Fair Access and Equity of Individualized Interventions for Ultrarare Genetic Conditions
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Since application of the first individualized therapy in 2019, development of these bespoke treatments has expanded rapidly. Individualized therapies—including directly administered vectored gene therapy, genetically modified cellular therapies, and antisense oligonucleotides, among others—refer to products designed to treat one to a few individuals based on their specific molecular diagnosis. This technology offers 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 development and 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? In this ELSI Friday Forum, we explore these and other ethical considerations arising in this new therapeutic landscape.
Panelist: Ingrid Holm, MD, MPH (Boston Children’s Hospital, Harvard Medical School)
Panelist: Alison Bateman-House, MPH, PhD (NYU Langone Health, University of Arizona)
Moderator: Meghan Halley, PhD, MPH (Stanford University)
Please join fellow audience members for an informal discussion in a Zoom meeting immediately following the forum. The link will be placed in the Zoom Chat during the forum. For those who cannot attend the live event, the forum will be recorded and archived on the ELSIhub Video page. Closed captioning will be provided for live and recorded events.
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