Health Economic Studies Research Articles

Economic Evaluations of Magnetic Resonance Image-Guided Radiotherapy (MRIgRT): A Systematic Review.

Objectives: This review systematically summarizes the evidence on the economic impact of magnetic resonance image-guided RT (MRIgRT). Methods: We systematically searched INAHTA, MEDLINE, and Scopus up to March 2022 to retrieve health economic studies. Relevant data were extracted on study type, model inputs, modeling methods and economic results. Results: Five studies were included. Two studies performed a full economic assessment to compare the cost-effectiveness of MRIgRT with other forms of image-guided radiation therapy. One study performed a cost minimization analysis and two studies performed an activity-based costing, all comparing MRIgRT with X-ray computed tomography image-guided radiation therapy (CTIgRT). Prostate cancer was the target condition in four studies and hepatocellular carcinoma in one. Considering the studies with a full economic assessment, MR-guided stereotactic body radiation therapy was found to be cost effective with respect to CTIgRT or conventional or moderate hypofractionated RT, even with a low reduction in toxicity. Conversely, a greater reduction in toxicity is required to compete with extreme hypofractionated RT without MR guidance. Conclusions: This review highlights the great potential of MRIgRT but also the need for further evidence, especially for late toxicity, whose reduction is expected to be the real added value of this technology.

Leveraging Comprehensive Cancer Registry Data to Enable a Broad Range of Research, Audit and Patient Support Activities.

Simple SummaryRegistry data has the potential to support a broad range of research, audit and education initiatives. Here, we describe the experience and learnings of a series of large multi-institutional cancer registries that leverage real-world clinical data for a range of purposes, that informs the conduct and output of each registry in a virtuous cycle. Lessons learnt include the need for careful and continuous curation of information being collected, regular database updates, and the need for a continued focus on data quality. As a standalone resource, each registry has supported numerous projects, but linkage with external datasets with patients in common has enhanced the research potential. Multiple projects have linked registry data with matched tissue specimens to support the discovery and valiation of prognostic and predictive markers in the tumour and blood specimens. Registry-based biomarker trials have been successfully supported, generating novel and practice-changing data. Registry-based clinical trials, particularly studies exploring the best use of drug options are now complementing the research conducted in traditional clinical trials. More recent projects supported by the registries include health economic studies, personalised patient education material, and increased consumer engagement, including consumer entered data.Traditional cancer registries have often been siloed efforts, established by single groups with limited objectives. There is the potential for registry data to support a broad range of research, audit and education initiatives. Here, we describe the establishment of a series of comprehensive cancer registries across the spectrum of common solid cancers. The experience and learnings of each registry team as they develop, implement and then use collected data for a range of purposes, that informs the conduct and output of other registries in a virtuous cycle. Each registry is multi-site, multi-disciplinary and aims to collect data of maximal interest and value to a broad range of enquiry, which would be accessible to any researcher with a high-quality proposal. Lessons learnt include the need for careful and continuous curation of data fields, with regular database updates, and the need for a continued focus on data quality. The registry data as a standalone resource has supported numerous projects, but linkage with external datasets with patients in common has enhanced the audit and research potential. Multiple projects have linked registry data with matched tissue specimens to support prognostic and predictive biomarker studies, both validation and discovery. Registry-based biomarker trials have been successfully supported, generating novel and practice-changing data. Registry-based clinical trials, particularly randomised studies exploring the optimal use of available therapy options are now complementing the research conducted in traditional clinical trials. More recent projects supported by the registries include health economic studies, personalised patient education material, and increased consumer engagement, including consumer entered data.

The Economic Burden of Localized Prostate Cancer and Insights Derived from Cost-Effectiveness Studies of the Different Treatments.

