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

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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.

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Notes

  1. A small number of RDPOs websites were not functioning or not adequate for the purpose of the study.

  2. There were no differences between RDPOs that responded to our survey and all identified RDPOs when compared (on the basis of website data) on a number of variables, including age, disease prevalence, and indications of research goals.

  3. Our interviewees are referred to by pseudonyms in this article.

  4. Only about 15 per cent of survey respondents said that research was the top priority of their RDPO.

  5. Funding guidelines developed by Retina Australia (an organisation for patients with retinitis pigmentosa and other rare retinal dystrophies) state that, although researchers will retain intellectual property rights, “the researcher will enter into negotiations with Retina Australia to determine what is a fair and reasonable proportion of the profits to be paid to Retina Australia in recognition of its contribution in funding, should the researcher file for a patent or enter into commercial development based wholly or in part on research wholly or partially funded by Retina Australia”, www.retinaaustralia.com.au/…/RADoc7ResearchGrantsFundingGuideline, accessed 10 February 2014.

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Acknowledgements

We are extremely grateful to everyone who participated in this study. We thank the Biosocieties’ reviewers for their very helpful comments on an earlier version of this manuscript. The authors declare they have no conflict of interest.

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Pinto, D., Martin, D. & Chenhall, R. Chasing cures: Rewards and risks for rare disease patient organisations involved in research. BioSocieties 13, 123–147 (2018). https://doi.org/10.1057/s41292-017-0061-4

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