, fairly new and runs completely with volunteering members, we have strived to access opportunities because we see a need and many gaps in many areas of our systems.

Rare disease organisation leader

Getting involved in advocacy

In the case studies about Action Plan development and the legalisation of mitochondrial donation, both RVA and the Mito Foundation drove positive change by co-ordinating and supporting collective advocacy to the Australian Government. However, effective advocacy can happen in many other ways.

Most people in rare disease advocacy become involved through need rather than choice. Advocacy is often driven by recognising a gap or unmet need and then exploring ways to address these gaps. Examples include:

  • A parent seeing a promising clinical trial overseas and advocating for a site in Australia
  • A person seeing that treatments for their condition are available overseas and advocating for access to this treatment for themselves and their rare disease community
  • A clinician wanting to improve clinical care and have access to the latest diagnostics
  • A person wanting to speak up about their rights to obtain appropriate, timely, and accessible health care, in line with the Australian Charter of Healthcare Rights
  • A health professional, passionate about delivering high quality holistic care, wanting to write a submission for an enquiry into accessible disability services.

Potential challenges in rare disease advocacy

Every vulnerable community gets tired of advocating and asking to be respected, but really, the responsibilities need to start to shift toward the sector to understand why that respect is not there in the first place.

Person living with a rare disease

Living with a rare disease requires resilience, tenacity, pragmatism, and optimism, as does engaging in rare disease advocacy.25 There are unique challenges in rare disease advocacy, as highlighted in RVA’s Engaged, Ethical and Effective: A Guide For Rare Disease Organisation Leaders in Australia.25

These challenges, which also apply to advocates who are health professionals, researchers, and other rare disease stakeholders, include:

  • Lack of data/evidence
  • Lack of knowledge and awareness
  • Lack of resources
  • The need to learn ‘on-the-go’
  • Health and support systems that are complex and fragmented
  • Small number of people with the rare disease.

Rare Voices Australia’s Engaged, Ethical and Effective: A Guide For Rare Disease Organisation Leaders in Australia is a useful tool for rare disease groups/organisations. It contains resources and checklists, including advice on advocacy and community engagement.

Advocating for changes to Government policy

Government policy is the basic agreed principles by which government is guided.26 Government policies can have a significant impact on the rare disease sector, for example, by determining the funding and operation of the health services caring for people living with a rare disease.

The Action Plan is an Australian Government policy framework and the key policy framework for rare disease in Australia. Generally, a policy framework provides an overarching structure that guides how policies and procedures will be developed, approved, communicated and reviewed.27 Policy frameworks also communicate a course of action for a particular issue or group of people.

Aligning advocacy for changes in Government policy with the Action Plan is criticial to ensure effective implementation.

A wide range of Australian Government policies relate to rare disease. These include:

  • Health technology assessments
  • Medical research funding
  • Data generation and management
  • Genomic testing
  • Newborn screening
  • Hiring and training the health workforce
  • Management of chronic conditions
  • Hospital funding
  • Disability
  • Education and employment.

To advocate effectively, it is important to first understand these different policy areas, the relevant legislation, policies and processes, and key decision makers. As Australia has a federated health system, understanding the different responsibilities of each level of government (State and Federal) is essential to targeted and effective advocacy.

Australia’s health system is made up of both public and private service providers. All levels of government, non-government organisations, health insurers, individuals (via philanthropic funding) can fund health service research, design, delivery, and evaluation. More information about the Australian health care system can be found in the Toolkit companion document How the Health Care System Works in Australia.

Tips and strategies for effective advocacy

As the national peak body for Australians living with a rare disease, RVA offers guidance to groups/organisations on effective rare disease advocacy. RVA acknowledges that there are many different advocacy styles and approaches.25

As most rare disease advocacy is likely to be ongoing and have multiple strategic and long-term goals, RVA recommends placing strong relationships at the centre of any advocacy approach. Although public aggressive approaches that ambush people may result in quick wins, they may jeopardise future advocacy. Robust discussions, which are sometimes necessary, are always best engaged in privately and in an appropriate manner. Fostering trust and credibility with key stakeholders and decision makers are key to securing meetings.

