PharmacoEconomics OpenPub Date : 2025-07-01Epub Date: 2025-04-22DOI: 10.1007/s41669-025-00575-1
Sophie van Beekhuizen, Menglu Che, Loraine Monfort, Mahmoud Hashim, Ali Azough, Nicole Kubitz, Adrian Griffin, Martin Price
{"title":"Indirect Treatment Comparisons in EUnetHTA Relative Effectiveness Assessments: Learnings and Recommendations for the Implementation of EU Joint Clinical Assessments.","authors":"Sophie van Beekhuizen, Menglu Che, Loraine Monfort, Mahmoud Hashim, Ali Azough, Nicole Kubitz, Adrian Griffin, Martin Price","doi":"10.1007/s41669-025-00575-1","DOIUrl":"10.1007/s41669-025-00575-1","url":null,"abstract":"<p><strong>Background: </strong>Beginning in January 2025, all new active substances must undergo evaluation of relative clinical effectiveness through European Union (EU) joint clinical assessments (JCAs). In the absence of head-to-head data, indirect treatment comparisons (ITCs) become indispensable in meeting the numerous population, intervention, comparators and outcomes (PICO) criteria to support decision-making.</p><p><strong>Objective: </strong>This study examined ITCs in European Network for Health Technology Assessment (EUnetHTA) relative effectiveness assessments (REAs) to obtain valuable insights into their potential implications for future JCAs.</p><p><strong>Methods: </strong>The EUnetHTA website was hand-searched for REAs of pharmaceutical products between 2010 and 2021. Information on PICO, ITC methods, ITC limitations/critiques, and relative effectiveness conclusions were systematically extracted. On the basis of the final EUnetHTA critiques, suitability of ITC evidence was categorised by the current study authors as appropriate, unsuitable or unclear.</p><p><strong>Results: </strong>Twenty-three REAs were identified. Twelve REAs included an ITC, of which six were in oncology indications. Across the REAs, 64 comparisons were required, with a median of four comparators per REA (range 1-18). In total, 25 comparisons were informed by indirect evidence; the suitability of ITCs was categorised as unclear in all but one of the 25 comparisons.</p><p><strong>Conclusion: </strong>Multiple analyses and ITCs were necessary to address multiple PICOs. Although most ITCs were categorised as unclear within the REAs, they were still considered appropriate to inform decision-making. The EU JCA process will most likely require health technology developers to use various ITC approaches to address the multiple PICOs requested, recognising the inherent limitations of these methodologies. Efforts to address potential challenges for EU JCA should focus on supporting JCA assessors/co-assessors and national HTA agencies in the evaluation and interpretation of ITCs to enable decision-making.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"597-609"},"PeriodicalIF":2.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12209061/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144009731","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2025-07-01Epub Date: 2025-04-15DOI: 10.1007/s41669-025-00576-0
Caleb Dixon, Thomas Price, Elin Gruffydd, Andrew Lloyd, Ernest H Law
{"title":"Patient Validation of Estimation of Health Utility Values in Alopecia Areata.","authors":"Caleb Dixon, Thomas Price, Elin Gruffydd, Andrew Lloyd, Ernest H Law","doi":"10.1007/s41669-025-00576-0","DOIUrl":"10.1007/s41669-025-00576-0","url":null,"abstract":"<p><strong>Background: </strong>Current patient-reported burden measures, which guide decision-making, may not be sensitive to pertinent impacts of alopecia areata (AA). The UK public previously valued patient/caregiver vignettes using the time trade-off (TTO) method in interviews; however, the public may not fully understand the impact of AA. Therefore, this study aimed to evaluate the content validity of the vignettes using TTO interviews with patients with AA and to compare patient-derived utilities for each health state with the utilities derived from the UK public.