Value in Health最新文献

{"title":"Stakeholders’ Perspectives on Medication Adherence Enhancing Interventions","authors":"Bijan J. Borah PhD ,&nbsp;Lisa J. Pieretti MBA ,&nbsp;Alan J. Balch PhD ,&nbsp;Rajvi J. Wani PhD ,&nbsp;Christopher J. Daly PharmD ,&nbsp;Dalia Dawoud PhD ,&nbsp;David Hutchins MBA ,&nbsp;Mickaël Hiligsmann PhD ,&nbsp;Andrew M. Peterson PharmD, PhD ,&nbsp;Tamas Agh MD, PhD","doi":"10.1016/j.jval.2025.01.022","DOIUrl":"10.1016/j.jval.2025.01.022","url":null,"abstract":"<div><div>With an approximately 50% prevalence rate, medication nonadherence is a significant healthcare challenge that increases the risk of potentially avoidable adverse events and associated costs ranging from $949 to $44 190 per person annually. The ISPOR Medication Adherence and Persistence Special Interest Group conducted a systematic literature review (SLR) in 2023 to evaluate measures used in assessing medication adherence enhancing interventions (MAEIs). The findings revealed that although medication adherence and clinical outcomes were frequently measured, economic and patient-reported outcomes were underexplored. Importantly, the SLR also highlighted the need for consensus-based criteria to assess MAEIs.</div><div>This commentary supplements the SLR findings by presenting perspectives from key stakeholders: patients, manufacturers, providers, health technology assessment agencies, and payers. These stakeholders emphasize the importance of demonstrating improved clinical outcomes, health-related quality of life, and potential cost savings through MAEIs. Recognizing the significance of medication adherence for patient outcomes and regulatory and coverage decisions, manufacturers are starting to utilize artificial intelligence for developing personalized adherence strategies. Providers are addressing nonadherence through patient-centered approaches, health equity training, and value-based contracts. In addition to these new adherence-improving initiatives, MAEIs also need to consider personalized adherence strategies based on individual socioeconomic factors and social needs to improve their effectiveness. Future studies should focus on quantifying the costs associated with MAEI implementation and how such costs can be borne by different stakeholders, which will be crucial for large-scale adoption and sustainability of MAEIs.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 5","pages":"Pages 676-679"},"PeriodicalIF":4.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143912071","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Price Effects of Biosimilars in the United States","authors":"Mireia Jofre-Bonet PhD ,&nbsp;Alistair McGuire PhD ,&nbsp;Victoria Dayer PharmD ,&nbsp;Joshua A. Roth PhD, MHA ,&nbsp;Sean D. Sullivan PhD","doi":"10.1016/j.jval.2025.02.008","DOIUrl":"10.1016/j.jval.2025.02.008","url":null,"abstract":"<div><h3>Objectives</h3><div>This study evaluates the impact of biosimilar competition on the price of reference products and biosimilars in the US pharmaceutical market. We focus on average sales price (ASP) dynamics since 2015 in Medicare Part B because Medicare is a critical payer stakeholder in biosimilar price dynamics.</div></div><div><h3>Methods</h3><div>Using Centers for Medicare and Medicaid Services data over 34 quarters beginning in 2015, we analyzed the price effects of biosimilar competition in 8 biologic product markets. First, we evaluated the effect of biosimilar competition on ASP. Second, we evaluated the effect of market concentration on ASP using the Herfindahl-Hirschman index. We used instrumental variable methods to address the endogeneity between competition and ASP. Across analyses, our outcome variable is price ratio—the ratio of ASP measured over time relative to the fixed ASP at study baseline.</div></div><div><h3>Results</h3><div>Our results showed a 10% to 13% decrease in the originator price ratio for entry of each additional biosimilar competitor, and a 1.8% decrease in the originator price ratio with a 10% decrease in market concentration at the lowest Herfindahl-Hirschman index observed in study period. These findings highlight the statistically significant effect of biosimilar competition and market concentration on prices in high-cost biologics drug markets.