Journal of Health Economics and Outcomes Research最新文献

{"title":"Costs of Adverse Event Management Associated with First-Line Cetuximab or Panitumumab in Metastatic Colorectal Cancer Patients in Saudi Arabia.","authors":"Gihan Elsisi, Hana Abdul Kareem, Abdelaziz Alaseiri, Abdullah Alsharm, Mohamed Al Garni, Hajer Al-Mudaiheem, Fouad Alnagar, Hazem Lotfy, Mohamed Ouda, Ahmed Elshehri","doi":"10.36469/001c.130878","DOIUrl":"https://doi.org/10.36469/001c.130878","url":null,"abstract":"<p><p><b>Introduction:</b> In Saudi Arabia, patients with metastatic colorectal cancer (mCRC) with wild-type RAS mutations may be treated with either cetuximab plus chemotherapy (CET + CT) or panitumumab plus chemotherapy (PAN + CT), which are epidermal growth factor receptor (EGFR) antibodies. This study calculated the costs of adverse event (AE) management linked to anti-EGFR treatment in Saudi Arabia's national health budget from payer and societal perspectives. <b>Methods:</b> An adaptation of a global model developed in Microsoft Excel® was performed to estimate the costs of AE management associated with the first-line treatment (CET + CT and PAN + CT) of RAS wild-type mCRC patients in Saudi healthcare settings. The frequencies of common and highly common AEs were sourced from the summaries of product characteristics of CET and PAN, whereas AE severity was captured from a meta-analysis. Unit costs in Saudi Riyal (SAR) were obtained from the National Guard of Health Affairs and Ministry of Health 2024 price lists. The model assumptions, inputs, and results were validated using a local Delphi panel. <b>Results:</b> Within the Saudi payer perspective model, treatment with CET + CT vs PAN + CT resulted in average population cost savings of SAR 9 246 133 (4 741 606 international dollars [Intl <math><mo>]</mo> <mo>)</mo> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>p</mi> <mi>e</mi> <mi>r</mi> <mo>-</mo> <mi>p</mi> <mi>a</mi> <mi>t</mi> <mi>i</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>c</mi> <mi>o</mi> <mi>s</mi> <mi>t</mi> <mi>s</mi> <mi>a</mi> <mi>v</mi> <mi>i</mi> <mi>n</mi> <mi>g</mi> <mi>s</mi> <mi>o</mi> <mi>f</mi> <mi>S</mi> <mi>A</mi> <mi>R</mi> <mn>20</mn> <mrow><mo> </mo></mrow> <mn>321</mn> <mo>(</mo> <mi>I</mi> <mi>n</mi> <mi>t</mi> <mi>l</mi></math> 10 421) for severe AEs, and average population cost savings of SAR 16 039 427 (Intl <math><mn>8</mn> <mrow><mo> </mo></mrow> <mn>225</mn> <mrow><mo> </mo></mrow> <mn>347</mn> <mo>)</mo> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>p</mi> <mi>e</mi> <mi>r</mi> <mo>-</mo> <mi>p</mi> <mi>a</mi> <mi>t</mi> <mi>i</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>c</mi> <mi>o</mi> <mi>s</mi> <mi>t</mi> <mi>s</mi> <mi>a</mi> <mi>v</mi> <mi>i</mi> <mi>n</mi> <mi>g</mi> <mi>s</mi> <mi>o</mi> <mi>f</mi> <mi>S</mi> <mi>A</mi> <mi>R</mi> <mn>35</mn> <mrow><mo> </mo></mrow> <mn>251</mn> <mo>(</mo> <mi>I</mi> <mi>n</mi> <mi>t</mi> <mi>l</mi></math> 18 077) for all-grade AEs. On the other hand, within the Saudi societal perspective model, treatment with CET + CT vs PAN + CT resulted in average population cost savings of SAR 11 386 314 (Intl <math><mn>5</mn> <mrow><mo> </mo></mrow> <mn>839</mn> <mrow><mo> </mo></mrow> <mn>135</mn> <mo>)</mo> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>p</mi> <mi>e</mi> <mi>r</mi> <mo>-</mo> <mi>p</mi> <mi>a</mi> <mi>t</mi> <mi>i</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>c</mi> <mi>o</mi> <mi>s</mi> <mi>t</mi> <mi>s</mi> <mi>a</mi> <mi>v</mi> <mi>i</mi> <mi>n</mi> <mi>g</mi> <mi>s</mi> <mi>o</mi> <mi>f</","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"106-112"},"PeriodicalIF":2.3,"publicationDate":"2025-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11890290/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586030","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 and Budget Impact Analysis of the Use of Faricimab in Diabetic Macular Edema and Neovascular Age-Related Macular Degeneration in Colombia.","authors":"Daniel Samacá-Samacá, Antonio Robles, Hugo Ocampo, Francisco J Rodríguez, Carolina Sardi-Correa, Laura Prieto-Pinto, Christian Bührer, Camilo Tamayo, David Rodríguez, Mauricio Hernández-Quintana","doi":"10.36469/001c.129832","DOIUrl":"10.36469/001c.129832","url":null,"abstract":"<p><p><b>Background:</b> Retinal