Simple SummaryProstate cancer is one of the most frequent and impacting malignant neoplasms for men. In particular, localized prostate cancer has a notably high incidence and prevalence, despite which a solid consensus on treatment and procedure of care has not yet been reached. This article aims to shed light on this challenge by characterizing the economic burden and cost-effectiveness of different treatment strategies for localized prostate cancer after analyzing published comparable data from studies conducted in OECD countries.Prostate cancer has huge health and societal impacts, and there is no clear consensus on the most effective and efficient treatment strategy for this disease, particularly for localized prostate cancer. We have reviewed the scientific literature describing the economic burden and cost-effectiveness of different treatment strategies for localized prostate cancer in OECD countries. We initially identified 315 articles, studying 13 of them in depth (those that met the inclusion criteria), comparing the social perspectives of cost, time period, geographical area, and severity. The economic burden arising from prostate cancer due to losses in productivity and increased caregiver load is noticeable, but clinical decision-making is carried out with more subjective variability than would be advisable. The direct cost of the intervention was the main driver for the treatment of less severe cases of prostate cancer, whereas for more severe cases, the most important determinant was the loss in productivity. Newer, more affordable radiotherapy strategies may play a crucial role in the future treatment of early prostate cancer. The interpretation of our results depends on conducting thorough sensitivity analyses. This approach may help better understand parameter uncertainty and the methodological choices discussed in health economics studies. Future results of ongoing clinical trials that are considering genetic characteristics in assessing treatment response of patients with localized prostate cancer may shed new light on important clinical and pharmacoeconomic decisions.

Long-term spill-over impact of COVID-19 on health and healthcare of people with non-communicable diseases: a study protocol for a population-based cohort and health economic study

IntroductionThe COVID-19 pandemic has a significant spill-over effect on people with non-communicable diseases (NCDs) over the long term, beyond the direct effect of COVID-19 infection. Evaluating changes in health outcomes,...

The health, cost and equity impacts of restrictions on the advertisement of high fat, salt and sugar products across the transport for London network: a health economic modelling study

BackgroundPolicies aimed at restricting the marketing of high fat, salt and sugar products have been proposed as one way of improving population diet and reducing obesity. In 2019, Transport for London implemented advertising restrictions on high fat, salt and sugar products. A controlled interrupted time-series analysis comparing London with a north of England control, suggested that the advertising restrictions had resulted in a reduction in household energy purchases. The aim of the study presented here was to estimate the health benefits, cost savings and equity impacts of the Transport for London policy using a health economic modelling approach, from an English National Health Service and personal social services perspective.MethodsA diabetes prevention microsimulation model was modified to incorporate the London population and Transport for London advertising intervention. Conversion of calorie to body mass index reduction was mediated through an approximation of a mathematical model estimating weight loss. Outcomes gathered included incremental obesity, long-term diabetes and cardiovascular disease events, quality-adjusted life years, healthcare costs saved and net monetary benefit. Slope index of inequality was calculated for proportion of people with obesity across socioeconomic groups to assess equity impacts.ResultsThe results show that the Transport for London policy was estimated to have resulted in 94,867 (4.8%) fewer individuals with obesity, and to reduce incidence of diabetes and cardiovascular disease by 2,857 and 1,915 cases respectively within three years post intervention. The policy would produce an estimated 16,394 additional quality-adjusted life-years and save £218 m in NHS and social care costs over the lifetime of the current population. Greater benefits (e.g. a 37% higher gain in quality-adjusted life-years) were expected to accrue to individuals from the most socioeconomically deprived groups compared to the least deprived.ConclusionsThis analysis suggests that there are considerable potential health and economic gains from restricting the advertisement of high fat, salt and sugar products. The population health and economic impacts of the Transport for London advertising restrictions are likely to have reduced health inequalities in London.

Health economics in Africa from 1991 to 2020: A systematic review.

