Our latest research, all in one place. Browse our collection of journal articles, reports and conference proceedings to see how we’re contributing to HEOR research. Remember to:
OBJECTIVES: Publications that report early Kaplan Meier (KM) from a clinical trial are useful for understanding how accurate trends seen in immature data are to that of end-of-trial publications. This immature data may not be reported or available. This de novo approach aimed to recreate early trial data from full published KM. If successful, this approach may be used to adjust trial evidence to reflect an earlier timepoint, allowing for before and after comparisons so that analysts can observe predictive data trends and characteristics.
METHODS: Published five-year KM from the ZUMA-1 trial for axicabtagene ciloleucel CAR-T therapy in refractory large B-cell lymphoma was digitised and pseudo individual participant data (IPD) was generated using the Guyot algorithm. The aim was to produce IPD that reflected 12-month, 18-month and 24-month minimum follow-ups, with the proportion censored at a given time informed by earlier ZUMA-1 trial publications.
The censoring indicator was changed until the desired proportion censored was reached, starting with the latest reported events in the five-year dataset. A truncated normal distribution was assumed when assigning the relevant follow-up time for the newly censored points. Generated early datasets were plotted and summary statistics produced, which were compared to equivalent outputs of early ZUMA-1 publications.
RESULTS: The generated truncated datasets resulted in KM that visually matched the KM reported in early ZUMA-1 publications. Median survival was not met in either dataset. The total number of events is 49 in the generated truncated 24-months minimum follow-up dataset and 50 in the ZUMA-1 early publication with 27-month median follow-up.
CONCLUSIONS: Publications reporting trial KM can be truncated to recreate earlier datasets to a reasonably accurate degree. A key feature of the published KM that allowed for this truncation was the small range from minimum to maximum follow-up timepoint, as to narrow the applied censoring to a more accurate range.
OBJECTIVES: There is a lack of consensus and transparency on using real-world evidence (RWE) in health technology assessment (HTA). In 2022, the National Institute for Health and Care Excellence (NICE) developed an RWE framework. This study explores the use of RWE databases in NICE technology appraisals (TAs) from April 2019.
METHODS: The NICE website was searched to source TAs from April 2019 to May 2024 that mentioned at least 1 of 10 identified RWE databases. Information on how RWE was used and how it was viewed by the External Assessment Group (EAG) and NICE committee were extracted.
RESULTS: 13 TAs mentioned at least 1 of the databases: Clinical Practice Research Datalink (12 TAs), OpenSAFELY (3 TAs), The Health Improvement Network (1 TA) and General Practice Extraction Service Data for Pandemic Planning and Research (1 TA). In 12 TAs, RWE data were used to inform or validate model parameters. In all TAs screened, the committee and EAG did not criticize the use of RWE data extracted from databases. Moreover, in three cases the EAG utilized RWE data to inform alternative base case parameter estimates. In two cases, the committee recommended the manufacturer use RWE data to validate a model assumption. Comments from the committee focused on the relevance and applicability of the RWE to the decision problem.
CONCLUSIONS: It is evident that decision makers respond positively to RWE when it has been correctly employed. For RWE to aid the decision-making process, data must be applicable and relevant to the population of interest. Correct utilization of RWE yields the potential to reduce parameter uncertainty. This study supports the use of RWE in HTA and demonstrates that a standard framework can ensure appropriate use of RWE. Future research could explore RWE use in other HTA agencies to strengthen the current study and broaden its applicability.
OBJECTIVES: The aim of this study was to investigate whether it is possible to elicit how United Kingdom (UK) public citizens might trade off human health and environmental outcomes.
METHODS: Using a representative adult population, a discrete choice experiment (DCE) was conducted on criteria impacting trade-off decisions between human health and environmental outcomes. Respondents were asked to make twelve choices that included four attributes: the impact on UK life expectancy, the impact on biodiversity, the impact on UK carbon emissions and location of environmental impacts. Data were analyzed using a conditional logit regression model.
RESULTS: 508 respondents completed the survey. A DCE found UK public citizens are willing to forgo human health to reduce environmental harm.
CONCLUSIONS: This research demonstrated it is possible to elicit the public’s view about trade-offs between health and the environment. Moreover, the public is willing to forgo human health to reduce environmental impact, propounding the importance of healthcare sustainability.
INTRODUCTION: The standard treatment for gastroesophageal reflux disease (GERD) is proton pump inhibitors (PPIs). In selected cases, Nissen fundoplication is offered as a surgical treatment option, but alternative endoscopic and minimally invasive surgical alternatives are emerging. RefluxStop is a new technology for the treatment of GERD.
RESEARCH DESIGN AND METHODS: A cost-effectiveness analysis of RefluxStop in comparison to PPI therapy and Nissen fundoplication in the Swedish healthcare setting was conducted using a Markov model and available comprehensive population and clinical trial-based long-term data. Benefits were measured in quality-adjusted life-years (QALYs). Uncertainty was determined by deterministic and probabilistic sensitivity analyses.
RESULTS: The base case incremental cost-effectiveness ratios (ICERs) for RefluxStop in comparison to PPIs and Nissen fundoplications were SEK 48,152 (€ 4,531) and SEK 62,966 (€ 5,925) per QALY gained, respectively. At a cost-effectiveness threshold of SEK 500,000 per QALY gained, RefluxStop has a high likelihood of being cost-effective, with probabilities of 96% and 100% against Nissen fundoplication and PPIs, respectively. The results of the model remained robust with sensitivity analysis.
CONCLUSIONS: RefluxStop may offer a highly cost-effective long-term treatment alternative for chronic GERD patients over lifelong PPI therapy, but also in comparison with laparoscopic Nissen fundoplication.
BACKGROUND: Transcatheter aortic valve implantation (TAVI) has shown similar or improved clinical outcomes compared with surgical aortic valve replacement (SAVR) in patients with symptomatic severe aortic stenosis at low risk for surgical mortality. This cost-utility analysis compared TAVI with SAPIEN 3 versus SAVR in symptomatic severe aortic stenosis patients at low risk of surgical mortality from the perspective of the Swedish healthcare system.
METHODS: A published, two-stage, Markov-based cost-utility model that captured clinical outcomes from the Swedish Web-system for Enhancement and Development of Evidence-based care in Heart disease Evaluated according to Recommended Therapies (SWEDEHEART) registry (2018-2020) was adapted from the perspective of the Swedish healthcare system using local general population mortality, utility and costs data. The model had a lifetime horizon. Model outputs included changes in direct healthcare costs and health-related quality of life from using TAVI as compared with SAVR.
RESULTS: TAVI with SAPIEN 3 resulted in lifetime costs per patient of 940,541 Swedish krona (SEK) and lifetime quality-adjusted life years (QALYs) per patient of 7.16, whilst SAVR resulted in lifetime costs and QALYs per patient of 821,380 SEK and 6.81 QALYs, respectively. Compared with SAVR, TAVI offered an incremental improvement of +0.35 QALY per patient at an increased cost of +119,161 SEK per patient over a lifetime horizon, resulting in an incremental cost-effectiveness ratio of 343,918 SEK per QALY gained.
CONCLUSION: TAVI with SAPIEN 3 is a cost-effective option versus SAVR for patients with symptomatic severe aortic stenosis at low risk for surgical mortality treated in the Swedish healthcare setting. These findings may inform policy decisions in Sweden for the management of this patient group.
