Health Service Perspective Research Articles

Comparative cost-effectiveness of termination of resuscitation rules for patients transported in cardiac arrest

AimTo compare the cost-effectiveness of termination-of-resuscitation (TOR) rules for patients transported in cardiac arrest. MethodsThe economic analyses evaluated cost-effectiveness of alternative TOR rules for OHCA from a National Health Service (NHS) and personal social services (PSS) perspective over a lifetime horizon. A systematic review was used to identify the different TOR rules included in the analyses. Data from the OHCAO outcomes registry, trial data and published literature were used to compare outcomes for the different rules identified. The economic analyses estimated discounted NHS and PSS costs and quality-adjusted life-years (QALYs) for each TOR rule, based on which incremental cost-effectiveness ratios (ICERs) were calculated. ResultsThe systematic review identified 33 TOR rules and the economic analyses assessed the performance of 29 of these TOR rules plus current practice. The most cost-effective strategies were the European Resuscitation Council (ERC) termination of resuscitation rule (ICER of £8,111), the Korean Cardiac Arrest Research Consortium 2 (KOC 2) termination of resuscitation rule (ICER of £17,548), and the universal Basic Life Support (BLS) termination of resuscitation rule (ICER of £19,498,216). The KOC 2 TOR rule was cost-effective at the established cost-effectiveness threshold of £20,000–£30,000 per QALY. ConclusionThe KOC 2 rule is the most cost-effective at established cost-effectiveness thresholds used to inform health care decision-making in the UK. Further research on economic implications of TOR rules is warranted to support constructive discussion on implementing TOR rules.

Cost-effectiveness of craniotomy versus decompressive craniectomy for UK patients with traumatic acute subdural haematoma

ObjectiveTo estimate the cost-effectiveness of craniotomy, compared with decompressive craniectomy (DC) in UK patients undergoing evacuation of acute subdural haematoma (ASDH).DesignEconomic evaluation undertaken using health resource use and outcome data...

EE309 Cost-Effectiveness Analysis of Velmanase Alfa in Patients with Alpha Mannosidosis from a UK Health Service Perspective

EE309 Cost-Effectiveness Analysis of Velmanase Alfa in Patients with Alpha Mannosidosis from a UK Health Service Perspective

A Model-Based Economic Evaluation of Hypothetical Treatments for Amyotrophic Lateral Sclerosis in the UK: Implications for Pricing of New and Emerging Health Technologies.

Amyotrophic lateral sclerosis (ALS) is a devastating disease which leads to loss of muscle function and paralysis. Historically, clinical drug development has been unsuccessful, but promising disease-modifying therapies (DMTs) may be on the horizon. The aims of this study were to estimate survival, quality-adjusted life-years (QALYs) and costs under current care, and to explore the conditions under which new therapies might be considered cost effective. We developed a health economic model to evaluate the cost effectiveness of future ALS treatments from a UK National Health Service and Personal Social Services perspective over a lifetime horizon using data from the ALS-CarE study. Costs were valued at 2021/22 prices. Two hypothetical interventions were evaluated: a DMT which delays progression and mortality, and a symptomatic therapy which improves utility only. Sensitivity analysis was conducted to identify key drivers of cost effectiveness. Starting from King's stage 2, patients receiving current care accrue an estimated 2.27 life-years, 0.75 QALYs and lifetime costs of £68,047. Assuming a 50% reduction in progression rates and a UK-converted estimate of the price of edaravone, the incremental cost-effectiveness ratio for a new DMT versus current care is likely to exceed £735,000 per QALY gained. Symptomatic therapies may be more likely to achieve acceptable levels of cost effectiveness. Regardless of efficacy, DMTs may struggle to demonstrate cost effectiveness, even at a low price. The cost effectiveness of DMTs is likely to be strongly influenced by drug price, the magnitude and durability of relative treatment effects, treatment starting/stopping rules and any additional utility benefits over current care.

