Download: The Science of Hope – rare disease studies that work

In Partnering & Investment, Regulation & Government, Whitepapers by Freya Smale

premier research, science of hope, rare disease, orphan drug, rare disease studies, rare disease conference, world orphan drug conference ,orphan drug congress , WODC

This guest blog post was provided by Premier Research.

Too few patients. Too little data. Enormous recruitment challenges. What now?

Rare disease studies face many of the same obstacles as other drug development projects. But getting around those obstacles often poses unique challenges.

Our white paper, The Science of Hope: The need, the challenges and three proven strategies for successful orphan drug development, spells out what you need to know to make your next rare disease study a success:

  • The huge need for new therapies.
  • Financial and regulatory incentives for developing Orphan Drugs.
  • Challenges to watch out for.
  • Proven strategies to overcome obstacles in three key areas of development.

To download ‘The Science of Hope’ whitepaper, click here.

Or visit to discover how our experienced rare disease study teams can guide you through the process from experimental molecule to FDA and EMA approval.

We’re pleased to be a sponsor of the 4th Annual World Orphan Drug Congress, November 14-15, 2013. We’re currently developing our panel discussion, and would love to hear from you about what you’d like to see us cover with our Geneva panel. Let us know in the comments below, or send us an email at


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