Cost-effectiveness Analysis Of Treatment Research Articles

Development of a novel patient reported outcome measure for health-related quality of life in amyotrophic lateral sclerosis (PROQuALS): study protocol

BackgroundPatient reported outcome measures (PROMs) can be used to assess the impact of health conditions upon an individual’s health-related quality of life (HRQoL). Whilst PROMs have been used to quantify the HRQoL impact of amyotrophic lateral sclerosis (ALS), existing instruments may not fully capture what matters to people living with ALS (plwALS) or be appropriate to be used directly to inform the cost-effectiveness of new treatments. This highlights a need for a new condition-specific PROM that can both capture what’s important to plwALS and be used in economic evaluation. This study has two key aims: 1) to produce a novel PROM for measuring HRQoL in plwALS (PROQuALS). 2) to value a set of items from the novel PROM to generate an associated preference-weighted measure (PWM) that will enable utility values to be generated.MethodsA mixed-methods study design will be conducted across three stages. Stage 1 involves concept elicitation and the generation of draft PROM content from a robust and comprehensive systematic review of HRQoL in ALS, with input from plwALS. Stage 2 consists of cognitive debriefing of the draft PROM content to ascertain its content validity (Stage 2a), followed by a psychometric survey (Stage 2b) to assess statistical performance. Evidence from Stage 2 will be used to make decisions on the final content and format of the novel PROM. Stage 3 will involve valuation and econometric modeling using health economics methods to generate preference weights, so a PWM derived from the novel PROM can be used in the cost-effectiveness analyses of treatments. Patient and clinical advisory groups will have critical, collaborative input throughout the project.DiscussionThe novel PROM will be designed to comprehensively assess important aspects of HRQoL to plwALS and to quantify HRQoL in terms of subjective impact. The PROQuALS measure will be available for use in research and healthcare settings. The associated PWM component will extend and enable the use of PROQuALS in cost-effective analyses of new treatments for ALS.Trial registrationNot applicable.

Correction to: Cost Effectiveness Analyses of Interventions for Osteoporosis in Men: A Systematic Literature Review

Correction to: Cost Effectiveness Analyses of Interventions for Osteoporosis in Men: A Systematic Literature Review

Medical Costs Associated With Diabetes Complications in Medicare Beneficiaries Aged 65 Years or Older With Type 1 Diabetes.

To estimate medical costs associated with 17 diabetes complications and treatment procedures among Medicare beneficiaries aged ≥65 years with type 1 diabetes. With use of the 2006-2017 100% Medicare claims database for beneficiaries enrolled in fee-for-service plans and Part D, we estimated the annual cost of 17 diabetes complications and treatment procedures. Type 1 diabetes and its complications and procedures were identified using ICD-9/ICD-10, procedure, and diagnosis-related group codes. Individuals with type 1 diabetes were followed from the year when their diabetes was initially identified in Medicare (2006-2015) until death, discontinuing plan coverage, or 31 December 2017. Fixed-effects regression was used to estimate costs in the complication occurrence year and subsequent years. The cost proportion of a complication was equal to the total cost of the complication, calculated by multiplying prevalence by the per-person cost divided by the total cost for all complications. All costs were standardized to 2017 U.S. dollars. Our study included 114,879 people with type 1 diabetes with lengths of follow-up from 3 to 10 years. The costliest complications per person were kidney failure treated by transplant ($77,809 in the occurrence year and $13,556 in subsequent years), kidney failure treated by dialysis ($56,469 and $41,429), and neuropathy treated by lower-extremity amputation ($40,698 and $7,380). Sixteen percent of the total medical cost for diabetes complications was for treating congestive heart failure. Costs of diabetes complications were large and varied by complications. Our results can assist in cost-effectiveness analysis of treatments and interventions for preventing or delaying diabetes complications in Medicare beneficiaries aged ≥65 years with type 1 diabetes.

Interventions delivered in secondary or tertiary medical care settings to improve routine vaccination uptake in children and young people: a scoping review

ObjectiveTo identify and analyse the interventions delivered opportunistically in secondary or tertiary medical settings, focused on improving routine vaccination uptake in children and young people.DesignScoping review.Search strategyWe searched CINAHL, Web...

Cost-effectiveness analysis of treatment with varenicline for nicotine dependence compared with smoking cessation without pharmacotherapy in the real world.

Smoking is an important public health issue. Although measures to support smoking cessation have been implemented worldwide, smokers often fail to quit smoking after receiving pharmacotherapies for nicotine dependence. The present study evaluated the cost-effectiveness of varenicline for smoking cessation compared with no pharmacotherapy using actual paid medical cost data in Japan. This was a retrospective cohort study of 3657 subjects who had quit smoking with varenicline or no pharmacotherapy. We extracted health examination and medical claim data from a health insurer database for the period 2012-2015. We calculated the incremental cost-effective ratio (ICER) of varenicline using actual paid medical costs for nicotine dependence and the number needed to treat to maintain smoking cessation compared with no pharmacotherapy, considering sex, age, income, and occupation. The 1- and 2-year smoking cessation maintenance rates were 69.7% and 62.4%, respectively. We found that 8.8% of subjects who quit smoking used varenicline for nicotine dependence and the cost per person was Japanese Yen (JPY) 52 177 (U.S. dollars [USD] 474; USD 1=JPY 110). The ICER of varenicline was dominant when comparing 2-year cessation with 1-year cessation. Male, age <40 years, low income, and manufacturing workers were the most cost-effective variables. The cost-effective variables of varenicline in the real world were investigated. The results of this study strengthen the evidence regarding which type of people should be targeted for measures to support smoking cessation using varenicline.

A cost-effectiveness analysis of front-line treatment strategies in early-stage follicular lymphoma

Recent data suggest the use of radiotherapy alone (RT) in Early-Stage Follicular Lymphoma is declining. Cost-effectiveness analysis of treatments has not been performed. We constructed a partitioning model (15-year horizon) to compare RT, combined-modality therapy (CMT) and immunochemotherapy with rituximab maintenance (ICT + RM) from a PET-staged cohort from the Australian Lymphoma Alliance. Lifetime direct health care costs, quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs) were calculated. AUD $75,000 was defined as the willingness-to-pay threshold (WTP). The direct healthcare costs were: RT $12,791, CMT $29,391 and ICT + RM $42,644. Compared with RT, CMT demonstrated minimal improvement in QALYs (+0.01) and an ICER well above the WTP threshold ($1,535,488). Compared with RT, ICT + RM demonstrated an improvement in QALYs (+0.41) with an ICER of $73,319. Modeling a 25% cost reduction with a rituximab biosimilar led to further ICER reductions with ICT + RM ($52,476). ICT + RM is cost-effective in early-stage FL from the Australian taxpayer perspective.

Resource Utilization Using Micro-Costing Method and Cost Effectiveness Analysis of Treatment of Acute Promyelocytic Leukemia with Generic Arsenic-Trioxide