This systematic review was conducted to identify, evaluate and characterize the overall progress of health economics research conducted for Africa. Health economics studies carried out from 1991 to 2020 for Africa were retrieved from the EconLit database using relevant searching strategies. According to the methodology of Preferred Reporting Items for Systematic Review and Meta-Analysis, qualified journal papers were included. Using bibliometrics, we ran a series of analyses on authorship, studied countries, affiliations, and countries of origin, journals, and research topics. A total of 2935 studies were screened, and 178 were included in this review. We observed that the determinants of illness is the most researched topics. The United States, World Bank, University of California Berkeley, are respectively the most influential countries, world organizations, and academic institutions in the field of health economics of Africa. HIV/AIDs is still the leading health issue in highly cited health economics studies in Africa. Health Policy and Planning is the most productive and academically influential journal, and Kenya is the most studied country by health economists among all African countries. African health systems are vulnerable compared to developed countries, as many of them are underfunded. The academic strength in Africa is much weaker than that of leading health economics counties. Even within the continent, the academic development and the attention it receives are uneven. More influential health economics studies of Africa should be published in addition to the disease focus of HIV/AIDS.

Is tilapia mortality a latent concern for the aquaculture sector of Bangladesh? An epidemiology and health economic impact study

Tilapia are the 3rd most culturedaquaculture species in global aquaculture, produced in over 170 countries. Bangladesh is the world's fourth-largest tilapia producer and yet only few studies have been conducted to understand factors associated with tilapia mortality and economic losses. Using an online tilapia epidemiology and health economics survey tool, we surveyed 565 tilapia farms in 15 of Bangladesh's most important tilapia-producing districts. The study examined a range of factors, including geographic locations, farm characteristics, water source, stocking, biosecurity measures, baseline and unusual mortality levels and characteristics. For the period January 2017 to February 2019 a total of 18.2% of farms (103 out of 565) reported having experienced unusual mortality, with an average mortality level of 23.2% (range 3 to 90). A number of factors were found to be significantly associated with reporting of unusual mortality occurrences, including farmer education level, farm size, farm biosecurity measures, baseline mortality level, farmer concern about baseline mortality, dead fish removal frequency and disposal method and antibiotic treatment. Farming region, water source, dead fish removal frequency, and antibiotic treatment were all found to be significantly associated with the level of unusual mortality, with water source and dead fish removal frequency remained significant in the multivariable model. Major clinical signs linked with these mortalities included skin erosions, hemorrhagic lesions, open wounds, skin discoloration, exophthalmos, abdominal distension, swelling, scale protrusion and eye opacification. Based on baseline and unusual mortality in tilapia, a total hidden loss of 875.7 million USD annually was estimated. To ensure the future sustainability of tilapia production, the authors recommend more investigations of unusual mortalities events with collection of metadata and clinical samples for disease diagnostics, coupled with nationwide farmer awareness campaigns.

Safety and efficacy of rituximab versus dimethyl fumarate in patients with relapsing-remitting multiple sclerosis or clinically isolated syndrome in Sweden: a rater-blinded, phase 3, randomised controlled trial