</p><p><strong>Methods: </strong>To understand their perspective surrounding trade-offs between quantity and quality of life, and to confirm the UK public's utility values, adult participants in the UK with AA currently experiencing scalp hair loss or with no scalp hair loss but experiencing hair loss in the last 12 months were recruited. In total, five AA vignettes that described health states by hair loss and its impacts and one caregiver vignette describing caring for an adolescent patient (aged 12-17 years) with AA were presented to participants in interviewer-guided sessions. Participants imagined themselves in the health state or as a caregiver, then valued each state using a visual analog scale (VAS) and traded time from 10 years in full health in 6-month increments to avoid living in each health state to evaluate the TTO utilities (0 [dead] to 1 [full health]).</p><p><strong>Results: </strong>Among participants (N = 50), mean VAS scores (Severity of Alopecia Tool [SALT] 0-10: 74.3; SALT 11-20: 59.0; SALT 21-49: 39.4; SALT 50-100: 29.6; SALT 50-100 [no eyebrows/eyelashes]: 26.0; and caregiver [adolescent SALT 50-100]: 58.6) and TTO utility values (SALT 0-10: 0.93; SALT 11-20: 0.87; SALT 21-49: 0.67; SALT 50-100: 0.54; SALT 50-100 [no eyebrows/eyelashes]: 0.45; and caregiver [adolescent SALT 50-100]: 0.81) were lower for states with greater hair loss, similar to the mean UK public utility values. Over a 10-year time horizon, the average patient with AA was willing to trade approximately 5 years of life (mean TTO utility, 0.537 [standard deviation, 0.448]) to avoid impacts associated with 50-100% scalp hair loss.</p><p><strong>Conclusions: </strong>This is the first known study to evaluate how patients with AA would make trade-offs between quantity of life and disease-specific quality of life. A high willingness to trade off duration of life was observed, consistent with the UK public study and supporting a high disease burden of AA.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"683-692"},"PeriodicalIF":2.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12209119/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144027804","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2025-07-01Epub Date: 2025-05-12DOI: 10.1007/s41669-025-00581-3
M Senni, E Paoletti, Ewa Stawowczyk, M Hale, A Ramirez de Arellano
{"title":"Economic Evaluation of Patiromer in Patients with Concomitant Heart Failure and Chronic Kidney Disease in Italy.","authors":"M Senni, E Paoletti, Ewa Stawowczyk, M Hale, A Ramirez de Arellano","doi":"10.1007/s41669-025-00581-3","DOIUrl":"10.1007/s41669-025-00581-3","url":null,"abstract":"<p><strong>Background: </strong>Hyperkalaemia (HK), in patients with heart failure (HF) with and without chronic kidney disease (CKD), is potentially life-threatening. Risk of HK is further heightened in those patients receiving renin-angiotensin-aldosterone system inhibitors (RAASi), used to reduce cardiovascular morbidity and mortality in HF. Patiromer, an oral potassium (K<sup>+</sup>) binder, has been shown to reduce the risk of HK and enable optimal RAASi dosing. We evaluated the cost-effectiveness of patiromer in HF patients with CKD in the Italian setting, utilising results from the recent DIAMOND clinical trial, which assessed long-term use of patiromer in HK management.</p><p><strong>Methods: </strong>An established Markov model was adapted to include data from DIAMOND using the National Health Service (NHS) perspective. In DIAMOND, patients received patiromer during a run-in period (up to 12 weeks) to achieve optimal RAASi without HK. However, this led to low mean K<sup>+</sup> concentrations in the placebo arm, resulting from a legacy effect of patiromer in the run-in phase of the trial. Therefore, the DIAMOND population was adjusted to a real-world population to better represent the K<sup>+</sup> levels in the standard of care (SoC) arm. Mean K<sup>+</sup> concentration for baseline and the patiromer arm was calculated from the overall population at baseline (screening phase) and after treatment (end of run-in period), respectively. Lifetime trajectories were estimated for quality-adjusted life years (QALYs), life years (LYs) and costs.</p><p><strong>Results: </strong>The economic evaluation model calculated a discounted total average cost per patient of €109,900 for patiromer and €64,847 for SoC. Patiromer generated a gain of 1.97 LYs (1.55 QALYs) compared with SoC. The incremental cost-effectiveness ratio (ICER) for patiromer was €29,060/QALY gained versus SoC.</p><p><strong>Conclusion: </strong>Applying DIAMOND data, patiromer is deemed to be cost-effective at a willingness-to pay threshold of €40,000 per QALY gained in Italy.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"693-706"},"PeriodicalIF":2.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12209094/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144022842","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2025-07-01Epub Date: 2025-03-19DOI: 10.1007/s41669-025-00573-3
