Chapter 5:Disease Advocacy Organisations

Patient support groups, voluntary health organisations and disease advocacy organisations (DAOs) are just a few of the names to describe the non-profit organisations that are working to accelerate discovery and development of rare disease interventions. These organisations run the gamut from simple support for people affected by a condition to full-blown research entities that rival some pharmaceutical companies in financing and capacity. In the early years of DAOs being involved in drug development the participation was largely indirect: DAOs influenced policy and culture. In recent times DAOs have had a more direct influence on rare disease research and drug development through funding, fundamental and applied research and access to disease expertise. With the advances that new technologies, a networked age, and a mature DAO culture offer, systemic change in rare disease drug development should be possible. It is clear that DAOs connect previously disparate research, invent new models for collaboration and use social media to integrate various components of the drug discovery system. DAOs were early pioneers in forging pathways into a very technical and difficult field. In the modern age, DAOs continue to lead the way in influencing, funding and enabling research and drug development and constitute a critical element in tackling rare diseases.