PharmacoEconomics Open最新文献

{"title":"Cost of Patients with Alzheimer's Disease in Spain According to Disease Severity.","authors":"Laura Gómez Maldonado, Ricardo de Mora-Figueroa, Angélica López-Angarita, Paulina Maravilla-Herrera, María Merino","doi":"10.1007/s41669-023-00451-w","DOIUrl":"10.1007/s41669-023-00451-w","url":null,"abstract":"<p><strong>Background: </strong>Alzheimer's disease (AD) has been associated with great healthcare and non-healthcare resource consumption. The aim of this study was to estimate the burden of AD in Spain according to disease severity from a societal perspective.</p><p><strong>Methods: </strong>A self-administered questionnaire was designed by the researchers and completed by the informal caregivers of patients with AD, reporting data on themselves as caregivers and on the AD patients for whom they care. The patients' sociodemographic and clinical data, their healthcare and non-healthcare resource consumption in the previous 12 months, and the impact of the disease on labor productivity were compiled. Data collected on informal caregivers included sociodemographic data and the impact of caring for a person with AD on their quality of life and labor productivity. Costs were estimated by multiplying the number of consumed resources by their unit prices. The cost of informal care was assessed using the proxy good method, and labor productivity losses were estimated using the human capital method. Costs were estimated by disease severity and are presented per patient per year in 2021 euros (€).</p><p><strong>Results: </strong>The study sample comprised 171 patients with AD aged 79.1 ± 7.4 years; 68.8% were female, time from diagnosis was 5.8 ± 4.1 years, diagnosis delay was 1.8 ± 2.3 years, and the mean Cumulative Illness Rating Scale-Geriatric (CIRS-G) total was score 8.2 ± 6.0. According to disease severity, 14% had mild cognitive impairment or mild AD, 43.9% moderate AD, and 42.1% severe AD. The average annual cost per patient was €42,336.4 in the most conservative scenario. The greatest proportion of this cost was attributed to direct non-healthcare costs (86%, €36,364.8), followed by direct healthcare costs (8.6%, €3647.1), social care costs (4.6%, €1957.1), and labor productivity losses (less than 1%, €367.4). Informal care was the highest cost item, representing 80% of direct non-healthcare costs and 69% of the total cost. The total direct non-healthcare cost and total cost were significantly higher in moderate to severe disease severities, compared to milder disease severity.</p><p><strong>Conclusions: </strong>AD poses a substantial burden on informal caregivers, the national healthcare system, and society at large. Early diagnosis and treatment to prevent disease progression could reduce this economic impact.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10781927/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"107591844","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":"United States Value Set for the Functional Assessment of Cancer Therapy-General Eight Dimensions (FACT-8D), a Cancer-Specific Preference-Based Quality of Life Instrument.","authors":"Madeleine T King, D A Revicki, R Norman, F Müller, R C Viney, A S Pickard, D Cella, J W Shaw","doi":"10.1007/s41669-023-00448-5","DOIUrl":"10.1007/s41669-023-00448-5","url":null,"abstract":"<p><strong>Objectives: </strong>To develop a value set reflecting the United States (US) general population's preferences for health states described by the Functional Assessment of Cancer Therapy (FACT) eight-dimensions preference-based multi-attribute utility instrument (FACT-8D), derived from the FACT-General cancer-specific health-related quality-of-life (HRQL) questionnaire.</p><p><strong>Methods: </strong>A US online panel was quota-sampled to achieve a general population sample representative by sex, age (≥ 18 years), race and ethnicity. A discrete choice experiment (DCE) was used to value health states. The valuation task involved choosing between pairs of health states (choice-sets) described by varying levels of the FACT-8D HRQL dimensions and survival (life-years). The DCE included 100 choice-sets; each respondent was randomly allocated 16 choice-sets. Data were analysed using conditional logit regression parameterized to fit the quality-adjusted life-year framework, weighted for sociodemographic variables that were non-representative of the US general population. Preference weights were calculated as the ratio of HRQL-level coefficients to the survival coefficient.</p><p><strong>Results: </strong>2562 panel members opted in, 2462 (96%) completed at least one choice-set and 2357 (92%) completed 16 choice-sets. Pain and nausea were associated with the largest utility weights, work and sleep had more moderate utility weights, and sadness, worry and support had the smallest utility weights. Within dimensions, more severe HRQL levels were generally associated with larger weights. A preference-weighting algorithm to estimate US utilities from responses to the FACT-General questionnaire was generated. The worst health state's value was -0.33.