Hariram Harshini, Gavan Sean P
Manchester Centre for Health Economics, Division of Population Health, Health Services Research and Primary Care, School of Health Sciences, Faculty of Biology, Medicine, and Health, https://ror.org/027m9bs27The University of Manchester, Manchester, UK.
Medical Student, Division of Medical Education, School of Medical Sciences, Faculty of Biology, Medicine and Health, The University of Manchester, Manchester, UK.
Int J Technol Assess Health Care. 2025 Jul 29;41(1):e56. doi: 10.1017/S0266462325100391.
Evaluate the extent to which delivery constraints were considered during the health technology assessment (HTA) of cell and gene therapies.
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).
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 ( = 8); testing constraints ( = 7); geographical constraints ( = 5); payment constraints ( = 5); maturity of developments in care ( = 4); and infrastructure constraints ( = 2).
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.
评估在细胞和基因疗法的卫生技术评估（HTA）过程中对递送限制因素的考虑程度。
从英国国家卫生与临床优化研究所技术评估和高度专业化技术领域的指导文件中识别出细胞和基因疗法的递送限制因素，截至2024年10月。采用归纳编码法识别指导文件中报告的递送限制因素。进行定量分析以确定报告递送限制因素的指导文件比例，以及这些限制因素在指导文件中的分布情况（频率、平均范围）。
确定了16份细胞和基因疗法的指导文件。13份指导文件（占样本的81.3%）报告了细胞和基因疗法递送方面的限制因素。共报告了31个递送限制因素的例子。每份指导文件的限制因素平均数为1.9（范围：0 - 6个限制因素）。报告的限制因素按六个不同主题分组：提供者经验（n = 8）；检测限制（n = 7）；地理限制（n = 5）；支付限制（n = 5）；护理发展成熟度（n = 4）；以及基础设施限制（n = 2）。
正式的HTA流程是识别细胞和基因疗法递送限制因素的一种有效方法。主动识别潜在的递送限制因素将有助于决策者、提供者和制造商制定改善细胞和基因疗法实施的策略。克服递送限制将增加在整个医疗系统中实现具有成本效益的细胞和基因疗法预期增量净健康效益的可能性。
