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Recent health care legislation in the United States has turned considerable focus to comparative effectiveness research (CER) domestically, though it has been a topic of discussion internationally for many years. Without a fully comprehensive and consistent definition of CER developed, much uncertainty and confusion surrounds its utilization. In addition, contention exists regarding the incorporation of cost and economic considerations as a component of CER. This discussion includes various suggested definitions of CER, methodological considerations, legislation and utilization, and the role of cost-effectiveness evaluations in CER. [ABSTRACT FROM AUTHOR]

Describes the development of the School-Based Rural Case Management: A Model to Prevent and Reduce Risk in a primary health care academic nursing center practice in West Virginia. Provision of guidance for the identification of health risks and health services; Assistance to the matching of appropriate services to the client by a school-based case management system.

Summary Findings from an exploratory factor analysis on the 21 item 'attitudes towards health care teams' (Heinemann et al., 1999) are reported. Using data collected as part of an innovative educational program on geriatric team training program in the United States we report an exploratory factor analyses for 913 student trainees. The geriatric interdisciplinary team training (GITT) program funded by a United States philanthropic foundation, The John A. Hartford Foundation of New York City, requires medicine, nursing, and social work students to learn about geriatric teams. A 3-factor solution with all 21-items is obtained. These factors are labeled to reflect normative team constructs: team value, team efficiency and shared leadership. Though conceptually these factors map onto those identified by Heinemann et al. (1999), some important philosophical and methodological differences are noted. Implications for interdisciplinary education and for the construct validity of this scale are discussed. [ABSTRACT FROM AUTHOR]

In spite of recent attempts to institute change, significant reform of the health care system in the U.S. continues to be one of the most difficult issues. Hospitals and other health care systems are increasingly challenged to control costs and tighten budgets. As a result of these economic pressures, hospitals and nursing departments have had to make difficult choices about the size and about the professional mix of the nursing staff. Moreover, because hospitalized patients are older, more acurately ill and have shorter lengths of stay, nurses are expected to provide more care yet prepare patients for discharge in less time.

Objectives: To conduct a cost analysis of adjunctive hydrocortisone therapy for severe septic shock from the perspective of a third-party payer in the United States.Design: Estimates of outcomes were aggregate data from the Adjunctive Corticosteroid Treatment in Critically Ill Patients with Septic Shock and Activated Protein C and Corticosteroids for Human Septic Shock trials. In these trials, the outcomes of interests were ICU length of stay, vasopressor-free days, ventilation-free days, and the proportion of patients receiving blood transfusion. Each outcome was monetized into a set of mutually exclusive components and was aggregated to estimate the cost-per-patient based on each trial. Cost inputs for each outcome were obtained from literature and adjusted based on the medical care consumer price index. To estimate the budget impact using adjunctive hydrocortisone therapy, per-patient avoided cost was multiplied by expected septic shock annual incidence. Deterministic one-way sensitivity analysis evaluated the robustness of the findings, and Monte Carlo simulation estimated 95% CI of the findings.Setting: A total of 103 medical-surgical ICU (69 for Adjunctive Corticosteroid Treatment in Critically Ill Patients with Septic Shock and 34 for Activated Protein C and Corticosteroids for Human Septic Shock).Patients: Adults greater than or equal to 18 years old with septic shock.Interventions: Adjunctive hydrocortisone therapy (hydrocortisone at a dose of 200 mg/d for 7 d for Adjunctive Corticosteroid Treatment in Critically Ill Patients with Septic Shock and hydrocortisone at a 50 mg IV bolus every 6 hr and fludrocortisone as a 50 μg tablet once daily).Measurements and Main Results: Per Adjunctive Corticosteroid Treatment in Critically Ill Patients with Septic Shock, adjunctive hydrocortisone therapy showed a 90-day monetized benefit of $8,111 (95% CI, $3,914-$12,307) per patient, driven by improvements in ICU-free days, vasopressor-free days, ventilation-free days, and blood transfusion proportion. The total estimated annual impact of adjunctive hydrocortisone therapy, in 2019 dollars, was $750 million. Per Activated Protein C and Corticosteroids for Human Septic Shock, adjunctive hydrocortisone therapy showed a 90-day monetized benefit of $25,539 per patient (95% CI, $22,853-$28,224), driven by improvements in ICU free-days, vasopressor-free days, and ventilation-free days. The total estimated annual impact of adjunctive hydrocortisone therapy, in 2019 dollars, was $2.3 billion. The deterministic one-way sensitivity analysis showed the cost of ICU stays to be the most influential factor in both analyses. The sensitivity analysis using the reported median showed a greater monetized benefit of $10,658 (Adjunctive Corticosteroid Treatment in Critically Ill Patients with Septic Shock) and $30,911 (Activated Protein C and Corticosteroids for Human Septic Shock) per patient.Conclusions: Using adjunctive hydrocortisone therapy yields a significant monetized benefit based on inputs from the Adjunctive Corticosteroid Treatment in Critically Ill Patients with Septic Shock and Activated Protein C and Corticosteroids for Human Septic Shock trials. [ABSTRACT FROM AUTHOR]

