. "Appendix F: Advocacy Group Approaches to Accelerating Research and Product Development: Illustrative Examples." Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press, 2011.
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Rare Diseases and Orphan Products: Accelerating Research and Development
TABLE F-1 Spending on Research or Research Grants for Selected Advocacy Organizations, 2008
Spending (millions of dollars)
Scleroderma Research Foundation
Friedreich’s Ataxia Research Alliance
International Rett Syndrome Foundation
Alpha 1 Foundation (research and detection)
Spinal Muscular Atrophy Foundation
Cystic Fibrosis Foundation
SOURCE: For Cystic Fibrosis Foundation and Scleroderma Research Foundation, 2008 financial statement. For Alpha 1 Foundation and Friedreich’s Ataxia Research Alliance, 2009 annual report. For other organizations, IRS form 990 for 2008 as posted on Guidestar.org. (Figures are from Part I, line 13, or Line 10 on the 990-EZ.) Amounts reported do not include fundraising, salaries, or other expenses.
gies that various organizations have developed, often based on a systematic assessment of the gaps in knowledge or resources and the contributions that organizations like theirs can make to bridge those gaps. Not featured but central to organizational research strategies for rare and common diseases alike are three strategic elements: raising funds, political advocacy, and engaging patients, families, and communities in these and other aspects of an organization’s work.
The examples of elements of research strategies for the following organizations are excerpted from materials on each group’s website; they thus may be worded to attract donors and inspire the community of patients, families, researchers, and other supporters. The accuracy of the excerpted materials has not been checked, and their use does not constitute a recommendation or endorsement. The formatting (e.g., font size, text highlighting) has been altered for consistency, and graphics and some details (as indicated) have been omitted.
EXAMPLE 1 LANDSCAPE ANALYSIS: INTERNATIONALRETT SYNDROME FOUNDATION
As part of an effort to understand in detail the environment of researchon Rett syndrome, the International Rett Syndrome Foundation (IRSF)undertook a landscape analysis, which in this case involved a detailedexamination of the focus of research funding by NIH and private entities.The group has recently applied the same analytic strategy to frontotemporaldementia. Rett syndrome (RTT) is a developmental disorder that is caused