Cost-effectiveness Studies Research Articles

Radiation Oncology Research in Asia: Current Status and a Peep Into the Future From the Federation of Asian Organizations for Radiation Oncology.

This survey was conducted to assess the current research practices among the 14 members of the Federation of Asian Organizations for Radiation Oncology (FARO) committee, to inform measures for research capacity building in these nations. A 19-item electronic survey was sent to two research committee members from the 14 representative national radiation oncology organizations (N = 28) that are a part of FARO. Thirteen of the 14 member organizations (93%) and 20 of 28 members (71.5%) responded to the questionnaire. Only 50% of the members stated that an active research environment existed in their country. Retrospective audits (80%) and observational studies (75%) were the most common type of research conducted in these centers. Lack of time (80%), lack of funding (75%), and limited training in research methodology (40%) were cited as the most common hindrances in conducting research. To promote research initiatives in the collaborative setting, 95% of the members agreed to the creation of site-specific groups, with head and neck (45%) and gynecological cancers (25%) being the most preferred disease sites. Projects focused on advanced external beam radiotherapy implementation (40%), and cost-effectiveness studies (35%) were cited as some of the potential areas for future collaboration. On the basis of the survey results, after result discussion and the FARO officers meeting, an action plan for the research committee has been created. The results from the survey and the initial policy structure may allow facilitation of radiation oncology research in the collaborative setting. Centralization of research activities, funding support, and research-directed training are underway to help foster a successful research environment in the FARO region.

Antibiotics for lower respiratory tract infection in children presenting in primary care: ARTIC-PC RCT.

Antimicrobial resistance is a global health threat. Antibiotics are commonly prescribed for children with uncomplicated lower respiratory tract infections, but there is little randomised evidence to support the effectiveness of antibiotics in treating these infections, either overall or relating to key clinical subgroups in which antibiotic prescribing is common (chest signs; fever; physician rating of unwell; sputum/rattly chest; shortness of breath). To estimate the clinical effectiveness and cost-effectiveness of amoxicillin for uncomplicated lower respiratory tract infections in children both overall and in clinical subgroups. Placebo-controlled trial with qualitative, observational and cost-effectiveness studies. UK general practices. Children aged 1-12 years with acute uncomplicated lower respiratory tract infections. The primary outcome was the duration in days of symptoms rated moderately bad or worse (measured using a validated diary). Secondary outcomes were symptom severity on days 2-4 (0 = no problem to 6 = as bad as it could be); symptom duration until very little/no problem; reconsultations for new or worsening symptoms; complications; side effects; and resource use. Children were randomised to receive 50 mg/kg/day of oral amoxicillin in divided doses for 7 days, or placebo using pre-prepared packs, using computer-generated random numbers by an independent statistician. Children who were not randomised could participate in a parallel observational study. Semistructured telephone interviews explored the views of 16 parents and 14 clinicians, and the data were analysed using thematic analysis. Throat swabs were analysed using multiplex polymerase chain reaction. A total of 432 children were randomised (antibiotics, n = 221; placebo, n = 211). The primary analysis imputed missing data for 115 children. The duration of moderately bad symptoms was similar in the antibiotic and placebo groups overall (median of 5 and 6 days, respectively; hazard ratio 1.13, 95% confidence interval 0.90 to 1.42), with similar results for subgroups, and when including antibiotic prescription data from the 326 children in the observational study. Reconsultations for new or worsening symptoms (29.7% and 38.2%, respectively; risk ratio 0.80, 95% confidence interval 0.58 to 1.05), illness progression requiring hospital assessment or admission (2.4% vs. 2.0%) and side effects (38% vs. 34%) were similar in the two groups. Complete-case (n = 317) and per-protocol (n = 185) analyses were similar, and the presence of bacteria did not mediate antibiotic effectiveness. NHS costs per child were slightly higher (antibiotics, £29; placebo, £26), with no difference in non-NHS costs (antibiotics, £33; placebo, £33). A model predicting complications (with seven variables: baseline severity, difference in respiratory rate from normal for age, duration of prior illness, oxygen saturation, sputum/rattly chest, passing urine less often, and diarrhoea) had good discrimination (bootstrapped area under the receiver operator curve 0.83) and calibration. Parents found it difficult to interpret symptoms and signs, used the sounds of the child's cough to judge the severity of illness, and commonly consulted to receive a clinical examination and reassurance. Parents acknowledged that antibiotics should be used only when 'necessary', and clinicians noted a reduction in parents' expectations for antibiotics. The study was underpowered to detect small benefits in key subgroups. Amoxicillin for uncomplicated lower respiratory tract infections in children is unlikely to be clinically effective or to reduce health or societal costs. Parents need better access to information, as well as clear communication about the self-management of their child's illness and safety-netting. The data can be incorporated in the Cochrane review and individual patient data meta-analysis. This trial is registered as ISRCTN79914298. This project was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 27, No. 9. See the NIHR Journals Library website for further project information.

