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Special Communication
February 2018

Ethical Challenges Confronted When Providing Nusinersen Treatment for Spinal Muscular Atrophy

Author Affiliations
  • 1Stanford Center for Biomedical Ethics, Stanford University, Stanford, California
  • 2Center for Biomedical Ethics and Humanities, Northwestern University, Chicago, Illinois
  • 3Center for Bioethics and Humanities, University of Colorado Anschutz Medical Campus, Aurora
  • 4Center for Bioethics, Harvard Medical School, Boston, Massachusetts
  • 5Office of Ethics, Boston Children’s Hospital, Boston, Massachusetts
  • 6Department of Neurology, Harvard Medical School, Boston, Massachusetts
  • 7Department of Critical Care Medicine, Boston Children’s Hospital, Boston, Massachusetts
  • 8Division of Neurology, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania
  • 9Department of Medical Ethics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania
JAMA Pediatr. 2018;172(2):188-192. doi:10.1001/jamapediatrics.2017.4409
Abstract

The US Food and Drug Administration’s December 2016 approval of nusinersen for the treatment of patients with all subtypes of spinal muscular atrophy ushered in a new era for patients with spinal muscular atrophy, their families, and all those involved in their care. The extreme cost of the medication and the complicated logistical requirements for administering nusinersen via lumbar puncture have created practical challenges that raise important ethical considerations. We discuss 6 challenges faced at the institutional level in the United States: cost, limited evidence, informed consent, treatment allocation, fair distribution of responsibilities, and transparency with stakeholders. These challenges must be understood to ensure that patients with spinal muscular atrophy benefit from treatment, are protected from harm, and are treated fairly.

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