PharmacoEconomics Open最新文献

{"title":"Clinicians' Preferences for Sphingosine-1-Phosphate Receptor Modulators in Multiple Sclerosis Based on Clinical Management Considerations: A Choice Experiment.","authors":"Alexander Keenan, Chiara Whichello, Hoa H Le, David M Kern, Gabriela S Fernandez, Vicky Turner, Anup Das, Matt Quaife, Amy Perrin Ross","doi":"10.1007/s41669-024-00510-w","DOIUrl":"10.1007/s41669-024-00510-w","url":null,"abstract":"<p><strong>Background: </strong>Four sphingosine-1-phosphate receptor (S1PR) modulators are currently available in the USA for treating relapsing forms of multiple sclerosis (MS). These S1PR modulators have similar efficacy. Clinicians may therefore consider other factors, such as clinical management considerations, when distinguishing among treatments. This study estimated which S1PR modulator clinicians would choose on the basis of a treatment's clinical management and quantified how individual aspects of clinical management might drive this choice.</p><p><strong>Methods: </strong>A multi-criteria decision analysis (MCDA) was conducted on the basis of clinical management preferences elicited in a discrete choice experiment (DCE) and real-world clinical management profiles of the S1PR modulators currently available to treat relapsing forms of MS (fingolimod, ozanimod, ponesimod, siponimod). The DCE was completed by neurologists in the USA experienced in treating MS and included eight clinical management attributes: first-dose observations, genotyping, liver function tests, eye exams, drug-drug interactions, interactions with antidepressants, interactions with foods high in tyramine, and immune system recovery time. Attribute levels were selected on the basis of S1PR modulator product labels. In the MCDA, partial MCDA scores were created for each attribute and summed to produce an overall MCDA score for each S1PR modulator.</p><p><strong>Results: </strong>The DCE was completed by 200 neurologists. The overall MCDA score was highest for ponesimod (4.78 points), followed by siponimod (4.10 points), fingolimod (3.61 points), and ozanimod (2.38 points). Having fewer drug-drug interactions contributed most to the overall scores (up to 1.56 points), followed by having no first-dose observations (0.95 points), the shortest immune system recovery time (0.94 points), and not interacting with foods high in tyramine (0.86 points).</p><p><strong>Conclusion: </strong>When considering clinical management convenience, the average US-based neurologist treating MS is likely to choose ponesimod over siponimod, fingolimod, or ozanimod. The strongest driver of preferences was the number of drug-drug interactions. This information can help inform recommendations for the treatment of MS and facilitate shared decision-making between clinicians and patients.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"857-867"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499474/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142081126","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-Utility Analysis of Adjuvant Olaparib for Germline BRCA1/2-Mutated, High-Risk HER2-Negative Early Breast Cancer in Spain.","authors":"Sergio Cedillo, Almudena González-Domínguez, Yoana Ivanova-Markova, Rafael López López, Sara López-Tarruella Cobo, José Alberto Peña Pedrosa","doi":"10.1007/s41669-024-00518-2","DOIUrl":"10.1007/s41669-024-00518-2","url":null,"abstract":"<p><strong>Objective: </strong>Here we estimate the cost-effectiveness of olaparib in the Spanish National Health Service (SNHS) as adjuvant treatment of early germline mutations in the BRCA1/2 genes (gBRCAm) HER2-negative (HER2neg) breast cancer (BC) with high risk of recurrence.</p><p><strong>Methods: </strong>A semi-Markov model was adapted to the Spanish healthcare setting, using the perspective of the SNHS, and a lifetime horizon. Two scenarios were compared: receiving olaparib versus standard of care (SoC) treatment. The model comprised five health states and included the clinical results of the OlympiA trial, along with the direct healthcare costs associated with the use of early BC and subsequent treatment resources (€2023). A discount rate of 3% was applied for future cost and quality-of-life outcomes. A probabilistic sensitivity analysis (PSA) was carried out.</p><p><strong>Results: </strong>The introduction of olaparib as adjuvant treatment for patients with early gBRCAm HER2neg BC with high risk of recurrence could involve an incremental cost of €44,273 and €50,164, with an improvement of 1.14 and 1.28 quality-adjusted life years (QALYs) for hormone receptor-positive (HR⁺) and triple-negative (TN) patients, respectively. Therefore, adjuvant olaparib could be cost-effective for early gBRCAm HER2neg BC, with an incremental cost-effectiveness ratio of €38,839/QALY and €39,084/QALY for HR⁺ and TN patients, respectively. The results from the PSA showed that 75.7% and 82.2% of the simulations fell below the €60,000/QALY threshold.