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...

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 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...

Objectives Policy makers pay more and more attention to the orphan drug market. Pharmaceutical companies are suspected to take advantage of the legislation. Moreover, granted an orphan designation (OD) could be associated with higher prices. The aim of this work is to assess the impact of the...

When entering the market, orphan drugs are associated with substantial prices and a high degree of uncertainty regarding safety and effectiveness. This makes decision making about the reimbursement of these drugs a complex exercise. To advance on this, the Dutch government introduced a...

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

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.

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.