Presentation (CTI)

OBJECTIVES: Highly specialised technologies (HST) and advanced therapy medicinal products (ATMPs) are gaining market prominence. However, current value assessments often overlook their full lifecycle value. This study evaluate the lifecycle value of HST and ATMPs approved by the National Institute for Health Care and Excellence (NICE)—across both patent-protected and post-patent phases.
METHODS: We reviewed NICE technology appraisals of HST and ATMPs—including gene therapies—published between January 2021 and June 2024. Key data included product characteristics, eligible populations, quality-adjusted life years (QALYs), incremental cost-effectiveness ratios (ICERs), health system costs/savings and opportunity costs (£15,000/QALY threshold). We used data from the literature to inform expected post-patent market dynamics and to address gaps/redactions in NICE appraisal reporting. Costs and QALYs across the product lifecycle were simulated using a multi-cohort modelling approach.
RESULTS: Of 340 appraisals, 13 HSTs and 6 gene therapies met inclusion criteria. Median QALY gains (6.9) exceeded historical averages (2000-2020) for non-ATMPs (0.49). The median ICER (£100,000/QALY) was higher than that for non-ATMPs (£28,555). Despite small eligible populations, HSTs/ATMPs yield lifecycle value comparable to non-ATMPs via substantial QALY gains or cost savings. The median share of value accruing to manufacturers was 150% while median net population health effects of approval was a loss of 8,374 QALYs. Shares of value accruing to manufacturers varied widely across products reflecting differences in cost-effectiveness thresholds applied, health system savings and expected post-patent market dynamics. Findings were sensitive to the time until off-patent availability and to the cost of producing the HST/ATMP.
CONCLUSIONS: NICE approved HSTs/ATMPs often displaced more health than they delivered over the product lifecycle. This underscores critical trade-offs between incentivising the development of, and access to, these products and system-wide opportunity costs. This research also emphasises the importance ensuring that health systems can access affordable versions of these technologies at patent expiry.