Cost-effectiveness of novel systemic therapies for advanced breast cancer: Global representativeness and influence of funding.

e18924 Background: Approval of expensive novel treatments increases costs of cancer care and undermines access, particularly in low and middle-income countries. As the most incident neoplasm, breast cancer (BC) therapies have an enormous budget impact if incorporated. Therefore, cost-effectiveness (CE) studies are essential to guide such decisions. Methods: A systematic literature search of Medline, Scopus, and Web of Science from January 1st 2012 to July 8th 2022 was conducted to identify CE studies of tumor-targeted systemic therapies for advanced BC. Articles without CE (or utility) ratio calculations were excluded. Information on the country and drug studied, authors’ conflicts of interests (COI) and funding, and authors' conclusions were manually reviewed by two independent investigators. Results: Of 1103 screened records, 74 studies comprising 212 CE comparisons were included. Most studied scenarios were from high-income countries (70%, 141/212), with only 10% from Latin America and none from Africa. The most studied drug classes were iCDK4/6 (23%), anti-HER2 directed therapy (21%), anti-PD1/PDL1 (12%) and endocrine therapy (11%). In 46% of the comparisons, a new drug combined with standard of care was compared to placebo and/or standard of care, and in 37% the compared treatment were alternative same class therapies. Nearly 46% (97/212) had pharmaceutical industry funding or author’s COI associated with industry. Overall, 56% of authors’ conclusions were unfavorable. Pharmaceutical industry related-studies were associated with high-income countries (75% vs. 64%, p = 0.006), same-class drug comparators (52% vs. 33%, p = 0.047), and favorable conclusions (60% vs. 15%, p < 0.001). A combination of drugs (addition to standard of care) was associated with unfavorable conclusions (56% vs 28%, p < 0.001). Conclusions: Industry-related studies target high-income countries' scenarios and are more likely to have favorable conclusions. As expected, the addition of drugs to standard treatments (combinations) is less likely to be cost-effective. Government funding in low and middle-income countries is needed to decrease the current literature gap and provide stakeholders with proper information to make incorporation decisions.

EE362 Systematic Review of Individual-Level Simulation in Cost-Effectiveness Analysis of Antiviral Treatments in Patients with Hepatitis B

We aimed to systematically review individual-level simulation (ILS) studies in recent cost-effectiveness (CE) studies of nucleoside antiviral agents (NAA) in patients with hepatitis B and suggest the advantages and applicability of this method.

Cost-Effectiveness of Screening to Identify Patients With Atrial Fibrillation: ASystematic Review.

Screening for Atrial Fibrillation (AF) is recommended for people aged above 65 years. Screening for AF in asymptomatic individuals can be beneficial by enabling earlier diagnosis and the commencement of interventions to reduce the risk of early events, thus improving patient outcomes. This study systematically reviews the literature about the cost-effectiveness of various screening methods for previously undiagnosed AF. Four databases were searched to identify articles that are cost-effectiveness studies conducted on screening for AF published from January 2000 to August 2022. The Consolidated Health Economic Evaluation Reporting Standards 2022 checklist was used to assess the quality of the selected studies. A previously published approach was used to assess the usefulness of each study for health policy makers. The database search yielded 799 results, with 26 articles meeting the inclusion criteria. Articles were categorised into four subgroups: (i) population screening, (ii) opportunistic screening, (iii) targeted, and (iv) mixed methods of screening. Most of the studies screened adults ≥65 years of age. Most studies were performed from a 'health care payer perspective' and almost all studies used 'not screening' as a comparator. Almost all screening methods assessed were found to be cost-effective in comparison to 'not screening'. The reporting quality varied between 58% to 89%. The majority of the studies were found to be of limited usefulness for health policy makers, as none of the studies made any clear statements about policy change or implementation direction. All approaches of AF screening were found to be cost-effective compared with no screening, while opportunistic screening was found to be the optimal approach in some studies. However, screening for AF in asymptomatic individuals is context specific and likely to be cost-effective depending on the population screened, screening approach, frequency, and the duration of screening.

Use of 18F-FDG PET/MRI as an Initial Staging Procedure for Nasopharyngeal Carcinoma.

