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Rare Diseases and Orphan Products Accelerating Research and Development (2010) / Chapter Skim
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Appendix A: Study Activities
Pages 283-290

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From page 283...
... 3. Examine current public policies relevant to product development for rare diseases, including the Orphan Drug Act, the Humanitarian Use De vice exemption, the approaches of the National Institutes of Health and the Food and Drug Administration, reimbursement policies, and other legislative and regulatory initiatives.
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From page 284...
... Three of these meetings included public sessions during which the committee heard from a range of interested parties, including government officials, basic and clinical researchers, representatives of advocacy groups that are engaged in supporting research and product development, and representatives of industry trade associations and individual companies involved in developing drugs and devices. (The agendas for the public sessions follow this overview of study activities.)
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From page 285...
... Coté, M.D., M.P.H., Director, FDA Office of Orphan Products Development 4:30 Adjourn open session INSTITUTE OF MEDICINE COMMITTEE ON ACCELERATING RARE DISEASES RESEARCH AND ORPHAN PRODUCT DEVELOPMENT Lecture Room, National Academy of Sciences Building 2101 C Street, N.W., Washington, DC November 23, 2009 -- Open Session 8:20 Welcome and introductions Thomas Boat, M.D., Committee Chair 8:30 Panel 1 National Organization for Rare Disorders Wayne Pines, Consultant
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From page 286...
... Diana R Wetmore, Ph.D., Vice President of Alliance Management Multiple Myeloma Research Foundation and Multiple Myeloma Research Consortium Susan Kelley, M.D., Chief Medical Officer Friedreich's Ataxia Research Alliance Jennifer Farmer, M.S., Executive Director Fanconi Anemia Research Fund (by phone)
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From page 287...
... ; Medical Director, Civitan International Research Center; Professor of Pediatric Neurology, University of Alabama at Birmingham Discussion 2:20 Open discussion 2:45 Adjourn open session Organizations submitting written statements to the committee In addition to the organizations presenting statements during the November meeting, the committee worked with the Genetic Alliance and the National Organization for Rare Disorders to solicit views from their members on issues before the committee. The following organizations responded: American Partnership for Eosinophilic Disorders Brown-Vialetto-Van Laere International Children's Tumor Foundation FRAXA Research Foundation (Fragile X)
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From page 288...
... Senior Consultant, Therapeutics for Rare and Neglected Diseases Program, National Institutes of Health Discussion 9:00 Update from FDA Debra Lewis, O.D., M.B.A. Associate Director, Office of Orphan Products Development, FDA Introduction to Humanitarian Device Exemption (HDE)
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From page 289...
... Assistant Professor, Treuman Katz Center for Pediatric Bioethics and Department of Pediatrics, University of Washington School of Medicine Discussion Noon Working lunch for committee and speakers and other invited guests Update from NIH Stephen Groft, Pharm.D. Director, Office of Rare Diseases Research, NIH 1:00 Translational and Clinical Research for Rare Diseases and Orphan Products Christopher P
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Key Terms

  • orphan products
  • diseases research
  • orphan product
  • genetic alliance
  • diseases clinical

  • rare disorders
  • accelerating rare
  • solicit views
  • orphan drug
  • public sessions

  • national organization
  • stephen groft
  • multiple myeloma
  • development lecture
  • medical devices

  • sciences building
  • written statements
  • ber meeting
  • nih rare
  • national human

  • bayview proteomics
  • alliance biobank
  • integrated national
  • executive vice
  • fraxa research

  • drug discovery
  • committee approximately
  • fragile x
  • medical technology
  • dave frohnmayer


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