Third-party Payers Research Articles

Multiple pricing for personal assistance services

Third-party payers often reimburse health care providers based on prospectively set prices. Although a key motivation of prospective payment is to contain costs, this paper shows that this aspect crucially depends on the design of the pricing scheme due to the well-known incentives of patient selection (or “dumping”). This paper provides a general theoretical framework where heterogeneous users are served by either private for-profit or public providers, each paid an hourly compensation by a third-party payer. The private, but not the public providers may select patients. It is demonstrated that this realistic feature of the model implies that total costs depends on the number of prices. The features of the model is illustrated using the Swedish system of personal assistance services as a motivating example. Numerical results show that marginal adjustments to the current uniform pricing scheme would lead to substantial savings.

Cost-utility evaluation of a health and social care integration programme for frail older adults in Argentina

PurposeOur objective was to evaluate the cost-utility of a health and social care integration programme for frail older adults in Buenos Aires, Argentina.Design/methodology/approachBased on a study of the programme’s effectiveness, a Markov model was conducted to assess its cost-utility. The active intervention was the health and social care integration programme, and the control was the best standard of care so far. The setting was the patients' home of residence. A third-party payer perspective and a lifelong time horizon were adopted. All transition probabilities, quality-adjusted life years (QALYs) and costs were estimated from the effectiveness study. A discount rate of 3.5% was applied to costs and benefits. Costs are expressed in international dollars (Int$), calculated according to the International Monetary Fund’s purchasing power parity rate. Different sensitivity analyses were performed. The model was built in Excel 365. Construct validity, verification during model construction and internal consistency of the results were assessed.FindingsThe programme had an average cost of Int$18,768.22/QALY, and the control Int$42,609.68/QALY. In the incremental analysis, the programme saved Int$26,436.10 and gained 0.81 QALYs over the control. In the sensitivity analyses, in 99.96% of cases, the programme was less costly and more effective.Practical implicationsThe cost savings can facilitate the scalability.Originality/valueThe health and social care integration programme for frail older adults was more effective and less costly than the best standard of care to date. This study contributes to the scarce evidence on the efficiency of integrated care strategies for frail older persons.

The Utility of the Schnur Sliding Scale in Adolescent Patients.

Although the Schnur Sliding Scale (SSS) was not intended to be used by third-party payors, it has become the national standard for coverage decisions regarding macromastia treatment in patients of all ages. Adolescents were neither included in the cohort that created the SSS nor represented in subsequent validation studies. In this prospective study, health-related quality of life surveys were administered to girls and young women 12 to 21 years of age before and after reduction mammaplasty to treat macromastia. The SSS was used preoperatively to estimate the amount of tissue to be resected. Before and after surgery, participants completed the Short Form-36, version 2 (SF-36); the Rosenberg Self-Esteem Scale (RSES); the Breast-Related Symptoms Questionnaire (BRSQ); and the Eating Attitudes Test-26. Demographic data were compared, and linear regressions were used to evaluate the effect of resection amounts meeting the SSS value on survey scores. Resection amounts fell below the SSS for 39 patients and above the SSS for 255 patients. The 2 groups had no differences in mean age or body mass index. Both groups had significant postoperative survey score improvements on the RSES, BRSQ, and 7 of 8 SF-36 domains (all P < 0.05). The groups had comparable postoperative survey scores on the RSES, BRSQ, and 8 of 8 SF-36 domains (all P > 0.05). Adolescents undergoing reduction mammaplasty above and below the SSS experienced comparable physical and psychosocial benefits. These findings underscore the need for third-party payors to broaden coverage for adolescent reduction mammaplasty, as the common coverage cutoff has no effect on overall postoperative benefit. Risk, II.

Budget Impact Analysis of Olaparib for the Management of Patients with Homologous Recombination Repair (HRR)-Mutated Castration-Resistant Metastatic Prostate Cancer in Argentina.

