The US Orphan Drug Act: rare disease research stimulator or commercial opportunity?

Health Policy. 2010 May;95(2-3):216-28. doi: 10.1016/j.healthpol.2009.12.001. Epub 2009 Dec 29.


Objectives: This study investigates issues associated with the United States Orphan Drug Act.

Methods: A comprehensive orphan drug database was compiled from FDA data and corporate annual reports of major pharmaceutical companies. Analysis allowed the generation of a descriptive orphan drug portrait as well as documentation of orphan drugs along their lifecycle.

Results: Currently, 2002 products have obtained orphan drug designation with 352 drugs obtaining FDA approval. Approximately 33% of orphan drugs are oncology products. On average, products obtain 1.7 orphan designations with approximately 70% obtaining a single designation. At least 9% of orphan drugs have reached blockbuster status with two-thirds having two or more designations. An additional 25 orphan drugs had sales exceeding US$ 100 million in 2008 alone. Since 1983, at least 14 previously discontinued products have been recycled as orphan drugs.

Conclusions: The United States Orphan Drug Act has created issues which, in some cases, have led to commercial and ethical abuses. Orphan Drug Act reform is necessary but current incentives, including 7 year market exclusivity, should be maintained in order to favour patients as well as economic prosperity. Suggested reforms include price regulation, subsidy paybacks for profitable drugs and the establishment of an International Orphan Drug Office.

MeSH terms

  • Annual Reports as Topic
  • Commerce / economics
  • Databases, Factual
  • Drug Approval / organization & administration*
  • Drug Industry / ethics
  • Drug Industry / organization & administration*
  • Health Care Reform
  • Health Services Needs and Demand
  • Humans
  • Marketing of Health Services
  • Motivation
  • Organizations / organization & administration
  • Orphan Drug Production* / economics
  • Orphan Drug Production* / ethics
  • Orphan Drug Production* / legislation & jurisprudence
  • Rare Diseases / drug therapy*
  • Research Support as Topic / ethics
  • Research Support as Topic / organization & administration*
  • United States
  • United States Food and Drug Administration
  • Vulnerable Populations