</p><p><strong>Conclusions: </strong>This value set provides US population utilities for health states defined by the FACT-8D for use in evaluating oncology treatments.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10781923/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138499147","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":"Developing a Natural History Model for Duchenne Muscular Dystrophy.","authors":"Jonathan Broomfield, M Hill, F Chandler, M J Crowther, J Godfrey, M Guglieri, J Hastie, J Larkindale, J Mumby-Croft, E Reuben, F Woodcock, K R Abrams","doi":"10.1007/s41669-023-00450-x","DOIUrl":"10.1007/s41669-023-00450-x","url":null,"abstract":"<p><strong>Background: </strong>The aim of this study was to pool multiple data sets to build a patient-centric, data-informed, natural history model (NHM) for Duchenne muscular dystrophy (DMD) to estimate disease trajectory across patient lifetime under current standard of care in future economic evaluations. The study was conducted as part of Project HERCULES, a multi-stakeholder collaboration to develop tools to support health technology assessments of new treatments for DMD.</p><p><strong>Methods: </strong>Health states were informed by a review of NHMs for DMD and input from clinicians, patients and caregivers, and defined using common outcomes in clinical trials and real-world practice. The primary source informing the NHM was the Critical Path Institute Duchenne Regulatory Science Consortium (D-RSC) database. This was supplemented with expert input obtained via an elicitation exercise, and a systematic literature review and meta-analysis of mortality data.</p><p><strong>Results: </strong>The NHM includes ambulatory, transfer and non-ambulatory phases, which capture loss of ambulation, ability to weight bear and upper body and respiratory function, respectively. The NHM estimates patients spend approximately 9.5 years in ambulatory states, 1.5 years in the transfer state and the remainder of their lives in non-ambulatory states. Median predicted survival is 34.8 years (95% CI 34.1-35.8).</p><p><strong>Conclusion: </strong>The model includes a detailed disease pathway for DMD, including the clinically and economically important transfer state. The NHM may be used to estimate the current trajectory of DMD in economic evaluations of new treatments, facilitating inclusion of a lifetime time horizon, and will help identify areas for further research.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10781931/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138452077","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":"Specialist Palliative Care and Health Care Costs at the End of Life.","authors":"Patricia Kenny, Dan Liu, Denzil Fiebig, Jane Hall, Jared Millican, Sanchia Aranda, Kees van Gool, Philip Haywood","doi":"10.1007/s41669-023-00446-7","DOIUrl":"10.1007/s41669-023-00446-7","url":null,"abstract":"<p><strong>Background/aims: </strong>The use and costs of health care rise substantially in the months prior to death, and although the use of palliative care services may be expected to lead to less costly care, the evidence is mixed. We analysed the costs of care over the last year of life and the extent to which these are associated with the use and duration of specialist palliative care (SPC) for decedents who died from cancer or another life-limiting illness.</p><p><strong>Methods: </strong>The decedents were participants in a cohort study of older residents of the state of New South Wales, Australia. Using linked survey and administrative health data from 2007 to 2016, two cohorts were identified: n = 10,535 where the cause of death was cancer; and n = 11,179 where the cause of death was another life-limiting illness. Costs of various types were analysed with separate risk-adjusted linear regression models for the last 1, 3, 6, 9 and 12 months before death and for both cohorts. SPC was categorised according to time to death from first contact with the service as 1-7 days, 7-30 days, 30-180 days and more than 180 days.</p><p><strong>Results: </strong>SPC use was higher among the cancer cohort (30.0%) relative to the non-cancer cohort (4.8%). The mean costs over the final year of life were AU$55,037 (SD 45,059) for the cancer cohort and AU$35,318 (SD 41,948) for the non-cancer cohort. Earlier use of SPC was associated with higher costs over the last year of life but lower costs in the last 1 and 3 months for both cohorts. Initiating SPC use more than 180 days before death was associated with a mean difference relative to the no SPC group of AU$15,590 (95% CI 10,617 to 20,562) and AU$13,739 (95% CI 733 to 26,746) over the last year of life for those dying from cancer and another illness, respectively. The same differences over the last month of life were - AU$2810 (95% CI -  3945 to -  1676) and - AU$4345 (95% CI -  6625 to - 2066). Admitted hospital care was the major driver of costs, with longer SPC associated with lower rates of death in hospital for both cohorts.