Supporting self-management of low back pain with an internet intervention with and without telephone support in primary care (SupportBack 2): a randomised controlled trial of clinical and cost-effectiveness

Low back pain is prevalent and a leading cause of disability. We aimed to determine the clinical and cost-effectiveness of an accessible, scalable internet intervention for supporting behavioural self-management (SupportBack). Participants in UK primary care with low back pain without serious spinal pathology were randomly assigned 1:1:1 using computer algorithms stratified by disability level and telephone-support centre to usual care, usual care and SupportBack, or usual care and SupportBack with physiotherapist telephone-support (three brief calls). The primary outcome was low back pain-related disability (Roland Morris Disability Questionnaire [RMDQ] score) at 6 weeks, 3 months, 6 months, and 12 months using a repeated measures model, analysed by intention to treat using 97·5% CIs. A parallel economic evaluation from a health services perspective was used to estimate cost-effectiveness. People with lived experience of low back pain were involved in this trial from the outset. This completed trial was registered with ISRCTN, ISRCTN14736486. Between Nov 29, 2018, and Jan 12, 2021, 825 participants were randomly assigned (274 to usual care, 275 to SupportBack only, 276 to SupportBack with telephone-support). Participants had a mean age of 54 (SD 15), 479 (58%) of 821 were women and 342 (42%) were men, and 591 (92%) of 641 were White. Follow-up rates were 687 (83%) at 6 weeks, 598 (73%) at 3 months, 589 (72%) at 6 months, and 652 (79%) at 12 months. For the primary analysis, 736 participants were analysed (249 usual care, 245 SupportBack, and 242 SupportBack with telephone support). At a significance level of 0·025, there was no difference in RMDQ over 12 months with SupportBack versus usual care (adjusted mean difference -0·5 [97·5% CI -1·2 to 0·2]; p=0·085) or SupportBack with telephone-support versus usual care (-0·6 [-1·2 to 0·1]; p=0·048). There were no treatment-related serious adverse events. The economic evaluation showed that the SupportBack group dominated usual care, being both more effective and less costly. Both interventions were likely to be cost-effective at a threshold of £20 000 per quality adjusted life year compared with usual care. The SupportBack internet interventions did not significantly reduce low back pain-related disability over 12 months compared with usual care. They were likely to be cost-effective and safe. Clinical effectiveness, cost-effectiveness, and safety should be considered together when determining whether to apply these interventions in clinical practice. National Institute for Health and Care Research Health Technology Assessment (16/111/78).

Cost-effectiveness of atrial fibrillation detection with insertable cardiac monitors in large-artery or small-vessel disease stroke in the United Kingdom

Abstract Background The randomised STROKE-AF trial demonstrated clinical effectiveness of continuous cardiac monitoring to detect atrial fibrillation (AF) with insertable cardiac monitors (ICMs) for patients with ischemic stroke attributed to large-artery or small-vessel disease (LAD/SVD). ICMs were ten-fold more effective at detecting AF compared to the standard of care (SoC). However, cost-effectiveness of this monitoring strategy is unknown. Purpose The aim of this economic evaluation was to assess the cost-effectiveness of long-term continuous cardiac monitoring via ICMs, for detection of AF (and, in event of AF diagnosis, subsequent anticoagulation), in patients with ischemic stroke attributed to large-or-small vessel disease compared to the SoC of conventional follow up in the United Kingdom. Methods We constructed a Markov model with a cycle length of three months and a lifetime horizon to conduct the cost-effectiveness analysis, from the National Health Service (NHS) perspective. Costs and health-related benefits were discounted at an annual rate of 3.5%. We expressed outcomes in terms of quality-adjusted life-years (QALYs). Patient characteristics in the analysis and detection probabilities were based on the STROKE-AF trial. Detection of AF resulted in a switch in treatment from antiplatelet to direct oral anticoagulant (DOAC). Risk of cerebrovascular events were dependent on a patient’s AF detection status and subsequent treatment. Scenario, deterministic, and probabilistic sensitivity analyses were conducted to test the robustness of the base-case incremental cost-effectiveness ratio (ICER) and account for uncertainties in the model. We stratified patients into risk groups based on CHA2DS2-VASc scores and risk of AF detection. Results The base case incremental cost-effectiveness ratio of the implantation of an ICM was £15,485 per QALY, substantially under the NICE willingness-to-pay threshold of £20,000-£30,000 per QALY. ICM implantations resulted in 0.139 QALYs gained per patient at an incremental cost of £2,147. There were 0.054 fewer stroke events per patient in the ICM arm versus SoC. Results were robust under sensitivity analysis. Conclusion ICMs were cost-effective compared to SoC for detection of AF in patients with LAD/SVD stroke in the United Kingdom driven largely by more stroke events avoided. Subgroup analysis showed that ICMs were more cost-effective in patients with higher CHA2DS2-VASc scores and those with a higher individual risk of AF.Markov Model SchematicSummary ICER Results and Events avoided

