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...

Purpose – The purpose of this paper is to show the link between the comparative effectiveness research (CER) and the interest in developing drugs for rare disease by the pharmaceutical industry. Total healthcare spending is on the rise without having a corresponding better health outcome. As...

Purpose – The purpose of this paper is to enrich empirical studies on institutional entrepreneurship (IE), more specifically those focusing on the role of the IE in building a new field (here the orphan drug [OD] field). This research addresses the main paradoxes of IE theories: 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