PharmacoEconomics Open最新文献

{"title":"Integrating Multi-Cancer Early Detection (MCED) Tests with Standard Cancer Screening: System Dynamics Model Development and Feasibility Testing.","authors":"Mussab Fagery, Hadi A Khorshidi, Stephen Q Wong, Özge Karanfil, Jon Emery, Maarten J IJzerman","doi":"10.1007/s41669-024-00533-3","DOIUrl":"https://doi.org/10.1007/s41669-024-00533-3","url":null,"abstract":"<p><strong>Background: </strong>Cancer screening plays a critical role in early disease detection and improving outcomes. In Australia, established screening protocols for colorectal, breast and cervical cancer have significantly contributed to timely cancer detection. However, the recent introduction of multi-cancer early detection (MCED) tests arguably can disrupt current screening, yet the extent to which these tests provide additional benefits remains uncertain. We present the development and initial validation of a system dynamics (SD) model that estimates the additional cancer detections and costs associated with MCED tests.</p><p><strong>Aim: </strong>This article describes the development of a simulation model built to evaluate the additional patient diagnoses and the economic impact of incorporating MCED testing alongside Australia's well-established standard of care (SOC) screening programs for colorectal, breast, cervical and lung cancers. The model was designed to estimate the additional number of patients diagnosed at each cancer stage (stage I, II, III, IV, or unknown) and the associated costs. This simulation model allows for the analysis of multiple scenarios under a plausible set of assumptions regarding population-level participation rates.</p><p><strong>Methods: </strong>An SD model was developed to represent the existing SOC national cancer screening pathways and to integrate potential clinical pathways that could be introduced by MCED tests. The SD model was built to investigate three scenarios for the use of MCED testing: firstly, to explore the viability of MCED testing as a substitute among individuals who are not opting for SOC screening for any reason; secondly, to implement MCED testing exclusively for individuals ineligible for SOC screening, yet have high-risk characteristics; and thirdly, to employ MCED testing after SOC screening to serve as a triaging/confirmatory tool for individuals receiving inconclusive test results. The three primary scenarios were constructed by varying diagnostic accuracy and uptake rates of MCED tests.</p><p><strong>Discussion: </strong>The clinical utility and outcomes of MCED testing for screening and early detection still lack comprehensive evidence. Nonetheless, this simulation model facilitates a thorough analysis of MCED tests within the Australian healthcare context, providing insights into potential additional detections and costs to the healthcare system, which may help prioritise future evidence development. The adaptable yet novel SD model presented herein is anticipated to be of considerable interest to industry, policymakers, consumers and clinicians involved in informing clinical and economic decisions regarding integrating MCED tests as cancer screening and early detection tools. The expected results of applying this SD model will determine whether using MCED testing in conjunction with SOC screening offers any potential benefits, possibly guiding policy decisions and clinica","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142471895","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}

{"title":"Costs of Illness for Huntington's Disease: A Systematic Review.","authors":"Divya Patil, Emily F Gorman, T Joseph Mattingly","doi":"10.1007/s41669-024-00531-5","DOIUrl":"https://doi.org/10.1007/s41669-024-00531-5","url":null,"abstract":"<p><strong>Background: </strong>Huntington's disease (HD) is associated with significant financial burden for patients and payers. The objective was to identify and quantify costs of HD by stage of disease progression.</p><p><strong>Methods: </strong>A systematic search of Medline, Embase, Scopus, and Cochrane Library was used to identify types of costs that are frequently reported for individuals diagnosed with HD and to quantify those costs across early, middle, and late stages of HD. Full-text, original research articles were included if they reported costs specific to HD burden. To standardize stage-level costs, Shoulson Fahn and Barthel Index scores were combined to estimate the cost for early, middle-, and late-stage HD.