Democratizing Education for Sickle Cell Disease Gene Therapy: A Community-Based Model for Creating Patient Education Materials.

Abstract

Introduction Deliberative democracy is an inclusionary approach to reaching consensus decision-making through participative and representative engagement. The Democratizing Education for Sickle Cell Disease Gene Therapy Project used a deliberative community engagement model to partner with patient advocacy and research community members within the field of sickle cell disease (SCD) gene therapy to create new, accessible patient education materials (PEMs) about SCD gene therapy. Objective Develop PEMs for sickle cell disease gene therapy and study the process of deliberative community engaged research Methods A study of the experiences of a multi-disciplinary group of participants including patients, patient advocates, health professionals, gene therapy researchers, industry and government members using a deliberative community engagement model to develop new PEMs. Multiple types of data were collected including survey data (three-time points), video and audio recordings of deliberations, and focus groups. Mixed-methods analysis were used to evaluate the experience of participating in the deliberative community engagement approach. Conclusion The experiences and views of participants reveal both the strengths and challenges of using the deliberative community engagement model to involve participants of diverse backgrounds, life experiences, and expertise. Project participants identified four key focus areas for PEM development: (1) types of gene therapy and alternative curative approaches; (2) social context of gene therapies; (3) treatment medical impact (risks & benefits); and (4) participation in gene therapy clinical trials. The findings from this project provide key insights on the strengths and challenges of a deliberative community engagement model that will be increasingly relevant as the field of gene therapy grows globally.