Abstract
This article investigates how the engagement of patients’ organisations (POs) in research relates to the dynamics of their activism in the area of rare diseases. It traces back how certain concerned families and groups elaborated rareness as an issue of equity and social justice, gave shape to what we call a ‘politics of numbers’ for stating the fact of rare diseases as a major public health problem, and promoted patients’ critical involvement in biomedical and therapeutic research as a solution for mainstreaming rare diseases in regular health systems. It then studies three Portuguese and three French POs, which point to the limits of the epidemiological notion of rareness for capturing the compounded and intersecting nature of the bio-psycho-social make-up of their conditions. It finally shows how these critics progressively lead to the emergence of an alternative politics, which we call a ‘politics of singularisation’. At the core of this politics stands a collective and ongoing profiling of conditions and patients, whose similarities and differences relates to the ubiquity of biological pathways and diseases categories. Our contention is that this ‘politics of singularisation’ not only pictures a politics of illnesses which questions the rationale for nosological classifications, but also, and consequently, affects the making of social links by suggesting the simultaneous identification of individual patients and constitution of collectives to which they partake while asserting their specificities.
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Notes
The AFM massively uses military metaphors such as “war on disease”, “our enemy”, and the likes, to capture its move from self-help to the fight against the disease, and the strategic role of its engagement in research on this route (Rabeharisoa and Callon, 1999). Formed in 1958 as the French association of people with myopathies, grouping parents of children suffering from DMD (Duchenne de Boulogne Muscular Disease) (Paterson and Barral, 1994), the AFM renamed itself the French association against myopathies in the 1980s (our emphasis).
There exist about 300 rare diseases POs in France, 200 of which are today members of the French Alliance, created in 2000 (www.alliance-maladies-rares.org).
Unless mentioned otherwise, the descriptions of conditions in this article are based on data collected and made available by Orphanet, a web portal on rare diseases and orphan drugs, which the biomedical community, the industry and POs contribute and refer to. Initiated by a few French scientists from the INSERM (Institut National de la Santé et de la Recherche Médicale – the French Institute for Health and Medical Research), Orphanet today has 35 partner countries all over the world (www.orpha.net).
Recently, the acronym CATCH 22, for “Cardiac, Abnormal face, Thymus, Cleft palate, Hypocalcemia”, has also been proposed (www.orpha.net).
This French popular expression designates Professors of medicine, who head clinical wards in public hospitals.
Destruction of the membranes of blood cells.
The second plan started in 2010, also for four years.
The Plan however was adopted with no financial commitment from the Portuguese government, which faced a dramatic budget crisis.
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Acknowledgements
This article is based on a European research project called EPOKS (European Patients’ Organizations in Knowledge Society), supported by the European Commission FP7. We warmly thank the leaders and representatives of patients’ organisations, the researchers and the clinicians who agreed to be interviewed. We owe a lot to our partners in this project, who took an active part in the discussion of our data and analysis. We are also grateful to the members of Centre de sociologie de l’innovation of Mines-ParisTech who offered thoughtful comments on a previous version of this article. Our final thanks go to the anonymous reviewers for their suggestions.
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Rabeharisoa, V., Callon, M., Filipe, A. et al. From ‘politics of numbers’ to ‘politics of singularisation’: Patients’ activism and engagement in research on rare diseases in France and Portugal. BioSocieties 9, 194–217 (2014). https://doi.org/10.1057/biosoc.2014.4
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DOI: https://doi.org/10.1057/biosoc.2014.4