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

What is networking and collaboration, and why is it so important in rare disease advocacy?

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.

Types of collaborations and partnerships in the rare disease sector

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.

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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.

How to seek out and establish collaborations and partnerships

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

Collaborating with people with lived experience

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.

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Networking to unify and amplify the voice of lived experience

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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.

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.

To learn more about collaborating with people with lived experience, see Approach 3: Co-design.

Collaborating with scientific and medical experts

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.

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Collaborating with the pharmaceutical and biotechnology industry

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

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

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