</div></div><div><h3>Conclusions</h3><div>Our findings demonstrate that the US biosimilars market has become highly competitive, and as a result, prices have declined significantly over the period 2015 to 2023. These price dynamics have generated substantial savings for Medicare; however, there is a potential threat to market sustainability if these price trend dynamics persist in the coming years.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 5","pages":"Pages 742-750"},"PeriodicalIF":4.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573884","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Early Cost-Effectiveness Analysis of Using Whole-Genome Sequencing for Patients With Castration-Resistant Prostate Cancer","authors":"Jinjing Fu MSc ,&nbsp;Nora Franzen PhD, MSc ,&nbsp;Eline Aas PhD ,&nbsp;J.C. Koen van der Mijn MD, PhD ,&nbsp;Pim J. van Leeuwen MD, PhD ,&nbsp;Valesca P. Retel PhD, MSc, MHA","doi":"10.1016/j.jval.2025.02.004","DOIUrl":"10.1016/j.jval.2025.02.004","url":null,"abstract":"<div><h3>Objectives</h3><div>This study aims to assess the potential cost-effectiveness of using whole-genome sequencing (WGS)-guided systemic therapy in metastatic castrate-resistant prostate cancer compared with the European Association of Urology guideline recommended diagnostics from a Dutch societal perspective.</div></div><div><h3>Methods</h3><div>A decision analytic model combining a decision tree and partitioned survival models was developed to link diagnostic results with subsequent biomarker-guided treatments. Two diagnostic strategies, WGS and guideline-recommended practice—the genomic testing for breast cancer gene 1/2 (BRCA1/2) and deficient mismatch repair, were simulated to compare the health outcome and cost. Treatment effectiveness was estimated through survival analysis using published trial data. Sensitivity and scenario analyses were conducted to examine result robustness and to identify conditions under which WGS may be cost-effective.</div></div><div><h3>Results</h3><div>WGS identified an additional 21% of patients eligible for personalized therapy (PD-1/PDL-1 inhibitors and olaparib), resulting in an incremental increase in cost (€14 260) and quality-adjusted life years (QALY = 0.05). These results yielded an incremental cost-effectiveness ratio of €289 625 per QALY gained. WGS would become cost-effective if the cost of biomarker-guided therapies decreases by 62% and when identifying a proportion of 23% more patients with actional targets.</div></div><div><h3>Conclusions</h3><div>Our findings suggest that future treatments with improved efficacy and reduced cost could potentially make the WGS strategy cost-effective. Its unaccounted potential value to identify prognostic biomarkers, diagnostic alternatives, and patient heterogeneity should be addressed in future research and considered for optimal implementation. New reimbursement options are needed considering the high prices of biomarker-guided therapies that drive the incremental cost-effectiveness ratio.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 5","pages":"Pages 720-729"},"PeriodicalIF":4.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573890","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Are Updated COVID-19 Vaccines Still Relevant for All Adult Age Groups? An Economic Evaluation of the Monovalent XBB.1.5 Vaccine in Australia","authors":"Charles Ebuka Okafor PhD ,&nbsp;Syed Afroz Keramat PhD ,&nbsp;Namal N. Balasooriya PhD ,&nbsp;Echezona H. Dioji MSc","doi":"10.1016/j.jval.2025.01.014","DOIUrl":"10.1016/j.jval.2025.01.014","url":null,"abstract":"<div><h3>Objectives</h3><div>There is a progressive decline in the relative effectiveness of updated COVID-19 vaccines. Surveillance reports in Australia have also shown a wide variation in the disease severity and mortality across age groups. This study aimed to perform a cost-utility analysis of the monovalent XBB.1.5 vaccine relative to no updated booster vaccine between September 2023 and August 2024.