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The involvement of patient organisations in rare disease research: a mixed methods study in Australia

Pinto, Deirdre, Martin, Dominique and Chenhall, Richard 2016, The involvement of patient organisations in rare disease research: a mixed methods study in Australia, Orphanet journal of rare diseases, vol. 11, pp. 1-15, doi: 10.1186/s13023-016-0382-6.

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Title The involvement of patient organisations in rare disease research: a mixed methods study in Australia
Author(s) Pinto, Deirdre
Martin, DominiqueORCID iD for Martin, Dominique orcid.org/0000-0001-9363-0770
Chenhall, Richard
Journal name Orphanet journal of rare diseases
Volume number 11
Article ID 2
Start page 1
End page 15
Total pages 15
Publisher BioMed Central
Place of publication London, Eng.
Publication date 2016
ISSN 1750-1172
Keyword(s) rare diseases
patient organisations
research
Australia
research policy
patient and public involvement
consumer involvement
Summary Background: We report here selected findings from a mixed-methods study investigating the role of Australian rare disease patient organisations (RDPOs) in research. Despite there being many examples of RDPOs that have initiated and supported significant scientific advances, there is little information – and none at all in Australia – about RDPOs generally, and their research-related goals, activities, and experiences. This information is a pre-requisite for understanding what RDPOs bring to research and how their involvement could be strengthened.

Methods: We reviewed 112 RDPO websites, conducted an online survey completed by 61 organisational leaders, and interviewed ten leaders and two key informants. Quantitative and qualitative data were analysedusing basic descriptive statistics and content analysis, respectively.

Results: Although most are small volunteer-based groups, more than 90 % of the surveyed RDPOs had a goal to promote or support research on the diseases affecting their members. Nearly all (95 %) had undertaken at least one research-related activity – such as providing funding or other support to researchers – in the previous five years. However, RDPO leaders reported considerable challenges in meeting their research goals. Difficulties most frequently identified were insufficient RDPO resources, and a perceived lack of researchers interested in studying their diseases. Other concerns included inadequate RDPO expertise in governing research “investments”, and difficulty engaging researchers in the organisation’s knowledge and ideas. We discuss these perceived challenges in the light of two systemic issues: the proliferation of and lackof collaboration between RDPOs, and the lack of specific governmental policies and resources supporting rare disease research and patient advocacy in Australia.

Conclusion: This study provides unique information about the experiences of RDPOs generally, rather than experiences retrospectively reported by RDPOs associated with successful research. We describe RDPOs’ valuable contributions to research, while also providing insights into the difficulties for small organisations trying to promote research. The study is relevant internationally because of what it tells us about RDPOs; however, we draw attention to specific opportunities in Australia to support RDPOs’ involvement in research, for the benefit of current and future generations affected by rare diseases.
Language eng
DOI 10.1186/s13023-016-0382-6
Field of Research 110399 Clinical Sciences not elsewhere classified
1199 Other Medical And Health Sciences
Socio Economic Objective 920109 Infectious Diseases
HERDC Research category C1.1 Refereed article in a scholarly journal
ERA Research output type C Journal article
Copyright notice ©2016, The Authors
Free to Read? Yes
Use Rights Creative Commons Attribution licence
Persistent URL http://hdl.handle.net/10536/DRO/DU:30083280

Document type: Journal Article
Collections: Faculty of Health
School of Medicine
Open Access Collection
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Every reasonable effort has been made to ensure that permission has been obtained for items included in DRO. If you believe that your rights have been infringed by this repository, please contact drosupport@deakin.edu.au.