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PharmacoEconomics
Journal Prestige (SJR): 1.998

Citation Impact (citeScore): 3
Number of Followers: 27

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ISSN (Print) 1170-7690 - ISSN (Online) 1179-2027
Published by Adis

[21 journals]

A Systematic Literature Review of the Economic Evaluations of Treatments
for Patients with Chronic Myeloid Leukemia
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  Abstract: Background and Objectives The management of chronic myeloid leukemia is associated with an extensive economic burden, and as novel interventions are being tested in this disease, understanding the comparative effectiveness is of interest. Findings and conclusions of this important issue continue to evolve with improvements in clinical research and economic understanding. This systematic literature review aims to conduct a comprehensive assessment of economic evaluations in chronic phase chronic myeloid leukemia. Methods Embase®, MEDLINE®, and the National Health Service Economic Evaluation Database were searched on 4 July, 2022 to identify economic evaluations of chronic myeloid leukemia. Health technology assessment websites and key conference proceedings were also searched. Economic evaluations comparing treatment options in adult patients with chronic phase chronic myeloid leukemia were included. The quality of the studies were assessed using Drummond’s checklists. Results The search retrieved 47 studies and 16 health technology assessments that fulfilled the eligibility criteria. Most were cost-utility analyses (23 studies and 11 health technology assessments) and were from the USA (n = 15) and China (n = 7). Twenty-seven studies and six health technology assessments included only patients with chronic phase chronic myeloid leukemia. Most models had a Markov structure, a 1 year to lifetime time horizon, and a 1-month cycle length. Commonly assessed treatments were various tyrosine kinase inhibitors (imatinib, nilotinib, dasatinib, bosutinib, and ponatinib) and other interventions such as interferon-α, hydroxyurea, and allogeneic stem cell transplant. Conclusions In patients with newly diagnosed chronic myeloid leukemia, imatinib regimens were cost effective, mostly owing to the availability of generics. Nilotinib and dasatinib were generally cost effective as second-line agents for patients who were resistant or intolerant to imatinib. Though progress has been made to better characterize the cost effectiveness of first-line and second-line chronic myeloid leukemia therapies, the paucity of published cost-effectiveness studies of third-line treatments increases the uncertainty associated with economic evaluations of later lines of therapy.
  PubDate: 2022-12-01
Cost Effectiveness of the First‐in‐Class ARNI (Sacubitril/Valsartan)
for the Treatment of Essential Hypertension in a Chinese Setting
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  Abstract: Objective The aim of this study was to model the potential long-term disease progression and pharmacoeconomic value of sacubitril/valsartan for the treatment of essential hypertension from a Chinese healthcare system perspective. Methods A Markov cohort model with five health states was constructed to simulate the incidence of acute cardiovascular events and cost per quality-adjusted life-year (QALY) gained with sacubitril/valsartan compared with allisartan isoproxil and valsartan over a lifetime horizon with an annual cycle. Multivariable risk regression models derived from China-PAR data accompanied by hazard ratios were used to transform the dual mechanism of sacubitril/valsartan to lower blood pressure and left ventricular mass index into long-term fatal and non-fatal cardiovascular risks. Efficacy data were calculated using a network meta-analysis integrated by the results of clinical trials. Healthcare costs were determined from a real-world study and published literature, supplemented by expert opinion. Utilities were derived from literature. Both costs and health outcomes were discounted at 5.0% annually, and prices corresponded to 2021. Model validation, deterministic and probabilistic sensitivity analyses were conducted to test the robustness of results. Results For simulated patients with hypertension, sacubitril/valsartan reduced the rates of myocardial infarction by 6.67% and 6.39%, stroke by 9.38% and 8.98%, and heart failure hospitalization by 9.92% and 9.62% relative to allisartan isoproxil and valsartan, respectively. It was also associated with gains in life expectancy among hypertensive individuals of 0.362–0.382 years. Eventually, lifetime costs per patient were CN¥59,272 (US$9187) for sacubitril/valsartan, CN¥54,783 (US$8492) for allisartan isoproxil, and CN¥56,714 (US$8791) for valsartan; total QALYs were 11.38, 11.24, and 11.25, respectively. The incremental cost-effectiveness ratio was CN¥31,805/QALY (US$4930/QALY) compared with allisartan isoproxil, and CN¥19,247/QALY (US$2983/QALY) compared with valsartan, both of which are below the one time per-capita GDP of CN¥80,976/QALY (US$12,551/QALY) in China. Similar results were obtained in various extensive sensitivity analysis scenarios. Conclusions This was the first study to evaluate the cost effectiveness of sacubitril/valsartan in the treatment of hypertension. Sacubitril/valsartan compares favorably with allisartan isoproxil and valsartan in the Chinese setting, which is mainly due to its higher efficacy resulting in fewer cardiovascular events and ultimately less related mortality over time. The results could inform deliberations regarding reimbursement and access to this treatment in China and may provide reference for facilitating more reasonable and efficient allocation of limited resources in such low- and middle-income countries.
