meeting with researchers and clinicians to develop was developed the shared agenda A research agenda was developed • Nierse Netherlands NMDs Patient A staff member of VSN was a member of the A research agenda was developed • • (2013) organizations team developing the agenda [137] Germany Retinitis Pigmentosa Patient Pro Retina, a German Retinitis Pigmentosa Not specified • organizations patient group, formulated research priorities for (2005) the scientific community [42] Initiation of research Ferguson United States Chromosome 18 deletion Patients and A mother of a patient noticed improvement in Researchers tested the theory, finding it to be • • families (2002) symptoms after taking human growth correct her child’s [19] hormone and developed a theory to explain her observations based on available scientific literature Mai United States Li-Fraumeni syndrome Patients and Patients participated in a workshop around Patients and families identified 6 priorities for • • families (2012) (LFS) clinical research on LFS at the National Institutes clinical research [57] of Health with scientists and physicians, sharing A new advocacy group was established to • their experiences and their goals facilitate effective communication between LFS families and the clinical and scientific members of the research consortium Netherlands Patient The Dutch Addison and Cushing Society The NVACP-requested study eventually led to Addison’s disease; • • disease organizations (NVACP) requested an independent research the establishment of a research project on Cushing’s (2005) facility to complete a study on improved drug delayed release tablets [42] administration methods for the treatment of disease Addison’s Panofsky United States Rare diseases in general Patient Patient organizations develop close relationships provides benefits for patient • • “Sociability” (2011) organizations with, and between, scientists to organizations and can help scientists develop (i.e., “sociability”) [145] influence the research process stronger research networks and improved Additional mechanisms utilized include productivity • resources; collective mobilization; timing; lay expertise; and organization controls Note: according to the author, without • sociability, these additional mechanisms are insufficient for influencing research Patient Not specified Rare diseases in general Patient Patient organizations promote the running of a Not specified • Partner organizations trial by gathering researchers; demonstrating [20] interest in new treatments; and bringing together research teams
meeting with researchers and clinicians to develop the shared agenda was developed • A research agenda was developed Nierse (2013) [137] Netherlands NMDs Patient organizations • A staff member of VSN was a member of the team developing the agenda • A research agenda was developed (2005) [42] Germany Retinitis Pigmentosa Patient organizations • Pro Retina, a German Retinitis Pigmentosa patient group, formulated research priorities for the scientific community Not specified Initiation of research Ferguson (2002) [19] United States Chromosome 18 deletion Patients and families • A mother of a patient noticed improvement in her child’s symptoms after taking human growth hormone and developed a theory to explain her observations based on available scientific literature • Researchers tested the theory, finding it to be correct Mai (2012) [57] United States Li-Fraumeni syndrome (LFS) Patients and families • Patients participated in a workshop around clinical research on LFS at the National Institutes of Health with scientists and physicians, sharing their experiences and their goals • Patients and families identified 6 priorities for clinical research • A new advocacy group was established to facilitate effective communication between LFS families and the clinical and scientific members of the research consortium (2005) [42] Netherlands Addison’s disease; Cushing’s disease Patient organizations • The Dutch Addison and Cushing Society (NVACP) requested an independent research facility to complete a study on improved drug administration methods for the treatment of Addison’s disease • The NVACP-requested study eventually led to the establishment of a research project on delayed release tablets Panofsky (2011) [145] United States Rare diseases in general Patient organizations • Patient organizations develop close relationships with, and between, scientists (i.e., “sociability”) to influence the research process • Additional mechanisms utilized include resources; collective mobilization; timing; lay expertise; and organization controls • Note: according to the author, without sociability, these additional mechanisms are insufficient for influencing research • “Sociability” provides benefits for patient organizations and can help scientists develop stronger research networks and improved productivity Patient Partner [20] Not specified Rare diseases in general Patient organizations • Patient organizations promote the running of a trial by gathering researchers; demonstrating interest in new treatments; and bringing together research teams Not specified
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