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.