  PubDate: 2022-12-01
Conceptual Framework for Optimised Proxy Value Set Selection Through
Supra-National Value Set Development for the EQ-5D Instruments
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  Abstract: Background Preference differences between countries and populations justify the use of country-specific value sets for the EQ-5D instruments. There are no clear criteria based on which the selection of value sets for countries without a national value set should be made. As part of the European PECUNIA project, this study aimed to identify factors contributing to differences in preference-based valuations and develop supra-national value sets for homogenous country clusters in Europe. Methods A literature review was conducted to identify factors relevant to variations in the EQ-5D-3L/5L health state valuations across countries. Factors fulfilling the pre-specified criteria of validity, reliability, international feasibility and comparability were used to group 27 European Union member states, the European Free Trade Association countries and the UK. Clusters of countries were developed based on the frequency of their appearance in the same grouping. The supra-national value sets were estimated for these clusters from the coefficients of existing published valuation studies using the ordinary least-squares model. Results Ten factors were identified from 69 studies. From these, five grouping variables: (1) culture and religion; (2) linguistics; (3) healthcare system typology; (4) healthcare system financing; and (5) sociodemographic aspects were derived to define the groups of homogenous countries. Frequency-based grouping revealed five cohesive clusters: English-speaking, Nordic, Central-Western, Southern and Eastern European. Conclusions European countries were clustered considering variables that may relate to differences in health state valuations. Supra-national value sets provide optimised proxy value set selection in the lack of a national value set and/or for regional decision making.
  PubDate: 2022-12-01
Efficiency of the EmERGE Pathway of Care in Five European HIV Centres
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  Abstract: Objective We aimed to calculate the efficiency of the EmERGE Pathway of Care in five European HIV clinics, developed and implemented for medically stable people living with HIV. Methods Participants were followed up for 1 year before and after implementation of EmERGE, between April 2016 and October 2019. Micro-costing studies were performed in the outpatient services of the clinics. Unit costs for outpatient services were calculated in national currencies and converted to US$ 2018 OECD purchasing parity prices to enable between clinic comparisons in terms of outcomes and costs. Unit costs were linked to the mean use of services for medically stable people living with HIV, before and after implementation of EmERGE. Primary outcome measures were CD4 count and viral load; secondary outcomes were patient activation (PAM13) and quality of life (PROQOL-HIV). Out-of-pocket expenditure data were collected. Results There were 2251 participants: 87–93% were male, mean age at entry was 41–47 years. Medically stable people living with HIV had outpatient visits in four sites which decreased by 9–31% and costs by 5–33%; visits and costs increased by 8% in one site, which had to revert back to face-to-face visits. Antiretroviral drugs comprised 83–91% of annual costs: the Portuguese site had the highest antiretroviral drug costs in US$ purchasing parity prices. Primary and secondary outcome measures of participants did not change during the study. Conclusions EmERGE is acceptable and provided cost savings in different socio-economic settings. Antiretroviral drug costs remain the main cost drivers in medically stable people living with HIV. While antiretroviral drug prices in local currencies did not differ that much between countries, conversion to US$ purchasing parity prices revealed antiretroviral drugs were more expensive in the least wealthy countries. This needs to be taken into consideration when countries negotiate drug prices with pharmaceutical vendors. Greater efficiencies can be anticipated by extending the use of the EmERGE Pathway to people with complex HIV infection or other chronic diseases. Extending such use should be systematically monitored, implementation should be evaluated and funding should be provided to monitor and evaluate future changes in service provision.