B-cell depleting therapies are highly efficacious in relapsing-remitting multiple sclerosis but one such therapy, rituximab, is not approved for multiple sclerosis and no phase 3 trial data are available. We therefore examined the safety and efficacy of rituximab compared with dimethyl fumarate in patients with relapsing-remitting multiple sclerosis to obtain data that might allow inclusion of rituximab in treatment guidelines. RIFUND-MS was a multicentre, rater-blinded, active-comparator, phase 3, randomised controlled trial done at 17 Swedish university and community hospitals. Key inclusion criteria for participants were: age 18-50 years; relapsing-remitting multiple sclerosis or clinically isolated syndrome according to prevailing McDonald criteria; 10 years or less since diagnosis; untreated or only exposed to interferons or glatiramer acetate; and with clinical or neuroradiological disease activity in the past year. Patients were automatically randomly assigned (1:1) by the treating physician using a randomisation module in the Swedish multiple sclerosis registry, without stratification, to oral dimethyl fumarate 240 mg twice daily or to intravenous rituximab 1000 mg followed by 500 mg every 6 months. Relapse evaluation, Expanded Disability Status Scale rating, and assessment of MRI scans were done by examining physicians and radiologists masked to treatment allocation. The primary outcome was the proportion of patients with at least one relapse (defined as subacute onset of new or worsening neurological symptoms compatible with multiple sclerosis with a duration of more than 24 h and preceded by at least 30 days of clinical stability), assessed in an intention-to-treat analysis using log-binomial regression with robust standard errors. This trial is registered at ClinicalTrials.gov, NCT02746744. Between July 1, 2016, and Dec 18, 2018, 322 patients were screened for eligibility, 200 of whom were randomly assigned to a treatment group (100 assigned to rituximab and 100 assigned to dimethyl fumarate). The last patient completed 24-month follow-up on April 21, 2021. 98 patients in the rituximab group and 97 patients in the dimethyl fumarate group were eligible for the primary outcome analysis. Three (3%) patients in the rituximab group and 16 (16%) patients in the dimethyl fumarate group had a protocol-defined relapse during the trial, corresponding to a risk ratio of 0·19 (95% CI 0·06-0·62; p=0·0060). Infusion reactions (105 events [40·9 per 100 patient-years]) in the rituximab group and gastrointestinal reactions (65 events [47·4 per 100 patient-years]) and flush (65 events [47·4 per 100 patient-years]) in the dimethyl fumarate group were the most prevalent adverse events. There were no safety concerns. RIFUND-MS provides evidence that rituximab given as 1000 mg followed by 500 mg every 6 months is superior to dimethyl fumarate in preventing relapses over 24 months in patients with early relapsing-remitting multiple sclerosis. Health economic and long-term safety studies of rituximab in patients with multiple sclerosis are needed. Swedish Research Council.

Further support for rituximab in relapsing multiple sclerosis

B cells have long been implicated in multiple sclerosis and, in the past few years, ocrelizumab and ofatumumab, drugs that target anti-CD20-expressing B cells, have been approved for use. The original anti-CD20 therapy, rituximab, is used off-label in some countries, and results from a clinical trial (RIFUND-MS) by Anders Svenningsson and colleagues 1 Svenningsson A Frisell T Burman J et al. Safety and efficacy of rituximab versus dimethyl fumarate in patients with relapsing-remitting multiple sclerosis or clinically isolated syndrome in Sweden: a rater-blinded, phase 3, randomised controlled trial. Lancet Neurol. 2022; 21: 693-703 Summary Full Text Full Text PDF PubMed Scopus (1) Google Scholar published in The Lancet Neurology help to fill a gap in our knowledge about how useful this monoclonal antibody might be for patients with relapsing-remitting multiple sclerosis. Safety and efficacy of rituximab versus dimethyl fumarate in patients with relapsing-remitting multiple sclerosis or clinically isolated syndrome in Sweden: a rater-blinded, phase 3, randomised controlled trialRIFUND-MS provides evidence that rituximab given as 1000 mg followed by 500 mg every 6 months is superior to dimethyl fumarate in preventing relapses over 24 months in patients with early relapsing-remitting multiple sclerosis. Health economic and long-term safety studies of rituximab in patients with multiple sclerosis are needed. Full-Text PDF

Enhancing Health Equity Through Cancer Health Economics Research.

Cancer displays some of the largest health-equity concerns of all diseases. This paper draws attention to how health economics research can assess strategies to reduce or even eliminate health disparities and provides pivotal examples of existing research as well as areas for future contributions. The paper also highlights critical data limitations that currently restrain the impact health economics research could have. We then explore new areas of inquiry where economic research is sparse but could have an important impact on health equity, particularly in topics involving Medicare and Medicaid policies that expand reimbursement and generosity of coverage. Health economics studies are notably absent from policies and practices surrounding clinical trials, representing an opportunity for future research. We urge health economics researchers to consider experiments, interventions, and assessments through primary data collection; we further encourage the formulation of multidisciplinary teams to ensure that health economics skills are well melded with other areas of expertise. These teams are needed to maximize novelty and rigor of evidence. As policies are promulgated to address disparities in cancer, involvement of economics in a multidisciplinary context can help ensure that these policies do not have unintended impacts that may deepen inequities.

Research on the Economics of Cancer-Related Health Care: An Overview of the Review Literature.

