title: 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.
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.
authors.affiliation: Centre for Biomedical Ethics and Law, Faculty of Medicine, KU Leuven, Kapucijnenvoer 35, Leuven, Belgium.
authors.forename: Wim
authors.initials: W
authors.lastname: Pinxten
authors.forename: Yvonne
authors.initials: Y
authors.lastname: Denier
authors.forename: Marc
authors.initials: M
authors.lastname: Dooms
authors.forename: Jean-Jacques
authors.initials: JJ
authors.lastname: Cassiman
authors.forename: Kris
authors.initials: K
authors.lastname: Dierickx
mesh_terms: D001675:Bioethics; D004992:Ethics, Medical; D005060:Europe / Q000453:epidemiology; D017408:Guidelines as Topic; D006291:Health Policy / Q000191:economics / Q000331:legislation & jurisprudence; D006297:Health Services Accessibility / Q000458:organization & administration; D006801:Humans; D009014:Morals; D009965:Orphan Drug Production / Q000191:economics* / Q000331:legislation & jurisprudence*; D035583:Rare Diseases / Q000188:drug therapy / Q000191:economics; D012106:Research / Q000191:economics; D012109:Research Support as Topic; D040841:Resource Allocation / Q000458:organization & administration*