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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.
BACKGROUND AND AIMS: The NICE Highly Specialised Technology (HST) programme evaluates interventions for very rare conditions within the UK. This review aimed to analyse previous NICE HST appraisals and determine commonly used methods to overcome uncertainties relating to health-related quality of life (HRQoL) and disease burden for people with rare diseases and their caregivers. The review also aimed to identify areas where further methodological development is required.
APPROACH AND RESULTS: A targeted review of all previous NICE HST appraisals published by the 28th February 2022, in which at least one committee meeting had taken place, was conducted. A total of 24 appraisals were included (17 fully completed and seven ongoing). Data were extracted by one reviewer. The evidence review group (ERG) and committee comments were compared against the NICE reference case and synthesised to identify the following methodological uncertainties that occurred most commonly: using alternatives to the EuroQol-5 Dimension (EQ-5D), sourcing HRQoL data from single-arm studies, measuring caregiver disutilities and estimating disease burden.
CONCLUSIONS: This review has highlighted the need for new methodology to reflect the impact of the diseases on people with rare diseases and their families. The review identified the following methodological requirements: alternative approaches that should be used when EQ-5D is not appropriate, methods to evaluate paediatric HRQoL and methods to quantify disease burden. This review also highlights the need to establish clear recommendations on the estimation of utilities across different rare diseases.