</p><p><strong>Conclusion: </strong>Early initiation of SPC was associated with higher costs over the last year of life and lower costs over the last months of life. This was the case for both the cancer and non-cancer cohorts, and appeared to be largely attributed to reduced hospitalisation. Although further investigation is required, our results suggest that expanding the availability of SPC services to provide more equitable access could enable patients to spend more time at their usual place of residence, reduce pressure on inpatient services and facilitate death at home when that is preferred.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10781921/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71425814","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":"Economic Burden of Delayed Diagnosis in Patients with Pulmonary Arterial Hypertension (PAH).","authors":"Hilary M DuBrock, Hayley D Germack, Marjolaine Gauthier-Loiselle, Jörg Linder, Ambika Satija, Ameur M Manceur, Martin Cloutier, Patrick Lefebvre, Sumeet Panjabi, Robert P Frantz","doi":"10.1007/s41669-023-00453-8","DOIUrl":"10.1007/s41669-023-00453-8","url":null,"abstract":"<p><strong>Background: </strong>The aim of this study was to assess health care resource utilization (HRU) and costs associated with delayed pulmonary arterial hypertension (PAH) diagnosis in the United States.</p><p><strong>Methods: </strong>Eligible adults with newly diagnosed PAH from Optum's de-identified Clinformatics^® Data Mart Database (2016-2021) were assigned to mutually exclusive cohorts based on time between first PAH-related symptom and first PAH diagnosis (i.e., ≤12 months' delay, >12 to ≤24 months' delay, >24 months' delay). All-cause HRU and health care costs per patient per month (PPPM) were assessed during the first year following diagnosis and compared across cohorts using regression analysis adjusted for baseline covariates. Sensitivity analyses were conducted to assess outcomes during all available follow-up post-diagnosis.</p><p><strong>Results: </strong>Among 538 patients (mean age: 65.6 years; 60.6% female), 60.8% had ≤12 months' delay, 23.4% had a delay of >12 to ≤24 months, and 15.8% had >24 months' delay. Compared with ≤12 months, delays of >12 to ≤24 months and >24 months were associated with increased hospitalizations (incidence rate ratio [95% confidence interval]: 1.40 [1.11-1.71] vs 1.71 [1.29-2.12]) and outpatient visits (1.17 [1.06-1.30] vs 1.26 [1.08-1.41]). Longer delays were also associated with more intensive care unit (ICU) stays and 30-day readmissions. Diagnosis delays translated into excess costs PPPM of US$3986 [1439-6436] for >12 to ≤24 months and US$5366 [2107-8524] for >24 months compared with ≤12 months' delay; increased hospitalization costs (US$3248 [1108-5135] and US$4048 [1401-6342], respectively) being the driver. Sensitivity analyses yielded similar trends.</p><p><strong>Conclusions: </strong>Delayed PAH diagnosis is associated with significant incremental economic burden post-diagnosis, driven by hospitalizations including ICU stays and 30-day readmissions, highlighting the need for increased awareness and a potential benefit of earlier screening.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10781905/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138047779","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":"Early Cost-Effectiveness Analysis of Cytoreductive Surgery and Hyperthermic Intraperitoneal Chemotherapy for Gastric Cancer Patients with Limited Peritoneal Carcinomatosis.","authors":"Joost G E Verbeek, Karen van der Sluis, Marieke A Vollebergh, Johanna W van Sandick, Wim H van Harten, Valesca P Retèl","doi":"10.1007/s41669-023-00454-7","DOIUrl":"10.1007/s41669-023-00454-7","url":null,"abstract":"<p><strong>Background: </strong>Gastric cancer patients with peritoneal carcinomatosis (PC) have a poor prognosis, with a median overall survival of 10 months when treated with systemic chemotherapy only. Cohort studies showed that cytoreductive surgery with hyperthermic intraperitoneal chemotherapy (CRS/HIPEC) might improve the prognosis for gastric cancer patients with limited PC. Besides generating trial data on clinical effectiveness, it is crucial to timely collect information on economic aspects to guide the reimbursement decision-making process. No previous data have been published on the cost(-effectiveness) of CRS/HIPEC in this group of patients. Therefore, we performed an early model-based cost-effectiveness analysis of CRS/HIPEC for gastric cancer patients with limited PC in the Dutch setting.