. "4 Discovery Research for Rare Diseases and Orphan Product Development." 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
will interfere with the ability of these groups to target their resources and collaborate effectively.
The following chapter shifts the focus from basic research to the preclinical and clinical development investigations that are required to establish safety and efficacy and otherwise meet regulatory standards for approval of pharmaceuticals and biologics. It concludes with additional recommendations for resource sharing and collaboration.
