Discussions last week at an FDA workshop on Orphan drug development, and on BioCentury This Week television, highlighted the consensus among patient advocates, regulators and drug developers about the barriers that are holding back development of new therapies for rare conditions - and the lack of consensus on strategies for overcoming those barriers.

Orphan drug development faces many of the problems that hinder the creation of new therapies for larger indications, including a paucity of qualified biomarkers and patient-reported outcome (PRO) endpoints, but small patient populations make such obstacles both more acute and more difficult to overcome.