Who rules rare disease associations? A framework to understand their action

The inner structure of so‐called ‘patients’ organisations’ has been accorded relatively little attention with respect to their increasing role in the medical world. This comparative study in France of eight such organisations, matching six rare disorders, explores the issue of power and decision making through the description of the entities that make up the organisation (and especially which stakeholders are represented), their mutual relationships, the temporal scope of collective action, and the concrete achievements of the organisation. Two main types of organisation are distinguished: ‘pluralistic’ organisations (that bring together a broad array of different stakeholders who are willing to work together) and ‘monistic’ organisations (where a single category of stakeholders firmly takes the lead). Their operations are very different and result from the association’s composition. A key finding is that both the usual opposition between lay and expert and the reference to the diseases’ characteristics prove to be irrelevant to understanding these organisations. Rather, the composition of the leading group is crucial.