Lifetime cost-effectiveness of first metatarsophalangeal joint fusion using patient reported outcomes from National Foot and Ankle Registry data in the UK.

ABSTRACTBackgroundThis study investigated the quality and clinical outcomes of the British Orthopaedic Foot and Ankle Society (BOFAS) registry first metatarsophalangeal joint (MTPJ) arthrodesis pathway.MethodsA prospective cohort study using data derived from the BOFAS registry. Adults aged ≥ 18 years with a record of undergoing first MTPJ arthrodesis in the UK from 29/08/2014 to 31/10/2019. The pre‐ and post‐treatment pathway was evaluated by analysing Patient Reported Outcome Measures (PROMs) at baseline, 6 months and 12 months intervals. Consistency of data capture and completeness were explored using means, SD, medians and IQR for continuous variables and frequencies for categorical variables.ResultsThe mean age of the study population (n = 459) was 64.1 (± 12.1) years and 98.9% of the study cohort were female. Completeness of data collection was low for some items (e.g., medication 46%, surgical procedures 52%). Baseline completion of PROMs was moderate with 52.5% of participants providing MOXFQ (Manchester–Oxford Foot Questionnaire) pain and walking/standing scores at baseline. However, follow‐up response rates declined substantially to 27.2% at 6 months and 15.7% at 12 months. Improvement in PROMs by 12 months following surgery was statistically and clinically significant (p < 0.001), with median scores of 10 [IQR: 0–20] for MOXFQ pain, 5.5 [0–21] for walking/standing, 0 [0–19] for social interaction, 5 [1–31] for NRS pain and 0.8 [0.7–1.0] for EQ‐5D‐5L.ConclusionThe analysis highlights the clinical benefits of first metatarsophalangeal joint (first MTPJ) fusion surgery, with improvements in pain intensity, walking/standing ability, social interaction and quality of life. The BOFAS registry serves as a valuable tool for collecting patient‐reported outcome measure (PROM) data, providing important insights into treatment effectiveness and participant well‐being. Strengthening the data collection capabilities of the BOFAS registry could further enhance our understanding of the benefits of first MTPJ fusion and inform future treatment strategies.

Using real world evidence to generate cost-effectiveness analysis of fibrinolytic therapy in patients with ST-segment elevation myocardial infarction in Thailand

BackgroundThe Quality Adjusted Life Year (QALY), based on health-related quality of life measures such as euroqol 5 dimensions (EQ-5D), is an important outcome in rheumatoid arthritis (RA) studies, as it...

While smoking is common among those experiencing homelessness, the effectiveness of an e-cigarette intervention to reduce smoking in this population is unclear. To determine the cost-effectiveness of providing an e-cigarette for smoking cessation in homeless support centres compared to usual care. A multicentre two-arm cluster randomised controlled trial, with data collection time points at baseline, 4, 12 and 24 weeks post baseline. Adults (aged 18+) who smoked daily and accessed 32 homeless support centres across six areas of Great Britain received either e-cigarette intervention (n = 239 in 16 centres) or usual care (n = 236 in 16 centres) by centre (cluster) randomisation. The intervention was the provision of an e-cigarette starter kit plus 4 weeks' supply of e-liquids. The usual care comprised very brief advice for smoking cessation and signposting to local Stop Smoking Services. The total costs included costs of intervention/usual care, costs of smoking cessation outside of the trial and costs of general healthcare services use over 24 weeks. Quality-adjusted life-years were derived from EuroQol-5 Dimensions, five-level version administered at each data collection point. An incremental cost-effectiveness ratio was calculated for 24 weeks using the difference between groups in total costs and quality-adjusted life-years, with cost-effectiveness acceptability curve constructed based on bootstrap to examine uncertainty. A long-term model was employed to project a lifetime incremental cost-effectiveness ratio with probabilistic sensitivity analysis to examine uncertainty. The analysis over 24 weeks was based on research team records and data collected via self-reported questionnaires. Unit costs for valuation were extracted from published secondary sources. The parameters of the long-term model were based on the 24-week results and published secondary sources. Mean intervention costs were estimated at £92 [standard error (SE) £0] per participant and mean usual care costs at £50 (SE £0) per participant. Mean total costs per participant were estimated at £3859 (SE £441) in the e-cigarette group and £2716 (SE £386) in the usual care group. Mean quality-adjusted life-years were estimated at 0.303 (SE 0.008) in the e-cigarette group and 0.295 (SE 0.010) in the usual care group. Adjusting for baseline covariates and respective baseline values, e-cigarette group were £1267 (95% confidence interval £600 to £1938) more costly and yielded 0.007 (95% confidence interval -0.017 to 0.027) more quality-adjusted life-years than usual care. The incremental cost-effectiveness ratio was calculated at £181,000 per quality-adjusted life-year gain, with probability of intervention being cost-effective between the incremental cost-effectiveness ratio thresholds of £20,000-30,000 per quality-adjusted life-year gain at 0.9-3.5%. The lifetime model projected the incremental cost-effectiveness ratio at £38,360 per quality-adjusted life-year gained, with the probability of intervention being cost-effective between £20,000 and £30,000 from 47.6% to 49.6%. The imbalance in missing data led to some uncertainty in the results, and healthcare costs recorded in the trial may not reflect the health needs of this population. Providing e-cigarettes for smoking cessation in homeless support centres was more costly than usual care, but the small increase in quality-adjusted life-years was not significant. Future work should aim to maximise quit rates while being cost-effective and therefore implementable. This article presents independent research funded by the National Institute for Health and Care Research (NIHR) Public Health Research programme as award number NIHR132158.

Background: The main results of COURAGE showed no differences in all-cause mortality or non-fatal MI (primary endpoint), the composite of death, MI or stroke, or hospitalization for ACS (secondary endpoints) during 4.6 year median follow-up in the 2,287 patients with stable coronary artery disease (CAD) randomized to optimal medical therapy (OMT) with or without percutaneous coronary intervention (PCI). There was a benefit to PCI in quality of life measured with the Seattle Angina Questionnaire. This study focuses on cost-effectiveness of PCI. Methods: Resource utilization including initial and follow-up hospitalizations were assigned a DRG and then costs assigned from Medicare reimbursement. Medication costs were assessed from Redbook average wholesale price. Survival was estimated for patients from Framingham data. Survival was quality adjusted from utility measured by standard gamble. Cost and outcome were discounted 3%. Costs after the trial period are Medicare average costs. Cost-effectiveness is expressed as an incremental cost effectiveness ratio (ICER), of cost per life year (LY) or quality adjusted life year (QALY) gained. The distribution of the ICER was assessed by bootstrap. Results: The added cost of PCI is approximately $10,000, without significant gain in LY or QALYs. The ICER varied from just over $150,000 to just under $300,000 per LY or QALY gained with PCI. A large minority of the distributions of the ICERs found PCI to be dominated by OMT, that is OMT offering better outcome at lower cost. Conclusions: The addition of PCI to OMT as an initial management strategy costs significantly more without offering an advantage in survival or QALYs. The ICER is high compared to conventional benchmarks, and the distribution includes infinity and low probability of being below $50,000/QALY gained. PCI in addition to OMT is not a cost-effective initial management strategy for symptomatic, chronic coronary artery disease.

Gastric cancer is the fifth most common malignancy and second leading cause of cancer-related mortality. Chemotherapy options for patients who fail first-line treatment are limited. Thus the objective of this study was to assess the cost-effectiveness of second-line treatment options for patients with advanced or metastatic gastric cancer. Cost-effectiveness analysis using a Markov model to compare the cost-effectiveness of six possible second-line treatment options for patients with advanced gastric cancer who have failed previous chemotherapy: irinotecan, docetaxel, paclitaxel, ramucirumab, paclitaxel plus ramucirumab, and palliative care. The model was performed from a third-party payer's perspective to compare lifetime costs and health benefits associated with studied second-line therapies. Costs included only relevant direct medical costs. The model assumed chemotherapy cycle lengths of 30 days and a maximum number of 24 cycles. Systematic review of literature was performed to identify clinical data sources and utility and cost data. Quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs) were calculated. The primary outcome measure for this analysis was the ICER between different therapies, where the incremental cost was divided by the number of QALYs saved. The ICER was compared with a willingness-to-pay (WTP) threshold that was set at $50,000/QALY gained, and an exploratory analysis using $160,000/QALY gained was also used. The model's robustness was tested by using 1-way sensitivity analyses and a 10,000 Monte Carlo simulation probabilistic sensitivity analysis (PSA). Irinotecan had the lowest lifetime cost and was associated with a QALY gain of 0.35 year. Docetaxel, ramucirumab alone, and palliative care were dominated strategies. Paclitaxel and the combination of paclitaxel plus ramucirumab led to higher QALYs gained, at an incremental cost of $86,815 and $1,056,125 per QALY gained, respectively. Based on our prespecified WTP threshold, our base case analysis demonstrated that irinotecan alone is the most cost-effective regimen, and both paclitaxel alone and the combination of paclitaxel and ramucirumab were not cost-effective (ICER more than $50,000). Both 1-way sensitivity analyses and PSA demonstrated the model's robustness. PSA illustrated that paclitaxel plus ramucirumab was extremely unlikely to be cost-effective at a WTP threshold less than $400,000/QALY gained. Irinotecan alone appears to be the most cost-effective second-line regimen for patients with gastric cancer. Paclitaxel may be cost-effective if the WTP threshold was set at $160,000/QALY gained.

Evidence-based medicine within a budget -where should we spend our money?

First metatarsophalangeal (MTP) joint fusion is a widely accepted surgical intervention for treating severe arthritis, deformities, and instability of the first MTP joint. This paper provides a review of a single surgeon's experience with continuous compression implants (CCI), which offer a notable advantage by providing uniform compression across a larger surface area of the fusion site compared to plate and screw constructs. This design potentially reduces soft tissue irritation and, consequently, the need for subsequent implant removal. It also saves on cost and has the potential to reduce the length of surgery. A retrospective review was conducted on 27 patients (n=36 feet) who underwent primary first metatarsophalangeal joint (MTPJ) fusion using continuous compression implants (CCI) between March 2020 and April 2024 at Bradford Royal Infirmary. Patient data were collected from the surgeon's logbook and medical records. The outcomes analyzed included the fusion rate and complications. Statistical analysis was performed using SPSS version 22.0, with p<0.05 considered significant. The mean age of the cohort was 60.24 years (range 41-90), with 88.88% female. The ratio of left to right was 70%. The mean follow-up duration was 27 months (range 6-48 months).Complete fusion of the first MTPJ was achieved in 34 out of 36 feet (94.4%). Nonunion occurred in one patient, while delayed union was observed in another. Clinically, 35 out of 36 patients (97.3%) reported satisfaction with the procedure, with one patient requiring metalwork removal and revision due to loosening. Early results show that the rate of fusion achieved by using the CCI for the first MTPJ arthrodesis in our series was comparable to that of other devices quoted in the literature.

The clinical features of hepatocellular carcinoma (HCC) and the medical environment are diverse in different geographic areas. The aim of this study is to evaluate the cost-effectiveness of the surveillance of HCC in different medical circumstances. The Markov model focused on variables that differ from country to country and may change in the future, especially in regards to the proportion of small HCC detected incidentally. The target population was 45-year-old patients with Child-Pugh class A cirrhosis, and the intervention was surveillance with ultrasonography every 6 months. The additional cost of the surveillance was $US15 100, the gain in quality-adjusted life years (QALYs) was 0.50 years, and the incremental cost-effectiveness ratio (ICER) was $US29 900/QALY in a base-case analysis (annual incidence of HCC = 4%). If 40% of small HCC were detected incidentally without surveillance, the gain in QALY decreased to 0.15 and the ICER increased to $US47 900/QALY. The increase in the annual incidence of HCC to 8% resulted in the increase of QALYs to 0.81, and the decrease of the ICER to $US25 400/QALY. The adoption of liver transplantation increased the gain in QALYs and the ICER to 0.84 and $US59 900/QALY, respectively. The gain in QALYs and the ICER due to the surveillance of HCC varies between different patient subgroups and it critically depends on the rate of small HCC detected incidentally without surveillance, as well as the annual incidence of HCC and the adoption of liver transplantation.

BackgroundScreening program tend to recognized patients in their early stage and consequently improve health outcomes. Cost-effectiveness of the abdominal aortic aneurysm (AAA) screening program has been scarcely studied in developing countries. We sought to evaluate the cost-effectiveness of a screening program for the abdominal aortic aneurysm (AAA) in men aged over 65 years in Iran.MethodsA Markov cohort model with 11 mutually exclusive health statuses was used to evaluate the cost-effectiveness of a population-based AAA screening program compared with a no-screening strategy. Transitions between the health statuses were simulated by using 3-month cycles. Data for disease transition probabilities and quality of life outcomes were obtained from published literature, and costs were calculated based on the price of medical services in Iran and the examination of the patients’ medical records. The outcomes were life-years gained, the quality-adjusted life-year (QALY), costs, and the incremental cost-effectiveness ratio (ICER). The analysis was conducted for a lifetime horizon from the payer’s perspective. Costs and effects were discounted at an annual rate of 3%. Uncertainty surrounding the model inputs was tested with deterministic and probabilistic sensitivity analyses.ResultsThe mean incremental cost of the AAA screening strategy compared with the no-screening strategy was $140 and the mean incremental QALY gain was 0.025 QALY, resulting in an ICER of $5566 ($14,656 PPP) per QALY gained. At a willingness-to-pay of 1 gross domestic product (GDP) per capita ($5628) per QALY gained, the probability of the cost-effectiveness of AAA screening was about 50%. However, at a willingness-to-pay of twice the GDP per capita per QALY gained, there was about a 95% probability for the AAA screening program to be cost-effective in Iran.ConclusionsThe results of this study showed that at a willingness-to-pay of 1 GDP per capita per QALY gained, a 1-time AAA screening program for men aged over 65 years could not be cost-effective. Nevertheless, at a willingness-to-pay of twice the GDP per capita per QALY gained, the AAA screening program could be cost-effective in Iran. Further, AAA screening in high-risk groups could be cost-effective at a willingness-to-pay of 1 GDP per capita per QALY gained.

The standard treatment for a non-union of the hallux metatarsophalangeal joint fusion has been to revise the fusion. Revision fusion is technically more demanding, often involving bone grafting, more substantial fixation and prolonged period of immobilization postoperatively. We present data to suggest that removal of hardware and debridement alone is an alternative treatment option. A case note review identified patients with a symptomatic non-union after hallux metatarsophalangeal joint (MTPJ) fusion. It is our practice to offer these patients revision fusion or removal of hardware and debridement. For the seven patients that chose hardware removal and were left with a pseudarthrosis, a matched control group was selected from patients who had had successful fusions. Three outcome scores were used. Hallux valgus and dorsiflexion angles were recorded. One hundred thirty-nine hallux MTPJ arthrodeses were carried out. Fourteen non-unions were identified. The rate of non-union in males and following previous hallux MTPJ surgery was 19% and 24%, respectively. In females undergoing a primary MTPJ fusion, the rate was 2.4%. Twelve non-union patients were reviewed at 27 months (mean). Eleven patients had elected to undergo removal of hardware and debridement. Four patients with pseudarthrosis were unhappy with the results and proceeded to either revision fusion or MTPJ replacement. Seven non-union patients, who had removal of hardware alone, had outcome scores marginally worse compared to those with successful fusions. Removal of hardware alone is a reasonable option to offer as a relatively minor procedure following a failed arthrodesis of the first MTPJ. This must be accepted on the proviso that in this study four out of 11 (36%) patients proceeded to a revision first MTPJ fusion or first MTPJ replacement. We also found that the rate of non-union in primary first MTPJ fusion was significantly higher in males and those patients who had undergone previous surgery.

