Social Pharmaceutical Innovation for Rare Diseases: Towards a Conceptual Definition and Research Program

Conor Douglas (York University, Canada)

Panel: Biosocialities, patient organizations, and activism in the area of rare diseases

Abstract: There is something wrong with the way in which the pharmaceutical industry researches and develops drugs. For instance, of the some 7000 currently identified rare diseases only about 570 treatments have been approved by the US Food and Drug Administration to treat about 11% (or 838) of those rare diseases. As a result millions of patients around the world still go untreated. To be sure some of the challenges facing research and development in this space are scientific and technical. However, there are also a number of critical issues that are thwarting more rapid development of, and ultimate access to, drugs for rare diseases that are more associated with social, economic and political (regulatory) factors that have come to characterize the current innovation paradigm within the pharmaceutical industry. This presentation argues that the current model of pharmaceutical innovation alone will not deliver the quantity of products needed to address the unmet needs faced by rare disease patients, nor at a price point that is sustainable for healthcare systems. As a consequence, radical transformations are needed across the pharmaceutical research, development and deployment life-cycle that stand to offer alternative, supplementary and hopefully transformative pathways to a greater number of increasingly accessible treatments. Here we present how principles of social innovation (SI) have been developed and deployed in other sectors “to serve demands which neither the state nor markets would or can meet”, which is seen to be the case in rare diseases. Our aim is to show how those principles can be applied in the pharmaceutical sector – in what we are calling social pharmaceutical innovation (or SPIN) – to help address the unmet needs of rare disease patients as well as their families and caregivers. The presentation will briefly introduce social innovation and its key features before demonstrating our understanding of SPIN, why it is important, and how we hope to foster it.
This presentation is part of a larger international collaborative project entitle “Social Pharmaceutical Innovation for Unmet Medical Needs” with partners from the University of Sao Paulo (Brazil), Mines ParisTech at the Université PSL (France), and Utrecht University (Netherlands). The Canadian component of the study is funded through the Social Sciences and Humanities Research Council (SSHRC), and more information about the project can also be seen here: