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Jan 13, 2014
 |  BioCentury  |  Regulation

Orphan barriers

FDA workshop highlights hurdles to expanding Orphan drug space

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.

Congress and FDA have already taken extraordinary measures to bolster the commercial and scientific feasibility of creating new drugs for rare diseases. The question now is whether these measures are sufficient to address the still enormous unmet need.

There are no FDA-approved therapies for 95% of the estimated 7,000 rare diseases, and advances in precision medicine are creating new subpopulations that fall under the 200,000-patient Orphan threshold (see BioCentury This Week, Jan. 5).

Nevertheless, the initial surge of Orphan drugs wouldn't have occurred in the absence of market exclusivity and tax benefits provided by the Orphan Drug Act. And FDA's expedited development tools such as breakthrough therapies, Fast Track designation, accelerated approval and Priority Review, have been applied extensively to Orphan drug applications.

In opening remarks at FDA's Jan. 6-7 meeting on Complex Issues in Rare Disease Drug Development, FDA's Richard Moscicki noted that more than 36% of novel drugs the agency approved in 2013 had Orphan designations. He added that most products...

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