Cost-effectiveness of PCSK9 inhibition in addition to standard lipid-lowering therapy in patients at high risk for vascular disease

4591 Background: EV-302 and CheckMate 901 demonstrated significant survival benefit with enfortumab vedotin-pembrolizumab (EVPembro) and gemcitabine-cisplatin-nivolumab (GemCisNivo) as 1L therapy, respectively, in patients (pts) with la/mUC. However, whether these treatments are cost-effective or not remains unclear. Methods: A three-state Markov model (progression-free, progression and death) was developed. State utilities were derived from published literature. State transition probabilities were informed from point-probabilities and hazard ratios for OS and PFS obtained from the latest follow-up of eligible trials. Average sales price (2024 USD) for individual treatments (excluding ancillary charges) were obtained from the center of medicare and medicaid services (US payer’s perspective). Treatment strategies were modeled in accordance with the dose/schedule reported in the eligible trials. Pts who received GemCis or Gem-carboplatin (GemCarbo) and did not progress were modeled to receive Avelumab maintenance. Half-cycle corrected costs and utilities were accrued over a 10-year lifetime horizon and were discounted at 3%. Monte Carlo simulation was used to estimate quality adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs). A willingness-to-pay threshold (WTP) of 100,000$ per QALY was used. Results: In cisplatin-eligible pts, GemCisNivo demonstrated a 0.8 QALY gain over GemCis at an ICER of 21,127 ($/QALY). GemCisNivo was also associated with an incremental net monetary benefit (INMB) of 63,099$ relative to GemCis. EVPembro demonstrated a 2.4 QALY gain over GemCis at an ICER of 915,283 and a 1.6 QALY gain over GemCisNivo at an ICER of 1,406,308. In cisplatin-ineligible pts, EVPembro demonstrated a 2.4 QALY gain over Gem-carboplatin (GemCarbo) at an ICER of 934,761. Price threshold analyses demonstrated that EVPembro is likely to be cost-effective at a cost of 12,344$ for cisplatin-eligible and 13,374$ per cycle for cisplatin-ineligible pts (original cost: 35,665.60$ per cycle). Sensitivity analysis using a total of 12 cycles for EVPembro showed cost-effectiveness at an ICER of 69,594 relative to GemCisNivo in cisplatin-eligible pts, and at an ICER of 53,099 relative to GemCarbo in cisplatin-ineligible pts. Conclusions: In terms of US payer’s perspective, GemCisNivo is most likely to be cost-effective in cisplatin-eligible pts at the WTP of 100,000 $/QALY. Limiting the number of cycles for EVPembro may render it as a cost-effective 1L treatment in la/mUC pts regardless of cisplatin-eligibility status.

