Panelists:
Ingrid Holm, MD, MPH (Boston Children's Hospital, Harvard Medical School)
Alison Bateman-House, MPH, PhD (NYU Langone Health, University of Arizona)
Moderated by: Meghan Halley, PhD, MPH (Stanford University)
Abstract: Since application of the first individualized therapy in 2019, development of bespoke treatments has expanded rapidly. ‘Individualized therapies’ refer to products designed to treat one to a few individuals based on their specific molecular diagnosis, including directly administered vectored gene therapy, genetically modified cellular therapies, and antisense oligonucleotides. Individualized therapies offer a particularly exciting opportunity for patients with so-called “n-of-1” or “ultrarare” diseases, which lack incentives for drug development through traditional pathways. However, the high cost of their development and the inherently small number of patients eligible to receive each new therapy raise complex ethical concerns related to equity and access. How should research resources be allocated across the many thousands of ultrarare diseases eligible for this approach? Within disease communities, how should the specific gene targets be selected, and who should make these decisions? When and how should additional eligible patients be allowed to access newly developed therapies? Further, given the highly technical nature of the development process, will it ever be possible to safely expand access outside of elite academic medical centers? This ELSI Friday Forum explores these and other ethical considerations arising in this new therapeutic landscape.
ELSI Friday Forum is sponsored by NHGRI
Location and Address
Online