The PRIME Institute of the College of Pharmacy, University of Minnesota, recently released preliminary research findings indicating a trend of extraordinary pharmaceutical industry pricing of drug products in the United States (U.S.). According to researchers at the PRIME Institute, such extraordinary price increases are defined as any price increase that is equal to, or greater than, 100% at a single point in time. In some instances, PRIME Institute researchers found that drugs exhibiting extraordinary price increases are categorized as "orphan drugs" (...) (or blood-related biologic treatments) and often life-saving or life-sustaining for treating the cause or symptoms of diseases affecting fewer than 200,000 people in the U.S., or where there is prevalence of less than 5 per 10,000 people afflicted with a disease or symptoms in the community. Because of extraordinary price increases for orphan drugs — some exceeding 1000% at a single point in time -this article addresses two interrelated questions: Are extraordinary orphan drug price increases socially responsible behavior? If so, are the pharmaceutical industry's policies providing orphan drug access to American consumers in dire need of available life-sustaining and life-enhancing pharmaceuticals considered "socially responsible" behavior? The author concludes, after an interdisciplinary analysis of the legal, economic, sociopolitical, and ethical dimensions of orphan drug pricing, that they are not socially responsible – unless justified by cost and availability of health care marketplace/patient options. Furthermore, the author recommends a socially responsible industry strategic approach to insure that patients ultimately receive -regardless of cost -timely access to life-saving and life-sustaining orphan drugs. (shrink)

Torbjörn Tännsjö has written a clear and thought-provoking book on healthcare priority setting. He argues that different branches of ethical theory—utilitarianism, egalitarianism, and prioritarianism—are in general agreement on real-world healthcare priorities, and that it is human irrationality that stands in the way of complying with their recommendations. While I am generally sympathetic to the overall project and line of argumentation taken by the book, this paper raises two concerns with Tännsjö’s argument. First, that he is wrong to set aside deontic (...) constraints as irrelevant or as pointing in the same direction as consequentialism. Secondly, that his argument against prioritarianism in favor of utilitarianism is insufficient and under-developed. Given these problems, I conclude that we should welcome Tännsjö’s contribution but with these qualifications in mind. (shrink)

Individuals living with rare conditions are faced with important challenges derived from the rarity of their conditions and aggravated by the low priority given to rare disease research. However, current realities of rare disease research require consideration of the relationship between subjectivity and ‘traditional’ objectivity. Objectivity in research has traditionally been associated with processes and descriptions that are independent of the investigator. The need for researchers to provide unbiased knowledge and achieve a balance between objectivity and the underlying values in (...) nursing and scientific research requires an examination of how objectivity is conceptualized within the context of rare disease research. The aim of this paper is to examine scientific objectivity in rare disease research from a philosophical viewpoint and, in doing so, demonstrate the need to redefine it to reflect the current scientific environment. As such, healthcare providers working on this field need to redefine objectivity around ethical and moral obligation to advance science in an equitable manner with the end goal to produce knowledge that is trustworthy and beneficial to our patients. (shrink)

Orphan drug policy often gives ‘special treatment’ to rare diseases, by giving additional priority or making exceptions to specific drugs, based on the rarity of the conditions they aim to treat. This essay argues that the goal of orphan drug policy should be to make prevalence irrelevant to funding decisions. It aims to demonstrate that it is severity, not prevalence, which drives our judgments that important claims are being overlooked when treatments for severe rare diseases are not funded. It shows (...) that prioritising severity avoids problems caused by prioritising rarity, and that it is compatible with a range of normative frameworks. The implications of a severity-based view for drug development are then derived. The severity-based view also accounts for what is wrong with how the current system of drug development unfairly neglects common diseases that burden the developing world. Lastly, the implications of a severity-based view for current orphan drug policies are discussed. There are no data in this work. (shrink)