Sheryl Hui-Xian Ng, Palvinder Kaur, Laurence Lean Chin Tan, Mervyn Yong Hwang Koh, Andy Hau Yan Ho, Allyn Hum, Woan Shin Tan
{"title":"Healthcare Expenditure Trajectories in the Last 5 Years of Life: A Retrospective Cohort Study of Decedents with Advanced Cancer and End-Stage Organ Diseases.","authors":"Sheryl Hui-Xian Ng, Palvinder Kaur, Laurence Lean Chin Tan, Mervyn Yong Hwang Koh, Andy Hau Yan Ho, Allyn Hum, Woan Shin Tan","doi":"10.1007/s41669-025-00573-3","DOIUrl":"10.1007/s41669-025-00573-3","url":null,"abstract":"<p><strong>Background: </strong>Patients with nonmalignant end-stage organ diseases often incur healthcare costs in the last year of life that are disproportionately higher than in the period prior. Studies on healthcare expenditure (HCE) trends in these patients have largely focused on the final year of life, and examining a longer-term trajectory could better support healthcare professionals to target the timing and methods of care management. In this study, we aim to describe the HCE trajectories of end-stage organ disease (ESOD) over the last 5 years of life, compared against advanced cancer (AC).</p><p><strong>Methods: </strong>We conducted a retrospective cohort study to profile decedents who had either a primary or secondary diagnosis of AC, advanced dementia, severe liver disease, as well as heart failure (HF), end-stage renal failure (ESRF), or respiratory failure (RF) in the last 5 years of their lives using a regional health system database in Singapore. Hospital-based HCE and utilization for each diagnosis group was reported cumulatively for 5 years, by year and by month. The proportion of the 5-year HCE incurred in each year was also reported.</p><p><strong>Results: </strong>Across all conditions, monthly HCE started to increase rapidly around 3 years prior to death, with 80% of the 5-year expenditure incurred in the same period. Expenditure among patients with ESODs other than dementia was £12,787 to £21,019 higher in comparison with patients with AC. Patients with RF incurred the highest 5-year HCE, driven by inpatient admissions.</p><p><strong>Conclusions: </strong>Our findings highlight the importance of examining HCE trends in ESOD and their cost drivers over multiple years prior to the last year of life to inform healthcare policy and to review care processes to ensure appropriate and efficient end-of-life (EOL) care.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"661-670"},"PeriodicalIF":2.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12209486/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664067","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Optimizing Diabetic Retinopathy Screening at Primary Health Centres in India: A Cost-Effectiveness Analysis.","authors":"Neha Purohit, Parul Chawla Gupta, Sandeep Buttan, Akashdeep Singh Chauhan, Ranjan Kumar Choudhury, Vishali Gupta, Atul Kotwal, Shankar Prinja","doi":"10.1007/s41669-025-00572-4","DOIUrl":"10.1007/s41669-025-00572-4","url":null,"abstract":"<p><strong>Background: </strong>The eye care package under the Ayushman Bharat comprehensive primary healthcare programme includes annual population-based screening for diabetic retinopathy (DR) using non-mydriatic fundus cameras at the primary health centres (PHCs) in India. However, there can be several implementation models for introduction of a systematic screening programme for DR.</p><p><strong>Objectives: </strong>This study aims to assess the cost effectiveness of screening for DR in comparison with the usual-care scenario without a DR screening programme, and to determine cost-effective approaches for implementation of annual population-based screening for DR by optometrists at PHCs in India in terms of screening modalities (face-to-face vs tele-supported screening [screening followed by transfer and remote grading of images by ophthalmologists] vs artificial intelligence [AI]-supported screening) and target population groups for screening.