  PubDate: 2022-12-01
Cost-Effectiveness Analysis of Prostate Cancer Screening in the UK: A
Decision Model Analysis Based on the CAP Trial
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  Abstract: Background and Objective Most guidelines in the UK, Europe and North America do not recommend organised population-wide screening for prostate cancer. Prostate-specific antigen-based screening can reduce prostate cancer-specific mortality, but there are concerns about overdiagnosis, overtreatment and economic value. The aim was therefore to assess the cost effectiveness of eight potential screening strategies in the UK. Methods We used a cost-utility analysis with an individual-based simulation model. The model was calibrated to data from the 10-year follow-up of the Cluster Randomised Trial of PSA Testing for Prostate Cancer (CAP). Treatment effects were modelled using data from the Prostate Testing for Cancer and Treatment (ProtecT) trial. The participants were a hypothetical population of 10 million men in the UK followed from age 30 years to death. The strategies were: no screening; five age-based screening strategies; adaptive screening, where men with an initial prostate-specific antigen level of < 1.5 ng/mL are screened every 6 years and those above this level are screened every 4 years; and two polygenic risk-stratified screening strategies. We assumed the use of pre-biopsy multi-parametric magnetic resonance imaging for men with prostate-specific antigen ≥ 3 ng/mL and combined transrectal ultrasound-guided and targeted biopsies. The main outcome measures were projected lifetime costs and quality-adjusted life-years from a National Health Service perspective. Results All screening strategies increased costs compared with no screening, with the majority also increasing quality-adjusted life-years. At willingness-to-pay thresholds of £20,000 or £30,000 per quality-adjusted life-year gained, a once-off screening at age 50 years was optimal, although this was sensitive to the utility estimates used. Although the polygenic risk-stratified screening strategies were not on the cost-effectiveness frontier, there was evidence to suggest that they were less cost ineffective than the alternative age-based strategies. Conclusions Of the prostate-specific antigen-based strategies compared, only a once-off screening at age 50 years was potentially cost effective at current UK willingness-to-pay thresholds. An additional follow-up of CAP to 15 years may reduce uncertainty about the cost effectiveness of the screening strategies.