Purpose. A systematic review examining the economic effects of pharmacist-provided direct patient care on health outcomes in the United States was conducted. Methods. A comprehensive literature search was conducted using 13 academic and medical databases. Studies were included in the analysis if they described pharmacist-provided direct patient care, used comparison groups, evaluated economic outcomes, and were conducted in the United States. Outcome results were categorized as (1) favorable, indicating significant improvement as a result of pharmacists' interventions or services, (2) not favorable, indicating significant improvement as a result of nonpharmacist care, (3) mixed, having favorable results on one measure of a study variable but not favorable results or no effect on another, (4) having no effect, indicating no significant difference between pharmacists' interventions or services and the comparison, or (5) unclear, indicating the outcome could not be determined based on presented data. Results. Of the 56,573 citations considered, a total of 126 studies met the criteria for inclusion in this systematic review. Results favoring pharmacist-provided care were found in 20 studies (15.9%), mixed results were seen in 53 studies (42.1%), no effect was found in 6 studies (4.8%), and unclear results were found in 47 studies (37.3%). Conclusion. A majority of studies examining the economic effects of pharmacist-provided direct patient care in the United States were limited by their partial cost analyses, study design, and other analysis considerations. A majority of the 20 studies that found positive economic benefits examined pharmacists' interventions involving technical methods or multimodal approaches. [ABSTRACT FROM AUTHOR]

Introduction: Postpartum Medicaid eligibility extensions may increase access to healthcare for low-income women. However, its implications for healthcare utilization are unknown., Methods: We analyzed the linked-infant birth certificate and claims data of women whose childbirths were paid for by Medicaid between 2016 and 2019 in Arizona, United States. We evaluated associations between postpartum care visits and Medicaid insurance type and assessed effect modification by the delivery route and type of residence., Results: Women with pregnancy-related Medicaid insurance were less likely to attend postpartum visits, with an adjusted odds ratio (aOR) of 0.70 and a 95% confidence interval (CI) of 0.66 to 0.74 than those with continuous Medicaid insurance. Younger age, rural residence [aOR 0.83, CI 0.78, 0.88], vaginal delivery route [aOR 0.11, CI 0.10, 0.12], and the absence of complications during/after childbirth [aOR 0.58, CI 0.49, 0.70] were associated with the absence of postpartum care visit. Low-income women who lost their pregnancy-related Medicaid coverage after 60 days in Arizona experienced lower rates of postpartum care utilization., Discussion: Interventions to improve postpartum utilization should be considered beyond extending postpartum Medicaid coverage for low-income women., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. The author(s) declared that they were an editorial board member of Frontiers, at the time of submission. This had no impact on the peer review process and the final decision., (Copyright © 2024 Okechukwu, Abraham, Okechukwu, Magrath, Marrero, Farland and Alaofe.)

Aim: To estimate the budget impact of adding a toripalimab regimen as a treatment option to the existing pembrolizumab regimen, both including gemcitabine and cisplatin, in untreated recurrent/metastatic nasopharyngeal carcinoma (R/M NPC) using the published wholesale acquisition cost (WAC) and average sales price (ASP)., Methods: Budget impact analysis comparing a treatment mix "without" versus "with" the toripalimab regimen in the US eligible annual incident R/M NPC population, a 3-year time horizon, toripalimab/pembrolizumab market splits of 60/40 (Y1) and 80/20 (Y2/3), and medication adjustments for discontinuation or progression. Cost inputs included drugs, administration, and adverse event (AE) management. The models were replicated for a hypothetical 1-million-member health plan in which costs per-member-per-month (PMPM) and per-member-per-year (PMPY) were estimated. One-way (OWSA) and probabilistic sensitivity analyses (PSA) as well as scenario analyses were performed., Results: In the "without" scenario, the 3-year WAC-based costs for the pembrolizumab regimen total $1,449,695,333 ($1,305,632,448 for treatment and $144,062,885 for managing AEs). In the "with" scenario, total 3-year costs for pembrolizumab decline to $380,012,135 with toripalimab adding $885,505,900 ($779,206,567 for treatment and $106,299,333 for AE management). Annual net savings range from $46,526,152 in 2024 to $71,194,214 in 2026, for 3-year savings of $184,177,298. Associated net savings in a 1-million-member health plan are $543,068 over 3 years with savings of $0.045 PMPM and $0.543 PMPY. The ASP-based model shows similar patterns with 3-year net savings of $174,235,983 in the US incident population and savings of $0.043 PMPM and $0.514 PMPY in a 1-million-member health plan. The PSA support base case findings; OWSA and scenario analyses reveal how parameter variability impacts results., Conclusion: Savings between $174 million and $184 million can be achieved from treating 60% of R/M NPC patients in year 1 and 80% in years 2 and 3 with the toripalimab regimen over a similar pembrolizumab regimen.