Cost-Effectiveness of the Baveno VI Criteria Compared With Endoscopy for High-Risk Varices in Patients With Child-Pugh A Cirrhosis

Although upper gastrointestinal endoscopy (EGD) remains the gold standard for detecting varices in cirrhosis, the Baveno VI criteria proposed a combination of transient elastography and platelet count that could rule out high-risk varices, therefore sparing the need for an endoscopy, with significant potential cost savings. We performed a cost-effectiveness analysis of the Baveno VI criteria compared with EGD in the diagnosis of high-risk varices in cirrhosis. We built an analytical decision model to estimate the cost and benefits of using the Baveno VI criteria compared with EGD in patients with Child-Pugh A cirrhosis. The analysis was performed from the UK National Health Service perspective, over 1, 5, and 20 years. A Markov model was populated with data from published evidence. Outcomes were measured in terms of quality-adjusted life years (QALYs) and avoided deaths. The analyses were repeated for Canada and Spain, using relevant cost inputs. The Baveno VI criteria were cost effective compared with endoscopy in all analyses. For 1000 patients, they produced 0.16 additional QALYs at an incremental cost of £326 ($443.41) over 5 years, resulting in an incremental cost of £2081 ($2830) per additional QALY gained. The incremental net monetary benefit of Baveno VI compared with EGD was £2808 ($3819) over 5 years per patient. Baveno VI criteria also were cost effective in Canada and Spain. Deterministic and probabilistic sensitivity analysis supported these findings. The findings demonstrate that the Baveno VI criteria are cost effective, suggesting that they should be considered for widespread implementation on the basis of safety, appropriateness, and economic grounds.

Cost-effectiveness of methenamine hippurate compared with antibiotic prophylaxis for the management of recurrent urinary tract infections in secondary care: a multicentre, open-label, randomised, non-inferiority trial

ObjectivesTo estimate the cost-effectiveness of methenamine hippurate compared with antibiotic prophylaxis in the management of recurrent urinary tract infections.DesignMulticentre, open-label, randomised, non-inferiority trial.SettingEight centres in the UK, recruiting from June...

Digital variance angiography in patients undergoing lower limb arterial recanalization: cost-effectiveness analysis within the English healthcare setting.

Aim: Digital variance angiography (DVA) is a recently developed image processing method capable of improving image quality compared with the traditionally used digital subtraction angiography (DSA), among patients undergoing lower limb x-ray angiography. This study aims to explore the potential cost-effectiveness of DVA from an English National Health Service perspective. Materials & methods: A two-part economic model, consisting of a decision tree and a Markov model, was developed to consider the costs and health outcomes associated with the use of DVA as part of current practice imaging, compared with x-ray angiography using standard DSA. The model explored the impact of DVA on the development of acute kidney injury (AKI), chronic kidney disease and radiation-induced cancer over a lifetime horizon. Both deterministic and probabilistic analyses were performed to assess the cost per quality-adjusted life-year (QALY). Results: Base-case results indicate that DVA results in cost savings of £309 per patient, with QALYs also improving (+0.025) over a lifetime. As shown in sensitivity analysis, a key driver of model results is the relative risk (RR) reduction of contrast-associated acute kidney injury associated with use of DVA. The intervention also decreases the risk of carcinoma over a lifetime. Scenario analyses show that cost savings range from £310 to £553, with QALY gains ranging from 0.048 to 0.109 per patient. Conclusion: The use of DVA could result in a decrease in costs and an increase in QALYs over a lifetime, compared with existing imaging practice. The potential for this technology to offer an economically viable alternative to existing image processing methods, through a reduction in contrast media volume and radiation exposure, has been demonstrated.

Economic evaluation of a water fluoridation scheme in Cumbria, UK.