</div></div><div><h3>Methods</h3><div>A cost-utility analysis was performed using a Markov model from the healthcare system perspective for 3 different age groups (18-64, 65-74, and ≥75 years). Costs and outcomes with the monovalent XBB.1.5 vaccine were compared with no updated booster dose for 1 year. Health outcomes were expressed as quality-adjusted life years (QALYs), whereas costs were presented in 2023 Australian dollars. Aggregate distributional cost-effectiveness analysis and sensitivity analyses were performed. The willingness-to-pay threshold was set at A$50 000/QALY.</div></div><div><h3>Results</h3><div>The updated vaccine was dominant for the 18 to 64 years group and cost-effective for the 65 to 74 years group (incremental cost-effectiveness ratio [ICER] = A$10 786/QALY) and ≥75 years group (ICER = A$36,531/QALY) relative to no updated booster vaccine. The major determinants of the ICER was the vaccine uptake rate. There was inequality in health benefits between the older First Nations versus nonindigenous Australians. The results were robust to simultaneous changes in the parameters’ values.</div></div><div><h3>Conclusions</h3><div>The monovalent XBB.1.5 vaccine is cost-effective and still relevant for all adult age groups in Australia. Based on current evidence, the study findings support the promotion of booster vaccination for Australian adults.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 5","pages":"Pages 730-741"},"PeriodicalIF":4.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143374782","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Do Machine Learning Approaches Perform Better Than Regression Models in Mapping Studies? A Systematic Review","authors":"Tianqi Hong PhD ,&nbsp;Shitong Xie PhD ,&nbsp;Xinran Liu MSc ,&nbsp;Jing Wu PhD ,&nbsp;Gang Chen PhD","doi":"10.1016/j.jval.2024.12.010","DOIUrl":"10.1016/j.jval.2024.12.010","url":null,"abstract":"<div><h3>Objectives</h3><div>To identify how machine learning (ML) approaches were implemented in mapping studies and to determine the extent to which ML improved performance compared with regression models (RMs).</div></div><div><h3>Methods</h3><div>A systematic literature search was conducted in 12 databases from inception to December 2023 to identify studies that applied ML to develop mapping algorithms. A data template was applied to extract data set information, source and target measures, ML approaches and RMs, mapping types (direct vs indirect), goodness-of-fit indicators (mean absolute error, mean squared error, root mean squared error, R-squared, and intraclass correlation coefficient), and validation methods. Differences in goodness-of-fit indicators between ML and RMs were summarized. Potential advantages and challenges for ML were further discussed.</div></div><div><h3>Results</h3><div>Thirteen mapping studies were identified, in which both ML and RM were adopted. Bayesian networks were the most frequently used ML approach (n = 6), followed by the least absolute shrinkage and selection operator (n = 4). The ordinary least square model was the most used RM (n = 8), followed by the censored least absolute deviation and multinomial logit models (n = 5 each). The average improvement in the goodness-of-fit of ML compared with that of RMs by indicators were 0.007 (mean absolute error), 0.004 (mean squared error), 0.058 (R-squared), 0.016 (intraclass correlation coefficient), and −0.0004 (root mean squared error).</div></div><div><h3>Conclusions</h3><div>There is an increasing number of studies using ML in developing mapping algorithms. Generally, a minor improvement of goodness-of-fit was observed compared with RMs when using mean-based comparisons. Issues such as how to interpret, apply, and externally validate the ML-based outputs would affect their implementation. Future studies are warranted to verify advantages of ML approaches.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 5","pages":"Pages 800-811"},"PeriodicalIF":4.