The aim of this study was to perform a budget impact analysis (BIA) of introducing olaparib treatment for adult patients with metastatic castration-resistant prostate cancer in Argentina. A BIA model was used to estimate the cost difference between the current scenario (without olaparib) and the new scenario (incorporation of olaparib) for a third-party payer over a 5-year time horizon. The budgetary impact is estimated at the national health system level and by healthcare sectors in Argentina. Input parameters were obtained from the literature and validated by local expert opinion. Direct medical costs were obtained from both the Institute for Clinical Effectiveness and Health Policy (IECS) unit cost database and public data in Argentina. The microcosting estimation was used for key variables of the analysis. All costs are reported in US dollars (US$) as for October 2022 (1 US$ = 152.59 Argentine pesos). One-way sensitivity analyses and scenario analyses were conducted to evaluate the model robustness. The incorporation of olaparib, with a wholesale price per pack of US$3176, was associated with a weighted average of the budget impact per member per month (PMPM) of US$0.0191 for the national health system, being slightly higher than the estimated budgeted high impact threshold (US$0.0153). The PMPM budget impact for a 5-year average ranged between US$0.007 (public sector) and US$0.033 (private sector). The duration of treatment with olaparib was the most influential parameter in the budget impact results. The introduction of olaparib for the treatment of metastatic castration-resistant prostate cancer has a high budget impact for Argentina's health system. These findings are informative to support policy decisions aimed to expand the current treatment landscape for prostate cancer.

Expedited versus standard postpartum discharge in patients with hypertensive disorders of pregnancy and its effect on the postpartum course

BackgroundHospital stay after an uncomplicated delivery is typically 2 days for vaginal birth and 3 days for cesarean birth. Health maintenance organizations and third-party payers have encouraged shorter maternity stays. The safety of earlier discharge is unclear particularly when it comes to patients diagnosed with hypertensive disorders of pregnancy (HDP). ObjectiveTo examine whether expedited discharge amongst patients with HDP will have a negative effect on postpartum readmission rate and blood pressure related complications. Study designThis was a single academic center retrospective cohort study of patients with HDP (gestational hypertension, preeclampsia, or chronic hypertension) for 2 epochs: 2015–2018, prior to implementation of an expedited discharge policy, and 2019–2020 after hospital wide implementation of expedited postpartum discharge. The expedited discharge policy entailed patients being discharged home as soon as day 1 after a vaginal delivery and day 2 after a cesarean delivery. The primary outcome was unplanned health care utilization postpartum, defined as emergency department (ED) visits, unscheduled clinic visits, and hospital readmission. Secondary outcomes were planned postpartum visits attendance, antihypertensive medication initiation after discharge, and blood pressure control throughout the first year. Bivariable and multivariable logistic regression analyses were run to evaluate the association between expedited discharge and primary and secondary outcomes. ResultsA total of 1,441 patients were included in the analysis. There were no statistically significant differences in the rate of unplanned health care utilization (11.3% in the standard postpartum discharge group vs. 13.8% in the expedited discharge group, P=.17). Systolic and diastolic blood pressures did not differ between the groups at 1–2 weeks, six weeks, and one year postpartum. Patients in the expedited discharge group were more likely to attend the 1–2-week postpartum blood pressure check (58.7% vs. 51.7%, P=.02, adjusted OR 1.33, 95% CI 1.08–1.77). Other secondary outcomes did not differ between the two cohort groups. ConclusionIn this single academic center study, expedited discharge after delivery in patients with HDP was not associated with a higher rate of unplanned healthcare utilization postpartum.

Occupational Therapy Practice Guidelines for Adults Living With and Beyond Cancer

Abstract Importance: Occupational therapy practitioners play an important role in addressing the impairments, occupational performance, and participation needs of adults living with and beyond cancer. Objective: These Practice Guidelines aim to assist occupational therapy practitioners, educators, and researchers in applying evidence and expert opinion to occupational therapy practice across the cancer continuum of care, including identifying gaps in care. Key focus areas include psychosocial function, fatigue, cognition, pain, sleep, and physical activity. These guidelines can serve as a reference for policymakers, health care professionals, health care managers, regulators, third-party payers, and managed care organizations to manage, reimburse, and set policy. Method: We examined, synthesized, and integrated the results of systematic reviews, supporting literature, and expert opinion to develop and provide clinical recommendations for practice in the care of adults living with and beyond cancer. Results: Sixteen systematic reviews with moderate to strong strength of evidence published between 2019 and 2022 served as the foundation for these Practice Guidelines. Conclusions and Recommendations: To support symptom management for adults living with and beyond cancer, occupational therapy practitioners can consider using psychosocial interventions for anxiety, depression, posttraumatic growth, and overall psychological distress. Practitioners can consider using physical activity interventions for cancer-related fatigue, cognitive performance, and sleep. Practitioners could also consider using interventions for cognition, pain management, and self-management for the improvement of cancer-related symptoms. Plain-Language Summary: These Practice Guidelines provide strong and moderate levels of evidence for occupational therapy practitioners to support adults living with and beyond cancer. They provide specific guidance for addressing pain, cancer-related fatigue, cancer-related cognitive impairments, psychosocial well-being, sleep, and physical activity. The guidelines also include case examples and an infographic to support occupational therapy practitioners in integrating evidence to support interventions in cancer care, and they highlight gaps and expert opinions in practice for continued clinically relevant research.