We have previously reported on the challenges of treating AML in India, the major challenge being the cost of treatment (Chepsy et al. BJH 2015). With conventional treatment of AML it is recognized that the major part of this cost is not related to the cost of the individual chemotherapeutic drugs, but rather from the high cost of supportive care to take patients through repeated cycles of prolonged neutropenia induced by conventional chemotherapy. In contrast with arsenic trioxide (ATO) based regimens in acute promyelocytic leukemia (APL), when expensive patented ‘innovator’ ATO was used, the reported 3 year direct pharmacy cost of drugs was higher with an ATO+ATRA regime compared with a conventional ATRA+chemotherapy regimen (Euro 46,700 Vs 6,700), though the cost of supportive care was a third of conventional therapy (Kruse M et al. PLoS One. 2015). There is limited data on the cost effectiveness of using generic ATO, as available in India, in the treatment of APL (10 mg vial costs US $7 in India versus US $ 676 in North America)In a single center, retrospective study, 30 newly diagnosed APL (ND-APL) patients, treated with ATO and 16 relapsed APL (R-APL) patients, treated on a phase II trial with a ATO based regimen followed by an autologous stem cell transplant (regimens as previously reported by us; Mathews et al. Blood 2012 and Mathews et al. Blood 2016 abstract) were evaluated. In the absence of patients being treated at our center with a conventional ATRA+chemotherapy regimen (APL-CT) the cost of treatment for APL with such a regimen was assumed to be similar to the cost that would be incurred to treat a non-M3 AML patient at our center with a conventional AML chemotherapy schedule. Incremental cost effectiveness ratio was calculated using weighted costs with weighing factor obtained from survival data for 60 patients from our database for APL ATO group and from the AIDA 2000 trial for APL-CT group.A three phase micro-costing method (figure 1) used four cost centers involved in providing patient care: (i) chemotherapy administration (ii) supportive care (iii) treatment of complications (iv) laboratory services. For each of these cost centers there were four cost drivers: (i) drugs / reagents (ii) personnel (iii) ward (iv) instruments / overheads. The actual cost of cost drivers was found out by micro-costing and summed up to obtain the unit cost of each cost center (m). Each time a patient used a cost center this unit cost would be incurred. Next, the frequency of utilization (n) of each cost center was found out from the patient database and multiplied by the unit cost of the cost center to obtain the total cost of treatment in each protocol (m x n) (For illustration and international comparison all cost were converted to US$).In ND-APL patients, the mean cost of induction and consolidation was $5078.5±1581 and $902±212 respectively while in APL-CT it was $9812.1±3239 and $11803.1±4872 respectively (exchange rate 1 US$=61.87 Indian rupees). Our data illustrated that APL-CT group incurred higher costs mainly due to the greater duration of in-patient care that was required in this group and since they had higher rate of infectious complications (Table 1). During maintenance phase, in ND-APL, there were no in-patient stays and the entire chemotherapy was given on outpatient basis and the average cost was $2074±146 and the cost of following up these patients for an additional three years was $704.6±289. The mean lifetime cost of treatment of ND-APL using ATO was $8759.2±2078 while using APL-CT was $24393±5528. In R-APL patients, the total cost of re-induction and consolidation was $3498±1220 and $2274±372 respectively. The mean cost of an autologous-SCT was $4318±159. The average lifetime cost of treatment of relapsed APL patients using ATO based regimen followed by an autologous SCT was $12269.7±4915. The weighted cost per living for ATO and CT was estimated to be $12426 and $28330 respectively. The incremental cost effectiveness ratio showed ATO to be cheaper by $13359 for each life saved compared to conventional chemotherapy.ATO based regimen in ND-APL and R-APL are associated with significant reduction in resource utilization and costs of treatment and is also cost-effective as compared to conventional ATRA+chemotherapy. With the world still debating the best treatment for APL, generic ATO based regimen offers a significant opportunity for resource constrained countries to treat all patients with a diagnosis of APL. [Display omitted] DisclosuresNo relevant conflicts of interest to declare.

Quantifying spillover benefits in value assessment: a case study of increased graft survival on the US kidney transplant waitlist

Aim To quantify the wider impacts of increased graft survival on the size of the kidney transplant waitlist and health and economic outcomes. Materials and methods The analysis employed known steady-state solutions to a double-queueing system as well as simulations of this system. Baseline input parameters were sourced from the Organ Procurement and Transplant Network and the United States Renal Data System. Three increased graft survival scenarios were modeled: decreases in repeat transplant candidates joining the waitlist of 25%, 50%, and 100%. Results Under the three scenarios, we estimated that the US waitlist size would decrease from 91,822 to 85,461 (6.9% decrease), 80,073 (12.8% decrease), and 69,340 (24.4% decrease), respectively. Patient outcomes improved, with lifetime quality-adjusted life years (QALYs) for a 1-year cohort of transplant recipients increasing by 10,010, 16,888, and 43,345 over the three scenarios. Discounted lifetime costs for the cohort in the new steady state were lower by $1.6 billion, $2.3 billion, and $9.0 billion for each scenario, respectively. Spillover impacts (i.e. benefits that accrued beyond the patients who directly experienced increased graft survival) accounted for 58–65% of the QALY gains and ranged from cost increases of 3.3% to decreases of 5.5%. Limitations The model is a simplification of reality and does not account for the full degree of patient heterogeneity occurring in the real world. Health economic outcomes are extrapolated based on the assumption that the median patient is representative of the overall population. Conclusions Increasing graft survival reduces demand from repeat transplants candidates, allowing additional candidates to receive transplants. These spillover impacts decrease waitlist size and shorten wait times, leading to improvements in graft and patient survival as well as quality-of-life. Cost-effectiveness analyses of treatments that increase kidney graft survival should incorporate spillover benefits that accrue beyond the direct recipient of an intervention.