Compared with the conventional work-up (CWU) including computed tomography (CT) of the chest and abdomen, MRI of the head and neck, and skeletal scintigraphy, positron emission tomography (PET)/MRI might improve diagnostic accuracy, shorten the work-up time, and reduce false-positive (FP) findings in patients with nasopharyngeal carcinoma (NPC). However, evidence of cost-effectiveness is needed for the adoption of PET/MRI for the initial staging in NPC. To evaluate the cost-effectiveness and clinical value of PET/MRI as an initial staging procedure for NPC. Retrospective cohort cost effectiveness study. Three hundred forty-three patients with a median age of 51 (13-81) years underwent PET/MRI before treatment (the PET/MRI group) and the remaining 677 patients with a median age of 55 (15-95) years only underwent CWU (the CWU group). There were 80 (23.3%) females and 193 (28.5%) females in the PET/MRI and CWU groups, respectively. 3-T integrated PET/MRI system, diffusion-weighted echo-planar imaging (b = 0 and 1000 s/mm2 ) and [18F] fluorodeoxyglucose PET. The primary end point was the FP rate. Costs were determined as issued in 2021 by the Medical Insurance Administration Bureau of Zhejiang, China. Incremental cost effectiveness ratio (ICER) measured cost of using PET/MRI per percent of patients who avoided a FP. A P-value <0.05 was considered statistically significant. For the whole group, the de novo metastatic disease rate was 5.2% (53/1020). A total of 187 patients with FP results were observed. Significantly more patients with FP results were observed in the CWU group compared to the PET/MRI group (25.6% vs. 4.1%). The ICER was $54 for each percent of patients avoiding a FP finding. Compared with CWU, PET/MRI may reduce the FP risk. Furthermore, PET/MRI may be cost-effective as an initial staging procedure for NPC. 3 TECHNICAL EFFICACY: Stage 6.

An economic evaluation of first-line cryoballoon ablation versus antiarrhythmic drug therapy for the treatment of paroxysmal atrial fibrillation from an English NHS perspective

Abstract Funding Acknowledgements Type of funding sources: Other. Main funding source(s): This cost-effectiveness study was funded by Medtronic Background The UK National Institute of Care Excellence (NICE) recommend the use of pulmonary vein isolation (PVI) to treat paroxysmal atrial fibrillation (PAF) in those who have not responded to drug treatment. Recently, three randomized controlled trials have demonstrated that as an initial rhythm control strategy, PVI with cryoballoon ablation, reduces atrial arrhythmia recurrence compared to antiarrhythmic drug (AAD) therapy in patients with symptomatic PAF. Purpose To evaluate the cost-effectiveness of first-line cryoablation versus first-line optimized AADs in an English National Health Service (NHS) setting. Methods Individual patient-level data from 703 participants with untreated PAF recruited into Cryo-FIRST, STOP AF First and EARLY-AF were used to derive equations to predict the following outcomes: rates of ablation, AF recurrence and resolution, AF-related hospital attendance, and health-related quality of life (HRQoL) utilities. Where parameters could not be sourced from the trial data, inputs were taken from the published literature or derived using clinical expert opinion. The cost-effectiveness model (CEM) was a hybrid of a decision tree with a one-year time horizon and a Markov model (three-month cycle length) with a lifetime time horizon and was developed from the perspective of the English NHS. Health benefits were expressed in quality-adjusted life years (QALYs), and all benefits and costs were discounted at 3.5% per year in line with NICE requirements. Uncertainty in the CEM inputs was explored using probabilistic sensitivity analysis. The results include an initial 12-week blanking period for all studies. Results The three-monthly rate of AF recurrence was reduced on average by 46.7% (p&amp;lt;0.001) in those treated with cryoablation. Similarly, the monthly rate of receiving an ablation following initial treatment was reduced by 72.8% (p&amp;lt;0.001) in the cryoablation arm. Furthermore, the average cryoablation patient was associated with a 4.3% (p=0.025) increase in their HRQoL. While the likelihood of failure was greater in the AAD group, in those who failed initial treatment, there was no difference in the rate of AF symptom resolution. The CEM indicates that cryoablation is more effective (+0.17 QALYs) and more costly (+£1,414) over a lifetime compared to optimized AADs. Cryoablation resulted in an Incremental Cost-Effectiveness Ratio of £8,435 with a 78.5% probability of being cost-effective at a willingness-to-pay threshold of £20,000 per QALY gained. Individuals in both treatment arms were predicted to receive ~1.2 ablations over a lifetime regardless of initial treatment. However, there was a 45% relative reduction in the amount of time spent in symptomatic AF states for those initially treated with cryoablation. Conclusions AF rhythm control in drug naïve patients with cryoballoon ablation is cost-effective compared to optimized AADs in an English NHS setting.