We reviewed current literature reviews regarding economics of cancer-related health care to identify focus areas and gaps. We searched PubMed for systematic and other reviews with the Medical Subject Headings "neoplasms" and "economics" published between January 1, 2010, and April 1, 2020, identifying 164 reviews. Review characteristics were abstracted and described. The majority (70.7%) of reviews focused on cost-effectiveness or cost-utility analyses. Few reviews addressed other types of cancer health economic studies. More than two-thirds of the reviews examined cancer treatments, followed by screening (15.9%) and survivorship or end-of-life (13.4%). The plurality of reviews (28.7%) cut across cancer site, followed by breast (20.7%), colorectal (11.6%), and gynecologic (8.5%) cancers. Specific topics addressed cancer screening modalities, novel therapies, pain management, or exercise interventions during survivorship. The results indicate that reviews do not regularly cover other phases of care or topics including financial hardship, policy, and measurement and methods.

Evaluating the quality of the economic evidence in colorectal cancer genomics studies.

The increase in the use of genome-based screening and diagnostic tests adds to the overall costs of oncologic care for colorectal cancer. This, in turn, has resulted in an increase in published economic analyses. Aim: To perform a systematic literature review of the available economic evidence evaluating the value of genomic testing for colorectal cancer and appraise the quality of the economic studies conducted to date. Methods: A systematic review of the literature for economic studies of colorectal cancer genomics from January 2006 through October 2020, and evaluation of study quality using the Quality of Health Economic Studies (QHES) instrument was conducted. The validated QHES was then applied to a final set of articles that met eligibility criteria. Results: Our search of the literature initially yielded 12,859 records. A final set of 49 articles met our inclusion criteria. The QHES score ranged from 24 to 100, with an average score of 82. Most of the studies (n=40, 82%) scored above 75 and were considered of good quality. Conclusion: Our analysis revealed that most of the economic analyses of colorectal cancer genomic molecular diagnostics in the literature may be of good quality. There is, however, some variation in methodological rigor between the articles.

P-731 Costs and effects accounted for in economic evaluations of Medically Assisted Reproduction: a systematic review

Abstract Study question Which costs and effects must be considered in an economic evaluation of Medically Assisted Reproduction (MAR) and to which extent do published studies consider these? Summary answer Current economic evaluations of MAR focus on technical efficiency using narrow short-term perspectives and excluding relevant costs/effects, and hence, fail to provide adequate ‘value-for-money’ information. What is known already Due to limited budgets of health systems and patients, decision-makers need to understand which MAR interventions are cost-effective. However, it is complex to evaluate MAR from an economic perspective, as MAR treatment involves women, men, their relationship, and potential children born. Hence, it remains unclear which costs and effects related to these different stakeholders should be accounted for, and over which time horizon. In this study, we performed a systematic review of health economic studies in MAR and compared the results with a predefined value-framework providing 12 cost categories and 9 effect categories. Study design, size, duration A systematic review was performed of articles (published 2010-2021) in PubMed Central, Embase, Web of Science CC, CINAHL, Cochrane (CENTRAL), HTA and NHS EED. Using the PICO framework, full economic evaluations (cost-utility analysis (CUA), cost-benefit analysis (CBA), cost-minimization analysis (CMA) and cost-effectiveness analysis (CEA)) were selected and assessed. We identified 66 economic evaluations of MAR, including the analysis of 81 Incremental Cost-Effectiveness Ratios (ICER) as 15 studies presented more than one ICER. Participants/materials, setting, methods Our value-framework lists 12 cost-categories (infertility diagnosis, MAR, pregnancy, delivery, post-natal -care, basic/complicated childcare, care for involuntary childlessness, productivity losses (MAR/pregnancy/childcare), patient out-of-pocket expenses, broader long-term social costs) and 9 effect-categories (health child/parents short- and long-term, well-being child/parents short- and long-term, broader long-term social benefits) of MAR, indicating which costs/effects are relevant for different analytical methods (CEA/CUA/CBA), time-horizons (<1 year/1-5 years/>5 years) and perspectives (health system/patient/society). All ICERs were analysed based on their included costs/effects. Main results and the role of chance There is a large scope of costs and effects potentially relevant to understand the economic value of MAR. Of the 66 included studies, 88% performed a CEA whereas 2% performed a CUA. Most ICERs expressed cost-effectiveness as cost per live birth (52%) or cost per pregnancy (24%) whereas one study expressed the result as cost per QALY. 24% of all ICERs adopted the broader societal perspective, whereas 61% adopted the narrower health system perspective. Time horizons considered were very heterogeneous, with 68% not looking further than one year after treatment and 1% considering a time horizon of a lifetime. Only the direct costs of MAR and the short-term medical outcomes of born children were consistently accounted for in all ICERs. The following significant costs and effects were largely neglected: costs related to diagnostics, pregnancy and delivery (including complications), postnatal follow-up care and childcare; indirect costs (patient out-of-pocket expenses, productivity losses); indirect effects of infertility on the health of wish parents; longer term costs and effects of a live birth. These significant cost and effects were excluded due to the limited scope set by most studies, adopting short time horizons and narrow perspectives. Limitations, reasons for caution The systematic review relied on available databases of published scientific articles. Therefore, economic evaluations published in the grey literature (government reports, unpublished manuscripts, etc.) may have been missed. Only articles published in English were included. Wider implications of the findings As MAR has broad implications for many stakeholders, future studies should adopt more accurate costing-methods (using our value-framework) and focus on allocative efficiency. Hence, the debate about the value-for-money of MAR and about how large MAR budgets should be relative to those of other disease areas, will be better informed. Trial registration number Not applicable

