The U.S. Food & Drug Administration (FDA) recently approved two novel gene therapies (GT) that aim to effectively cure sickle cell disease (SCD), a notoriously debilitating and historically marginalized condition that affects more than 100,000 Americans. While society has largely heralded this milestone in clinical genomics and SCD care as a revolutionary step towards restorative justice, many underlying issues complicate the clinical integration of GT for SCD. As such, this research aimed to: 1) explore diverse stakeholders' perspectives around the clinical implementation of GT for SCD; 2) identify urgent ethical and practical issues threatening this pivotal moment in SCD care and genomic medicine; and 3) recommend interventions that advance restorative justice throughout the U.S. SCD community. Data was collected through a targeted literature review structured around eight key stakeholder groups, as well as participation in relevant listening sessions and discussions with thought leaders. Four overarching priorities arose as key themes that need to be addressed for the ethical and successful integration of GT: a) safety and efficacy; b) accessibility and equity; c) shared decision-making; and d) large-scale feasibility. Furthermore, this research uncovered more than 15 ethical issues and 20 recommended interventions specific to the SCD community’s integration of GT. A comprehensive framework was created to synthesize and disseminate these timely and actionable research findings to key stakeholders; this framework is publicly accessible online via bit.ly/GTSCDFramework. Future activities include soliciting feedback from key stakeholders and adapting the framework to meet the evolving needs of the SCD community, as well as other disease populations seeking restorative justice in the era of clinical genomics.
Authors: Tessa Youngner, Harvard Medical School Center for Bioethics; Hadley Smith, Harvard Medical School and Harvard Pilgrim Health Care Institute