  PubDate: 2022-12-01
Cost Effectiveness of Adding Pembrolizumab to Platinum and
Fluoropyrimidine-Based Chemotherapy as First-Line Treatment for Advanced
Esophageal Cancer: A US Healthcare Payer’s Perspective
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  Abstract: Background and Objective Pembrolizumab plus cisplatin and fluorouracil demonstrated superior efficacy and comparable safety compared with fluorouracil and cisplatin (FP) as first-line treatment for locally advanced unresectable or metastatic carcinoma of the esophagus and gastroesophageal junction adenocarcinoma in a phase III trial (KEYNOTE-590). This study evaluated the cost effectiveness of pembrolizumab plus FP versus FP and versus a blended chemotherapy comparator including FP, carboplatin plus paclitaxel, FOLFOX, FOLFIRI, docetaxel plus FP, trastuzumab plus FP, and trastuzumab plus FOLFOX from a US healthcare payer’s perspective. Methods A partitioned survival model was developed with three health states (progression-free, progressive disease, and death). Overall survival, progression-free survival, time on treatment, and adverse events were informed by patient-level data from KEYNOTE-590. The blended chemotherapy comparator reflected the current US treatment landscape and was assumed to have similar efficacy and safety as FP. Health utilities were estimated using linear mixed-effects models based on EQ-5D-5L data from the trial. Resource use and cost inputs (2020 US dollars) were based on US standard sources and literature. Costs, life-years, and quality-adjusted life-years (QALYs) discounted at 3.0% per year and incremental cost-effectiveness ratio were outcomes in the model. Sensitivity and scenario analyses were conducted to assess the robustness of base-case results. Results Compared with FP, pembrolizumab plus FP produced a mean gain of 0.86 life-year and 0.77 QALY with additional costs of $112,630 over 37.6 years, yielding an incremental cost-effectiveness ratio of $147,097 per QALY. Results were similar when the intervention was pembrolizumab plus alternative chemotherapies or when blended chemotherapy became the comparator. Results were most sensitive to different overall survival extrapolation approaches. Conclusions Our analysis suggests that pembrolizumab plus chemotherapy extended life-years and QALYs and is cost effective compared with chemotherapy alone as a first-line treatment for advanced esophageal cancer in the USA given a willingness-to-pay threshold of $150,000 per QALY.
  PubDate: 2022-12-01
Estimating the Relationship Between EQ-5D-5L and EQ-5D-3L: Results from a
UK Population Study
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  Abstract: Objectives The aim of this study was to estimate the relationship between EQ-5D-3L and EQ-5D-5L, in both directions, using a single model. Methods An online survey containing both variants of EQ-5D, with randomised ordering, was administered to a large UK sample in 2020. A joint statistical model of the ten EQ-5D responses (five at 5L, five at 3L), using a multi-equation ordinal regression framework was estimated. The joint model ensures mappings in either direction are fully consistent with the information in the sample and satisfy Bayes’ rule. Three extensions enhance model flexibility: a copula specification allows differing degrees of correlation between the 3L and 5L responses at the upper and lower extremes of health; a normal mixture residual distribution gives flexibility in the distributional form of responses; and a common factor captures correlations in responses across the five dimensions. Results Almost 50,000 responses were received. Thirty-five percent of respondents reported an existing medical condition. Ninety percent of possible 3L and 43% of possible 5L health states were observed. The preferred model specification includes age, sex and the responses to the EQ-5D instrument. Close alignment to the observed data was observed both in within-sample and out-of-sample comparisons. Conclusion The results from this study provide a means of translating evidence to or from EQ-5D-3L to or from 5L based on a large-scale UK population survey with randomised ordering. Mapping can be performed either using descriptive system responses, individual utility scores or summary statistics.
  PubDate: 2022-11-30
Findings from a Roundtable Discussion with US Stakeholders on Valuation of
the EQ-5D-Y-3L
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  Abstract: Objectives The International Valuation Protocol for the valuation of the EQ-5D-Y-3L provides baseline guidance, but country-specific context is also important. This study aimed to obtain US stakeholders’ input on key considerations for youth valuation in the US. Methods A total of 14 stakeholders representing various backgrounds were identified via the investigators’ networks. A 2-h online meeting was held to discuss (1) the need for a US value set for the EQ-5D-Y-3L; (2) willingness to pay more for quality-adjusted life-year (QALY) gains for children versus adults; (3) sampling strategies; (4) framing perspectives; and (5) other challenges. The session was recorded, transcribed, and summarized. Results Several stakeholders supported paying more for QALY gains for children in recognition of their potential future contributions to society, as well as to avoid potential undervaluation and promote access to innovative treatments. Concerns regarding possible double counting, lack of data to showcase long-term benefits, and dangers of paying more for certain subgroups were also expressed. Most of the stakeholders felt that adolescents could relate to a 10-year-old’s perspective better than adults and were capable of self-completing valuation tasks, and thus should be directly included in the valuation study. There were concerns that adults would be inconsistent in their views about a 10-year-old, partly depending on their status as a parent. Conclusions US stakeholders provided insights relevant to youth valuation in a US context and were open to continued dialogue with investigators. This study could be useful to investigators who are conducting youth valuation studies in different countries and seeking stakeholder input.