diseases are major contributors to disability, significantly affecting patients' quality of life. Diabetic macular edema (DME) and neovascular age-related macular degeneration (nAMD) represent a significant disease and economic burden in Colombia. Assessing the economic evaluation of available treatments is essential for the efficient allocation of healthcare resources. <b>Objective:</b> To estimate the cost-effectiveness and budget impact of using faricimab for the treatment of patients with DME and nAMD within the Colombian health system. <b>Methods:</b> This study used a 25-year Markov cohort model to estimate the cost-effectiveness of faricimab vs aflibercept, ranibizumab, and brolucizumab. We used the methodological guidelines of the local health technology assessment agency for conducting the cost-effectiveness and budget impact analysis. Transition probabilities and injection frequencies were derived from the literature. Drug prices were retrieved from public local databases. Quality-adjusted life years (QALYs) were assessed. The potential patient population for the budget impact analysis was estimated based on disease prevalence and expert consultations. <b>Results:</b> Faricimab treat-and-extend (T&E) was dominant vs aflibercept T&E (+0.22 QALYs), ranibizumab T&E (+0.55 QALYs), and brolucizumab for 8 to 12 weeks (+0.06 QALYs) in DME, generating cost savings (in US dollars) of <math><mn>3849</mn> <mo>,</mo></math> 1375, and <math><mn>2824</mn> <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>I</mi> <mi>n</mi> <mi>n</mi> <mi>A</mi> <mi>M</mi> <mi>D</mi> <mo>,</mo> <mi>f</mi> <mi>a</mi> <mi>r</mi> <mi>i</mi> <mi>c</mi> <mi>i</mi> <mi>m</mi> <mi>a</mi> <mi>b</mi> <mi>a</mi> <mi>l</mi> <mi>s</mi> <mi>o</mi> <mi>s</mi> <mi>h</mi> <mi>o</mi> <mi>w</mi> <mi>e</mi> <mi>d</mi> <mi>d</mi> <mi>o</mi> <mi>m</mi> <mi>i</mi> <mi>n</mi> <mi>a</mi> <mi>n</mi> <mi>c</mi> <mi>e</mi> <mi>v</mi> <mi>s</mi> <mi>a</mi> <mi>f</mi> <mi>l</mi> <mi>i</mi> <mi>b</mi> <mi>e</mi> <mi>r</mi> <mi>c</mi> <mi>e</mi> <mi>p</mi> <mi>t</mi> <mi>a</mi> <mi>s</mi> <mi>n</mi> <mi>e</mi> <mi>e</mi> <mi>d</mi> <mi>e</mi> <mi>d</mi> <mo>(</mo> <mo>+</mo> <mn>0.12</mn> <mi>Q</mi> <mi>A</mi> <mi>L</mi> <mi>Y</mi> <mi>s</mi> <mo>)</mo> <mo>,</mo> <mi>r</mi> <mi>a</mi> <mi>n</mi> <mi>i</mi> <mi>b</mi> <mi>i</mi> <mi>z</mi> <mi>u</mi> <mi>m</mi> <mi>a</mi> <mi>b</mi> <mi>a</mi> <mi>s</mi> <mi>n</mi> <mi>e</mi> <mi>e</mi> <mi>d</mi> <mi>e</mi> <mi>d</mi> <mo>(</mo> <mo>+</mo> <mn>0.05</mn> <mi>Q</mi> <mi>A</mi> <mi>L</mi> <mi>Y</mi> <mi>s</mi> <mo>)</mo> <mo>,</mo> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>b</mi> <mi>r</mi> <mi>o</mi> <mi>l</mi> <mi>u</mi> <mi>c</mi> <mi>i</mi> <mi>z</mi> <mi>u</mi> <mi>m</mi> <mi>a</mi> <mi>b</mi> <mn>8</mn> <mi>t</mi> <mi>o</mi> <mn>12</mn> <mi>w</mi> <mi>e</mi> <mi>e</mi> <mi>k</mi> <mi>s</mi> <mo>(</mo> <mo>+</mo>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"97-105"},"PeriodicalIF":2.3,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11884430/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573246","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":"The Effect of <i>Dobbs v. Jackson Women's Health Organization</i> on Clinical Diagnosis of Postpartum Depression.","authors":"Onur Baser, Yuanqing Lu, Facundo Sepulveda, Ariani Alemzadeh, Amy Endrizal","doi":"10.36469/001c.129633","DOIUrl":"https://doi.org/10.36469/001c.129633","url":null,"abstract":"<p><p><b>Background:</b> The 2022 US Supreme Court decision in <i>Dobbs v. Jackson Women's Health Organization</i> eliminated the constitutional right to abortion and activated trigger laws in 21 states, either banning or significantly restricting abortion access. This study estimated changes in postpartum depression (PPD) diagnoses after <i>Dobbs</i> in states with trigger laws vs those without. <b>Methods:</b> Medicaid data from Kythera Labs spanning December 2019 to June 2024 were utilized. Difference-in-difference models assessed changes in PPD diagnosis rates post-<i>Dobbs</i> (21 trigger states, 29 non-trigger states). <b>Results:</b> Women in trigger states were younger (mean, 26.53 vs 27.98 years), more likely to reside in low socioeconomic status areas (41.28% vs 24.42%) and less likely to have obstetrical complications (66.06% vs 77.36%), maternal complications (16.41% vs 18.9%), and lifestyle risk factors (13.58% vs 21.17%). Baseline PPD diagnosis rates were 8.51% in trigger states and 12.66% in non-trigger states. Post-<i>Dobbs</i>, PPD diagnosis rates were 10.20% in trigger states and 14.34% in non-trigger states. <b>Conclusions:</b> Overall, women in states with abortion trigger laws experienced a small positive but statistically insignificant increase in PPD diagnoses following <i>Dobbs</i> compared with those in non-trigger states.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"86-96"},"PeriodicalIF":2.3,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11869945/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143541814","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 Analysis of Nirmatrelvir/Ritonavir Compared with Molnupiravir in Patients at High Risk for Progression to Severe COVID-19 in Japan.","authors":"Akira Yuasa, Hiroyuki Matsuda, Yosuke Fujii, Tendai Mugwagwa, Yuki Kado, Mizuki Yoshida, Koichi Murata, Yoshiaki Gu","doi":"10.36469/001c.129067","DOIUrl":"10.36469/001c.129067","url":null,"abstract":"&lt;p&gt;&lt;p&gt;&lt;b&gt;Background:&lt;/b&gt; Nirmatrelvir/ritonavir (NMV/r) and molnupiravir are oral antiviral drugs approved for the treatment of early symptomatic patients with mild to moderate COVID-19 at high risk of progression to severe disease in Japan. &lt;b&gt;Objective:&lt;/b&gt; This study evaluated, from a Japanese payer perspective, the cost-effectiveness of NMV/r compared with molnupiravir among patients with COVID-19. &lt;b&gt;Methods:&lt;/b&gt; This cost-effectiveness model describes the COVID-19 disease history and the impact of antiviral treatment on short-term and long-term outcomes. Nirmatrelvir/ritonavir was compared with molnupiravir, and in the scenario analysis NMV/r was compared with standard of care over a lifetime horizon. &lt;b&gt;Results:&lt;/b&gt; When compared with molnupiravir, NMV/r showed higher quality-adjusted life years (QALYs) (15.752 vs 15.739) and higher total cost (¥6 248 014 vs ¥6 245 829 [US &lt;math&gt;&lt;mn&gt;44&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;136.86&lt;/mn&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt;&lt;/math&gt; 44 121.42]). The incremental cost-effectiveness ratio was ¥164 934 (US &lt;math&gt;&lt;mn&gt;1165.12&lt;/mn&gt; &lt;mo&gt;)&lt;/mo&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;Q&lt;/mi&gt; &lt;mi&gt;A&lt;/mi&gt; &lt;mi&gt;L&lt;/mi&gt; &lt;mi&gt;Y&lt;/mi&gt; &lt;mi&gt;g&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;g&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mo&gt;-&lt;/mo&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mo&gt;-&lt;/mo&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mo&gt;(&lt;/mo&gt; &lt;mi&gt;W&lt;/mi&gt; &lt;mi&gt;T&lt;/mi&gt; &lt;mi&gt;P&lt;/mi&gt; &lt;mo&gt;)&lt;/mo&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;J&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mo&gt;(&lt;/mo&gt; &lt;mrow&gt;&lt;mo&gt;¥&lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;5&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;000&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;000&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt;/&lt;/mo&gt;&lt;/mrow&gt; &lt;mi&gt;Q&lt;/mi&gt; &lt;mi&gt;A&lt;/mi&gt; &lt;mi&gt;L&lt;/mi&gt; &lt;mi&gt;Y&lt;/mi&gt; &lt;mo&gt;[&lt;/mo&gt; &lt;mi&gt;U&lt;/mi&gt; &lt;mi&gt;S&lt;/mi&gt;&lt;/math&gt; 35 320.71/QALY]). In the scenario analysis, the incremental cost-effectiveness ratio was ¥3 646 821 (US &lt;math&gt;&lt;mn&gt;25&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;761.66&lt;/mn&gt; &lt;mo&gt;)&lt;/mo&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;Q&lt;/mi&gt; &lt;mi&gt;A&lt;/mi&gt; &lt;mi&gt;L&lt;/mi&gt; &lt;mi&gt;Y&lt;/mi&gt; &lt;mi&gt;g&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mo&gt;.&lt;/mo&gt; &lt;mi&gt;O&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mo&gt;-&lt;/mo&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;i&lt;/","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"75-85"},"PeriodicalIF":2.3,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11864595/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143516031","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":"Clinical and Economic Outcomes in Patients With Alpha-1 Antitrypsin Deficiency in a US Medicare Advantage Population.","authors":"Nikhil Khandelwal, Jimmy Hinson, Trinh Nguyen, Alexjandro Daviano, Yihua Xu, Brandon T Suehs, Sally Higgins, Marie Sanchirico, J Michael Wells","doi":"10.36469/001c.127446","DOIUrl":"10.36469/001c.127446","url":null,"abstract":"<p><p><b>Background:</b> Alpha-1 antitrypsin deficiency (AATD) testing rates and associated clinical and economic outcomes data in the US Medicare population are limited. <b>Objective:</b> To characterize individuals with AATD, describe clinical outcomes/healthcare research utilization (HCRU) among individuals with chronic obstructive pulmonary disease (COPD) with or without AATD, and identify AATD testing rates among individuals newly diagnosed with COPD. <b>Methods:</b> This retrospective, observational analysis of claims data included individuals from the Humana Research Database (aged 18-89 years) enrolled in Medicare Advantage Prescription Drug plans. Three cohorts included individuals with evidence of AATD; individuals with COPD + AATD matched to individuals with COPD; and individuals with newly diagnosed COPD. AATD health-related outcomes, such as pulmonary and extrapulmonary conditions or events, and economic outcomes, including inpatient admissions, emergency department visits, and physician visits, were examined independently during the pre-index and post-index periods and compared between those with ATTD and without AATD. <b>Results:</b> We identified 1103 individuals with AATD (aged 67.2 ± 10.0 years, 56.3% women, 94.5% White); overall, 22.2% had exacerbations, respiratory distress, and respiratory failure. Individuals with COPD and AATD (n = 742) were matched to individuals with COPD (n = 7420), based on age (68 ± 9 years), sex (55.0% women), and race (97.2% White). The AATD group had a higher proportion of emphysema (47.4% vs 18.7%), COPD exacerbations (40.6% vs 24.7%), and cirrhosis (4.0% vs 1.3%) than the non-AATD group. All-cause inpatient admissions (31.7% vs 27.3%), COPD-specific inpatient admissions (7.4% vs 4.3%), and COPD-specific emergency department visits (19.5% vs 10.8%) were higher in individuals who had ATTD than in those without AATD. AATD testing rates among individuals with newly diagnosed COPD increased slightly over time (2015: 1.07%; 2020: 1.49%). Individuals with COPD and AATD had more comorbidities and higher HCRU. Testing rates increased slightly but remained low. <b>Discussion:</b> Further research is needed to assess the impact of improved AATD testing on those with COPD. <b>Conclusion:</b> Increased awareness, earlier testing, and treatment may reduce the healthcare burden of AATD in the US Medicare population.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"66-74"},"PeriodicalIF":2.3,"publicationDate":"2025-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11846658/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143483412","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Vignette Study to Derive Health-Related Quality-of-Life Weights for Individuals with Steroid Refractory Chronic Graft-versus-Host Disease Receiving Third-Line Therapy in the United Kingdom.","authors":"Emma Williams, Luke Skinner, Richard Hudson, Arunesh Sil, Katharina Ecsy, Elisheva Lew, Abdul Jabbar Omar Alsaleh, Elin Gruffydd, Andrew Lloyd, Daniele Avenoso","doi":"10.36469/001c.125546","DOIUrl":"10.36469/001c.125546","url":null,"abstract":"<p><p><b>Background:</b> Chronic graft-versus-host disease (cGvHD) - a potentially debilitating complication of allogeneic hematopoietic stem cell transplantation - is a rare condition. <b>Objectives:</b> This vignette-based study aimed to generate utility values to inform an economic model via an online survey wherein cGvHD health state (HS) vignettes were valued by the general UK population using the EQ-5D-5L and the EQ-5D-visual analog scale (EQ-5D VAS). <b>Methods:</b> This non-interventional health-related quality of life (HRQoL) study was conducted in 3 stages across the UK: the development, validation, and valuation of HS vignettes to generate utility values for cGvHD. Four HS for cGvHD were defined based on an economic model partitioning different treatment level responses in patients with cGvHD receiving third-line (3L) therapy (HS1: complete response, HS2: partial response, HS3: lack of