"I just wanted to speak to someone- and there was no one…": using Burden of Treatment Theory to understand the impact of a novel ATMP on early recipients.

Background: Advanced therapy medicinal products such as Chimeric antigen receptor T-cell therapy offer ground-breaking opportunities for the treatment of various cancers, inherited diseases, and chronic conditions. With development of these novel therapies continuing to increase it's important to learn from the experiences of patients who were among the first recipients of ATMPs. In this way we can improve the clinical and psychosocial support offered to early patient recipients in the future to support the successful completion of treatments and trials.

A scoping review of patient and public perspectives on cell and gene therapies.

The development and introduction of cell and gene therapies presents complex social and economic issues. Fully addressing these challenges requires engagement with patients and the public. A systematically conducted scoping review was undertaken to gauge current patient and public knowledge and perspectives, and as such inform requirements for future research, education and engagement activities.

Patient and public perspectives on cell and gene therapies: a systematic review.

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.