Monday, January 13, 2014
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.