response, and HS4: recurrent cGvHD). Draft vignettes were developed for each HS based on 4 previously published GvHD vignettes. The contents of the draft vignettes were reviewed for all aspects of cGvHD symptoms and functional impact and validated through semistructured interviews with 5 clinical experts. The 4 finalized HS vignettes were valued by 300 participants from the UK general population using EQ-5D-5L and EQ-5D VAS. <b>Results:</b> Previously published vignettes were used to develop the vignettes for the current study that described GvHD in the context of blood cancer and other rare blood disorders (n = 2 each) and included symptoms, functioning, and quality of life for a patient in the HS. The highest and lowest mean EQ-5D-5L utility scores were observed for HS1 (mean [95% CI]: 0.577 [0.558-0.595]) and HS4 (0.061 [0.034-0.088]), respectively. The EQ-5D-VAS showed the highest and lowest mean utility scores for HS1 (46.8 [44.9-48.6]) and HS4 (25.6 [23.4-27.7]), respectively. <b>Conclusion:</b> This study generated utility values for HS vignettes describing symptoms, functioning, and HRQoL for patients with cGvHD receiving 3L therapy. The utility values highlighted a substantial burden of cGvHD and HRQoL impact associated with the treatment response level. However, assessing concordance between utility estimates derived from the vignette-based method in a general population and those from patients with cGvHD is further warranted.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"58-65"},"PeriodicalIF":2.3,"publicationDate":"2025-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11844253/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143483408","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":"Drug Insurance and Psoriasis Severity: A Retrospective Cohort Study.","authors":"Laurence Mainville, Hélène Veillette, Paul R Fortin","doi":"10.36469/001c.127820","DOIUrl":"10.36469/001c.127820","url":null,"abstract":"<p><p><b>Background:</b> Prescription drug insurance in Canada is constituted of a patchwork of public and private insurance plans. The type of drug insurance may have a negative impact on access to treatment for patients covered by public plans compared with private plans. <b>Objectives:</b> In patients with psoriasis treated with advanced therapy in public vs private drug insurance groups, we compared: (1) psoriasis severity scores when an advanced therapy was prescribed, (2) psoriasis severity scores at follow-up, (3) treatment response, and (4) delay between prescription and first dose of advanced therapy. <b>Methods:</b> This unicentric, retrospective cohort study included patients suffering from psoriasis treated by advanced therapy, dermatologist-prescribed between September 2015 and August 2019, in a tertiary academic care center in Québec City, Canada. Data were collected from medical records. <b>Results:</b> Patients treated with an advanced therapy for psoriasis covered under the provincial public drug insurance plan (n = 78) and under a private drug plan (n = 93) did not differ regarding the studied outcomes. Patients' characteristics differed between groups. Patients in the public group were older (<i>P</i> < .0001), more socioeconomically deprived (<i>P</i> < .05), and more likely to benefit from compassion from the industry to access a prescribed medication free of charge (<i>P</i> < .0001) compared with patients from the privately insured group. <b>Discussion:</b> The high prevalence of compassionate programs from the industry in the public insurance group (42% vs 14%), and the high prevalence of psoriasis on difficult-to-treat areas (face, genitalia, and/or palmoplantar areas) in our cohort (85.4%) may mask differences in access to advanced therapy between the two groups. <b>Conclusions:</b> Prescribers of advanced therapy can be reassured, as we found no inequality in access or care based on patients' drug insurance coverage.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"51-57"},"PeriodicalIF":2.3,"publicationDate":"2025-02-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11807372/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143382656","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":"Are Trends in Economic Modeling of Pediatric Diabetes Mellitus up to Date with the Clinical Practice Guidelines and the Latest Scientific Findings?","authors":"Roque Cardona-Hernandez, Alberto de la Cuadra-Grande, Julen Monje, María Echave, Itziar Oyagüez, María Álvarez, Isabel Leiva-Gea","doi":"10.36469/001c.127920","DOIUrl":"10.36469/001c.127920","url":null,"abstract":"<p><p><b>Background:</b> Modeling techniques in the field of pediatrics present unique challenges beyond traditional model limitations, and sometimes difficulties in faithfully simulating the condition's evolution over time. <b>Objective:</b> This study aimed to identify whether economic modeling approaches in diabetes in pediatric patients align with the recommendations of clinical practice guidelines and the latest scientific evidence. <b>Methods:</b> A literature review was performed in March 2023 to identify modeling-based economic evaluations in diabetes in pediatric patients. Data were extracted and synthesized from eligible studies. Clinical practice guidelines for diabetes were gathered to compare their alignment with modeling strategies. Two endocrinology specialists provided insights on the latest findings in diabetes that are not yet included in the guidelines. A multidisciplinary group of experts agreed on the relevant themes to conduct the comparative analysis: parameter informing on glycemic control, diabetic ketoacidosis/hypoglycemia, C-peptide as prognostic biomarker, metabolic memory, age at diagnosis, socioeconomic status, pediatric-specific sources of risk equations, and pediatric-specific sources of utilities/disutilities. <b>Results:</b> Nineteen modeling-based studies (7 de novo, 12 predesigned models) and 34 guidelines were selected. Hemoglobin A1c was the main parameter to model the glycemic control; however, guidelines recommend the usage of complementary measures (eg, time in range) which are not included in economic models. Eight models included diabetic ketoacidosis (42.1%), 16 included hypoglycemia (84.2%), 2 included C-peptide (1 of those as prognostic factor) (10.5%) and 1 included legacy effect (5.3%). Neither guidelines nor models included recent findings, such as age at diagnosis or socioeconomic status, as prognostic factors. The lack of pediatric-specific sources for risk equations and utility/disutility values were additional limitations. <b>Discussion:</b> Economic models designed for assessing interventions in diabetes in pediatric patients should be based on pediatric-specific data and include novel adjuvant glucose-monitoring metrics and latest evidence on prognostic factors (C-peptide, legacy effect, age at diagnosis, socioeconomic status) to provide a more faithful reflection of the disease. <b>Conclusions:</b> Economic models represent useful tools to inform decision making. However, further research assessing the gaps is needed to enhance evidence-based health economic modeling that best represents reality.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"30-50"},"PeriodicalIF":2.3,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11797704/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143255734","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 Associated with Leukopenia and Neutropenia in Kidney Transplant Recipients Receiving Valganciclovir in the United States.","authors":"Qinghua Li, Vladimir Turzhitsky, Pamela Moise, Harry Jin, Kaylen Brzozowski, Irina Kolobova","doi":"10.36469/001c.125097","DOIUrl":"10.36469/001c.125097","url":null,"abstract":"<p><p><b>Background:</b> Cytomegalovirus prophylaxis in kidney transplant recipients (KTRs) is limited by post-transplant neutropenia and leukopenia (PTN/PTL). Despite its clinical significance, the healthcare resource utilization (HCRU) related to PTN/PTL remains poorly characterized. <b>Objective:</b> To evaluate HCRU among KTRs taking valganciclovir during their first year post-transplant. <b>Methods:</b> Using TriNetX Dataworks-USA, a federated, de-identified electronic medical record database, we identified adult KTRs who underwent their first kidney transplant from January 2012 to September 2020. All eligible patients were followed for 1 year. PTN/PTL was defined as absolute neutrophil count less than 1000/μL or white blood cell count less than 3500/μL. Multivariable logistic/Poisson regression models were used to assess the association between PTN/PTL and various