</p><p><strong>Methods: </strong>A mathematical model comprising a decision tree and Markov model was developed. An extensive review of published literature was undertaken to obtain model parameters. Primary data collection was done to derive quality-of-life values. We used a lifetime horizon, abridged societal perspective, and discounted future costs and consequences at an annual rate of 3%. The incremental cost-effectiveness ratio (ICER) was computed for alternative screening strategies. A willingness-to-pay equal to gross domestic product per capita equal to ₹171,498 (US$2182) was used to determine the cost-effective choice. Sensitivity analyses were performed to assess the impact of variation in input parameters on the ICER values.</p><p><strong>Results: </strong>All the annual screening strategies were found to have lower ICERs relative to usual care. Among the screening strategies, annual tele-supported screening in the population with diabetes duration ≥5 years was the most cost-effective strategy with an ICER value of ₹57,408 (US$730) per quality-adjusted life year (QALY) gained. At the national level, this strategy is likely to reduce the annual incidence of vision-threatening DR and blindness by 17.3%, and 38.5%, respectively, and would result in higher benefits in Indian states with higher epidemiological transition. Sensitivity analyses showed that if adequate glycaemic control is achieved in 79% of the diabetic population, annual AI-supported screening in individuals with a diabetes' duration of 10 years or more becomes the most cost-effective strategy.</p><p><strong>Conclusion: </strong>The results of the study suggest the prioritization of an annual tele-supported DR screening programme in India. They also highlight the importance of the adoption of an integrated approach and functional linkage between eye care and diabetes care, to intensify efforts directed at improving glycaemic control, and to facilitate early DR detection and management.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"627-638"},"PeriodicalIF":2.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12209073/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144005085","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Assessing the Value for Money of Enzyme Replacement Therapy in Gaucher Disease Types 1 and 3b: Can Expanded Coverage Be Justified?","authors":"Waranya Rattanavipapong, Thunyarat Anothaisintawee, Wanrudee Isaranuwatchai, Duangrurdee Wattanasirichaigoon, Thipwimol Tim-Aroon, Khunton Wichajarn, Achara Sathienkijkanchai, Pimlak Charoenkwan, Kanya Suphapeetiporn, Chanchai Traivaree, Chulaluck Kuptanon, Yot Teerawattananon","doi":"10.1007/s41669-025-00579-x","DOIUrl":"10.1007/s41669-025-00579-x","url":null,"abstract":"<p><strong>Background and objectives: </strong>The Health Intervention and Technology Assessment Program was commissioned to conduct a cost-utility and budget impact analysis of enzyme replacement therapy (ERT) for Gaucher disease types 1 and 3b. The findings from this assessment are to support the decision-making process regarding the potential expansion of ERT coverage within Thailand's public health system.</p><p><strong>Methods: </strong>The analysis compared the current policy, which provides treatment with imiglucerase only for patients with Gaucher disease type 1, as listed in the National List of Essential Medicine, with a proposed policy that extends coverage to include Gaucher disease types 1 and 3b with either imiglucerase or velaglucerase. Cost-utility analysis of these policy options was performed using decision tree and Markov models over a lifetime horizon from a societal perspective. The financial implications for the relevant budgetary authority over 5 years were estimated. The research methodology adheres rigorously to Thailand's health technology assessment guidelines.</p><p><strong>Results: </strong>The study found that the incremental cost-effectiveness ratios for treating both Gaucher disease types 1 and 3b are 6,769,000 and 9,359,000 baht per quality-adjusted life year (QALY) for imiglucerase and velaglucerase, respectively, which is well beyond Thailand's cost-effectiveness threshold of 160,000 baht per QALY. Such an expansion would incur an additional budgetary burden of approximately 81 million baht for imiglucerase and 138 million baht for velaglucerase. Increasing the rate of hematopoietic stem cell transplantation (HSCT) can improve the cost-effectiveness of the expansion.