e18356 Background: Carfilzomib plus dexamethasone (Kd) dosed once weekly at 70 mg/m2 (QW Kd70) was recently approved in the US for treating patients with relapsed and refractory multiple myeloma (RRMM). To assess the cost-effectiveness (CE) of QW Kd70 vs twice weekly Kd dosed at 27 mg/m2 (BIW Kd27), data from the phase 3 ARROW trial, which directly compared these regimens in patients with 2-3 prior lines of therapy were used. Methods: A partitioned survival model was developed for the CE analysis. Time to treatment discontinuation, progression-free survival, and overall survival (OS) were estimated from the ARROW trial. Long-term OS was extrapolated using Surveillance Epidemiology and End Results registry data after matching characteristics of patients in the registry and ARROW trial. Direct costs were estimated from a US health care payer perspective. Utilities collected in the ARROW trial using the five-level version of the EuroQol questionnaire (EQ-5D-5L) were applied to estimate the quality-adjusted life years (QALYs). Uncertainty was explored using sensitivity analyses. Two subgroups of patients refractory to lenalidomide or bortezomib were assessed. Main outcomes were mean life-years (LYs), QALYs, lifetime costs, and incremental cost-effectiveness ratios (ICERs). Results: For QW Kd70 and BIW Kd27, the model predicted mean LYs of 4.17 and 3.07 years, QALYs of 2.98 and 2.03 years, and mean total lifetime costs of $444,563 and $373,364, respectively. The incremental LYs gain, QALY gain, and incremental costs of QW Kd70 vs BIW Kd27 were estimated to be 1.10 years, 0.95 year, and $71,199, respectively, resulting in an ICER of $64,595 per LY gained and $75,204 per QALY gained. For patients refractory to lenalidomide and bortezomib, similar results were found with ICERs of $79,988 and $76,793, respectively. Conclusions: In line with ARROW trial results, this CE analysis showed that QW Kd70 is expected to provide considerable additional benefit in terms of LYs and QALYs gained compared with BIW Kd27. In the RRMM setting, QW Kd70 is cost-effective with ICERs below accepted willingness to pay thresholds in US and represents an efficient utilization of the health care budget.

This analysis aimed to evaluate the cost effectiveness of molnupiravir versus best supportive care for the treatment of older adult patients (aged ≥ 65 years) in Japan with mild to moderate COVID-19 who are at risk of disease progression leading to hospitalization, predominantly using input data derived from the Omicron era of the SARS-CoV2 pandemic. A decision-analytic model was developed, comprising a decision-tree model for the acute COVID-19 phase (30 days), followed by a lifetime Markov model. Inputs used to parametrize the model were derived from a database study conducted in Japan and a published systematic literature review of real-world studies, and from ad-hoc literature searches and other research (for disease progression, cost, and utility estimates). This analysis modelled death averted due to COVID-19 hospitalization as an indirect effect of molnupiravir (through preventing hospitalization). Costs were expressed in 2022 Japanese yen (¥; JPY), from the perspective of payers (the base case) or society (in a scenario analysis). Costs and QALYs were discounted at 2% per year. Cost effectiveness of molnupiravir versus best supportive care was primarily compared to a willingness-to-pay (WTP) threshold of ¥5,000,000 per quality-adjusted life year (QALY) gained. Treatment with molnupiravir is associated with a QALY gain of 0.018 and an incremental cost of ¥81,472 over best supportive care and is cost effective (with an incremental cost-effectiveness ratio [ICER] of ¥4,638,477) versus best supportive care based on the predefined WTP threshold of ¥5,000,000 per QALY gained. Molnupiravir leads to a reduction in the proportion of patients who die due to COVID-19 hospitalization (0.09% with molnupiravir vs 0.29% with best supportive care). Molnupiravir is also associated with lower costs associated with COVID-19 hospitalizations compared with best supportive care (¥22,527 vs ¥27,472). In a deterministic sensitivity analysis, the top five most sensitive parameters were baseline hospitalization rate, mortality benefit of molnupiravir, mortality rate in general ward, discount rate, and mortality rate in intensive care unit. In a probabilistic sensitivity analysis, at the predefined WTP threshold of ¥5,000,000 per QALY gained, molnupiravir had an 80% probability of being cost effective versus best supportive care. Molnupiravir is a cost-effective treatment option for the treatment of older adult outpatients (age ≥ 65 years) with symptomatic COVID-19 in Japan, relative to best supportive care.