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OBJECTIVE: We aimed to assess the carbon footprint and clinical outcomes of once-weekly semaglutide in people with overweight or obesity in the UK using a disease modelling approach.
METHODS: A per-patient carbon footprint analysis was conducted to estimate emissions related to obesity management with semaglutide 2.4 mg as an add on to diet and exercise versus diet and exercise alone. A Markov state-transition cohort model (Core Obesity Model) was used for the analysis. Data were sourced from STEP and SELECT trials across three populations: (1) body mass index [BMI] ≥ 30 or ≥ 27 with one or more obesity-related complications (BMI30+/BMI27+C), (2) BMI ≥ 27 with one or more complications, including type 2 diabetes mellitus (BMI27+C), and (3) BMI ≥ 27 with established cardiovascular disease (BMI27+CVD). Carbon emissions were estimated using resource-based and cost-based methods. Key outcomes included life-years, quality-adjusted life-years, and incremental carbon footprint effectiveness ratio.
RESULTS: In the BMI30+/BMI27+C and BMI27+C populations, semaglutide was dominant, yielding 0.30 and 0.25 additional life-years, 0.51 and 0.46 additional quality-adjusted life-years, while reducing lifetime carbon emissions by 1.8% (9850 vs 10,030 kg of CO2 equivalent [CO2e]) and 1.9% (9792 vs 9983 kg CO2e), respectively. Manufacturing emissions of semaglutide were offset by reductions in carbon emissions resulting from fewer obesity-related complications. In the BMI27+CVD population, semaglutide improved life-years by 0.56 and quality-adjusted life-years by 0.59, but emissions increased (14,700 vs 14,444 kg CO2e) because of longer survival and increased monitoring. Scenario and sensitivity analyses confirmed the robustness of the results.
CONCLUSIONS: Semaglutide offers both clinical and environmental benefits in obesity management, supporting the UK’s net-zero emissions goals.
BACKGROUND: Interpreting meaningful change in patient-reported outcome measures (PROMs) is essential for assessing hip replacement outcomes. The Oxford Hip Score (OHS) is widely used, yet statistically detectable change may not align with patient-perceived improvement.
OBJECTIVES: To compare Rasch-based classifications of OHS change, following Hobart’s effect size methodology, with anchor-based interpretations derived from patient Global Impression of Change (GIC).
METHODS: OHS data from a large hip replacement cohort (N = 184,509) were analysed using two complementary approaches. Rasch analysis transformed ordinal scores into interval-level person measures, and effect sizes with confidence intervals classified patients as significantly improved, non-significantly improved, unchanged, or worse. MIC was estimated using a five-point patient GIC. Median pre-operative, post-operative, and change scores were summarised per category.
RESULTS: Both methods demonstrated graded outcome patterns. Concordance was strongest for large improvements, with statistically significant change closely matching reports of being “much better.” Divergence occurred for modest improvement and worsening: patients often reported little or no benefit despite measurable OHS improvement.
CONCLUSIONS: Rasch-based and anchor-based approaches capture overlapping but distinct constructs. Integrating both provides a nuanced interpretation of PROM change after hip replacement.
Missing Health-Related Quality of Life (HRQoL) data in clinical studies risk propagating bias into health technology assessments (HTAs) and cost-utility analyses. Despite this, current National Institute for Health and Care Excellence (NICE) guidance offers no specific recommendations for handling missing HRQoL values. Using Monte Carlo simulations (1000 datasets), this study evaluated nine imputation methods, across the missing completely at random (MCAR), missing at random (MAR) and missing not at random (MNAR) assumptions at levels ranging from 5% to 50%. Performance was assessed using bias, variance, and coverage of the true HRQoL mean. While multiple imputation by chained equations (MICE)-based approaches performed best under MCAR and MAR, all methods showed bias under MNAR, with a delta-pattern mixture model performing the best (relative bias ≤2.1% at all missingness levels but coverage falls to 35.4% at 50% missingness). The choice of imputation method is key to preventing biased results from propagating into cost-effectiveness analysis, which in theory may lead to suboptimal reimbursement decisions and inefficient healthcare spending. To address the lack of explicit guidance from HTA bodies, we have developed a preliminary policy that could be used for HTA submissions: If the missingness pattern is not known and missingness ≤5%, it is suggested that most methods (except GLM) are acceptable (though care should be taken when using CCA and LOCF if MNAR is suspected). It is also suggested that MICE-based techniques are used as the base case for missingness >5%, and delta-PMM used as a sensitivity analysis when data are not MCAR. Further simulation studies would be required to strengthen the suggestions in this preliminary policy; however, the development of universal recommendations would lead to improved consistency and reliability of HTA globally.
BACKGROUND: RSVpreF is a vaccine that immunises against respiratory syncytial virus (RSV), a seasonal respiratory virus, that can lead to lower respiratory tract infections. In Switzerland, RSVpreF is indicated for the passive protection of infants against RSV following maternal immunisation.
OBJECTIVE: The Swiss Federal Office of Public Health commissioned a health technology assessment (HTA) of RSVpreF for maternal vaccination, evaluating the clinical and cost effectiveness of the vaccine compared with nirsevimab (administered directly to newborns and infants) or no RSV prophylaxis. This study reports the cost-effectiveness model developed for the Swiss HTA.