</p><p><strong>Results: </strong>A total of 692 abstracts were identifie,d and following abstract screening, a total of 80 full-text articles were reviewed for inclusion. Only five studies were included for extraction and synthesis including three from the USA, one from the United Kingdom (UK), and one from Peru. Annual inpatient, outpatient, drug costs, and caregiving costs all increased substantially as disease progressed. Outpatient costs were approximately 2.5 times greater than inpatient costs for early and middle stages of HD. Among all the costs associated with HD, annual caregiver cost emerges as the most significant costs in the economic burden of HD, ranging from a minimum of $6041 for early stage to a maximum of $133,200 for late-stage HD. Significant variation was observed across studies, especially comparing costs observed in Peru with the USA and UK.</p><p><strong>Conclusion: </strong>Outpatient costs exceed inpatient costs, especially in early and middle stages, underscoring the importance of outpatient care. All costs seem to rise rapidly, in a nonlinear fashion, as patients advance to later stages. While only two studies reported caregiver burden, these costs were significanly higher in the most severe stage, where patients were completely dependent on a caregiver. This review highlights the complexity of cost assessment in HD and underscores the need for consistent methods and further research to guide effective policy actions.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142471894","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}

{"title":"Cost Effectiveness of Sequencing Vedolizumab as First-Line Biologic in Ulcerative Colitis and Crohn's Disease in Canada: An Analysis Using Real-World Evidence from the EVOLVE Study.","authors":"Aren Fischer, Stephen Mac, Erica Stivelman Freiman, John K Marshall, Kim Rand, Juan M Ramos-Goñi","doi":"10.1007/s41669-024-00523-5","DOIUrl":"https://doi.org/10.1007/s41669-024-00523-5","url":null,"abstract":"<p><strong>Introduction: </strong>Vedolizumab is a gut-selective anti-lymphocyte trafficking biologic indicated for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) and Crohn's disease (CD) in Canada.</p><p><strong>Objective: </strong>The objective of this study was to evaluate the cost effectiveness of treatment sequencing for UC and CD from a public healthcare payer perspective, leveraging new real-world evidence from the literature and the EVOLVE study, a retrospective chart review.</p><p><strong>Methods: </strong>Using separate decision tree/Markov models to assess cost effectiveness for UC and CD, two sequencing approaches were estimated for adult patients (≥ 18 years) diagnosed with UC or CD who were biologic-naïve: vedolizumab as first-line biologic followed by anti-tumor necrosis factor (TNF)-α versus first-line anti-TNFα followed by vedolizumab. Treatment effectiveness (response and remission), surgery rates, dose escalation and regain of response and safety inputs were estimated from EVOLVE, a retrospective chart review of real-world data, and evidence synthesis from the literature, whereas costs and utilities were estimated from health technology assessment reports, clinical trials, and the literature. Biosimilar costs were used for anti-TNFα. Both models simulated a 5-year time horizon and discounted costs and outcomes at 1.5%. Probabilistic base-case analyses (n = 10,000) reported total costs (2023 Canadian dollars) and quality-adjusted life-years (QALYs). Several scenario analyses were conducted to explore robustness of results.</p><p><strong>Results: </strong>In UC, vedolizumab as a first-line biologic followed by anti-TNFα resulted in an incremental gain of 0.09 QALYs (2.46 vs. 2.55) and saved $7179 ($134,028 vs. $126,848), making this a dominant strategy compared with first-line anti-TNFα followed by vedolizumab. In CD, use of vedolizumab as a first-line biologic resulted in an incremental gain of 0.04 QALYs (3.35 vs. 3.39) at an incremental cost of $50,631 ($89,850 vs. $140,381) versus first-line anti-TNFα followed by vedolizumab (incremental cost-effectiveness ratio of $1,265,775 per QALY).</p><p><strong>Conclusions: </strong>Based on this analysis, sequencing vedolizumab as a first-line biologic prior to anti-TNFα in UC and CD provided additional clinical benefit to patients. In UC, vedolizumab as a first-line biologic also saved healthcare system costs compared with anti-TNFα, whereas in CD, vedolizumab provided incremental benefit at an incremental cost, which was not considered cost effective at a threshold of $50,000/QALY.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142392323","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}

