In recent years, the biopharmaceutical industry has seen an increase in the development of so-called orphan drugs for the treatment of rare and neglected diseases. This increase has been spurred on by legislation in the United States, Europe, and elsewhere designed to promote orphan drug...

This paper investigates the impact of the introduction of new orphan drugs on premature mortality from rare diseases using longitudinal, disease-level data obtained from a number of major databases. The analysis is performed using data from two countries: the United States (during the period...

This article explores how an Eastern European country could deal with orphan drugs access, combining EU policies with its own national settings. The cross-sectional observational study takes the total number of orphan drugs (61) available on EU level by March 2011, and then consecutively filters...

Understanding doctors' preferences for prioritizing treatment of rare diseases can provide an important context for policy makers who must decide whether to exempt rare disease treatments, which are often quite expensive, from standard cost-effectiveness criteria. We surveyed a random sample of...

Purpose – This article aims to provide a description of principal aspects of policy and practice associated with orphan drugs and treatments of rare diseases, and give perspectives for 2011 on new and emerging approaches for addressing patient access. Design/methodology/approach –...

Formal risk analysis techniques applied in managing construction project risks tend to focus on risks that lend themselves to 'objective' methods of economic analysis. Although subjective probabilities and Bayesian methods are applied successfully in other industries to manage 'subjective' risks...

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...