Cost of hospitalization for stroke in a low-middle-income country: Findings from a public tertiary hospital in the Philippines.

Determining the cost of hospitalization for acute stroke is important in the appropriate allocation of resources for public health facilities and in the cost effectiveness analysis of interventions. Despite being the second leading cause of mortality in the Philippines, there are no published data on the cost of stroke in the country. The study aims to determine the in-hospitalization cost for stroke (IHCS) in a tertiary public hospital in the Philippines and identify the factors influencing IHCS. The study was a retrospective review of the medical and billing records of the hospital. Adult patients admitted for acute stroke between 1 June 2017 and 31 May 2018 were included in the analysis. After the mean cost of stroke was determined, multivariate logistic regression analysis was done to determine demographic and clinical characteristics that were predictive of stroke cost. A total of 863 patient records were analyzed. The median in-hospitalization cost for stroke was PHP 17,141.50 or US$329.52. Independent determinants of higher cost include male sex (p = 0.021), stroke type (hemorrhagic stroke, p = 0.001; subarachnoid hemorrhage, p < 0.001), lower GCS on admission (p = 0.023), surgical intervention (p < 0.001), intravenous thrombolysis (p < 0.001), infection (p < 0.001), length of hospital stay (p < 0.001), and mechanical ventilation (p = 0.008). The study provided current data on the in-hospitalization cost of acute stroke in a public tertiary hospital in the Philippines. Male sex, stroke type, lower GCS on admission, surgical intervention, intravenous thrombolysis, infection, length of hospital stay, and mechanical ventilation were independent predictors of cost.

A cost effectiveness analysis of interventions to reduce residential radon exposure in Canada

The objective of this analysis is to estimate the incremental cost effectiveness ratios for the 2012 populations in Canada, each province/territory, and 17 census metropolitan areas, for practical radon mitigation scenarios to reduce residential radon exposures. Sixteen intervention scenarios compare radon mitigation implemented at differing rates in new and existing housing relative to preventive measures installed at construction, using three different radon mitigation thresholds. A period life-table analysis was conducted using data derived from two recent Canadian radon surveys, along with Canadian mortality and quality of life data. Analyses adopted a lifetime horizon and a discount rate of 1.5%. It is practical to reduce residential radon and associated lung cancer mortality in Canada, and the most cost effective scenario at each radon mitigation threshold is the combination of the activation of the preventive measures in new housing and mitigation of existing housing.

PP34 Costs Of Healthcare-Associated Infections In Latin America

IntroductionHealthcare-associated infections (HAI) are among the most common preventable health adverse event, associated with significant burden globally. Limited data on HAI costs in lower and middle-income countries is available. The aim of this study is to assess the cost, additional length-of-stay (LOS) and extra-mortality of HAI in the Latin American and Caribbean (LAC) Region.MethodsWe searched Medline/PubMed, Embase, Web of Science, Lilacs, Cochrane, National Health Service Economic Evaluation Database, Centre for Reviews and Dissemination, EconLit, and gray literature published in any language without restriction of date till July 2017. We included observational studies addressing the outcomes of interest, in which hospitalized patients with HAI are compared to those without HAI. The following study designs were included: quasi-experimental, controlled before-after, prospective and retrospective comparative cohort, case-control, and cross-sectional studies. We considered the following HAI-sites: surgical site infections (SSI), catheter-associated urinary-tract infections (CA-UTI), ventilator-associated pneumonia (VAP), and central line-associated bloodstream infection (CLA-BSI), as well as cross-infection (CI). Screening of citations, data extraction, and risk of bias assessment were conducted in duplicate by independent reviewers, according to the study protocol registered on PROSPERO. Reported costs were converted to USD considering official exchange rates.ResultsWe identified 4,339 citations. After removing duplicates, a total of 3,029 citations were screened for eligibility. A total of 87 studies from 17 countries were included. The majority (27.4 percent) reported on VAP, followed by CLA-BSI (21.2 percent), SSI (16.4 percent), and CA-UTI (14.4 percent). Most studies (46.7 percent) reported on incremental LOS, with an average of 14.8 days (range 0.9-49 days). Costs were reported by 25 percent of studies, with average incremental costs of USD 3,460 (range 49-12,155). Average extra-mortality of 15.6 percent (range -2.8-45.2 percent) was reported by 12.6 percent of studies.ConclusionsAvailable evidence from the LAC Region reports significant economic burden of HAI. This information will be useful for cost-effectiveness analysis of interventions aimed at reducing HAI economic and health burden.