Sodium phenylbutyrate-taurursodiol access, adherence and adverse event in patients with amyotrophic lateral sclerosis: Experience at one center in the United States.

Sodium phenylbutyrate-taurursodiol (PB-TURSO) was recently approved for treating amyotrophic lateral sclerosis (ALS). Third-party payors' coverage policies are evolving, and adverse events are just being fully assessed. The goals of this study were to evaluate patients' experiences in obtaining and continuing PB-TURSO and assess adverse events and medication adherence. Medical records of 109 ALS patients who were considered PB-TURSO candidates by the treating physician at a tertiary ALS clinic from October 2022 to May 2023 were reviewed. Data was recorded for demographics, clinical, and insurance information. A survey was e-mailed to patients asking about out-of-pocket expenses for PB-TURSO, financial assistance, medication start and (if applicable) stop dates, and reasons for discontinuation. Insurance information was available for 91 patients [57 males (62%); mean age 64.8 years (range 25.7-88)]. Of 79 who applied for insurance approval, 71 (90%) were approved; however, 19 required 1-3 appeals. Among 73 patients with available data about medication status, 54 started PB-TURSO and 19 did not, most commonly due to personal choice or out-of-pocket expenses. About 44% of patients (24/54) stopped taking PB-TURSO, primarily due to adverse events. Monthly out-of-pocket expenses varied from $0 to $3500 and 36 patients qualified for financial assistance. Administrative and nursing staff devoted 7.2 hours/week to the insurance authorization process. Most patients received insurance approval for PB-TURSO, but one-fourth required appeals. Some out-of-pocket costs were very high. Investment of staff time was substantial. These findings have implications for insurance coverage of, and adherence to, future ALS treatments.

Budget Impact Analysis of Olaparib in Combination with Bevacizumab for Maintenance Therapy for Ovarian Cancer in Argentina.

To perform a budget impact analysis (BIA) of introducing olaparib as maintenance therapy in women who have BRCA mutations (BRCAm) with platinum-sensitive recurrent ovarian cancer (PSROC) in combination with bevacizumab in Argentina. A BIA model was used to analyse over a 5-year time horizon the change in the health system's budget following the adoption of olaparib as maintenance therapy in BRCAm patients with PSROC. The BIA for each year was estimated by comparing the cost difference between the current scenario (treatment with bevacizumab) and the new scenario (the addition of olaparib) for a third-party payer. The BIA is estimated at the national health system level, and by healthcare sectors in Argentina (public sector, social security and private sector). International and national epidemiological data were used to determine the target patient population. Clinical efficacy, safety outcomes and duration of treatments were obtained from the pivotal clinical study report. Relevant direct medical costs were obtained from public data in Argentina and expert consultation. All the costs are reported in US dollars as of October 2022 ($1 = 152.59 Argentine pesos). A scenario analysis assessed the full coverage of the homologous recombination deficiency (HRD) test in Argentina. In addition, one-way sensitivity analysis was conducted to evaluate the model robustness. For a third-party payer with a cohort of 1,000,000 women covered, the estimated target population was 2 individuals in year 1 and 6 individuals in year 5. The incorporation of olaparib, with a wholesale price per pack of $3176.32, was associated with a weighted average of the budget impact per member per month (PMPM) of $0.062 for the national health system, being above the estimated health system budget impact threshold ($0.0153). By healthcare sector, the results of budget impact PMPM for year 5 ranged between $0.08 (public sector) and $0.114 (private sector). For all perspectives, the variables that most influenced the budget impact was the incidence of ovarian cancer, the drug acquisition cost and the treatment duration. The introduction of olaparib for the treatment of BRCAm women with PSROC has a high budget impact for all three health systems in Argentina.

