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According to Waxman, orphan drugs were once a story of hopelessness. 7 The impact of the Act on the lives of patients and their families has been expressed, both by individuals who took part in its passage and those who studied it retrospectively, by the idea of ‘hope’. The empowerment of patients living with rare diseases is also commonly cited by health care professionals working in the field of rare diseases and others as a major achievement of the Orphan Drug Act, which patient advocacy was crucial in passing. Some scholars view the legislation as creating a new ‘field’ or ‘agora’, in which multiple stakeholders with a shared interest in developing orphan drugs interact and cooperate-a development they view as a major achievement. 5 The legislation has also served as a model for law or policy internationally, inspiring similar enactments in the 1990s in Singapore, Japan, Australia and Europe. 4 Orphan products, which barely existed before the 1980s, now account for about a third of all newly-approved drugs and biologics. Whereas only 10 drugs for such diseases had been approved by the US Food and Drug Administration (FDA) in the decade before 1983, by 2010, the agency had approved more than 350.
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The impact of the Act is most commonly measured by the number of new treatments for rare diseases developed after its passage.
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3 By closely examining the longer history of orphan-drug legislation-not just the passage of the 1983 law but the three decades from the early 1960s, when the word ‘orphan’ was first used to describe a class of drugs, through the early 1990s, when orphan drugs were shown to be profitable, this paper presents a new perspective on both what has been achieved and also not achieved by adopting the market-oriented approach embodied in the Act. 2 However, a common criticism of the law is that it has resulted in extremely high prices for some orphan drugs. Waxman (D–CA) proudly describes it as ‘an example of government at its finest, demonstrating how Congress applies itself to solve overlooked, but deeply important, problems that affect millions of Americans’. The Act is often considered ‘one of the most successful pieces of health related legislation passed in the United States’. This paper examines the history of orphan drug policy, especially in relation to the US Orphan Drug Act of 1983. Orphan drugs, rare diseases, pharmaceutical market, drug regulation, United States Introduction