2025/01/01 by Harshini Hariram, Sean P. Gavan
Economics, Econometrics and Finance · Medicine · #Biomedical Ethics and Regulation #CAR-T cell therapy research #Health Systems, Economic Evaluations, Quality of Life
paper · pdf · doi:10.1017/s0266462325100391
openalex publication_date 2025/01/01 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/02
Abstract Objectives Evaluate the extent to which delivery constraints were considered during the health technology assessment (HTA) of cell and gene therapies. Methods Constraints on delivering cell and gene therapies were identified from guidance documents by the National Institute for Health and Care Excellence Technology Appraisal and Highly Specialised Technologies streams until October 2024. Inductive coding was performed to identify delivery constraints reported within the guidance documents. A quantitative analysis established the proportion of guidance documents that reported delivery constraints, and the distribution of these constraints across the guidance documents (frequency, mean range). Results Sixteen guidance documents for cell and gene therapies were identified. Thirteen guidance documents (81.3 percent of the sample) reported constraints on delivering cell and gene therapies. Thirty-one examples of delivery constraints were reported. The mean number of constraints per guidance document was 1.9 (range: 0–6 constraints). The reported constraints were grouped by six different themes: provider experience ( n = 8); testing constraints ( n = 7); geographical constraints ( n = 5); payment constraints ( n = 5); maturity of developments in care ( n = 4); and infrastructure constraints ( n = 2). Conclusion Formal HTA processes are one effective way to identify constraints on delivering cell and gene therapies. Proactive identification of potential delivery constraints will help decision-makers, providers, and manufacturers generate strategies that improve the implementation of cell and gene therapies. Overcoming delivery constraints will strengthen the likelihood of realizing the expected incremental net health benefit of cost-effective cell and gene therapies for patients across a healthcare system.