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Wednesday, June 17 • 9:30am - 10:30am
#310 L: Global Rare Disease Town Hall

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Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-20-662-L04-P; CME 1.00; IACET 1.00; RN 1.00

FDA and international regulators will address unique regulatory complexities and challenges specific to orphan product development. It will provide key information and updates about programs available to expedite orphan drug development and include audience Q&A.

Learning Objectives

Identify unique regulatory complexities and challenges specific to orphan drug development; Describe FDA and EMA/Health Canada programs available to expedite the development of orphan products; Recognize new regulatory initiatives and policy developments related to orphan drug review.


James E. Valentine, JD, MHS


Hylton Joffe, MD

Elizabeth Hart, MD

Janet Maynard, MD, MHS

Kelly Robinson, MSc

avatar for James Valentine

James Valentine

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... Read More →
avatar for Janet Maynard

Janet Maynard

Director, Office of Orphan Products, OC, FDA
Dr. Janet Maynard, as the Director of the Office of Orphan Products Development (OOPD), oversees legislatively mandated designation and grant programs intended to promote the development of products for rare diseases including, orphan drug, rare pediatric disease, and humanitarian... Read More →
avatar for Elizabeth Hart

Elizabeth Hart

Branch Chief, General Medicine 1, OTAT, CBER, FDA
Dr. Elizabeth Hart is the Branch Chief of General Medicine 1 in the Office of Tissue and Advanced Therapies in the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA). She completed her undergraduate medical training at the University of Pennsylvania... Read More →
avatar for Kelly Robinson

Kelly Robinson

Director, Centre for Evaluation of Radiopharmaceuticals and Biotherapeutics, Health Canada
Kelly joined Health Canada in 2001 and has experience in both the pre-market and post-market review and regulation of biologics and pharmaceuticals. Kelly is the Director of the Centre for Evaluation of Radiopharmaceuticals and Biotherapeutics in the Biologics and Genetic Therapies... Read More →
avatar for Hylton Joffe

Hylton Joffe

Acting Director, Office of Rare Diseases, Pediatrics, URM, CDER, FDA
Dr. Hylton Joffe is the acting director of FDA's Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in CDER's Office of New Drugs. Hylton received his medical degree from the University of Arizona then completed his internal medicine residency and endocrinology... Read More →

Wednesday June 17, 2020 9:30am - 10:30am EDT
TBD Virtual Event Horsham, PA 19044
  05: Patient Engagement, Forum