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Monday, June 19 • 11:00am - 12:15pm
#120: FDA Rare Disease Town Hall

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Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-17-541-L04-P; CME 1.25; IACET 1.25; RN 1.25

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.


James E. Valentine, JD, MHS


Gayatri R. Rao, JD, MD

Jonathan C. Goldsmith, MD, FACP

Ilan Irony, MD

Michael Pacanowski, PharmD, MPH

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 →

avatar for Jonathan Goldsmith

Jonathan Goldsmith

Associate Director for Rare Diseases, Office of New Drugs, CDER, FDA
Dr. Goldsmith is Associate Director for Rare Diseases in the Office of New Drugs/CDER/FDA. Prior to federal service, he was a tenured professor in academia and focused on clinical drug development in regulated industry, at NHLBI/NIH and at orphan disease foundations. He earned his... Read More →
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 →

Michael Pacanowski

Associate Director for Genomics and Targeted Therapy, OCP, CDER, FDA
Dr. Pacanowski is the Associate Director for Genomics and Targeted Therapy in the Office of Clinical Pharmacology at FDA. His team of translational scientists works to advance the use of pharmacogenomics and other biomarker innovations in drug development through review of investigational... Read More →
avatar for Gayatri Rao

Gayatri Rao

Director, Office of Orphan Products Development, Office of the Commissioner, FDA
Gayatri R. Rao is the Director for the Office of Orphan Products Development (OOPD) at FDA. The Office’s mission is to advance the development of promising drugs, biologics, devices, and medical foods for rare diseases. Dr. Rao oversees several programs that promote product development... Read More →

Monday June 19, 2017 11:00am - 12:15pm CDT
N227b McCormick Place 2301 South Martin Luther King Jr. Drive, Gate 4 Chicago, IL 60616
  07: SpecialPops, Forum