fda marks orphan drug act milestone: 30 year recognition january 2013
TRANSCRIPT
![Page 1: FDA Marks Orphan Drug Act Milestone: 30 Year Recognition January 2013](https://reader036.vdocuments.site/reader036/viewer/2022062407/56649da95503460f94a9630b/html5/thumbnails/1.jpg)
FDA Marks Orphan Drug Act Milestone:
30 Year RecognitionJanuary 2013
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One of Many Rare Disease Heroes…
Henry Waxman and Orrin Hatch
Author and cosponsors of the Orphan Drug Act (ODA)
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One of Many Rare Disease Heroes…
Abbey MeyersChampion for ODA and founder of
National Organization for Rare Disorders
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One of Many Rare Disease Heroes…
Media/Entertainment
IndustryRaising awareness about rare diseases
and the need for medical products
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One of Many Rare Disease Heroes…
Rare Disease Advocacy Groups
Organization, education, support, research, advocacy, and action
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One of Many Rare Disease Heroes…
FDA Orphan Incentive Programs
Advancing orphan products through orphan designations, grants, and
facilitation
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One of Many Rare Disease Heroes…
NIH Office of Rare Diseases
ResearchAdvancing rare disease information,
collaboration, and research
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One of Many Rare Disease Heroes…
Robert Campbell Jr, MD
representing Clinician/product developers and advocates;Developed the artificial rib humanitarian use
device
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One of Many Rare Disease Heroes…
Hemin and Desiree Lyon
representing1st approved orphan designated drug
and patient advocacy
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One of Many Rare Disease Heroes…
Jana Monacorepresenting
Parent advocacy for newborn screening and medical foods
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One of Many Rare Disease Heroes…
Industry-Investors-Academia
Developing products in the spirit of the ODA
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One of Many Rare Disease Heroes…
Sharon Terryrepresenting
Advocacy for innovative and collaborative approaches for rare disease products
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One of Many Rare Disease Heroes…
NIH Undiagnosed Diseases Program
Cross-cutting efforts to understand and treat rare diseases
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One of Many Rare Disease Heroes…
Nancy Wexler, PhD
representingPatient advocacy and gene
discovery/research
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One of Many Rare Disease Heroes…
Ashley Appellrepresenting
Patients living and thriving with a rare disease
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One of Many Rare Disease Heroes…
FDA Offices/Centers
Communication, diagnostic and therapeutic product evaluation, safety, and access
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One of Many Rare Disease Heroes…
Myozyme and Tiffany
Houserepresenting
Contributions of patient representatives to the FDA orphan approval process
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One of Many Rare Disease Heroes…
Ronald J. Bartekrepresenting
Family member founder of a rare disease advocacy group
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One of Many Rare Disease Heroes…
Pediatric Ventricular Assist Device-Berlin
Heartrepresenting
ODA Grants and Humanitarian Use Devices for pediatric rare diseases
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One of Many Rare Disease Heroes…
Denise Ney, PhDrepresenting
Clinical dietitian/medical foods researcher for metabolic disorders
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One of Many Rare Disease Heroes…
Hal Dietz, MDrepresenting
Clinical research in rare diseases
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One of Many Rare Disease Heroes…
FDA/CDER Rare Diseases Program
Focused regulatory science and education efforts for rare disease drug
development
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One of Many Rare Disease Heroes…
NIH TRND Program
Stimulating drug discovery and collaborative preclinical development for rare disease
drugs
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One of Many Rare Disease Heroes…
EMA/EURORDIS/IRDIRC/ICORD
representingCollaborative international rare disease
efforts
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One of Many Rare Disease Heroes…
Pearl O’Rourke, MD
representingIRB efforts on behalf of rare diseases
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One of Many Rare Disease Heroes…
Jouni Uitto, MD, PhD
representingBasic research in rare diseases
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One of Many Rare Disease Heroes…
George Harris Carter
representingPatient advocacy for drug development
and clinical trials
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One of Many Rare Disease Heroes…
Kalydeco
representingPersonalized medicines for rare diseases
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One of Many Rare Disease Heroes…
Leslie Boyer, MD
representingAcademic grantees successfully pursuing
biological marketing approvals
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One of Many Rare Disease Heroes…
Rodney Howell, MD
representingAdvocacy for newborn screening/early detection and treatment of congenital
disorders
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FDA Marks Orphan Drug Act Milestone:
30 Year RecognitionJanuary 2013