The addition of fluoride to community drinking water supplies has been a long-standing public health intervention to improve dental health. However, the evidence of cost-effectiveness in the UK currently lacks a contemporary focus, being limited to a period with higher incidence of caries. A water fluoridation scheme in West Cumbria, United Kingdom, provided a unique opportunity to study the contemporary impact of water fluoridation. This study evaluates the cost-effectiveness of water fluoridation over a 5-6 years follow-up period in two distinct cohorts: children exposed to water fluoridation in utero and those exposed from the age of 5. Cost-effectiveness was summarized employing incremental cost-effectiveness ratios (ICER, cost per quality adjusted life year (QALY) gained). Costs included those from the National Health Service (NHS) and local authority perspective, encompassing capital and running costs of water fluoridation, as well as NHS dental activity. The measure of health benefit was the QALY, with utility determined using the Child Health Utility 9-Dimension questionnaire. To account for uncertainty, estimates of net cost and outcomes were bootstrapped (10 000 bootstraps) to generate cost-effectiveness acceptability curves and sensitivity analysis performed with alternative specifications. There were 306 participants in the birth cohort (189 and 117 in the non-fluoridated and fluoridated groups, respectively) and 271 in the older school cohort (159 and 112, respectively). In both cohorts, there was evidence of small gains in QALYs for the fluoridated group compared to the non-fluoridated group and reductions in NHS dental service cost that exceeded the cost of fluoridation. For both cohorts and across all sensitivity analyses, there were high probabilities (>62%) of water fluoridation being cost-effective with a willingness to pay threshold of £20 000 per QALY. This analysis provides current economic evidence that water fluoridation is likely to be cost-effective. The findings contribute valuable contemporary evidence in support of the economic viability of water fluoridation scheme.

A Cost-Utility Analysis of Add-On Cannabidiol Versus Usual Care Alone for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in England and Wales.

The aim of this study was to evaluate the cost effectiveness of plant-derived highly purified cannabidiol (Epidyolex® in the UK; 100mg/mL oral solution) as an add-on treatment to usual care for the management of treatment-refractory seizures associated with tuberous sclerosis complex (TSC) in patients aged ≥2 years. A cohort-based model was developed using a National Health Service perspective and lifetime horizon. Health states were based on weekly seizure frequency and seizure-free days, utilizing patient-level data from the GWPCARE6 trial (ClinicalTrials.gov identifier: NCT02544763). Two independent regression models were applied to individual patient-level data to predict seizure-free days and seizure frequency. Healthcare resource utilization data were sourced from a Delphi panel, and patient and caregiver health-related quality of life values were elicited using vignettes valued by the general public. Outcomes relating to TSC-associated neuropsychiatric disorders were modeled with costs and quality-adjusted life-years sourced from published literature. In the base case, compared with usual care alone, 12mg/kg/day cannabidiol was associated with an incremental cost-effectiveness ratio (ICER) of £23,797. The National Institute for Health and Care Excellence disease severity modifier reduced the ICER to £19,831. Probabilities of cost effectiveness at willingness-to-pay thresholds of £20,000 and £30,000 were 30% and 52%, respectively, for the base case and 39% and 66%, respectively, for the disease severity modifier. Results were robust to sensitivity and scenario analyses. At 12mg/kg/day and an ICER threshold of £20,000-£30,000, we provide evidence for the cost effectiveness of add-on cannabidiol treatment for patients with TSC-associated seizures aged ≥2 years who are refractory to current treatment.

Effectiveness and cost-effectiveness of a web-based cardiac rehabilitation programme for people with chronic stable angina: protocol for the ACTIVATE (Angina Controlled Trial Investigating the Value of the ‘Activate your heart’ Therapeutic E-intervention) randomised controlled trial

IntroductionChronic stable angina is common and disabling. Cardiac rehabilitation is routinely offered to people following myocardial infarction or revascularisation procedures and has the potential to help people with chronic stable...

Talking in primary care (TIP): protocol for a cluster-randomised controlled trial in UK primary care to assess clinical and cost-effectiveness of communication skills e-learning for practitioners on patients’ musculoskeletal pain and enablement

IntroductionEffective communication can help optimise healthcare interactions and patient outcomes. However, few interventions have been tested clinically, subjected to cost-effectiveness analysis or are sufficiently brief and well-described for implementation in...

