The Use of Real-World Data for Estimating Relative Treatment Effects in NICE Health Technology Assessment Submissions: A Review

Yoojung Che (), Stephen Duffield and Manuel Gomes
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Yoojung Che: Research Department of Primary Care and Population Health, University College London
Stephen Duffield: National Institute for Health and Care Excellence
Manuel Gomes: Research Department of Primary Care and Population Health, University College London

PharmacoEconomics, 2025, vol. 43, issue 2, No 1, 123-131

Abstract: Abstract This paper investigates the current use of real-world data (RWD) for estimating relative treatment effects in National Institute for Health and Care Excellence (NICE) health technology assessment (HTA) submissions. This review included 64 HTA submissions, which accounted for approximately 11% of the total NICE HTA submissions between January 2016 and December 2023. The main sources of RWD considered in the submissions were disease registries and electronic health records. RWD were primarily used to provide an external control arm to enable comparisons within single-arm trials and to inform long-term treatment effects when extrapolating survival data beyond the trial follow-up. Adjustments for potential systematic differences between treatment groups have improved over time; however, approximately one-third of the submissions still relied on unadjusted treatment comparisons. We found that approximately 10% of NICE HTA submissions used RWD to directly inform treatment effects estimation. Over one-third of the submissions relied on naïve and/or unadjusted treatment comparisons, which may have introduced biases. To ensure that RWD provide sound evidence for HTA, submissions should follow published guidelines, including the NICE real-world evidence (RWE) framework.

Date: 2025
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DOI: 10.1007/s40273-024-01449-w

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