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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
  1. Wim Pinxten1,
  2. Yvonne Denier2,
  3. Marc Dooms3,
  4. Jean-Jacques Cassiman4,
  5. Kris Dierickx5
  1. 1Department of Medical Ethics and Philosophy of Medicine, Erasmus MC, Rotterdam, The Netherlands
  2. 2Centre for Biomedical Ethics and Law, Faculty of Medicine, KU Leuven, Leuven, Belgium
  3. 3Pharmacy Department, University Hospitals Leuven, Leuven, Belgium
  4. 4Department of Human Genetics, Faculty of Medicine, KU Leuven, Leuven, Belgium
  5. 5Centre for Biomedical Ethics and Law, Faculty of Medicine, KU Leuven, Leuven, Belgium
  1. Correspondence to Professor Yvonne Denier, Centre for Biomedical Ethics and Law, Faculty of Medicine, KU Leuven, Kapucijnenvoer 35 box 7001, Leuven 3000, Belgium; yvonne.denier{at}med.kuleuven.be

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.

  • Rare diseases
  • orphan drugs
  • resource allocation
  • ethical framework
  • regulation (EC) 141/2000
  • minors/parental consent
  • laws and cases
  • informed consent
  • newborns and minors
  • allocation of healthcare resources
  • distributive justice
  • medical ethics
  • tissue engineering
  • personalism
  • minors in medicine
  • genetic biobanking
  • ethics in genetic research
  • human genetics
  • empirical ethics
  • reproductive technology

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Footnotes

  • WP and YD contributed equally to this work.

  • Competing interests None.

  • Provenance and peer review Not commissioned; externally peer reviewed.

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