Background Rare diseases pose immense challenges for healthcare systems due to their low prevalence, associated disabilities, and attendant treatment costs. Advancements in gene therapy, such as treatments for Spinal Muscular Atrophy (SMA), have introduced novel therapeutic options, but the high...

We offer a theory of how the combination of budget constraints and insurance drives up prices. A natural context for our theory is the health care market, where drug prices can be very high. Our model predicts that monopoly prices for orphan drugs are inversely related to the prevalence up until...

The aim of this paper is to discuss the common features and specificity of Corporate Social Responsibility (CSR) practices of innovative transnational corporations (TNCs) acting in the pharmaceutical industry. The innovativeness of pharmaceutical firms is understood here as their ability to make...

The aim of this paper is to discuss the common features and specificity of Corporate Social Responsibility (CSR) practices of innovative transnational corporations (TNCs) acting in the pharmaceutical industry. The innovativeness of pharmaceutical firms is understood here as their ability to make...

We offer a theory of how the combination of budget constraints and insurance drives up prices. A natural context for our theory is the health care market, where drug prices can be very high. Our model predicts that monopoly prices for orphan drugs are inversely related to the prevalence up until...

Highlights the emergence of a "hybrid collective model" of collaboration between patients and experts through a systematic study in the field of rare diseases in France and Portugal, with attention to the reflexive work carried out by patients' organizations on the notion of rareness.