</p><p><strong>Conclusions: </strong>Olaparib as adjuvant treatment could be cost-effective in gBRCAm patients with early HER2neg BC in Spain.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"887-896"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499548/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142004967","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":"Comparison of Two Financial Incentives to Encourage the Use of Adalimumab Biosimilars: Results of a French Experiment Close to Clinicians.","authors":"Marion Tano, Pascal Paubel, Matthieu Ribault, Albane Degrassat-Théas","doi":"10.1007/s41669-024-00505-7","DOIUrl":"10.1007/s41669-024-00505-7","url":null,"abstract":"<p><strong>Background: </strong>In 2018, the French government introduced two mutually exclusive financial incentives to increase etanercept and insulin glargine biosimilar use. The general case redirects 20% of the price difference between the reference product and its biosimilars to hospitals for every biosimilar dispensed in community pharmacies from hospital prescriptions. The experimental case redirects 30% to prescribing clinical units after hospital selection. Adalimumab was added to these incentives in 2019, after its first biosimilar was launched.</p><p><strong>Objective: </strong>This retrospective observational study aimed to compare both general and experimental incentives after 19 months to assess the impact of directly incentivizing clinical units for adalimumab biosimilars.</p><p><strong>Method: </strong>The monthly number of adalimumab boxes dispensed in community pharmacies was linked to the corresponding hospital's prescription using IQVIA Xponent data from November 2017 to October 2020. The monthly mean rate of adalimumab biosimilars was compared between incentive groups and subgroups depending on hospitals' prior experience with the etanercept experimental case.</p><p><strong>Results: </strong>General case hospitals had a significantly lower mean biosimilar uptake (16.7% vs 23.2%, p = 0.029) than those included in the experimental case. Twenty-three of the 40 hospitals in the experimental case and ten out of the 91 hospitals studied in the general case had already taken part in the etanercept experiment. Biosimilar uptake was higher, but not statistically significant, for hospitals with prior experience in the adalimumab general case group (p = 0.086).</p><p><strong>Conclusions: </strong>This study confirmed that incentivizing close to physicians was more effective in increasing the biosimilar rate. It also suggested that previous incentive experience positively influenced biosimilar penetration.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"897-909"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499575/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141492949","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: 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}

{"title":"A Cost-Effectiveness Analysis of Adjuvant Nivolumab for Patients with Resected Esophageal Cancer or Gastroesophageal Junction Cancer in France.","authors":"Paul Casabianca, Marc Massetti, François-Emery Cotte, Romain Moreau, Sarah Kassahun, Prianka Singh, Inkyu Kim, Anne-Françoise Gaudin, Guillaume Piessen, Henri Leleu","doi":"10.1007/s41669-024-00500-y","DOIUrl":"10.1007/s41669-024-00500-y","url":null,"abstract":"<p><strong>Introduction: </strong>Esophageal and gastroesophageal junction cancer (EC/GEJC) is a poor prognosis disease with a high risk of recurrence even in patients curatively resected. Adjuvant nivolumab is currently used for patients with completely resected (R0) EC/GEJC who have residual pathologic disease following prior neoadjuvant chemoradiotherapy. This study aimed to determine the cost effectiveness of nivolumab in this indication in France according to the collective perspective excluding indirect costs.</p><p><strong>Materials and methods: </strong>A simplified four-health-state semi-Markov model was developed to model EC/GEJC patients who have residual disease after neoadjuvant chemoradiotherapy followed by R0 over a 15-year time horizon, comparing adjuvant nivolumab versus surveillance, which was the recommended French clinical practice before immunotherapy arrival. Time-to-recurrence (TTR) from CheckMate 577 was used to inform transition from disease-free to post-recurrence health state; patients who recurred were split according to the distribution of type of recurrence observed during the trial. Post-recurrence survival (PRS) according to the type of recurrence was derived from a real-world registry.