Search

Nitric oxide is a gas produced in the airways of asthmatic subjects and related to T2 inflammation. It can be measured as fractional nitric oxide (FeNO) in the exhaled air and used as a non-invasive, easy to evaluate, rapid marker. It is now widely used in many settings to determine airway inflammation. The aim of this narrative review is to report relationship between FeNO and the physiopathologic characteristics of asthmatic patients. Factors affecting FeNO levels have also been analysed as well as the impact of corticosteroid, target therapies and rehabilitation programs. Considering the availability of the test, spreading this method-ology to low income countries has also been considered as a possibility for evaluating airway inflammation and monitoring adherence to inhaled corticosteroid therapy. PubMed data search has been performed restricted to English language papers. Research was limited to studies in adults unless studies in children were the only ones reported for a particular issue. This revision could be useful to summarize the role of FeNO in relation to asthma charac-teristics and help in the use of FeNO in different clinical settings particularly in low income countries. (c) 2020 Sociedade Portuguesa de Pneumologia. Published by Elsevier Espana, S.L.U. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

Background A recently conducted matching-adjusted indirect comparison demonstrated that bosutinib improved progression-free survival, and delayed progression to advanced disease compared with dasatinib and nilotinib in patients with second line (2L) chronic-phase chronic myeloid leukemia (CP-CML). However, the long-term clinical and economic impact of using bosutinib versus dasatinib and nilotinib has not been evaluated. The objective was to determine the cost-effectiveness of bosutinib compared with dasatinib and bosutinib compared with nilotinib from a US payer perspective. Methods A cost-effectiveness model was developed using partition survival methods and three health states: progression-free, progression, and death. Trial data (individual patient-level and aggregate-level data) informed the progression-free and overall survival estimates. Costs included drugs and medical resource use. Utility values were obtained from literature. Sensitivity analyses (SAs) included one-way and probabilistic sensitivity analyses (PSAs). Results Comparing bosutinib versus dasatinib resulted in a gain of 0.4 discounted life years, 1.5 quality-adjusted life years (QALYs), and incremental costs of $28,459 (values in 2020 US dollars), for an incremental cost-effectiveness ratio (ICER) of $19,811/QALY gained. Comparing bosutinib versus nilotinib resulted in a gain of 0.8 discounted life-years, 1.8 QALYs, and incremental costs of $76,563, for an ICER of $41,932/QALY gained. Drug costs and extrapolation distribution type were the main drivers of the model in the one-way SAs. In the PSAs, bosutinib had >90% and >80% probabilities of being cost-effective at a willingness-to-pay threshold of $100,000/QALY versus dasatinib and nilotinib, respectively. Conclusions Our results suggest that compared with dasatinib and nilotinib, bosutinib may represent good value for money for treating 2L CP-CML patients. Supplementary Information The online version contains supplementary material available at 10.1007/s40258-021-00666-0.

Background Health Technology Assessment (HTA), which can support public drug reimbursement decisions will play a core function in the planned national Pharmacare program in Canada. To address existing barriers to the use of HTA, these must be ranked in order of priority. The goal of this study was to access the relative importance of known facilitators and barriers to the use of HTA in the context of the Canadian health care system, with attention to differences between regions and stakeholder groups. Methods We used the best-worst scaling object case approach to elicit a quantitative ranking of a list of 20 facilitators and 22 barriers. A sample of 68 Canadian HTA stakeholders, including members of expert committees, decision/policymakers, researchers/academics, and others participated in the study. Their task was to identify the most important and the least important item in 12 sub-sets of five facilitators and 14 sub-sets of five barriers. Findings Relative Importance Scores derived via hierarchical Bayes analysis revealed relations, engagement, and contact between stakeholders as most important on both the barrier and facilitator sides. Other top-ranked facilitators included the availably of credible and relevant research. Other top-ranked barriers included inconsistencies in the evidence and limited generalizability. The availability of HTA guidelines did not rank highly on either side. The main limitation of the study was the challenge with reaching the relevant respondents; this was mitigated by involving the national HTA agency in the research. Conclusion Canadian stakeholders consider the relationships within the HTA network among the most important. Policies should focus on strengthening these relationships. Future research should focus on the connectivity and distribution of knowledge and power within the HTA network.

Food policies for the prevention and management of diet-related non-communicable diseases (NCDs) have been increasingly relying on microsimulation models (MSMs) to assess effectiveness. Given the increased uptake of MSMs, this review aims to provide an overview of the characteristics of MSMs that link diets with NCDs. A comprehensive review was conducted in PubMed and Web of Knowledge. Inclusion criteria were: (i) findings from a MSM, (ii) diets, foods or nutrients as main exposure of interest, (iii) NCDs, such as overweight/obesity, type 2 diabetes, coronary heart disease, stroke or cancer as disease outcome for impact assessment. This review included information from 33 studies using MSM in analyzing diet and diverse food policies on NCDs. Hereby, most models employed stochastic, discrete-time, dynamic microsimulation techniques to calculate anticipated (cost-)effectiveness of strategies based on food pricing, food reformulation or dietary (lifestyle) interventions. Currently available models differ in the methodology used for quantifying the effect of the dietary changes on disease, and in the method for modelling disease incidence and mortality. However, all studies provided evidence that the models were sufficiently capturing the close-to-reality situation by justifying their choice of model parameters and validating externally their modelled disease incidence and mortality with observed or predicted event data. With the increasing use of various MSMs, between-model comparisons, facilitated by open access models and good reporting practices, would be important for judging model's accuracy, leading to continued improvement in the methodologies for developing and applying MSMs, and subsequently a better understanding of the results by policymakers. A statement of significance Given the advancement in the application of microsimulation modelling in evaluating food policies and measuring diet-related disease burdens, the present scoping review serves as an exercise to inform future modelling, hereby highlighting the need for transparency in model development, application and dissemination to advance and safeguard accuracy and relevance in modelling efforts.

Summary Background Actinic keratosis (AK) is a common premalignant skin condition that might have the ability to progress into squamous cell carcinoma. Due to the high incidence of AK, treatment of this disease significantly impacts healthcare spending. Objectives To determine which commonly prescribed field‐directed treatment is the most cost‐effective, when comparing 5‐fluorouracil (5‐FU) 5%, imiquimod (IMQ) 5%, ingenol mebutate (IM) 0·015% and methyl aminolaevulinate photodynamic therapy (MAL‐PDT) for AK in the head and neck region. Methods We performed an economic evaluation from a healthcare perspective. Data were collected alongside a single‐blinded, prospective, multicentre randomized controlled trial with 624 participants in the Netherlands. The outcome measure was expressed as the incremental cost‐effectiveness ratio, which is the incremental costs per additional patient with ≥ 75% lesion reduction compared with baseline. This trial was registered at ClinicalTrials.gov, number NCT02281682. Results The trial showed that 5‐FU was the most effective field treatment for AK in the head and neck region. Twelve months post‐treatment, the total mean costs for 5‐FU were significantly lower (€433) than the €728, €775 and €1621 for IMQ, IM and MAL‐PDT, respectively. The results showed that 5‐FU was a dominant cost‐effective treatment (more effective and less expensive) compared with the other treatments, 12 months post‐treatment. Conclusions Based on these results, we consider 5‐FU 5% cream as the first‐choice treatment option for multiple AKs in the head and neck area. What's already known about this topic? Due to the increasing incidence of actinic keratosis (AK), the recommended treatment results in a considerable socioeconomic burden for (dermatological) healthcare.Although cost‐effectiveness modelling studies have been performed in which different treatments for AK were compared, a prospective clinical trial comparing four frequently prescribed treatments on effectiveness and resource consumption within a time horizon of 12 months has never been conducted. What does this study add? This is the first study examining the cost‐effectiveness of 5‐fluorouracil 5% cream, imiquimod 5% cream, ingenol mebutate 0·015% gel and methyl aminolaevulinate photodynamic therapy, with data collected in a randomized controlled trial over a time horizon of 12 months.We found that 5‐fluorouracil was a dominant cost‐effective treatment (more effective and less costly), based on data from the Netherlands. Linked Comment: Steeb et al. Br J Dermatol 2020; 183:612., Linked Comment: Steeb et al. Br J Dermatol 2020; 183:612. Plain language summary available online

Background New pharmacological therapies for the treatment of cardiovascular disease (CVD) have emerged in recent years. The high rates of CVD and the need for long-term treatment to decrease risk factors makes cost-effectiveness crucial for their successful long-term implementation. Objective This study assessed cost-effectiveness studies of novel pharmacological treatments (ezetimibe, proprotein convertase subtilisin/kexin type 9 [PCSK9] inhibitors, omega-3 polyunsaturated fatty acids [n-3 PUFAs], and the cardiovascular polypill) compared with standard care for the secondary prevention of CVD. Methods We searched seven databases and the reference list of selected literature reviews for eligible cost-effective analyses (CEA) published between January 2009 and January 2020 that evaluated the above novel treatments versus standard care. Two independent reviewers performed the screening and evaluation in accordance with the Consolidated Health Economic Evaluation Reporting Standards statement. Cost results were adapted to 2018 US dollars (US$) to facilitate comparisons between studies. Consideration of cost-effectiveness was based on the original study criteria. Results Thirty-two studies were included in this review, most of them adopting a healthcare perspective. Studies evaluating ezetimibe, PCSK9 inhibitors and n-3 PUFAs assessed their addition to standard care compared with standard care alone, while studies analysing the polypill evaluated the replacement of multiple monotherapies for a fixed-dose combination. Ten studies reported on ezetimibe, fifteen evaluated PCSK9 inhibitors, five focused on n-3 PUFAs and seven on the polypill. From a healthcare perspective, ezetimibe was cost effective in 62.5% of the studies (incremental cost-effectiveness ratios [ICERs] ranged from US$27,195 to US$204,140), n-3 PUFAs in 60% (ICERs from US$57,128 to US$139,082) and the cardiovascular polypill in 100% (ICERs from dominant to US$30,731) compared with standard care. Conversely, only 10% of the studies considered PCSK9 inhibitors cost effective compared with standard care from a healthcare perspective (ICERs ranged from US$231,119 to US$1,223,831). Additionally, ezetimibe was cost effective in 50% of the studies, PCSK9 inhibitors in 33% and the polypill in 50% of the studies adopting a societal perspective. The key model-related parameters predicting cost-effectiveness included drug cost, time horizon, and the baseline risk of cardiovascular events. Conclusions Based on current pricing and willingness-to-pay thresholds, most CEA studies considered ezetimibe, n-3 PUFAs and the polypill to be cost effective compared with standard care but not PCSK9 inhibitors for secondary prevention of CVD.