Matching the model with the evidence: comparing discrete event simulation and state-transition modeling for time-to-event predictions in a cost-effectiveness analysis of treatment in metastatic colorectal cancer patients

BackgroundIndividual patient data, e.g. from clinical trials, often need to be extrapolated or combined with additional evidence when assessing long-term impact in cost-effectiveness modeling studies. Different modeling methods can be used to represent the complex dynamics of clinical practice; the choice of which may impact cost-effectiveness outcomes. We compare the use of a previously designed cohort discrete-time state-transition model (DT-STM) with a discrete event simulation (DES) model. MethodsThe original DT-STM was replicated and a DES model developed using AnyLogic software. Models were populated using individual patient data of a phase III study in metastatic colorectal cancer patients, and compared based on their evidence structure, internal validity, and cost-effectiveness outcomes. The DT-STM used time-dependent transition probabilities, whereas the DES model was populated using parametric distributions. ResultsThe estimated time-dependent transition probabilities for the DT-STM were irregular and more sensitive to single events due to the required small cycle length and limited number of event observations, whereas parametric distributions resulted in smooth time-to-event curves for the DES model. Although the DT-STM and DES model both yielded similar time-to-event curves, the DES model represented the trial data more accurately in terms of mean health-state durations. The incremental cost-effectiveness ratio (ICER) was €172,443 and €168,383 per Quality Adjusted Life Year gained for the DT-STM and DES model, respectively. ConclusionDES represents time-to-event data from clinical trials more naturally and accurately than DT-STM when few events are observed per time cycle. As a consequence, DES is expected to yield a more accurate ICER.

Cost-effectiveness of lumbar fenestration surgery in the Japanese universal health insurance system

BackgroundHealthcare costs are a global concern, and cost-effectiveness analyses of interventions have become important. However, data regarding cost-effectiveness are limited to a few medical fields. The purpose of our study was to examine the Japanese universal health insurance system cost per quality-adjusted life year (QALY) for lumbar fenestration surgery. MethodsForty-eight patients who underwent fenestration for lumbar degenerative spinal canal stenosis between July 2013 and September 2015 were included. Effectiveness was evaluated by measuring the EuroQOL 5-dimension (EQ-5D), Short-Form 8 physical component summary (PCS), and visual analog scale (VAS). Cost was analyzed from the perspective of the public healthcare payer. Effectiveness and cost were measured 1 year after surgery. QALYs were calculated by multiplying the utility value (EQ-5D) and life years. Only direct costs based on actual reimbursements were included. Cost per QALY with a 5-year time horizon with a 2% discount rate was estimated. Sensitivity analysis was performed by varying the time horizon (2 years or 10 years). ResultsMean total cost 1 year after fenestration surgery was 1,254,300 yen (standard deviation [SD], 430,000 yen; median, 1,172,300 yen). Operative cost was 406,800 yen (SD, 251,500 yen; median, 363,000 yen). Mean gained score was 0.21 for EQ-5D (SD, 0.18; median, 0.24), 11 for PCS (SD, 10; median, 12), and −43 for VAS (SD, 34; median, −38). Cost per QALY was 1,268,600 yen. Sensitivity analysis demonstrated that cost per QALY with a 10-year time horizon was 679,300 yen and that with a 2-year time horizon was 3,004,600 yen. ConclusionsCost per QALY of lumbar fenestration with a 5-year time horizon was 1,268,600 yen (11,532 US dollar), which was below the widely accepted benchmark (cost per QALY <5,000,000–6,500,000 yen (50,000 US dollars)). Fenestration is a cost-effective intervention.

39. Cost-effectiveness analysis for treatment of cirrhotic hepatitis C patients with combination of sofosbuvir and velpatasvir versus current treatment regimens in Punjab state

39. Cost-effectiveness analysis for treatment of cirrhotic hepatitis C patients with combination of sofosbuvir and velpatasvir versus current treatment regimens in Punjab state

Psychometric evaluation of the Chinese version of the Child Health Utility 9D (CHU9D-CHN): a school-based studyin China.