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143374860","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Newer Glucagon-Like Peptide-1 Receptor Agonists Are Associated With Improved Glycemic Control in US Adults With Type 2 Diabetes: A Population-Level Time Series Analysis","authors":"Lei Lv PhD ,&nbsp;Yan Wang ScD ,&nbsp;Lin Xie MS ,&nbsp;Josh Noone PhD ,&nbsp;Sara Alvarez PharmD ,&nbsp;Yuehan Zhang PhD ,&nbsp;Yan Song PhD ,&nbsp;Daniel M. Rotroff PhD","doi":"10.1016/j.jval.2025.01.018","DOIUrl":"10.1016/j.jval.2025.01.018","url":null,"abstract":"<div><h3>Objectives</h3><div>The population-level impact of newer glucagon-like peptide-1 receptor agonist (GLP-1 RA) treatments on patients with type 2 diabetes remains unclear. Therefore, this study investigated the association of newer GLP-1 RAs with population-level diabetes-related outcomes in US adults with type 2 diabetes.</div></div><div><h3>Methods</h3><div>This was a noninterventional, retrospective analysis of administrative insurance claims data obtained from Optum’s deidentified Clinformatics® Data Mart Database between January 2004 and December 2022. Individual patient data were aggregated into monthly time series at the population level. Associations between the proportion of patients on newer GLP-1 RAs of interest (dulaglutide, once-weekly or oral semaglutide, and tirzepatide) and average hemoglobin A1c (HbA1c), HbA1c &lt;7.0%, and HbA1c &lt;8.0% were assessed using time series regressions. Time-based trends in population-level outcomes were assessed using interrupted time series analyses. All analyses were adjusted for potential confounders.</div></div><div><h3>Results</h3><div>Time series regression analyses demonstrated that a higher proportion of patients taking newer GLP-1 RAs was associated with a lower average HbA1c and a higher proportion of patients with HbA1c &lt;7.0% (after 5 months of treatment) and &lt;8.0% (after 2 months of treatment). Results from the interrupted time series analyses demonstrated that population-level glycemic control has been improving since the first newer GLP-1 RA (dulaglutide) was approved. This trend has persisted, even after the COVID-19 emergency declaration.</div></div><div><h3>Conclusions</h3><div>A higher percentage of patients receiving newer GLP-1 RAs was associated with significantly improved population-level glycemic control. The newer GLP-1 RA period displayed improved population-level glycemic control compared with the older GLP-1 RA period.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 5","pages":"Pages 712-719"},"PeriodicalIF":4.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143415371","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An Acquired Taste: Latent Class Analysis to Compare Adolescent and Adult Preferences for EQ-5D-Y-3L Health States","authors":"Jonathan L. Nazari PharmD ,&nbsp;Juan M. Ramos-Goñi PhD ,&nbsp;Ning Yan Gu PhD ,&nbsp;A. Simon Pickard PhD","doi":"10.1016/j.jval.2025.01.020","DOIUrl":"10.1016/j.jval.2025.01.020","url":null,"abstract":"<div><h3>Objectives</h3><div>US stakeholders advised including adolescents in the valuation study for the EQ-5D-Y-3L, a step toward greater acknowledgment of children in informing societal values. This study aimed to assess the relative contribution of adolescent and adult preferences to a model when combined.</div></div><div><h3>Methods</h3><div>Discrete choice experiment data were collected from an online sample of 1529 adults and 714 adolescents (ages 11-17). Each respondent completed 15 discrete choice experiment tasks, which were analyzed using latent class models representing varying number of preference classes. Within the best-fitting model, the contribution of each class was determined by the scale-adjusted class share (SACS), combining the class’s proportion of respondents (class share) and the magnitude of coefficients (within-class scale). We estimated the contribution of adolescent and adult respondents to SACS for each class, with lower SACS representing less contribution to the combined model.</div></div><div><h3>Results</h3><div>The best-fitting model described 6 classes. Adults had higher contribution to all except 1 class, accounting for 78.7% of the total contribution. After adjusting for the unequal sample size of adolescent and adult respondents, adults contributed approximately 65.0%, and adolescents contributed 35.0% of the weights toward a combined model.</div></div><div><h3>Conclusions</h3><div>Adolescents showed diminished, disproportionate representation in a combined model, due in part to more indifferent, less informative preferences for EQ-5D-Y-3L health states compared with adults. Latent class analysis showcases one approach to estimate and weight contributions from intentionally sampled subgroups in a combined model.