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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

Timothy Cote, MD MPH

Director, Office of Orphan Products Development, Food and Drug Administration, Rockville, MD, USA

Monday, Feb 23, 2009


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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


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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.


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IOM: Highest Hopes

Deliver an integrated vision of rare disease research and rare disease drug regulation.


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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.


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Summary: the IOM Processand the Future

  • Big thinking

  • Many inputs

  • High hopes


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