Neily Zakiyah,1,2 Rano K Sinuraya,1– 3 Arif SW Kusuma,2,4 Auliya A Suwantika,1,2,5 Keri Lestari1,2 1Department of Pharmacology and Clinical Pharmacy, Faculty of Pharmacy, Universitas Padjadjaran, Bandung, Indonesia; 2Center of Excellence in Higher Education for Pharmaceutical Care Innovation, Universitas Padjadjaran, Bandung, Indonesia; 3Unit of Global Health, Department of Health Sciences, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands; 4Department of Biological Pharmacy, Faculty of Pharmacy, Universitas Padjadjaran, Bandung, Indonesia; 5Center for Health Technology Assessment, Universitas Padjadjaran, Bandung, 40132, IndonesiaCorrespondence: Neily Zakiyah Tel/Fax +62 22-7796200Email neily.zakiyah@unpad.ac.idObjective: Sacubitril/valsartan is a relatively new medication that is more effective than the usual enalapril for heart failure patients with reduced ejection fraction. Therefore, this study aims to determine the cost-effectiveness of sacubitril/valsartan compared to enalapril in Indonesia’s healthcare system.Methods: In this study, a Markov decision-analytic model was developed to estimate the total cost, health outcomes, and cost-effectiveness of sacubitril/valsartan compared to enalapril from Indonesia’s healthcare perspective. The input parameters for the cost-effectiveness were predominantly from the PARADIGM-HF trial. Subsequently, the country-specific data were synthesized for medication and hospitalization costs, cardiovascular and non-cardiovascular death, as well as re-hospitalization rate. The incremental cost-effectiveness ratio (ICER) per quality-adjusted life years (QALYs) gained was estimated to determine the cost-effectiveness. Deterministic and probabilistic sensitivity analyses were conducted to assess the impact of parameter uncertainty.Results and Discussion: In the base case, sacubitril/valsartan was more costly and effective than enalapril with a total cost of IDR 91,783,325,865 (USD 6,487,522) vs IDR 68,101,971,241 (USD 4,813,653) and a total QALYs of 19,680 vs 18,795, resulting in an ICER of IDR 26,742,098 (USD 1890). Based on the willingness to pay threshold GDP per capita in Indonesia, it can be considered cost-effective. The most influential drivers of cost-effectiveness in deterministic sensitivity analysis were risk of mortality outside hospitalization, hospital admission rate, and cost of sacubitril/valsartan. The vast majority of simulation results from probabilistic analysis suggested that sacubitril/valsartan was likely resulted in higher cost and improved QALYs compared with enalapril, indicating the robustness of the model.Conclusion: Based on the current price in Indonesia, sacubitril/valsartan can be considered a cost-effective option, although this depends heavily on the willingness to pay threshold. Further studies that incorporate real-world evidence with sacubitril/valsartan are needed to inform the decision-making process.Keywords: cost-effectiveness, heart failure, Markov model, sacubitril/valsartan

Objectives There is emerging evidence supporting sequential therapy with an osteoanabolic followed by an antiresorptive in patients at high-risk of fragility fractures. This study assessed the cost-effectiveness of sequential treatment with abaloparatide (ABL) followed by alendronate (ALN) [(ABL/ALN)] compared with teriparatide (TPTD) followed by ALN (TPTD/ALN). Methods A previously validated Markov microsimulation model was adapted to estimate the cost-effectiveness of sequential ABL/ALN compared with sequential TPTD/ALN and no treatment with a lifetime horizon from the US payer perspective. Patients were assumed to receive ABL or TPTD for 18 months followed by 5 years of ALN in line with clinical recommendations. The effects of ABL on fracture risk were derived from the ACTIVExtend trial. The effects of TPTD were assumed to be maintained during subsequent ALN treatment, consistent with ACTIVExtend findings for ABL. Evaluation was completed for patients, aged 50–80 years with a BMD T-score ≤ −3.5 or with a T-score between −2.5 and −3.5 and a history of ≥ one osteoporotic fracture. Results In all simulated populations, sequential ABL/ALN therapy was dominant (lower costs, higher QALYs) compared with sequential TPTD/ALN therapy, resulting from the improved efficacy and lower drug price of ABL. Probabilistic sensitivity analyses suggested that ABL/ALN was dominant in at least 99% of the simulations. Compared to no treatment, the cost per QALY gained of ABL/ALN was always below $130,000. Conclusions Sequential ABL/ALN therapy is a cost-effective (dominant) strategy compared with sequential TPTD/ALN therapy for the treatment of US women at increased risk of fractures.

BACKGROUND: Health economic evaluations are often required before implementing a vaccination programme. Such evaluations rarely consider the historical context of a vaccination programme. We review the financial history of vaccination programmes in the Netherlands, and compare these to demographic and macroeconomic developments as well as avoided mortality burden.METHODS: Previously uncatalogued historical expenditures on the Dutch National Immunisation Programme (NIP) and influenza vaccination were obtained from official reports. Costs were adjusted for inflation using Consumer Price Indices and expressed in Euro of 2016. Estimates on mortality burden averted were obtained from previous research and used to calculate the ratio of expenses to averted mortality burden for vaccinations against diphtheria, tetanus, pertussis, polio, measles, mumps and rubella for birth cohorts 1953-1992.RESULTS: Developments towards a uniform government funded NIP started early 1950s with vaccinations against diphtheria, pertussis and tetanus, culminating in its official launch in 1957 together with polio vaccinations. Since the 1980s, expenditure increased nearly five-fold mostly due to the addition of new vaccines, while spending on already implemented vaccinations tended to decline. Overall, expenditure increased from € 5 million in 1957 to € 93 million in 2014. Relative to total healthcare expenditure, the NIP contributed little, ranging between 0.05% and 0.14%. Spending on influenza vaccination increased from € 37 million in 1996 to € 52 million in 2014, while relative to total healthcare expenditure it decreased from 0.069% to 0.055%. In 2014, 0.15% of healthcare expenditure and € 533 per birth was spent on vaccination programmes. Overall, for birth cohorts 1953-1992, € 5.4 thousand (95% confidence interval: 4.0-7.3) was expended per year-of-life-lost averted.CONCLUSION: The actual costs per year-of-life gained are more favorable than estimated here since averted medical costs were not included. Although expenditure on vaccination programmes increased substantially, the contribution to overall healthcare expenditure remained small.

Objectives: To determine how the value of new medicines is defined from the perspective of different stakeholders in nine European countries. Methods: We carried out an explorative survey by conducting in-depth qualitative interviews with a range of experts with health technology assessment, clinical provision, health economics, payer, academic and industry experience. Experts were asked to distribute 100 points over a predefined list of attributes related to value. In total, 30 interviews were conducted with seven academics, five clinicians, nine economists, five payers and four providers. Each of these categories was represented in responses from France, Germany, the Netherlands and the UK. Results: Comments on the interview guide were all positive. There was substantial variation in definitions of value provided by respondents. Most respondents indicated that delivering benefits to the patient is a prerequisite for a healthcare product to be of value, but only half included costs or resource use in their definition. Quantitative responses showed that effectiveness and efficacy were considered the most important attributes of value, followed by safety and side effects. Lower scores were given to other attributes, such as dignity and individualism, and invasiveness of the treatment. Resources and costs associated with complications and readmissions were considered important by academics and economists, but not clinicians. Conclusions: The interview findings indicate that the value of a drug is subjective in being perceived differently by different stakeholders, and for different treatment types. Future research will require evaluation of the views of a larger number of stakeholders in more and different countries to confirm these findings. © European Association of Hospital Pharmacists (unless otherwise stated in the text of the article) 2017. All rights reserved. No commercial use is permitted unless otherwise expressly granted.

Summary The use of gastro-resistant risedronate, a convenient dosing regimen for oral bisphosphonate therapy, seems a cost-effective strategy compared with weekly alendronate, generic risedronate, and no treatment for the treatment of postmenopausal women with osteoporosis in France. Introduction Gastro-resistant (GR) risedronate tablets are associated with improved persistence compared to common oral bisphosphonates but are slightly more expensive. This study assessed its cost-effectiveness compared to weekly alendronate and generic risedronate for the treatment of postmenopausal women with osteoporosis in France. Methods A previously validated Markov microsimulation model was used to estimate the lifetime costs (expressed in €2017) per quality-adjusted life-years (QALY) of GR risedronate compared with weekly alendronate, generic risedronate, and no treatment. Pooled efficacy data for bisphosphonates derived from a previous meta-analysis were used for all treatment options, and persistence data (up to 3 years) were obtained from a large Australian longitudinal study. Evaluation was done for high-risk women 60–80 years of age, with a bone mineral density (BMD) T-score ≤ − 2.5 and/or prevalent vertebral fractures. Results In all of the simulated populations, GR risedronate was cost-effective compared to alendronate, generic risedronate, and no treatment at a threshold of €60,000 per QALY gained. In women with a BMD T-score ≤ − 2.5 and prevalent vertebral fractures, the cost per QALY gained of GR risedronate compared to alendronate, generic risedronate, and no treatment falls below €20,000 per QALY gained. In women aged 75 years and older, GR risedronate was even shown to be dominant (more QALYs, less costs) compared to alendronate, generic risedronate, and no treatment. Conclusion This study provides the first economic results about GR risedronate, suggesting that it represents a cost-effective strategy compared with weekly alendronate and generic risedronate for the treatment of postmenopausal women with osteoporosis in France.

Objective To study cost-effectiveness of an interleukin (IL)-17A inhibitor secukinumab, with other biologics and apremilast in patients with Psoriatic arthritis (PsA) from payer perspective in Finland. Methods In this semi-Markov model, subcutaneous (SC) secukinumab was compared with SC treatments etanercept and its biosimilar, certolizumab pegol, adalimumab and its biosimilar, golimumab, ustekinumab, intravenous (IV) treatment infliximab, as well as oral non-biologic apremilast. Patients without prior exposure (naïve) to biologics and without moderate to severe psoriasis were considered for secukinumab 150 mg group. Secukinumab 300 mg group included naïve patients with moderate to severe psoriasis and all patients with prior biologic exposure. The PsA Response Criteria (PsARC) at 12-week was primary criteria for treatment response. Other clinical as well as cost related model inputs were derived from relevant clinical trials as well as Finnish publications. The key model outcomes were quality-adjusted life years and incremental cost-effectiveness ratio. An annual 3% discount rate was applied to all future costs and benefits. Model input variations were assessed through sensitivity analyses and alternative scenario analyses. Results For a lifetime horizon (60 years), secukinumab 150 mg dominated all branded SC biologics and apremilast with highest QALY of 8.01 and lowest lifetime cost of €187,776, while it was cost-effective against IV infliximab among biologic-naïve patients without moderate to severe psoriasis. Secukinumab 300 mg was cost-effective against all branded SC biologics and apremilast and dominated IV infliximab among biologic-naïve patients with moderate to severe psoriasis, while it was cost-effective in biologic experienced patients. With the one-way sensitivity analysis, PsARC response, drug acquisition cost, and health assessment questionnaire score were the most important parameters affecting the outcomes. Across all treatment groups, patients on secukinumab were most likely to achieve highest net monetary benefit than other competitors in probabilistic sensitivity analysis. With alternative scenario analysis, results largely remained unchanged. Conclusions Secukinumab is a cost-effective treatment for PsA patients from a Finnish payer’s perspective. Electronic supplementary material The online version of this article (10.1186/s12962-018-0162-3) contains supplementary material, which is available to authorized users.

Neglected tropical diseases (NTDs) remain a significant cause of morbidity and mortality in many low-income and middle-income countries. Several NTDs, namely lymphatic filariasis, onchocerciasis, schistosomiasis, soil-transmitted helminthiases (STH) and trachoma, are predominantly controlled by preventive chemotherapy (or mass drug administration), following recommendations set by the WHO. Over one billion people are now treated for NTDs with this strategy per year. However, further investment and increased domestic healthcare spending are urgently needed to continue these programmes. Consequently, it is vital that the cost-effectiveness of preventive chemotherapy is understood. We analyse the current estimates on the cost per disability-adjusted life year (DALY) of the preventive chemotherapy strategies predominantly used for these diseases and identify key evidence gaps that require further research. Overall, the reported estimates show that preventive chemotherapy is generally cost-effective, supporting WHO recommendations. More specifically, the cost per DALY averted estimates relating to community-wide preventive chemotherapy for lymphatic filariasis and onchocerciasis were particularly favourable when compared with other public health interventions. Cost per DALY averted estimates of school-based preventive chemotherapy for schistosomiasis and STH were also generally favourable but more variable. Notably, the broader socioeconomic benefits are likely not being fully captured by the DALYs averted metric. No estimates of cost per DALY averted relating to community-wide mass antibiotic treatment for trachoma were found, highlighting the need for further research. These findings are important for informing global health policy and support the need for continuing NTD control and elimination efforts.

Background: The Dietary Approach to Stop Hypertension (DASH) and potassium supplementation have been shown to reduce the risk of death with a functioning graft (DWFG) and renal graft failure in renal transplant recipients (RTR). Unfortunately, a key problem for patients is the adherence to these diets. The aim of this study is to evaluate the cost-effectiveness and budget impact of higher adherence to either the DASH or potassium supplementation. Methods: A Markov model was used to simulate the life course of 1000 RTR in the Netherlands. A societal perspective with a lifetime time horizon was used. The potential effect of improvement of dietary adherence was modelled in different scenarios. The primary outcomes are the incremental cost-effectiveness ratio (ICER) and the budget impact. Results: In the base case, improved adherence to the DASH diet saved 27,934,786 and gained 1880 quality-adjusted life years (QALYs). Improved adherence to potassium supplementation saved €1,217,803 and gained 2901 QALYs. Both resulted in dominant ICERs. The budget impact over a five-year period for the entire Dutch RTR population was €8,144,693. Conclusion: Improving dietary adherence in RTR is likely to be cost-saving and highly likely to be cost-effective compared to the current standard of care in the Netherlands.

Background Genetic testing has potential roles in identifying whether an individual would have risk of adverse drug reactions (ADRs) from a particular medicine. Robust cost-effectiveness results on genetic testing would be useful for clinical practice and policy decision-making on allocating resources effectively. This study aimed to update a systematic review on economic evaluations of pharmacogenetic testing to prevent ADRs and critically appraise the quality of reporting and sources of evidence for model input parameters. Methods We searched studies through Medline via PubMed, Scopus and CRD’s NHS Economic Evaluation up to October 2019. Studies investigating polymorphism-based pharmacogenetic testing, which guided drug therapies to prevent ADRs, using economic evaluation methods were included. Two reviewers independently performed data extraction and assessed the quality of reporting using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) guidelines and the quality of data sources using the hierarchy of evidence developed by Cooper et al. Results Fifty-nine economic evaluations of pharmacogenetic testing to avoid drug-induced ADRs were found between 2002 and 2018. Cost-utility and cost-effectiveness analyses were the most common methods of economic evaluation of pharmacogenetic testing. Most studies complied with the CHEERS checklist, except for single study-based economic evaluations which did not report uncertainty analysis (78%). There was a lack of high-quality evidence not only for estimating the clinical effectiveness of pharmacogenetic testing, but also baseline clinical data. About 14% of the studies obtained clinical effectiveness data of testing from a meta-analysis of case-control studies with direct comparison, which was not listed in the hierarchy of evidence used. Conclusions Our review suggested that future single study-based economic evaluations of pharmacogenetic testing should report uncertainty analysis, as this could significantly affect the robustness of economic evaluation results. A specific ranking system for the quality of evidence is needed for the economic evaluation of pharmacogenetic testing of ADRs. Differences in parameters, methods and outcomes across studies, as well as population-level and system-level differences, may lead to the difficulty of comparing cost-effectiveness results across countries.

Cost-effectiveness analysis is widely adopted as an analytical framework to evaluate whether health care interventions represent value for money, and its use in dentistry is increasing. Traditionally, in cost-effectiveness analysis, one assumes that the decision maker&rsquo, s maximum willingness to pay (WTP) for health gain is equivalent to his minimum willingness to accept (WTA) monetary compensation for health loss. It has been documented in the literature that losses are weighted higher than equivalent gains, i.e., that WTA exceeds WTP for the same health condition, resulting in a WTA/WTP ratio greater than 1. There is a knowledge gap of published WTA/WTP ratios for dental interventions in the literature. We therefore conducted a (i) systematic review of published WTA-WTP estimates in dentistry (MEDLINE, Web of Science, Cochrane Library, London, UK) and (ii) a patient-level analysis of WTA/WTP ratios of included studies, and (iii) we demonstrate the impact of a WTA-WTP disparity on cost-effectiveness analysis. Out of 55 eligible studies, two studies were included in our review. The WTA/WTP ratio ranged from 2.58 for discontinuing water fluoridation to 5.12 for mandibular implant overdentures, indicating a higher disparity for implant rehabilitations than for dental public health interventions. A WTA-WTP disparity inflates the cost-effectiveness of dental interventions when there is a substantial risk of both lower costs and health outcomes. We therefore recommend that in these cases the results of cost-effectiveness analyses are reported using different WTA/WTP ratios in a sensitivity analysis.

