Research Article: What Is the Purpose of the Orphan Drug Act?

Date Published: January 3, 2017

Publisher: Public Library of Science

Author(s): Matthew Herder

Abstract: Matthew Herder suggests it may be time to re-examine the purpose of the U.S. Orphan Drug Act.

Partial Text: Before the ODA became law, Congress heard diverse views about which R&D “orphans” the legislation should attempt to rescue [10]. Some witnesses focused on rare diseases during Congressional hearings, whereas others advocated for “orphan medical devices and medically necessary foods” [10]. Still others spoke in favor of “drugs for less developed countries,” or vaccines, which manufacturers had moved away from due to high product liability concerns at that time. Worried that this lack of consensus might undermine the bill’s progress, the ODA’s authors made a political choice to focus on rare diseases [10].

What should be done? One idea is for the FDA to try yet again to cut down on the practice of salami slicing and, in turn, to better discriminate between genuine and artificial rare diseases. In 1992, the FDA first purported to curb salami slicing by requiring that, for subsets of common diseases to be considered rare, they needed to be “medically plausible” [16], a term it failed to define. Twenty-one years later, the FDA finally promulgated more promising regulations [17] that hold drug manufacturers to a higher standard of evidence. When seeking an orphan drug indication, manufacturers must now show not only why one subset of a disease should be targeted by their drug, but also why the drug is inappropriate outside the selected subset [18]. However, the findings of Kesselheim and colleagues [4] suggest that these new regulations—or the FDA’s application of them—may not be adequate to the task. Another potentially more fruitful approach is to limit orphan drug designation to disease pathways rather than the rarity of the disease per se [4].



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