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

P. S. Arno, Karen A. Bonuck, M. Davis

Research output: Contribution to journalArticle

26 Citations (Scopus)

Abstract

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
Volume73
Issue number2
StatePublished - 1995

Fingerprint

Orphan Drug Production
Anti-HIV Agents
orphan
Rare Diseases
AIDS
drug
Disease
Pharmaceutical Preparations
Private Sector
subsidy
Motivation
History
sales
private sector
Population
incentive
act
reform

ASJC Scopus subject areas

  • Health Policy
  • Nursing(all)
  • Health(social science)
  • Health Professions(all)

Cite this

Rare diseases, drug development, and AIDS : the impact of the Orphan Drug Act. / Arno, P. S.; Bonuck, Karen A.; Davis, M.

In: The Milbank quarterly, Vol. 73, No. 2, 1995, p. 231-252.

Research output: Contribution to journalArticle

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