  PubDate: 2022-11-29
Extensions of Health Economic Evaluations in R for Microsoft Excel Users:
A Tutorial for Incorporating Heterogeneity and Conducting Value of
Information Analyses
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  Abstract: Abstract Advanced health economic analysis techniques currently performed in Microsoft Excel, such as incorporating heterogeneity, time-dependent transitions and a value of information analysis, can be easily transferred to R. Often the outputs of survival analyses (such as Weibull regression models) will estimate the impacts of correlated patient characteristics on patient outcomes, and are utilised directly as inputs for health economic decision models. This tutorial provides a step-by-step guide of how to conduct such analyses with a Markov model developed in R, and offers a comparison with established analyses performed in Microsoft Excel. This is done through the conversion of a previously published Microsoft Excel case study of a hip replacement surgery cost-effectiveness model. We hope that this paper can act as a facilitator in switching decision models from Microsoft Excel to R for complex health economic analyses, providing open-access code and data, suitable for future adaptation.
  PubDate: 2022-11-28
Role of Economic Evaluations on Pricing of Medicines Reimbursed by the
Italian National Health Service
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  Abstract: Objective The main objective of this study was to explore the extent to which the incremental cost-effectiveness ratio (ICER), alongside other factors, predicts the final outcome of medicine price negotiation in Italy. The second objective was to depict the mean ICER of medicines obtained after negotiation. Methods Data were extracted from company dossiers submitted to the Italian Medicines Agency (AIFA) from October 2016 to January 2021 and AIFA’s internal database. Beta-based regression analyses were used to test the effect of ICER and other variables on the outcome of price negotiation (ΔP), defined as the percentage difference between the list price requested by manufacturers and the final price paid by the Italian National Health Service (INHS). Results In our dataset of 48 pricing and reimbursement procedures, the ICER before negotiation was one of the variables with a major impact on the outcome of negotiation when ≥ 40,000€/QALY. As resulting from multiple regression analyses, the effect of the ICER on ΔP seemed driven by medicines for non-onco-immunological and non-rare diseases. Overall, the negotiation process granted mean incremental costs of €64,688 and mean incremental QALYs of 1.96, yielding an average ICER of €33,004/QALY. Conclusions This study provides support on the influence of cost-effectiveness analysis on price negotiation in the Italian context, providing an estimate of the mean ICER of reimbursed medicines, calculated using net confidential prices charged by the INHS. The role and use of economic evaluations in medicines pricing should be further improved to get the best value for money.
  PubDate: 2022-11-25
The Egyptian EQ-5D-5L Extensive Pilot Study: Lessons Learned
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  Abstract: Objectives To investigate the effect of an extensive pilot phase in improving protocol compliance, face validity, reduction of interviewer effect and prediction errors in the composite time trade-off (cTTO) data elicited as part of the EQ-5D-5L valuation study in Egypt. Methods This study used the cTTO data and quality control (QC) reports from the Egyptian EQ-5D-5L valuation study. Three-level mixed models were estimated to test whether interviewer effects were reduced during the pilot phase and subsequent rounds of collected cTTO data. Ordinary least square (OLS) regression analysis was conducted for each interviewer separately to test whether the mean absolute error (MAE) improved as interviewers completed more interviews. Moreover, improvement in protocol compliance, face validity and reduction of prediction errors in the cTTO data were tested. Results 1180 interviews were conducted by nine interviewers and included in the final analysis, of which 206 interviews were pilot and 974 interviews were actual. There was substantial improvement in the face validity and reduction of prediction errors in the cTTO data where the MAE of the actual data was 0.37, which is much lower than that of the pilot data, which was 0.44. However, there was an initial high level of protocol compliance in terms of the four indicators of the QC tool and the variance attributed to the interviewers was small throughout the whole study. Conclusions This study clarified the benefits of the pilot phase and the strict implementation of the QC tool in improving the face validity and the prediction accuracy of the cTTO data. However, a more extensive pilot phase may be more beneficial in EQ-5D-5L valuation studies that have issues initially with protocol compliance and interviewer effects.