METHODS: The model used a Markov model structure, a 1-year time horizon, a one-month cycle length, a Swiss healthcare payers' perspective, and a 3% discount rate for costs and health benefits. Health states included no RSV infection, RSV infection leading to hospitalisation, primary care visits or emergency department visits, post-RSV infection, and death. MATISSE informed RSVpreF efficacy, and MELODY and HARMONIE informed nirsevimab efficacy. Health outcomes were expressed as quality-adjusted life years (QALYs). Probabilistic and deterministic sensitivity analyses were undertaken to explore the impact of parameter uncertainty on the model results.
RESULTS: When compared with nirsevimab, RSVpreF reduces total costs (CHF −9,878,738) and QALYs (−18.4), with an incremental cost-effectiveness ratio (ICER) of 536,008 per QALY (N = 38,844). Compared with no RSV prophylaxis, RSVpreF increases total costs (CHF +470,441) and QALYs (+27.1), with an ICER of 17,377 per QALY (N = 38,844).
CONCLUSIONS: RSVpreF is highly likely to be cost-effective versus both comparators at a range of plausible threshold values. The results of the model contributed to guidance updates in Switzerland, with RSVpreF now reimbursed by mandatory health insurance.
PURPOSE: Qualitative accounts and studies employing disease-specific outcome measures have demonstrated that people with dystrophic epidermolysis bullosa (DEB) display impaired health-related quality of life (HRQoL) due to wound-related challenges. There is a paucity of utility data necessary to inform the health economic evaluation of emerging treatments for cutaneous open wounds.
METHODS: The objective was to implement a vignette study with a representative sample of the United Kingdom (UK) general population (N = 1139) and people with DEB (N = 44) to estimate utility values. Vignettes were defined by various attributes, including the number and area of wounds, pain, itch and hours spent managing wounds. Participants valued the vignettes from the perspective of a patient or a caregiver proxy using the EQ-5D-3L or EQ-5D-Y-3L, respectively.
RESULTS: The mean utilities elicited from the patient perspective ranged from 0.491 to − 0.448 and 0.457 to − 0.462 for the general population (N = 627) and DEB (N = 31) sample, respectively. The utilities elicited from the proxy perspective ranged from 0.443 to − 0.543 and 0.079 to − 0.550 in the general population (N = 512) and DEB (N = 13) sample, respectively.
CONCLUSION: The findings join a limited evidence base indicating that the symptoms associated with the wounds experienced by people with DEB have a considerable detrimental effect on their HRQoL.
AIMS: Asymptomatic severe aortic stenosis (aSAS) is associated with morbidity, with mortality increasing once symptoms develop. Benefits of early aortic valve replacement (AVR) over clinical surveillance (CS) with delayed AVR upon symptom onset have been demonstrated, and it is now recommended from the latest European Guidelines if procedural risk is low (Class IIa, Level A). The aim of this study is to estimate the economic impact of transcatheter aortic valve implantation (TAVI) for the treatment of aSAS.
METHODS AND RESULTS: A cost-utility analysis compared TAVI using the SAPIEN 3/SAPIEN 3 Ultra valve vs. CS in patients with aSAS across nine European countries. A lifetime Markov model captured peri-procedural and long-term outcomes across three health states: alive and well, stroke, and death. Inputs were derived from the EARLY TAVR trial (NCT03042104) and literature sources. Clinical event rates were estimated via parametric survival analysis. Transcatheter aortic valve implantation treatment for patients with aSAS was modelled to be the economically dominant strategy for treating aSAS across all nine European healthcare systems, with cost-effectiveness probabilities ranging from 97.3% (UK) to 99.9% (Belgium). Incremental results per person included cost savings from −£1788 (UK) to −CHF15 802 (Switzerland), quality-adjusted life years gains of 0.18 (Germany, UK) to 0.23 (Switzerland), and life-year gains of 0.12 (UK) to 0.17 (Belgium). Annual discounting of upfront TAVI costs vs. delayed AVR costs drove results.
CONCLUSION: For patients with aSAS, early intervention with TAVI is estimated to deliver greater health benefits and reduce costs compared with CS. These findings support policies promoting early detection and timely intervention before symptom onset.
AIM: Late-onset Pompe disease (LOPD) is a rare lysosomal disease primarily impacting muscle strength andrespiratory function. LOPD has a substantial burden despite the availability of alglucosidase alfa (alg). Patients often require mobility and respiratory support over time. Cipaglucosidase alfa in combinationwith miglustat (cipa + mig) is one of two more recently approved treatments for adults with LOPD. Given limited data on the lifetime trajectory to mobility and respiratory support in LOPD, a patient-level simulation model was developed to compare the long-term impact of cipa + mig with alg on these outcomes.
MATERIALS & METHODS: The patient-level simulation predicts lifetime mobility and respiratorydisease progression outcomes based on the 6-min walk distance and % predicted forced vital capacity for alg and cipa + mig for the overall LOPD population using available data and assumptions from experienced clinicians. PROPEL/PROPEL open-label extension (NCT03729362) and ATB200-02 (NCT02675465) studies informed outcomes for four years with cipa + mig and one year with alg. French Pompe disease registry data were used thereafter.
RESULTS: Based on the available data and clinical assumptions, the model predicts cipa + mig slows the overall progression of LOPD, allowing patients an additional 2.72 years without mobility or respiratory support compared with alg. People receiving alg may be wheelchair dependent and require invasive respiratory support for an additional 2.57 and 1.55 years, respectively.
CONCLUSION: Cipa + mig may delay disease progression compared with alg over the lifetime of a patient with LOPD, which would increase the amount of time spent without mobility and respiratory support dependency.