{"title":"Cost-Effectiveness of Fruquintinib for Refractory Metastatic Colorectal Cancer in the USA.","authors":"Dong-Won Kang, Patricio B Lynn, Li Wang, Shouhao Zhou, Chan Shen","doi":"10.1007/s41669-024-00529-z","DOIUrl":"https://doi.org/10.1007/s41669-024-00529-z","url":null,"abstract":"<p><strong>Background: </strong>The FRESCO-2 trial established the efficacy and safety of fruquintinib in patients with refractory metastatic colorectal cancer. However, its cost-effectiveness in the US context is not well documented.</p><p><strong>Objective: </strong>This study evaluates the cost-effectiveness of fruquintinib versus placebo for this patient population from the perspective of US payers.</p><p><strong>Methods: </strong>We developed a partitioned survival model on the basis of patient-level data reconstructed from the survival curves of the FRESCO-2 trial. Parametric estimation was conducted to estimate long-term clinical outcomes and medical costs over a lifetime horizon. Cost inputs and utilities were sourced from public data and previous literature. We used a discount rate of 3.0% per year for both clinical outcomes and costs. We adopted an incremental cost-effectiveness ratio (ICER) threshold of US$100,000 per quality-adjusted life-year (QALY) gained. We performed sensitivity and scenario analyses to examine the robustness of cost-effectiveness results.</p><p><strong>Results: </strong>Fruquintinib treatment resulted in incremental gains of 0.108 life years (LYs) and 0.073 QALYs compared with the placebo, at an additional cost of US$112,294, primarily driven by medication expenses. The ICER for fruquintinib versus placebo was calculated at US$1,037,855 per LY and US$1,546,619 per QALY gained, exceeding the predefined cost-effectiveness threshold. The cost-effectiveness results were robust across all sensitivity and scenario analyses.</p><p><strong>Conclusion and relevance: </strong>Despite the survival benefit, fruquintinib was not cost-effective compared with the placebo in patients with refractory metastatic colorectal cancer in the US setting, on the basis of the conventional willingness-to-pay threshold. Our findings may provide a basis for informing the pricing and reimbursement decisions regarding fruquintinib.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142392324","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}

{"title":"Cost-Utility and Budget Impact Analysis of Adding SGLT-2 Inhibitors to Standard Treatment in Type 2 Diabetes Patients with Heart Failure: Utilizing National Database Insights from Thailand.","authors":"Tanawan Kongmalai, Juthamas Prawjaeng, Phorntida Hadnorntun, Pattara Leelahavarong, Usa Chaikledkaew, Ammarin Thakkinstian, Varalak Srinonprasert","doi":"10.1007/s41669-024-00526-2","DOIUrl":"https://doi.org/10.1007/s41669-024-00526-2","url":null,"abstract":"<p><strong>Background: </strong>Heart failure (HF) in type 2 diabetes (T2D) patients poses a significant clinical and financial burden. While sodium-glucose cotransporter-2 inhibitors (SGLT2i) have shown cardiovascular benefits in trials, they are not included in Thailand's National List of Essential Medicines (NLEM), and their value-for-money remains unassessed.</p><p><strong>Objective: </strong>This study aims to evaluate the cost-utility of adding SGLT2i to the standard treatment for T2D-HF patients in Thailand.</p><p><strong>Methods: </strong>A Markov model with 3-month cycles and a lifetime horizon was conducted from a societal perspective. Efficacy data came from a systematic review and meta-analysis. Transition probabilities and direct medical costs were derived from the National Health Security Office database, while direct non-medical costs and utility were collected through patient interviews at Siriraj hospital to reflect Thailand's context. The main outcomes were incremental costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER). A sensitivity and budget impact analysis were also performed.</p><p><strong>Results: </strong>Canagliflozin led to the highest increase in QALYs, at 1.21 years, followed by dapagliflozin (0.54 years) and empagliflozin (0.06 years). Collectively, SGLT2i yielded an increase of 0.41 QALYs. Canagliflozin incurred the highest additional treatment cost at United States dollars (US$)5600, followed by dapagliflozin (US$3547) and empagliflozin (US$2694). The ICERs for canagliflozin, dapagliflozin, empagliflozin, and overall SGLT2i were US$4632, US$6430, US$48,952, and US$8480 per QALY gained, respectively. SGLT2i were not cost-effective compared with Thailand's willingness-to-pay threshold of US$4564 per QALY gained. However, threshold analysis indicates that the costs of canagliflozin, dapagliflozin, empagliflozin, and overall SGLT-2i should be reduced by 2.3%, 38.2%, 90.2%, and 55.6%, respectively, to be cost-effective. Budget impact analysis revealed that the total budget for treating T2D patients with HF over 5 years is US$15.6 million.</p><p><strong>Conclusions: </strong>Adding SGLT2i to standard treatment reduced HF hospitalization and mortality rates and improved QALYs in T2D-HF patients. Nevertheless, they would not be cost-effective at current prices in Thailand.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142292818","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}

