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

March 28 @ 8:00 am - 4:30 pm



March 28, 2018, 8:00 a.m.-4:15 p.m.
Amicus Therapeutics, 1 Cedarbrook Drive, Cranbury, NJ

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.

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:

  • Jeffrey Barrett, MD, Ph.D., Vice President, Translational Informatics, Sanofi
  • 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
  • Susan McCune, M.D., Director, Office of Pediatric Therapeutics, Office of the Commissioner, U.S. Food and Drug Administration
  • 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
  • Robert “Skip” Nelson, M.D., Ph.D., Sr. Director, Pediatric Drug Development, Child Health Innovation Leadership Dept. (CHILD),
  • 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

To view the full agenda, click here.

Save the date and plan to join us on March 28 for this unique event. Registration will open shortly.


March 28
8:00 am - 4:30 pm
Event Category:


Amicus Therapeutics
6 Cedar Brook Drive
Cranbury, NJ United States
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