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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.


Ronald Bartek

President/Co-Founder, 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; CIA analyst of East-West balance; graduate, U.S. Military Academy, West Point; Army officer, Korea & Vietnam; MA, Russian Area Studies, Georgetown

Linda Deal

Head of Patient-Centered Outcomes Measurement, Pfizer Inc
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 for the treatment of rare, serious and life-threatening conditions.
avatar for Jonathan Goldsmith

Jonathan Goldsmith

Associate Director for Rare Diseases, Office of New Drugs, CDER, FDA
Dr. Goldsmith is the Associate Director for Rare Diseases in the Office of New Drugs/CDER/FDA. Prior to his federal service, he had an extensive career in academia as a tenured professor, in regulated industry where he focused on clinical drug development, at the NHLBI/NIH and with orphan disease foundations. Dr. Goldsmith earned his medical degree from NYU School of Medicine, received his post-graduate training in Internal Medicine at Vanderbilt... Read More →
avatar for David Schubert

David Schubert

Vice President of Regulatory & Quality, Stealth BioTherapeutics
David Schubert is the Vice President of Regulatory and Quality at Stealth BioTherapeutics Inc., a company developing novel and innovative mitochondrial targeted therapies for both rare and common diseases. Previously, Mr. Schubert was the Vice President of Regulatory Affairs and Quality Assurance for MediVector Inc. At MediVector, Mr. Schubert was responsible for the development and execution of US and EU regulatory and quality activities for a... Read More →

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