{"title":"Estimation of Health State Utility Values for Immunoglobulin A Nephropathy: A Time Trade-Off Analysis.","authors":"Zheng-Yi Zhou, Mark E Bensink, Nisha C Hazra, Chunyi Xu, Bruce Hendry, Claire C Sharpe, Mo Zhou","doi":"10.1007/s41669-024-00527-1","DOIUrl":"https://doi.org/10.1007/s41669-024-00527-1","url":null,"abstract":"<p><strong>Background: </strong>Immunoglobulin A nephropathy (IgAN) is a rare progressive disease that can lead to kidney failure. The current study aimed to estimate health state utility values for IgAN from a UK societal perspective.</p><p><strong>Methods: </strong>We used the time trade-off (TTO) method to derive utility values for various health states in IgAN, defined based on chronic kidney disease (CKD) stage, proteinuria, dialysis, and nephrotic syndrome (CKD stages 1-4, proteinuria < 1 g/day vs ≥ 1 g/day; CKD stage 5, dialysis vs non-dialysis). We developed health state vignettes to describe typical symptoms and quality-of-life impairments of IgAN. Eligible participants from the UK general public completed a computer-assisted telephone interview. Estimated TTO utility values were reviewed against visual analogue scale (VAS)-derived values.</p><p><strong>Results: </strong>In total, 200 participants were included in the study (mean age, 48.9 years; female, 59.0%). Mean (standard deviation [SD]) utility values were 0.84 (0.17) and 0.71 (0.23) for CKD stage 1/2 with proteinuria < 1 g/day and with proteinuria ≥ 1 g/day, respectively; 0.68 (0.23) and 0.61 (0.25) for CKD stage 3; and 0.55 (0.26) and 0.49 (0.27) for CKD stage 4. Mean (SD) utility of CKD stage 5 with and without dialysis was 0.38 (0.30) and 0.42 (0.28), respectively. The mean (SD) utility value of nephrotic syndrome was 0.43 (0.33).</p><p><strong>Conclusions: </strong>Our results indicated that various IgAN health states are associated with impaired health status, with substantial utility decrements related to disease progression, elevated proteinuria, and nephrotic syndrome.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142292828","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}

{"title":"Healthcare Resource Utilization and Associated Costs During the First 5 Years After Diagnosis and at the End of Life: A Nationwide Cohort Study of Patients with Multiple Myeloma in Finland.","authors":"Mikko Kosunen, Jarno Ruotsalainen, Alvar Kallio, Roope Metsä, Paavo Raittinen, Leena Lehmus, Maarit J Korhonen, Timo Purmonen","doi":"10.1007/s41669-024-00524-4","DOIUrl":"https://doi.org/10.1007/s41669-024-00524-4","url":null,"abstract":"<p><strong>Background: </strong>The burden associated with the treatment of patients with multiple myeloma (MM) is expected to increase due to the aging population. Thus, policymakers and clinicians need a holistic view of the healthcare resource use (HCRU) and costs associated with MM and its treatment for informed decision making. However, nationwide information on HCRU and costs due to MM is scarce in Finland. The aim of this study was to determine healthcare resource utilization, patterns of service use and associated costs among Finnish patients with MM during the first 5 years from their first diagnosis and at end of life.</p><p><strong>Methods: </strong>Data on patients newly diagnosed with MM and receiving treatment for it in Finland in 2015-2019 was sourced from comprehensive nationwide registers. Data on all-cause and MM-specific HCRU including inpatient stays, outpatient visits and contacts, emergency care visits and home care were obtained separately from specialized and primary care registers. HCRU costs were assessed by multiplying the numbers of primary and specialized care contacts by respective national unit costs. For reimbursed outpatient medication and reimbursed sick leave, data on actual costs were collected. All registry data were linked via unique personal identifiers, and follow-up time was up to 5 years.</p><p><strong>Results: </strong>Altogether, 1615 patients were included in the analyses. In the 5-year follow-up period, patients had on average 96 healthcare contacts per patient-year (PPY) and the mean all-cause healthcare costs were €46,000 PPY. Around 47% of these costs originated from reimbursed outpatient medication and the rest from healthcare contacts. Over half (60%) of the contacts occurred in primary care but most of the costs were associated with specialized care. Additionally, 29% of contacts were MM-specific, but they were responsible for 58% of the costs. The HCRU was highest during the first year after diagnosis, levelled off during the follow-up and then increased significantly during the last year of patients' lives. The number of all-cause healthcare contacts PPY was approximately 53% higher, and the respective costs were 5% higher during the last year of a patient's life when compared with the first year after diagnosis. During the last 12 months (N = 417) and 6 months (N = 505) of life and during palliative care (N = 145), the most common healthcare contact was home care.</p><p><strong>Conclusions: </strong>During active treatment, MM is primarily treated in the specialized care setting, with outpatient medication and visits to specialized care being the main cost drivers. These results can be utilized to estimate the need for care and expected costs over time due to MM and in health economic evaluations concerning MM.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142140742","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}