Contains fulltext : 225314.pdf (Publisher’s version ) (Open Access) OBJECTIVES: This study analysed the cost-effectiveness of two different attachments for the 2-implant overdenture (2IOD) in edentulous mandibles. MATERIALS AND METHODS: When considering alternative treatments, cost-effectiveness analysis is an important factor for stakeholders (patient, clinician, social security, insurance company, etc.). A general practice population (n = 116) was treated between 2003 and 2013 with a mandibular 2IOD with 2 different ball/stud attachment systems, one spherical (Group D) and one cylindrical (Group L). Patient well-being was assessed with OHIP-14-Total (OHIP-14-T), at intake and annually up to 5 years, to calculate the health effect. Initial and maintenance costs of both treatments were inventoried. The cost-effectiveness was compared. Annual discount rates of 4% and 1.5% were applied to future costs and health outcomes, following Dutch guidelines. Prices were adjusted to the year 2003. To offset the uncertainty in relevant input parameters, a sensitivity analysis was performed using bootstrap analysis. Significance was set at p

International audience; Mother-to-child transmission (MTCT) of hepatitis B virus (HBV) is a key component of the hepatitis B burden worldwide. Despite its efficacy to prevent HBV transmission, infant vaccination is not enough to control HBV MTCT. Additional efforts are urgently needed to evaluate and scale-up preventive strategies especially in endemic countries, which are most affected. This review highlights the efficacy and barriers of the currently validated measures for the prevention of HBV MTCT and proposes alternatives adapted to resource-limited settings to eventually achieve HBV elimination worldwide.

Objectives: To assess the cost effectiveness of home telemonitoring (HTM) and nurse telephone support (NTS) compared with usual care (UC) in the management of patients with chronic heart failure, from a third-party payer's perspective. Methods: We developed a Markov model with a 20-year time horizon to analyze the cost effectiveness using the original study (Trans-European Network-Home-Care Management System) and various data sources. A probabilistic sensitivity analysis was performed to assess the decision uncertainty in our model. Results: In the original scenario (which concerned the cost inputs at the time of the original study), HTM and NTS interventions yielded a difference in quality-adjusted life-years (QALYs) gained compared with UC: 2.93 and 3.07, respectively, versus 1.91. An incremental net monetary benefit analysis showed (sic)7,697 and (sic)13,589 in HTM and NTS versus UC at a willingness-to-pay (WTP) threshold of (sic)20,000, and (sic)69,100 and (sic)83,100 at a WTP threshold of (sic)80,000, respectively. The incremental cost-effectiveness ratios were (sic)12,479 for HTM versus UC and (sic)8,270 for NTS versus UC. The current scenario (including telenurse cost inputs in NTS) yielded results that were slightly different from those for the original scenario, when comparing all New York Heart Association (NYHA) classes of severity. NTS dominated HTM, compared with UC, in all NYHA classes except NYHA IV. Conclusions: This modeling study demonstrated that HTM and NTS are viable solutions to support patients with chronic heart failure. NTS is cost-effective in comparison with UC at a WTP of (sic)9000/QALY or higher. Like NTS, HTM improves the survival of patients in all NYHA classes and is cost-effective in comparison with UC at a WTP of (sic)14,000/QALY or higher.

Background Men who have sex with men require special attention for human papillomavirus vaccination given elevated infection risks and the development of, in particular, anal cancer. Objective Our purpose was to review the cost effectiveness of human papillomavirus vaccination for both currently vaccine-eligible and non-eligible individuals, particularly the men-who-have-sex-with-men population, and synthesize the available evidence. Methods We systematically searched for published articles in two main databases (PubMed and EMBASE). Screening and data extraction were performed by two independent reviewers. The risk of bias was assessed using a validated instrument (Bias in Economic Evaluation, ECOBIAS). Methodological aspects, study results, and sensitivity analyses were extracted and synthesized to generate a consistent overview of the cost effectiveness of human papillomavirus vaccination in the men-who-have-sex-with-men population. Results From 770 identified articles, four met the inclusion criteria. Across the studies, human papillomavirus vaccination showed incremental cost-effectiveness ratios ranging from dominant to US$96,146 and US$14,000 to US$18,200 for tertiary prevention and primary prevention, respectively. The incremental cost-effectiveness ratio seemed most sensitive to vaccine efficacy, vaccine costs, and the incidence of anal cancer in the selected target populations. Conclusion This review presents the human papillomavirus vaccine, both as a primary and adjuvant (tertiary) vaccination, as a potentially cost-effective strategy for preventing mainly-but not limited to only-anal cancer in men-who-have-sex-with-men populations.

PURPOSE: In this study, we describe current practices in the management of hemorrhoidal disease in the Netherlands.METHODS: A validated online survey was performed among Dutch surgeons and residents treating hemorrhoidal disease. Contact details were retrieved from the Dutch Association for Surgery resulting in 619 contacts. Only doctors who were treating hemorrhoidal disease regularly were asked to complete the questionnaire. The following items were assessed: initial treatment, recurrence, complications, and follow-up.RESULTS: In total, 133 respondents completed the survey. Ninety percent of the respondents started with rubber band ligation (RBL) as the first treatment in low-grade hemorrhoidal disease. In case of recurrence, 64% of the respondents repeated RBL three times before switching to a more invasive treatment modality. In grade III hemorrhoidal disease, the respondents preferred more invasive techniques: a sutured hemorrhoidopexy was performed in 24%, Doppler-guided hemorrhoidal artery ligation (DG-HAL) in 9%, stapled hemorrhoidopexy in 19%, and the traditional hemorrhoidectomy in 31% of the patients, respectively. The majority of the respondents (39%) reported a mild complication in 5-10% of the patients. The most reported complication was pain. Nearly all the respondents (98%) reported a major complication in less than 5% of the patients. The majority of the patients (57%) were seen in outpatient clinics 6 weeks post-treatment.CONCLUSION: This Dutch survey showed areas of common practice for primary treatment of hemorrhoidal disease. However, it also demonstrated varying practices regarding recurrent hemorrhoidal disease. Practical guidelines are required to support colorectal surgeons in the Netherlands.

INTRODUCTION: Curettage is more effective than expectant management in women with suspected incomplete evacuation after misoprostol treatment for first-trimester miscarriage. The cost-effectiveness of curettage vs. expectant management in this group is unknown.MATERIAL AND METHODS: From June 2012 until July 2014 we conducted a randomized controlled trial and parallel cohort study in the Netherlands, comparing curettage with expectant management in women with an incomplete evacuation of the uterus after misoprostol treatment for first-trimester miscarriage. Successful treatment was defined as a sonographic finding of an empty uterus 6 weeks after study entry, or an uneventful course. Cost-effectiveness and cost-utility analyses were performed. We included costs of healthcare utilization, informal care and lost productivity. Cost-effectiveness planes and cost-effectiveness acceptability curves were estimated using bootstrapping.RESULTS: We included 256 women from 27 hospitals; 95 curettage and 161 expectant management. Treatment was successful in 96% of the women treated with curettage vs. 83% of the women after expectant management (mean difference 13%, 95% confidence interval 5-20). Mean costs were significantly higher in the curettage group (mean difference €1157; 95% C confidence interval €955-1388). The incremental cost-effectiveness ratio for curettage vs. expectant management was €8586 per successfully treated woman. The cost-effectiveness acceptability curve showed that at a willingness-to-pay of €18 200/extra successfully treated women, the probability that curettage is cost-effective is 95%.CONCLUSIONS: Curettage is not cost-effective compared with expectant management in women with an incomplete evacuation of the uterus after misoprostol treatment. This indicates that curettage in this group should be restrained.

Coronary artery disease (CAD) is a major cause of death and disability. Several diagnostic tests, such as myocardial perfusion scintigraphy (MPS), are accurate for the detection of CAD, as well as having prognostic value for the prediction of cardiovascular events. Nevertheless, the diagnostic and prognostic value of these tests should be cost-effective and should lead to improved clinical outcome. We have reviewed the literature on the cost-effectiveness of MPS in different circumstances: (i) the diagnosis and management of CAD; (ii) comparison with exercise electrocardiography (ECG) and other imaging tests; (iii) as gatekeeper to invasive coronary angiography (ICA), (iv) the impact of appropriate use criteria; (v) acute chest pain, and (vi) screening of asymptomatic patients with type-2 diabetes. In total 57 reports were included. Although most non-invasive imaging tests are cost-effective compared with alternatives, the data conflict on which non-invasive strategy is the most cost-effective. Different definitions of cost-effectiveness further confound the subject. Computer simulations of clinical diagnosis and management are influenced by the assumptions made. For instance, diagnostic accuracy is often defined against an anatomical standard that is wrongly assumed to be perfect. Conflicting data arise most commonly from these incorrect or differing assumptions.

Purpose To provide an overview of model characteristics and outcomes of model-based economic evaluations concerning chemotherapy and targeted therapy (TT) for metastatic breast cancer (MBC); to assess the quality of the studies; to analyse the association between model characteristics and study quality and outcomes. Methods PubMED and NHS EED were systematically searched. Inclusion criteria were as follows: English or Dutch language, model-based economic evaluation, chemotherapy or TT as intervention, population diagnosed with MBC, published between 2000 and 2014, reporting life years (LY) or quality-adjusted life-year (QALY) and an incremental cost-effectiveness ratio. General characteristics, model characteristics and outcomes of the studies were extracted. Quality of the studies was assessed through a checklist. Results 24 studies were included, considering 50 comparisons (20 concerning chemotherapy and 30 TT). Seven comparisons were represented in multiple studies. A health state-transition model including the following health states: stable/progression-free disease, progression and death was used in 18 studies. Studies fulfilled on average 14 out of the 26 items of the quality checklist, mostly due to a lack of transparency in reporting. Thirty-one per cent of the incremental net monetary benefit was positive. TT led to higher iQALY gained, and industry-sponsored studies reported more favourable cost-effectiveness outcomes. Conclusions The development of a disease-specific reference model would improve the transparency and quality of model-based cost-effectiveness assessments for MBC treatments. Incremental health benefits increased over time, but were outweighed by the increased treatment costs. Consequently, increased health benefits led to lower value for money. Electronic supplementary material The online version of this article (doi:10.1007/s10549-017-4374-6) contains supplementary material, which is available to authorized users.