Comparison of standard charges for complex proton therapy among Medicare Prospective Payment System (PPS) –exempt and –non-exempt cancer centers.

e23039 Background: Proton therapy is one of the most expensive treatment modalities offered to cancer patients, and only 44 proton therapy centers are operational in the U.S. Of those, 5 are Medicare Prospective Payment System (PPS)-exempt and are reimbursed based on their reported costs rather than predetermined prices. These PPS-exempt cancer centers set their adjusted payment rates to reflect the expense required for providing specialized care. We hypothesized that PPS-exempt centers would have higher rates for complex proton therapy delivery compared to non-exempt centers. Methods: We obtained online standard charge files for 44 proton therapy centers in the U.S. We used the CPT code 77525, the per fraction technical charge billed for complex proton treatment delivery, to extract the gross price (base charge for service), de-identified minimum and maximum negotiated price (lowest/highest charge negotiated with third-party payers), and cash price (charge for cash payment of service) per fraction of complex proton therapy. Prices were compared between PPS-exempt and non-exempt proton therapy centers using Wilcoxon/Kruskal Wallis test with a p-value &lt; 0.05 considered significant. Results: Of the 44 proton therapy centers, 39 (88.6%) had publicly available standard charge files, and 5 (11.4%) are private practice not required to provide a standard charge file. Five of the 39 (12.8%) centers are PPS-exempt, and the remaining 34 (87.2%) are PPS non-exempt. Four (80%) PPS-exempt centers had their gross price, minimum and maximum negotiated price, and cash price available in their standard charge file for CPT 77525, while 23 (67.6%) PPS non-exempt centers had their gross price and cash price listed and 19 (55.9%) had the minimum and maximum price available. The median gross charge per fraction for complex proton treatment delivery was $8,035.25 (IQR $7,022.00-$9,427.50) at PPS-exempt versus $6,434.00 (IQR $5,213.00-$12,350.00) at PPS non-exempt centers. The median minimum price was $656.47 (IQR $178.04-$1,353.23) at PPS-exempt versus $1,294.70 (IQR $734.20-$2,267.46) at PPS non-exempt centers. The median maximum price was $8,120.10 (IQR $5,576.40-$11,529.86) at PPS-exempt versus $5,814.00 (IQR $4,830.00-$9,524.70) at PPS non-exempt centers. The median cash price was $5,360.65 (IQR $2,979.51-$6,897.08) at PPS-exempt versus $4,170.40 (IQR $2,828.00-$5,640.00) at PPS non-exempt centers. Conclusions: In summary, 80% of PPS-exempt and 67.6% of PPS non-exempt centers had publicly available standard charge files. Median charges for PPS-exempt centers are higher for gross charge, maximum price, and cash price, however did not meet statistical significance. [Table: see text]

An economic analysis of low-energy contact x-ray brachytherapy for treatment of rectal cancer.

e15622 Background: Health Technology Wales sought to evaluate the clinical and cost-effectiveness of contact x-ray brachytherapy (CXB) in conjunction with external-beam radiotherapy (EBRT), compared to EBRT alone for early-stage (T2–3b, N0–1, M0) rectal cancer in a surgically fit population. Methods: An economic analysis was performed using a decision analytical, state transition model. Analysis was performed from a third-party payer perspective over a life-time horizon. The model was populated using data from the Organ Preservation in Early Rectal Adenocarcinoma (OPERA) trial. Results: . CXB was estimated to provide 0.2 quality-adjusted life years (QALYs) per patient at an additional cost of $1118 per person. CXB was cost-effective compared with external beam boost at a cost of $5623 per QALY gained. These findings did not change in deterministic sensitivity analysis and CXB was cost-effective in over 90% of probabilistic sensitivity analyses. Conclusions: CXB is likely to be cost-effective with a high degree of certainty in this patient population.

Screening for Bacterial Vaginosis Prior to Delivery: A Cost-Effectiveness Study.