MSR48 Estimating Event-Specific Probabilities and Conditional Time-to-Event Distributions from Censored Competing Risks Data

Accurate modelling of competing risk data is crucial in health economic modelling studies. Although different approaches have been investigated for a variety of contexts, the important case of censored data remains not fully explored. We fill this gap by defining and evaluating a “sample the event first and time-to-event second” approach.

Estimating population norms for the health-related quality of life of adults in southern Jiangsu Province, China

Although national health-related quality of life population norms had been published based on the EuroQol 5-Dimensions 5-levels scale, China is a vast country with diverse cultural and social development in various regions. Therefore, regional population norms may better reflect the health status of residents in a given area. The purpose of the study was to derive the HRQoL population norm for adult general population in southern Jiangsu Province using the EQ-5D-5L scale and explore potential influencing factors. The data were based on a cross-sectional survey conducted in Liyang City from March 2019 to July 2020. EQ-5D-5L utility scores based on Chinese value set and EQ-VAS scores were used to assess HRQoL. The Tobit regression model and generalized linear model were performed to identify the association among potential covariates and HRQoL. The means (95% confidence interval) of the EQ-5D-5L utility scores and EQ-VAS scores were 0.981(0.980–0.983) and 83.6(83.2–83.9), respectively. Younger people (≤ 40 years old) were more likely to experience problems with anxiety or depression. Additionally, women had lower HRQoL scores although multivariate analysis found no statistical difference between the sexes. Lower HRQoL was associated with advanced age, lower socioeconomic status, no spouse, lack of regular physical activities, smoking cessation, and chronic non-communicable diseases. Subjects who declared that they were afflicted by diseases presented significantly lower utility scores, ranging from 0.823 (0.766–0.880) for memory-related diseases to 0.978 (0.967–0.989) for hepatic diseases. Regional population norms of HRQoL are needed in the health economic study owing to the great socioeconomic differences across regions in China. The present study provides HRQoL population norms for adults in southern Jiangsu. These norm values could help policy makers better allocate limited health resources and prioritize service plans.

Preliminary study on the existing problems in health economic evaluation study of acupuncture in China

The literature of health economic evaluation study in the field of acupuncture in China was systematically summarized and analyzed, and the existing problems in the current research were discussed from the aspects of research perspective, cost calculation scope, data analysis method selection. Moreover, the key points of the health economic evaluation research were summarized, and the research objectives, the relationship between the expected research results and data analysis methods and the process of thinking were sorted out, and several suggestions for research report writing were proposed, aiming to provide a reference for the quality improvement of the acupuncture health economic evaluation research in China.