Background: Few interventions are known to reduce the incidence of respiratory failure that occurs following elective surgery (postoperative respiratory failure; PRF). We previously reported risk factors associated with PRF that occurs within the first 5 days after elective surgery (early PRF; E-PRF); however, PRF that occurs six or more days after elective surgery (late PRF; L-PRF) likely represents a different entity. We hypothesized that L-PRF would be associated with worse outcomes and different risk factors than E-PRF., Methods: This was a retrospective matched case-control study of 59,073 consecutive adult patients admitted for elective non-cardiac and non-pulmonary surgical procedures at one of five University of California academic medical centers between October 2012 and September 2015. We identified patients with L-PRF, confirmed by surgeon and intensivist subject matter expert review, and matched them 1:1 to patients who did not develop PRF (No-PRF) based on hospital, age, and surgical procedure. We then analyzed risk factors and outcomes associated with L-PRF compared to E-PRF and No-PRF., Results: Among 95 patients with L-PRF, 50.5% were female, 71.6% white, 27.4% Hispanic, and 53.7% Medicare recipients; the median age was 63 years (IQR 56, 70). Compared to 95 matched patients with No-PRF and 319 patients who developed E-PRF, L-PRF was associated with higher morbidity and mortality, longer hospital and intensive care unit length of stay, and increased costs. Compared to No-PRF, factors associated with L-PRF included: preexisiting neurologic disease (OR 4.36, 95% CI 1.81-10.46), anesthesia duration per hour (OR 1.22, 95% CI 1.04-1.44), and maximum intraoperative peak inspiratory pressure per cm H 2 0 (OR 1.14, 95% CI 1.06-1.22)., Conclusions: We identified that pre-existing neurologic disease, longer duration of anesthesia, and greater maximum intraoperative peak inspiratory pressures were associated with respiratory failure that developed six or more days after elective surgery in adult patients (L-PRF). Interventions targeting these factors may be worthy of future evaluation., (© 2022. The Author(s).)

BACKGROUND: Ibrutinib, acalabrutinib, and zanubrutinib have shown improvements in efficacy and safety over conventional chemoimmunotherapy in refractory or relapsed mantle cell lymphoma (R/R MCL). OBJECTIVE: To evaluate the comparative cost-effectiveness of the Bruton's tyrosine kinase inhibitors (BTKIs) and estimate the expected value of (partial) perfect information (EV[P]PI) in terms of net health benefits (NHBs) and net monetary benefits (NMBs) forgone. METHODS: Using a two-state Markov model (progression-free; progression or death), we estimated in base-case and probabilistic sensitivity analyses (PSAs) the incremental cost-effectiveness (ICER) and cost-utility ratios (ICUR) of, respectively, progression-free survival (PFS) life-years (PFLYs) and PFS quality-adjusted LY (PFQALY) gained (g) against 3-year and 5-year time horizons. A willingness-to-pay threshold of $150,000/PFQALY was used to assess the probability of being cost-effective in the PSA. EVPI was calculated from the respective NHBs and NMBs. RESULTS: Compared with ibrutinib, acalabrutinib yielded a 3-year ICER of $90,571 (PSA = $88,588)/PFLYg and ICUR of $117,098 ($110,063)/PFQALYg, whereas zanubrutinib yielded a 3-year ICER of $58,422 ($58,907)/PFLYg and ICUR of $73,027 ($73,634)/PFQALYg. The corresponding 5-year estimates were ICER of $73,918 ($74,189)/PFLYg and ICUR of $90,512 ($90,844)/PFQALYg for acalabrutinib and ICER of $48,641 ($48,732)/PFLYg and ICUR of $61,612 ($63,727)/PFQALYg for zanubrutinib. Compared with zanubrutinib, treatment with acalabrutinib yielded a 3-year ICER of $144,633 ($134,964)/PFLYg and ICUR of $197,227 ($166,109)/PFQALYg; the corresponding 5-year estimates were $117,579 ($118,161)/PFLYg and $136,144 ($136,818)/PFQALYg. The EVPI/patient was an NHB of 0.036 PFQALYs and NMB of $3,602 forgone, resulting in a population EVPI of $134,766,957 forgone. The EVPPIs/patient for effectiveness were NHB of 0.015 PFQALYs and NMB of $1,479, with corresponding values of 0.032 and $3,187 for costs and 0.015 and $1,519 for health-related quality of life forgone. CONCLUSIONS: This early cost-effectiveness analysis based on phase I/II clinical trials of BTKIs in R/R MCL suggests an additional PFS benefit for second-generation BTKIs compared with ibrutinib. However, the relative uncertainty due to the lack of direct trial evidence may lead to an opportunity cost or lost health benefits if the current evidence is adopted to compare between these products. Additional evidence is needed to address the relative efficacy of the BTKIs. DISCLOSURES: A. McBride serves on speakers bureaus for Coherus BioSciences and Merck. He is now at Bristol-Myers Squibb in a position unrelated to this study. I. Abraham is joint equity owner in Matrix45. By company policy, owners and employees are prohibited from owning equity in client and sponsor organizations (except through mutual funds or other independently administered collective investment instruments), contracting independently with client and sponsor organizations, or receiving compensation independently from such organizations. Matrix45 provides similar services to biopharmaceutical, diagnostics, and medical device companies on a nonexclusivity basis. Of relevance to this article, Matrix45 has not provided any services to this study. I. Abraham is the quantitative methods editor of JAMA Dermatology and deputy editor-in-chief of the Journal of Medical Economics. The remaining authors have no relevant financial or nonfinancial interests to disclose.

