Rare diseases, drug development, and AIDS: the impact of the Orphan Drug Act.

P. S. Arno, K. Bonuck, M. Davis

Research output: Contribution to journalReview articlepeer-review

32 Scopus citations


The Orphan Drug Act provides public subsidies and incentives to spur the development of drugs for rare diseases--drugs that the private sector might otherwise consider unprofitable to produce. Although the act has achieved numerous successes, the high prices and extraordinary sales generated by some orphan drugs lead to a pivotal policy question: how can the act be used to meet the legislative goal of stimulating drug development for small patient populations without resulting in prices that make drugs inaccessible? This question is explored using the example of AIDS drugs, many of which received subsidies under the act, to illustrate central points. The history of the act, its weaknesses, and strategies for reform are described as well.

Original languageEnglish (US)
Pages (from-to)231-252
Number of pages22
JournalThe Milbank quarterly
Issue number2
StatePublished - 1995

ASJC Scopus subject areas

  • Health Policy
  • Public Health, Environmental and Occupational Health


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