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Wednesday, June 29 • 4:00pm - 5:15pm
#376: Pediatric Rare Disease Drug Development

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Limited Capacity seats available

Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-593-L01-P; CME 1.25; IACET 1.25; RN 1.25

This session will focus on the critical need for developing medicine for orphan diseases impacting the pediatric population, review global pediatric orphan drug development challenges, and provide information on various strategies designed to overcome these challenges.

Learning Objectives

Specify updates in regulatory policy and industry activities in pediatric orphan drug development; Discuss key clinical development challenges in demonstrating substantial evidence of effectiveness for orphan diseases in pediatrics; Review initiatives to address clinical development and regulatory approval challenges.


Kinnari Patel, PharmD, MBA, RPh


FDA Perspective
Mary Dianne Murphy, MD

CDER Perspective
Lynne P. Yao, MD

Industry Perspective
Kinnari Patel, PharmD, MBA, RPh

avatar for Kinnari Patel

Kinnari Patel

Vice President, Head of Regulatory, Pharmacovigilance, and Compliance, Rocket Pharmaceuticals


Mary Murphy

Director, Office of Pediatric Therapeutics, Office of Special Medical Programs, FDA
Dianne Murphy, MD, FAAP, is Director of the Office of Pediatric Therapeutics (OPT) Office of the Commissioner at FDA. Dr. Murphy was a medical officer at Bethesda Naval; Dir. of the virology laboratory at the U. of Tenn., Knoxville & tenured Professor at the U. of Florida, Jacksonville... Read More →
avatar for Lynne Yao

Lynne Yao

Director, Division of Pediatric and Maternal Health, Office of New Drugs, CDER, FDA, United States
Lynne Yao, MD, is the Director, Office of New Drugs, Division of Pediatric and Maternal Health in the Center for Drug Evaluation and Research, US Food and Drug Administration. The Division of Pediatric and Maternal Health oversees quality initiatives within the Office of New Drugs... Read More →

Wednesday June 29, 2016 4:00pm - 5:15pm EDT
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 17: Rare-Orphan Diseases, Session