The dynamics of “Orphanization” and the move towards precision medicine

Paul Martin, Andy Bartlett, Jin Ding, Matthew S. Hanchard and Eva Hilberg

Chapter 5 in Handbook of Pharmaceutical Science and Technology Studies, 2026, pp 75-88 from Edward Elgar Publishing

Abstract: Abstract: This chapter explores the dynamics of “orphanization” – a sociotechnical transition in a key segment of the pharmaceutical sector driven by the growth of orphan drugs for rare diseases. Focusing on developments in the US and Europe, it examines how the orphan drug model, catalyzed by regulatory incentives, has reshaped pharmaceutical innovation, regulatory standards, business strategies, and patient engagement. Orphan drugs, which are increasingly central to the biopharmaceutical sector, now make up about half of all new drug approvals in the US and are characterized by very high prices, posing challenges for healthcare sustainability and access. The chapter traces changes in knowledge production, including the development of molecular biomarkers, precision medicine, new clinical trial designs, and real-world evidence, as well as the rise of expedited regulatory pathways and conditional approvals. Patient organizations, once peripheral, have become active partners in governance and knowledge creation, reshaping the politics and economics of rare disease drug development. The analysis highlights the opportunities and systemic risks alike arising from orphanization, including global access inequities and shifts in the pharmaceutical market. As rare disease and precision medicine converge, the chapter reflects on the broader implications for policy, equity, and the future structure of pharmaceutical innovation systems.

Keywords: Innovation; Orphanization; Sociotechnical transition; Governance; Targeted therapies; Rare disease; Knowledge production (search for similar items in EconPapers)
Date: 2026
ISBN: 9781035338443
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