A fair share for the orphans: ethical guidelines for a fair distribution of resources within the bounds of the 10-year-old European Orphan Drug Regulation: Figure 1

Abstract

For a significant number of patients, there exists no, or only little, interest in developing a treatment for their disease or condition. Especially with regard to rare diseases, the lack of commercial interest in drug development is a burning issue. Several interventions have been made in the regulatory field in order to address the commercial disinterest in these conditions. However, existing regulations mainly focus on the provision of incentives to the sponsors of clinical trials of orphan drugs, and leave unanswered the overarching question about the rightful place of orphan drugs in resource allocation systems. In this article, we analyse the ethical aspects of funding research and development in the field of rare diseases. We then propose an ethical framework that can help health policy makers move forward in the difficult matter of fairly allocating resources for the prevention, diagnosis and treatment of rare diseases.

Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework

“…Three papers explicitly addressed the ‘life threatening or chronically debilitating’ nature of a condition, which forms part of the EU’s orphan-drug legislation [42, 56, 59]. The authors discussed ethical arguments for favouring the worst-off, “even when only minor gains can be achieved and the cost is very high” [42].…”

“…The authors discussed ethical arguments for favouring the worst-off, “even when only minor gains can be achieved and the cost is very high” [42]. Pinxten et al [56] argued that developing and supplying orphan drugs complies with the ‘core biomedical objectives’ of health care because “these patients have urgent, objective medical needs and because their lives are in danger when they do not receive the necessary care … from a biomedical perspective, there are no valid reasons to exclude rare diseases from publicly funded healthcare”.…”

“…In six papers, the authors explored the impact of orphan drugs on the distribution of health across competing patient populations [18, 31, 42, 52, 55, 56]. It was argued that debates around whether orphan drugs should receive special status must consider opportunity costs [18].…”

Global pharmacogenomics: Where is the research taking us?

“…This health gap between rich and poor populations led the United Nations to include drug development for the least advantaged under Objective 8 of the Millennium Development Goals (MDGs; United Nations (UN), 2013). To date, other national initiatives and policies such as the US Orphan Drug Act (1983) or the EU regulatory framework to stimulate research on rare diseases (2000) have failed to reduce local or global inequalities in access to drugs for rare or neglected diseases (Davies, Neidle, & Taylor, 2012;Pinxten et al, 2012).…”

“…Yet, the success of pharmacogenomics drugs or other interventions have thus far been mitigated (Ma & Lu, 2011). For such promises to translate to the bedside, the upstream scientific researchwhether conducted in university or industry laboratories -must first demonstrate interest and investment in these diseases (Pinxten, Denier, Dooms, Cassiman, & Dierickx, 2012).…”