From unmet medical needs to dilemmas of access to treatment: The case of rare diseases and orphan drugs

Carlos Novas

Chapter 23 in Handbook of Pharmaceutical Science and Technology Studies, 2026, pp 363-375 from Edward Elgar Publishing

Abstract: This chapter examines legislation, regulations, and processes of judicialization related to pharmaceuticals intended for the treatment of rare diseases. I focus on the intersection between law, governance, and Science and Technology Studies (STS) by examining the passage of the US Orphan Drug Act (1983). This case study explores how, throughout the 1970s, drugs intended to treat small patient populations were considered of limited commercial value, and pharmaceutical companies claimed to have insufficient economic incentives to focus on this market. The US Orphan Drug Act (1983) provides a case study of how scientists, physicians, patients’ organizations, US Food and Drug Administration regulators, politicians, and the pharmaceutical industry worked together to create a legislative framework that incentivizes the development of drugs intended for rare diseases. This case study further highlights the role of social, regulatory, and legislative institutions in creating a market to satisfy unmet medical needs. This chapter provides an overview of the success of this legislation in increasing the supply of orphan drugs and discusses concerns related to the high cost of these therapies. The second part of the chapter focuses on access to orphan drugs in the South American context as it highlights the regulatory and institutional challenges of meeting the healthcare needs of rare disease patients. The examples of Brazil and Chile will be used as they foreground the role of right to health legislation and the Courts in shaping access to orphan drugs. The analysis of orphan drugs, regulation and judicialization in this chapter draws upon STS and governmentality literatures.

Keywords: Orphan drugs; Rare diseases; Patient organizations; Biopolitics; Governmentality; Judicialization (search for similar items in EconPapers)
Date: 2026
ISBN: 9781035338443
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