BACKGROUND: Chemotherapy-induced myelosuppression, which commonly manifests as neutropenia, anemia, and/or thrombocytopenia, is a frequent and severe complication of standard treatment regimens for patients with extensive-stage small cell lung cancer (ES-SCLC). Trilaciclib is a first-in-class myeloprotective therapy indicated to decrease the incidence of chemotherapy-induced myelosuppression when administered prior to a platinum-/etoposide-containing regimen or topotecan-containing regimen for ES-SCLC. OBJECTIVE: To estimate the budget impact of administering trilaciclib prior to chemotherapy to manage chemotherapy-induced myelosuppression in adults with ES-SCLC from a US payer perspective. METHODS: A budget impact model was developed to assess the impact of introducing trilaciclib to a hypothetical 1 million-member health insurance plan. The model compared 2 market scenarios: a current scenario of standard treatments for ES-SCLC without trilaciclib, and an alternative scenario of standard treatment plus trilaciclib. Population, clinical, and cost inputs were derived from published literature and trilaciclib clinical trial data. Model outcomes included the number of myelosuppressive adverse events (AEs), costs of treatment, costs of AE management, total cost, and per-member per-month (PMPM) costs. The budget impact of trilaciclib was calculated as the difference in cost (2021 US dollars) between the 2 scenarios over a 1- to 5-year time horizon. Scenario and deterministic sensitivity analyses were conducted to assess uncertainty around key model inputs. RESULTS: An estimated total of 301 patients were eligible for treatment with trilaciclib over a 5-year period. The use of trilaciclib was estimated to reduce the number of myelosuppressive AEs over a 5-year period (events avoided included 108 for neutropenia, 7 for febrile neutropenia, 23 for anemia, and 46 for thrombocytopenia) compared with the scenario without trilaciclib. The adoption of trilaciclib was associated with a cost saving of $801,254 ($0.013 PMPM) over 5 years. The acquisition cost for trilaciclib ($3,704,199) was offset by the reduction in AE management cost ($4,282,748) and reduction in prophylactic granulocyte colony-stimulating factor use ($222,704). The cost savings associated with trilaciclib began in year 1 (total $34,388; $0.003 PMPM) and accrued over time. CONCLUSIONS: The acquisition cost of trilaciclib is projected to be offset by a reduction in the costs of managing AEs related to myelosuppression when added to standard chemotherapy regimens for ES-SCLC. The net budget impact of trilaciclib is estimated to be a cost saving. DISCLOSURES: This research was funded by G1 Therapeutics, Inc., and implemented by ZS Associates, an independent consultancy that collated the model inputs and performed the budget impact analysis. The study sponsor was involved in the study design; collection, analysis, and interpretation of data; writing of the report; and the decision to submit the report for publication. The journal open access fee was funded by G1 Therapeutics, Inc. Moran, Chioda, and Huang are employed by G1 Therapeutics, Inc. Chioda and Huang report stocks and stock options for G1 Therapeutics, Inc. Goyal and Deniz are employed by ZS Associates. Goyal reports consulting fees from G1 Therapeutics, Inc. Abraham reports consulting fees from Coherus, G1 Therapeutics, Inc. (unrelated to this study and manuscript), Mylan/Viatris, and Sandoz and participation on a data safety monitoring board or advisory board for G1 Therapeutics, Inc. MacDonald reports consulting fees from Coherus, G1 Therapeutics, Inc. (unrelated to this study and manuscript), Mylan/Viatris, and Sandoz. Deniz reports no disclosures. A synopsis of the current study was presented in poster format at the Virtual AMCP Annual Meeting, April 12-16, 2021.

