Ethical Frontiers in Biotechnology: Hyper-Personalized Medicine

September 13, 2019

Over 22 million patients in the U.S. live with rare genetic diseases for which there are no FDA-approved treatments. Over 3.5 million of these patients are children who will die before the age of five. While genome sequencing can help diagnose these patients, it is far too long and costly to develop treatments for their orphan diseases through usual forms of scientific and pharmaceutical investment.

Using a case study from Boston Children’s Hospital, Drs. Timothy Yu and Insoo Hyun discuss the scientific and ethical challenges facing single-patient (N=1) trials, as well as the opportunities presented by this entrée into what may be termed “hyper-personalized medicine.”