ObjectivesThe aim of this study was to conduct a cost-effectiveness analysis comparing conventional removable partial dentures (RPDs) and implant-supported RPDs (ISRPDs) treatment in patients with an edentulous maxilla and a bilateral free-ending situation in the mandible.Material and methodsThirty subjects were included. A new RPD was made and implant support was provided 3months later. Treatment costs (opportunity costs and costs based on tariffs) were calculated. Treatment effect was expressed by means of the Dutch Oral Health Impact Profile questionnaire (OHIP-NL49), a chewing ability test (Mixing Ability Index, MAI) and a short-form health survey measuring perceived general health (SF-36), which was subsequently converted into quality-adjusted-life-years (QALYs). The incremental cost-effectiveness ratio (ICER) was the primary outcome measure of cost-effectiveness, comparing both treatment strategies.ResultsThe mean total opportunity costs were Euro981 (95% CI Euro971-Euro991) for the RPD treatment and Euro2.480 (95% CI Euro2.461-Euro2.500) for the ISRPD treatment. The total costs derived from the national tariff structure were Euro850 for the RPD treatment and Euro2.610 for the ISRPD treatment. The ICER for OHIP-NL49 and MAI using the opportunity costs was Euro80 and Euro786, respectively. When using the tariff structure, corresponding ICERs were Euro94 and Euro921. The effect of supporting an RPD with implants when expressed in QALYs was negligible; hence an ICER was not determined.ConclusionsIt is concluded that depending on the choice of outcome measure and monetary threshold, supporting an RPD with implants is cost-effective when payers are willing to pay more than Euro80 per OHIP point gained. Per MAI point gained, an additional Euro786 has to be invested.

Objective: To assess the effectiveness of curettage versus expectant management in women with an incomplete evacuation of the uterus after misoprostol treatment for first trimester miscarriage.Study design: We conducted a multicenter cohort study alongside a randomized clinical trial (RCT) between June 2012 until July 2014. 27 Dutch hospitals participated. Women with an incomplete evacuation after misoprostol treatment for first trimester miscarriage who declined to participate in the RCT, received treatment of their preference; curettage (n = 65) or expectant management (n = 132). A successful outcome was defined as an empty uterus on sonography at six weeks or uneventful clinical follow-up. We furthermore assessed complication rate and (re)intervention rateResults: Of the 197 women who declined to participate in the RCT, 65 preferred curettage and 132 expectant management. A successful outcome was observed in 62/65 women (95%) in the surgical group versus 112/132 women (85%) in the expectant group (RR 1.1, 95% CI 1.03-1.2), with complication rates of 6.2% versus 2.3%, respectively (RR 2.7, 95% CI 0.6-12).Conclusion: In women with an incomplete evacuation of the uterus after misoprostol treatment, expectant management is an effective and safe option. This finding could restrain the use of curettage in women that have used misoprostol in the treatment of first trimester miscarriage. (C) 2017 Elsevier B.V. All rights reserved.

Background: This study aims to evaluate the long-term cost-effectiveness of add-on cognitive behavior therapy (CBT) for the prevention of psychosis for individuals at ultrahigh risk (UHR) of psychosis.Method: The Dutch Early Detection and Intervention randomized controlled trial was used, comparing routine care (RC; n = 101) with routine care plus CBT for UHR (here called CBTuhr; n = 95). A cost-effectiveness analysis was conducted with treatment response (defined as proportion of averted transitions to psychosis) as an outcome and a cost-utility analysis with quality-adjusted life years (QALYs) gained as a secondary outcome.Results: The proportion of averted transitions to psychosis was significantly higher in the CBTuhr condition (with a risk difference of 0.122; b = 1.324, SEb = 0.017, z = 7.99, P < 0.001). CBTuhr showed an 83% probability of being more effective and less costly than RC by -US$ 5777 (savings) per participant. In addition, over the 4-year follow-up period, cumulative QALY health gains were marginally (but not significantly) higher in CBTuhr than for RC (2.63 vs. 2.46) and the CBTuhr intervention had a 75% probability of being the superior treatment (more QALY gains at lower costs) and a 92% probability of being cost-effective compared with RC at the Dutch threshold value (US$ 24 560; €20 000 per QALY).Conclusions: Add-on preventive CBTuhr had a high likelihood (83%) of resulting in more averted transitions to psychosis and lower costs as compared with RC. In addition, the intervention had a high likelihood (75%) of resulting in more QALY gains and lower costs as compared to RC.

Background & aims The Oligo Element Sore Trial has shown that supplementation with a disease-specific nutritional formula enriched with arginine, zinc, and antioxidants improves pressure ulcer (PU) healing in malnourished patients compared to an isocaloric–isonitrogenous support. However, the use of such a nutritional formula needs to be supported also by a cost-effectiveness evaluation. Methods This economic evaluation – from a local healthcare system perspective – was conducted alongside a multicenter, randomized, controlled trial following a piggy-back approach. The primary efficacy endpoint was the percentage of change in PU area at 8 weeks. The cost analysis focused on: the difference in direct medical costs of local PU care between groups and incremental cost-effectiveness ratio (ICER) of nutritional therapy related to significant study endpoints (percentage of change in PU area and ≥40% reduction in PU area at 8 weeks). Results Although the experimental formula was more expensive (mean difference: 39.4 Euros; P < 0.001), its use resulted in money saving with respect to both non-nutritional PU care activities (difference, −113.7 Euros; P = 0.001) and costs of local PU care (difference, −74.3 Euros; P = 0.013). Therefore, given its efficacy it proved to be a cost-effective intervention. The robustness of these results was confirmed by the sensitivity analyses. Conclusion The use of a disease-specific oral nutritional formula not only results in better healing of PUs, but also reduces the costs of local PU care from a local healthcare system perspective

Background: Children with intestinal failure (IF) depend on parenteral nutrition (PN). The goal in the treatment of IF is to wean children off PN through intestinal rehabilitation (IR). Although the healthcare burden of IF is enormous, to our knowledge there has been no previous cost-effectiveness analysis in pediatric IF including IR.Objective: We sought to determine the cost-effectiveness of IR in terms of costs and life-years.Design: We simulated the treatment of IF in children in a discrete-event model. Data for this model were derived from patient records, the Dutch Registry of Intestinal Failure and Transplantation, the Intestinal Transplant Registry, and the literature. The time horizon of the model was 40 y. Simulated patients were enrolled at a rate of 40 patients/mo for 10 y. Actual costs were calculated for hospital admissions, surgical interventions, endoscopies, PN, and immunosuppressive medication. We evaluated the cost-effectiveness of IR by comparing 1 scenario with IR with 1 scenario without IR. In the scenario with IR, a proportion of patients who represented those with the ability to wean off PN were assigned to IR. In the scenario without IR, all patients progressed to. home PN (HPN). In both scenarios, a proportion of patients receiving HPN were eventually eligible for an intestinal transplantation.Results: IR prolonged survival; the mean number of life-years per patient was 19.4 in the scenario with IR compared with 18.2 in the scenario without IR. Average total costs per patient were 819,292 in the scenario with IR compared with 1,176,830 in the scenario without IR (equivalent to 1,129,230 US$ and 1,622,025 US$, respectively, in January 2014); costs mainly included hospital admissions and PN.Conclusions: On the basis of our simulations, we concluded that IR improved the survival of children with IF and was associated with cost savings. Therefore, we consider IR to be a cost-effective treatment for children with IF.

Introduction: We develop a framework to model disease progression across Alzheimer's disease (AD) and to assess the cost-effectiveness of future disease-modifying therapies (DMTs) for people with mild cognitive impairment (MCI) due to AD.Methods: Using data from the US National Alzheimer's Coordinating Center, we apply survival analysis to estimate transition from predementia to AD dementia and ordered probit regression to estimate transitions across AD dementia stages. We investigate the cost-effectiveness of a hypothetical treatment scenario for people in MCI due to AD.Results: We present an open-access model-based decision-analytic framework. Assuming a modest DMT treatment effect in MCI, we predict extended life expectancy and a reduction in time with AD dementia.Discussion: Any future DMT for AD is expected to pose significant economic challenges across all health-care systems, and decision-analytic modeling will be required to assess costs and outcomes. Further developments are needed to inform these health policy considerations. (C) 2019 The Authors. Published by Elsevier Inc. on behalf of the Alzheimer's Association.

Objectives Emerging evidence supports sequential therapy with anabolic followed by antiresorptive in patients at high-risk of fragility fractures. This study assessed the cost-effectiveness of sequential treatment with abaloparatide (ABL) followed by alendronate (ALN) [(ABL/ALN)] compared to ALN monotherapy and to sequential treatment starting with antiresorptive therapy (ALN/ABL/ALN). Methods A previously validated Markov microsimulation model was used to estimate the cost-effectiveness of sequential ABL/ALN compared to ALN monotherapy and to sequential ALN/ABL/ALN from a lifetime US payer perspective. In line with practice guidelines, patients were assumed to receive ABL for 18 months followed by 5 years of ALN, or ALN monotherapy for 5 years, or a sequence of ALN for 2 years followed by 18 months of ABL and then by 3 years ALN. Evaluation was conducted for patients aged 50-80 years old with a BMD T-score ≤-3.5 and without a history of prior fracture, or with a T-score between -2.5 and -3.5 and a history of ≥ 1 osteoporotic fracture. Results Sequential ABL/ALN was cost-effective (threshold of US$150,000 per QALY) vs generic ALN monotherapy in women ≥60 years with a BMD T-score ≤−3.5 and in women with BMD T-score between −2.5 and −3.5 and history of osteoporotic fracture. In all simulated populations, sequential ABL/ALN therapy was dominant (lower costs, more QALYs) compared with sequential ALN/ABL/ALN, resulting from delayed effect of ABL in patients previously treated with an antiresorptive agent. Conclusions Sequential ABL/ALN therapy is cost-effective vs ALN monotherapy for US postmenopausal women aged ≥60 years at increased risk of fractures.