Risk-based lung cancer screening in heavy smokers: a benefit–harm and cost-effectiveness modeling study

BackgroundAnnual screening through low-dose computed tomography (LDCT) is recommended for heavy smokers. However, it is questionable whether all individuals require annual screening given the potential harms of LDCT screening. This study examines the benefit–harm and cost-effectiveness of risk-based screening in heavy smokers and determines the optimal risk threshold for screening and risk-stratified screening intervals.MethodsWe conducted a comparative cost-effectiveness analysis in China, using a cohort-based Markov model which simulated a lung cancer screening cohort of 19,146 heavy smokers aged 50 ~ 74 years old, who had a smoking history of at least 30 pack-years and were either current smokers or had quit for < 15 years. A total of 34 risk-based screening strategies, varying by different risk groups for screening eligibility and screening intervals (1-year, 2-year, 3-year, one-off, non-screening), were evaluated and were compared with annual screening for all heavy smokers (the status quo strategy). The analysis was undertaken from the health service perspective with a 30-year time horizon. The willingness-to-pay (WTP) threshold was adopted as three times the gross domestic product (GDP) of China in 2021 (CNY 242,928) per quality-adjusted life year (QALY) gained.ResultsCompared with the status quo strategy, nine risk-based screening strategies were found to be cost-effective, with two of them even resulting in cost-saving. The most cost-effective strategy was the risk-based approach of annual screening for individuals with a 5-year risk threshold of ≥ 1.70%, biennial screening for individuals with a 5-year risk threshold of 1.03 ~ 1.69%, and triennial screening for individuals with a 5-year risk threshold of < 1.03%. This strategy had the highest incremental net monetary benefit (iNMB) of CNY 1032. All risk-based screening strategies were more efficient than the status quo strategy, requiring 129 ~ 656 fewer screenings per lung cancer death avoided, and 0.5 ~ 28 fewer screenings per life-year gained. The cost-effectiveness of risk-based screening was further improved when individual adherence to screening improved and individuals quit smoking after being screened.ConclusionsRisk-based screening strategies are more efficient in reducing lung cancer deaths and gaining life years compared to the status quo strategy. Risk-stratified screening intervals can potentially balance long-term benefit–harm trade-offs and improve the cost-effectiveness of lung cancer screenings.

Primary Sjögren's syndrome in Italy: Real-world evidence of a rare disease through administrative healthcare data

ObjectivesPrimary Sjögren's syndrome (pSS) is a systemic autoimmune disease with significant impact on morbidity, mortality, and quality of life. This study aimed to evaluate epidemiology, healthcare needs and related costs of pSS patients from the Italian National Health Service perspective. MethodsFrom the Fondazione Ricerca e Salute's database (∼5 million inhabitants/year), pSS prevalence in 2018 was calculated. Demographics, mean healthcare consumptions and direct costs at one year following index date (first in-hospital diagnosis/disease waiver claim) were analysed through an individual direct matched pair case-control analysis (age, sex, residency). ResultsIn Italy, 3.8/10,000 inhabitants were identified as affected by pSS (1,746 case: 1,746 controls) in 2018. In the year following index date, 53.7% of cases and 42.7% of controls received ≥1 drug (p<0.001); mean per capita cost was €501 and €161, respectively (p<0.01). At least one hospitalization occurred to 7.8% of cases and 3.9% of controls (p<0.001) with mean per capita costs of €416 and €129, respectively (p = 0.46). At least one outpatient specialist service was performed in 49.8% of cases and 30.6% of controls (p<0.001); mean per capita costs were €200 and €75, respectively (p<0.01). Overall, mean annual costs were €1,171 per case and €372 per control (p < 0.01). ConclusionAccording to results of this population-based study, the prevalence of pSS in Italy appears to be consistent with the definition of rare disease. Patients with pSS have higher pharmacological, in-hospital and outpatient specialist care needs, leading to three-times higher overall cost for the INHS, compared to the general population.

The health service perspective on determinants of success in allied health student research project collaborations: a qualitative study guided by the Consolidated Framework for Implementation Research