  PubDate: 2022-11-25
Psychometric Properties of Generic Preference-Weighted Measures for
Children and Adolescents: A Systematic Review
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  Abstract: Introduction Preference-weighted measures (PWMs)—also referred to as preference-based measures in the literature—of health status/health-related quality of life plays an essential role in estimating quality-adjusted life-years (QALY) for use in economic evaluations of healthcare products and interventions. However, as PWMs are first and foremost intended to accurately reflect respondent health status, they should ideally demonstrate good psychometric properties for the population in question. This study aimed to systematically review published evidence on the measurement properties of commonly used PWMs for children and adolescents. Methods Three electronic databases (PubMed, Medline, and PsycINFO) were searched for articles assessing the psychometric properties (content validity, construct validity—including convergent validity and known-group validity, test-retest reliability, and responsiveness) of the PWMs of interest (AQoL-6D, CHU9D, HUI2, HUI3, and EQ-5D-Y). The COsensus-based Standards for the selection of health Measurement INstruments methodology (COSMIN) guidelines were used to assess (a) the methodological quality of the studies included and (b) the psychometric performance of the instruments covered. Data were analysed overall as well as by population (country and disease group) and perspective (self-report or proxy-report). The study protocol was registered in the International Prospective Register of Systematic Reviews (PROSPERO) database (CRD42021277296). Results In total, 53 articles were included in this systematic review. Health Utilities Index (HUI) was tested only in patient populations, CHU9D was most frequently tested in general population samples, while EQ-5D-Y was tested in both populations. Overall, there was high-quality evidence supporting sufficient construct validity for all instruments except AQoL-6D. Evidence supporting test-retest and responsiveness was scarce. There was high-quality evidence supporting sufficient responsiveness of HUI2 and HUI3, and inconsistent test-retest reliability of CHU9D and EQ-5D-Y. Evidence for content validity was minimal and therefore not extracted and synthesized for any PWMs. Conclusion This review provides updated evidence on the measurement properties of existing generic PWMs for children and adolescents. High-quality evidence for all relevant psychometric properties and across a range of populations was not available for any of the instruments included, indicating that further work is needed in this direction. This study has identified some of the most noticeable evidence gaps for each of the individual measures. Users can use this information to guide their decision on the choice of PWM to administer.
  PubDate: 2022-11-21
The Societal Cost of Schizophrenia: An Updated Systematic Review of
Cost-of-Illness Studies
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  Abstract: Background Schizophrenia imposes a substantial economic burden on society. This updated systematic review aims to collate the latest societal cost of schizophrenia across countries by reviewing recent cost-of-illness (COI) studies. Methods An electronic search was conducted across several databases (MEDLINE, Embase, PsycINFO, Cochrane Database of Systematic Reviews, Health Management Information Consortium, and System for Information on Grey Literature) to identify COI studies published from 2016 to 2022. Two independent reviewers selected studies for inclusion. The cost components and estimates reported by included studies were descriptively summarised. All costs were converted to US dollars (2022 values). Study quality was assessed using a checklist adapted from Larg & Moss. Results Twenty-four studies were included (5 from the update review and 19 from the original review), of which only two were conducted for low- and middle-income countries (LMICs). Widespread methodological heterogeneity among included studies was observed. The annual societal cost per person varied from US$819 in Nigeria to US$94,587 in Norway. Productivity losses accounted for 32–83% of the overall societal cost, whilst direct healthcare cost made up 11–87%. The reporting quality of included studies varied. Conclusion This review highlights the substantial economic burden of schizophrenia and a lack of COI studies for LMICs. Recommendations on future research, and good practices on improving the methodological and reporting quality of COI research for schizophrenia are provided.