{"title":"Correction: Updated Public Health Impact and Cost Effectiveness of Recombinant Zoster Vaccine in Canadian Adults Aged 50 Years and Older.","authors":"Sydney George, Justin Carrico, Katherine A Hicks, Dessi Loukov, Cheryl Ng, Jessica Regan, Nikolaos Giannelos","doi":"10.1007/s41669-024-00502-w","DOIUrl":"10.1007/s41669-024-00502-w","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"785"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362412/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141627302","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":"Cost and Quality of Life of Disability Progression in Multiple Sclerosis Beyond EDSS: Impact of Cognition, Fatigue, and Limb Impairment.","authors":"Jürgen Wasem, Yanic Heer, Eleni Karamasioti, Erwan Muros-Le Rouzic, Giuseppe Marcelli, Danilo Di Maio, Stefan Braune, Gisela Kobelt, Paul Dillon","doi":"10.1007/s41669-024-00501-x","DOIUrl":"10.1007/s41669-024-00501-x","url":null,"abstract":"<p><strong>Background and objective: </strong>Understanding the socioeconomic burden of multiple sclerosis (MS) is essential to inform policymakers and payers. Real-world studies have associated increasing costs and worsening quality of life (QoL) with disability progression. This study aims to further evaluate the impact of cognition, fatigue, upper and lower limb function (ULF, LLF) impairments, and disease progression per Expanded Disability Status Scale (EDSS) level, on costs and QoL.</p><p><strong>Methods: </strong>This was a cross-sectional cohort study including 20,988 patients from the German NeuroTransData MS registry from 2009 to 2019. QoL analyses were based on EQ-5D-5L. Cost analyses included indirect/direct medical and non-medical costs. Eight subgroups, ranging from 439 to 1812 patients were created based on presence of measures for disease progression (EDSS), cognition (Symbol Digit Modalities Test [SDMT]), fatigue (Modified Fatigue Impact 5-Item Scale [MFIS-5]), ULF (Nine-Hole Peg Test [9HPT]), and LLF (Timed 25-Foot Walk [T25FW]). Multivariable linear regression assessed the independent effect of each test's score on QoL and costs, while adjusting for EDSS and 12 other confounders.</p><p><strong>Results: </strong>Lower QoL was associated with decreasing cognition (p < 0.001), worsening ULF (p = 0.025), and increasing fatigue (p < 0.0001); however, the negative impact of LLF worsening on QoL was not statistically significant (p = 0.54). Higher costs were associated with decreasing cognition (p < 0.001), worsening of ULF (p = 0.0058) and LLF (p = 0.049), and increasing fatigue (p < 0.0001). Each 1-scale-step worsening function of SDMT, MFIS-5, 9HPT, and T25FW scores resulted in €170, €790, €330, and €520 higher costs, respectively. Modeling disability progression based on SDMT, MFIS-5, 9HPT, and T25FW scores as an interaction with EDSS strata found associations with lower QoL and higher costs at variable EDSS ranges.</p><p><strong>Conclusions: </strong>Disease progression in MS measured by 9HPT, SDMT, and MFIS-5 had a significant negative impact on QoL and broad socioeconomic costs independent of EDSS. T25FW had a significant negative association with costs. Cognition, fatigue, ULF, and LLF have stronger impact on costs and QoL in patients with higher EDSS scores. Additional determinants of MS disability status, including SDMT, MFIS-5, 9HPT, and T25FW, should be considered for assessing cost effectiveness of novel therapeutics for MS.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"665-678"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362420/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141469931","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":"Correction: NICE's Pathways Pilot: Pursuing Good Decision Making in Difficult Circumstances.","authors":"Dawn Lee, Darren Burns, Ed Wilson","doi":"10.1007/s41669-024-00499-2","DOIUrl":"10.1007/s41669-024-00499-2","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"783"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362439/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141248087","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}