</p><p><strong>Conclusions: </strong>The study concludes that expanding ERT with either imiglucerase or velaglucerase to treat both Gaucher disease types 1 and 3b is not cost-effective at current prices in Thailand; however, it could become cost-effective with a reduction of approximately 60% in drug prices or if all eligible patients undergo HSCT.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"611-626"},"PeriodicalIF":2.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12209059/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144037695","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2025-07-01Epub Date: 2025-04-09DOI: 10.1007/s41669-025-00569-z
Carlos Chivardi, Alejandro Zamudio-Sosa, Marta Wilson-Barthes, Fernando Alarid-Escudero, Monica Gandhi, Kenneth H Mayer, Don Operario, Omar Galárraga
{"title":"Cost-Effectiveness of Using Conditional Economic Incentives to Improve Pre-exposure Prophylaxis Adherence Among Male Sex Workers.","authors":"Carlos Chivardi, Alejandro Zamudio-Sosa, Marta Wilson-Barthes, Fernando Alarid-Escudero, Monica Gandhi, Kenneth H Mayer, Don Operario, Omar Galárraga","doi":"10.1007/s41669-025-00569-z","DOIUrl":"10.1007/s41669-025-00569-z","url":null,"abstract":"<p><strong>Introduction: </strong>Conditional economic incentives can improve medication-taking behaviors among populations at risk of contracting human immunodeficiency virus (HIV). However, there are no data on the cost-effectiveness of incentive programs for improving pre-exposure prophylaxis (PrEP) adherence among male sex workers (MSWs) who have one of the highest HIV acquisition rates. Our objective was to assess the cost-effectiveness of incentive programs to improve adherence to pre-exposure prophylaxis (PrEP) among male sex workers METHODS: We conducted an economic evaluation of the PrEP Seguro randomized pilot trial in Mexico (ClinicalTrials.gov: NCT03674983). Among n = 110 MSWs, those randomized to the intervention received tiered incentives based on PrEP drug levels in scalp hair measured at three clinic visits over 6 months. The intervention led to a 28.7% increase in scalp hair PrEP concentration, consistent with increased adherence (p = 0.05). Here we use a micro-costing approach from the health system perspective to calculate costs. Quality-adjusted life years (QALYs) were estimated from the number of HIV infections averted through sufficient PrEP adherence (tenofovir concentration > 0.011 ng/mg corresponding to greater than or equal to three weekly doses). Incremental cost-effectiveness ratios (ICERs) estimated the cost/QALY gained owing to the intervention.</p><p><strong>Results: </strong>The mean cost per patient was US $165.53 and $179.55 among standard care and incentive patients, respectively. Over 6 months of follow-up, 62% of standard care patients and 78% of incentive recipients were PrEP adherent. After the program, the lifetime average QALYs gained per infection avoided were 9.17 (minimum, maximum: 7.5, 10.8) and 9.84 (minimum, maximum: 8.05, 11.6) among standard care and incentive patients, respectively. The 6-month ICER was US $20.92/QALY gained by the intervention, which was highly cost-effective at a willingness-to-pay of US $8655 (Mexico's 2020 per capita gross domestic product (GDP)).</p><p><strong>Discussion: </strong>Using behavioral economics approaches for enhancing adherence to HIV prevention may offer health and fiscal benefits through reduced HIV incidence. Fully powered implementation trials can determine future cost-effectiveness of scaling up incentives for PrEP adherence among high-risk populations.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"649-659"},"PeriodicalIF":2.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12209064/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144041152","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Hiroaki Mamiya, Kittima Wattanakamolkul, Nan Li, David Bin-Chia Wu, Mariko Hirozane, Ataru Igarashi