0550 Health-Economic Implications of Defined Improvements in Restless Leg Syndrome Severity: A Model-Based Exploratory Analysis based on Prior Publication Data

Abstract Introduction Restless leg syndrome (RLS) is a debilitating condition associated with reduced health-related quality of life (HRQoL) and increased resource utilization. Our objective was to provide model-based estimates of quality-adjusted life year (QALY) gain and reduction in healthcare utilization that might be expected for changes in symptom severity. Methods We identified two health-economic studies which reported severity-specific quality-of-life and resource utilization. Happe et al. 2009 reported HRQoL using EQ-5D measurements stratified by International Restless Legs Scale (IRLS) severity (n=519, 63.6% female, 64.2 years) and Durgin et al. 2015 reported healthcare utilization stratified by RLS severity (no/mild/moderate/severe RLS) (n=2,392, 56.9% female, 56.0 years). We fitted regression models to support IRLS-specific estimates of EQ-5D and healthcare resource utilization. These models (polynomial for EQ-5D, linear for resource utilization) were applied to calculate QALY gain, and events avoided for ER visits, hospitalizations, and healthcare provider (HCP) visits over 5 years for IRLS improvement of 1-10 points, from a baseline of 30, 25, and 20. Results At five years, a 1, 3, 5, 7, and 10-point IRLS reduction corresponded to a QALY gain of 0.09, 0.25, 0.40, 0.52, and 0.69 for a baseline of 30, 0.07, 0.19, 0.29, 0.37, and 0.47 for a baseline of 25, and 0.04, 0.12, 0.18, 0.22, and 0.26 for a baseline of 20. The events avoided over 5 years with an IRLS reduction of 1, 3, 5, 7, and 10 points respectively were 1.12, 3.37, 5.62, 7.87, and 11.25 HCP visits, 0.09, 0.26, 0.43, 0.61, and 0.87 ER visits, and 0.04, 0.11, 0.18, 0.26, and 0.36 hospitalizations. Conclusion This exploratory analysis based on two health-economic studies confirms the substantial quality-of-life and healthcare cost burden associated with RLS. The data provide guidance on the magnitude of potential quality-of-life improvement and cost reduction that might be associated with defined improvement in RLS severity. Further investigation may be warranted for the factors that link change in IRLS and healthcare resource utilization, such as sleep deficits or cardiovascular risks. Support (If Any) Funding was provided by Noctrix Health

A randomized controlled phase III study comparing hadrontherapy with carbon ions versus conventional radiotherapy – including photon and proton therapy – for the treatment of radioresistant tumors: the ETOILE trial

BackgroundSome cancers such as sarcomas (bone and soft tissue sarcomas) and adenoid cystic carcinomas are considered as radioresistant to low linear energy transfer radiation (including photons and protons) and may therefore beneficiate from a carbon ion therapy. Despite encouraging results obtained in phase I/II trials compared to historical data with photons, the spread of carbon ions has been limited mainly because of the absence of randomized medical data. The French health authorities stressed the importance of having randomized data for carbon ion therapy.MethodsThe ETOILE study is a multicenter prospective randomized phase III trial comparing carbon ion therapy to either advanced photon or proton radiotherapy for inoperable or macroscopically incompletely resected (R2) radioresistant cancers including sarcomas and adenoid cystic carcinomas.In the experimental arm, carbon ion therapy will be performed at the National Center for Oncological Hadrontherapy (CNAO) in Pavia, Italy. In the control arm, photon or proton radiotherapy will be carried out in referent centers in France.The primary endpoint is progression-free survival (PFS). Secondary endpoints are overall survival and local control, toxicity profile, and quality of life. In addition, a prospective health-economic study and a radiobiological analysis will be conducted.To demonstrate an absolute improvement in the 5-year PFS rate of 20% in favor of carbon ion therapy, 250 patients have to be included in the study.DiscussionSo far, no clinical study of phase III has demonstrated the superiority of carbon ion therapy compared to conventional radiotherapy, including proton therapy, for the treatment of radioresistant tumors.Trial registrationClinicalTrials.gov identifier: NCT02838602. Date of registration: July 20, 2016. The posted information will be updated as needed to reflect protocol amendments and study progress.

