For anyone affected by a rare disease worldwide, and for all of us working towards an Orphan Drug Policy in Canada, the below thesis is an excellent source of information and I think, well worth the read. Below is the abstract, click the link for the full thesis: https://circle.ubc.ca/handle/2429/39881
“In the context of Canada’s publicly funded universal health care system, access to potentially life-saving and/or life lengthening orphan drugs costing anywhere from $100,000.00 to $850,000.00 per patient per year is a complicated matter. This study is an anthropological examination of the debates surrounding ‘expensive drugs for rare diseases’, a term that has come to represent the costly treatments developed for rare metabolic diseases like Mucopolysaccharidosis, Pompe Disease, Fabry Disease, and Phenylketonuria.
This study was conducted in British Columbia, Canada. It is based on several months of participant observation in hospital, industry, and patient advocacy contexts, as well as 14 semi-structured interviews conducted with the different stakeholders in the debate: patients and families, health care professionals, representatives of the provincial government’s Ministry of Health, pharmaceutical companies, and patient advocacy groups. This study looks at discussions of authority, responsibility, and rights to health care/health technology. It examines how complex systems of relationships shape these discussions in a particular time and place, and how the competing cultural models of publicly funded health care and profit-based pharmaceutical policy and industry operate in the context of extremely expensive drugs. The body of literature on orphan drugs in the social sciences/humanities is very underdeveloped, and there are no known comprehensive social scientific/ethnographic studies of the metaphors, constructs, and cultural context of debates surrounding orphan drugs/expensive drugs for rare diseases. This study attempts to fill some of these gaps by looking at the complexities of different stakeholder arguments and their structural and discursive context.
In attempting to reconcile and solve the problems of accessibility to EDRD, the different stakeholders directly implicated in the debate mobilize culturally shaped notions evidence, accountability, fairness, and responsibility. This study demonstrates that the problems, pitfalls, and provisional solutions articulated by the different people implicated in this debate throw in to relief the many contradictions between orphan drug policies, neglected diseases, drug regulation/assessment practices, and the relationship between pharmaceuticals and society. These frameworks and competing cultural models are creating tensions that may be irreconcilable with a publicly funded health care system.”