Cost-Effectiveness of Forgoing Postoperative Catheter Angiography After Microsurgical Occlusion of Spinal Dorsal Intradural Arteriovenous Fistulas

Spinal dorsal intradural arteriovenous fistulas (DI-AVFs) represent 70% of all spinal vascular lesions. Diagnostic tools include pre- and postoperative digital subtraction angiography (DSA) and intraoperative indocyanine green videoangiography (ICG-VA). ICG-VA has a high predictive value in DI-AVF occlusion, but postoperative DSA remains a core component of postoperative protocols. The aim of this study was to evaluate the potential cost reduction of forgoing postoperative DSA after microsurgical occlusion of DI-AVFs. Cohort-based cost effectiveness study of all DI-AVFs within a prospective, single-center cerebrovascular registry from January 1, 2017, to December 31,2021. Complete data including intraoperative ICG-VA and costs were available for 11 patients. Mean (SD) age was 61.5 (14.8) years. All DI-AVFs were treated with microsurgical clip ligation of the draining vein. ICG-VA showed complete obliteration in all patients. Postoperative DSA was performed for 6 patients and confirmed complete obliteration. Mean (SD) cost contributions for DSA and ICG-VA were $11,418 ($4,861) and $12 ($2), respectively. Mean (SD) total costs were $63,543 ($15,742) and $53,369 ($27,609) for patients who did and did not undergo postoperative DSA, respectively. Comorbidity status was identified as the main driver of total cost (P= 0.01 after adjusting for postoperative DSA status). ICG-VA is a powerful diagnostic tool in demonstrating microsurgical cure of DI-AVFs, with a negative predictive value of 100%. Eliminating postoperative DSA in patients with confirmed DI-AVF obliteration on ICG-VA may yield substantial cost savings, in addition to sparing patients the risk and inconvenience of a potentially unnecessary invasive procedure.

Cost-effectiveness of a 3-year tele-messaging intervention for positive airway pressure use.

To evaluate the cost-effectiveness of a 3-year tele-messaging intervention for positive airway pressure (PAP) use in obstructive sleep apnea (OSA). A post hoc cost-effectiveness analysis (from US payers' perspective) of data from a 3-month tele-OSA trial, augmented with 33 months of epidemiologic follow-up. Cost-effectiveness was compared among 3 groups of participants with an apnea-hypopnea index of at least 15 events/hour: (1) no messaging (n = 172), (2) messaging for 3 months (n = 124), and (3) messaging for 3 years (n = 46). We report the incremental cost (2020 US$) per incremental hour of PAP use and the fraction probability of acceptability based on a willingness-to-pay threshold of $1825 per year ($5/day). The use of 3 years of messaging had similar mean annual costs ($5825) compared with no messaging ($5889; P = .89) but lower mean cost compared with 3 months of messaging ($7376; P = .02). Those who received messaging for 3 years had the highest mean PAP use (4.11 hours/night), followed by no messaging (3.03 hours/night) and 3 months of messaging (2.84 hours/night) (all P < .05). The incremental cost-effectiveness ratios indicated that 3 years of messaging showed lower costs and greater hours of PAP use compared with both no messaging and 3 months of messaging. Based on a willingness-to-pay threshold of $1825, there is a greater than 97.5% chance (ie, 95% confidence) that 3 years of messaging is acceptable compared with the other 2 interventions. Long-term tele-messaging is highly likely to be cost-effective compared with both no and short-term messaging, with an acceptable willingness-to-pay threshold. Future long-term cost-effectiveness studies in a randomized controlled trial setting are warranted.

Cost-Effectiveness Analyses in AML: What Have We Learned, How Should This Impact Patient Care, and What Needs to Be Done in the Future?

Acute myeloid leukemia (AML) is the most common acute leukemia in adults in the United States and has seen the approval of several novel agents over the past decade. Similar to treatments for other hematologic and solid malignancies, these novel agents are costly. In the setting of finite financial resources in the healthcare system, the concept of cost-effectiveness analyses has been developed to compare the estimated costs and associated benefits expected with different interventions (eg, drugs, diagnostic tests, procedures). Although drug approvals in the United States are not based on budgetary considerations, cost-effectiveness analyses can inform health policy decisions, resource allocation, and societal debates. However, such analyses are only capturing parts of the costs and benefits to the healthcare system, payers, and consumers, and are based on modeling assumptions with inherent limitations. In addition, cost-effectiveness analyses for several of the novel agents approved for treatment of AML are limited and have reported conflicting results. This review uses cost-effectiveness analyses of azacitidine/venetoclax and liposomal cytarabine/daunorubicin as examples to review considerations and best practices when conducting and interpreting such studies. To ensure adequate interpretability of cost-effectiveness studies, transparency in the model inputs/assumptions, data sources, and funding is of great importance, as evidenced by the discrepant conclusions across studies. Furthermore, the perspective and the healthcare system from which a cost-effectiveness analysis is conducted are important to consider because practice patterns and drug prices between countries can be variable. However, with advances in health economic modeling techniques, adherence to best practices, and increasing public interest in these types of studies, cost-effectiveness analyses can become an important tool to inform various stakeholders in the healthcare system to allocate limited resources most efficiently.