Manual therapy versus advice to stay active for nonspecific back and/or neck pain: a cost-effectiveness analysis

BackgroundLow back and neck pain are the most common musculoskeletal disorders worldwide, and imply suffering and substantial societal costs, hence effective interventions are crucial. The aim of this study was to evaluate the cost-effectiveness of manual therapy compared with advice to stay active for working age persons with nonspecific back and/or neck pain.MethodsThe two interventions were: a maximum of 6 manual therapy sessions within 6 weeks, including spinal manipulation/mobilization, massage and stretching, performed by a naprapath (index group), respectively information from a physician on the importance to stay active and on how to cope with pain, according to evidence-based advice, at 2 occasions within 3 weeks (control group). A cost-effectiveness analysis with a societal perspective was performed alongside a randomized controlled trial including 409 persons followed for one year, in 2005. The outcomes were health-related Quality of Life (QoL) encoded from the SF-36 and pain intensity. Direct and indirect costs were calculated based on intervention and medication costs and sickness absence data. An incremental cost per health related QoL was calculated, and sensitivity analyses were performed.ResultsThe difference in QoL gains was 0.007 (95% CI − 0.010 to 0.023) and the mean improvement in pain intensity was 0.6 (95% CI 0.068–1.065) in favor of manual therapy after one year. Concerning the QoL outcome, the differences in mean cost per person was estimated at − 437 EUR (95% CI − 1302 to 371) and for the pain outcome the difference was − 635 EUR (95% CI − 1587 to 246) in favor of manual therapy. The results indicate that manual therapy achieves better outcomes at lower costs compared with advice to stay active. The sensitivity analyses were consistent with the main results.ConclusionsOur results indicate that manual therapy for nonspecific back and/or neck pain is slightly less costly and more beneficial than advice to stay active for this sample of working age persons. Since manual therapy treatment is at least as cost-effective as evidence-based advice from a physician, it may be recommended for neck and low back pain. Further health economic studies that may confirm those findings are warranted.Trial registration Current Controlled Trials ISRCTN56954776. Retrospectively registered 12 September 2006, http://www.isrctn.com/ISRCTN56954776.

The Health Economics of Metastatic Hormone-Sensitive and Non-Metastatic Castration-Resistant Prostate Cancer-A Systematic Literature Review with Application to the Canadian Context.

Background: Health economic evaluations are needed to assess the impact on the healthcare system of emerging treatment patterns for advanced prostate cancer. The objective of this study is to review the scientific literature identifying cost-effectiveness and cost analyses that are assessing treatments for metastatic hormone-sensitive prostate cancer (mHSPC) and nonmetastatic castration-resistant prostate cancer (nmCRPC). Methods: On 29 June 2021, we searched the scientific (MEDLINE, Embase, and EBSCO) and grey literature for health economic studies targeting mHSPC and nmCRPC. We used the CHEC-extended checklist and the Welte checklist for risk-of-bias assessment and transferability analysis, respectively. Results: We retained 20 cost-effectiveness and 4 cost analyses in the mHSPC setting, and 14 cost-effectiveness and 6 cost analyses in the nmCRPC setting. Docetaxel in combination with androgen deprivation therapy (ADT) was the most cost-effective treatment in the mHSPC setting. Apalutamide, darolutamide, and enzalutamide presented similar results vs. ADT alone and were identified as cost-effective treatments for nmCRPC. An increase in costs as patients transitioned from nmCRPC to mCRPC was noted. Conclusions: We concluded that there is an important unmet need for health economic evaluations in the mHSPC and nmCRPC setting incorporating real-world data to support healthcare decision making.