Clinical Research: Phase 1 - Phase 4

Mapping the New Landscape of Orphan Drug Development

Position compounds for clinical and commercial success

Despite aggressive orphan drug development over the past 15 years, the pharma industry — dissuaded by small patient populations, high costs, and limited commercial potential — has barely put a dent in the 7,000+ known rare diseases.

In this white paper, we look at opportunities in the field of rare disease treatments and outline strategies to maximize the odds of success. Topics include:

  • The often poorly understood incentives for orphan drug development: government grants, tax credits, market exclusivity provisions, and more
  • Avenues for expedited drug development, including fast track and breakthrough designations, priority review, and accelerated approval
  • Regulatory strategies such as early regulatory engagement, educating regulators and setting precedents, and creating a unique development algorithm for each compound

The outlook for orphan drugs is brighter today than ever. Download the white paper for more tips on seizing this unprecedented opportunity.