G-FORCE: the effectiveness of group psychotherapy for Cluster-C personality disorders: protocol of a pragmatic RCT comparing psychodynamic and two forms of schema group therapy

BackgroundCluster-C personality disorders (PDs), characterized by a high level of fear and anxiety, are related to high levels of distress, societal dysfunctioning and chronicity of various mental health disorders. Evidence for the optimal treatment is extremely scarce. Nevertheless, the need to treat these patients is eminent. In clinical practice, group therapy is one of the frequently offered approaches, with two important frameworks: schema therapy and psychodynamic therapy. These two frameworks suggest different mechanisms of change, but until now, this has not yet been explored. The purpose of the present G-FORCE trial is to find evidence on the differential (cost)effectiveness of two forms of schema group therapy and psychodynamic group therapy in the routine clinical setting of an outpatient clinic and to investigate the underlying working mechanisms and predictors of outcome of these therapies.MethodsIn this mono-centre pragmatic randomized clinical trial, 290 patients with Cluster-C PDs or other specified PD with predominantly Cluster-C traits, will be randomized to one of three treatment conditions: group schema therapy for Cluster-C (GST-C, 1 year), schema-focused group therapy (SFGT, 1.5 year) or psychodynamic group therapy (PG, 2 years). Randomization will be pre-stratified on the type of PD. Change in severity of PD (APD-IV) over 24 months will be the primary outcome measure. Secondary outcome measures are personality functioning, psychiatric symptoms and quality of life. Potential predictors and mediators are selected and measured repeatedly. Also, a cost-effectiveness study will be performed, primarily based on a societal perspective, using both clinical effects and quality-adjusted life years. The time-points of assessment are at baseline, start of treatment and after 1, 3, 6, 9, 12, 18, 24 and 36 months.DiscussionThis study is designed to evaluate the effectiveness and cost-effectiveness of three formats of group psychotherapy for Cluster-C PDs. Additionally, predictors, procedure and process variables are analysed to investigate the working mechanisms of the therapies. This is the first large RCT on group therapy for Cluster-C PDs and will contribute improving the care of this neglected patient group. The absence of a control group can be considered as a limitation.Trial registrationCCMO, NL72826.029.20. Registered on 31 August 2020, first participant included on 18 October 2020.

The Clinical Application and Progress of Mirogabalin on Neuropathic Pain as a Novel Selective Gabapentinoids.

Neuropathic pain is a complex sort of pain that is detrimental to individuals' health, both physically and mentally, but merely a small portion of them could witness pain alleviation. Mirogabalin, by distinctive binding characteristics of voltage-gated calcium channels, has won approval from the Japanese authority as a third member of gabapentinoids in Japan. Our review was aimed at encompassing the bulk of clinical research on mirogabalin, which included clinical trials, special considerations, coadministration studies, case reports, and cost-effectiveness studies. A review was carried out on a series of platforms, such as PubMed, MEDLINE, and Scopus, up to December 2021 using the keywords as follows: "mirogabalin OR mirogabalin besylate OR Tarlige OR DS-5565" AND "neuropathic pain OR Neuropathy." Mirogabalin demonstrated analgesic activity and manageable adverse reactions and provides a new alternative for individuals with PHN or DPNP in 3 phase II and 4 III trials. Mirogabalin alleviated pain markedly in comparison with placebo. Administration of mirogabalin on a long-term basis is a flexible dosage regimen for patients with PHN. It is noteworthy that mirogabalin should be administrated cautiously when combined with probenecid and cimetidine on account of a slight increase in pharmacodynamics effects of mirogabalin. The development of mirogabalin allows further optimization of individual treatment strategies so as to provide more therapeutic choices in this medical domain.

Prophylactic Mesh Placement With Ileal Conduit: A Cost-effectiveness Analysis

To determine the cost-effectiveness of mesh placement in patients undergoing ileal conduit urinary diversion for bladder cancer. Long-term studies have shown that parastomal hernias (PSH) occur in more than half of all stomas. Mesh prophylaxis has been shown to reduce PSH after end-colostomy and ileal conduits. However, no cost-effectiveness studies on mesh prophylaxis have been performed for this population. We created a Markov model incorporating the costs and effectiveness of mesh prophylaxis for patients undergoing radical cystectomy and ileal conduit construction. Costs were obtained from the literature and adjusted to 2022 US dollars. Effectiveness was measured in quality-adjusted life years (QALY). 1- and 2-way sensitivity analyses were performed to test the robustness of our model. In stage I-IV bladder cancer, prophylactic mesh placement was costlier, but more effective in providing quality of life compared with no mesh placement at index surgery. Average incremental cost between the 2 strategies across all stages was an additional $897 when mesh was utilized. Incremental effectiveness averaged 0.49 additional QALY across all stages. This resulted in an incremental cost-effectiveness ratio of $2114.71/QALY. Sensitivity analyses indicated that benefit of mesh placement was sensitive to the probability of mesh infection. In patients undergoing ileal conduit urinary diversion for bladder cancer, mesh prophylaxis at the time of radical cystectomy is an overall cost-effective strategy in preventing PSH for patients presenting with all stages of bladder cancer.

