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. 2025 Jul 29;41(1):e56.
doi: 10.1017/S0266462325100391.

Constraints on delivering cell and gene therapies identified during technology appraisal by the National Institute for Health and Care Excellence

Harshini Hariram  1   2 , Sean P Gavan  1
Affiliations

Constraints on delivering cell and gene therapies identified during technology appraisal by the National Institute for Health and Care Excellence

Harshini Hariram et al. Int J Technol Assess Health Care. .

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.

Keywords: advanced therapy medicinal product; cell therapy; constraint; gene therapy; implementation.

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Conflict of interest statement

HH declares no conflicts of interest. SPG declares that his institution has received research grants from the National Institute for Health and Care Research (NIHR), Innovate UK, and LifeArc for research into the health economics of cell and gene therapies outside of the submitted work.

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