HCRU types. <b>Results:</b> A total of 8791 KTRs were identified, of whom 6219 (70.7%) developed PTN/PTL at a mean of 5.7 months post-transplantation. Hospitalizations, rehospitalizations, emergency room visits, outpatient appointments, packed red blood cell transfusions, and granulocyte-colony stimulating factor administration were more prevalent among KTRs with PTN/PTL (61.1% vs 49.5%, 24.5% vs 14.1%, 35.2% vs 28.9%, 30.4 vs 26.2 visits, 22.3% vs 17.6%, 23.4% vs 2.2%, respectively; P < .001). Adjusted analyses confirmed that PTN/PTL correlated with increased HCRU across all categories. <b>Conclusions:</b> KTRs who developed PTN/PTL had significantly higher HCRU. Further studies are needed to evaluate strategies addressing PTN/PTL for KTRs.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"22-29"},"PeriodicalIF":2.3,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11784900/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143080474","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":"Glycogen Storage Disease Type Ia: A Retrospective Claims Analysis of Complications, Resource Utilization, and Cost of Care.","authors":"Eliza Kruger, Justin Nedzesky, Nina Thomas, Jeffrey D Dunn, Andrew A Grimm","doi":"10.36469/001c.125886","DOIUrl":"10.36469/001c.125886","url":null,"abstract":"<p><p><b>Background:</b> Glycogen storage disease type Ia (GSDIa) is a rare inherited disorder resulting in potentially life-threatening hypoglycemia, metabolic abnormalities, and complications often requiring hospitalization. <b>Objective:</b> This retrospective database analysis assessed the complications, resource utilization, and costs in a large cohort of patients with GSDIa. <b>Methods:</b> We conducted a retrospective cohort study of GSDIa patients and matched non-GSDIa comparators utilizing the PharMetrics® Plus database. International Classification of Diseases, Tenth Revision (ICD-10) diagnosis codes in any billing position for inpatient and outpatient claims (January 2016-February 2020) were identified for complications related to GSDIa. Healthcare use and costs were assessed by setting of care (inpatient, outpatient, physician office, emergency department, and pharmacy). <b>Results:</b> Overall, 557 patients with GSDIa and 5570 matched comparators (male, 63%; adults, 67%) were identified. The most frequent complications in patients with GSDIa vs comparators included anemia due to enzyme disorders (odds ratio, 4.0 × 103; 95% confidence interval, 555.9-2.8 × 104), hepatocellular adenoma (305.9; 41.6-2.2 × 104), liver transplantation (164.6; 21.8-1.2 × 103), and gastrostomy (152.2; 61.1-379.2), as well as acidosis (45.5; 29.4-70.3), hepatomegaly (43.6; 29.1-65.3), hyperuricemia (23.6; 11.9-46.9), and hypoglycemia (20.2; 14.3-28.7). Chronic complications (eg, gout, osteoarthritis, chronic kidney disease, and neoplasms) were more common in adults with GSDIa, whereas acute complications (eg, poor growth, gastrostomy, seizure, and hypoglycemia) were more common in children with GSDIa. Patients with GSDIa more often required hospitalization (0.53 vs 0.06 hospitalizations per patient per year) vs comparators, including 2 or more hospitalizations (26.6% vs 2.3%), longer length of stay (3.1 vs 0.4 days), and more annual visits in all care settings, including 4.3 times more visits in the emergency department. Mean annual total healthcare costs were higher for GSDIa patients vs comparators ( <math><mn>33</mn> <mrow><mo> </mo></mrow> <mn>910</mn> <mi>v</mi> <mi>s</mi></math> 4410). <b>Discussion:</b> In this large, retrospective database analysis, complications observed among patients with GSDIa were consistent with prior reports and demonstrate the chronic and progressive nature of the disease. Resource utilization was substantial in GSDIa patients, and mean annual total healthcare costs were almost 8 times higher than those of comparators. <b>Conclusions:</b> GSDIa is associated with numerous potentially serious and sometimes fatal complications, extensive resource utilization, and high management costs.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"13-21"},"PeriodicalIF":2.3,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11716495/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142950020","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}