</p><p><strong>Results: </strong>Adjuvant treatment with nivolumab led to an incremental survival gain of 1.19 years (+ 34%), mostly in the disease-free state, an incremental cost of €48,634 and QALY of 0.98 resulting in an incremental cost-utility ratio (ICUR) of €49,572/QALY with limited uncertainty. 'Cure assumption' at 5 years had an important impact on the results (€41,115/QALY; - 17%), as that tends to increase life-years and QALYs while costs remain the same. Probabilistic sensitivity analyses confirmed reference ICUR (€52,542/QALY) with 80% probability of nivolumab being cost effective at a willingness-to-pay threshold of €75,000/QALY.</p><p><strong>Conclusions: </strong>Our analysis suggests that adjuvant nivolumab is cost effective in the treatment of EC/GEJC patients who have residual disease after neoadjuvant CRT followed by R0 resection. Compared with previously evaluated cost-effectiveness analyses for other immune-checkpoint inhibitors indicated in metastatic settings, ICUR appears particularly low in this early setting thanks to the important impact on health outcomes and capped treatment duration.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"689-699"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362429/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141535054","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":"Mapping Payment and Pricing Schemes for Health Innovation: Protocol of a Scoping Literature Review.","authors":"Vittoria Ardito, Ludovico Cavallaro, Michael Drummond, Oriana Ciani","doi":"10.1007/s41669-024-00496-5","DOIUrl":"10.1007/s41669-024-00496-5","url":null,"abstract":"<p><strong>Introduction: </strong>Innovative pricing and payment/reimbursement schemes have been proposed as one part of the solution to the problem of patient access to new health technologies or to the uncertainty about their long-term effectiveness. As part of a Horizon Europe research project on health innovation next generation pricing and payment models (HI-PRIX), this protocol illustrates the conceptual and methodological steps related to a scoping review aiming at investigating nature and scope of pricing and payment/reimbursement schemes applied to, or proposed for, existing or new health technologies.</p><p><strong>Methods: </strong>A scoping review of literature will be performed according to the PRISMA guidelines for scoping reviews (PRISMA-ScR) guidelines. The search will be conducted in three scientific databases (i.e., PubMed, Web of Science, and Scopus), over a 2010-2023 timeframe. The search strategy is structured around two blocks of keywords, namely \"pricing and payment/reimbursement schemes,\" and \"innovativeness\" (of the scheme type or scheme use). A simplified search will be replicated in the gray literature. Studies illustrating pricing and payment/reimbursement schemes with a sufficient level of details to explain their characteristics and functioning will be deemed eligible to be considered for data synthesis. Pricing and payment/reimbursement schemes will be classified according to several criteria, such as their purpose, nature, governance, data collection needs, and foreseen distribution of risk. The results will populate a publicly available online tool, the Pay-for-Innovation Observatory.</p><p><strong>Discussion: </strong>The findings of this review have the potential to offer a comprehensive toolkit with a variety of pricing and payment schemes to policymakers and manufacturers facing reimbursement and access decisions.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"765-772"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362434/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141076629","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":"Modeling First-Line Daratumumab Use for Newly Diagnosed, Transplant-Ineligible, Multiple Myeloma: A Cost-Effectiveness and Risk Analysis for Healthcare Payers.","authors":"Diana Beatriz Bayani, Yihao Clement Lin, Chandramouli Nagarajan, Melissa G Ooi, Allison Ching Yee Tso, John Cairns, Hwee Lin Wee","doi":"10.1007/s41669-024-00503-9","DOIUrl":"10.1007/s41669-024-00503-9","url":null,"abstract":"<p><strong>Background and objective: </strong>This study aimed to assess the cost-effectiveness of two regimens regarded as the standard of care for the treatment of newly diagnosed, transplant-ineligible multiple myeloma in Singapore: (1) daratumumab, lenalidomide, and dexamethasone and (2) bortezomib, lenalidomide, and dexamethasone. Additionally, it aimed to explore potential strategies to manage decision uncertainty and mitigate financial risk.