Individual-based models (IBMs) informing public health policy should be calibrated to data and provide estimates of uncertainty. Two main components of model-calibration methods are the parameter-search strategy and the goodness-of-fit (GOF) measure; many options exist for each of these. This review provides an overview of calibration methods used in IBMs modelling infectious disease spread. We identified articles on PubMed employing simulation-based methods to calibrate IBMs informing public health policy in HIV, tuberculosis, and malaria epidemiology published between 1 January 2013 and 31 December 2018. Articles were included if models stored individual-specific information, and calibration involved comparing model output to population-level targets. We extracted information on parameter-search strategies, GOF measures, and model validation. The PubMed search identified 653 candidate articles, of which 84 met the review criteria. Of the included articles, 40 (48%) combined a quantitative GOF measure with an algorithmic parameter-search strategy–either an optimisation algorithm (14/40) or a sampling algorithm (26/40). These 40 articles varied widely in their choices of parameter-search strategies and GOF measures. For the remaining 44 (52%) articles, the parameter-search strategy could either not be identified (32/44) or was described as an informal, non-reproducible method (12/44). Of these 44 articles, the majority (25/44) were unclear about the GOF measure used; of the rest, only five quantitatively evaluated GOF. Only a minority of the included articles, 14 (17%) provided a rationale for their choice of model-calibration method. Model validation was reported in 31 (37%) articles. Reporting on calibration methods is far from optimal in epidemiological modelling studies of HIV, malaria and TB transmission dynamics. The adoption of better documented, algorithmic calibration methods could improve both reproducibility and the quality of inference in model-based epidemiology. There is a need for research comparing the performance of calibration methods to inform decisions about the parameter-search strategies and GOF measures., Author summary Calibration—that is, “fitting” the model to data—is a crucial part of using mathematical models to better forecast and control the population-level spread of infectious diseases. Evidence that the mathematical model is well-calibrated improves confidence that the model provides a realistic picture of the consequences of health policy decisions. To make informed decisions, Policymakers need information about uncertainty: i.e., what is the range of likely outcomes (rather than just a single prediction). Thus, modellers should also strive to provide accurate measurements of uncertainty, both for their model parameters and for their predictions. This systematic review provides an overview of the methods used to calibrate individual-based models (IBMs) of the spread of HIV, malaria, and tuberculosis. We found that less than half of the reviewed articles used reproducible, non-subjective calibration methods. For the remaining articles, the method could either not be identified or was described as an informal, non-reproducible method. Only one-third of the articles obtained estimates of parameter uncertainty. We conclude that the adoption of better-documented, algorithmic calibration methods could improve both reproducibility and the quality of inference in model-based epidemiology.

Introduction Cystic fibrosis (CF) is a life-limiting, hereditable condition, with the highest prevalence in Europe. CF treatments have led to improvements in clinical symptoms, disease management and decelerated disease progression. However, little is known about the health state utility (HSU) associated with CF disease states, adverse events, and changes in disease severity. Although HSU data have contributed to existing health economic modelling studies, a lack of such data have been highlighted. This systematic review aims to provide a summary of HSU-related research in CF and highlight related research gaps. Methods Online searches were performed in six databases and studies in any of the following categories were included: (1) estimation of HSUs in CF; (2) mapping studies between patient-reported outcome measures (PROMs) and HSUs; (3) economic evaluations on the management of CF that report primary HSU data; and (4) any CF clinical trial that reported HSU as an outcome. Results A total of 17 studies were reviewed, of which 12 provided HSU values for specific CF populations. The remaining five articles provided HSU data that were broken down by CF relevant health states, including lung transplantations, pulmonary exacerbation (PEx) events and forced expiratory volume in 1 s (FEV1). Conclusion Current HSU data in CF are limited and there is considerable scope for further research, both in providing HSU values for CF and in investigating methods for HSU elicitation/evaluation in CF populations. Electronic supplementary material The online version of this article (10.1007/s41669-019-0144-1) contains supplementary material, which is available to authorized users.

ObjectiveTo evaluate a potential reduction in injury related healthcare costs when using the ‘11+ Kids’ injury prevention programme compared with a usual warmup in children’s football.MethodsThis cost effectiveness analysis was based on data collected in a cluster randomised controlled trial over one season from football teams (under-9 to under-13 age groups) in Switzerland. The intervention group (INT) replaced their usual warmup with ‘11+ Kids’, while the control group (CON) warmed up as usual. Injuries, healthcare resource use and football exposure (in hours) were collected prospectively. We calculated the mean injury related costs in Swiss Francs (CHF) per 1000 hours of football. We calculated the cost effectiveness (the direct net healthcare costs divided by the net health effects of the ‘11+ Kids’ intervention) based on the actual data in our study (trial based) and for a countrywide implementation scenario (model based).ResultsCosts per 1000 hours of exposure were CHF228.34 (95% CI 137.45, 335.77) in the INT group and CHF469.00 (95% CI 273.30, 691.11) in the CON group. The cost difference per 1000 hours of exposure was CHF−240.66 (95%CI −406.89, −74.32). A countrywide implementation would reduce healthcare costs in Switzerland by CHF1.48 million per year. 1002 players with a mean age of 10.9 (SD 1.2) years participated. During 76 373 hours of football, 99 injuries occurred.ConclusionThe ‘11+ Kids’ programme reduced the healthcare costs by 51% and was dominant (ie, the INT group had lower costs and a lower injury risk) compared with a usual warmup. This provides a compelling case for widespread implementation.

Purposes Intervention mapping (IM) is a protocol for developing effective behavior change interventions. It has been used for 10 years to develop work disability prevention (WDP) interventions, but it is not known to what extent and with what success. The main objective of this study was to review the effectiveness of these interventions. Secondary objectives were to review their fidelity to the IM protocol, their theoretical frameworks and their content. Methods A search strategy was conducted in MEDLINE, Web of Science, PsycINFO, Pascal, Francis, and BDSP. All titles and abstracts were reviewed. A standardized extraction form was developed. All included studies were reviewed by two reviewers blinded to each other. Results Eight WDP interventions were identified aimed at return to work (RTW; n = 6) and self-management at work (n = 2). RTW interventions targeted workers with stress-related mental disorders (n = 1), low back pain (n = 1), musculoskeletal disorders (n = 1), cancer (n = 2) and gynecological surgery (n = 1). The fidelity to the IM protocol was weaker for the participatory planning group. Matrices of change, change methods, and applications were systematically reported. The main theoretical frameworks used were the attitude-social influence-self efficacy model (n = 4) and the theory of planned behavior (n = 2). Half of the interventions included a workplace component (n = 4). Two interventions were reported as effective, and one partially effective. Conclusion The IM protocol is used in WDP since 2007. The participative dimension appears underused. Few theoretical frameworks were used. Implications are to better consider the stakeholders involvement, and mobilize theoretical frameworks with greater attempts to intervene on the work environment.

Background The financial burden of chronic kidney disease (CKD) is increasing due to the ageing population and increased prevalence of comorbid diseases. Our aim was to evaluate age-related differences in health care use and costs in Stage G4/G5 CKD without renal replacement therapy (RRT), dialysis and kidney transplant patients and compare them to the general population. Methods Using Dutch health care claims, we identified CKD patients and divided them into three groups: CKD Stage G4/G5 without RRT, dialysis and kidney transplantation. We matched them with two controls per patient. Total health care costs and hospital costs unrelated to CKD treatment are presented in four age categories (19–44, 45–64, 65–74 and ≥75 years). Results Overall, health care costs of CKD patients ≥75 years of age were lower than costs of patients 65–74 years of age. In dialysis patients, costs were highest in patients 45–64 years of age. Since costs of controls increased gradually with age, the cost ratio of patients versus controls was highest in young patients (19–44 years). CKD patients were in greater need of additional specialist care than the general population, which was already evident in young patients. Conclusion Already at a young age and in the earlier stages of CKD, patients are in need of additional care with corresponding health care costs far exceeding those of the general population. In contrast to the general population, the oldest patients (≥75 years) of all CKD patient groups have lower costs than patients 65–74 years of age, which is largely explained by lower hospital and medication costs.

AIMS: With evidence supporting the use of preventive interventions for prediabetes populations and the use of novel biomarkers to stratify the risk of progression, there is a need to evaluate their cost-effectiveness across jurisdictions. Our aim is to summarize and assess the quality and validity of decision models and model-based economic evaluations of populations with prediabetes, to evaluate their potential use for the assessment of novel prevention strategies and to discuss the knowledge gaps, challenges and opportunities.MATERIALS AND METHODS: We searched Medline, Embase, EconLit and NHS EED between 2000 and 2018 for studies reporting computer simulation models of the natural history of individuals with prediabetes and/or we used decision models to evaluate the impact of treatment strategies on these populations. Data were extracted following PRISMA guidelines and assessed using modelling checklists. Two reviewers independently assessed 50% of the titles and abstracts to determine whether a full text review was needed. Of these, 10% was assessed by each reviewer to cross-reference the decision to proceed to full review. Using a standardized form and double extraction, each of four reviewers extracted 50% of the identified studies.RESULTS: A total of 29 published decision models that simulate prediabetes populations were identified. Studies showed large variations in the definition of prediabetes and model structure. The inclusion of complications in prediabetes (n = 8) and type 2 diabetes (n = 17) health states also varied. A minority of studies simulated annual changes in risk factors (glycaemia, HbA1c, blood pressure, BMI, lipids) as individuals progressed in the models (n = 7) and accounted for heterogeneity among individuals with prediabetes (n = 7).CONCLUSIONS: Current prediabetes decision models have considerable limitations in terms of their quality and validity and do not allow evaluation of stratified strategies using novel biomarkers, highlighting a clear need for more comprehensive prediabetes decision models.

