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Wednesday, June 19 • 4:30pm - 5:30pm
#375: Sustainability of Rare Disease Drug Development

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Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-674-L04-P; CME 1.00; RN 1.00

Panelists will reflect on the past forty years of the orphan drug act including advancements and impediments. The panel will discuss what is needed in the future to continue to advance rare disease drug development to treat as many patients as possible.

Learning Objectives

Discuss the outcomes, challenges, and opportunities in the next forty years of the Orphan Drug Act; Identify policy advancements that need to be made to continue innovative rare disease drug development.

Chair

Darcy Frear, PhD

Speaker

Panelist
Katherine Donigan, PhD

Panelist
Patroula Smpokou, MD

Panelist
Karin Hoelzer, DVM, PhD

Panelist
Julia Tierney, JD


Speakers
avatar for Darcy Frear

Darcy Frear

Associate Director, Bridgebio, United States
Dr. Darcy Frear is currently the associate director for Regulatory Policy and Intelligence at BridgeBio. Prior to joining BridgeBio in 2022, Darcy began her regulatory career at Genentech supporting program management in oncology and US regulatory policy. Subsequently, she joined... Read More →
KD

Katherine Donigan

Senior Director, Science and Regulatory Policy, Sarepta, United States
Dr. Katherine Donigan is currently a Senior Director of Science and Regulatory Policy at Sarepta Therapeutics. Prior to joining Sarepta, Dr. Donigan was a Senior Director of Science and Regulatory at Biotechnology Innovation Organization (BIO) and spent 7 years in regulatory policy... Read More →
KH

Karin Hoelzer

Senior Director, Policy and Regulatory Affairs, NORD, United States
Dr. Karin Hoelzer directs Policy and Regulatory Affairs for the National Organization for Rare Disorders (NORD®). She works closely with pharmaceutical and biotech partners to ensure public policy supports innovative approaches and new treatments, and adequately incorporates patient... Read More →
avatar for Patroula Smpokou

Patroula Smpokou

Lead Physician, Division of Rare Diseases/Medical Genetics, OND, CDER, FDA, United States
Patroula Smpokou, MD, is a board-certified pediatric clinical geneticist working in the Division of Rare Diseases and Medical Genetics at CDER. Her work involves the clinical review and regulation of products intended for rare genetic diseases. Prior to joining FDA, she was a practicing... Read More →
avatar for Julia Tierney

Julia Tierney

Deputy Center Director for Strategy, Policy, and Legislation, CBER, FDA, United States
JULIE TIERNEY is the Deputy Center Director for Strategy, Policy and Legislation for the Food and Drug Administration’s (FDA’s) Center for Biologics Evaluation and Research (CBER). Ms. Tierney has held several senior roles in the Office of the Commissioner and CBER during her... Read More →

Wednesday June 19, 2024 4:30pm - 5:30pm PDT
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  09: Regulatory, Forum