</p><p><strong>Methods: </strong>We constructed a two-state (alive-dead) Markov transition model to evaluate costs and clinical outcomes from a Dutch healthcare perspective. Clinical outcomes, transition probabilities and utilities were derived from literature and verified by clinical experts in the field. Costs were measured using two available representative cohorts (2010-2017): one 'systemic chemotherapy only' cohort and one 'CRS/HIPEC' cohort (n = 10 each). Incremental cost-utility ratios (ICURs) were expressed as Euros per quality-adjusted life-year (QALY). We performed probabilistic and deterministic sensitivity, scenario, and value-of-information analyses using a willingness-to-pay (WTP) threshold of €80,000/QALY, which reflects the Dutch norm for severe diseases.</p><p><strong>Results: </strong>In the base-case analysis, CRS/HIPEC yielded more QALYs (increment of 0.68) and more costs (increment of €34,706) compared with systemic chemotherapy only, resulting in an ICUR of €50,990/QALY. The probability that CRS/HIPEC was cost effective compared with systemic chemotherapy alone was 64%. To reduce uncertainty, the expected value of perfect information amounted to €4,021,468. The scenario analyses did not alter the results and showed that treatment costs, lifetime health-related quality of life and overall survival had the largest influence on the model.</p><p><strong>Conclusions: </strong>The presented early cost-effectiveness analysis suggests that adding CRS/HIPEC to systemic chemotherapy for gastric cancer patients with limited PC has a good chance of being cost-effectiveness compared with systemic chemotherapy alone when using a WTP of €80,000/QALY. However, there is substantial uncertainty in view of the current available data on effectiveness. Results from the ongoing phase III PERISCOPE II trial are therefore crucial for further decisions on treatment policy and its cost-effectiveness.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10781926/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138461419","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":"Preferences for Living Arrangements in Dementia: A Discrete Choice Experiment.","authors":"Christian Speckemeier, Carina Abels, Klemens Höfer, Anja Niemann, Jürgen Wasem, Anke Walendzik, Silke Neusser","doi":"10.1007/s41669-023-00452-9","DOIUrl":"10.1007/s41669-023-00452-9","url":null,"abstract":"<p><strong>Background: </strong>Dementia affects about 55 million people worldwide. Demographic change and shifting lifestyles challenge the organization of dementia care. A discrete choice experiment (DCE) was conducted to elicit preferences for living arrangements in dementia in urban and rural regions of Germany.</p><p><strong>Methods: </strong>Preliminary work included review of previous literature and focus groups. The DCE consists of seven attributes (group size, staff qualifications, organization of care, activities offered, support of religious practice, access to garden, consideration of food preferences) with three levels each. Individuals from the general population between the ages of 50 and 65 years were identified through population registration offices in three rural municipalities and one urban area, and 4390 individuals were approached via postal survey. A hierarchical Bayesian mixed logit model was estimated and interactions with sociodemographic characteristics were investigated.</p><p><strong>Results: </strong>A total of 428 and 412 questionnaires were returned by rural and urban respondents, respectively. Access to a garden was perceived as the most important attribute (average importance 36.0% in the rural sample and 33.4% in the urban sample), followed by consideration of food preferences (15.8%, 17.8%), staff qualification (14.6%, 15.3%), care organization (11.4%, 12.3%), group size (12.2%, 11.1%), and range of activities (8.0%, 10.1%). The attribute relating to religious practice was given the least importance (2.1%, 0%). Preferences vary according to gender, age, religious beliefs, experience as an informal caregiver, and migrant background.</p><p><strong>Conclusion: </strong>Heterogeneous preferences for living arrangements for people with dementia were identified. The expansion of concepts with access to natural environments for persons with dementia might be a viable option for the formal care market in Germany. Further research is needed to meet the challenges of setting up and designing innovative living arrangements for people with dementia. Preferences vary by gender, age, religious beliefs, experience as an informal caregiver, and migrant background.