Abstract

Cell and gene therapies offer opportunities for treating disease with potential to restore function, and cure disease. However, they are not without risk and pose complex logistical, economic, ethical and social challenges for health systems. Here we report our systematic review of the current evidence on patient and public knowledge and perspectives of cell and gene therapies, to inform future research, education and awareness raising activities. We screened 10,735 titles and abstracts, and evaluated the full texts of 151 publications. The final selection was 35 publications. Four themes were generated from the narrative synthesis of the study findings namely: (1) Knowledge and understanding of cell and gene therapies, (2) Acceptance of cell and gene therapies (3) Understanding of risk and benefits of therapy, and (4) Information needs and current sources of information. As potential funders or future recipients, it is important that the public and patients are aware of these therapies, understand the issues involved, and can contribute to the debate. This review highlights the need for appropriate patient and public education on the various aspects of cell and gene therapies. High quality studies exploring patient and public opinions and experiences of cell and gene therapy are required. Patient and public perceptions of these therapies, alongside evidence of clinical and cost-effectiveness, will be central to their uptake and use.

Highlights

  • Cell and gene therapies offer opportunities for treating disease with potential to restore function, and cure disease

  • The European Medicines Agency (EMA) refers to cell and gene therapies along with tissue-engineered medicines as Advanced Therapy Medicinal Products (ATMPs)[7]

  • It is crucial that health systems, regulatory bodies and researchers are aware of the knowledge and perspectives of patients and the public as this insight may guide information provision, patient and public involvement (PPI) activities and inform the development and delivery of targeted educational interventions

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Summary

Introduction

Cell and gene therapies offer opportunities for treating disease with potential to restore function, and cure disease They are not without risk and pose complex logistical, economic, ethical and social challenges for health systems. We report our systematic review of the current evidence on patient and public knowledge and perspectives of cell and gene therapies, to inform future research, education and awareness raising activities. New cell, gene and tissue-engineered therapies have been developed to treat various cancers, inherited diseases and some chronic conditions[1] They offer opportunities for the treatment of disease and injury, to restore function, and in some cases offer cures[1,2,3,4]. We provide descriptions of the cell, gene and tissue-engineered therapies in the Supplementary Table 1

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