This ELSI Friday Forum took place on September 8, 2023.
Since application of the first individualized therapy in 2019, development of these bespoke treatments has expanded rapidly. Individualized therapies—including antisense oligonucleotides and others that may be developed—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)
- Moderator: Meghan Halley, PhD, MPH (Stanford University)
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Videos in Series

ELSI Friday Forum - Population Descriptors in Genomic Research: Applying the NASEM Recommendations

ELSI Friday Forum - Legal and Policy Challenges to Privacy in the Post-Genomic and Post-Dobbs Era

ELSI Friday Forum - Value and Values in Payment for Gene Therapies

ELSI Friday Forum - Visual Storytelling in ELSI Research

ELSI Friday Forum - Indigenizing Genomics and Advancing Indigenous Data Sovereignty

ELSI Friday Forum - Wrestling with Social and Behavioral Genomics

ELSI Friday Forum - Genetic Advantages in Sports: When Do They Count as "Doping"?

ELSI Friday Forum - Genetics and AI: Group Privacy and Fairness

ELSI Friday Forum - Advocacy and Allyship in ELSI: Opportunities and Challenges

ELSI Friday Forum - Decoloniality and Genetic Ancestry: Situating the "African Genome"

ELSI Friday Forum - Legal Challenges to Newborn Screening Research

ELSI Friday Forum - Impact of SCOTUS's Dobbs Decision on Prenatal Genomics Research and Practice

ELSI Friday Forum - Addressing Algorithmic Harms: Practices and Provocations for Health AI

ELSI Friday Forum - Balancing Data Privacy and Data Sharing: Normative and Technical Approaches

ELSI Friday Forum - Genomic Imaginaries: Sparking Dialogue between ELSI and Literary Studies

ELSI Friday Forum - Ensuring Equitable Use of New Genetic Technologies: Lessons from Eugenics

ELSI Friday Forum - New ACMG Guidance on Carrier Screening: More or Less Equitable?

ELSI Friday Forum - Benefit-sharing and Pharmaceutical Development in Africa: What Does Equity Mean?
ELSI Friday Forum - Genomic Data Sharing: Putting Principles and Policy into Practice
ELSI Friday Forum - Current Legal Challenges to Abortion: Implications for Prenatal Genetics
ELSI Friday Forum - Ethical Challenges in Novel Gene Therapies for Sickle Cell Disease

ELSI Friday Forum - Widening the Lens: Using Arts in ELSI Research

ELSI Friday Forum - Migrant DNA: Context, Ethics and Legal Issues

ELSI Friday Forum - Genomics and Infectious Disease: Scientific and ELSI Issues of COVID

ELSI Friday Forum - ELSI Friday Forum: Biobanking in the Era of COVID

ELSI Friday Forum - Addressing Racism in Research and Clinical Practice
