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Wednesday, June 29 • 2:00pm - 3:00pm
#353: The Utility of Natural History Studies in Drug Development and Approval

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Limited Capacity seats available

Component Type: Session
Level: Intermediate
CE: CME 1.00; IACET 1.00; RN 1.00

A well-designed natural history study can help in designing an efficient drug development program and support marketing approval. This session will discuss the utility of natural history studies and strategies for designing them.

Learning Objectives

Recognize the ways in which a natural history study can guide each stage of drug development; Identify features of natural history registries that will support drug development activities; Summarize the use of natural history controls to support important FDA drug approvals.


James E. Valentine, JD


FDA Draft Guidance and the Utility of Natural History Studies in the Development of Drugs for Rare Diseases
Jonathan C. Goldsmith, MD, FACP

Considerations in the Use of National History Studies: Development and Registration Perspective
Camilla V. Simpson, MS

Use of Historical Controls to Support Drug Approvals
James E. Valentine, JD

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 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 Camilla Simpson

Camilla Simpson

Group Vice President Regulatory Affairs, BioMarin Pharmaceutical Inc.
Camilla Simpson is a Global Regulatory Affairs executive with 20+ years in the Biopharmaceutical Industry. She is currently working for BioMarin, where she leads a team of 150 professionals. Prior to this she was with Shire Pharmaceuticals, and Abbott Laboratories. She is experienced... Read More →

Wednesday June 29, 2016 2:00pm - 3:00pm EDT
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 17: Rare-Orphan Diseases, Session