Melbourne School of Population and Global Health - Theses
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ItemThe cure depends on us: the role of Australian patient organisations in rare disease research( 2015)In this thesis, I explore how Australian patient organisations devoted to specific rare diseases are involved in biomedical research. As with patient organisations for more common diseases, many rare disease patient organisations (RDPOs) have broadened their historical focus on self-help and now seek to advance research on “their” diseases. RDPOs are thought to have special opportunities to influence researchers because they can provide resources – such as funding, study participants, and disease-related knowledge – which are often scarce in rare disease research. RDPOs’ involvement in research is important to theorists concerned with the “public shaping” of science, but it also has immense practical significance. Collectively, rare diseases are estimated to affect six to eight per cent of the population; and most are life-threatening or chronically disabling conditions with no specific or effective treatments. The need to accelerate therapy development, and the absence of government or industry-funded research for many rare conditions, underpins growing international interest in strengthening collaborations between RDPOs and researchers. In the first study of RDPOs in Australia, I reviewed 112 RDPO websites, conducted an online survey completed by 61 RDPO leaders, and interviewed ten RDPO leaders and two key informants. Consistent with international literature and empirical case studies, I found that RDPOs are highly motivated to support research. However, while some Australian RDPOs have helped to advance the understanding and treatment of their diseases, I argue that RDPOs are far from the robust challengers of the research establishment portrayed in the literature. In fact, RDPO leaders face considerable difficulties in upholding organisational interests in their various forms of engagement with academic and industry researchers. For example, leaders may struggle to direct RDPO funding in ways which best meet the goals of the organisation; they may lack the expertise or power to assert their knowledge and ideas when collaborating with researchers; and they may run the risk of real or perceived conflicts of interest in financial relationships with pharmaceutical companies. These problems are related to RDPOs’ limited funds and capabilities, the competing interests of researchers, and the marginalisation of rare diseases in Australian health and research policy. I argue that my study provides a more realistic picture of the challenges faced by “ordinary” RDPOs than previous studies focusing on the successes of large, well-resourced organisations and groups with highly driven, entrepreneurial leaders. Despite the difficulties I identify, I contend that RDPOs have much to contribute to research. They can also play an important role in providing their members with information about biomedical research, including the risks and benefits of participating in mainstream and novel forms of research – such as participant-led clinical trials and crowd-sourced health studies. I suggest that there are considerable opportunities in Australia, building on the findings of this study and international initiatives, to support and safeguard RDPOs’ involvement in research, for the benefit of Australian research and people affected by rare diseases.