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...

Without changes in the current policies, pharmaceutical companies will eventually cease responding to the incentives to develop orphan drugs, because they will increasingly be uncertain whether the drugs, if developed, will be reimbursed. </AbstractSection> Copyright Springer-Verlag Berlin Heidelberg 2014

To encourage the development of orphan drugs, the European Union has implemented specific policies in 2000. However, the political, social, scientific and economic context has changed since the implementation of these policies. For that reason, the aim of this article is to evaluate orphan drug...

This study investigates the relative influence of the regional availability of health care resources (measured by physician densities, number of health care centers) on health care quality (measured by delay in diagnosis), based on data for the rare disease Marfan Syndrome.

Innovative work behavior is a core demand of healthcare professionals who treat patients with rare diseases. In healthcare services, determinants of innovative work behavior are not completely detected. This paper focuses on how the existence of guidelines and the flexibility of healthcare...