Trk 17: Rare-Orphan Diseases [
clear filter]
10:30am EDT
#227: Unique Global Regulatory Considerations and Drug Development Incentives in Rare Disease and Orphan Drug Development
Chair
Patient Advocate / Secretary, Canadian Organization For Rare Disorders (CORD)
Maureen Smith’s M.Ed. (Educational Psychology) interest in patient advocacy stems from a rare disease diagnosis at the age of 8. She is the Secretary of the Canadian Organization for Rare Disorders (CORD), co-chair of the Rare/Orphan Disease program track for the Drug Information...
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Speakers IP
Senior Research Scientist, UBC: An Express Scripts Company
Irene Pan, MSc, is a Senior Research Scientist with UBC: An Express Scripts Company. With over 12 years of clinical research experience, Ms. Pan has conducted numerous studies in a wide range of therapeutic indications including ophthalmology, neurology, cardiology, oncology and respiratory...
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Director, Global Regulatory Affairs, Pfizer Inc
15 years of experience across public and government (FDA) sectors with an MBA in biotechnology and an MS in biochemistry. Global experience leading reg strategy with drugs (NCE, line extensions, drug: drug codev, biologic products, and combination products [drug: device]), in all...
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Tuesday June 28, 2016 10:30am - 11:30am EDT
103C
Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
Trk 17: Rare-Orphan Diseases,
Session
- Credit Type ACPE, CME, IACET, RN
- Audience Intermediate
- Featured Topics Approval Pathways, Regulatory Harmonization-Convergence
- Interest Area Rare - Orphan Diseases, Regulatory Affairs
- format json
- Interest Area Rare / Orphan Diseases,Regulatory Affairs
- Featured Topics Approval Pathways,Regulatory Harmonization/Convergence
- Credit Type ACPE, CME, IACET, RN
- Tags Session
2:00pm EDT
#252: Capturing Real-World Data in Rare Diseases
Chair
Medical Affairs Lead, ASPIRE Unit, Actelion
15 years’ experience across medical affairs, product development strategy and new product planning, regulatory affairs, business development, strategy: Former Board President, International Pemphigus and Pemphigoid Foundation (rare disease foundation); Senior Medical Director, Archimedes...
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Speakers
Senior Director, Medical Affairs Research Operations, PPD
Donny Chen, senior director of medical affairs research operations at PPD, has spent the last 18 years designing and spearheading observational research studies in a variety of therapeutic areas, with particular expertise in strategy, analysis and project management. Mr. Chen graduated...
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Senior Director, Clinical Trial Management, Medpace
Derenda Nichols has more than 25 years experience in the CRO industry. Her diverse background includes monitoring and site management, regulatory affairs, medical writing, global project management, operations and most recently, executive management. She has successfully participated...
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Tuesday June 28, 2016 2:00pm - 3:00pm EDT
103C
Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
2:00pm EDT
#353: The Utility of Natural History Studies in Drug Development and Approval
Chair
Attorney, Hyman, Phelps & McNamara, PC
James Valentine, JD, MHS is an attorney at Hyman, Phelps & McNamara where he assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters, including new drug and biologic development and approval issues. Mr. Valentine has been central...
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Speakers
Associate Director for Rare Diseases, Office of New Drugs, CDER, FDA
Dr. Goldsmith is Associate Director for Rare Diseases in the Office of New Drugs/CDER/FDA. Prior to federal service, he was a tenured professor in academia and focused on clinical drug development in regulated industry, at NHLBI/NIH and at orphan disease foundations. He earned his...
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Group Vice President Regulatory Affairs, BioMarin Pharmaceutical Inc.
Camilla Simpson is a Global Regulatory Affairs executive with 20+ years in the Biopharmaceutical Industry. She is currently working for BioMarin, where she leads a team of 150 professionals. Prior to this she was with Shire Pharmaceuticals, and Abbott Laboratories. She is experienced...
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Wednesday June 29, 2016 2:00pm - 3:00pm EDT
103C
Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
4:00pm EDT
#376: Pediatric Rare Disease Drug Development
Chair
Vice President, Head of Regulatory, Pharmacovigilance, and Compliance, Rocket Pharmaceuticals
Speakers MM
Director, Office of Pediatric Therapeutics, Office of Special Medical Programs, FDA
Dianne Murphy, MD, FAAP, is Director of the Office of Pediatric Therapeutics (OPT) Office of the Commissioner at FDA. Dr. Murphy was a medical officer at Bethesda Naval; Dir. of the virology laboratory at the U. of Tenn., Knoxville & tenured Professor at the U. of Florida, Jacksonville...
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Director, Division of Pediatric and Maternal Health, Office of New Drugs, CDER, FDA, United States
Lynne Yao, MD, is the Director, Office of New Drugs, Division of Pediatric and Maternal Health in the Center for Drug Evaluation and Research, US Food and Drug Administration. The Division of Pediatric and Maternal Health oversees quality initiatives within the Office of New Drugs...
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Wednesday June 29, 2016 4:00pm - 5:15pm EDT
103C
Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA