Journal of Health Economics and Outcomes Research最新文献_第8页

The Effects of Adverse Events and Associated Costs on Value-Based Care for Metastatic Pancreatic Ductal Adenocarcinoma. 不良事件和相关费用对转移性胰腺导管腺癌基于价值的护理的影响。

IF 2.3 Q2 ECONOMICS

Journal of Health Economics and Outcomes Research

Pub Date : 2024-12-18 eCollection Date: 2024-01-01 DOI: 10.36469/001c.124367

Prachi Bhatt, Jared Hirsch, Paul Cockrum, George Kim, Gabriela Dieguez

Background: Rising oncology healthcare costs have led to value-based care reimbursement models that coordinate care and improve quality while reducing overall spending. These models are increasingly important for traditional Medicare and other payers. Objectives: To compare the incidence of adverse events (AEs), AE-associated excess costs, and total cost of care (TCOC) of 3 cohorts receiving first-line treatment for metastatic pancreatic ductal adenocarcinoma (mPDAC). Methods: We conducted a retrospective analysis of administrative claims data from 2018 to 2022 using the Medicare 100% Research Identifiable Files. We examined 3 cohorts receiving mPDAC treatment: FOLFIRINOX (FFX) (oxaliplatin, irinotecan, leucovorin, 5-FU bolus and infusion); modified FFX, (5-FU infusion only); and gemcitabine/nab-paclitaxel (gem/abrax). We compared the incidence of clinically significant AEs, TCOC, components of TCOC, and costs related to AEs/treatment toxicity. Results: Patient AE rates ranged from 6.2% to 51.7%. AEs occurred more frequently in patients receiving FFX with all 4 components. Patients receiving brand name gem/abrax had lower rates of febrile neutropenia (6.2%) and neutropenia (22.2%) than those receiving FFX with no 5-FU bolus (febrile neutropenia, 9.9%; neutropenia, 36.9%) and FFX with all 4 components (febrile neutropenia, 6.9%; neutropenia, 30.4%). Rates of most nonhematologic AEs were higher in patients receiving FFX with all 4 components, with diarrhea occurring in 28.3%, abdominal pain in 31.5%, and nausea/vomiting in 41.5% of patients. TCOC was lower in the gem/abrax cohort: <math><mn>6505</mn> <mi>v</mi> <mi>s</mi> <mi>F</mi> <mi>F</mi> <mi>X</mi> <mi>w</mi> <mi>i</mi> <mi>t</mi> <mi>h</mi> <mi>n</mi> <mi>o</mi> <mn>5</mn> <mo>-</mo> <mi>F</mi> <mi>U</mi> <mi>b</mi> <mi>o</mi> <mi>l</mi> <mi>u</mi> <mi>s</mi> <mo>(</mo></math> 6995) and FFX with all 4 components ( <math><mn>7142</mn> <mo>)</mo> <mi>p</mi> <mi>e</mi> <mi>r</mi> <mi>a</mi> <mi>d</mi> <mi>m</mi> <mi>i</mi> <mi>n</mi> <mi>i</mi> <mi>s</mi> <mi>t</mi> <mi>r</mi> <mi>a</mi> <mi>t</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mo>.</mo> <mi>T</mi> <mi>h</mi> <mi>e</mi> <mi>d</mi> <mi>e</mi> <mi>v</mi> <mi>e</mi> <mi>l</mi> <mi>o</mi> <mi>p</mi> <mi>m</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>o</mi> <mi>f</mi> <mi>a</mi> <mi>n</mi> <mi>y</mi> <mi>s</mi> <mi>t</mi> <mi>u</mi> <mi>d</mi> <mi>i</mi> <mi>e</mi> <mi>d</mi> <mi>h</mi> <mi>e</mi> <mi>m</mi> <mi>a</mi> <mi>t</mi> <mi>o</mi> <mi>l</mi> <mi>o</mi> <mi>g</mi> <mi>i</mi> <mi>c</mi> <mi>A</mi> <mi>E</mi> <mi>w</mi> <mi>a</mi> <mi>s</mi> <mi>a</mi> <mi>s</mi> <mi>s</mi> <mi>o</mi> <mi>c</mi> <mi>i</mi> <mi>a</mi> <mi>t</mi> <mi>e</mi> <mi>d</mi> <mi>w</mi> <mi>i</mi> <mi>t</mi> <mi>h</mi> <mi>a</mi> <mi>m</mi> <mi>e</mi> <mi>a</mi> <mi>n</mi> <mi>e</mi> <mi>x</mi> <mi>c</mi> <mi>e</mi> <mi>s</mi> <mi>s</mi> <mi>c</mi> <mi>o</mi> <mi>s</mi> <mi>t</mi> <mi>o</mi> <mi>f</mi></math> 5993 per administration,

背景：不断上升的肿瘤医疗保健费用导致了基于价值的护理报销模式，协调护理和提高质量，同时减少总体支出。这些模式对传统医疗保险和其他支付者越来越重要。目的：比较3个队列接受转移性胰腺导管腺癌（mPDAC）一线治疗的不良事件（ae）发生率、ae相关的超额费用和总护理成本（TCOC）。方法：我们使用医疗保险100%研究可识别文件对2018年至2022年的行政索赔数据进行回顾性分析。我们检查了3个接受mPDAC治疗的队列：FOLFIRINOX (FFX)（奥沙利铂、伊立替康、亚叶酸素、5-FU丸和输注）；改良的FFX（仅5-FU输注）；吉西他滨/nab-紫杉醇（gem/abrax）。我们比较了临床显著ae的发生率、TCOC、TCOC成分以及与ae /治疗毒性相关的费用。结果：AE发生率为6.2% ~ 51.7%。所有4种成分均接受FFX治疗的患者发生不良事件的频率更高。接受gem/abrax品牌治疗的患者发热性中性粒细胞减少率（6.2%）和中性粒细胞减少率（22.2%）低于接受FFX治疗但不服用5-FU的患者(发热性中性粒细胞减少，9.9%；中性粒细胞减少症，36.9%)和所有4种成分的FFX(发热性中性粒细胞减少症，6.9%；嗜中性白血球减少症,30.4%)。所有4种成分均接受FFX治疗的患者中，大多数非血液学不良事件发生率较高，其中腹泻发生率为28.3%，腹痛发生率为31.5%，恶心/呕吐发生率为41.5%。TCOC较低的宝石/ abrax群:6505 v s F F X w i t h n o 5 - F U l b o s(6995)和FFX所有4组件(7142)p e r d m我n s t r t i o n。T h e d e v e l o p m e n T o f n y s T u i e d h e m T o d l o g i c a e w s s s o c i T e d w i T h m e n e x c e s s c o s T o f 5993 /政府,而任何的发展研究nonhematological AE与平均per-administration超过3665美元的成本。讨论：如果目标是降低TCOC，以最小化化疗费用为目的的治疗决策可能导致次优决策。我们的研究表明，FFX比gem/abrax更昂贵（每次给药的TCOC）。接受gem/abrax治疗的患者年龄较大，基线Charlson合并症指数评分较高；然而，在造成成本差异方面，其他因素可能也很重要。结论：无论药物成本如何，化疗导致ae显著增加与较高的TCOC相关。

{"title":"The Effects of Adverse Events and Associated Costs on Value-Based Care for Metastatic Pancreatic Ductal Adenocarcinoma.","authors":"Prachi Bhatt, Jared Hirsch, Paul Cockrum, George Kim, Gabriela Dieguez","doi":"10.36469/001c.124367","DOIUrl":"10.36469/001c.124367","url":null,"abstract":"Background: Rising oncology healthcare costs have led to value-based care reimbursement models that coordinate care and improve quality while reducing overall spending. These models are increasingly important for traditional Medicare and other payers. Objectives: To compare the incidence of adverse events (AEs), AE-associated excess costs, and total cost of care (TCOC) of 3 cohorts receiving first-line treatment for metastatic pancreatic ductal adenocarcinoma (mPDAC). Methods: We conducted a retrospective analysis of administrative claims data from 2018 to 2022 using the Medicare 100% Research Identifiable Files. We examined 3 cohorts receiving mPDAC treatment: FOLFIRINOX (FFX) (oxaliplatin, irinotecan, leucovorin, 5-FU bolus and infusion); modified FFX, (5-FU infusion only); and gemcitabine/nab-paclitaxel (gem/abrax). We compared the incidence of clinically significant AEs, TCOC, components of TCOC, and costs related to AEs/treatment toxicity. Results: Patient AE rates ranged from 6.2% to 51.7%. AEs occurred more frequently in patients receiving FFX with all 4 components. Patients receiving brand name gem/abrax had lower rates of febrile neutropenia (6.2%) and neutropenia (22.2%) than those receiving FFX with no 5-FU bolus (febrile neutropenia, 9.9%; neutropenia, 36.9%) and FFX with all 4 components (febrile neutropenia, 6.9%; neutropenia, 30.4%). Rates of most nonhematologic AEs were higher in patients receiving FFX with all 4 components, with diarrhea occurring in 28.3%, abdominal pain in 31.5%, and nausea/vomiting in 41.5% of patients. TCOC was lower in the gem/abrax cohort: <math><mn>6505</mn> <mi>v</mi> <mi>s</mi> <mi>F</mi> <mi>F</mi> <mi>X</mi> <mi>w</mi> <mi>i</mi> <mi>t</mi> <mi>h</mi> <mi>n</mi> <mi>o</mi> <mn>5</mn> <mo>-</mo> <mi>F</mi> <mi>U</mi> <mi>b</mi> <mi>o</mi> <mi>l</mi> <mi>u</mi> <mi>s</mi> <mo>(</mo></math> 6995) and FFX with all 4 components ( <math><mn>7142</mn> <mo>)</mo> <mi>p</mi> <mi>e</mi> <mi>r</mi> <mi>a</mi> <mi>d</mi> <mi>m</mi> <mi>i</mi> <mi>n</mi> <mi>i</mi> <mi>s</mi> <mi>t</mi> <mi>r</mi> <mi>a</mi> <mi>t</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mo>.</mo> <mi>T</mi> <mi>h</mi> <mi>e</mi> <mi>d</mi> <mi>e</mi> <mi>v</mi> <mi>e</mi> <mi>l</mi> <mi>o</mi> <mi>p</mi> <mi>m</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>o</mi> <mi>f</mi> <mi>a</mi> <mi>n</mi> <mi>y</mi> <mi>s</mi> <mi>t</mi> <mi>u</mi> <mi>d</mi> <mi>i</mi> <mi>e</mi> <mi>d</mi> <mi>h</mi> <mi>e</mi> <mi>m</mi> <mi>a</mi> <mi>t</mi> <mi>o</mi> <mi>l</mi> <mi>o</mi> <mi>g</mi> <mi>i</mi> <mi>c</mi> <mi>A</mi> <mi>E</mi> <mi>w</mi> <mi>a</mi> <mi>s</mi> <mi>a</mi> <mi>s</mi> <mi>s</mi> <mi>o</mi> <mi>c</mi> <mi>i</mi> <mi>a</mi> <mi>t</mi> <mi>e</mi> <mi>d</mi> <mi>w</mi> <mi>i</mi> <mi>t</mi> <mi>h</mi> <mi>a</mi> <mi>m</mi> <mi>e</mi> <mi>a</mi> <mi>n</mi> <mi>e</mi> <mi>x</mi> <mi>c</mi> <mi>e</mi> <mi>s</mi> <mi>s</mi> <mi>c</mi> <mi>o</mi> <mi>s</mi> <mi>t</mi> <mi>o</mi> <mi>f</mi></math> 5993 per administration, ","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"161-167"},"PeriodicalIF":2.3,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11664866/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142882196","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}

引用次数: 0

Could the Inflation Reduction Act Maximum Fair Price Hurt Patients? 《通货膨胀削减法案》最大限度地提高公平价格会伤害患者吗？

IF 2.3 Q2 ECONOMICS

Journal of Health Economics and Outcomes Research

Pub Date : 2024-11-27 eCollection Date: 2024-01-01 DOI: 10.36469/001c.125251

Anne M Sydor, Esteban Rivera, Robert Popovian

Background: The Inflation Reduction Act's Medicare Drug Price Negotiation Program allows the federal government to negotiate caps for select medications. These price caps may reduce revenue for the pharmacy benefit managers (PBMs) that negotiate the actual price paid for medicines in the U.S. To offset the resulting pressure on their profit margins, it is possible that PBMs would, in turn, increase patients' out-of-pocket costs for medicines with capped prices. The model presented here evaluates how increased out-of-pocket costs for the anticoagulants apixaban (Eliquis) and rivaroxaban (Xarelto) could impact patients financially and clinically. Methods: Copay distributions for all 2023 prescription fills for apixaban and rivaroxaban managed by the 3 largest PBMs, CVS Caremark, Express Scripts International, and Optum Rx, were used to approximate current copay costs. Increased out-of-pocket costs were modeled as a shift of all apixaban and rivaroxaban prescriptions to the highest copay tier. The known linear relationship between copay costs and treatment abandonment was used to calculate the potential resulting increase in treatment abandonment. Known rates of morbidity and mortality due to abandoning anticoagulants were used to estimate resulting increases in morbidity and mortality. Results: If the 3 largest PBMs all shifted costs onto patients by moving all apixaban and rivaroxaban prescriptions to the highest formulary tier, Tier 6, patients' copay amount would increase by $235 t o$ 482 million for apixaban and $105 t o$ 206 million for rivaroxaban. Such an increase could lead to 169 000 to 228 000 patients abandoning apixaban and 71 000 to 93 000 abandoning rivaroxaban. The resulting morbidity and mortality could include up to an additional 145 000 major cardiovascular events and up to 97 000 more deaths. Conclusion: The Medicare Price Negotiation Program could impact patients negatively if it causes PBMs to increase patients' out-of-pocket costs for medicines. Policymakers should closely monitor changes in overall affordability, including all patient out-of-pocket expenditures, for medications in the program. Preemptive measures should be considered to ensure that the most vulnerable citizens are not placed in precarious situations, leading to poorer health outcomes.

背景：《减少通货膨胀法案》的医疗保险药品价格谈判项目允许联邦政府就选定药品的价格上限进行谈判。这些价格上限可能会减少在美国协商药品实际支付价格的药品福利管理机构（PBMs）的收入。为了抵消由此对其利润率造成的压力，PBMs可能会反过来增加患者购买价格上限药品的自付费用。本文提出的模型评估了抗凝药物阿哌沙班（Eliquis）和利伐沙班（Xarelto）的自付费用增加对患者经济和临床的影响。方法：使用CVS Caremark、Express Scripts International和Optum Rx这3家最大的药品管理公司管理的所有2023种阿哌沙班和利伐沙班处方的共付分布来估计当前的共付成本。增加的自付费用被建模为所有阿哌沙班和利伐沙班处方转移到最高的共付等级。已知的共同支付费用和放弃治疗之间的线性关系被用来计算可能导致的放弃治疗的增加。已知的因放弃抗凝剂而导致的发病率和死亡率被用来估计由此导致的发病率和死亡率的增加。结果：如果最大的3家pbm将所有阿哌沙班和利伐沙班的处方全部转移到最高处方级第6层，将成本转移到患者身上，阿哌沙班患者的共付金额将增加2.35亿至4.82亿美元，利伐沙班患者的共付金额将增加1.05亿至2.06亿美元。这种增加可能导致16.9万至22.8万名患者放弃阿哌沙班，7.1万至9.3万名患者放弃利伐沙班。由此造成的发病率和死亡率可能包括多达14.5万例重大心血管事件和多达9.7万例死亡。结论：医疗保险价格谈判项目如果导致药品管理机构增加患者自付药品费用，可能会对患者产生负面影响。政策制定者应密切监测总体可负担性的变化，包括该计划中所有患者的自付费用。应考虑采取先发制人的措施，确保最脆弱的公民不会处于不稳定的境地，从而导致较差的健康结果。

{"title":"Could the Inflation Reduction Act Maximum Fair Price Hurt Patients?","authors":"Anne M Sydor, Esteban Rivera, Robert Popovian","doi":"10.36469/001c.125251","DOIUrl":"10.36469/001c.125251","url":null,"abstract":"Background: The Inflation Reduction Act's Medicare Drug Price Negotiation Program allows the federal government to negotiate caps for select medications. These price caps may reduce revenue for the pharmacy benefit managers (PBMs) that negotiate the actual price paid for medicines in the U.S. To offset the resulting pressure on their profit margins, it is possible that PBMs would, in turn, increase patients' out-of-pocket costs for medicines with capped prices. The model presented here evaluates how increased out-of-pocket costs for the anticoagulants apixaban (Eliquis) and rivaroxaban (Xarelto) could impact patients financially and clinically. Methods: Copay distributions for all 2023 prescription fills for apixaban and rivaroxaban managed by the 3 largest PBMs, CVS Caremark, Express Scripts International, and Optum Rx, were used to approximate current copay costs. Increased out-of-pocket costs were modeled as a shift of all apixaban and rivaroxaban prescriptions to the highest copay tier. The known linear relationship between copay costs and treatment abandonment was used to calculate the potential resulting increase in treatment abandonment. Known rates of morbidity and mortality due to abandoning anticoagulants were used to estimate resulting increases in morbidity and mortality. Results: If the 3 largest PBMs all shifted costs onto patients by moving all apixaban and rivaroxaban prescriptions to the highest formulary tier, Tier 6, patients' copay amount would increase by <math><mn>235</mn> <mi>t</mi> <mi>o</mi></math> 482 million for apixaban and <math><mn>105</mn> <mi>t</mi> <mi>o</mi></math> 206 million for rivaroxaban. Such an increase could lead to 169 000 to 228 000 patients abandoning apixaban and 71 000 to 93 000 abandoning rivaroxaban. The resulting morbidity and mortality could include up to an additional 145 000 major cardiovascular events and up to 97 000 more deaths. Conclusion: The Medicare Price Negotiation Program could impact patients negatively if it causes PBMs to increase patients' out-of-pocket costs for medicines. Policymakers should closely monitor changes in overall affordability, including all patient out-of-pocket expenditures, for medications in the program. Preemptive measures should be considered to ensure that the most vulnerable citizens are not placed in precarious situations, leading to poorer health outcomes.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"154-160"},"PeriodicalIF":2.3,"publicationDate":"2024-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11612897/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142769717","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}

引用次数: 0

We Are on the Verge of Breakthrough Cures for Type 1 Diabetes, but Who Are the 2 Million Americans Who Have It? 我们即将找到治疗 1 型糖尿病的突破性方法，但谁是 200 万美国患者？

IF 2.3 Q2 ECONOMICS

Journal of Health Economics and Outcomes Research

Pub Date : 2024-11-12 eCollection Date: 2024-01-01 DOI: 10.36469/001c.124604

Rebecca Smith, Samara Eisenberg, Aaron Turner-Pfifer, Jacqueline LeGrand, Sarah Pincus, Yousra Omer, Fei Wang, Bruce Pyenson

Background: Two million Americans have type 1 diabetes (T1DM). Innovative treatments have standardized insulin delivery and improved outcomes for patients, but patients' access to such technologies depends on social determinants of health, including insurance coverage, proper diagnosis, and appropriate patient supports. Prior estimates of US prevalence, incidence, and patient characteristics have relied on data from select regions and younger ages and miss important determinants. Objectives: This study sought to use large, nationally representative healthcare claims data sets to holistically estimate the size of the current US population with T1DM and investigate geographic nuances in prevalence and incidence, patient demographics, insurance coverage, and device use. This work also aimed to project T1DM population growth over the next 10 years. Methods: We used administrative claims from 4 sources to identify prevalent and incident T1DM patients in the US, as well as various demographic and insurance characteristics of the patient population. We combined this data with information from national population growth projections and literature to construct an actuarial model to project growth of the T1DM population based on current trends and scenarios for 2024, 2029, and 2033. Results: We estimated 2.07 million T1DM patients nationally across all insurance coverages in our 2024 baseline model year: 1.79 million adults (≥20 years) and 0.28 million children. This represents a US T1DM prevalence rate of 617 per 100 000 and an incidence rate of 0.016%. By 2033, we project the US population with T1DM will grow by about 10%, reaching approximately 2.29 million patients. Discussion: Our results showed important differences in T1DM prevalence and incidence across regions, payers, and ethnic groups. This suggests studies based on more geographically concentrated data may miss important variation in prevalence and incidence across regions. It also indicates T1DM prevalence tends to vary by income, consistent with several international studies. Conclusions: Accurate projections of T1DM population growth are critical to ensure appropriate healthcare coverage and reimbursement for treatments. Our work supports future policy and research efforts with 2024, 2029, and 2033 projections of demographics and insurance coverage for people with T1DM.

背景：两百万美国人患有 1 型糖尿病（T1DM）。创新疗法规范了胰岛素给药方式，改善了患者的治疗效果，但患者能否获得这些技术取决于健康的社会决定因素，包括保险范围、正确诊断和适当的患者支持。之前对美国患病率、发病率和患者特征的估计依赖于特定地区和较低年龄段的数据，忽略了重要的决定因素。研究目标：本研究试图利用具有全国代表性的大型医疗索赔数据集来全面估算当前美国 T1DM 患者的规模，并调查患病率和发病率、患者人口统计学、保险覆盖率和设备使用方面的地域差异。这项工作还旨在预测未来 10 年 T1DM 患者的增长情况。方法：我们利用来自 4 个来源的行政索赔来确定美国 T1DM 患者的患病率和发病率，以及患者群体的各种人口和保险特征。我们将这些数据与国家人口增长预测和文献信息相结合，构建了一个精算模型，根据当前趋势和 2024 年、2029 年和 2033 年的情景预测 T1DM 人口的增长。结果：我们估计，在 2024 基准模型年，全国所有保险范围内的 T1DM 患者将达到 207 万人：179万成人（≥20岁）和28万儿童。这意味着美国的 T1DM 患病率为每 10 万人中有 617 人，发病率为 0.016%。我们预计，到 2033 年，美国 T1DM 患者人数将增长约 10%，达到约 229 万人。讨论我们的研究结果表明，T1DM 的患病率和发病率在不同地区、付款人和种族群体之间存在重大差异。这表明，基于更集中的地理数据进行的研究可能会遗漏不同地区患病率和发病率的重要差异。它还表明，T1DM 的患病率往往因收入而异，这与多项国际研究结果一致。结论对 T1DM 人口增长的准确预测对于确保适当的医疗保险和治疗报销至关重要。我们的工作通过对 2024 年、2029 年和 2033 年 T1DM 患者的人口统计和保险覆盖率的预测，为未来的政策和研究工作提供了支持。

{"title":"We Are on the Verge of Breakthrough Cures for Type 1 Diabetes, but Who Are the 2 Million Americans Who Have It?","authors":"Rebecca Smith, Samara Eisenberg, Aaron Turner-Pfifer, Jacqueline LeGrand, Sarah Pincus, Yousra Omer, Fei Wang, Bruce Pyenson","doi":"10.36469/001c.124604","DOIUrl":"10.36469/001c.124604","url":null,"abstract":"Background: Two million Americans have type 1 diabetes (T1DM). Innovative treatments have standardized insulin delivery and improved outcomes for patients, but patients' access to such technologies depends on social determinants of health, including insurance coverage, proper diagnosis, and appropriate patient supports. Prior estimates of US prevalence, incidence, and patient characteristics have relied on data from select regions and younger ages and miss important determinants. Objectives: This study sought to use large, nationally representative healthcare claims data sets to holistically estimate the size of the current US population with T1DM and investigate geographic nuances in prevalence and incidence, patient demographics, insurance coverage, and device use. This work also aimed to project T1DM population growth over the next 10 years. Methods: We used administrative claims from 4 sources to identify prevalent and incident T1DM patients in the US, as well as various demographic and insurance characteristics of the patient population. We combined this data with information from national population growth projections and literature to construct an actuarial model to project growth of the T1DM population based on current trends and scenarios for 2024, 2029, and 2033. Results: We estimated 2.07 million T1DM patients nationally across all insurance coverages in our 2024 baseline model year: 1.79 million adults (≥20 years) and 0.28 million children. This represents a US T1DM prevalence rate of 617 per 100 000 and an incidence rate of 0.016%. By 2033, we project the US population with T1DM will grow by about 10%, reaching approximately 2.29 million patients. Discussion: Our results showed important differences in T1DM prevalence and incidence across regions, payers, and ethnic groups. This suggests studies based on more geographically concentrated data may miss important variation in prevalence and incidence across regions. It also indicates T1DM prevalence tends to vary by income, consistent with several international studies. Conclusions: Accurate projections of T1DM population growth are critical to ensure appropriate healthcare coverage and reimbursement for treatments. Our work supports future policy and research efforts with 2024, 2029, and 2033 projections of demographics and insurance coverage for people with T1DM.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"145-153"},"PeriodicalIF":2.3,"publicationDate":"2024-11-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11566618/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142648041","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}

引用次数: 0

Retrospective Analysis of RSV Infection in Pediatric Patients: Epidemiology, Comorbidities, Treatment, and Costs in Dubai (2014-2023). 儿科患者 RSV 感染的回顾性分析：迪拜的流行病学、并发症、治疗和成本（2014-2023 年）。

IF 2.3 Q2 ECONOMICS

Journal of Health Economics and Outcomes Research

Pub Date : 2024-11-05 eCollection Date: 2024-01-01 DOI: 10.36469/001c.123889

Jean Joury, Nawal Al Kaabi, Sara Al Dallal, Bassam Mahboub, Mostafa Zayed, Mohamed Abdelaziz, Jennifer Onwumeh-Okwundu, Mark A Fletcher, Subramanyam Kumaresan, Badarinath C Ramachandrachar, Mohamed Farghaly

Background: Infections attributable to respiratory syncytial virus (RSV) are a major cause of hospitalization among young children worldwide. Despite substantial clinical and economic burden, real-world data associated with RSV infections in the United Arab Emirates (UAE) are limited. Objectives: This study aimed to assess among children (<18 years) diagnosed with RSV the epidemiology, seasonality, comorbidities, treatment patterns, length of hospital stay, healthcare resource utilization (HCRU), and costs associated with pediatric infection in Dubai, UAE. Methods: This 10-year retrospective cohort study (Jan. 1, 2014-Sept. 30, 2023) utilized Dubai Real-World Database, a private insurance claims database. Patients aged <18 years with a first-episode diagnosis claim (primary or secondary, or a hospital admission) for RSV any time during the index period (Jan. 1, 2014-June 30, 2023) were included. Outcomes were analyzed during a 3-month follow-up. Patients were stratified into 3 cohorts: Cohort 1 (<2 years), Cohort 2 (2 to <6 years), and Cohort 3 (6 to <18 years). Results: Of 28 011 patients identified, 25 729 were aged <18 years with RSV infection. An increasing trend in reported cases was observed from 2014 to 2022, with an average annual increase of 55%. Half of study patients (49.3%) belonged to Cohort 1, with a mean age of 0.6 years, while less than 2% had known risk factors and 22% of the patients in cohort 1 were hospitalized. In Cohort 1, 32.0% had upper respiratory tract infections, 39.4% had lower respiratory tract infections, and 44.4% of patients had an "other respiratory disease." The average length of hospitalization was about 4 days across all cohorts. The total hospitalization cost was highest in patients <2 years, amounting to US $9 798 174 (m e d i a n, U S$ 2241.30). Conclusion: Among the RSV patients, 49.3% were <2 years of age and few had recognized risk factors. Among patients <2 years, 22% were hospitalized, with an average hospital stay of 4 days; the cost of hospitalization totaled US $9 798 174. These findings can inform healthcare stakeholders about future policy measures and the need for effective preventive strategies.

背景：呼吸道合胞病毒（RSV）感染是全球幼儿住院治疗的主要原因。尽管造成了巨大的临床和经济负担，但阿拉伯联合酋长国（UAE）与 RSV 感染相关的实际数据却很有限。研究目的本研究旨在评估阿联酋儿童（方法：这项为期 10 年（2014 年 1 月 1 日至 2023 年 9 月 30 日）的回顾性队列研究利用了迪拜真实世界数据库（一个私人保险索赔数据库）。患者年龄结果在确定的 28 011 名患者中，25 729 人的年龄为 9 798 174（m e d i a n , U S 2241.30）。结论在 RSV 患者中，49.3% 的人是

{"title":"Retrospective Analysis of RSV Infection in Pediatric Patients: Epidemiology, Comorbidities, Treatment, and Costs in Dubai (2014-2023).","authors":"Jean Joury, Nawal Al Kaabi, Sara Al Dallal, Bassam Mahboub, Mostafa Zayed, Mohamed Abdelaziz, Jennifer Onwumeh-Okwundu, Mark A Fletcher, Subramanyam Kumaresan, Badarinath C Ramachandrachar, Mohamed Farghaly","doi":"10.36469/001c.123889","DOIUrl":"10.36469/001c.123889","url":null,"abstract":"Background: Infections attributable to respiratory syncytial virus (RSV) are a major cause of hospitalization among young children worldwide. Despite substantial clinical and economic burden, real-world data associated with RSV infections in the United Arab Emirates (UAE) are limited. Objectives: This study aimed to assess among children (<18 years) diagnosed with RSV the epidemiology, seasonality, comorbidities, treatment patterns, length of hospital stay, healthcare resource utilization (HCRU), and costs associated with pediatric infection in Dubai, UAE. Methods: This 10-year retrospective cohort study (Jan. 1, 2014-Sept. 30, 2023) utilized Dubai Real-World Database, a private insurance claims database. Patients aged <18 years with a first-episode diagnosis claim (primary or secondary, or a hospital admission) for RSV any time during the index period (Jan. 1, 2014-June 30, 2023) were included. Outcomes were analyzed during a 3-month follow-up. Patients were stratified into 3 cohorts: Cohort 1 (<2 years), Cohort 2 (2 to <6 years), and Cohort 3 (6 to <18 years). Results: Of 28 011 patients identified, 25 729 were aged <18 years with RSV infection. An increasing trend in reported cases was observed from 2014 to 2022, with an average annual increase of 55%. Half of study patients (49.3%) belonged to Cohort 1, with a mean age of 0.6 years, while less than 2% had known risk factors and 22% of the patients in cohort 1 were hospitalized. In Cohort 1, 32.0% had upper respiratory tract infections, 39.4% had lower respiratory tract infections, and 44.4% of patients had an \"other respiratory disease.\" The average length of hospitalization was about 4 days across all cohorts. The total hospitalization cost was highest in patients <2 years, amounting to US <math><mn>9</mn> <mrow><mo> </mo></mrow> <mn>798</mn> <mrow><mo> </mo></mrow> <mn>174</mn> <mo>(</mo> <mi>m</mi> <mi>e</mi> <mi>d</mi> <mi>i</mi> <mi>a</mi> <mi>n</mi> <mo>,</mo> <mi>U</mi> <mi>S</mi></math> 2241.30). Conclusion: Among the RSV patients, 49.3% were <2 years of age and few had recognized risk factors. Among patients <2 years, 22% were hospitalized, with an average hospital stay of 4 days; the cost of hospitalization totaled US $9 798 174. These findings can inform healthcare stakeholders about future policy measures and the need for effective preventive strategies.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"133-144"},"PeriodicalIF":2.3,"publicationDate":"2024-11-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11580195/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142687047","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}

引用次数: 0

Assessing the Fiscal Burden of Overweight and Obesity in Japan through Application of a Public Economic Framework. 通过应用公共经济框架评估日本超重和肥胖的财政负担。

IF 2.3 Q2 ECONOMICS

Journal of Health Economics and Outcomes Research

Pub Date : 2024-11-04 eCollection Date: 2024-01-01 DOI: 10.36469/001c.123991

Ataru Igarashi, Cillian Copeland, Nikos Kotsopoulos, Riku Ota, Silvia Capucci, Daisuke Adachi

Introduction: Obesity continues to represent a significant public health concern, with a broad impact from both a health and economic perspective. Objective: This analysis assesses the fiscal consequences of overweight and obesity (OAO) in Japan by capturing obesity-attributable lost tax revenue and increased government transfers using a government perspective. Methods: The fiscal burden of OAO was estimated using an age-specific prevalence model, which tracked the Japanese population across different body mass index (BMI) categories. The model was populated with fiscal data for Japan, including employment activity and government spending, to calculate tax revenue and transfer costs. A targeted literature review was conducted to identify data estimating the impact of OAO on employment, income, sick leave, retirement, and mortality. These modifiers were applied to Japanese epidemiological and fiscal projections to calculate government tax revenue and spending. The incremental impact of reducing OAO in the general population was subsequently calculated. Results were estimated based on the 2023 Japanese working-age population aged 18 to 70 years. Results: The total fiscal burden of OAO in Japan, defined as BMI of at least 25, is estimated at US <math><mn>13.41</mn> <mi>b</mi> <mi>i</mi> <mi>l</mi> <mi>l</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mo>(</mo> <mrow><mo>¥</mo></mrow> <mn>1925</mn> <mi>b</mi> <mi>i</mi> <mi>l</mi> <mi>l</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mo>)</mo> <mo>,</mo> <mi>r</mi> <mi>e</mi> <mi>p</mi> <mi>r</mi> <mi>e</mi> <mi>s</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>i</mi> <mi>n</mi> <mi>g</mi> <mn>0.4</mn></math> 6.3 billion (¥901 billion) and <math><mn>1.2</mn> <mi>b</mi> <mi>i</mi> <mi>l</mi> <mi>l</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mo>(</mo> <mrow><mo>¥</mo></mrow> <mn>179</mn> <mi>b</mi> <mi>i</mi> <mi>l</mi> <mi>l</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mo>)</mo> <mi>i</mi> <mi>n</mi> <mi>d</mi> <mi>i</mi> <mi>r</mi> <mi>e</mi> <mi>c</mi> <mi>t</mi> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>i</mi> <mi>n</mi> <mi>d</mi> <mi>i</mi> <mi>r</mi> <mi>e</mi> <mi>c</mi> <mi>t</mi> <mi>t</mi> <mi>a</mi> <mi>x</mi> <mi>r</mi> <mi>e</mi> <mi>v</mi> <mi>e</mi> <mi>n</mi> <mi>u</mi> <mi>e</mi> <mo>,</mo> <mi>r</mi> <mi>e</mi> <mi>s</mi> <mi>p</mi> <mi>e</mi> <mi>c</mi> <mi>t</mi> <mi>i</mi> <mi>v</mi> <mi>e</mi> <mi>l</mi> <mi>y</mi> <mo>,</mo> <mi>d</mi> <mi>u</mi> <mi>e</mi> <mi>t</mi> <mi>o</mi> <mi>l</mi> <mi>o</mi> <mi>w</mi> <mi>e</mi> <mi>r</mi> <mi>e</mi> <mi>m</mi> <mi>p</mi> <mi>l</mi> <mi>o</mi> <mi>y</mi> <mi>m</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>i</mi> <mi>n</mi> <mi>c</mi> <mi>o</mi> <mi>m</mi> <mi>e</mi> <mi>c</mi> <mi>o</mi> <mi>m</mi> <mi>b</mi> <mi>i</mi> <mi>n</mi> <mi>e</mi> <mi>d</mi> <mi>w</mi> <mi>i</mi> <mi>t</mi> <mi>h</mi> <mi>h</mi> <mi>i</mi> <mi>g</mi> <mi>h</mi> <mi>e</mi> <mi>r</mi> <mi>s</mi> <mi>i</mi> <mi>c</mi> <mi>k</mi> <mi>l</mi

导言：肥胖症仍然是一个重大的公共卫生问题，对健康和经济都有广泛的影响。分析目的本分析从政府角度出发，通过捕捉肥胖导致的税收损失和政府转移支付的增加，评估超重和肥胖（OAO）在日本造成的财政后果。方法：使用特定年龄流行率模型估算 OAO 的财政负担，该模型跟踪不同体重指数 (BMI) 类别的日本人口。该模型使用了日本的财政数据，包括就业活动和政府支出，以计算税收和转移成本。我们进行了有针对性的文献回顾，以确定估算 OAO 对就业、收入、病假、退休和死亡率影响的数据。这些修正因素被应用于日本的流行病学和财政预测，以计算政府税收和支出。随后计算了减少 OAO 对普通人群的增量影响。结果以 2023 年日本 18 至 70 岁的工作年龄人口为基础进行估算。结果：日本 OAO 的总财政负担（定义为体重指数至少为 25）估计为 134.1 亿美元（1925 亿日元），分别为 463 亿美元（9010 亿日元）和 1.2 b i l l i o n ( ¥ 179 b i l l i o n ) i n d i r e c t a n d i n d i r e c t a x r e v n u e ， r e s p e c t i v e l y ， d u e t l o w e r e m p l o y m e n t a n d i n c o m e c o m b i n e d w i t h i g h e r s i c k l e a v e 。由于提前退休的人数增加，预计将增加 54 亿美元（7,690 亿日元）的养老金支出。结论：尽管 OAO 对健康的影响有据可查，但这项财政分析表明，OAO 对医疗保健系统和更广泛的政府账户都造成了巨大的经济负担。旨在减少人口肥胖的政策有可能通过增加就业和减少公共开支使政府账户受益，从而抵消实施这些政策的成本。

{"title":"Assessing the Fiscal Burden of Overweight and Obesity in Japan through Application of a Public Economic Framework.","authors":"Ataru Igarashi, Cillian Copeland, Nikos Kotsopoulos, Riku Ota, Silvia Capucci, Daisuke Adachi","doi":"10.36469/001c.123991","DOIUrl":"https://doi.org/10.36469/001c.123991","url":null,"abstract":"Introduction: Obesity continues to represent a significant public health concern, with a broad impact from both a health and economic perspective. Objective: This analysis assesses the fiscal consequences of overweight and obesity (OAO) in Japan by capturing obesity-attributable lost tax revenue and increased government transfers using a government perspective. Methods: The fiscal burden of OAO was estimated using an age-specific prevalence model, which tracked the Japanese population across different body mass index (BMI) categories. The model was populated with fiscal data for Japan, including employment activity and government spending, to calculate tax revenue and transfer costs. A targeted literature review was conducted to identify data estimating the impact of OAO on employment, income, sick leave, retirement, and mortality. These modifiers were applied to Japanese epidemiological and fiscal projections to calculate government tax revenue and spending. The incremental impact of reducing OAO in the general population was subsequently calculated. Results were estimated based on the 2023 Japanese working-age population aged 18 to 70 years. Results: The total fiscal burden of OAO in Japan, defined as BMI of at least 25, is estimated at US <math><mn>13.41</mn> <mi>b</mi> <mi>i</mi> <mi>l</mi> <mi>l</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mo>(</mo> <mrow><mo>¥</mo></mrow> <mn>1925</mn> <mi>b</mi> <mi>i</mi> <mi>l</mi> <mi>l</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mo>)</mo> <mo>,</mo> <mi>r</mi> <mi>e</mi> <mi>p</mi> <mi>r</mi> <mi>e</mi> <mi>s</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>i</mi> <mi>n</mi> <mi>g</mi> <mn>0.4</mn></math> 6.3 billion (¥901 billion) and <math><mn>1.2</mn> <mi>b</mi> <mi>i</mi> <mi>l</mi> <mi>l</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mo>(</mo> <mrow><mo>¥</mo></mrow> <mn>179</mn> <mi>b</mi> <mi>i</mi> <mi>l</mi> <mi>l</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mo>)</mo> <mi>i</mi> <mi>n</mi> <mi>d</mi> <mi>i</mi> <mi>r</mi> <mi>e</mi> <mi>c</mi> <mi>t</mi> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>i</mi> <mi>n</mi> <mi>d</mi> <mi>i</mi> <mi>r</mi> <mi>e</mi> <mi>c</mi> <mi>t</mi> <mi>t</mi> <mi>a</mi> <mi>x</mi> <mi>r</mi> <mi>e</mi> <mi>v</mi> <mi>e</mi> <mi>n</mi> <mi>u</mi> <mi>e</mi> <mo>,</mo> <mi>r</mi> <mi>e</mi> <mi>s</mi> <mi>p</mi> <mi>e</mi> <mi>c</mi> <mi>t</mi> <mi>i</mi> <mi>v</mi> <mi>e</mi> <mi>l</mi> <mi>y</mi> <mo>,</mo> <mi>d</mi> <mi>u</mi> <mi>e</mi> <mi>t</mi> <mi>o</mi> <mi>l</mi> <mi>o</mi> <mi>w</mi> <mi>e</mi> <mi>r</mi> <mi>e</mi> <mi>m</mi> <mi>p</mi> <mi>l</mi> <mi>o</mi> <mi>y</mi> <mi>m</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>i</mi> <mi>n</mi> <mi>c</mi> <mi>o</mi> <mi>m</mi> <mi>e</mi> <mi>c</mi> <mi>o</mi> <mi>m</mi> <mi>b</mi> <mi>i</mi> <mi>n</mi> <mi>e</mi> <mi>d</mi> <mi>w</mi> <mi>i</mi> <mi>t</mi> <mi>h</mi> <mi>h</mi> <mi>i</mi> <mi>g</mi> <mi>h</mi> <mi>e</mi> <mi>r</mi> <mi>s</mi> <mi>i</mi> <mi>c</mi> <mi>k</mi> <mi>l</mi","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"125-132"},"PeriodicalIF":2.3,"publicationDate":"2024-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11555824/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142621935","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}

引用次数: 0

Real-World HbA1c Changes Among Type 2 Diabetes Mellitus Patients Initiating Treatment With a 1.0 Mg Weekly Dose of Semaglutide for Diabetes. 开始每周服用 1.0 毫克塞马鲁肽治疗的 2 型糖尿病患者的实际 HbA1c 变化。

IF 2.3 Q2 ECONOMICS

Journal of Health Economics and Outcomes Research

Pub Date : 2024-11-04 eCollection Date: 2024-01-01 DOI: 10.36469/001c.124111

Noelle N Gronroos, Caroline Swift, Monica S Frazer, Andrew Sargent, Michael Leszko, Erin Buysman, Sara Alvarez, Tyler J Dunn, Josh Noone

Background: Medical management of patients with type 2 diabetes mellitus (T2DM) is complex because of the chronic nature of the disease and its associated comorbidities. Injectable once-weekly semaglutide for diabetes (OW sema T2D) is a type of glucagon-like peptide-1 receptor agonist approved for the treatment of patients with T2DM. Objectives: To describe patient characteristics and HbA1c changes for patients prescribed 1.0 mg maintenance dose OW sema T2D. Methods: This retrospective study included adult patients with T2DM with a pre-index glycated hemoglobin (HbA1c) of at least 7%, initiating treatment with OW sema T2D between January 1, 2018, and December 31, 2019, and prescribed a 1.0 mg maintenance dose. Patients were identified in the Optum Research Database and were included if they had continuous health plan enrollment for at least 12 months prior to (pre-index) and at least 12 months following (post-index) the date of the first OW sema T2D claim (index). Dose at initiation and prescriber specialty were captured. Change in HbA1c between the latest post-index and pre-index HbA1c measurement was calculated among all patients and among those with at least 90 days of continuous treatment (persistent patients). Results: A total of 2168 patients were included in this study. On average, patients were taking 13.5 different classes of medications. The majority of patients had lipid metabolism disorder (90.8%), hypertension (86.6%), diabetes with complications (86.8%), or other nutritional/endocrine/metabolic disorders (72.5%). The mean HbA1c reduction was 1.2% (P < .001). Patients persistent with OW sema T2D (n =1280) had a mean HbA1c reduction of 1.4% (P < .001). The mean (SD) days covered with a 1.0 mg maintenance dose was 236.1 (94.1) days. Discussion: Despite being medically complex, the patients in this real-world study experienced significant reductions in HbA1c following initiation of OW sema T2D. Conclusions: A 1.0 mg maintenance dose of OW sema T2D is an effective treatment for T2DM in the real world.

背景：2 型糖尿病（T2DM）患者的医疗管理非常复杂，因为这种疾病具有慢性性质，并伴有相关的并发症。每周注射一次的糖尿病用塞马鲁肽（OW sema T2D）是一种胰高血糖素样肽-1受体激动剂，已被批准用于治疗T2DM患者。研究目的描述处方 1.0 毫克维持剂量 OW sema T2D 患者的特征和 HbA1c 变化。方法：这项回顾性研究纳入了糖化血红蛋白（HbA1c）指数前至少为 7%、在 2018 年 1 月 1 日至 2019 年 12 月 31 日期间开始接受 OW sema T2D 治疗并处方 1.0 毫克维持剂量的 T2DM 成年患者。患者在 Optum 研究数据库中被识别，如果他们在首次 OW sema T2D 索偿（索引）日期之前（索引前）至少 12 个月、之后（索引后）至少 12 个月内连续加入了医疗保险，则被纳入其中。开始用药时的剂量和处方专业均被记录。计算所有患者和至少持续治疗 90 天的患者（持续治疗患者）在指数后和指数前最近一次 HbA1c 测量之间的 HbA1c 变化。结果本研究共纳入了 2168 名患者。患者平均服用 13.5 种不同类型的药物。大多数患者患有脂质代谢紊乱（90.8%）、高血压（86.6%）、糖尿病并发症（86.8%）或其他营养/内分泌/代谢紊乱（72.5%）。HbA1c 平均降低了 1.2%（P 讨论）：尽管病情复杂，但在这项真实世界研究中，患者在开始接受 OW sema T2D 治疗后，HbA1c 明显降低。结论在现实世界中，1.0 毫克维持剂量的 OW sema T2D 是治疗 T2DM 的有效方法。

{"title":"Real-World HbA1c Changes Among Type 2 Diabetes Mellitus Patients Initiating Treatment With a 1.0 Mg Weekly Dose of Semaglutide for Diabetes.","authors":"Noelle N Gronroos, Caroline Swift, Monica S Frazer, Andrew Sargent, Michael Leszko, Erin Buysman, Sara Alvarez, Tyler J Dunn, Josh Noone","doi":"10.36469/001c.124111","DOIUrl":"10.36469/001c.124111","url":null,"abstract":"Background: Medical management of patients with type 2 diabetes mellitus (T2DM) is complex because of the chronic nature of the disease and its associated comorbidities. Injectable once-weekly semaglutide for diabetes (OW sema T2D) is a type of glucagon-like peptide-1 receptor agonist approved for the treatment of patients with T2DM. Objectives: To describe patient characteristics and HbA1c changes for patients prescribed 1.0 mg maintenance dose OW sema T2D. Methods: This retrospective study included adult patients with T2DM with a pre-index glycated hemoglobin (HbA1c) of at least 7%, initiating treatment with OW sema T2D between January 1, 2018, and December 31, 2019, and prescribed a 1.0 mg maintenance dose. Patients were identified in the Optum Research Database and were included if they had continuous health plan enrollment for at least 12 months prior to (pre-index) and at least 12 months following (post-index) the date of the first OW sema T2D claim (index). Dose at initiation and prescriber specialty were captured. Change in HbA1c between the latest post-index and pre-index HbA1c measurement was calculated among all patients and among those with at least 90 days of continuous treatment (persistent patients). Results: A total of 2168 patients were included in this study. On average, patients were taking 13.5 different classes of medications. The majority of patients had lipid metabolism disorder (90.8%), hypertension (86.6%), diabetes with complications (86.8%), or other nutritional/endocrine/metabolic disorders (72.5%). The mean HbA1c reduction was 1.2% (P < .001). Patients persistent with OW sema T2D (n =1280) had a mean HbA1c reduction of 1.4% (P < .001). The mean (SD) days covered with a 1.0 mg maintenance dose was 236.1 (94.1) days. Discussion: Despite being medically complex, the patients in this real-world study experienced significant reductions in HbA1c following initiation of OW sema T2D. Conclusions: A 1.0 mg maintenance dose of OW sema T2D is an effective treatment for T2DM in the real world.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"118-124"},"PeriodicalIF":2.3,"publicationDate":"2024-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11539928/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142590761","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}

引用次数: 0

Healthcare Resource Utilization Among Patients With Agitation in Alzheimer Dementia. 阿尔茨海默型痴呆症躁动患者的医疗资源使用情况。

IF 2.3 Q2 ECONOMICS

Journal of Health Economics and Outcomes Research

Pub Date : 2024-10-29 eCollection Date: 2024-01-01 DOI: 10.36469/001c.124455

Christie Teigland, Zulkarnain Pulungan, David Bruhn, Steve Hwang

Background: Agitation in Alzheimer dementia is common, but the associated healthcare burden remains unclear. Objective: This retrospective analysis evaluated baseline characteristics, healthcare resource utilization, and costs among patients with agitation in Alzheimer dementia and those without agitation in Alzheimer dementia. Methods: Medicare beneficiaries from 100% of the Medicare Fee-for-Service claims database (2009-2016) with 2 or more claims 30 or more days apart for both Alzheimer's disease and dementia and continuous enrollment with medical/pharmacy coverage for 6 months before and 12 months after the index diagnosis were included. Patients with agitation in Alzheimer dementia were identified by 2 or more claims 14 or more days apart using International Classification of Diseases-9-CM/-10-CM codes based on the provisional International Psychogeriatric Association agitation definition. Patients with severe psychiatric disorders were excluded. Two cohorts of patients (with and without agitation) were then defined, and patient characteristics, healthcare resource utilization, and costs were compared in a descriptive exploratory analysis. Results: Of 2 684 704 Fee-for-Service patients with Alzheimer dementia, 769 141 met all inclusion criteria; among these, 281 042 (36.5%) had agitation. The mean age in patients with and without agitation in Alzheimer dementia was 83 years. Most patients in both groups were female, but the proportion of males was slightly higher in the agitation in Alzheimer dementia group (30.3% vs 28.2%, respectively). Patients with agitation in Alzheimer dementia were more likely than those without agitation in Alzheimer dementia to have lower socioeconomic status (dual eligibility for Medicaid, 45.0% vs 41.7%, respectively) or be disabled (10.5% vs 9.4%). Overall, healthcare costs were higher in the agitation in Alzheimer dementia population compared with those without agitation in Alzheimer dementia (mean cost PPPY, $32 322 a n d$ 30 121, respectively), with the largest differences observed in inpatient and post-acute care costs. Conclusions: These exploratory findings underscore the substantial economic burden of agitation in Alzheimer dementia and highlight the need for treatment options for the agitation in Alzheimer dementia population to improve associated health outcomes.

背景：阿尔茨海默型痴呆症患者的躁动很常见，但相关的医疗负担仍不清楚。目的：本回顾性分析评估了阿尔茨海默氏症躁动患者的基线特征、医疗资源利用率和成本：这项回顾性分析评估了阿尔茨海默型痴呆症躁动患者和无躁动患者的基线特征、医疗资源利用率和费用。方法：纳入100%的医疗保险付费服务索赔数据库（2009-2016年）中的医疗保险受益人，这些受益人须同时患有阿尔茨海默病和痴呆症，且在确诊前6个月和确诊后12个月内连续投保医疗/药物保险，且索赔时间相隔30天或以上。阿尔茨海默氏症痴呆症患者中的躁动患者是根据国际老年心理协会的临时躁动定义，使用国际疾病分类-9-CM/-10-CM 编码，通过相隔 14 天或更长时间的 2 份或更多索赔来确定的。患有严重精神障碍的患者除外。然后定义了两组患者（有躁动和无躁动），并在描述性探索分析中比较了患者特征、医疗资源利用率和成本。研究结果在 2 684 704 名付费服务的阿尔茨海默痴呆症患者中，有 769 141 人符合所有纳入标准；其中有 281042 人（36.5%）有躁动。阿尔茨海默氏症痴呆症患者的平均年龄为 83 岁。两组患者中大多数为女性，但阿尔茨海默型痴呆患者中男性比例略高（分别为 30.3% 和 28.2%）。与阿尔茨海默型痴呆症患者相比，阿尔茨海默型痴呆症激越症患者更有可能社会经济地位较低（分别为 45.0% 对 41.7%）或身患残疾（10.5% 对 9.4%）。总体而言，阿尔茨海默型痴呆症患者的医疗费用高于无阿尔茨海默型痴呆症患者（PPPY 平均费用分别为 32 322 美元和 30 121 美元），其中住院和急性期后护理费用的差异最大。结论：这些探索性发现强调了阿尔茨海默型痴呆症患者躁动所造成的巨大经济负担，并突出表明有必要为阿尔茨海默型痴呆症患者提供治疗方案，以改善相关的健康状况。

{"title":"Healthcare Resource Utilization Among Patients With Agitation in Alzheimer Dementia.","authors":"Christie Teigland, Zulkarnain Pulungan, David Bruhn, Steve Hwang","doi":"10.36469/001c.124455","DOIUrl":"10.36469/001c.124455","url":null,"abstract":"Background: Agitation in Alzheimer dementia is common, but the associated healthcare burden remains unclear. Objective: This retrospective analysis evaluated baseline characteristics, healthcare resource utilization, and costs among patients with agitation in Alzheimer dementia and those without agitation in Alzheimer dementia. Methods: Medicare beneficiaries from 100% of the Medicare Fee-for-Service claims database (2009-2016) with 2 or more claims 30 or more days apart for both Alzheimer's disease and dementia and continuous enrollment with medical/pharmacy coverage for 6 months before and 12 months after the index diagnosis were included. Patients with agitation in Alzheimer dementia were identified by 2 or more claims 14 or more days apart using International Classification of Diseases-9-CM/-10-CM codes based on the provisional International Psychogeriatric Association agitation definition. Patients with severe psychiatric disorders were excluded. Two cohorts of patients (with and without agitation) were then defined, and patient characteristics, healthcare resource utilization, and costs were compared in a descriptive exploratory analysis. Results: Of 2 684 704 Fee-for-Service patients with Alzheimer dementia, 769 141 met all inclusion criteria; among these, 281 042 (36.5%) had agitation. The mean age in patients with and without agitation in Alzheimer dementia was 83 years. Most patients in both groups were female, but the proportion of males was slightly higher in the agitation in Alzheimer dementia group (30.3% vs 28.2%, respectively). Patients with agitation in Alzheimer dementia were more likely than those without agitation in Alzheimer dementia to have lower socioeconomic status (dual eligibility for Medicaid, 45.0% vs 41.7%, respectively) or be disabled (10.5% vs 9.4%). Overall, healthcare costs were higher in the agitation in Alzheimer dementia population compared with those without agitation in Alzheimer dementia (mean cost PPPY, <math><mn>32</mn> <mrow><mo> </mo></mrow> <mn>322</mn> <mi>a</mi> <mi>n</mi> <mi>d</mi></math> 30 121, respectively), with the largest differences observed in inpatient and post-acute care costs. Conclusions: These exploratory findings underscore the substantial economic burden of agitation in Alzheimer dementia and highlight the need for treatment options for the agitation in Alzheimer dementia population to improve associated health outcomes.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"118-124"},"PeriodicalIF":2.3,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11539929/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142590758","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}

引用次数: 0

Costs of Potential Medication Wastage Due to Switching Treatment Among People With Multiple Sclerosis. 多发性硬化症患者因更换治疗方案而造成的潜在药物浪费成本。

IF 2.3 Q2 ECONOMICS

Journal of Health Economics and Outcomes Research

Pub Date : 2024-10-28 eCollection Date: 2024-01-01 DOI: 10.36469/001c.123336

Darin T Okuda, Achal Patel, Robert Schuldt, Ibraheem Abioye, Nicole G Bonine

Background: Unused medications negatively impact healthcare resource utilization and environmental safety, contribute substantially to annual healthcare expenditures, and may ultimately affect patient health outcomes. People with multiple sclerosis (PwMS) commonly switch disease-modifying therapies (DMTs), leading to medication wastage and substantial costs for insurers and patients. Objectives: To estimate the cost associated with potential medication wastage (PMW) in a subcohort of PwMS receiving oral or self-injectable US Food and Drug Administration-approved DMTs who switched DMTs in a calendar year in the United States. Methods: This retrospective cohort study included adults with MS and used PharMetrics® Plus claims data from 2017 to 2021. PwMS were required to have 12 months of continuous eligibility for the entire year and a claim for at least 2 unique DMTs during the same calendar year. The PMW cohort was defined as those who had an aggregate overlap in days' supply across DMT switches within the year; those in the non-PMW cohort did not. The cost of PMW for insurers and PwMS due to overlap was calculated only at the point of switch to the new DMT and defined as the cost of the remaining days' supply of the prior DMT. Results: The number of PwMS meeting the inclusion criteria was 1762 in 2017, 1947 in 2018, 1679 in 2019, 1461 in 2020, and 1782 in 2021. Approximately 95% of PwMS switched DMTs once within single calendar years, and 25% (n = 381-464) contributed to PMW. For those who had overlapping DMT supply, it was estimated that 34% to 38% of the DMT being switched from was potentially wasted. The total cost of PMW paid by the insurer and PwMS ranged from $1 200 866 t o$ 1 489 859. While most of the total cost ( $1 172 140 -$ 1 450 328) was paid by the insurer, PwMS still owed substantial amounts ( $28 726 -$ 74 578). Across all PwMS, the per person per year cost ranged from $716 t o$ 846. The estimated wastage and associated costs were consistent across all study years. Conclusions: DMT switching is common among PwMS, resulting in PMW and high costs to patients and insurers.

背景：未使用的药物会对医疗资源的利用和环境安全产生负面影响，大大增加每年的医疗支出，并可能最终影响患者的健康状况。多发性硬化症患者（PwMS）通常会更换改变病情疗法（DMTs），从而导致药物浪费，并给保险公司和患者带来巨额费用。目标：估算多发性硬化症患者可能更换药物的相关成本：对美国食品药品管理局批准的接受口服或自我注射 DMTs 的多发性硬化症患者在一个日历年度内更换 DMTs 的亚群中与潜在药物浪费（PMW）相关的成本进行估算。研究方法：这项回顾性队列研究包括多发性硬化症成人患者，使用的是 2017 年至 2021 年的 PharMetrics® Plus 索赔数据。多发性硬化症患者必须在全年连续获得 12 个月的治疗资格，并且在同一日历年内至少申请过 2 种不同的 DMT。PMW队列的定义是，在一年中，DMT转换的总供应天数有重叠的患者；而非PMW队列的患者则没有重叠。保险公司和 PwMS 因重叠而产生的 PMW 成本仅在转用新 DMT 时计算，并定义为之前 DMT 剩余供应天数的成本。结果：符合纳入标准的 PwMS 人数在 2017 年为 1762 人，2018 年为 1947 人，2019 年为 1679 人，2020 年为 1461 人，2021 年为 1782 人。约 95% 的 PwMS 在单个日历年内更换过一次 DMT，25%（n = 381-464）对 PMW 有贡献。对于那些DMT供应重叠的患者，据估计34%至38%的DMT可能被浪费。由保险公司和 PwMS 支付的 PMW 总成本从 1 200 866 到 1 489 859 不等。虽然总费用的大部分（1 172 140 - 1 450 328）由保险公司支付，但 PwMS 仍拖欠大量费用（28 726 - 74 578）。在所有 PwMS 中，每人每年的费用从 716 到 846 不等。在所有研究年份中，估计的流失率和相关费用是一致的。结论DMT 转换在 PwMS 中很常见，会导致 PMW，并给患者和保险公司带来高额费用。

{"title":"Costs of Potential Medication Wastage Due to Switching Treatment Among People With Multiple Sclerosis.","authors":"Darin T Okuda, Achal Patel, Robert Schuldt, Ibraheem Abioye, Nicole G Bonine","doi":"10.36469/001c.123336","DOIUrl":"10.36469/001c.123336","url":null,"abstract":"Background: Unused medications negatively impact healthcare resource utilization and environmental safety, contribute substantially to annual healthcare expenditures, and may ultimately affect patient health outcomes. People with multiple sclerosis (PwMS) commonly switch disease-modifying therapies (DMTs), leading to medication wastage and substantial costs for insurers and patients. Objectives: To estimate the cost associated with potential medication wastage (PMW) in a subcohort of PwMS receiving oral or self-injectable US Food and Drug Administration-approved DMTs who switched DMTs in a calendar year in the United States. Methods: This retrospective cohort study included adults with MS and used PharMetrics® Plus claims data from 2017 to 2021. PwMS were required to have 12 months of continuous eligibility for the entire year and a claim for at least 2 unique DMTs during the same calendar year. The PMW cohort was defined as those who had an aggregate overlap in days' supply across DMT switches within the year; those in the non-PMW cohort did not. The cost of PMW for insurers and PwMS due to overlap was calculated only at the point of switch to the new DMT and defined as the cost of the remaining days' supply of the prior DMT. Results: The number of PwMS meeting the inclusion criteria was 1762 in 2017, 1947 in 2018, 1679 in 2019, 1461 in 2020, and 1782 in 2021. Approximately 95% of PwMS switched DMTs once within single calendar years, and 25% (n = 381-464) contributed to PMW. For those who had overlapping DMT supply, it was estimated that 34% to 38% of the DMT being switched from was potentially wasted. The total cost of PMW paid by the insurer and PwMS ranged from <math><mn>1</mn> <mrow><mo> </mo></mrow> <mn>200</mn> <mrow><mo> </mo></mrow> <mn>866</mn> <mi>t</mi> <mi>o</mi></math> 1 489 859. While most of the total cost ( <math><mn>1</mn> <mrow><mo> </mo></mrow> <mn>172</mn> <mrow><mo> </mo></mrow> <mn>140</mn> <mo>-</mo></math> 1 450 328) was paid by the insurer, PwMS still owed substantial amounts ( <math><mn>28</mn> <mrow><mo> </mo></mrow> <mn>726</mn> <mo>-</mo></math> 74 578). Across all PwMS, the per person per year cost ranged from <math><mn>716</mn> <mi>t</mi> <mi>o</mi></math> 846. The estimated wastage and associated costs were consistent across all study years. Conclusions: DMT switching is common among PwMS, resulting in PMW and high costs to patients and insurers.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"103-108"},"PeriodicalIF":2.3,"publicationDate":"2024-10-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11523564/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545867","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}

引用次数: 0

Adherence to First-Line Intravesical Bacillus Calmette-Guérin Therapy in the Context of Guideline Recommendations for US Patients With High-Risk Non-muscle Invasive Bladder Cancer. 美国高风险非肌层浸润性膀胱癌患者根据指南建议坚持一线膀胱内卡介苗疗法

IF 2.3 Q2 ECONOMICS

Journal of Health Economics and Outcomes Research

Pub Date : 2024-10-28 eCollection Date: 2024-01-01 DOI: 10.36469/001c.124208

Franklin D Gaylis, Bruno Emond, Ameur M Manceur, Anabelle Tardif-Samson, Laura Morrison, Dominic Pilon, Patrick Lefebvre, Lorie A Ellis, Hiremagalur Balaji, Andrea Ireland

Background: Bacillus Calmette-Guérin (BCG) can reduce recurrence and delay progression among patients with high-risk non-muscle invasive bladder cancer (NMIBC), but is associated with a substantial emotional, physical, and social burden. Objectives: This study evaluated the adequacy of first-line intravesical BCG treatment among high-risk NMIBC patients in the United States, including the subgroup with carcinoma in situ (CIS) of the bladder. Methods: Adults with high-risk NMIBC treated with BCG were selected from de-identified MarketScan® Commercial, Medicare, and Medicaid Databases (1/1/2010-2/28/2021). Adequacy of BCG induction and maintenance was evaluated from the first BCG claim until the end of the patient's observation, using a previously published claims-based algorithm (induction: ≥5 instillations within 70 days; induction and maintenance: ≥7 instillations within 274 days of first instillation) and a definition based on the landmark Southwest Oncology Group (SWOG) trial (induction: ≥5 instillations without gaps >7 days; followed by ≥2 instillations at month 3, 6, and every 6 months thereafter). Proportions of patients with adequate BCG induction and maintenance were reported overall and compared between those with and without CIS. Results: Of 5803 high-risk NMIBC patients treated with first-line BCG (mean age, 67.3 years; 20.6% female), 930 (16.0%) had CIS. After first-line BCG, 56.6% received another treatment. Although 86.9% had adequate BCG induction based on the claims-based algorithm (SWOG, 73.6%), only 41.5% had adequate BCG induction and maintenance (SWOG, 1.6%). Similar trends were observed for patients with and without CIS, with higher adherence to guidelines for patients with CIS (adequate induction using claims-based algorithm: 90.3% vs 86.2%; adequate induction and maintenance: 50.8% vs 39.7%, all P < .001). A greater proportion of CIS patients than non-CIS patients had cystectomy (CIS, 14.4%, non-CIS, 8.5%; P < .001) after first-line BCG. Discussion: Among patients with NMIBC treated with first-line intravesical BCG, most received adequate BCG induction but less than half had adequate BCG maintenance. BCG treatment was also inadequate for patients with CIS, with only half of patients receiving adequate BCG maintenance and a higher proportion undergoing cystectomy following first-line BCG. Conclusions: Results emphasize the need for additional treatment options for patients with NMIBC.

背景：卡介苗（BCG）可减少高危非肌层浸润性膀胱癌（NMIBC）患者的复发并延缓病情进展，但会给患者带来巨大的精神、身体和社会负担。研究目的本研究评估了美国高危 NMIBC 患者（包括膀胱原位癌 (CIS) 亚组）接受膀胱内卡介苗一线治疗的充分性。治疗方法从去标识化的 MarketScan® 商业、医疗保险和医疗补助数据库（1/1/2010-2/28/2021）中筛选出接受卡介苗治疗的高危 NMIBC 成人患者。使用之前公布的基于索赔的算法（诱导：70 天内≥5 次灌注；诱导和维持：首次灌注后 274 天内≥7 次灌注）和基于具有里程碑意义的西南肿瘤学组 (SWOG) 试验的定义（诱导：≥5 次灌注且间隔时间不超过 7 天；随后在第 3 个月和第 6 个月进行≥2 次灌注，之后每 6 个月进行一次灌注），对卡介苗诱导和维持的充分性进行了评估，评估时间从首次卡介苗索赔开始，直至患者观察结束。报告了获得适当卡介苗诱导和维持治疗的患者比例，并对有 CIS 和无 CIS 的患者进行了比较。结果：在5803名接受一线卡介苗治疗的高危NMIBC患者中（平均年龄67.3岁；20.6%为女性），930人（16.0%）患有CIS。一线卡介苗治疗后，56.6%的患者接受了其他治疗。虽然根据索赔算法，86.9% 的患者接受了适当的卡介苗诱导治疗（SWOG，73.6%），但只有 41.5% 的患者接受了适当的卡介苗诱导和维持治疗（SWOG，1.6%）。在有 CIS 和无 CIS 的患者中观察到了类似的趋势，有 CIS 的患者对指南的依从性更高（使用基于索赔的算法进行充分诱导的比例为 90.3% vs 86.2%；使用基于索赔的算法进行充分诱导的比例为 90.3% vs 86.2%）：90.3% vs 86.2%；充分诱导和维持：讨论：在接受一线膀胱内卡介苗治疗的 NMIBC 患者中，大多数接受了充分的卡介苗诱导治疗，但只有不到一半的患者接受了充分的卡介苗维持治疗。CIS患者的卡介苗治疗也不充分，只有一半的患者得到了充分的卡介苗维持治疗，而且在一线卡介苗治疗后接受膀胱切除术的比例较高。结论：研究结果表明，NMIBC 患者需要更多的治疗方案。

{"title":"Adherence to First-Line Intravesical Bacillus Calmette-Guérin Therapy in the Context of Guideline Recommendations for US Patients With High-Risk Non-muscle Invasive Bladder Cancer.","authors":"Franklin D Gaylis, Bruno Emond, Ameur M Manceur, Anabelle Tardif-Samson, Laura Morrison, Dominic Pilon, Patrick Lefebvre, Lorie A Ellis, Hiremagalur Balaji, Andrea Ireland","doi":"10.36469/001c.124208","DOIUrl":"10.36469/001c.124208","url":null,"abstract":"Background: Bacillus Calmette-Guérin (BCG) can reduce recurrence and delay progression among patients with high-risk non-muscle invasive bladder cancer (NMIBC), but is associated with a substantial emotional, physical, and social burden. Objectives: This study evaluated the adequacy of first-line intravesical BCG treatment among high-risk NMIBC patients in the United States, including the subgroup with carcinoma in situ (CIS) of the bladder. Methods: Adults with high-risk NMIBC treated with BCG were selected from de-identified MarketScan® Commercial, Medicare, and Medicaid Databases (1/1/2010-2/28/2021). Adequacy of BCG induction and maintenance was evaluated from the first BCG claim until the end of the patient's observation, using a previously published claims-based algorithm (induction: ≥5 instillations within 70 days; induction and maintenance: ≥7 instillations within 274 days of first instillation) and a definition based on the landmark Southwest Oncology Group (SWOG) trial (induction: ≥5 instillations without gaps >7 days; followed by ≥2 instillations at month 3, 6, and every 6 months thereafter). Proportions of patients with adequate BCG induction and maintenance were reported overall and compared between those with and without CIS. Results: Of 5803 high-risk NMIBC patients treated with first-line BCG (mean age, 67.3 years; 20.6% female), 930 (16.0%) had CIS. After first-line BCG, 56.6% received another treatment. Although 86.9% had adequate BCG induction based on the claims-based algorithm (SWOG, 73.6%), only 41.5% had adequate BCG induction and maintenance (SWOG, 1.6%). Similar trends were observed for patients with and without CIS, with higher adherence to guidelines for patients with CIS (adequate induction using claims-based algorithm: 90.3% vs 86.2%; adequate induction and maintenance: 50.8% vs 39.7%, all P < .001). A greater proportion of CIS patients than non-CIS patients had cystectomy (CIS, 14.4%, non-CIS, 8.5%; P < .001) after first-line BCG. Discussion: Among patients with NMIBC treated with first-line intravesical BCG, most received adequate BCG induction but less than half had adequate BCG maintenance. BCG treatment was also inadequate for patients with CIS, with only half of patients receiving adequate BCG maintenance and a higher proportion undergoing cystectomy following first-line BCG. Conclusions: Results emphasize the need for additional treatment options for patients with NMIBC.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"109-117"},"PeriodicalIF":2.3,"publicationDate":"2024-10-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11523569/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545866","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}

引用次数: 0

Patient Experience of Living With Hemophilia A: A Conceptual Model of Humanistic and Symptomatic Experience in Adolescents, Adults, and Children. A 型血友病患者的生活体验：青少年、成人和儿童的人文和症状体验概念模型》（Patient Experience of Living With Hemophilia A: A Conceptual Model of Humanistic and Symptomatic Experience in Adolescents, Adults, and Children.

IF 2.3 Q2 ECONOMICS

Journal of Health Economics and Outcomes Research

Pub Date : 2024-10-18 eCollection Date: 2024-01-01 DOI: 10.36469/001c.123374

Zalmai Hakimi, Rakhee Ghelani, Linda Bystrická, Nana Kragh, Patrick Marquis, Jameel Nazir, Nadine McGale

Background: People living with hemophilia A face challenges impacting their daily lives despite treatment innovations. Previous studies have explored perceptions and treatment experiences; however, there is a lack of an evidence-based, comprehensive model to identify concepts (clinical, physical, and psychological functioning) relevant for people with hemophilia A (PwHA). Objectives: The aim of this qualitative study was to address the question: What is the humanistic and symptomatic experience of adolescents, adults, and children living with hemophilia A and what is the impact of hemophilia A on their quality of life? Methods: Participants, identified through patient associations in the UK, were male PwHA and caregivers of male PwHA receiving prophylactic treatment. Qualitative research was conducted involving semistructured telephone interviews with PwHA and caregivers between April 2020 and September 2020 in the UK. Standard analytical techniques of conceptual model development were used. Results: Of 30 participants, 23 were PwHA and 7 were caregivers. A conceptual model was produced describing patient experience of symptoms, physical functioning, treatment experiences, and the impact of symptoms and treatment on daily lives. Participants reported hemophilia-related symptoms, including bleeding, pain, and joint stiffness, as well as difficulties engaging with social and leisure activities. They also reported protection from bleeds provided by their treatment, relief from symptoms, and the resultant sense of normality. Concepts were broadly relevant across all age groups; however, psychological impacts were reported only by adult PwHA, and caregivers reported impacts related to outdoor activities, play, and education. Participants indicated that their ideal treatment would be delivered orally. Discussion: This study highlights the range of symptoms experienced by PwHA across a broad range of age groups, thus enabling the evaluation of relevant concepts across different stages of life. The research supports development of a conceptual model documenting symptoms, impacts, and treatment experience relevant to PwHA. Conclusion: Insights gathered through the interviews and resulting conceptual model support development of new therapies to address the physical and social challenges identified by PwHA and highlight a need for novel hemophilia A treatments that can ease treatment administration, provide adequate level of protection, and enable life to be lived normally.

背景：尽管治疗方法有所创新，但 A 型血友病患者仍面临着影响其日常生活的挑战。以往的研究探讨了患者的看法和治疗经验；然而，目前还缺乏一个以证据为基础的综合模型来确定与 A 型血友病患者（PwHA）相关的概念（临床、身体和心理功能）。研究目的：本定性研究旨在解决以下问题：患有 A 型血友病的青少年、成人和儿童有哪些人文和症状体验，A 型血友病对他们的生活质量有哪些影响？研究方法：通过英国患者协会确定的参与者是男性血友病患者和接受预防性治疗的男性血友病患者的照顾者。定性研究于 2020 年 4 月至 2020 年 9 月在英国对 PwHA 和护理人员进行了半结构化电话访谈。研究中使用了概念模型开发的标准分析技术。结果：在 30 位参与者中，23 位是 PwHA，7 位是护理人员。建立的概念模型描述了患者的症状体验、身体功能、治疗体验以及症状和治疗对日常生活的影响。参与者报告了与血友病有关的症状，包括出血、疼痛和关节僵硬，以及参与社交和休闲活动的困难。他们还报告了治疗提供的出血保护、症状缓解以及由此产生的正常感。这些概念与所有年龄组的人都有广泛的相关性；但是，只有成年 PwHA 报告了心理影响，而护理人员则报告了与户外活动、游戏和教育有关的影响。参与者表示，他们理想的治疗方式是口服治疗。讨论：本研究强调了不同年龄组的 PwHA 所经历的症状范围，从而能够对不同生命阶段的相关概念进行评估。这项研究支持建立一个概念模型，记录与 PwHA 相关的症状、影响和治疗体验。结论：通过访谈和由此产生的概念模型收集到的观点支持开发新的疗法，以应对 PwHA 所面临的身体和社会挑战，并突出了对新型 A 型血友病治疗方法的需求，这种治疗方法可简化治疗管理，提供充分的保护，并使患者能够正常生活。

{"title":"Patient Experience of Living With Hemophilia A: A Conceptual Model of Humanistic and Symptomatic Experience in Adolescents, Adults, and Children.","authors":"Zalmai Hakimi, Rakhee Ghelani, Linda Bystrická, Nana Kragh, Patrick Marquis, Jameel Nazir, Nadine McGale","doi":"10.36469/001c.123374","DOIUrl":"10.36469/001c.123374","url":null,"abstract":"Background: People living with hemophilia A face challenges impacting their daily lives despite treatment innovations. Previous studies have explored perceptions and treatment experiences; however, there is a lack of an evidence-based, comprehensive model to identify concepts (clinical, physical, and psychological functioning) relevant for people with hemophilia A (PwHA). Objectives: The aim of this qualitative study was to address the question: What is the humanistic and symptomatic experience of adolescents, adults, and children living with hemophilia A and what is the impact of hemophilia A on their quality of life? Methods: Participants, identified through patient associations in the UK, were male PwHA and caregivers of male PwHA receiving prophylactic treatment. Qualitative research was conducted involving semistructured telephone interviews with PwHA and caregivers between April 2020 and September 2020 in the UK. Standard analytical techniques of conceptual model development were used. Results: Of 30 participants, 23 were PwHA and 7 were caregivers. A conceptual model was produced describing patient experience of symptoms, physical functioning, treatment experiences, and the impact of symptoms and treatment on daily lives. Participants reported hemophilia-related symptoms, including bleeding, pain, and joint stiffness, as well as difficulties engaging with social and leisure activities. They also reported protection from bleeds provided by their treatment, relief from symptoms, and the resultant sense of normality. Concepts were broadly relevant across all age groups; however, psychological impacts were reported only by adult PwHA, and caregivers reported impacts related to outdoor activities, play, and education. Participants indicated that their ideal treatment would be delivered orally. Discussion: This study highlights the range of symptoms experienced by PwHA across a broad range of age groups, thus enabling the evaluation of relevant concepts across different stages of life. The research supports development of a conceptual model documenting symptoms, impacts, and treatment experience relevant to PwHA. Conclusion: Insights gathered through the interviews and resulting conceptual model support development of new therapies to address the physical and social challenges identified by PwHA and highlight a need for novel hemophilia A treatments that can ease treatment administration, provide adequate level of protection, and enable life to be lived normally.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"95-102"},"PeriodicalIF":2.3,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11523570/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545868","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}

引用次数: 0