Background: To assess the cost-effectiveness of new treatments in Germany, the efficiency frontier (EF) method has been developed. We compared the cost-effectiveness analysis using international standards and the German methodology, using the heart failure drug sacubitril/valsartan as an example.Methods: A previously developed Markov model was adapted to include 4 treatment options: no treatment, enalapril, candesartan, and sacubitril/valsartan. The internationally used incremental cost-effectiveness ratio (ICER) was calculated, as well as cost-effectiveness acceptability curves. Additionally, EFs, net monetary benefits (NMBs), and price-acceptability curves were created according to German guidelines. All analyses were performed from the perspective of the German Statutory Health Insurance.Results: The base-case ICER for sacubitril/valsartan compared to enalapril is (sic)19 300/quality-adjusted life-year. On the cost-effectiveness acceptability curve, sacubitril/valsartan is most likely to be cost-effective, out of all included comparators, from a hypothetical willingness-to-pay threshold of (sic)18 250/quality-adjusted life-year onward. No EF could be constructed for the base case. Taking the uncertainty of the input parameters into account for the probabilistic sensitivity analysis, a NMB of around -(sic)14 000 was calculated, depending on the outcome considered, with the NMB being zero at a daily price for sacubitril/valsartan ranging from (sic)1.52 to (sic)1.67.Conclusion: We calculated an ICER for Germany, comparable to previously published cost-effectiveness analyses for Europe, which widely concluded sacubitril/valsartan to be cost-effective. Using the German EF approach, a considerable discount needs to be applied before sacubitril/valsartan can be considered cost-effective.

Background According to some studies, interventions can prevent or delay frailty, but their effect in preventing adverse outcomes in frail community-dwelling older people is unclear. The aim is to investigate the effect of an intervention on adverse outcomes in frail older adults. Methods A systematic review and meta-analysis of Medline, Embase, the Cochrane Library, and Social Sciences Citation Index. Randomized controlled studies that aimed to treat frail community-dwelling older adults, were included. The outcomes were mortality, hospitalization, formal health costs, accidental falls, and institutionalization. Several sub-analyses were performed (duration of intervention, average age, dimension, recruitment). Results Twenty-five articles (16 original studies) were included. Six types of interventions were found. The pooled odds ratios (OR) for mortality when allocated in the experimental group were 0.99 [95% CI: 0.79, 1.25] for case management and 0.78 [95% CI: 0.41, 1.45] for provision information intervention. For institutionalization, the pooled OR with case management was 0.92 [95% CI: 0.63, 1.32], and the pooled OR for information provision intervention was 1.53 [95% CI: 0.64, 3.65]. The pooled OR for hospitalization when allocated in the experimental group was 1.13 [95% CI: 0.95, 1.35] for case management. Further sub-analyses did not yield any significant findings. Conclusion This systematic review and meta-analysis does not provide sufficient scientific evidence that interventions by frail older adults can be protective against the included adverse outcomes. A sub-analysis for some variables yielded no significant effects, although some findings suggested a decrease in adverse outcomes. Trial registration Prospero registration CRD42016035429. Electronic supplementary material The online version of this article (10.1186/s12877-018-0936-7) contains supplementary material, which is available to authorized users.

Background Enterobacteriaceae are a common cause of hospital infections. Carbapenems are a clinically effective treatment of such infections. However, resistance is on the rise. In particular, carbapenemase-producing carbapenem-resistant Enterobacteriaceae (CP-CRE) are increasingly common. In order to limit spread in clinical settings, screening and isolation is being recommended, but many different screening methods are available. We aimed to compare the impact and costs of three algorithms for detecting CP-CRE carriage. Methods We developed an individual-based simulation model to compare three screening algorithms using data from a UK National Health Service (NHS) trust. The first algorithm, “Direct PCR”, was highly sensitive/specific and quick (half a day), but expensive. The second, “Culture + PCR”, was relatively sensitive/specific but slower, requiring 2.5 days. A third algorithm, “PHE”, repeated the “Culture + PCR” three times with an additional PCR. Scenario analysis was used to compare several levels of CP-CRE prevalence and coverage of screening, different specialities as well as isolation strategies. Our outcomes were (1) days that a patient with CP-CRE was not detected and hence not isolated (“days at risk”), (2) isolation bed days, (3) total costs and (4) mean cost per CP-CRE risk day averted per year. We also explored limited isolation bed day capacity. Results We found that although a Direct PCR algorithm would reduce the number of CP-CRE days at risk, the mean cost per CP-CRE risk day averted per year was substantially higher than for a Culture + PCR algorithm. For example, in our model of an intensive care unit, during a year with a 1.6% CP-CRE prevalence and 63% screening coverage, there were 508 (standard deviation 15), 642 (14) and 655 (14) days at risk under screening algorithms Direct PCR, Culture + PCR and PHE respectively, with mean costs per risk day averted of £192, £61 and £79. These results were robust to sensitivity analyses. Conclusions Our results indicate that a Culture + PCR algorithm provides the optimal balance of cost and risk days averted, at varying isolation, prevalence and screening coverage scenarios. Findings from this study will help clinical organisations determine the optimal screening approach for CP-CRE, balancing risk and resources. Electronic supplementary material The online version of this article (10.1186/s12916-018-1117-4) contains supplementary material, which is available to authorized users.

Background In 2012, the World Health Organization set goals for the elimination of onchocerciasis transmission by 2020 in selected African countries. Epidemiological data and mathematical modelling have indicated that elimination may not be achieved with annual ivermectin distribution in all endemic foci. Complementary and alternative treatment strategies (ATS), including vector control, will be necessary. Implementation of vector control will require that the ecology and population dynamics of Simulium damnosum (sensu lato) be carefully considered. Methods We adapted our previous SIMuliid POPulation dynamics (SIMPOP) model to explore the impact of larvicidal insecticides on S. damnosum (s.l.) biting rates in different ecological contexts and to identify how frequently and for how long vector control should be continued to sustain substantive reductions in vector biting. SIMPOP was fitted to data from large-scale aerial larviciding trials in savannah sites (Ghana) and small-scale ground larviciding trials in forest areas (Cameroon). The model was validated against independent data from Burkina Faso/Côte d’Ivoire (savannah) and Bioko (forest). Scenario analysis explored the effects of ecological and programmatic factors such as pre-control daily biting rate (DBR) and larviciding scheme design on reductions and resurgences in biting rates. Results The estimated efficacy of large-scale aerial larviciding in the savannah was greater than that of ground-based larviciding in the forest. Small changes in larvicidal efficacy can have large impacts on intervention success. At 93% larvicidal efficacy (a realistic value based on field trials), 10 consecutive weekly larvicidal treatments would reduce DBRs by 96% (e.g. from 400 to 16 bites/person/day). At 70% efficacy, and for 10 weekly applications, the DBR would decrease by 67% (e.g. from 400 to 132 bites/person/day). Larviciding is more likely to succeed in areas with lower water temperatures and where blackfly species have longer gonotrophic cycles. Conclusions Focal vector control can reduce vector biting rates in settings where a high larvicidal efficacy can be achieved and an appropriate duration and frequency of larviciding can be ensured. Future work linking SIMPOP with onchocerciasis transmission models will permit evaluation of the impact of combined anti-vectorial and anti-parasitic interventions on accelerating elimination of the disease. Electronic supplementary material The online version of this article (10.1186/s13071-018-2864-y) contains supplementary material, which is available to authorized users.

Objectives: Few empirical analyses of the impact of organised prostate cancer (PCa) screening on healthcare costs exist, despite cost-related information often being considered as a prerequisite to informed screening decisions. Therefore, we estimate the differences in register-based costs of publicly funded healthcare in the two arms of the Finnish Randomised Study of Screening for Prostate Cancer (FinRSPC) after 20 years. Methods: We obtained individual-level register data on prescription medications, as well as inpatient and outpatient care, to estimate healthcare costs for 80,149 men during the first 20 years of the FinRSPC. We compared healthcare costs for the men in each trial arm and performed statistical analysis. Results: For all men diagnosed with PCa during the 20-year observation period, mean PCa-related costs appeared to be around 10% lower in the screening arm (SA). Mean all-cause healthcare costs for these men were also lower in the SA, but differences were smaller than for PCa-related costs alone, and no longer statistically significant. For men dying from PCa, although the difference was not statistically significant, mean all-cause healthcare costs were around 10% higher. When analysis included all observations, cumulative costs were slightly higher in the CA; however, after excluding extreme values, cumulative costs were slightly higher in the SA. Conclusions: No major cost impacts due to screening were apparent, but the FinRSPC's 20-year follow-up period is too short to provide definitive evidence at this stage. Longer term follow-up will be required to be better informed about the costs of, or savings from, introducing mass PCa screening. (C) 2018 Elsevier Ltd. All rights reserved.

