PharmacoEconomics Open最新文献

{"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":"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":"83-92"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11717742/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142292828","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":"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":"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":"147-160"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11717771/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142471895","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 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":"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":"41-56"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11718032/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142392323","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-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":"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":"93-101"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11718036/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142392324","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":"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":"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":"57-68"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11717749/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142140742","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-Effectiveness of Three Different New-Generation Drug-Eluting Stents in the Randomised BIO-RESORT Trial at 3 Years.","authors":"Eline H Ploumen, Martijn J Oude Wolcherink, Rosaly A Buiten, Tineke H Pinxterhuis, Carine J M Doggen, Carl E Schotborgh, Peter W Danse, Martijn Scholte, K Gert van Houwelingen, Paolo Zocca, Xavier G L V Pouwels, Clemens von Birgelen","doi":"10.1007/s41669-024-00539-x","DOIUrl":"10.1007/s41669-024-00539-x","url":null,"abstract":"<p><strong>Background and objective: </strong>Evidence on health economic outcomes for percutaneous coronary intervention (PCI) comparing different contemporary drug-eluting stents (DES) with each other is scarce, as most previous randomised DES trials did not assess such aspects. This prespecified health economic evaluation of the Comparison of Biodegradable Polymer and Durable Polymer Drug-Eluting Stents in an All Comers Population (BIO-RESORT) trial aimed to compare at 3-year follow-up both health effects and costs of PCI with one of three new-generation drug-eluting stents (DES) in patients with obstructive coronary artery disease.</p><p><strong>Methods: </strong>The randomised BIO-RESORT trial assessed in 3514 patients the ultrathin-strut biodegradable polymer Orsiro sirolimus-eluting stent (SES) and very-thin-strut Synergy everolimus-eluting (EES) stent versus the thin-strut durable polymer Resolute Integrity zotarolimus-eluting stent (ZES). In the current analysis, we used the perspective of a health insurer in the Netherlands. The main endpoints were quality-adjusted life years (QALYs), and costs for each treatment strategy. Bootstrapping with 5000 resamples was performed to capture the uncertainty of results.</p><p><strong>Results: </strong>Mean QALYs for each stent group were 2.566 for the SES, 2.551 for the EES, and 2.550 for the ZES. Mean costs per strategy were €14,670 for the SES, €14,946 for the EES, and €15,069 for the ZES. The SES had the highest probability of being cost-effective for every willingness-to-pay threshold up to €100,000 per QALY. Furthermore, in 79% of modelling scenarios, the SES was more effective and cheaper than ZES.</p><p><strong>Conclusion: </strong>At 3-year follow-up, PCI with the SES had the highest probability of being cost-effective due to greater effectiveness and lower costs compared with the ZES and EES. These findings suggest that, due to the overall high volume of coronary stenting in clinical practice, use of this SES could result in substantial cost savings, complemented by slight additional health benefits.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"137-145"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11718029/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142546692","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":"Digital Versus Paper-Based Consent from the UK NHS Perspective: A Micro-costing Analysis.","authors":"Rachel Houten, Mohammad Iqbal Hussain, Antony P Martin, Nick Ainsworth, Claudia Lameirinhas, Alexander W Coombs, Simon Toh, Christopher Rao, Edward St John","doi":"10.1007/s41669-024-00536-0","DOIUrl":"10.1007/s41669-024-00536-0","url":null,"abstract":"<p><strong>Background: </strong>The paper-based consent pathway can be associated with missing information, error, and inadequate patient comprehension. Digital consent addresses some of these limitations. However, limited research has been conducted to understand relative costs and consequences associated with adopting digital consent pathways. The aim of this study was to compare the relative costs of digital consent pathways with paper-based consent pathways in UK National Health Service (NHS) clinical practice.</p><p><strong>Method: </strong>A micro-costing study was conducted from the UK NHS perspective. Multi-stakeholder involvement contributed to understanding how the paper-based consent pathway varies by department and hospital setting. Sensitivity analyses were conducted to identify the key cost drivers and scenario analyses explored the effect of consent timing and hospital digital readiness. Potential advantages and disadvantages of digital consent were also considered, such as possible impacts associated with consent-related litigation.</p><p><strong>Results: </strong>The cost per consent episode is approximately £0.90 more expensive when completed on paper. The ordering or printing of paper consent forms, and the transportation of forms to storage and back to clinic are process steps that would not be necessary with digital consent. Sensitivity and scenario analyses indicated consultation duration had the greatest impact on the relative costs of both pathways. Per litigation claim prevented, an average of £201,590 could be saved.</p><p><strong>Conclusions: </strong>Digital consent is potentially cost saving for the NHS. Consent for elective procedures is recommended in advance of the day of surgery, and digital consent used in this scenario demonstrated the greatest savings. Consultation duration was estimated to have the greatest impact on the relative costs of both pathways, which should be a focus of further investigation.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"27-39"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11717759/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583260","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":"Costs of Adverse Events in Patients with Advanced or Metastatic Renal Cell Carcinoma with First-Line Treatment.","authors":"Yan Chen, Ella X Du, Manasvi Sundar, Keith A Betts, Xin Yin, Samantha Eiffert, Karen Beauchamp, Andrew Delgado, Lisa Rosenblatt","doi":"10.1007/s41669-024-00534-2","DOIUrl":"10.1007/s41669-024-00534-2","url":null,"abstract":"<p><strong>Aim: </strong>This study evaluated costs associated with adverse events (AEs) in previously untreated real-world patients with advanced renal cell carcinoma (aRCC) in the USA.</p><p><strong>Materials and methods: </strong>This retrospective longitudinal cohort study analyzed data from the Merative MarketScan Research Database (1 January 2014-30 September 2021). Adult patients with aRCC receiving first-line systemic treatments for aRCC (tyrosine kinase inhibitors [TKIs], or combination therapies of TKIs and immunotherapy) on or after the date of aRCC diagnosis were included. A total of 27 AEs of interest were included based on a review of product labels of the first-line treatments included in the study and identified using International Classification of Diseases, Ninth/Tenth Revision, Clinical Modification codes. Incremental costs associated with AEs between cases and controls (unadjusted and adjusted for relevant baseline characteristics) were estimated by two-part modeling. Analyses were performed over three AE cost assessment periods (7, 14, and 30 days).</p><p><strong>Results: </strong>The study included 1681 patients with aRCC (mean [standard deviation; SD] age, 60.8 [10.6] years; 73.1% male), of which 1542 (91.7%) had at least one AE. AEs were mostly diagnosed in the outpatient (OP) setting. For most AEs, cases had significantly higher unadjusted and adjusted costs than controls. Costs associated with AEs ranged from < 300 US dollars (USD) for proteinuria to nearly 60,000 USD for hypophosphatemia. Seventeen AEs had adjusted 30-day costs exceeding 10,000 USD; of these, nine (pancreatitis, acute kidney injury, dyspnea, hypotension, hyperkalemia, hypomagnesemia, hyponatremia, hypophosphatemia, and neutrophil decreased/neutropenia) had 30-day costs exceeding 20,000 USD.</p><p><strong>Limitations: </strong>The study was subject to limitations of all observational analyses of claims data (e.g., residual confounding). Observed cost differences may not have been solely attributable to an AE of interest. Study findings may not be generalizable to aRCC patient populations outside the USA.</p><p><strong>Conclusion: </strong>Most patients experienced at least one AE after initiation of first-line treatment with a TKI or combination therapies of TKIs and immunotherapy. There were substantial costs associated with AEs. Considering both safety and efficacy profiles when selecting optimal treatments can potentially mitigate healthcare costs for aRCC.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"125-136"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11718028/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142590803","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":"CMS's Drug Price Negotiation Program 2023 Patient-Focused Listening Sessions: A Descriptive Analysis of Speaker Remarks.","authors":"Julie A Patterson, Tyler D Wagner, Rayan K Salih, Gabri'el D Shabazz, Jonathan D Campbell","doi":"10.1007/s41669-024-00530-6","DOIUrl":"10.1007/s41669-024-00530-6","url":null,"abstract":"<p><strong>Background: </strong>The US Centers for Medicare and Medicaid Services (CMS) held patient-focused listening sessions in Fall 2023 for each of the first ten drugs selected for the Inflation Reduction Act's (IRA) Drug Price Negotiation Program (DPNP). This study aimed to quantitatively describe speaker input at the sessions, including the absolute and relative time allocated to key areas of interest for the DPNP.</p><p><strong>Methods: </strong>In this descriptive analysis of speaker remarks from ten CMS-hosted patient-focused listening sessions, speaker demographics were examined using video streams, time-stamped transcripts, public data, contextual clues, validated tools, and visual assessment when necessary. Audio recordings were transcribed using a speech-to-text application programming interface, followed by human review for accuracy. Transcripts included speaker identification and sentence-level timestamping. Two researchers coded each sentence into predefined categories on the basis of CMS's optional discussion topics, DPNP goals, and potential unintended consequences of the IRA: speaker background, evidence about selected drugs or therapeutic alternatives, drug prices, patient access/benefit design, therapeutic advancements, and other. The total and proportion of time spent by category were calculated per speaker.</p><p><strong>Results: </strong>Out of an anticipated 200 speaker slots, 106 total speakers (median per session: 10; range 5-17) and 70 unique speakers participated, most of whom were patients (n = 38, 54.2%), female (n = 40, 57.1%), and below Medicare age (n = 40, 57.1%). The most popular categories discussed included evidence about the selected drug or therapeutic alternatives (median per slot: 36.5% of time; range 0-93.0%) and patient access/benefit design (median per slot: 12.1%; range 0-75.5%). CMS received a median of 12.3 total minutes of evidence about selected drugs or therapeutic alternatives per listening session across all speakers (range per session 3.9-22.6 minutes).</p><p><strong>Conclusions: </strong>The first listening sessions provided an important, but limited, first step toward patient engagement with the DPNP. Speaker demographics suggest the sessions fell short of capturing the voices of diverse and Medicare-representative patient populations. While speakers often focused their time on patient experience and evidence, CMS received less than 15 minutes of patient-focused evidence about therapeutic alternatives for more than half of the drug listening sessions. CMS has indicated its intent to improve upon the design of the patient-focused listening sessions for drugs selected in the second round of the DPNP. Future iterations should prioritize robust, two-way dialogue, transparency surrounding the impact of patient input on CMS's price determination process, and improvements to event logistics and outreach that facilitate the participation of diverse patients and stakeholders.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"103-113"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11717761/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625722","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":"Health Economic Evaluation of Antimicrobial Stewardship, Procalcitonin Testing, and Rapid Blood Culture Identification in Sepsis Care: A 90-Day Model-Based, Cost-Utility Analysis.","authors":"Wendy I Sligl, Charles Yan, Jeff Round, Xiaoming Wang, Justin Z Chen, Cheyanne Boehm, Karen Fong, Katelynn Crick, Míriam Garrido Clua, Cassidy Codan, Tanis C Dingle, Connie Prosser, Guanmin Chen, Alena Tse-Chang, Daniel Garros, David Zygun, Dawn Opgenorth, John M Conly, Christopher J Doig, Vincent I Lau, Sean M Bagshaw","doi":"10.1007/s41669-024-00538-y","DOIUrl":"10.1007/s41669-024-00538-y","url":null,"abstract":"<p><strong>Objective: </strong>We evaluated the cost-effectiveness of a bundled intervention including an antimicrobial stewardship program (ASP), procalcitonin (PCT) testing, and rapid blood culture identification (BCID), compared with pre-implementation standard care in critically ill adult patients with sepsis.</p><p><strong>Methods: </strong>We conducted a decision tree model-based cost-effectiveness analysis alongside a previously published pre- and post-implementation quality improvement study. We adopted a public Canadian healthcare payer's perspective. Two intensive care units in Alberta with 727 adult critically ill patients were included. Our bundled intervention was compared with pre-implementation standard care. We collected healthcare resource use and estimated unit costs in 2022 Canadian dollars (CAD) over a time horizon from study entry to hospital discharge or death. We calculated the incremental net monetary benefit (iNMB) of the intervention group compared with the pre-intervention group. The primary outcome was cost per sepsis case. Secondary outcomes included readmission rates, Clostridioides difficile infections, mortality, and lengths of stay. Uncertainty was investigated using cost-effectiveness acceptability curves, cost-effectiveness plane scatterplots, and sensitivity analyses.</p><p><strong>Results: </strong>Mean (standard deviation [SD]) cost per index hospital admission was CAD $83,251 ($107,926) for patients in the intervention group and CAD $87,044 ($104,406) for the pre-intervention group, though the difference ($3,793 [$7,897]) was not statistically significant. Costs were higher in the pre-intervention group for antibiotics, readmissions, and C. difficile infections. The intervention group had a lower mean expected cost; $110,580 ($108,917) compared with pre-intervention ($125,745 [$113,210]), with a difference of $15,165 ($8278). There were no statistically significant differences in quality adjusted life years (QALYs) between groups. The iNMB of the intervention group compared with pre-intervention was greater than $15,000 for willingness-to-pay (WTP) per QALY values of between $0 and $100,000. In our sensitivity analysis, the intervention was most likely to be cost-effective in roughly 56% of simulations at all WTP thresholds.</p><p><strong>Conclusions: </strong>Our bundled intervention of ASP, PCT, and BCID among adult critically ill patients with sepsis was potentially cost-effective, but with substantial decision uncertainty.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"15-25"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11718022/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142668597","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}