Chasing cures: Rewards and risks for rare disease patient organisations involved in research

Abstract

Rare disease patient organisations (RDPOs) provide funding, logistic support and ideas for biomedical research. The literature, however, largely consists of case studies of successful RDPOs, and theoretical analyses of their potential influence on biomedical science. RDPOs’ involvement is portrayed as an example of patient empowerment and democratisation of research that allows some societal groups to challenge researchers’ traditional control of research agendas. Little is known about whether this optimistic view reflects common experiences of RDPOs or how research involvement impacts on RDPOs themselves. In our mixed methods study of a broad group of Australian RDPOs, organisational leaders identified psychological and practical benefits of supporting research but, in contrast with prevailing accounts, often struggled to uphold organisational and personal interests when engaging with researchers. Leaders reported difficulty fundraising for research and allocating funds in ways likely to support the RDPOs’ goals. They had concerns about promoting ‘false hope’ and noted conflicts between researchers’ and patients’ interests. Some were disillusioned with research and frustrated by lack of opportunities for genuine involvement in decision-making. These insights have important implications for how patient group alliances, researchers and policymakers could strengthen the engagement and contributions of RDPOs, which have become vital stakeholder groups in research.