Insulin Pump and Closed Loop Systems' Use in People Living with Diabetes: A Narrative Review of Clinical and Cost-Effectiveness to Enable Access to Technology and Meet the Need of Payers.

The use of continuous subcutaneous insulin infusion delivery via insulin pumps is today considered standard of care for type 1 diabetes (T1D). Closed-loop systems combining continuous glucose monitoring (CGM) with automated algorithm-driven insulin delivery have been shown to be safe and efficacious in randomized controlled studies and in real-life studies in both pediatric and adult individuals with T1D. Implementation of hybrid closed-loop (HCL) systems have shown incremental effectiveness with further reduction of hypoglycemia and hyperglycemia. Although less extensively studied in type 2 diabetes (T2D), insulin pumps have demonstrated their effectiveness on glucose control together with the reduction in insulin needs and a neutral effect on weight. Recent studies have also shown promising results with the use of HCL in T2D. Cost-effectiveness studies both in T1D and T2D have shown that pump is cost effective in several countries, leading to improvements in quality adjusted life years. Insulin pumps are currently reimbursed for T1D in many European countries, but only in a few for individuals with T2D. HCL systems are to be evaluated in future trials performed in T2D to compare their incremental efficacy and cost effectiveness in comparison with available intensification tools which include multiple daily insulin injections, metformin, SGLT-2 inhibitors and GLP-1 receptor agonists. There is a need for updated guidelines for the use of CSII and HCL in individuals living with T2D based on the emerging evidence, identifying, and recommending for the people who'd benefit the most, which would eventually form a basis for the reimbursement and health policies. This article is protected by copyright. All rights reserved.

Different vaccination strategies for preventing coronavirus disease 2019 in Kenya: A dynamic modelling study of health impact and cost-effectiveness

Background: Vaccination can significantly reduce the health impact and economic burden of coronavirus disease 2019 (COVID-19), but vaccination levels for COVID-19 in most African countries lag far behind global averages. We assessed the cost-effectiveness of different COVID-19 vaccination strategies in Kenya and determined the optimal vaccination strategy. Methods: Using a dynamic transmission model, we divided the population into three groups: 0-18 years, 19-58 years and 58+ years. We assessed the effectiveness and cost-effectiveness of three vaccination strategies at different numbers of daily vaccinations based on previous studies and public databases. Nine scenarios were modeled and compared to no-continuation-vaccination to calculate the number of averted diseases, averted deaths, and net benefits of different vaccination strategies. One-way sensitivity analysis and probabilistic sensitivity analysis were conducted to assess the stability of our findings. Results: Compared to no-continuation-vaccination for various vaccination scenarios, all vaccination strategies were found to be effective and cost-saving. The incremental net benefit ranged from 0.235 billion USD to 2.305 billion USD, and prioritizing vaccination boosters for individuals aged 19-58 was identified as the most cost-effective option. On the other hand, prioritizing vaccination for the unvaccinated population aged 58 and above could potentially reduce COVID-19 related deaths by 1.59%-56.60%, which was the most effective approach in avoiding cause-specific deaths. However, all vaccination strategies were found to be ineffective in controlling the infection trend when compared to no intervention under different vaccination scenarios, with only 474,318-5,306,865 infections potentially being prevented. Conclusion: Timely and widespread vaccination against COVID-19 in Kenya is effective and cost-effective, a specific vaccination strategy should be selected based on decision-making needs. Priority vaccination for the elderly without vaccination may be more cost-effective compared with other vaccination strategies.

Clinical Perspectives on Using Remote Measurement Technology in Assessing Epilepsy, Multiple Sclerosis, and Depression: Delphi Study

Background Multiple sclerosis (MS), epilepsy, and depression are chronic central nervous system conditions in which remote measurement technology (RMT) may offer benefits compared with usual assessment. We previously worked with clinicians, patients, and researchers to develop 13 use cases for RMT: 5 in epilepsy (seizure alert, seizure counting, risk scoring, triage support, and trend analysis), 3 in MS (detecting silent progression, detecting depression in MS, and donating data to a biobank), and 5 in depression (detecting trends, reviewing treatment, self-management, comorbid monitoring, and carer alert). Objective In this study, we aimed to evaluate the use cases and related implementation issues with an expert panel of clinicians external to our project consortium. Methods We used a Delphi exercise to validate the use cases and suggest a prioritization among them and to ascertain the importance of a variety of implementation issues related to RMT. The expert panel included clinicians from across Europe who were external to the project consortium. The study had 2 survey rounds (n=23 and n=17) and a follow-up interview round (n=9). Data were analyzed for consensus between participants and for stability between survey rounds. The interviews explored the reasons for answers given in the survey. Results The findings showed high stability between rounds on questions related to specific use cases but lower stability on questions relating to wider issues around the implementation of RMT. Overall, questions on wider issues also had less consensus. All 5 use cases for epilepsy (seizure alert, seizure counting, risk scoring, triage support, and trend analysis) were considered beneficial, with consensus among participants above the a priori threshold for most questions, although use case 3 (risk scoring) was considered less likely to facilitate or catalyze care. There was very little consensus on the benefits of the use cases in MS, although this may have resulted from a higher dropout rate of MS clinicians (50%). Participants agreed that there would be benefits for all 5 of the depression use cases, although fewer questions on use case 4 (triage support) reached consensus agreement than for depression use cases 1 (detecting trends), 2 (reviewing treatment), 3 (self-management), and 5 (carer alert). The qualitative analysis revealed further insights into each use case and generated 8 themes on practical issues related to implementation. Conclusions Overall, these findings inform the prioritization of use cases for RMT that could be developed in future work, which may include clinical trials, cost-effectiveness studies, and the commercial development of RMT products and services. Priorities for further development include the use of RMT to provide more accurate records of symptoms and treatment response than is currently possible and to provide data that could help inform patient triage and generate timely alerts for patients and carers.