BackgroundA research culture in health care organisations is associated with improved healthcare performance. Allied health (AH) students undertake research training as part of their professional degree qualifications. This may include participation in research projects, sometimes undertaken in association with health services. Co-supervision of these projects by health service staff provides research capacity building opportunities and staff-centred outcomes for the individuals involved, as well as improvements in clinical knowledge and practice within the local area. Also, publications from these projects contribute to the wider evidence base. Identification of barriers and facilitators to engagement in, and conduct of, these projects may optimise systems for improved health service outcomes.MethodsThis formative evaluation used the Consolidated Framework for Implementation Research (CFIR) to guide analysis of qualitative data obtained from semi-structured interviews with health service-employed allied health professionals, including clinicians and research fellows, who had supervised students on clinical-related research placements within the previous five years.ResultsEleven AH clinicians described 18 collaborative projects with 24 students from five AH disciplines across four universities. Three health service-employed AH research fellows described their involvement in these and other student research projects. Twenty key determinant constructs were identified and mapped across all five CFIR domains. Facilitators included health service cosmopolitanism, project adaptability and implementation climate (compatibility). Health service-employed research fellows provided readiness for implementation and a facilitator for project execution. The main barriers identified were cost to staff in workload and personal time and aspects related to project complexity. Differing student characteristics affected the relative advantage of collaborative projects in positive and negative manners.ConclusionsThis study describes the facilitators and barriers to the conduct of collaborative AH student research projects. Addressing these determinants when establishing each new project may enable health services to optimise communication, role delineation and project success, and thus ultimately, healthcare performance and patient care.

An Economic Evaluation of a Streamlined Day-Case Atrial Fibrillation Ablation Protocol and Conventional Cryoballoon Ablation versus Antiarrhythmic Drugs in a UK Paroxysmal Atrial Fibrillation Population.

Symptom control for atrial fibrillation can be achieved by catheter ablation or drug therapy. We assessed the cost effectiveness of a novel streamlined atrial fibrillation cryoballoon ablation protocol (AVATAR) compared with optimised antiarrhythmic drug (AAD) therapy and a conventional catheter ablation protocol, from a UK National Health Service (NHS) perspective. Data from the AVATAR study were assessed to determine the cost effectiveness of the three protocols in a two-step process. In the first stage, statistical analysis of clinical efficacy outcomes was conducted considering either a three-way comparison (AVATAR vs. conventional ablation vs. optimised AAD therapies) or a two-way comparison (pooled ablation protocol data vs. optimised AAD therapies). In the second stage, models assessed the cost effectiveness of the protocols. Costs and some of the clinical inputs in the models were derived from within-trial cost analysis and published literature. The remaining inputs were derived from clinical experts. No significant differences between the ablation protocols were found for any of the clinical outcomes used in the model. Results of a within-trial cost analysis show that AVATAR is cost-saving (£1279 per patient) compared with the conventional ablation protocol. When compared with optimised AAD therapies, AVATAR (pooled conventional and AVATAR ablation protocols efficacy) was found to be more costly while offering improved clinical benefits. Over a lifetime time horizon, the incremental cost-effectiveness ratio of AVATAR was estimated as £21,046 per quality-adjusted life-year gained (95% credible interval £7086-£71,718). The AVATAR streamlined protocol is likely to be a cost-effective option versus both conventional ablation and optimised AAD therapy in the UK NHS healthcare setting.

Cost-utility analysis of sequential therapy with sorafenib followed by regorafenib versus single-line therapies in advanced hepatocellular carcinoma.

529 Background: Regorafenib is the only licensed second-line therapy available for patients with advanced hepatocellular carcinoma (HCC) that progress on first-line sorafenib in the UK. We evaluated the lifetime cost-effectiveness of a sequence of treatment with sorafenib followed by regorafenib (SOR-REG) compared with licensed single-line therapies for which there is no clear sequencing options available, including atezolizumab plus bevacizumab (ATEZO+BEV), which has become the standard of care in first-line treatment, as well as lenvatinib (LEN), followed by best supportive care (BSC). Methods: Assuming a UK National Health Service and Social Services perspective, we developed a probabilistic individual-level semi-Markov model with states reflecting progression-free survival, progression following first-line treatment, further progression following second-line treatment, and death. First-line overall survival (OS) and progression free survival (PFS) were informed by fractional polynomials network meta-analysis of published trials. Individual patient data from a phase 3 trial (RESORCE) on regorafenib and BSC informed OS and PFS in the second-line setting. Costs and utilities were sourced from published studies. Cost-effectiveness was summarized using incremental net monetary benefit (INMB), which could be positive or negative depending on direction of expected benefit associated with SOR-REG, and cost-effectiveness acceptability curves at willingness-to-pay (WTP) thresholds. Results: We found 1000 probabilistic samples and 1000 patients were sufficient for convergence. Lifetime costs were highest with ATEZO-BEV ([£80,963 [95% CI: £11,022, £97,369]), followed by SOR-REG (£38,373 [£31,064, £43,420]) and LEN (£37,351 [£5,020, £55,800]). Quality-adjusted life years (QALYs) were highest with ATEZO+BEV (1.021 [0.152, 1.239), followed by SOR-REG (0.942 [0.802, 1.057]) and LEN (0.755, [0.094, 1.092]). At a WTP threshold of £30,000 / QALY gained, SOR-REG had an INMB of £40,211 (95% CI -£2,099, £52,731) and £4,589 (-£8,044, £14,481) versus ATEZO+BEV and LEN, respectively. Conclusions: A sequence of SOR-REG for patients with advanced HCC is cost-effective at WTP thresholds of £30,000 / QALY gained due to lower lifetime costs and broadly comparable QALY gains with single-line ATEZO-BEV and LEN in the UK. Broader second-line treatment options are needed for patients receiving ATEZO+BEV and LEN as first-line therapies.

