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Developing Rare Disease Regulatory Strategy Under Current Global Regulatory Statutes: A Stakeholder Discussion
March 28, 2018 @ 8:00 am - 4:30 pm
March 28, 2018, 8:00 a.m.-4:15 p.m.
Amicus Therapeutics, 1 Cedarbrook Drive, Cranbury, NJ
This event is at capacity. Please contact Debbie Mennito at DMennito@BioNJ.org if you would be interested in being placed on a wait list should any spots open up.
Mark your calendar and plan to join us on March 28, 2018 for a brand-new program entitled, Developing Rare Disease Regulatory Strategy Under Current Global Regulatory Statutes: A Stakeholder Discussion, created in collaboration with Amicus Therapeutics, Inc.
The pursuit of research in rare disease populations presents unique challenges as well as opportunities in developing research protocols and regulatory pathways to ensure that innovation reaches patients who are often in desperate circumstances. Hear the latest on regulatory strategy and development considerations from FDA and industry experts who address these challenges on a daily basis. To view the full agenda, click here.
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:
- Karim Azer, Ph.D., Head, Quantitative Systems Pharmacology, Sanofi U.S.
- Mark Gastonguay, Ph.D., FISoP, CEO, Metrum Research Group, LLC
- Joe Giuliano, Executive Director, Global Medical Operations and Patient Registry, Amicus Therapeutics, Inc.
- Charbel Haber, Ph.D., Vice President, Global Regulatory Sciences, Biogen
- David Jacoby, M.D., Ph.D., Vice President, Head, Clinical Science, BioMarin Pharmaceuticals
- Franklin K. Johnson, Ph.D., Sr. Director, Clinical Pharmacology, Amicus Therapeutics, Inc.
- Vivian Kessler, RAC, Executive Director, Global Regulatory Affairs, Amicus Therapeutics, Inc.
- Qing Liu, Ph.D., Sr. Director, Biostatistics and Data Management, Amicus Therapeutics, Inc.
- Susan McCune, M.D., Director, Office of Pediatric Therapeutics, Office of the Commissioner at the Food and Drug Administration
- Scott McGoohan, JD, Director of U.S. Regulatory Policy and Intelligence at, Vertex Pharmaceuticals
- Andrew E. Mulberg, M.D., FAAP, CPI, Vice President, Head, Global Regulatory Affairs, Amicus Therapeutics, Inc.
- Lily Mulugeta, PharmD, Clinical Reviewer, Division of Pediatrics, CDER, U.S. Food and Drug Administration
- Robert “Skip” Nelson, M.D., Ph.D., Senior Director, Pediatric Drug Development, Child Health Innovation Leadership Department, Johnson & Johnson
- Nita Patel, RN, Sr. Director, Patient & Professional Advocacy, Amicus Therapeutics, Inc.
- Grace Pavlath, M.D., Ph.D., Sr. Vice President, Scientific Program Director, Muscular Dystrophy Association
- Christina Bucci-Rechtweg, M.D., Global Head, Pediatric & Maternal Health Policy, Drug Regulatory Affairs, Novartis Pharmaceuticals
- Klaus Romero, M.D., MS, FCP, Director of Clinical Pharmacology and Quantitative Medicine, Critical Path Institute
- Vikram Sinha, Ph.D, Assoc. Vice President, Quantitative Pharmacology & Pharmacometrics, Merck Research Laboratories, Merck & Co.
- Sheela Sitaraman, Ph.D., PMP, Senior Director, Program Management at Amicus Therapeutics, Inc.
- John Spaltro, Ph.D., Director, Global Regulatory Affairs, Amicus Therapeutics, Inc.
- Ben Stockham, Head, International Regulatory Affairs, Amicus Therapeutics, Inc.