Ambient inhalable particulate matter (PM) is a serious health concern worldwide, but especially so in China where high PM concentrations affect huge populations. Atmospheric processes and emission sources cause spatial and temporal variations in PM concentration and chemical composition, but their influence on the toxicological characteristics of PM are still inadequately understood.In this study, we report an extensive chemical and toxicological characterization of size-segregated urban air inhalable PM collected in August and October 2013 from Nanjing, and assess the effects of atmospheric processes and likely emission sources. A549 human alveolar epithelial cells were exposed to day- and nighttime PM samples (25, 75, 150, 200, 300 mu g/ml) followed by analyses of cytotoxicity, genotoxicity, cell cycle, and inflammatory response.PM10-2.5 and PM0.2 caused the greatest toxicological responses for different endpoints, illustrating that particles with differing size and chemical composition activate distinct toxicological pathways in A549 cells. PM10-2.5 displayed the greatest oxidative stress and genotoxic responses; both were higher for the August samples compared with October. In contrast, PM0.2 and PM2.5-1.0 samples displayed high cytotoxicity and substantially disrupted cell cycle; August samples were more cytotoxic whereas October samples displayed higher cell cycle disruption. Several components associated with combustion, traffic, and industrial emissions displayed strong correlations with these toxicological responses. The lower responses for PM1.0-0.2 compared to PM0.2 and PM2.5-1.0 indicate diminished toxicological effects likely due to aerosol aging and lower proportion of fresh emission particles rich in highly reactive chemical components in the PM1.0-0.2 fraction.Different emission sources and atmospheric processes caused variations in the chemical composition and toxicological responses between PM fractions, sampling campaigns, and day and night. The results indicate different toxicological pathways for coarse-mode particles compared to the smaller particle fractions with typically higher content of combustion-derived components. The variable responses inside PM fractions demonstrate that differences in chemical composition influence the induced toxicological responses.

Introduction: The study aimed to determine the room for improvement of a perfect cerebrospinal fluid (CSF) biomarker and the societal incremental net monetary benefit of CSF in subjects with mild cognitive impairment (MCI) assuming a hypothetical disease-modifying Alzheimer's disease (AD) treatment.Methods: A decision model compared current practice to a perfect biomarker and to two strategies positioning CSF as add- on test when current practice concluded the presence or absence of AD.Results: The simulated MCI population was aged on average 68.3 and 49% had AD. The room for improvement by the perfect CSF test was 0.39 quality adjusted life years, V33,622 ($ 43,372) savings, 2.0 potential beneficial treatment years, and 1.3-year delay in dementia conversion.Discussion: The results indicated more potential benefit from a biomarker positioned to verify subjects who are not expected to have AD (i.e., to prevent undertreatment) rather than to verify subjects expected to have AD (prevent overtreatment). Sensitivity analyses explored different CSF positions. (C) 2015 The Alzheimer's Association. Published by Elsevier Inc. All rights reserved.

BackgroundAlthough there is evidence for the effectiveness of interventions for psychosis among ultra-high-risk (UHR) groups, health economic evaluations are lacking. This study aimed to determine the cost effectiveness and cost–utility of cognitive–behavioural therapy (CBT) to prevent first-episode psychosis.MethodThe Dutch Early Detection and Intervention Evaluation study was a randomized controlled trial of 196 UHR patients with an 18-month follow-up. All participants were treated with routine care (RC) for non-psychotic disorders. The experimental group (n = 95) received add-on CBT to prevent first-episode psychosis. We report the intervention, medical and travel costs, as well as costs arising from loss of productivity. Treatment response was defined as psychosis-free survival and quality-adjusted life years (QALYs) gained.ResultsIn the cost-effectiveness analysis, the proportion of averted psychoses was significantly higher in the CBT condition (89.5% v. 76.2%). CBT showed a 63.7% probability of being more cost effective, because it was less costly than RC by US$844 (£551) per prevented psychosis. In the cost–utility analysis, QALY health gains were slightly higher for CBT than for RC (0.60 v. 0.57) and the CBT intervention had a 52.3% probability of being the superior treatment because, for equal or better QALY gains, the costs of CBT were lower than those of RC.ConclusionsAdd-on preventive CBT for UHR resulted in a significant reduction in the incidence of first psychosis. QALY gains show little difference between the two conditions. The CBT intervention proved to be cost saving.

Addition of the HPV vaccine to available cytological screening has been proposed to increase HPV-related cancer prevention. A comprehensive review on this combined strategy implemented in the Netherlands is lacking. For this review, we therefore analyzed all relevant studies on cost-effectiveness of HPV vaccines in combination with cervical screening in the Netherlands. Most of the studies agree that vaccination in pre-sexual-activity periods of life is cost-effective. Based on published sensitivity analyses, the incremental cost-effectiveness ratio was found to be mainly driven by vaccine cost and discount rates. Fewer vaccine doses, inclusion of additional benefits of these vaccines to prevent HPV-related non-cervical cancers and vaccination of males to further reduce the burden of HPV-induced cancers are three relevant options suggested to be investigated in upcoming economic evaluations.

Item does not contain fulltext PURPOSE: The objective of this study was to estimate the expected cost-utility and cost-effectiveness of the ketogenic diet (KD), vague nerve stimulation (VNS) and care as usual (CAU), using a decision analytic model with a 5-year time horizon. METHODS: A Markov decision analytical model was constructed to estimate the incremental costs, quality-adjusted life years (QALYs) and successfully treated patient (i.e. 50% or more seizure reduction) of the treatment strategies KD, VNS and CAU, from a health care perspective. The base case considered children with intractable epilepsy (i.e. two or more antiepileptic drugs had failed) aged between 1 and 18 years. Data were derived from literature and expert meetings. Deterministic and probabilistic sensitivity analyses were performed. RESULTS: Our results suggest that KD is more effective and less costly, and thus cost-effective compared with VNS, after 12 months. However, compared to CAU, neither KD nor VNS are cost-effective options, they are both more effective but also more expensive (euro346,899 and euro641,068 per QALY, respectively). At 5 years, VNS is cost-effective compared with KD and CAU (euro11,378 and euro68,489 per QALY, respectively) and has a 51% probability of being cost-effective at a ceiling ratio of euro80,000 per QALY. CONCLUSIONS: Our results suggest that on average the benefits of KD and VNS fail to outweigh the costs of the therapies. However, these treatment options should not be ignored in the treatment for intractable epilepsy in individual or specific groups of patients. There is a great need for high quality comparative studies with large patient samples which allow for subgroup analyses, long-term follow-up periods and outcome measures that measure effects beyond seizure frequency (e.g. quality of life). When this new evidence becomes available, reassessment of the cost-effectiveness of KD and VNS in children with intractable epilepsy should be carried out.

Background Many children stand to benefit from being asthma-free for life with primary (i.e., prenatally started) prevention addressing one environmental exposure in a unifaceted (UF) approach or at least two in a multifaceted (MF) approach. We assessed the cost-effectiveness of primary prevention programmes for Dutch children in a decision-analytic framework. Methods A decision-analytic tree model analysing healthcare costs and asthma cases prevented was developed to compare usual care (UC) with two UF and three MF programmes on the primary prevention of asthma amongst children. Programmes were evaluated through incremental cost-effectiveness ratios and net monetary benefits. Decision and parameter uncertainty were subjected to value-of-information analyses. Results The current UC and one of three MF programmes dominated the other alternatives. The MF programme was more costly but also more effective than UC at an incremental cost-effectiveness ratio of (sic)8,209.20/additional asthma case prevented. The value of perfect information to reduce uncertainty was (sic)291.6M at its lowest. Most of the uncertainty in the cost-effectiveness threshold was attributable to the probability and cost estimates for low-risk children. Conclusion This study supports the feasibility of a structured programme that simultaneously addresses exposure to house dust mites, pet dander, environmental tobacco, and breast-feeding as a cost-effective alternative to UC in the primary prevention of asthma amongst children.

BACKGROUND: Asthma medication cost-effectiveness analyses (CEAs) lack the qualitative assessment regarding whether they capture the National Institutes for Health (NIH) 2012 recommended outcomes necessary to allow robust cross-study comparisons.OBJECTIVE: We aimed to assess the current asthma outcomes used in CEAs and recommend a direction for improvement.METHODS: We performed a systematic search using electronic databases including PubMed, EMBASE, Tufts CEA registry, Cochrane, and NHSEED from January 2010 through December 2015. Key words included (1) cost-effectiveness, cost-utility, economic evaluation, health economics, or cost-benefit AND (2) asthma. All CEA studies evaluating 1 or more asthma medication were included. Authors assessed each CEA study with respect to asthma-specific NIH outcome recommendations including core (hospitalizations, emergency department visits, outpatient visits, medication, interventions costs), supplemental (visit categories and work/school absence), and emerging (academic/job-related) asthma outcomes. Besides outcomes of each CEA, issues that could prevent robust cross-study comparison were identified and thematically summarized.RESULTS: A total of 12 pre-NIH and 14 post-NIH recommendation CEAs were included. Eleven (91.7%) and 14 (100%) of the pre-/post-NIH studies included at least 1 core outcome, respectively. Of the 26 total studies, 7 (26.9%) included asthma-specific outpatient visit categories, 6 (23.1%) included asthma school or work absences, 5 (19.2%) included respiratory health care use, and none of the studies included emerging outcomes. Other issues that hamper cross-study comparison include lack of standardized cost data, time frames, quality-of-life measures, and incorporation of adherence.CONCLUSIONS: Although the use of NIH-recommended asthma core outcomes has improved, there is still room for improvement in using supplemental and emerging outcomes. To allow robust cross-study comparisons, future work should focus on further standardizing of data sources and methods.