The objective of this study was to compare the cost and effectiveness of three strategies for screening and/or treating bacterial vaginosis (BV) during pregnancy prior to delivery: (1) the current standard of care was neither test nor treat for BV (Treat None); (2) test all patients for BV at 36 weeks' gestation; treat if positive (Test Treat); and (3) treat all patients undergoing cesarean delivery with intravenous metronidazole at time of surgery (Treat All Cesarean). Effectiveness was defined as avoidance of postpartum surgical site infection (SSI). A decision analytic cost-effectiveness model was designed from a third-party payer perspective using clinical and cost estimates obtained from the literature, American College of Surgeons National Surgical Quality Improvement Program participant use file (2005-2019), 2019 National Vital Statistics, Medicare costs, and wholesale drug costs. Cost estimates were inflated to 2020 U.S. dollars. For this study, effectiveness was defined as avoidance of postpartum SSIs. The base case analysis that is the current standard of care of not routinely testing and treating patients for BV (Treat None) was the most expensive and least effective strategy, with a mean cost of $59.16 and infection rate of 3.71%. Empirically treating all patients for BV without testing (Treat All Cesarean) was the most effective and the least expensive strategy, with a mean cost of $53.50 and an infection rate of 2.75%. Testing all patients for BV and treating those positive for BV (Test Treat) was also relatively inexpensive and effective, with an infection rate of 2.94% and mean cost of $57.05. Compared with Treat None, we would expect the Treat All Cesarean strategy to reduce the infection rate by 26%. These findings suggest that treating pregnant patients with intravenous metronidazole at time of cesarean delivery could be an effective and cost-saving strategy. Testing and treating for BV could also be considered a reasonable strategy, as it has the added benefit of preserving antibiotic stewardship. In no analysis was the standard of care strategy of neither testing nor treating for BV before delivery the preferred strategy. · BV colonization may increase surgical site infection risk after cesarean section.. · Treatment of BV before or during delivery may be cost-saving strategies as treatment could prevent costs associated with infection.. · Further study is needed to best balance the risk of surgical site infection with antibiotic stewardship..

Epidemiology and comprehensive economic impact of atrial fibrillation and associated stroke in Slovakia.

Atrial fibrillation (AF) is the most common sustained cardiac arrhythmia. It is also a major risk factor for ischemic stroke. The main objective of our study was to identify direct and indirect costs of AF and AF-related stroke in Slovakia. We conducted a retrospective population-based study of AF and stroke related costs both from the third-party healthcare payers and societal perspective. The prevalence and incidence of AF and stroke were determined from central government run healthcare database. Further we estimated both indirect and direct costs of AF and stroke. All costs and healthcare resources were assessed from 2015 through 2019 and were expressed in the respective year. Over the 5-year study period, the prevalence of AF increased by 26% to a total of 149,198 AF cases in 2019, with an estimated total annual economic burden of €66,242,359. Direct medical costs accounted for 94% of the total cost of AF. The total cost of treating patients with stroke in 2019 was estimated at €89,505,669. As a result, the medical costs of stroke that develops as a complication of AF have been estimated to be €25,734,080 in 2019. Our study shows a substantial economic burden of AF and AF-related stroke in Slovakia. In view of the above, both screening for asymptomatic AF in high-risk populations and effective early management of AF with a focused on thromboprophylaxis rhythm control should be implemented.

Bleeds and resource use in hemophilia B: retrospective observational study.

To describe people with hemophilia B (PWHB) in the US who experience bleeds despite factor replacement therapy and to quantify the associated burden from the third-party payer perspective. Observational study of adult male PWHB treated with factor IX replacement therapy identified from the PharMetrics Plus claims data from 2010 to 2019. Patients with medically recorded bleeds (MRBs) were identified using diagnostic codes. Rates and rate ratios of inpatient admissions, emergency department (ED) visits, and outpatient visits among PWHB with and without MRBs were estimated. The presence of comorbidities was identified using diagnostic codes, and the analysis was stratified by age group. There were 345 PWHB with MRBs and 252without MRBs. More than half of PWHB with MRBs (56.8%) had 1 or more comorbidity vs 39.3% of PWHB without MRBs. The prevalence of anxiety and depression was high in PWHB, regardless of bleed status and age group, whereas the prevalence of other comorbidities increased with age group. The rate of all-cause inpatient admissions for PWHB with MRBs was 14.8 per 100 person-years (95%CI, 12.8-17.1), 2.5 times higher than for PWHB without MRBs. The rate of all-cause ED visits for PWHB with MRBs was 67.6per 100 person-years (95% CI, 63.2-72.3), 2.7 times higher than for those without MRBs. This study reports significant resource use and clinical burden among PWHB who seek medical care. PWHB with MRBs had considerable all-cause resource use compared with PWHB without MRBs. The prevalence of mental illness was consistently high across all age groups.