</p><p><strong>Methods: </strong>A cost-effectiveness analysis from the healthcare system perspective was conducted using a partitioned survival model to estimate lifetime costs and quality-adjusted life years (QALYs) associated with daratumumab-based treatment and the bortezomib-based regimen. The analysis used data from the MAIA and SWOG S0777 trials and incorporated local real-world data where available. Sensitivity analyses were performed to evaluate the robustness of the findings, and a risk analysis was conducted to analyze various payer strategies in terms of their payer strategy and uncertainty burden (P-SUB), which account for the decision uncertainty and the additional cost of choosing a suboptimal intervention.</p><p><strong>Results: </strong>The incremental cost-effectiveness ratio (ICER) for daratumumab, lenalidomide, and dexamethasone (DRd) compared with bortezomib, lenalidomide, and dexamethasone (VRd) was US $90,364 per QALY gained. The results were sensitive to variations in survival for DRd, postprogression treatment costs, cost of hospice care, and hazard ratio for progression-free survival. The scenarios explored indicated that structural assumptions, such as the time horizon of the analysis, significantly influenced the results due to uncertainties arising from immature trial data and treatment efficacy over time. Among the various payer strategies compared, an upfront price discount for daratumumab emerged as the best approach with the lowest P-SUB at US $14,708.</p><p><strong>Conclusion: </strong>In conclusion, this study finds that daratumumab as a first-line treatment for myeloma exceeds the cost-effectiveness threshold considered in this evaluation. An upfront price reduction is the recommended strategy to manage uncertainties and mitigate financial risks. These findings highlight the importance of targeted payer strategies to address specific types and sources of uncertainty.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"651-664"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362436/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141427407","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":"Using a Patient-Centered Multicriteria Decision Analysis to Assess the Value of Multiple Sclerosis Treatments in the US: A Study Protocol.","authors":"Surachat Ngorsuraches, Tim C Lai, Rebecca Habermann, Yolanda Wheeler, William Meador","doi":"10.1007/s41669-024-00509-3","DOIUrl":"10.1007/s41669-024-00509-3","url":null,"abstract":"<p><strong>Objective: </strong>The engagement of patients and family caregivers in value assessment is pivotal since they provide valuable contributions to assessment acceptability and relevance. The proposed study aims to use patient-centered techniques and multicriteria decision analysis (MCDA) to evaluate the values of disease-modifying therapies (DMTs) for multiple sclerosis (MS) from the perspectives of patients and family caregivers living in three 'Deep South' States of the US-Alabama, Louisiana, and Mississippi.</p><p><strong>Methods: </strong>This study will follow guidance from the Patient-Centered Outcomes Research Institute (PCORI) for patient engagement and two best practice reports for MCDA from the Professional Society for Health Economics and Outcomes Research (ISPOR) to complete value assessment. Throughout the study, we will engage multiple stakeholders, including patients, family caregivers, healthcare providers, and payers. Forty patients with MS and their family caregivers from Alabama, Louisiana, and Mississippi will be invited to participate in this study. We will intensively train them for value assessment knowledge and MCDA before we engage them in MCDA to determine the value of DMTs for MS.</p><p><strong>Discussions: </strong>Our approach differs from common MCDA since we incorporated a patient-centered framework in this study. Unlike previous studies only briefly inform or prepare participants before the MCDA process, in this study, we will provide basic value assessment trainings for patients and family caregivers to ensure they can effectively engage throughout the patient-centered MCDA process. We expect this study will demonstrate that the patient-centered MCDA approach is feasible and likely leads to improved patients' and family caregivers' engagement in value assessment.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"773-781"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362406/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141563959","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}