  PubDate: 2022-11-21
Estimating an EQ-5D-Y-3L Value Set for China
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  Abstract: Introduction The standard EQ-5D-Y-3L valuation protocol applies DCE data as the primary preference source to model the relative importance of dimensions while cTTO data served to anchor the DCE coefficients onto the QALY scale. This study aims to estimate an EQ-5D-Y-3L value set for China following this protocol, but with a larger cTTO design to better understand the role of cTTO data in estimating EQ-5D-Y-3L value sets. Methods In total, 150 choice sets and 28 EQ-5D-Y-3L health states were valued using DCE and cTTO methods with two independent samples, respectively. General public from 14 different regions were recruited using quota sampling method to achieve representativeness. We compared two modelling strategies: (1) fit the DCE data with mixed logit model with correlated coefficients and a subsequent mapping procedure for anchoring; (2) fit the DCE and TTO data jointly in a hybrid model. Two evaluation criteria (1) coefficient significance and monotonicity; (2) prediction accuracy of the observed cTTO values were used to select the value set. Results In total, 1476 individuals participated in the study, with 1058 participated the DCE interview and 418 participated the cTTO interview. The highest mean TTO value was 0.924 for state 11112 and the lowest mean TTO value was − 0.088 for state 33333. The hybrid model with an A3 term performed the best and was selected as the value set. Discussion Following the international protocol and using a larger cTTO design, this study established the EQ-5D-Y-3L value set using a hybrid model for China. Future EQ-5D-Y-3L valuation study could consider using a larger cTTO design for estimating the value set.
  PubDate: 2022-11-18
Analytical Considerations When Anchoring Discrete Choice Experiment Values
Using Composite Time Trade-Off Data: The Case of EQ-5D-Y-3L
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  Abstract: Abstract Discrete choice experiments (DCEs) are becoming increasingly used to elicit preferences for children’s health states. However, DCE data need to be anchored to produce value sets, and composite time trade-off (cTTO) data are typically used in the context of EQ-5D-Y-3L valuation. The objective of this paper is to compare different anchoring methods, summarise the characteristics of the value sets they produce, and outline key considerations for analysts. Three anchoring methods were compared using data from published studies: (1) rescaling using the mean value for the worst health state; (2) linear mapping; and (3) hybrid modelling. The worst state rescaling value set had the largest range. The worst state rescaling and linear mapping value sets preserved the relative importance of the dimensions from the DCE, whereas the hybrid model value set did not. Overall, the predicted values from the hybrid model value set were more closely aligned with the cTTO values. These findings are relatively generalisable. Deciding upon which anchoring approach to use is challenging, as there are numerous considerations. Where cTTO data are collected for more than one health state, anchoring on the worst health state will arguably be suboptimal. However, the final choice of approach may require value judgements to be made. Researchers should seek input from relevant stakeholders when commencing valuation studies to help guide decisions and should clearly set out their rationale for their preferred anchoring approach in study outputs.