Costs and Projected Effect of a Federally Qualified Health Center-Based Mailed Colorectal Cancer Screening Program in Texas.

Mailed stool testing for colorectal cancer (CRC) may improve screening uptake and reduce the incidence and mortality of CRC, especially among patients at federally qualified health centers (FQHCs). To expand screening programs it is important to identify cost-effective approaches. We developed a decision-analytic model to estimate the cost, effects on screening and patient outcomes (CRCs detected, CRCs prevented, CRC deaths prevented), and cost-effectiveness of implementing a state-wide mailed stool testing program over 5 years among unscreened, age-eligible (aged 50-75 y) patients at FQHCs in Texas. We compared various outreach strategies and organizational structures (centralized, regional, or a hybrid). We used data from our existing regional mailed stool testing program and recent systematic reviews to set parameters for the model. Costs included start-up and ongoing activities and were estimated in 2022 US dollars from the perspective of a hypothetical third-party payer. Cost-effectiveness was assessed by using both incremental and average cost-effectiveness ratios. Using either a statewide centralized or hybrid organizational configuration to mail stool tests to newly eligible FQHC patients and patients who have responded at least once since program inception is likely to result in the best use of resources over 5 years, enabling more than 110,000 additional screens, detecting an incremental 181 to 194 CRCs, preventing 91 to 98 CRCs, and averting 46 to 50 CRC deaths, at a cost of $10 million to $11 million compared with no program. A statewide mailed stool testing program for FQHC patients can be implemented at reasonable cost with considerable effects on CRC screening outcomes, especially when its structure maximizes program efficiency while maintaining effectiveness.

Cost-effectiveness of Rapid On-site Evaluation During Navigational Bronchoscopy

BackgroundPeripheral pulmonary lesions (PPL) are common, with more than 1.6 million PPLs incidentally identified in the United States annually. Navigational bronchoscopy (NB) is a cornerstone of the diagnostic evaluation of PPLs. Intraprocedural rapid on-site evaluation (ROSE) of biopsies obtained during NB is widely used, but the data for its utility are contradictory. The cost-effectiveness of ROSE has yet to be established and as such, ROSE currently has variable implementation between institutions and is not adequately reimbursed by payors. Research QuestionIs ROSE cost-effective during NB for PPL from a third-party payor perspective? Study Design and MethodsA cost-effectiveness model was constructed comparing NB for PPL with versus without ROSE from a third-party payor perspective. The base case is a 60-year-old operative candidate with a 2 cm pulmonary nodule without radiographic mediastinal or hilar lymphadenopathy referred for NB. Cost per quality adjusted life-year gained was the primary outcome. Inputs for the model were estimated from published literature. One-way deterministic sensitivity analyses were conducted on all parameters. Probabilistic sensitivity analysis was performed. ResultsThe use of ROSE resulted in a gain of 0.01 quality-adjusted life years and cost an additional $466. At a willingness to pay (WTP) threshold of $100,000/life-year, ROSE was cost-effective with an incremental cost effectiveness ratio (ICER) of $44,465.88. Sensitivity analyses on the sensitivity of NB with and without ROSE show that ROSE must increase the diagnostic sensitivity of the procedure by 3% to become cost-effective. InterpretationThe use of ROSE during navigational bronchoscopy for PPL is cost-effective for third-party payors at a WTP threshold of $100,000/life year and should be reimbursed at a higher rate. The cost-effectiveness of ROSE hinges on the additional diagnostic sensitivity gained by using ROSE.

Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States.

Gene therapies for sickle cell disease (SCD) may offer meaningful benefits for patients and society. This study evaluated the cost-effectiveness of lovotibeglogene autotemcel (lovo-cel), a one-time gene therapy administered via autologous hematopoietic stem cell transplantation, compared with common care for patients in the United States (US) with SCD aged ≥12years with ≥4 vaso-occlusive events (VOEs) in the past 24months. We developed a patient-level simulation model accounting for lovo-cel and SCD-related events, complications, and mortality over a lifetime time horizon. The pivotal phase1/2 HGB-206 clinical trial (NCT02140554) served as the basis for lovo-cel efficacy and safety. Cost, quality-of-life, and other clinical data were sourced from HGB-206 data and the literature. Analyses were conducted from US societal and third-party payer perspectives. Uncertainty was assessed through probabilistic sensitivity analysis and extensive scenario analyses. Patients treated with lovo-cel were predicted to survive 23.84years longer on average (standard deviation [SD], 12.80) versus common care (life expectancy, 62.24 versus 38.40years), with associated discounted patient quality-adjusted life-year (QALY) gains of 10.20 (SD, 4.10) and direct costs avoided of $1,329,201 (SD, $1,346,446) per patient. Predicted societal benefits included discounted caregiver QALY losses avoided of 1.19 (SD, 1.38) and indirect costs avoided of $540,416 (SD, $262,353) per patient. Including lovo-cel costs ($3,282,009 [SD, $29,690] per patient) resulted in incremental cost-effectiveness ratios of $191,519 and $124,051 per QALY gained from third-party payer and societal perspectives, respectively. In scenario analyses, the predicted cost-effectiveness of lovo-cel also was sensitive to baseline age and VOE frequency and to the proportion of patients achieving and maintaining complete resolution of VOEs. Our analysis of lovo-cel gene therapy compared with common care for patients in the US with SCD with recurrent VOEs estimated meaningful improvements in survival, quality of life, and other clinical outcomes accompanied by increased overall costs for the health care system and for broader society. The predicted economic value of lovo-cel gene therapy was influenced by uncertainty in long-term clinical effects and by positive spillover effects on patient productivity and caregiver burden.

Genotype-driven asthma prescribing of inhaled corticosteroids and long-acting β2-agonist: A cost-effectiveness analysis.

Predicting response to inhaled corticosteroids (ICSs) + long-acting β2-agonist (LABA) by previously detecting the presence of Arg16Gly ADRB2 genotype is a strategy that could reduce and optimize the management of asthmatic patients. There is a need for economic evaluations to facilitate the implementation of such tests. This research aims to evaluate the cost-effectiveness of Arg16Gly ADRB2 screening in children with asthma in Colombia. From the perspective of a third-party payer, we conducted a cost-effectiveness analysis to determine the cost and quality-adjusted life-years (QALYs) of genotype-driven asthma prescribing based on the Arg16Gly ADRB2 genotype versus current treatment based on no genetic testing. Using four state-transition models, we estimate cost and QALYs employing micro-simulation modeling with a time horizon of 10 years and a cycle length of 1 week. Cost-effectiveness was assessed at a willingness-to-pay (WTP) value of US$5180. The mean incremental cost of strategy genetic testing versus no genetic testing is US$ -6809. The mean incremental benefit of strategy genetic testing is 16 QALYs. The incremental net monetary benefit of strategic genetic testing versus no genetic testing is US$ 88,893. Genetic testing is the strategy with the highest expected net benefit. The outcomes derived from our primary analysis remained robust when subjected to variations in all underlying assumptions and parameter values. Genetic testing of Arg16Gly ADRB2 is a cost-effective strategy to address asthma management in asthmatic children requiring ICS+LABA. This result should encourage the generation of more evidence and the incorporation of such evidence into clinical practice guidelines for pediatric asthma.

Cost-effectiveness analysis of durvalumab, tremelimumab, and etoposide-platinum in first-line treatment of extensive-stage small cell lung cancer.

Durvalumab plus etoposide-platinum (DEP) showed sustained overall survival improvements in patients with extensive-stage small-cell lung cancer (ES-SCLC) compared to etoposide-platinum (EP), but adding tremelimumab to DEP (DTEP) did not significantly improve outcomes. A third-party payer perspective is taken here to evaluate the cost-effectiveness of DTEP, DEP, and EP for ES-SCLC. The cost-effectiveness was evaluated by partitioning survival models into 3 mutually exclusive health states. In this model, clinical characteristics and outcomes were obtained from the CASPIAN. Model robustness was evaluated through 1-way deterministic and probabilistic sensitivity analyses. Outcome measurements included costs, quality-adjusted life-years (QALYs), incremental cost-effectiveness ratio, life-years, incremental net health benefit, and incremental net monetary benefit. The analysis was conducted with a 10-year lifetime horizon in a United States setting. Compared with EP, DEP, and DTEP were associated with an increment of 0.480 and 0.313 life-years, and an increment of 0.247 and 0.165 QALYs, as well as a $139,788 and $170,331 increase in cost per patient. The corresponding ICERs were $565,807/QALY and $1033,456/QALY, respectively. The incremental net health benefit and incremental net monetary benefit of DEP or DTEP were -0.685 QALYs and -$102,729, or -0.971 QALYs and -$145,608 at a willingness to pay threshold of $150,000/QALY, respectively. Compared with DTEP, DEP was dominated. DTEP and DEP were 100% unlikely to be cost-effective if the willingness to pay threshold was $150,000/QALY. DEP was cost-effective compared to EP when durvalumab was priced below $0.994/mg. Compared with EP, DEP, and DTEP were unlikely to be considered cost-effective across all subgroups. DEP and DTEP were not cost-effective options in the first-line treatment for ES-SCLC compared with EP, from the third-party payer perspective in the United States. Compared with DTEP, DEP was dominated.

