Presentation (CTI)

OBJECTIVES: Cost-utility analyses of biologics in chronic disease typically extrapolate long-term treatment response from short trial follow-up, introducing uncertainty into lifetime estimates. Prior dupilumab analyses for severe chronic rhinosinusitis with nasal polyps (CRSwNP) relied on expert opinion or simple extrapolation beyond the one-year trial duration. This analysis used the AROMA global real-world registry (NCT04959448) to inform long-term response to dupilumab, replacing extrapolation-based assumptions in the National Institute for Health and Care Excellence (NICE) appraisal TA1134.
METHODS: A lifetime Markov model with a 1-year decision tree (NHS and Personal Social Services [PSS] perspective; 3.5% discount rate) compared dupilumab plus established clinical management (ECM) versus ECM alone in adults with ≥1 prior sinus surgery and 22-item sinonasal outcomes test (SNOT-22) score ≥50. Short-term efficacy (weeks 0-52) was derived from pooled SINUS-24/-52 trials (NCT02912468/NCT02898454). From year 2, sustained response to dupilumab was estimated from AROMA registry data propensity-matched to the SINUS trials, using a linear trendline excluding the first 12 months; registry response was based on maintained SNOT-22 improvement (≥8.9 points) without rescue therapy (systemic corticosteroids/sinus surgery), as nasal polyp score was not collected. This was compared with a conventional trial-extrapolated approach.
RESULTS: By 5 years, modelled loss of disease control with dupilumab reached ~28% under constant trial extrapolation versus ~20% under the AROMA-informed method; by 10 years, ~52% versus ~28%, reflecting more durable real-world response. NICE accepted the AROMA-informed long-term efficacy in TA1134; the committee’s preferred probabilistic incremental cost-effectiveness ratio was £24,846 per QALY gained, within the range NICE considers cost-effective.
CONCLUSIONS: Disease-specific registries can replace modelled long-term extrapolation in cost-effectiveness analyses (CEAs), reducing reliance on expert opinion or arbitrary extrapolation. NICE acceptance of AROMA-informed long-term efficacy demonstrates that real-world registry evidence can meet HTA standards with appropriate matched cohort methodology, with implications for biologic CEAs in other chronic inflammatory diseases.