Aim: To estimate cost-savings from conversion to biosimilar pegfilgrastim-cbqv that could be reallocated to provide budget-neutral expanded access to AC (doxorubicin/cyclophosphamide) and TCH (docetaxel/carboplatin/trastuzumab) in breast cancer (BC) patients. Methods: Simulation modeling in panels of 20,000 BC and 5000 HER2+ (HER2+ BC) patients, varying treatment duration (one-six cycles) and conversion rates (10-100%), to estimate cost-savings and additional AC and TCH treatment that could be provided. Results: In 20,000 patients, cost-savings of $1,083 per-patient per-cycle translate to $21,652,064 (one cycle) to $129,912,397 (six cycles). Savings range from $5,413,016 to $32,478,097, respectively, in the 5000-patient HER2+ BC panel. Conclusion: Conversion to pegfilgrastim-cbqv could save up to $130 million and provide more than 220,000 additional cycles of antineoplastic treatment on a budget-neutral basis to BC patients.

In this population-based study, we used the SEER database (1985-2015) to examine survival outcomes in chronic lymphocytic leukemia (CLL) patients followed up to the era of advanced treatments including targeted therapies. Data were extracted for patients 15 years or older with a primary diagnosis of CLL. A period analysis was performed to estimate 5- and 10-year relative survival rates for patients diagnosed during different calendar periods from 1985 to 2015. A mixture cure model was used to examine long-term survivors' proportions among patients diagnosed in 1985-2015 and for two cohorts diagnosed in 2000-2003, followed up to 2012 and 2004-2007, and followed up to 2015. Cox proportional hazard modeling was used for the two cohorts to estimate hazard ratios (HRs) of death adjusted for gender and age. The 5-year and 10-year age-adjusted relative survival rate ranged between 73.7 and 89.4% and from 51.6% to "not reached," respectively, for calendar periods of 1985-1989 to 2010-2014. The long-term survivor proportions varied by age and gender from 0 to 59%. The HRs (95%CI) for the 2004-2007 cohort in comparison to the 2000-2003 cohort were 0.58 (0.43-0.78), 0.58 (0.48-0.70), 0.57 (0.49-0.0.67), 0.68 (0.54-0.85), and 0.83 (0.68-1.02) for the age categories of 45-54, 55-64, 65-74, 75-84, and ≥ 85 years, respectively. Overall, relative survival improved significantly for CLL patients diagnosed between 1985 and 2015. These improvements were markedly better following the introduction of targeted therapies., (© 2021. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.)

Aim: To compare the incidence of febrile neutropenia and related outcomes of prophylactic same-day versus next-day pegfilgrastim/pegfilgrastim-cbqv in patients with lymphoma receiving cyclophosphamide, hydroxydaunorubicin, vincristine, prednisone (CHOP)-like chemotherapy. Methods: Retrospective, real-world, single-institution study. Results: 93 patients received 460 cycles of CHOP-like chemotherapy. The incidence of febrile neutropenia and grade 3/4 chemotherapy-induced neutropenia was 5 and 16.5%, respectively. In 401 cycles pegfilgrastim was administered same-day versus 12 cycles next-day. Febrile neutropenia occurred in 17 cycles versus 0 cycles (p = 1.00) and grade 3/4 chemotherapy-induced neutropenia in 65 cycles (16.2%) versus 1 cycle (16.7%; p = 1.00) with same-day versus next-day pegfilgrastim administration, respectively. Conclusion: Pegfilgrastim may be safely administered on the same day as chemotherapy in patients with lymphoma receiving CHOP-like chemotherapy.

Background: The risk of immune-related(ir)-hypothyroidism in older patients with advanced melanoma treated with anti-CTLA4 or anti-PD1 therapies is poorly understood, especially in the real-world setting. Research design and methods: We identified older patients (≥65 years) diagnosed with advanced melanoma between 2011-2015 and treated with anti-CTLA4 or anti-PD1 agents in the SEER-Medicare database. Applying probability-of-treatment-weighting for confounder adjustment and proportional hazards models, we estimated the risk of ir-hypothyroidism between treatment initiation and up to 90 days from last dose between anti-PD1 and anti-CTLA4 users. Results: Of 210 older patients with advanced melanoma identified, 164 received anti-CTLA4 (ipilimumab) and 46 anti-PD1 agents (11 nivolumab, 35 pembrolizumab). There was no statistically significant difference in ir-hypothyroidism risk between anti-PD1 and anti-CTLA4 users (HR=2.15, 95%CI=0.83-5.53). Pairwise medication comparisons showed a lower risk among ipilimumab versus nivolumab (HR=0.15, 95%CI=0.06-0.40) and pembrolizumab versus nivolumab users (HR=0.13, 95%CI=0.03-0.55). Sensitivity analyses using an all-stages melanoma cohort did not show a difference in ir-hypothyroidism risk between medication classes and individual medications. Conclusions: This retrospective claims data analysis revealed no statistically significant difference in ir-hypothyroidism risk between anti-CTLA4 or anti-PD1 users. However, patients with advanced melanoma treated with ipilimumab or pembrolizumab may have a lower ir-hypothyroidism risk compared to nivolumab users.

Aims: For this economic analysis, we aimed to model: (1) the cost-efficiency of prophylaxis with biosimilar pegfilgrastim-bmez for chemotherapy-induced (febrile) neutropenia (CIN/FN) compared to reference pegfilgrastim, and (2) the expanded access to CIN/FN prophylaxis and anti-neoplastic treatment that could be achieved with biosimilar cost-savings on a budget-neutral basis. Methods: In a hypothetical panel of 20,000 cancer patients receiving CIN/FN prophylaxis and using the average sales price (ASP) for the second quarter of 2019 for reference pegfilgrastim, we: conducted an ex ante simulation from the payer perspective of the cost-savings of 10-100% conversion from reference to biosimilar pegfilgrastim-bmez using drug price discounting ranging from 10-35%; estimated the budget-neutral expanded access to biosimilar pegfilgrastim-bmez enabled by these cost-savings; and estimated the budget-neutral expanded access to anti-neoplastic treatment with pembrolizumab. The simulations were replicated using fourth quarter 2019 wholesale acquisition cost (WAC) for reference pegfilgrastim and biosimilar pegfilgrastim-bmez in a post facto analysis. Results: In ASP simulations, cost-savings of using pegfilgrastim-bmez over reference pegfilgrastim in a 20,000 patient panel range from $1.3 M (at 15% price discount) to $3 M (35%) at 10% conversion rate and from $6.4 M to $14.9 M, respectively, at 50% conversion. These savings could provide prophylaxis with pegfilgrastim-bmez to an additional 352 (15% discount) to 1,076 patients (35%) at 10% conversion or 1,764-5,384, respectively, at 50% conversion. Alternatively, savings could be reallocated for anti-neoplastic treatment with pembrolizumab to 3 (15% discount) to 9 (35%) patients at 10% conversion or 19-45, respectively, at 50% conversion. When utilizing WAC, cost-savings range from $4.6 M (10% conversion) to $23.1 M (50%) which could provide pegfilgrastim-bmez to an additional 1,174 (10% conversion) to 5,873 patients (50%). Conclusions: Prophylaxis with biosimilar pegfilgrastim-bmez increases the value of cancer care by generating significant cost-savings that could be reallocated to provide expanded access to CIN/FN prevention and anti-neoplastic therapy on a budget-neutral basis.

Purpose: Clinical trials often exceed their anticipated enrollment periods, and study sites often do not meet accrual goals. We previously reported the development and validation of a single-site accrual prediction model. Here, we describe the expansion of this methodology at 16 cancer centers (CCs) and compare an overall model versus site-specific models., Methods: This retrospective cohort study used data from treatment and supportive care intervention studies permanently closed to accrual between 2009 and 2015 at 16 United States-based CCs. Center and ClinicalTrials.gov data were used to generate both site-specific and random effects mixed models (random effect: institution). Accrual predictions were generated from each model and compared with the accrual prediction of the disease team (DT)., Results: Sixteen institutions submitted 5,787 eligible trials (range, 93 to 697 trials per institution). Local accrual ranged from 363 to 6,716 participants; 1,053 studies (18%) accrued no participants. Actual average accrual was 8.5 participants (median, four participants). Site-specific models predicted accrual at 99% of actual and correctly predicted whether a study would accrue four or more participants 73% of the time versus DT prediction of 58%. Correlation at the category level was 30%; model sensitivity and specificity were 83% and 62%, respectively. The overall model predicted accrual 93% of actual and correctly predicted accrual of four or more participants 66% of the time, with a correlation at the category level of 28%., Conclusion: Both regression models predicted clinical trial accrual at least as or more accurately than DT at all but one center. Site-specific models generally performed slightly better than the random effects model. This study confirms the previous finding that this method is an accurate and objective metric that can be easily implemented to improve clinical research resource allocation across multiple centers.

Importance: A phase 2 trial comparing talimogene laherparepvec plus ipilimumab vs ipilimumab monotherapy in patients with advanced unresectable melanoma found no differential benefit in progression-free survival (PFS) but noted objective response rates (ORRs) of 38.8% (38 of 98 patients) vs 18.0% (18 of 100 patients), respectively., Objective: To perform an economic evaluation of talimogene laherparepvec plus ipilimumab combination therapy vs ipilimumab monotherapy., Design, Setting, and Participants: For PFS, cost-effectiveness and cost-utility analyses using a 2-state Markov model (PFS vs progression or death) was performed. For ORRs, cost-effectiveness analysis of the incremental cost of 1 additional patient achieving objective response was performed. In this setting based on a US payer perspective (2017 US dollars), participants were patients with advanced unresectable melanoma., Main Outcomes and Measures: The PFS life-years and PFS quality-adjusted life-years were determined, and the associated incremental cost-effectiveness ratios (ICERs) and incremental cost-utility ratios (ICURs) were estimated. Also estimated was the ICER per 1 additional patient (out of 100 treated patients) achieving objective response. Base-case analyses were validated by sensitivity analyses., Results: In PFS analyses, the cost of talimogene laherparepvec plus ipilimumab ($494 983) exceeded the cost of ipilimumab monotherapy ($132 950) by $362 033. The ICER was $2 129 606 per PFS life-years, and the ICUR was $2 262 706 per PFS quality-adjusted life-year gained. Probabilistic sensitivity analyses yielded an ICER of $1 481 208 per PFS life-year gained and an ICUR of $1 683 191 per PFS quality-adjusted life-year gained. In 1-way sensitivity analyses, the PFS hazard ratio and the utility of response were the most influential parameters. Talimogene laherparepvec plus ipilimumab has a 50% likelihood of being cost-effective at a willingness-to-pay threshold of $1 683 191 per PFS quality-adjusted life-year gained. In ORR analyses, talimogene laherparepvec plus ipilimumab ($474 904) vs ipilimumab alone ($132 810), a $342 094 difference, yielded an ICER of $1 629 019 per additional patient achieving objective response. In subgroup analyses by disease stage and BRAFV600E mutation status, ICERs ranged from $1 069 044 to $17 104 700 per 1 additional patient achieving objective response., Conclusions and Relevance: The cost to gain 1 additional progression-free quality-adjusted life-year, 1 additional progression-free life-year, or to have 1 additional patient attain objective response is about $1.6 million. This amount may be beyond what payers typically are willing to pay. Combination therapy of talimogene laherparepvec plus ipilimumab does not offer an economically beneficial treatment option relative to ipilimumab monotherapy at the population level. This should not preclude treatment for individual patients for whom this regimen may be indicated.

Background: Treatments for metastatic pancreatic cancer include monotherapy with gemcitabine (GEM); combinations of GEM with oxaliplatin (OX + GEM), cisplatin (CIS + GEM), capecitabine (CAP + GEM), or nab-paclitaxel (NAB-P + GEM); and the non-GEM combination FOLFIRINOX. Combination therapies have yielded better survival outcomes than GEM alone. A sponsor-independent economic evaluation of these regimens has not been conducted for USA., Objective: The objective of this study was to estimate the cost utility and cost effectiveness of these regimens from the payer perspective for USA., Methods: A three-state Markov model (progression-free, progressed disease, death) simulating the total costs and health outcomes (quality-adjusted life-years; life-years) was developed to estimate the incremental cost-utility and cost-effectiveness ratios. FOLFIRINOX clinical data were obtained from trial and indirect estimates were obtained from network meta-analyses. Lifetime horizon and 3%/year discount rates were used., Results: FOLFIRINOX was the most expensive regimen and GEM the least costly regimen. Compared to GEM, all but one (CIS + GEM) regimen were found to be more effective in quality-adjusted life-years and life-years. Compared to GEM, the incremental cost-utility ratios for CAP + GEM, OX-GEM, NAB-P + GEM, and FOLFIRINOX, were US$180,503, US$197,993, US$204,833, and US$265,718 per additional quality-adjusted life-year, respectively; and the incremental cost-effectiveness ratios were US$88,181, US$87,620, US$135,683, and US$167,040 per additional life-year, respectively. A probabilistic sensitivity analysis confirmed the base-case analysis., Conclusions: This sponsor-independent economic evaluation for USA found that OX + GEM, CAP + GEM, FOLFIRINOX, and NAB-P + GEM, but not CIS + GEM, were more expensive but also more effective than GEM alone in terms of quality-adjusted life-years and life-years gained. The NAB-P + GEM regimen appears to be the most cost effective in USA at a willingness-to-pay threshold of US$200,000/quality-adjusted life-year.

The European Medicines Agency (EMA), under a strictly regulated pathway, has approved several biosimilar products since 2005, including biosimilar versions of the erythropoiesis-stimulating agent (ESA) epoetin alfa since 2007. Subsequent to these approvals, the use of biosimilar epoetin alfa in the management of renal anemia has grown steadily throughout Europe. With the enactment of the US Biologics Price Competition and Innovation Act of 2009, a US Food and Drug Administration regulatory approval process for biosimilars was legalized. Thus, biosimilar erythropoietin products are expected to be available for prescription in the USA by mid-decade, presumably at a price that is competitive with the originator brand-name reference products. In this paper, we describe the status of originator and biosimilar ESAs, review the clinical development and regulatory approval of biosimilar erythropoietins in Europe, and summarize relevant efficacy and safety information of biosimilar erythropoietins in relation to their reference products to provide a background for US nephrologists as they appraise biosimilar erythropoietins as treatment options for renal anemia. Key lessons learned from Europe are that (a) EMA-approved biosimilar erythropoietins have comparable efficacy and safety profiles to their reference product erythropoietin; (b) pharmacovigilance preapproval and postapproval are critical, especially with regard to immunogenicity and vascular thromboembolic events; (c) strict preapproval and postapproval requirements must guide the regulatory pathway for biosimilars; and (d) high-quality manufacturing and production processes must be established to ensure quality biosimilar products. The availability of biosimilar erythropoietins in the USA will provide nephrologists with alternative effective, and potentially more affordable, treatment options for patients with renal anemia.

Facilities that provide care to Alzheimer's disease patients are under unrelenting pressure to document the quality of nursing care they provide to various stakeholders. Unfortunately, little consensus exists nor is guidance given as to how to measure the quality of nursing care. Regulations and standards exist but are seldom translated into systematic outcome measures that assist nurses and facilities to measure, report,and manage the quality of care they provide to residents in general and Alzheimer's patients in particular. This article offers practical ad-vice on conceptualizing quality of nursing care to Alzheimer's patients and the selection of outcome measures to collect, analyze, use, and re-port quality of nursing care data.

Background: Elder neglect accounts for over 70% of all adult protective services reports in the nation annually, and it has been estimated that there are over 70,000 new cases each year. The purpose of this study was to conduct elder neglect research in the emergency department (ED), using a dyadic vulnerability/risk-profiling framework for elder neglect., Methods: Patients were recruited through four EDs in New York and Tampa from the beginning of February 2001 through the end of September 2003. Demographics, a Mini-Mental Status Examination score, and an initial elder assessment screen were collected. The diagnosis of neglect was then made by a Neglect Assessment Team (NAT) comprising a nurse, physician, and social worker, with extensive clinical experience in elder neglect., Results: Of the 3664 ED screens of adults 70 years and older, 405 (11%) met the inclusion criteria and agreed to participate. Neglect was diagnosed by the NAT in 86 of the 405 cases reviewed. Demographic differences between neglect versus no neglect cases were examined using Fisher's exact test, and differences emerged between the 2 groups., Conclusion: This study documents the underreporting of cases of neglect as evidenced by differences in diagnoses by screeners versus experts. The research assistants screened positive for neglect in 5% (N=22) of the 405 cases. The NAT made the diagnosis of neglect in 22% (86/389) of the cases. This markedly different rate of neglect may mean that ED screens are important but may underestimate the true number of cases. Conversely, an NAT may make the diagnosis of neglect in an older adult more often given a higher sensitivity and a more robust knowledge base of the problem.

Self-efficacy is an important determinant of health behavior that can be targeted for intervention. Little effort has been given to the development of valid measures for self-efficacy with medication taking for adherence research. The purpose of this study was to determine the criterion validity of the Long-Term Medication Behavior Self-Efficacy Scale (LTMBSES). Individual patient data from 6 existing adherence studies in transplant, hyperlipidemia, and AIDS/HIV patients (n = 1021) were pooled. Validity was determined by assessing the relation between the LTMBSES score and medication adherence--both self-reported and electronically monitored. A weak relationship was found between the LTMBSES score and adherence, which can possibly be attributed to a ceiling effect, caused by a too homogeneous population and/or a failure of the scale to challenge patients. Generalized Estimating Equations revealed that the total average self-efficacy score predicted reported medication adherence (p < .0001). The Receiver Operating Characteristic curve revealed the area under the curve was 0.67, indicating a significant (p < .0001), but poor predictive capability. Evidence for criterion validity of the Long-Term Medication Behavior Self-Efficacy Scale is not yet convincing. Future research should focus on: (1) validation in a population with a more heterogenous level of adherence, and (2) making the scale more challenging by referring to "always taking the medication without exception."

This article describes Nurses Improving Care for Healthsystem Elders (NICHE), a project begun in 1992 with four pilot hospitals. These pilot hospitals gathered baseline data using a geriatric institutional assessment profile (GIAP) with a pre- and postdesign to capture changes in staff attitudes, knowledge, and perceptions of the care of older adults. Based on the success of the pilot effort, NICHE, now in its eighth year, has evolved into a program that involves 32 health systems comprising 105 hospitals nationally. To date, more than 10,000 GIAPs have been collected by NICHE hospital staff. All NICHE settings are able to benchmark their GIAP data against comparable institutions (eg, urban, rural, university, community settings) to understand how they compare and then interpret the data at their unique sites. The opportunities for continuous quality improvement through the NICHE program are described.