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Sunday, June 26 • 9:00am - 5:00pm
#41: A Rare Disease Program Case Study: Strategies for Successful Outcomes

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Limited Capacity seats available

Component Type: Tutorial
CE: ACPE 6.50 Application UAN: 0286-0000-16-507-L01-P; CME 6.50; IACET 6.50; RN 6.50

An additional registration fee is required for all preconference tutorials.  Register today for this tutorial.

Working through a multifaceted case study, this one-day, intensive tutorial will address heads-on the unique challenges associated with the development of rare disease therapies. Successful outcomes are a direct result of early planning, design and engagement with patients, patient advocacy groups, treating physicians, specialists, and regulators.

This interactive case study will emphasize how “spending” time in early planning assures appropriate study outcome measures, timely enrollment of clinical trials, and successful regulatory interactions which affect the bottom line and improve the potential for developing therapies for rare disease patients faster.

Who should attend?

This tutorial is designed for professionals working with rare disease therapies including by not exclusive to researchers from academia and industry, senior managers, regulatory affairs specialists, clinical trialists, government officials and investors focused on the future of rare disease and orphan product development.

Learning Objectives

• Examine rare disease clinical and regulatory program issues and challenges;
• Discuss special considerations for study endpoint identification and development;
• Distinguish differences between working with FDA on the development of rare disease therapies as compared to more common diseases;
• Develop ideas for educating, building visibility and understanding of a rare disease programs with key stakeholders.

avatar for Ronald Bartek

Ronald Bartek

Co-Founder/Founding President, Friedreich's Ataxia Research Alliance (FARA)
Co-founder/President, FARA, Board of Directors, NORD; NIH/NINDS National Advisory Council; partner/president, government affairs firm; 20 yrs federal service in defense, foreign policy & intelligence; U.S. Delegation to Intermediate-Range Nuclear Forces (INF) Treaty talks, Geneva... Read More →
avatar for Linda Deal

Linda Deal

Head of Patient-Centered Outcomes Measurement, Pfizer Inc.
Linda currently serves as PCOA Lead working across Pfizer’s therapeutic categories addressing efforts to develop, validate and use COAs as endpoints for providing evidence of medical treatment benefit differentiation, labeling and value. Linda has served patients and the pharmaceutical... Read More →
avatar for Jessica Foley

Jessica Foley

Head of Regulatory Services, NovusLife, LLC
Jessica is a senior regulatory professional and consultant with 10 years of experience in the global biopharmaceutical industry. She provides strategic product development and approval strategies to pharmaceutical and biotechnology companies with a focus on investigational therapies... 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 David Schubert

David Schubert

Vice President of Regulatory and Quality, Stealth BioTherapeutics
David Schubert is the Vice President of Regulatory and Quality at Stealth BioTherapeutics Inc. Previously, Mr. Schubert held positions at MediVector Inc., Logical Therapeutics Inc., Coley Pharmaceutical Group, Genzyme and Cubist Pharmaceuticals. Mr. Schubert has over 30 years of experience... Read More →

Sunday June 26, 2016 9:00am - 5:00pm EDT
112AB Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA