PharmacoEconomics最新文献

{"title":"Exploring the Generalizability of Foreign Cost-Effectiveness Analysis to Spain Using Data From a Scoping Review of Multinational Studies.","authors":"Lidia García-Pérez, Ignacio Abásolo-Alessón, Miguel Ángel Negrín-Hernández","doi":"10.1007/s40273-025-01541-9","DOIUrl":"https://doi.org/10.1007/s40273-025-01541-9","url":null,"abstract":"<p><strong>Objective: </strong>This study examines the generalizability of foreign economic evaluations to the Spanish healthcare system. The research aims to describe the cross-country adaptation methods identified in a scoping review of multinational cost-utility analyses and to examine the probability of concordant funding decisions between Spanish and foreign results, as well as to identify factors influencing generalizability.</p><p><strong>Methods: </strong>First, a scoping review of multinational studies reporting cost-utility analyses for at least two countries, including Spain, was conducted using MEDLINE, PubMed, Embase and Web of Science in April 2025. Data related to transferability were extracted and a narrative synthesis was performed. Second, a dataset of case comparisons-each defined as a technology against a comparator in a specific population-was developed from the identified studies. Each foreign comparison was matched to its Spanish equivalent within the same study. Incremental cost-effectiveness ratios (ICERs) were converted to 2024 Spanish Euros and compared against a threshold of €30,000 per quality-adjusted life year (QALY). A multilevel logit model was used, with a binary variable indicating decision concordance between Spanish and foreign ICERs/dominance as the dependent variable. We also analysed the distances in the incremental costs and incremental QALYs between countries using a log-normal bivariate model. Country-specific and other study-related factors were considered as independent variables in both models.</p><p><strong>Results: </strong>The review included 57 studies. Most were funded by drug manufacturers and conducted in Europe. The majority of authors did not specify their reasons for selecting countries. All but three studies attempted to use local costs, probabilities and/or epidemiological data. Twelve studies incorporated country-specific utilities. A total of 644 comparisons were analysed; 142 were Spanish results and 502 were foreign results with their Spanish equivalents. The cost-effectiveness plane quadrant of the foreign result matched the Spanish result in 84% of cases. Assuming a threshold of €30,000 per QALY, the funding decisions were the same in 93% of cases. The probability of decision concordance was higher when the study was conducted in a Eurozone country or in the United Kingdom. Sensitivity analysis showed the variability of decisions depending on the selected cost-effectiveness threshold. Similar variables were found as relevant factors explaining the distance in the incremental QALYs analysis.</p><p><strong>Conclusion: </strong>Foreign cost-effectiveness results of those studies analysing drugs from Eurozone countries such as France, Germany, Italy, or from the United Kingdom can often be generalizable and provide meaningful insights for decision making in Spain. However, these findings should not be used as a reason to avoid country-specific studies if they are feasible. Further resear","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145137974","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Eladocagene Exuparvovec for the Treatment of Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd): An Economic Evaluation from a US Perspective.","authors":"Rongrong Zhang, Thomas O'Connell, Berrin Monteleone, Yixi Teng, Paul Wuh-Liang Hwu, Paul Castellano, Ioannis Tomazos","doi":"10.1007/s40273-025-01542-8","DOIUrl":"https://doi.org/10.1007/s40273-025-01542-8","url":null,"abstract":"<p><strong>Background: </strong>Recently, the gene therapy eladocagene exuparvovec received accelerated approval from the US Food and Drug Administration (as eladocagene exuparvovec-tneq) for treatment of aromatic L-amino acid decarboxylase deficiency (AADCd), a rare, infantile-onset disorder characterized by developmental delays.</p><p><strong>Objectives: </strong>To conduct a US, modified societal perspective cost-utility analysis comparing eladocagene exuparvovec versus best supportive care (BSC).</p><p><strong>Methods: </strong>Multistate survival modeling was implemented tracking disease progression from a \"no motor function\" health state to achievement of motor-function improvements, measured by: (1) multiples of the meaningful score difference (MSD) of the Peabody Developmental Motor Scales-Second Edition (PDMS-2) total score and (2) motor milestones. Eladocagene exuparvovec trials informed clinical inputs. Health-state utilities were from a US time-trade-off study that valued AADCd quality-of-life impacts. Outcomes were discounted (3%); costs were reported in 2024 US dollars. Scenario analyses, characterizing alternative approaches of the multistate survival model analyses and probabilistic sensitivity analysis to assess the impact of parameter uncertainty, were conducted.</p><p><strong>Results: </strong>Discounted incremental quality-adjusted life-years (QALYs) for eladocagene exuparvovec were 20.83 (multiples of the MSD of total PDMS-2) and 18.44 (motor milestones). Incremental cost per QALY ranged from $199,007-$224,104. The scenario and sensitivity analyses results supported the validity of the base case analysis.</p><p><strong>Conclusions: </strong>Eladocagene exuparvovec is associated with considerable QALY gains compared with BSC. Within the context of other ultra-rare and/or one-time treatments, eladocagene exuparvovec provides substantial clinical improvements at lower cost than many other rare-disease treatments. Findings from this study highlight that eladocagene exuparvovec is an important treatment option for patients with AADCd.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145131494","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness Analysis of Aztreonam-Avibactam (ATM-AVI) Versus Colistin + Meropenem (COL + MER) for the Treatment of Infections Caused by Metallo-β-Lactamase (MBL)-Producing Enterobacterales in Italy.","authors":"Marco Falcone, Xiyu Bao, Fionn Woodcock, Roberto Di Virgilio, Maria Alejandra Vidal Pereira, Michal Kantecki, Maria Gheorghe","doi":"10.1007/s40273-025-01528-6","DOIUrl":"https://doi.org/10.1007/s40273-025-01528-6","url":null,"abstract":"<p><strong>Background and objective: </strong>Aztreonam-avibactam (ATM-AVI) is a novel combination antibiotic approved in Europe for the treatment of complicated intra-abdominal infection, hospital-acquired pneumonia, including ventilator-associated pneumonia; complicated urinary tract infection, including pyelonephritis and for infections due to aerobic Gram-negative organisms with limited treatment options. This analysis assessed the cost effectiveness of ATM-AVI ± metronidazole versus colistin + meropenem (COL + MER) for the treatment of patients with complicated intra-abdominal infection and hospital-acquired pneumonia/ventilator-associated pneumonia, including infections with suspected metallo-β-lactamase-producing Enterobacterales from the public payer perspective in Italy using phase III trial data.</p><p><strong>Methods: </strong>The cost-effectiveness analysis adopted a decision tree model to simulate the clinical pathway of complicated intra-abdominal infection and hospital-acquired pneumonia/ventilator-associated pneumonia, followed by a Markov model to capture lifetime health outcomes on cured patients, with costs valued in 2024 Euros and discounted at 3%. The model captures the impact of resistant pathogens and side effects (i.e. nephrotoxicity). Model uncertainty was assessed using a probabilistic and deterministic sensitivity analysis.</p><p><strong>Results: </strong>The ATM-AVI treatment sequence (ATM-AVI ± metronidazole followed by cefiderocol after treatment failure) had improved clinical outcomes and higher cure rates, shorter hospital stays and higher quality-adjusted life-year gains compared with the COL + MER sequence (COL + MER followed by cefiderocol after treatment failure). The incremental cost-effectiveness ratio in the ATM-AVI sequence was dominant for complicated intra-abdominal infection and was €1552 per quality-adjusted life-year for hospital-acquired pneumonia/ventilator-associated pneumonia, well below the willingness-to-pay threshold of €30,000 in Italy.</p><p><strong>Conclusions: </strong>Our analysis suggests that ATM-AVI is expected to be a cost-effective use of Italian healthcare resources for treating suspected metallo-β-lactamase-producing Enterobacterales, including complicated intra-abdominal infection and hospital-acquired pneumonia/ventilator-associated pneumonia.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145125574","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Consequence Analysis of Natalizumab Compared with Other High-Efficacy Treatments in Patients with Relapsing-Remitting Multiple Sclerosis.","authors":"Luca Prosperini, Vincenzo Brescia Brescia Morra, Carla Fornari, Laura Santoni, Daria Perini, Roberto Bergamaschi, Paolo Angelo Cortesi","doi":"10.1007/s40273-025-01539-3","DOIUrl":"https://doi.org/10.1007/s40273-025-01539-3","url":null,"abstract":"<p><strong>Background: </strong>Advances in the availability and regimen optimization of highly effective disease-modifying treatments (DMTs) for relapsing-remitting multiple sclerosis (RRMS) have led to questions about their comparative worth.</p><p><strong>Objectives: </strong>This study evaluates the costs and effects of natalizumab versus other highly effective DMTs and the impact, in terms of times and costs, of the new subcutaneous natalizumab formulation versus the intravenous formulation in patients with RRMS in Italy.</p><p><strong>Methods: </strong>This is a cost-consequence analysis from the Italian national health service and societal perspectives. A Markov model was developed to assess clinical and cost outcomes related to disease and DMTs. The model simulated two scenarios: one comparing natalizumab extended-dose regimen and ofatumumab and ocrelizumab, focusing on efficacy outcomes and costs, and one comparing intravenous and subcutaneous natalizumab with a focus on administration resource consumption, times, and costs. Model input data came from the literature.</p><p><strong>Results: </strong>DMTs had similar clinical and social outcomes: natalizumab slightly reduced disease progression, increased quality-adjusted life-years, and reduced the impact on days of productivity loss and informal care. Natalizumab also resulted in statistically significant 5-year cost reductions compared with ocrelizumab and ofatumumab. Subcutaneous natalizumab improved resource consumption compared with intravenous natalizumab, saving the time of healthcare professionals, patients, and caregivers and reducing administration costs. The subcutaneous formulation was associated with statistically significant total direct and indirect cost reductions at 5 years.</p><p><strong>Conclusion: </strong>6-week dosing regimen of natalizumab showed a slight improvement of clinical and social outcomes and a statistically significant cost reduction compared with ocrelizumab and ofatumumab over a 5-year simulation. Moreover, subcutaneous administration reduced administration times and costs.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145086627","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Heterogeneity in Economic Value of SGLT2is for Type 2 Diabetes: Subgroup Modeling Cost-Effectiveness Analyses.","authors":"Kah Suan Chong, Chun-Ting Yang, Chi-Chuan Wang, Huang-Tz Ou, Shihchen Kuo","doi":"10.1007/s40273-025-01536-6","DOIUrl":"https://doi.org/10.1007/s40273-025-01536-6","url":null,"abstract":"<p><strong>Background and objective: </strong>Although heterogeneous treatment effects of sodium-glucose cotransporter 2 inhibitors (SGLT2is) have been revealed, the heterogeneous economic value of SGLT2is in real-world type 2 diabetes (T2D) populations with diverse clinical characteristics remains unclear. We conducted subgroup cost-effectiveness analyses of SGLT2is versus dipeptidyl peptidase 4 inhibitors (DPP4is) among patients with T2D.</p><p><strong>Methods: </strong>A multi-state transition model was used to estimate the incremental cost-effectiveness ratios (ICERs, in US$ per quality-adjusted life-years [QALYs] gained) and value-based pricing (VBP) among patients with T2D stratified by age, estimated glomerular filtration rate (eGFR), glycated hemoglobin (HbA1c), and body mass index (BMI) over 5 years and a Lifetime horizon from a healthcare sector perspective, with both costs and quality-adjusted Life years discounted at 3% annually. Model inputs included treatment effects derived from analysis of individual-level data in Taiwan, and health utilities and costs sourced from published Literature of Taiwanese populations. Deterministic and probabilistic sensitivity analyses across subgroups were performed. All costs were standardized to 2022.</p><p><strong>Results: </strong>Over 5 years, the ICERs of SGLT2is versus DPP4is were as follows: age subgroups (< 65 versus ≥ 65 years: $26,520 versus $2298/QALY-gained), eGFR subgroups (60 ~ < 90 versus ≥ 90 ml/min/1.73 m²: $7700 versus $12,884/QALY-gained), HbA1c subgroups (< 8.5 versus ≥ 8.5%: $7001 versus $9488/QALY-gained), and BMI subgroups (< 30 versus ≥ 30 kg/m²: $7266 versus $9714/QALY-gained). Over a lifetime, the ICERs became lower, ranging from $2369/QALY-gained for those aged ≥ 65 years to $4239/QALY-gained for those aged < 65 years. Over 5 years, the annual VBP of SGLT2is ranged from $310 for those aged < 65 years to $1267 for those aged ≥ 65 years.</p><p><strong>Conclusions: </strong>Our analysis suggests that adopting SGLT2is over DPP4is for T2D is highly cost-effective across patient subgroups, particularly for the elderly and patients with mild renal impairment.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145054929","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Eco Friendly and Budget Smart: An Economic and Environmental Evaluation of Alternative PD-1 and PD-L1 Inhibitor Dosing Regimens.","authors":"Leo Karlsson, Joseph Ciccolini, Rob Ter Heine, Maddalena Centanni","doi":"10.1007/s40273-025-01535-7","DOIUrl":"https://doi.org/10.1007/s40273-025-01535-7","url":null,"abstract":"<p><strong>Background: </strong>Immune checkpoint inhibitors (ICIs) are clinically beneficial but associated with high costs that represent a growing challenge for healthcare budgets and may affect affordability, especially in resource-limited settings. Moreover, the healthcare sector is a significant source of greenhouse gas emissions, and medication-related waste-such as that from vial-based therapies-has been identified as a contributing factor. Alternative dosing strategies could reduce the environmental and financial impact of ICI therapy while maintaining clinical safety and efficacy.</p><p><strong>Methods: </strong>Population pharmacokinetic simulations were performed using virtual cohorts representative of the original cancer populations treated with ICIs. The analysis was conducted from a Western European hospital perspective, using Dutch public data to estimate costs (based on volume-dependent pricing) and carbon emissions from drug production, travel, and medical waste.</p><p><strong>Results: </strong>Under the US Food and Drug Administration exposure-matching criteria, optimized dosing regimens reduced drug costs by up to €23,311 (- 28%) and carbon emissions by up to 255 kgCO₂e (- 30%) per patient, depending on the drug and dosing strategy. Using a broader therapeutic window approach, cost savings reached up to €40,135 (- 69%) and carbon reductions up to 501 kgCO₂e (- 63%) per patient. Incorporating vial sharing further increased potential cost savings to €5,721 per patient (- 31%). All estimates reflect European pricing and emissions factors, modeled over an 8-month treatment period.</p><p><strong>Conclusions: </strong>These findings suggest that optimizing dosing strategies can yield meaningful economic and environmental benefits in ICI therapy while maintaining drug exposure within levels defined by US Food and Drug Administration criteria or broader therapeutic windows. A user-friendly application developed in this study allows users to generate virtual populations and evaluate tailored dosing strategies, facilitating practical implementation in diverse healthcare settings.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145030215","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"HTA Evidence in Rare Diseases: Just Rare or Also Special?","authors":"Anirban Basu, Simu K Thomas, Richard H Chapman, Jason Spangler","doi":"10.1007/s40273-025-01538-4","DOIUrl":"https://doi.org/10.1007/s40273-025-01538-4","url":null,"abstract":"<p><p>Manufacturers of orphan drugs face several obstacles in meeting health technology assessment requirements because of poor availability of natural history data, small sample sizes, single-arm trials, and a paucity of established disease-specific endpoints. There is a need for specific considerations and modified approaches in health technology assessments that would account for the challenges in orphan drug development. Multistakeholder collaborations can benefit patients, their families, and the broader society and reduce the inequity faced by patients with rare diseases.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145024009","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Highlights from the Manifesto on the Health Economics of Cardiovascular Disease Prevention.","authors":"Zanfina Ademi, Sheridan E Rodda, Karl Vivoda, Susan Hennessy, Olive Fenton, James S Ware","doi":"10.1007/s40273-025-01537-5","DOIUrl":"https://doi.org/10.1007/s40273-025-01537-5","url":null,"abstract":"<p><p>Cardiovascular disease (CVD) is a major contributor to the health and economic burden of disease globally. In this paper we discuss the literature on the health economics of the prevention and early intervention in CVD. We reveal the large economic impact of CVD and provide the economic argument supporting the calls for early detection and diagnosis of CVD outlined in the Global Heart Hub's patient-led Manifesto for Change. Many challenges in conducting cost-effectiveness analyses of interventions for CVD prevention are identified, as well as the emerging statistical and economic methods to help overcome these issues. Lastly, we acknowledge the profound disparities in cardiovascular health faced by minority or underserved populations, and the important role that prevention and early intervention can play in improving health equity.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145023980","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Global Assessment of Health Utilities Associated with Pneumococcal Disease in Children-Targeted Literature Reviews.","authors":"Min Huang, Jipan Xie, Hela Romdhani, Yan Song, Sun Lee, Daisy Liu, Elamin Elbasha, Salini Mohanty, Donna Rowen, Matthew S Kelly","doi":"10.1007/s40273-025-01504-0","DOIUrl":"10.1007/s40273-025-01504-0","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Pneumococcal disease can significantly impact the quality of life (QoL) of children. Health utilities are used to measure the disease burden and calculate quality-adjusted life year (QALY) estimates. These estimates provide critical inputs in economic evaluations of pneumococcal vaccines in children.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;This study aimed to synthesize utility values used in cost-utility analyses (CUAs) of pediatric pneumococcal vaccines and to summarize published utility studies on pneumococcal disease and post-meningitis sequelae (PMS) in children on a global scale.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Two targeted literature reviews were conducted to identify CUAs of pediatric pneumococcal vaccines and original studies on health utilities of pneumococcal disease and PMS. Both literature reviews identified relevant studies using published reviews, supplemented by de novo searches conducted in MEDLINE in June 2024 to cover periods not included in those reviews. References from published literature reviews on QoL of pneumococcal disease and CUAs were screened to identify additional original utility studies. Health utility values applied in the CUAs were summarized and the source studies for these utilities were reviewed. For original utility studies, methods and utility estimates were summarized for each condition.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The study identified 45 CUAs of pediatric pneumococcal vaccines in North America and Europe published from 2004 to 2024, and 21 original utility studies on pneumococcal disease or PMS in children published globally from 1994 to 2017. QALY decrement was the most common utility input in CUAs. Most CUAs referenced an earlier CUA for utility inputs, which were often sourced from one or two original utility studies for each health state. Most source studies were published more than two decades ago; some common source studies were conducted in adults. Utility estimates from original studies showed considerable variability, with ranges of -0.330 to 0.6882 for meningitis, -0.331 to 0.93 for non-meningitis invasive pneumococcal disease (IPD), -0.054 to 0.71 for inpatient pneumonia, 0.412-0.82 for outpatient pneumonia, 0.389-0.97 for acute otitis media (AOM)/simple AOM, 0.434-0.540 for recurrent AOM, -0.33 to 0.89 for neurological deficits, and 0.217-0.97 for hearing loss. Variability in methods, including in the surveyed population, utility elicitation method, and use of different country-specific preference weights, substantially impacted utility values. Overall, the methods were not suitable for temporary health states. Additionally, many studies used instruments that have not been validated in children.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Original utility studies demonstrated that pneumococcal disease and PMS are associated with impaired QoL in children; however, there was considerable variability in utility estimates across studies, reflecting the inherent methodologica","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1001-1045"},"PeriodicalIF":4.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144128234","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Innovation Headroom for a Highly Accurate PD-L1 Companion Diagnostic in Non-small Cell Lung Cancer.","authors":"Toluwase Akinsoji, Nick Dragojlovic, Cécile Darviot, Michel Meunier, Mark Harrison, Larry D Lynd","doi":"10.1007/s40273-025-01512-0","DOIUrl":"10.1007/s40273-025-01512-0","url":null,"abstract":"<p><strong>Background and objective: </strong>Companion diagnostics (CDx) are critical to precision medicine. Developing and commercializing new CDx faces regulatory and economic challenges. This study aims to illustrate the utility of an early health technology assessment in quantifying the unmet clinical need and commercial opportunity created by the limited accuracy of existing programmed cell death ligand 1 CDx.</p><p><strong>Methods: </strong>The study uses an early health technology assessment and market sizing to assess the potential value of a novel programmed cell death ligand 1 CDx for non-small cell lung cancer (NSCLC). Decision tree-based cost-effectiveness models were used to evaluate clinical and economic outcomes for improved programmed cell death ligand 1 testing in atezolizumab-treated patients with stage II-IIIA and metastatic NSCLC from a US payer perspective in 2023 US Dollars. Three strategies were examined: standard care, new CDx for cytology specimens only, and new CDx for all patients. Commercial opportunities from the perspectives of diagnostics and pharmaceutical manufacturers were assessed using headroom and threshold analyses.</p><p><strong>Results: </strong>Headroom analyses indicated that a new CDx is not cost effective for metastatic NSCLC but holds significant value for stage II-IIIA NSCLC. Assuming perfect sensitivity and specificity, the incremental cost-effectiveness ratio for the new CDx in stage II-IIIA NSCLC was $57,650/quality-adjusted life-year and $54,950/quality-adjusted life-year for cytology specimens only and all patients, respectively. A threshold analysis showed that at a $500 price point, the new CDx is cost effective at sensitivity levels of 0.9 for all patients and 0.8 for cytology only. The total addressable US market for the CDx manufacturer was estimated at $2.6 million per year with a $500/test kit price.</p><p><strong>Conclusions: </strong>A novel, highly accurate CDx for stage II-IIIA NSCLC could provide significant value to patients, payers, and manufacturers.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1135-1145"},"PeriodicalIF":4.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144333728","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}