Orphan Drugs: Supporting Innovation to Address Unmet Medical Need

August - 2009

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Introduction

The Orphan Drug Act (ODA), which celebrated its twenty fifth anniversary in 2008, has been universally heralded as an overwhelming success. Since its enactment, there have been over 300 products approved as "orphan" drugs. Most of these treat extremely rare disorders suffered by relatively small patient populations; orphan diseases are those diseases which have patient populations under 200,000 in the United States. While many orphan drugs are small molecule products, many others are biologicals. Biological therapeutic agents are usually composed of amino acids, peptides, proteins, nucleic acids or other macromolecules that are produced endogenously in higher order organisms. If Congress creates an abbreviated approval pathway for follow on biological products (socalled "biosimilars"), it must consider the impact of this policy on economic incentives for innovator drug companies' development of orphan biologicals. Unfortunately, existing biosimilars proposals do not provide specific protection for rare disease products.

This article will review the ODA, current legislative efforts on biosimilars, and the effects of those efforts on orphan biological development. We propose a possible amendment to pending biosimilar legislation that would preserve the incentives for continued orphan biological development.