The Child Health Utility 9D (CHU9D), a new generic preference-based health-related quality of life (HRQoL) instrument, was developed specifically for the application in cost-effectiveness analyses of treatments and interventions for children and adolescents. The main objective of this study was to examine the psychometric property of the Chinese version of CHU9D (CHU9D-CHN) in a large school-based sample in China. Data were collected using a multi-stage sampling method from third-to-ninth-grade students in Shaanxi Province, China. Participants self-completed a hard-copy questionnaire including the CHU9D-CHN instrument, the Pediatric Quality of Life Inventory™ 4.0 Generic Core Scales (PedsQL), information on socio-demographic characteristics and self-reported health status. The psychometric properties of the CHU9D-CHN, including the internal consistency, 2-week test-retest reliability, convergent and known-groups validity were studied. A total of 1912 students participated in the survey. The CHU9D-CHN internal consistency and test-retest reliability were good to excellent with a Cronbach's alpha of 0.77 and an intra-class correlation coefficient of 0.65, respectively. The CHU9D utility scores moderately correlated with the PedsQL total scores (r = .57, P < .001), demonstrating good convergent validity. Difference of the CHU9D utility scores among the different participants with levels of self-reported general health, health services utilisation and left-behind status demonstrated good construct validity. The findings demonstrated adequate psychometric performance for the CHU9D-CHN. The CHU9D-CHN was a satisfactory, reliable and valid instrument to measure and value HRQoL for children and adolescents in China.

Cost-effectiveness analysis of treatment with non-curative or palliative intent for hepatocellular carcinoma in the real-world setting.

Hepatocellular carcinoma (HCC) presentation is heterogeneous necessitating a variety of therapeutic interventions with varying efficacies and associated prognoses. Poor prognostic patients often undergo non-curative palliative interventions including transarterial chemoembolization (TACE), sorafenib, chemotherapy, or purely supportive care. The decision to pursue one of many palliative interventions for HCC is complex and an economic evaluation comparing these interventions has not been done. This study evaluates the cost-effectiveness of non-curative palliative treatment strategies such as TACE alone or TACE+sorafenib, sorafenib alone, and non-sorafenib chemotherapy compared with no treatment or best supportive care (BSC) among patients diagnosed with HCC between 2007 and 2010 in a Canadian setting. Using person-level data, we estimated effectiveness in life years and quality-adjusted life years (QALYs) along with total health care costs (2013 US dollars) from the health care payer’s perspective (3% annual discount). A net benefit regression approach accounting for baseline covariates with propensity score adjustment was used to calculate incremental net benefit to generate incremental cost-effectiveness ratio (ICER) and uncertainty measures. Among 1,172 identified patients diagnosed with HCC, 4.5%, 7.9%, and 5.6%, received TACE alone or TACE+sorafenib, sorafenib, and non-sorafenib chemotherapy clone, respectively. Compared with no treatment or BSC (81.9%), ICER estimates for TACE alone or TACE+sorafenib was $6,665/QALY (additional QALY: 0.47, additional cost: $3,120; 95% CI: -$18,800-$34,500/QALY). The cost-effectiveness acceptability curve demonstrated that if the relevant threshold was $50,000/QALY, TACE alone or TACE+sorafenib, non-sorafenib chemotherapy, and sorafenib alone, would have a cost-effectiveness probability of 99.7%, 46.6%, and 5.5%, respectively. Covariates associated with the incremental net benefit of treatments are age, sex, comorbidity, and cancer stage. Findings suggest that TACE with or without sorafenib is currently the most cost-effective active non-curative palliative treatment approach to HCC. Further research into new combination treatment strategies that afford the best tumor response is needed.