The Promise and Challenges of Practice-Oriented Research: A Commentary on the Special Issue.

At the centre of POR is the concept of collaboration between patients, therapists, agencies, and third-party payers. For this commentary, I review the articles of the special issue with attention to both the opportunities and challenges offered by practice-oriented research (POR).I also reviewed some previous research on practice-research networks and how that research might inform POR. The use of routine outcome monitoring (ROM), artificial intelligence (AI), and program evaluation (PE) models show promise for advancing POR. However, each comes with its challenges. The use of ROM to improve patient outcomes has research support. However, researchers have identified problems with implementing ROM because of low uptake by clinicians and because clinicians may see ROM as a potential intrusion. AI shows promise to improve patient outcomes by potentially providing therapists with immediate and nuanced data to inform interventions and interpersonal stances specific to each patient. However, the scaling up of AI runs the risk of dehumanizing psychological interventions. PE may provide a context for allowing therapists to engage in POR to address real-world processes and outcomes of mental health interventions. However, like ROM PE faces the challenge of trust among clinicians and patients who may be reluctant to participate. Despite these challenges, and because of efforts to nurture and maintain respectful collaborations, articles in this special issue highlight how POR can play a pivotal role in bridging the gap between theory and practice.

Nivolumab plus ipilimumab versus the EXTREME regimen in recurrent/metastatic squamous cell carcinoma of the head and neck: a cost-effectiveness analysis

In the CheckMate 651 study, nivolumab plus ipilimumab versus EXTREME (cisplatin/carboplatin + cetuximab + fluorouracil) regimen was compared for effectiveness. It is not known whether these immunotherapy agents are cost-effective for recurrent or metastatic squamous cell carcinomas of the head and neck (R/M SCCHN). The purpose of this study was to compare the cost-effectiveness of nivolumab plus ipilimumab with EXTREME in the first-line setting from the standpoint of third-party payers in the United States. The projecting of costs and outcomes over 15 years was done using a three-state partitioned survival model discounted by 3% per year. Long-term extrapolation of CheckMate 651 was used to model progression-free survival and overall survival (OS). The incremental net health benefit (INHB), incremental net monetary benefit (INMB), quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER) were calculated. The uncertainty and stability of the model were accounted for via one-way and probabilistic sensitivity analyses. As compared with nivolumab plus ipilimumab, EXTREME was associated with an increase of 0.154 life-years and 0.076 QALYs, as well as a cost increase of $572 per patient. The corresponding ICERs were $7545/QALY along with the values of INMB and INHB were $113,267 and 0.076 QALYs, respectively, at a willingness to pay (WTP) threshold of $150,000/QALY. The probability of nivolumab plus ipilimumab being cost-effective was > 99% in patients with combined positive score (CPS) ≥ 1, CPS 1–19, or CPS ≥ 20. Moreover, hazard ratio for OS and body weight were the most sensitive parameters for the model. According to sensitivity analyses, these results were generally robust. In overall populations with R/M SCCHN, the EXTREME regimen is cost-effective compared with nivolumab plus ipilimumab. Given a WTP threshold of $150,000 per QALY, the probability of the EXTREME regiment being cost-effective compared with nivolumab and ipilimumab, was 64%. Importantly, there was heterogeneity in the cost-effectiveness probabilities, based on primary sites and expression levels of PD-L1. Therefore, tailored treatment based on individual patient and clinical characteristics, remains important, and may impact the cost-effectiveness of the regimens under study.