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Pennsylvania Convention Center

Jun 26, 2016 9:00 AM - Jun 26, 2016 5:00 PM

1101 Arch Street, , Philadelphia, PA 19107 , USA

#41: A Rare Disease Program Case Study: Strategies for Successful Outcomes


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 workshop 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.

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