Approach 1: Networking and collaboration. How to find your allies and experts.

This approach includes the following key tools:

  1. The World Bank’s Community of Practice Toolkit
  2. Rare Voices Australia’s Policy page
  3. Partnership/collaboration checklist
  4. Considerations when planning collaborations with people living with a rare disease
  5. Involve Australia’s Guidelines for Community Involvement in Genomic Research
  6. Medicines Australia’s Working Together Guide
  7. Steps to evaluate and improve collaboration

As rare diseases are so rare, people living with a rare disease may feel isolated and alone. While there are many different rare diseases, they share countless commonalities. The Action Plan highlights that due to the unique challenges of living with a rare disease, individuals and organisations in the rare disease sector need to work together.

There are many types of collaborations and partnerships.

In a formal partnership between two or more organisations, there is shared understanding and typically a written agreement between the organisations making the roles and responsibilities of each organisation clear. For example, RVA partners with over 100 rare disease-specific organisations.

A community of practice is a group of people who come together regularly to work towards common goals. These are particularly helpful in rare disease, where knowledge may be scarce. A shared forum can help fill gaps and solve problems that cannot be resolved in isolation. For example, the Australian Rare Disease Organisations Community of Practice (ARDO-CoP) and the Rare Disease Project ECHO® Clinical Community of Learning Practice, formed as part of the RArEST Project.

If you’ve got other people around you that are trying to do similar things … there will be opportunities for us to continue to work together on specific issues. And that’s really powerful.”

RArEST Australian Rare Disease Organisations Community of Practice (ARDO-CoP) member

Networks and alliances across organisations and/or countries are particularly important for rare diseases, since each rare disease typically has a small number of patients, families, and experts. For example, Rare Diseases International’s Global Network for Rare Diseases and Genomics in the Community.

The World Bank’s Community of Practice Toolkit provides guidance and resources for setting up and running a more formal community of practice.

Panels, boards, and working groups can be established to address specific challenges, share expertise, or guide policy and practice for rare diseases. Clinicians may be invited to join boards to provide clinical expertise, and individuals may be asked to provide lived experience and personal insights into the priorities of people living with a rare disease.

Formal consultations are usually initiated by governments, regulatory bodies, or international organisations. In a formal consultation, a range of individuals and organisations are asked to provide their input.

The Policy page on RVA’s website provides several sample submissions for government consultations.

Research partnerships are partnerships between groups conducting research into rare diseases and one or more individuals or organisations within the rare disease sector, for example, a peak body, rare disease organisation, university, research institute, hospital, health care network, or biotechnology company.

RVA’s Research Partnership Guidelines show how RVA ensures that research partnerships are aligned with an organisation’s policies and goals. Other organisations may consider developing similar guidelines and processes.

The formal collaborations formed between the Australian Government, RVA, their partner organisations and people living with a rare disease helped shape the Action Plan and ensure the priorities of all groups were heard and incorporated.

“Working in partnership with Australians living with a rare disease was key and helped identify the goals and actions of the Action Plan.”

Rare Voices Australia

Many partnerships and collaborations are cross-sectoral, that is, they bring together individuals and organisations from different parts of society.

For example, a collaboration may be between the government, health services, or non-government organisations, including rare disease groups/organisations. Cross-sectoral or multi-sectoral collaborations aim to incorporate a wide range of experiences, opinions and expertise into policies, services, and resources.

When considering starting a partnership or collaboration, the following checklist may be helpful.

Partnership/collaboration checklist

  • Does your potential partner’s vision and priorities align with those outlined in the Action Plan?
  • Does their mission align with yours?
  • What aspect of rare diseases do they address? (for example, clinical, policy, other advocacy)
  • What expertise are they offering?
  • Is the collaborator part of your organisation or another one?
  • Are there any risks?
  • Are there any conflicts of interest? If so, how will these conflicts of interest be managed?

Including people with lived experience of a rare disease and their voices in all aspects of health care, including policy development, research, system design, the development of education materials, and care planning, has numerous benefits.29 These include ensuring health care is person-centred and efficient, resources are applicable and useful, and research is aligned with the needs of the rare disease community.

Rare disease groups/organisations often work beyond capacity and are staffed by volunteers who may be living with a rare disease or caring for someone who has a rare disease. It is recommended that people’s capacity, health literacy, accessibility needs, and practical constraints are considered when asking for input.

Networking to unify and amplify the voice of lived experience

In each of the four case studies, partnerships with people living with a rare disease helped inform the goals and priorities of the advocacy.

People living with a rare disease/rare disease groups/organisations and are often involved in:

  • Leading or chairing a committee or working party
  • Presenting training or education
  • Designing or conducting research projects
  • Developing and reviewing, strategy, research, or resources
  • Selection panels and grant review panels
  • Focus groups or interviews.