RVA has several useful tools to help groups/organisations get started on their advocacy journey. More resources to help with advocacy are highlighted at the bottom of this section.

Rare Voices Australia’s recommended advocacy style25

  • Polite persistence as a starting point
  • Relationship based
  • Robust conversations, when needed, held privately
  • Solutions-focused/pragmatic
  • Evidence-based
  • Collaborative and dependent on building and maintaining relationships
  • Credible and independent
  • Make time to understand and leverage unique positioning
  • Person-centred with a multi-stakeholder approach
  • A balance of long- and short-term approaches

Advocacy Self-Evaluation. This was written for groups/organisations but could be adapted for individual advocacy or the work of informal alliances.

Identify your advocacy strengths and development areas using this self evaluation tool. Tick each box that applies to your organisation.

  • Advocacy is aligned with the strategic objectives of the organisation.
  • Advocacy reflects the priorities of the community.
  • Advocacy is informed by a comprehensive understanding of the policies, frameworks and decision makers that influence key advocacy issues.
  • Evidence and data to support advocacy positions has been collected from reputable sources with the relevant expertise (for example, scientific and medical advisory committees, clinicians).
  • Other organisations that are advocating on similar issues have been identified, including opportunities to work together.
  • Consideration has been given to the advocacy style that is most appropriate for the organisation’s goals (for example, long-term, relationship based or single issue, quick wins).
  • Advocacy is informed by the values and ethical principles of the organisation.

Reproduced from Engaged, Ethical and Effective: A Guide For Rare Disease Organisation Leaders in Australia with permission from RVA.25

Tips and strategies to help ensure advocacy is strategic, targeted, and credible

Have a clear objective/s Be informed Identify a suitable advocacy style Follow up
• What is/are the problems/issues?

• What action/ resolution are you seeking?
• Who can influence this issue?

• Are there policies or legislation that are relevant?

• What evidence demonstrates the problem and supports proposed solutions?

• Are there others with similar interests I should work with?

• What are some potential objections/ barriers to my request?
• Authentic styles, aligned with your organisation’s strategic intent and values that take into account the complexity of the issue and the number of stakeholders you may need to influence, will be most effectiveRepeatedly, persistently,
Reproduced from Engaged, Ethical and Effective: A Guide For Rare Disease Organisation Leaders in Australia with permission from RVA.25

Aligning advocacy with the National Strategic Action Plan for Rare Diseases

It is helpful to develop an advocacy strategy that aligns with the messaging and language in the Action Plan. This helps the rare disease sector to communicate a consistent and unified message which can strengthen all messages. RVA’s resource Amplifying Advocacy Using the National Strategic Action Plan for Rare Diseases provides information, case studies and examples to show how the Action Plan can be used for advocacy. The template below is an excerpt from this guide and provides examples of specific advocacy activities that can be supported by using the Action Plan.

Ways to use the Action Plan in advocacy activitiesUpcoming activity
Submissions into upcoming inquiries and Government consultationsExample: submission lodged for the Parliamentary Inquiry into approval processes for new drugs and novel medical technologies in Australia
When writing to Members of Parliament at State and Federal levelExample: write to my local member of Parliament to request a meeting to discuss my community’s rare disease
When writing funding/sponsorship submissions, project proposals, and policy submissionsExample: RVA referring to the Action Plan when requesting support from the RVA Round Table of Companies
Communicating with your community about your organisation’s goals and prioritiesExample: in media releases, social media posters, e-newsletters etc
When providing consumer comments for health technology assessment processesExample: when submitting comments to PBAC consumer comments
Reproduced from Amplifying Advocacy Using the National Strategic Action Plan for Rare Diseases with permission from RVA.28
PBAC, Pharmaceutical Benefits Advisory Committee; RVA, Rare Voices Australia.

Further reading