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Rare Diseases and Orphan Products: Accelerating Research and Development
new models of “venture philanthropy” to bridge the gulf between basic research findings and approved therapies (see, e.g., IOM, 2008, 2009b; Kelley, 2009; Ashlock, 2010).
In sum, rare diseases have a profound impact on patients and families, but patients and families, in turn, have an impact on the world around them when they organize with others to inform their communities, influence public policy, and stimulate research. Sometimes separately but also in concert, rare disease organizations and their umbrella organizations have worked together on a broad agenda that includes funding for research and technological innovations that will identify the mechanisms of rare diseases and translate these findings into studies that ultimately lead to better ways to prevent, diagnose, and treat these diseases. As this report illustrates, the confluence of scientific advances and policy initiatives provides new opportunities to accelerate this progress.