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 5","pages":"Pages 781-789"},"PeriodicalIF":4.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143426296","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development and Testing of the Psychometric Properties of 20 Bolt-On Items for the EQ-5D-5L Across 31 Rare Diseases","authors":"Richard Huan Xu PhD ,&nbsp;Fanni Rencz PhD ,&nbsp;Ruiqi Sun MPhil ,&nbsp;Dong Dong PhD ,&nbsp;Shuyang Zhang MD","doi":"10.1016/j.jval.2025.01.006","DOIUrl":"10.1016/j.jval.2025.01.006","url":null,"abstract":"<div><h3>Objectives</h3><div>Our objective was to develop and assess the psychometric properties of relevant bolt-on items for the EQ-5D-5L in patients with rare diseases (RDs).</div></div><div><h3>Methods</h3><div>Nineteen new EQ-5D-5L bolt-ons were developed based on literature review, expert input, and qualitative interviews and focus groups with patients, caregivers, and representatives of patient associations. A nationwide, cross-sectional, web-based survey in China included patients or caregivers of patients with 31 RDs in China (n = 9190). In each RDs, participants completed the EQ-5D-5L and 3 of 20 (1 existing and 19 newly developed) bolt-ons. Ceiling, explanatory power, convergent, divergent, and known-group validity were examined.</div></div><div><h3>Results</h3><div>Among the bolt-ons, itching had the lowest ceiling (6.5%), whereas social relationships had the highest (42.2%). The absolute reduction in the ceiling of the EQ-5D-5L with the addition of any bolt-ons was limited, ranging from 0 (respiratory problems) to 8.3% points (isolation). Dignity and vitality resulted in the largest increase in explained variance in EQ VAS. The isolation, fertility, and visual acuity bolt-ons showed good divergent validity from the EQ-5D-5L items. There was strong convergent validity between SF-12 and conceptually related bolt-ons (eg, physical health composite and muscle problems bolt-on). Various bolt-ons improved the known-groups validity in specific patient groups, eg, Huntington’s disease (oral expressions), scleroderma (dexterity), myasthenia gravis (muscle problems), neuromyelitis optica and multiple sclerosis (fatigue), Marfan syndrome (self-image), and Pompe disease (safety).</div></div><div><h3>Conclusion</h3><div>The EQ-5D-5L shows sufficient validity in most RDs, but incorporating relevant, specific bolt-ons could enhance its ability to more comprehensively assess health-related quality of life in these patients.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 5","pages":"Pages 769-780"},"PeriodicalIF":4.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143067692","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Capturing the Additional Cardiovascular Benefits of SGLT2 Inhibitors and GLP-1 Receptor Agonists Beyond the Control of Traditional Risk Factors in People With Diabetes","authors":"Shu Niu MS ,&nbsp;Dawei Guan MS, MD ,&nbsp;Lizheng Shi PhD ,&nbsp;Vivian Fonseca PhD, MD ,&nbsp;Mikael Svensson PhD ,&nbsp;Mohammed K. Ali MD, MSc, MBA ,&nbsp;Yan V. Sun PhD ,&nbsp;Xin Hu PhD ,&nbsp;Chang Su PhD ,&nbsp;Carl Yang PhD ,&nbsp;Hui Shao PhD, MD","doi":"10.1016/j.jval.2025.01.015","DOIUrl":"10.1016/j.jval.2025.01.015","url":null,"abstract":"<div><h3>Objectives</h3><div>This study aimed to quantify the additional cardioprotective effects of sodium-glucose cotransporter 2 inhibitors (SGLT2is) and glucagon-like peptide-1 receptor agonists (GLP-1RAs) beyond the traditional risk factors control in individuals with type 2 diabetes. This helps calibrate the Building, Relating, Assessing, and Validating Outcomes (BRAVO) diabetes simulation model to capture the total cardiovascular benefits of new diabetes medications accurately.