Subtitle

The FDA's Uncertain Interpretation of the Orphan Drug Act

Document Type

Article

Publication Date

Winter 1999

Abstract

In this article we examine the Orphan Drug Act and one of the most important incentives it provides for the development of new drugs for rare diseases, a seven-year period of market exclusivity for new orphan drugs. The FDA has had difficulty interpreting the market exclusivity provisions of the Orphan Drug Act since its enactment. The continuing uncertainties about the scope of protection that the Act provides for innovative products threaten the basic purposes of the Act. The FDA's recent interpretation of the Act raises numerous questions: Should market protection from the Orphan Drug Act depend on the physical and functional properties of the drug, or should the protection depend on the design of the clinical trial? Should there be a nexus between biochemical similarity and clinical superiority, or should a competing drug be brought to the market if its application to the FDA is based on a differently designed clinical trial? Should the market protection afforded by the Orphan Drug Act be commensurate with the effort required to bring the drug to the market? Should the market protection afforded by the Orphan Drug Act be analogous to the scope of patent protection? Finally, should the FDA provide clear rules, analogous to the FDA Guidance Concerning Demonstration of Comparability of Human Biological Products, to assist orphan drug sponsors in their efforts to determine the non-comparability of two orphan drugs?

The article provides a brief overview of the Orphan Drug Act and its incentives for pharmaceutical research in general and the biotechnology industry in particular. The article also provides an introduction to the general scientific context of the problem of determining when two drugs are the same or different and the history of such Orphan Drug Act controversies. The article then uses the most recent major controversy under the Act, the approval of two variant forms of interferon-alpha (and the rejection of a third form) to examine several approaches to the problem of distinguishing similar biotechnology drugs. In particular, we suggest that rules similar to the FDA's policies concerning generic drugs and the comparability of biological molecules from different manufacturing sources should be used to provide guidance under the Act. We also conclude that guidelines for "non-comparability" testing, particularly focused on bioavailability and bioequivalence, could significantly reduce the uncertainties which have continually plagued the FDA's administration of the Orphan Drug Act and would bolster the Act's incentives for innovative medical treatments for rare diseases.

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