A Review of Economic Studies of Amyotrophic Lateral Sclerosis (ALS) in the United States (P3-8.015)

<h3>Objective:</h3> This literature review aims to illustrate our current understanding of the societal costs of ALS healthcare, including costs borne by patients, health systems, and payors. <h3>Background:</h3> The chronic, progressive nature of ALS places a large economic burden on patients and the healthcare system; a 2014 retrospective cost-of-illness study reports a total national cost of $246–433 million. True costs may be higher given that studies tend not to focus on patient costs, particularly non-medical costs, and philanthropy. <h3>Design/Methods:</h3> A PubMed search using keywords (ALS, cost-of-illness, economic evaluation, cost study/analysis) yielded 654 articles published between 2000–2022. 496 references were excluded after title and abstract review. Full-text review eliminated 146 articles that were performed outside of the US (n=70), did not address costs (n=43) or ALS (n=31), or were duplicates (n=2). <h3>Results:</h3> The twelve papers included in the review revealed a fragmented understanding of ALS-associated costs, limited to medical costs derived from hospital records and insurance databases and omitting patient out-of-pocket expenses (i.e., copays/coinsurance, home/vehicle modifications, appointment-related travel), which are believed to be large cost drivers. Notably, one of the few cost-of-illness studies that measured non-medical costs found that, on average annually, patients and families spent $17,889 on non-medical out-of-pocket costs and lost $14,682 in foregone income. <h3>Conclusions:</h3> There remains a need for prospective longitudinal studies estimating ALS-related expenses in relation to disease course, including medical and non-medical patient out-of-pocket costs, and cost-effectiveness studies to determine how to best allocate funds and resources. Additional work is also required to understand the economic impact of novel therapies that slow disease progression. Economic evaluations of ALS can address the urgent need for more accessible evidence-based cost information, which may point to strategies to alleviate the burden of sizeable out-of-pocket expenses concentrated over a short period of time and equip patients to make fully informed decisions about their care. <b>Disclosure:</b> Miss Gautam has nothing to disclose. The institution of Dr. Hollin has received research support from MT Pharma. The institution of Dr. Hollin has received research support from Patient-Centered Outcomes Research Institute. The institution of Dr. Hollin has received research support from Shriners Children’s. The institution of Dr. Hollin has received research support from Burroughs Wellcome Fund (BWF) Innovation in Regulatory Science. The institution of Dr. Hollin has received research support from National Institutes of Health. Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ITF. Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MTPA. Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Samus. Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MTPA. Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amylym. Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biohaven. Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cytokinetics.

Cost of exome analysis in patients with intellectual disability: a micro-costing study in a French setting

BackgroundWith the development of next generation sequencing technologies in France, exome sequencing (ES) has recently emerged as an opportunity to improve the diagnosis rate of patients presenting an intellectual disability (ID). To help French policy makers determine an adequate tariff for ES, we aimed to assess the unit cost per ES diagnostic test for ID from the preparation of the pre-analytical step until the report writing step and to identify its main cost drivers.MethodsA micro-costing bottom-up approach was conducted for the year 2018 in a French setting as part of the DISSEQ study, a cost-effectiveness study funded by the Ministry of Health and performed in collaboration with the GAD (Génétique des Anomalies du Développement), a genetic team from the Dijon University Hospital, and a public sequencing platform, the Centre National de Recherche en Génomique Humaine (CNRGH). The analysis was conducted from the point of view of these two ES stakeholders. All of the resources (labor, equipment, disposables and reagents, reusable material) required to analyze blood samples were identified, collected and valued. Several sensitivity analyses were performed.ResultsThe unit nominal cost per ES diagnostic test for ID was estimated to be €2,019.39. Labor represented 50.7% of the total cost. The analytical step (from the preparation of libraries to the analysis of sequences) represented 88% of the total cost. Sensitivity analyses suggested that a simultaneous price decrease of 20% for the capture kit and 50% for the sequencing support kit led to an estimation of €1,769 per ES diagnostic test for ID.ConclusionThis is the first estimation of ES cost to be done in the French setting of ID diagnosis. The estimation is especially influenced by the price of equipment kits, but more generally by the organization of the centers involved in the different steps of the analysis and the time period in which the study was conducted. This information can now be used to define an adequate tariff and assess the efficiency of ES.Trial registrationClinicalTrials.gov identifier NCT03287206 on September 19, 2017.

