The economics of rare-disease research is a consistent obstacle to progress. Although there are more than 10,000 rare diseases that collectively affect over 30 million people in the United States, each one impacts fewer than 200,000. Such small market sizes create little incentive for drugmakers to invest the billions of dollars needed to develop new treatments. Innovative policies that allow biotech companies to defy market forces - such as the Pediatric Rare Disease Priority Review (PPRV) program, the Platform Technology Designation program, and the Orphan Drug Act (ODA) - are essential to advancing medicine for some of the most vulnerable and neglected patient populations. This session will examine successful policies from multiple perspectives - government, industry, and patient advocacy - and build a foundation for addressing continuing obstacles to drug development in rare disease.