Estimating the value of future research into thromboprophylaxis for women during pregnancy and after delivery: a value of information analysis

BackgroundRisk assessment models (RAMs) are used to select women at increased risk of venous thromboembolism (VTE) during pregnancy and the puerperium for thromboprophylaxis. ObjectivesTo estimate the value of potential future studies that would reduce the decision uncertainty associated with offering thromboprophylaxis according to available RAMs in the following groups: high-risk antepartum women (eg, prior VTE), unselected postpartum women, and postpartum women with risk factors (obesity or cesarean delivery). MethodsA decision-analytic model was developed to simulate clinical outcomes, lifetime costs, and quality-adjusted life-years for different thromboprophylaxis strategies, including thromboprophylaxis for all, thromboprophylaxis for none, and RAM-based thromboprophylaxis. The expected value of perfect information analysis was used to determine which factors are associated with high decision uncertainty. The value of future research studies was estimated using expected value of sample information analysis. Costs were assessed from a health and social services perspective. ResultsThe expected value of perfect information analysis identified high decision uncertainty for high-risk antepartum women (£21.8 million) and obese postpartum women (£13.4 million), which was largely attributable to uncertainty regarding the effectiveness of thromboprophylaxis in reducing VTE. A randomized controlled trial of thromboprophylaxis compared with none in obese postpartum women is likely to have substantial value (£2.8 million; 300 participants per arm). A trial in women with previous VTE would have higher value but would be less acceptable. ConclusionFuture research should focus on estimating the effectiveness of thromboprophylaxis in obese postpartum women with additional risk factors who have not had a previous VTE.

Cost-Utility Analysis of Total Ankle Replacement Compared with Ankle Arthrodesis for Patients Aged 50-85 Years with End-Stage Ankle Osteoarthritis: The TARVA Study.

Patients with end-stage ankle osteoarthritis suffer from reduced mobility and quality of life and the main surgical treatments are total ankle replacement (TAR) and ankle fusion (AF). Our aim was to calculate the mean incremental cost per quality-adjusted life-year (QALY) of TAR compared with AF in patients with end-stage ankle osteoarthritis, over 52 weeks and over the patients' lifetime. We conducted a cost-utility analysis of 282 participants from 17 UK centres recruited to a randomised controlled trial (TARVA). QALYs were calculated using index values from EQ-5D-5L. Resource use information was collected from case report forms and self-completed questionnaires. Primary analysis was within-trial analysis from the National Health Service (NHS) and Personal Social Services (PSS) perspective, while secondary analyses were within-trial analysis from wider perspective and long-term economic modelling. Adjustments were made for baseline resource use and index values. Total cost at 52 weeks was higher in the TAR group compared with the AF group, from the NHS and PSS perspective (mean adjusted difference £2539, 95% confidence interval [CI] £1142, £3897). The difference became very small from the wider perspective (£155, 95% CI - £1947, £2331). There was no significant difference between TAR and AF in terms of QALYs (mean adjusted difference 0.02, 95% CI - 0.015, 0.05) at 52 weeks post-operation. The incremental cost-effectiveness ratio (ICER) was £131,999 per QALY gained 52 weeks post-operation. Long-term economic modelling resulted in an ICER of £4200 per QALY gained, and there is a 69% probability of TAR being cost effective at a cost-effectiveness threshold of £20,000 per QALY gained. TAR does not appear to be cost effective over AF 52 weeks post-operation. A decision model suggests that TAR can be cost effective over the patients' lifetime but there is a need for longer-term prospectively collected data. Clinical trial registration ISRCTN60672307 and ClinicalTrials.gov NCT02128555.