It is important to be aware of and remove any barriers to participation for people living with a rare disease and their families/carers. This will involve discussion with each individual, but typically would include considerations such as making the timing of meetings family-friendly, ensuring interactions with other stakeholders feel welcoming and safe, ensuring the meeting spaces are accessible, or holding meetings virtually so travel is not required. Consumer remuneration may be a potential way to remove barriers. For example, Health Consumers NSW suggest appropriate rates according to the level of consumer engagement or involvement.

Considerations when planning collaborations with people living with a rare disease

  • Are the people living with a rare disease representing their whole community, or just their individual experience?
  • Is it better to seek a diverse range of individual perspectives, or work with representatives from organisations who already represent a wide range of individuals?
  • If working with individuals, how diverse is the group and how can you support diverse representation?
  • Are there specific access needs for those with communication difference and how can those be addressed?
  • If working with groups/organisations, how do they seek and reflect the perspectives of those they represent, including priority populations?
  • Is there any additional training or support that may be helpful for the individual or group and how could that be provided?

Involve Australia’s Guidelines for Community Involvement in Genomic Research provides genomic researchers in Australia with practical information for involving community members effectively and meaningfully in research projects.

It is important to include all groups in the rare disease community. This includes priority populations such as:
• People with a disability
• Aboriginal and Torres Strait Islander people
• People from culturally and linguistically diverse backgrounds
• People living with an undiagnosed rare disease
• People experiencing socio-economic disadvantage
• People from regional, rural, and remote regions.

It may not always be possible to directly engage with members of all priority populations. In this case, consider collaborating with the peak bodies representing these communities. This is particularly important for Aboriginal and Torres Strait Islander people, and culturally and linguistically diverse communities, where cultural safety is critical. Peak bodies and health advocacy organisations with broad representation include the Consumers Health Forum of Australia, the Australian Multicultural Health Collaborative, the National Rural Health Alliance and National Aboriginal Community Controlled Health Organisation (NACCHO).

The RVA Education webinar Developing the Rare Disease Resources Collections for Priority Populations provides insights into developing resources for people living with a rare disease from priority populations, including working with community and peak bodies who have established relationships and trust with priority populations.

All advocacy benefits from the involvement of credible and expert scientific and medical advisers.25 Scientific and medical advisers can be involved in many different ways. Examples include helping to ensure information and educational resources are accurate and factual, assisting with submissions, or gaining the support of their colleagues and institutions to drive change in their own settings.

Collaborating isn’t just about building relationships – it helps to get things done.

“With many people and groups involved they each contribute a few pieces of the puzzle – it can’t be done alone.”

Clinician, Sydney Children’s Hospitals Network

Many pharmaceutical and biotechnology companies fund basic and clinical research into rare diseases. They may also fund support programs for people living with a rare disease, educational programs for health care professionals, or projects to build health literacy among people living with a rare disease.

Working with these companies can provide much-needed financial support, however, it is important to ensure that priorities align, and there is mutual benefit for all involved. Chapter 6 in RVA’s Engaged, Ethical and Effective: A Guide for Rare Disease Organisations provides additional insights and advice on grant funding and sponsorship from the pharmaceutical and biotechnology industry.

Medicines Australia’s Working Together Guide provides guidance on how the pharmaceutical and biotechnology industry can work with consumer groups.

A valuable and productive collaboration between a rare disease group/organisation and a pharmaceutical/biotechnology company requires a strong relationship that is based on core principles that include:30

  1. Respect for independence
  2. Achieving and maintaining public trust
  3. Open communication
  4. Confidentiality
  5. Accountability
  6. Successful collaborations that focus on the health consumer and their carers
  7. Management of conflicts of interest.

The steps outlined in the tool below may help with evaluating and improving collaborations.31

Steps to evaluate and improve collaboration

Step 1 – Find a common purpose

  • Partners have compared their respective organisation’s goals and advocacy objectives
  • All partners are aware of their part to play

Step 2 – Increase collaborative literacy

  • Partners have a shared understanding that collaboration is vital to the success of their work and that each partner has equally valuable expertise to offer

Step 3 – Knowing who is ‘in the tent’

  • Partners know who their partners are and why they have those particular partners

Step 4 – Monitor development

  • Partners monitor the extent to which their partnership has formed, developed, and transformed over time.

Step 5 – Assess the strength of the partnership

  • Partners are aware of the current and potential future strength of the partnership
  • Decisions are made about continuing the strength of the partnership or how to strengthen the partnership

Step 6 – Assess collaboration on an ongoing basis 

  • Partners regularly assess if others are still invested in, and working towards, a common purpose
  • The quality of the collaborations, for example decision-making processes and ensuring partnerships are equal, authentic, and productive, has been considered

Adapted from the Handbook on Planning, Evaluating, and Improving Collaboration for Oral Health Programs.31

It keeps networks connected and maintains relationships

“[we brought] together stakeholders regularly to update and share learnings and education.”

Clinician, SCHN

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