Small Numbers, Big Concerns: Practices and Organizational Arrangements in Rare Disease Drug Repurposing

Abstract

Because of their small market size, many rare diseases lack treatments. Although government incentives exist for the development of drugs for rare diseases, these interventions have yielded insufficient progress. Drawing on an in-depth case study of rare diseases therapies, we explore how the practices of two nonprofit organizations allowed them to circumvent the endemic market and government failures involving positive externalities by using generic drug repurposing—i.e., seeking new therapeutic applications for existing generic drugs. Beyond elucidating the potential of generic drug repurposing for those suffering from rare diseases, our discoveries provide important insights into the mutual constitution of organizational arrangements for societal challenges and the practices they host. By showing how organizational arrangements can both reinforce and extend practices such that they enable practitioners to achieve a standard of excellence, our study advances practice theory and research on the comparative efficacy of alternative organizational arrangements for tackling societal challenges.