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Free Whitepaper:

Maintaining Momentum in Orphan Drug Development

In 2011, more than one third of new drug approvals in the United States were for orphan drugs, equating to more than 400 therapies in development to treat or prevent rare diseases.

Orphan drug development is now a focus of large pharmaceutical companies in search of therapies with “niche busting” potential.

This paper outlines:

  • Legislation and market potential related to orphan drug development
  • Strategies for utilizing disease communities and venture philanthropy in initiating orphan drug trials
  • Overcoming development challenges of orphan drug trials, including the unique patient populations and poorly-defined clinical endpoints
  • Collaboration tactics between patients, regulatory agencies, CROs, sponsors and advocacy groups to encourage a successful orphan drug trial

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