NIH Office of Rare Disease Research (ORDR)

The Office of Rare Diseases Research (ORDR) was established in 1993 within the Office of the Director of the National Institutes of Health (NIH), the Federal focal point for health research. ORDR coordinates and supports rare diseases research, responds to research opportunities for rare diseases, and provides information on rare diseases. In 2002, the Congress and President Bush established ORDR and its responsibilities in statute by enacting and signing Public Law 107–280, The Rare Diseases Act of 2002.

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FDA Office of Orphan Products Development (OOPD)

 The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products (drugs, biologics, devices, or medical foods) that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.  In fulfilling that task, OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. The office also works on rare disease issues with the medical and research communities, professional organizations, academia, governmental agencies, industry, and rare disease patient groups.
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NIH: National Center for Advancing Translational Sciences (NCATS)

Christopher P. Austin, M.D.,  named director of NCATS

The mission of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) is to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions.

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NIH’s Cures Acceleration Network (CAN)

The Cures Acceleration Network (CAN) was authorized to advance the development of high need cures and reduce significant barriers between research discovery and clinical trials. To achieve these objectives, CAN provides the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) with new flexibilities in its funding authorities.

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FDA’s Critical Path Initiative

The Critical Path Initiative (CPI) is FDA’s national strategy to drive innovation in the scientific processes through which medical products are developed, evaluated, and manufactured.

The Initiative was launched in March 2004, with the release of FDA’s landmark report Innovation/Stagnation: Challenge and Opportunity on the Critical Path to New Medical Products. The publication diagnosed the reasons for the widening gap between scientific discoveries that have unlocked the potential to prevent and cure some of today’s biggest killers, such as diabetes, cancer, and Alzheimer’s, and their translation into innovative medical treatments.