{"title":"Including Productivity as an Element to Reflect Value of the Treatment: A Systematic Review of Published Health Economic Evaluations.","authors":"Hiroaki Mamiya, Kittima Wattanakamolkul, Nan Li, David Bin-Chia Wu, Mariko Hirozane, Ataru Igarashi","doi":"10.1007/s41669-025-00589-9","DOIUrl":"https://doi.org/10.1007/s41669-025-00589-9","url":null,"abstract":"<p><strong>Background: </strong>Productivity is utilised inconsistently in value assessment and poses methodological and conceptual challenges for estimation. This systematic literature review aims to understand how productivity gain/loss for patients and caregivers was measured in health economic evaluation, identify the preferred instruments to capture productivity gain/loss and assess their properties.</p><p><strong>Methods: </strong>A systematic literature review was conducted using PubMed, Ovid MEDLINE, Embase, Japan-specific (J-STAGE and Ichushi Web) and economic literature databases (Cost-Effectiveness Analysis (CEA) Registry, Paediatric Economic Database Evaluation (PEDE) and National Health Service Economic Evaluation Database (NHS EED)). Additional relevant data were identified using clinical trial registries and grey literature searches. Studies published between January 2021 and December 2023 which presented economic evaluations with outcomes relevant to productivity measures were included. Instruments were selected for practical relevance and frequent use in studies, while research-specific and single-study questionnaires were excluded.</p><p><strong>Results: </strong>Among 1431 published health economic evaluations with productivity in the assessment, 152 were included in this review. These studies were almost equally distributed over the 3-year period, with a slightly higher number in 2023 versus 2021 [54 (35.5%) versus 48 (31.6%)]. In total, 118/152 studies reported patient measures only, 14/152 reported caregiver measures only and 20/152 reported both patient and caregiver measures. Based on the inclusion criteria, 20 of the 88 identified instruments were further investigated in this review. The Work Productivity and Activity Impairment Questionnaire (WPAI) [23 (15.1%)], Institute for Medical Technology Assessment (iMTA) Productivity Cost Questionnaire (iPCQ) [23 (15.1%)] and Trimbos/iMTA Questionnaire for Costs associated with Psychiatric Illness (TIC-P) [(8 (5.3%)] were the most commonly used instruments. All three instruments were reported as valid and reliable. All three instruments reported absenteeism, presenteeism and overall work impairment. WPAI additionally included information on daily activity impairment. The human capital approach (n = 18) and the friction cost approach (n = 17) were utilised to assign monetary value to the lost productivity.</p><p><strong>Conclusions: </strong>This review identified a significant number of health economic evaluations between 2021 and 2023, which included productivity loss in the assessment. Various instruments were used to measure productivity loss in these studies, while a few instruments were commonly used across studies. To ensure consistent measurement of productivity, it is crucial to establish a standard tailored to a country or region, considering differences across countries or regions. Furthermore, necessary adaptations should be made on the basis of the specific health in","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-06-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144476163","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Suzanne J Baron, Serge Korjian, C Michael Gibson, Matthew R Reynolds
{"title":"Cost-Effectiveness of the CADScor System in Low-Risk Patients Presenting to the Emergency Department with Chest Pain.","authors":"Suzanne J Baron, Serge Korjian, C Michael Gibson, Matthew R Reynolds","doi":"10.1007/s41669-025-00590-2","DOIUrl":"https://doi.org/10.1007/s41669-025-00590-2","url":null,"abstract":"<p><strong>Background: </strong>Studies have shown that up to 13% of patients presenting to the emergency department (ED) with low-risk chest pain receive further cardiac testing beyond an electrocardiogram and serial cardiac enzymes. The CADScor System is a US Food and Drug Administration (FDA)-cleared device that uses ultra-sensitive phonocardiography to evaluate the risk of significant coronary artery disease (CAD).</p><p><strong>Objective: </strong>To evaluate the relative cost-effectiveness of the CADScor system compared with other diagnostic modalities for the evaluation of low-risk chest pain in patients presenting to the ED in the USA.