Patient power? The influence of Rare Disease Patient Organisation on the development of gene therapy and genome editing
Paul Martin (University of Sheffield)
Ilke Turkmendag (Newcastle University)
Paper short abstract:
This paper will map the emergence of sociotechnical networks in the UK and US related to gene therapy and genome editing, the way in which patient organisations have become embedded in the field, and how the claims of patients suffering from rare diseases are shaping the technology.
Paper long abstract:
Over the last 20 years the search for treatments for rare genetic diseases has become a priority despite the very small number of people affected by any one condition. A major focus has been the development of gene-based therapies for monogenic conditions, with a number starting to be licenced for use after more than 25 years of clinical testing. More recently, powerful new genome editing techniques offer to revolutionise the treatment of genetic diseases. Key to both the increased priority given to rare diseases and the development of these technologies has been the advocacy of Rare Disease Patient Organisations (RDPOs) who are active in supporting research and innovation, and making claims for more resources and permissive regulation. This raises important questions about research priorities, who decides them, how best to engage patients, and the regulation and future development of technologies that have the power to modify future generations. This paper will describe pilot research that maps the emergence of sociotechnical networks in the UK and US from 2000-2017 related to these new therapies, the way in which patient organisations have become centrally embedded in the field through their support for innovation, and how the claims of patients suffering from rare diseases are shaping the technology. In particular, the analysis will focus on how networks of actors, resources, and techniques associated with gene therapy are providing a powerful template for the emergence of genome editing. This has important implications for the governance of and prospects for human germ line engineering.
Recombining life: sociotechnical intersections in the making of genome editing