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10781908/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138295698","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":"Acknowledgement to Referees.","authors":"","doi":"10.1007/s41669-023-00456-5","DOIUrl":"https://doi.org/10.1007/s41669-023-00456-5","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138830891","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Retrospective Comparison of Survival Projections for CAR T-Cell Therapies in Large B-Cell Lymphoma.","authors":"Elisabeth F P Peterse, Elisabeth J M Verburg-Baltussen, Alexa Stewart, Fei Fei Liu, Christopher Parker, Maarten Treur, Bill Malcolm, Sven L Klijn","doi":"10.1007/s41669-023-00435-w","DOIUrl":"10.1007/s41669-023-00435-w","url":null,"abstract":"<p><strong>Background: </strong>Durable remission has been observed in patients with relapsed or refractory (R/R) large B-cell lymphoma (LBCL) treated with chimeric antigen receptor (CAR) T-cell therapy. Consequently, hazard functions for overall survival (OS) are often complex, requiring the use of flexible methods for extrapolations.</p><p><strong>Objectives: </strong>We aimed to retrospectively compare the predictive accuracy of different survival extrapolation methods and evaluate the validity of goodness-of-fit (GOF) criteria-based model selection for CAR T-cell therapies in R/R LBCL.</p><p><strong>Methods: </strong>OS data were sourced from JULIET, ZUMA-1, and TRANSCEND NHL 001. Standard parametric, mixture cure, cubic spline, and mixture models were fit to multiple database locks (DBLs), with varying follow-up durations. GOF was assessed using the Akaike information criterion and Bayesian information criterion. Predictive accuracy was calculated as the mean absolute error (MAE) relative to OS observed in the most mature DBL.</p><p><strong>Results: </strong>For all studies, mixture cure and cubic spline models provided the best predictive accuracy for the least mature DBL (MAE 0.013‒0.085 and 0.014‒0.128, respectively). The predictive accuracy of the standard parametric and mixture models showed larger variation (MAE 0.024‒0.162 and 0.013‒0.176, respectively). With increasing data maturity, the predictive accuracy of standard parametric models remained poor. Correlation between GOF criteria and predictive accuracy was low, particularly for the least mature DBL.</p><p><strong>Conclusions: </strong>Our analyses demonstrated that mixture cure and cubic spline models provide the most accurate survival extrapolations of CAR T-cell therapies in LBCL. Furthermore, GOF should not be the only criteria used when selecting the optimal survival model.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10721757/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10495709","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness of Recombinant Zoster Vaccine for the Prevention of Herpes Zoster in Hematopoietic Stem Cell Transplant Recipients and Other Immunocompromised Adults in the United States.","authors":"Ahmed Salem, Elizabeth M La, Desmond Curran, Brandon J Patterson, Justin Carrico, Stéphane Lorenc, Katherine A Hicks, Sara Poston, Christopher F Carpenter","doi":"10.1007/s41669-023-00438-7","DOIUrl":"10.1007/s41669-023-00438-7","url":null,"abstract":"<p><strong>Introduction: </strong>Immunocompromised (IC) adults are at increased risk of developing herpes zoster (HZ) and HZ-related complications due to therapy or underlying disease. This study evaluated the cost effectiveness of recombinant zoster vaccine (RZV) versus no vaccine for the prevention of HZ in hematopoietic stem cell transplant (HSCT) recipients and other IC adults aged ≥ 18 years in the United States (US).</p><p><strong>Methods: </strong>A static Markov model simulated cohorts of IC individuals using a 1-year cycle length and 30-year time horizon to estimate the cost effectiveness of RZV. Inputs were sourced from clinical trial results and publicly available sources/literature. Modeled populations included US adult HSCT recipients (base case), patients with human immunodeficiency virus (HIV), patients with breast cancer, patients with Hodgkin's lymphoma, and renal transplant recipients. The model reported societal costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). Sensitivity and threshold analyses were conducted.</p><p><strong>Results: </strong>In the base case of 19,671 US adult HSCT recipients, RZV resulted in total societal cost savings of US$0.1 million and 109 incremental QALYs versus no vaccine. RZV was a 'dominant strategy' versus no vaccine because vaccination resulted in cost savings with QALY gains. RZV was also cost saving in renal transplant recipients, and cost effective at a willingness-to-pay threshold of US$100,000 per QALY gained in patients with HIV, breast cancer, and Hodgkin's lymphoma, with ICERs of US$33,268, US$67,682, and US$95,972 per QALY gained, respectively, versus no vaccine.</p><p><strong>Conclusions: </strong>Model results show RZV is potentially cost saving for the prevention of HZ in US adult HSCT recipients and US adults with selected immunocompromising conditions, and cost effective for others, supporting the use of RZV to prevent HZ and HZ-related complications in IC adults.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10721768/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71425813","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}