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ICORD 2009: Inching towards Integration--- the NIH and FDA seek IOM Input on a National Rare Disease Policy

ICORD 2009: Inching towards Integration--- the NIH and FDA seek IOM Input on a National Rare Disease Policy. Timothy Cote, MD MPH Director, Office of Orphan Products Development, Food and Drug Administration, Rockville, MD, USA Monday, Feb 23, 2009. Research Impulse Creative Peer-governance

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ICORD 2009: Inching towards Integration--- the NIH and FDA seek IOM Input on a National Rare Disease Policy

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  1. ICORD 2009:Inching towards Integration---the NIH and FDA seek IOM Input on a National Rare Disease Policy Timothy Cote, MD MPH Director, Office of Orphan Products Development, Food and Drug Administration, Rockville, MD, USA Monday, Feb 23, 2009

  2. Research Impulse Creative Peer-governance Output: transformative ideas Require patients for data generation Regulatory Impulse Standardized Statutory governance Output: transformative actions Utilize data to make best decisions in service to patient Research and RegulationSeparate yet Symbiotic

  3. Problems • Funded research data often insufficient/inadequate for regulatory action. GMP, GCP, GLP are often ignored. • Rare disease clinical trails are unconventional: smaller size, alternative methods, unique situations. Regulatory bodies strength in standardization can hamper advancement.

  4. IOM: Highest Hopes Deliver an integrated vision of rare disease research and rare disease drug regulation.

  5. Other FDA hopes from the IOM • Analysis of existing FDA data to optimize drug development. • Determinants of maturation from designation to marketing approval • Best way to find/use abandoned orphans of promise. • Means of advancing drugs for neglected tropical diseases.

  6. Summary: the IOM Processand the Future • Big thinking • Many inputs • High hopes

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