  PubDate: 2022-11-18
The Costs of Dementia in Europe: An Updated Review and Meta-analysis
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  Abstract: Background and Objective The prevalence of dementia is increasing, while new opportunities for diagnosing, treating and possibly preventing Alzheimer’s disease and other dementia disorders are placing focus on the need for accurate estimates of costs in dementia. Considerable methodological heterogeneity creates challenges for synthesising the existing literature. This study aimed to estimate the costs for persons with dementia in Europe, disaggregated into cost components and informative patient subgroups. Methods We conducted an updated literature review searching PubMed, Embase and Web of Science for studies published from 2008 to July 2021 reporting empirically based cost estimates for persons with dementia in European countries. We excluded highly selective or otherwise biased reports, and used a random-effects meta-analysis to produce estimates of mean costs of care across five European regions. Results Based on 113 studies from 17 European countries, the estimated mean costs for all patients by region were highest in the British Isles (73,712 EUR), followed by the Nordics (43,767 EUR), Southern (35,866 EUR), Western (38,249 EUR), and Eastern Europe and Baltics (7938 EUR). Costs increased with disease severity, and the distribution of costs over informal and formal care followed a North-South gradient with Southern Europe being most reliant on informal care. Conclusions To our knowledge, this study represents the most extensive meta-analysis of the cost for persons with dementia in Europe to date. Though there is considerable heterogeneity across studies, much of this is explained by identifiable factors. Further standardisation of methodology for capturing resource utilisation data may further improve comparability of future studies. The cost estimates presented here may be of value for cost-of-illness studies and economic evaluations of novel diagnostic technologies and therapies for Alzheimer’s disease.
  PubDate: 2022-11-15
Response to Comment on “An Integrated
Pharmacokinetic-Pharmacodynamic-Pharmacoeconomic Modeling Method to
Evaluate Treatments for Adults with Schizophrenia”
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  PubDate: 2022-11-11
Comment on “An Integrated
Pharmacokinetic–Pharmacodynamic–Pharmacoeconomic Modeling Method to
Evaluate Treatments for Adults with Schizophrenia”
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  PubDate: 2022-11-11
Getting the Price Right: Lessons for Medicare Price Negotiation from Peer
Countries
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  Abstract: Abstract The USA pays more for brand-name prescription drugs than any other country and new legislation from August 2022 gives Medicare the authority to directly negotiate certain drug prices with manufacturers starting in 2026—something the federal insurer had been prohibited from doing for its prior history. As the USA prepares for negotiations, we therefore surveyed how comparable industrialized countries use statutory requirements and procedures to negotiate brand-name drug prices. Guidance documents, regulations, government and academic publications were reviewed to identify the process of negotiating drug prices in peer countries that have been cited as potential examples for US payment reform: Australia, Canada, France, Germany, and the UK. Processes for arriving at a final price for a drug generally fall under three approaches: statutory rebates, setting a maximum price, and arbitration between national (public) insurers and manufacturers. Each approach to price negotiation could be adopted by Medicare and reduce spending even if Medicare does not adopt an exclusionary or closed formulary. Much remains to be determined about how the new price negotiation authority in the USA will be implemented, and policymakers can learn from comparator countries’ statutory and regulatory strategies for price negotiation.
  PubDate: 2022-11-09
Economic Implications of Endometriosis: A Review
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  Abstract: Abstract Endometriosis is a chronic inflammatory disease that can have serious physical and emotional consequences for patients in terms of pain, quality of life, and infertility. Despite affecting about 10% of women, the pathophysiology and economic impact of the disease are not fully understood. This study aimed to review and summarize research articles quantifying the direct and indirect costs of endometriosis in the context of current national and international treatment guidelines. A search including the terms ‘endometriosis’ AND ‘costs’ OR ‘cost of illness’ OR ‘cost analysis’ OR ‘economic burden’ was performed, focusing on studies published between January 2000 and May 2022. Total costs, costs of primary and secondary care, productivity losses, and indirect costs were reported. The medical costs of endometriosis were principally registered in secondary care settings, where surgery was the main cost driver. There was considerable variability of populations and study settings, with the overall direct medical cost range of endometriosis from US$1459 to US$20,239 (2022) per patient per year. An increasing trend has been reported in secondary care costs over time; however, not enough data were available at this time to evaluate inpatient and outpatient costs versus treatment strategies. Similarly, further research is required to evaluate the costs and potential savings associated with new therapies. Numerous studies have evaluated the indirect costs of endometriosis in recent years, finding costs between US$4572 and US$14,079 (2022). Currently, limited data are available on the economic burden of the disease at the patient level.
  PubDate: 2022-11-08