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Developing Rare Disease Regulatory Strategy Under Current Global Regulatory Statutes: A Stakeholder Discussion

2.90909090909
3.1 from 12 votes
Date: 28 March, 2018
Location: Cranbury, NJ, USA

Topics to be discussed include:
Overall Challenges of Conducting Clinical Trials in the Rare Disease Population
Concept and Design Considerations in Rare Disease Clinical Development Plans
Understanding the Role of Extrapolation of Data from Varying Age Cohorts: Regulatory Requirements for Pediatric/Rare Disease Drug Development
Novel Statistical Design Considerations in Rare Disease
Drug Development, Pre-Competitive Interactions and Concepts Development

The prestigious list of speakers includes:
Mark Gastonguay, Ph.D., CEO, Metrum Research Group, LLC
Joe Giuliano, Sr. Director, Global Medical Operations and Patient Registry, Medical Affairs, Amicus Therapeutics
David Jacoby, M.D., Executive Group Medical Director, BioMarin Pharmaceuticals
Frank Johnson, Ph.D., Sr. Director, Clinical Pharmacology, Amicus Therapeutics
Vivian Kessler, RAC, Sr. Director, Global Regulatory Affairs, Amicus Therapeutics
Qing Liu, Ph.D., Sr. Director, Biostatistics and Data Management, Amicus Therapeutics
Scott McGoohan, JD, Director, U.S. Regulatory Policy & Intelligence, Vertex Pharmaceuticals
Andrew Mulberg, M.D., Vice President, Global Regulatory Affairs, Amicus Therapeutics
Lily (Yeruk) Mulugeta, PharmD, Clinical Reviewer, Division of Pediatrics, CDER, FDA
Nita Patel, RN, Sr. Director Patient Advocacy, Amicus Therapeutics
Grace Pavlath, M.D., Sr. Vice President, Scientific Program Director, Muscular Dystrophy Association
Christina Bucci-Rechtweg, M.D., Head, Pediatric & Maternal Health Policy Global Drug Development Regulatory Affairs Pediatric Therapeutic Area, Novartis Pharmaceuticals Corporation
Klaus Romero, M.D., Director of Clinical Pharmacology and Quantitative Medicine, Critical Path Institute
Swati Sathe, M.D., Director Clinical Research, Amicus Therapeutics
Vikram Sinha, Ph.D., Assoc. Vice President, Quantitative Pharmacology & Pharmacometrics, Merck & Co.
Sheela Sitaraman, Ph.D., Sr. Director, Program Management, Amicus Therapeutics
John Spaltro, Ph.D., Director, Global Regulatory Affairs, Amicus Therapeutics
Ben Stockham, Head International Regulatory Affairs, Amicus Therapeutics
Kristin Van Goor, Ph.D., Sr. Director, Global Regulatory Policy, Biogen

2.90909090909
3.1 from 12 votes