Recomendações multidisciplinares portuguesas sobre o pedido de DXA e indicação de tratamento de prevenção das fraturas de fragilidade

Objective: To establish Portuguese recommendations regarding the indications for ordering bone densitometry measurement to (DXA) and for starting treatment to prevent osteoporotic fractures. Methods: A multidisciplinary panel, representing a wide range of medical specialties and patient associations related to osteoporosis, as well as national experts in this field and in health economics, met to develop recommendations based on available evidence and expert consensus. Recently obtained data on the epidemiologic, economic and quality-of-life aspects of osteoporotic fractures in Portugal were used to support decisions. Results: Ten recommendations were developed covering the issues of indications for ordering DXA measurements and whom to treat with anti-fracture medications. Thresholds for assessment and intervention are based on the cost-effectiveness analysis of interventions at different thresholds of a ten-year probability of osteoporotic fractures, calculated with the Portuguese version of FRAX® (FRAX®Port), and taking into account Portuguese epidemiologic and economic data. Limitations of FRAX® are highlighted and guidance for appropriate adjustment is provided, when possible. Conclusions: Cost-effectiveness thresholds for DXA examination and drug intervention aiming at fragility fracture prevention are provided for the Portuguese population. These are practical, based on national epidemiological and economic data, evidence-based, and supported by a multidisciplinary panel of experts and scientific societies. Implementation of these recommendations has the possibility to assure the most effective use of health resources in the prevention of osteoporotic fractures in Portugal.

Integration of Patients' Decision-Making in Cost-Effectiveness Analysis of Diabetes Interventions: A Simulation Study Using System Dynamics Modeling

Markov-based and discrete event simulation models are widely used in health economic analysis to estimate the long term effects of treatment on clinical and economic outcomes. However, none of them simultaneously integrate patients’ economic resources and their medical decision making in the simulation. This study applied systems dynamics modeling into cost-effectiveness analysis (CEA) of glycemic control treatment. It explored the nonlinear dynamic impacts of patients’ behavior on the cost effectiveness of different interventions to better understand the complexity of diabetes patients’ medical choices in the real world and their impacts on the clinical and economic consequences.Our results showed that the cost-effectiveness ratio varied with patients’ out-of-pocket payment threshold for health care. It suggests that patients’ decision making of cost-related non-adherence with medication should be considered while conducting CEA for treatment of complex chronic condition like diabetes.This study offers insight into the current dynamics of health care costs and outcomes. While providing a framework for CEA with integration of diabetes treatment adherence, it opens up ways of evaluate the complexity of health care for chronic condition in the real world.

Response to “Putting the cart before the horse: A cost effectiveness analysis of treatments for stuttering in young children requires evidence that the treatments analyzed were effective”

The letter by Bergporsdottir and Ingham critically reviews the design and results of the RESTART-study, our randomized trial into the effectiveness and cost-effectiveness of two treatments for early stuttering in which nearly 200 children participated. The authors put three arguments forward to allege that we have not shown that the Lidcombe Program (LP) or the RESTART-DCM based treatment is more (cost-)effective than no treatment. They conclude that, based on their arguments, our results ‘have no value for clinical management’. We would like to respond to what we perceive to be an inappropriate and incorrect conclusion. In fact, the RESTART-study was designed to meet the increased clinical demand for more studies in the field of developmental stuttering in order to enhance clinical care and strengthen the position of SLPs in reimbursement decision making. We will review the three arguments of Bergporsdottir and Ingham below.

Putting the cart before the horse: A cost effectiveness analysis of treatments for stuttering in young children requires evidence that the treatments analyzed were effective

PurposeTo investigate the validity of findings from a recent study reported in this journal by de Sonneville-Koedoot, Bouwmans, Franken, and Stolk (2015) on the cost effectiveness of two programs for treating young children who stutter. MethodsThe de Sonneville-Koedoot, Bouwmans et al. study was based directly on the results obtained in an earlier study, known as the RESTART-study, which compared the outcomes from the Lidcombe Program and a Demands and Capacities Model program. The methodology of the RESTART-study was critically reviewed. ResultsThe absence of an untreated control group in the RESTART-study makes the results of that study uninterpretable. An inappropriate comparison made with the Yairi and Ambrose (2005) Illinois Study findings failed to resolve the control group problem. Furthermore, the criteria used to classify treated children as “non-stuttering” was also shown to be confounded. The foregoing problems meant that neither treatment program could be shown to be more effective than no treatment. Conclusionde Sonneville-Koedoot, Bouwmans et al’s findings, which compared the cost effectiveness of two treatments for young children who stutter, have no value for clinical management because the treatments investigated were not shown to be more effective than no treatment.