Evaluation of the sugar-sweetened beverage tax in Oakland, United States, 2015-2019: A quasi-experimental and cost-effectiveness study.

While a 2021 federal commission recommended that the United States government levy a sugar-sweetened beverage (SSB) tax to improve diabetes prevention and control efforts, evidence is limited regarding the longer-term impacts of SSB taxes on SSB purchases, health outcomes, costs, and cost-effectiveness. This study estimates the impact and cost-effectiveness of an SSB tax levied in Oakland, California. An SSB tax ($0.01/oz) was implemented on July 1, 2017, in Oakland. The main sample of sales data included 11,627 beverage products, 316 stores, and 172,985,767 product-store-month observations. The main analysis, a longitudinal quasi-experimental difference-in-differences approach, compared changes in beverage purchases at stores in Oakland versus Richmond, California (a nontaxed comparator in the same market area) before and 30 months after tax implementation (through December 31, 2019). Additional estimates used synthetic control methods with comparator stores in Los Angeles, California. Estimates were inputted into a closed-cohort microsimulation model to estimate quality-adjusted life years (QALYs) and societal costs (in Oakland) from 6 SSB-associated disease outcomes. In the main analysis, SSB purchases declined by 26.8% (95% CI -39.0 to -14.7, p < 0.001) in Oakland after tax implementation, compared with Richmond. There were no detectable changes in purchases of untaxed beverages or sweet snacks or purchases in border areas surrounding cities. In the synthetic control analysis, declines in SSB purchases were similar to the main analysis (-22.4%, 95% CI -41.7% to -3.0%, p = 0.04). The estimated changes in SSB purchases, when translated into declines in consumption, would be expected to accrue QALYs (94 per 10,000 residents) and significant societal cost savings (>$100,000 per 10,000 residents) over 10 years, with greater gains over a lifetime horizon. Study limitations include a lack of SSB consumption data and use of sales data primarily from chain stores. An SSB tax levied in Oakland was associated with a substantial decline in volume of SSBs purchased, an association that was sustained more than 2 years after tax implementation. Our study suggests that SSB taxes are effective policy instruments for improving health and generating significant cost savings for society.

Economic analysis of antenatal screening for human T-cell lymphotropic virus type 1 in Brazil: an open access cost-utility model

Human T-cell lymphotropic virus type 1 (HTLV-1) is a retrovirus that causes severe diseases, such as aggressive cancer or progressive neurological disease. HTLV-1 affects mainly people in areas with low human development index and can be transmitted from mother to child, primarily through breastfeeding. Refraining from breastfeeding is an effective intervention to reduce the risk of infection in infants. However, HTLV-1 antenatal screening is not offered globally. According to WHO, the scarcity of cost-effectiveness studies is considered one of the major barriers to the implementation of policies to prevent HTLV-1 infection. Therefore, this study aimed to assess the cost-effectiveness of antenatal screening and postnatal interventions to prevent HTLV-1 mother-to-child transmission in Brazil and to develop an open-access, editable, mathematical model that can be used by other countries and regions to assess different scenarios. In this cost-utility analysis, we constructed a decision tree and a Markov model to assess the cost-effectiveness of HTLV-1 antenatal screening and postnatal interventions (ie, avoidance of breastfeeding, by suppression of lactation with cabergoline, and provision of formula feed) to reduce transmission. For our model, we used data from Brazil and we took the perspective of the public health-care system to estimate costs. The implementation of both screening and interventions would result in the prevention of 1039 infections in infants every year in Brazil with an incremental cost-effectiveness ratio (ICER) of US$11 415 per quality-adjusted life-year (QALY). 88% of all probabilistic sensitivity analysis simulations had ICER values lower than the Brazilian cost-effectiveness threshold ($18 107·74 per QALY). HTLV-1 prevalence in pregnant women, the risk of HTLV-1 transmission when breastfeeding lasts for 6 months or more, and the cost of screening tests were the variables with the largest effect on ICER. HTLV-1 antenatal screening is cost-effective in Brazil. An open-access model was developed, and this tool could be used to assess the cost-effectiveness of such policy globally, favouring the implementation of interventions to prevent HTLV-1 mother-to-child transmission worldwide. None. For the Portuguese and Spanish translations of the abstract see Supplementary Materials section.