</div></div><div><h3>Methods</h3><div>We extracted patient characteristics and treatment efficacy data from 4 cardiovascular outcome trials (CVOTs) of SGLT2is and 4 CVOTs of GLP-1RAs completed before May 2023. Using the BRAVO diabetes simulation model, we translated reductions in traditional risk factors (ie, glycated hemoglobin, systolic blood pressure, low-density lipids, and body mass index) from the newer drugs into risk reductions in cardiovascular outcomes (ie, myocardial infarction [MI], stroke, congestive heart failure [CHF], and mortality), assuming that the drug-associated risk reductions were only driven by traditional risk factors. Then, we compared the simulated risk-factor-driven risk reductions of cardiovascular outcomes with observed risk reductions from the trials and calculated drug-specific incremental benefits (DIB).</div></div><div><h3>Results</h3><div>After accounting for the cardiovascular effects from traditional risk factors control, SGLT2is was associated with an additional 19% risk reduction in CHF (DIB: 0.81, 95% CI 0.72-0.90). Furthermore, the uncalibrated model predicted a risk reduction in stroke with SGLT2is, which was not observed in CVOTs. This discrepancy highlights the need for an SGLT2i-specific calibrator to align the simulation results with the observed outcomes. In contrast, no additional cardiovascular benefit was associated with GLP-1RAs after controlling for traditional risk factors.</div></div><div><h3>Conclusions</h3><div>Our study revealed that SGLT2is could further reduce CHF risk beyond the control of traditional risk factors but may offer additional pathways to offset the overall benefits of traditional risk factor control in stroke risk. No additional cardiovascular benefits were observed for GLP-1RAs beyond traditional risk factor control. The BRAVO model calibration enhances cardiovascular outcome prediction with these newer antidiabetic therapies.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 5","pages":"Pages 762-768"},"PeriodicalIF":4.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143374785","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evolving Concept of Value in Health Economics and Outcomes Research: Emerging Tools for Innovation and Access to Cell and Gene Therapies for Rare Diseases","authors":"Walter Toro MD, PhD ,&nbsp;Min Yang MD, PhD ,&nbsp;Anish Patel PharmD ,&nbsp;Su Zhang PhD ,&nbsp;Omar Dabbous MD ,&nbsp;Louis P. Garrison Jr. PhD","doi":"10.1016/j.jval.2024.12.006","DOIUrl":"10.1016/j.jval.2024.12.006","url":null,"abstract":"<div><h3>Objectives</h3><div>Recent scientific breakthroughs have propelled the development of disease-modifying and potentially curative cell and gene therapies (CGTs) for rare diseases, including those diseases previously considered untreatable. The unique characteristics of CGTs, however, pose challenges to the traditional methods of therapy value determination, reimbursement, and outcome evaluation used by regulatory and assessment agencies for product approval and market access. Notably, CGTs are one-time or short-course treatments, often first-in-class (precluding direct comparisons with effective alternatives), and have health benefits that are largely realized over time.</div></div><div><h3>Methods</h3><div>We summarized emerging health economics and outcomes research (HEOR) solutions in 5 areas pertaining to CGTs for rare diseases using examples from literature and recently approved therapies. These solutions include the collection of real-world data to generate real-world evidence, patient centricity and novel value assessment frameworks, evolution of economic evaluation methodologies, novel reimbursement models and management of affordability, and health equity and societal benefits.</div></div><div><h3>Results</h3><div>Advances in HEOR methods have informed the design and collection of long-term real-world data for CGT efficacy extrapolation, use of novel (including surrogate) endpoints, integration of the patient’s voice and preferences, and application of sophisticated statistical methodology and artificial intelligence/machine learning techniques.</div></div><div><h3>Conclusions</h3><div>Continuing innovation in HEOR is expected to contribute to improved health outcomes, reduced costs, and enhanced access, ultimately enabling more efficient delivery of CGTs to patients living with rare diseases.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 5","pages":"Pages 686-691"},"PeriodicalIF":4.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142915659","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}