</p><p><strong>Methods: </strong>A two-part economic model, consisting of a decision analytic tree followed by a short-term Markov model, was developed to compare 1-year costs and outcomes (e.g., quality-adjusted life year [QALY], false-negatives, and related cardiac events) associated with the CADScor System as the first-line test versus other noninvasive cardiac tests from the perspective of the US healthcare system (TreeAge Pro). Model inputs were derived from literature, and costs for treatment strategies were calculated from average US reimbursements associated with Current Procedural Terminology and Medicare Severity Diagnosis Related Group codes. Values were reported in 2023 US dollars (USD). Parameter uncertainty was assessed through a series of deterministic and one-way sensitivity analyses.</p><p><strong>Results: </strong>Base case results demonstrated that a CADScor-First strategy was cost saving compared with other noninvasive cardiac tests without a substantial difference in adverse events. Economic results were consistent when coronary artery disease (CAD) prevalence rates were varied from 2 to 30% and across a variety of sensitivity analyses. The overall cost savings were estimated to be $7.3-15.3 million USD per 10,000 patients with low-risk chest pain.</p><p><strong>Conclusions: </strong>Use of a CADScor-First strategy in the evaluation of patients with low-risk chest pain presenting to the ED may result in substantial cost savings for the US healthcare system. More research is needed to understand the long-term costs and outcomes of this strategy.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144476162","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Renée J G Arnold, Winona Tse, Kimberly Martin, Renee Kuan
{"title":"Real-World Analysis of Healthcare Utilization, Treatment Patterns, and Economic Burden in Medicare Beneficiaries with Parkinson's Disease: Implications by Levodopa Formulation and Disease Severity.","authors":"Renée J G Arnold, Winona Tse, Kimberly Martin, Renee Kuan","doi":"10.1007/s41669-025-00588-w","DOIUrl":"https://doi.org/10.1007/s41669-025-00588-w","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to evaluate healthcare utilization, costs, and treatment patterns of Medicare beneficiaries with Parkinson's disease (PD) treated with different carbidopa-levodopa regimens.</p><p><strong>Methods: </strong>A retrospective cohort study was conducted using 100% fee-for-service Medicare research identifiable claims data from 2017 to 2019. The study population included 201,241 Medicare beneficiaries aged 65-90 years with PD who received at least one prescription for a carbidopa-levodopa-containing regimen. Treatment patterns, healthcare resource utilization, and costs were analyzed, stratified by medication regimen containing levodopa and levodopa equivalent daily dose (LEDD), the latter as a proxy for disease severity.</p><p><strong>Results: </strong>Immediate release (IR) carbidopa-levodopa was the most common initial prescription (83%). Extended release (ER) formulations had the highest mean daily dose (1140 mg, although the equivalent dose in a non-ER formulation is approximately 570 mg, in line with the other three primary regimens). Treatment persistence and cost generally increased with higher LEDD. Concomitant medication use, particularly dopamine agonists, also increased with higher LEDD. Total 3-year per patient healthcare costs were lower for patients prescribed controlled release (CR) carbidopa-levodopa (US $98,650); compared with US $116,394 for patients prescribed IR carbidopa-levodopa; US $123,650 for those prescribed CR + IR carbidopa-levodopa; and US $125,802 for ER carbidopa-levodopa. Costs tended to increase with higher LEDD, primarily driven by outpatient care and medications.</p><p><strong>Conclusions: </strong>This study provides comprehensive real-world evidence on carbidopa-levodopa use in Medicare beneficiaries with PD. Findings highlight the need for individualized treatment approaches, considering both symptom control and healthcare costs. Future research should focus on prospective studies to assess long-term outcomes and economic impact of different treatment strategies in PD, considering disease severity and quality of life.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144310227","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}