Paper short abstract:

This communication looks at the emergence of orphan drugs markets, the intricacies between their testing and pricing, and debates on how to make rare diseases matter.

Paper long abstract:

In this communication, we look at the development of orphan drugs, e.g. drugs for rare diseases, as an interesting site for exploring the emergence of markets at the intersection of public health policies, regulatory measures, evidentiary practices, pricing mechanisms, patient activism, issues of social justice and moral sentiments.

Because of the small size of the targeted populations, orphan drugs could not pass the gold standard clinical trials that regulate the marketing of drugs, and were considered economically non viable by the industry. The 1983 American Orphan Drugs Act, and the 2000 European Directive on Orphan Medicinal Products, brought in economic incentives and scientific assistance to pharmaceutical and biotech firms willing to develop orphan drugs. These, and other legal measures, paved the way to the marketing of orphan drugs. They also raise criticisms against the "exceptional" regime, which some think rare disease patients and the industry benefit from, notably because orphan drugs tend to be very expensive and turn out to be "blockbusters of a new type", as some observers put it.

This communication draws on a preliminary fieldwork on initiatives launched by patient organizations in France, UK and in Europe, and on the dialogue these organizations establish with public authorities and the industry. We focus on how these initiatives and dialogue problematize the testing and the pricing of orphan drugs, and raise heated debates on how to make orphan drugs and rare diseases matter for the market and for society at large.