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Wednesday, June 27 • 8:00am - 9:15am
#313: Global Rare Disease Town Hall

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Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-18-632-L04-P; CME 1.25; IACET 1.25; RN 1.25

Rare disease drug development is exploding and, in light of both old and new incentives programs to encourage drug development, the challenges of bringing these products to market continue to persist. Exploring the scientific and regulatory issues with experts to provide insights from those with the most experience in both successful and failed rare disease drug proposals.This forum will address the unique regulatory complexities and challenges specific to orphan drug development. It will provide key information about programs available to expedite the development of orphan products and will include audience Q&A.

Learning Objectives

Identify unique regulatory complexities and challenges specific to orphan drug development; Describe FDA programs available to expedite the development of orphan products; Recognize FDA requirements related to orphan drug regulation under 21st Century Cures and PDUFA VI.


James E. Valentine


EMA Perspective
Kristina Larsson, MS

Perspectives on Rare Diseases and Gene Therapies
Ilan Irony, MD

FDA Perspective
Lucas Kempf, MD

avatar for Ilan Irony

Ilan Irony

Deputy Director, DCEPT, OTAT, CBER, FDA
After training at UCSF and NIH and years of practice in Internal Medicine and Endocrinology, Dr. Irony joined FDA CBER in 2000 as a clinical reviewer. He also worked in the Endocrine Division in CDER as a reviewer and team leader. In 2011, he returned to CBER first as branch chief... Read More →

Lucas Kempf

Acting Associate Director, Rare Diseases Program, OND, CDER, FDA
Dr. Lucas Kempf is the Acting Associate Director for the Rare Disease program in the OND immediate office. Prior to joining FDA in 2012, Lucas spent 8 years at NIH with a focus on neuroscience research, working to understand the genetics of neuropsychiatric disease and developing... Read More →
avatar for Kristina Larsson

Kristina Larsson

Head of Office for Orphan Medicines, European Medicines Agency (EMA)
Kristina Larsson joined the orphan team of the EMA as the Head of Office in July 2014. Before that she spent 8 years as a scientific officer in the scientific advice team of the EMA in charge of the Scientific Advice Working Party secretariat. Before joining the agency she worked... Read More →
avatar for James Valentine

James Valentine

Director, Hyman, Phelps & McNamara, PC, United States
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 →

Wednesday June 27, 2018 8:00am - 9:15am EDT
Room 205AB Boston Convention and Exhibition Center 415 Summer Street, Boston, MA 02210 USA
  09: Regulatory, Forum