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Objective: To estimate readiness of older rehabilitation users in the United States to participate in video-based telerehabilitation and assess disparities in readiness among racial and ethnic minoritized populations, socioeconomically disadvantaged populations, and rural-dwelling older adults., Design: Retrospective cohort study using nationally representative survey data from the National Health and Aging Trends Study from 2015 and 2020. Survey-weighted regression models, accounting for complex survey design, were used to generate estimates of readiness and evaluate disparities across racial and ethnic, socioeconomic, and geographic subgroups. Odds ratios (OR) and 95% confidence intervals (CIs) were estimated for each comparison., Setting: Home or community rehabilitation environments., Participants: A cohort of 5274 home or community-based rehabilitation users aged 70 years or older (N=5274), representing a weighted 33,576,313 older adults in the United States., Interventions: Not applicable., Main Outcome Measure(s): Video-based telerehabilitation readiness was defined consistent with prior work; unreadiness was defined as lacking ownership of internet-enabled devices, limited proficiency of use, or living with severe cognitive, visual, or hearing impairment. Telerehabilitation readiness was categorized as "Ready" or "Unready"., Results: Approximately 2 in 3 older rehabilitation users were categorized as ready to participate in video-based rehabilitation. Significantly lower rates of readiness were observed among those living in rural areas (OR=0.75, 95% CI: 0.60-0.94), financially strained individuals (OR=0.37, 95% CI: 0.26-0.53), and among individuals identifying as Black or Hispanic (as compared with non-Hispanic White older adults: Non-Hispanic Black [OR=0.23, 95% CI: 0.18-0.30]; Hispanic [OR=0.17, 95% CI: 0.11, 0.27])., Conclusions: Our findings highlight significant disparities in the readiness to uptake video-based telerehabilitation. Policy and practice interventions to address telerehabilitation readiness should focus not only on improving broadband access but also on technology ownership and training to ensure equitable adoption in populations with lower baseline readiness., (Copyright © 2024 American Congress of Rehabilitation Medicine. Published by Elsevier Inc. All rights reserved.)

Background: Studies evaluating surgical-site infection have had conflicting results with respect to the use of alcohol solutions containing iodine povacrylex or chlorhexidine gluconate as skin antisepsis before surgery to repair a fractured limb (i.e., an extremity fracture)., Methods: In a cluster-randomized, crossover trial at 25 hospitals in the United States and Canada, we randomly assigned hospitals to use a solution of 0.7% iodine povacrylex in 74% isopropyl alcohol (iodine group) or 2% chlorhexidine gluconate in 70% isopropyl alcohol (chlorhexidine group) as preoperative antisepsis for surgical procedures to repair extremity fractures. Every 2 months, the hospitals alternated interventions. Separate populations of patients with either open or closed fractures were enrolled and included in the analysis. The primary outcome was surgical-site infection, which included superficial incisional infection within 30 days or deep incisional or organ-space infection within 90 days. The secondary outcome was unplanned reoperation for fracture-healing complications., Results: A total of 6785 patients with a closed fracture and 1700 patients with an open fracture were included in the trial. In the closed-fracture population, surgical-site infection occurred in 77 patients (2.4%) in the iodine group and in 108 patients (3.3%) in the chlorhexidine group (odds ratio, 0.74; 95% confidence interval [CI], 0.55 to 1.00; P = 0.049). In the open-fracture population, surgical-site infection occurred in 54 patients (6.5%) in the iodine group and in 60 patients (7.3%) in the chlorhexidine group (odd ratio, 0.86; 95% CI, 0.58 to 1.27; P = 0.45). The frequencies of unplanned reoperation, 1-year outcomes, and serious adverse events were similar in the two groups., Conclusions: Among patients with closed extremity fractures, skin antisepsis with iodine povacrylex in alcohol resulted in fewer surgical-site infections than antisepsis with chlorhexidine gluconate in alcohol. In patients with open fractures, the results were similar in the two groups. (Funded by the Patient-Centered Outcomes Research Institute and the Canadian Institutes of Health Research; PREPARE ClinicalTrials.gov number, NCT03523962.)., (Copyright © 2024 Massachusetts Medical Society.)

Background and Objectives: Local excision (LE) for early-stage gastric cancer has expanded in the United States over recent years, however, national outcomes are unknown. The objective of the study was to evaluate national survival outcomes following LE for early-stage gastric cancer., Methods: Patients with resectable gastric adenocarcinoma between 2010 and 2016 were identified from the National Cancer Database then classified by LE curability into eCuraA (high) and eCuraC (low) according to Japanese Gastric Cancer Association guidelines. Demographics, clinical/provider descriptors, and perioperative/survival outcomes were extracted. Propensity-weighted cox proportional hazards regression assessed factors associated with overall survival., Results: Patients were stratified into eCuraA (N = 1167) and eCuraC (N = 13,905) subgroups. Postoperative 30-day mortality (0% vs 2.8%, p < 0.001) and readmission (2.3% vs 7.8%, p = 0.005) favored LE. Local excision was not associated with survival on propensity-weighted analyses. However, among eCuraC patients, LE was associated with higher likelihood of positive margins (27.1% vs 7.0%, p < 0.001), which was the strongest predictor of poor survival (HR 2.0, p < 0.001)., Conclusions: Although early morbidity is low, oncologic outcomes following LE are compromised for eCuraC patients. These findings support careful patient selection and treatment centralization in the early adoption phase of LE for gastric cancer., Competing Interests: Declaration of competing interest C. Daniel Mullins receives consulting income from Bayer, Merck, and Pfizer unrelated to this manuscript. Otherwise, the authors of this manuscript have no conflicts of interest to disclose., (Published by Elsevier Ltd.)

Introduction: Lower individual-level socioeconomic status (SES) and area-level SES have each been associated with poor survival outcomes among patients with multiple myeloma (MM). A body of literature suggests that individual-level SES may be differentially associated with mortality depending on area-level SES, and vice versa. This study assessed the effect of the cross-level interaction between individual low-income status and area deprivation on mortality among patients with MM., Materials and Methods: This retrospective cohort study used the Surveillance, Epidemiology, and End Results (SEER)-Medicare data (2006-2016). Individuals were defined as having low income if they were dually eligible for Medicare and Medicaid and/or if they received the Low-Income Subsidy. The county-level Social Deprivation Index (SDI) was linked to individual-level SEER-Medicare data and categorized into quintiles, from the least deprived (Quintile 1) to the most deprived (Quintile 5). Adjusted hazard ratios (HRs) for the associations between low-income status, area deprivation, and all-cause mortality were estimated from a mixed-effects Cox proportional-hazards (PH) model., Results: The mortality hazard was higher for individuals with low income than individuals without low income in all quintiles of area deprivation, with the exception of Quintile 5 (Quintile 1: HR 1.53 [95% confidence interval [CI]: 1.32-1.77]; Quintile 2: HR 1.17 [95%CI: 1.01-1.36]; Quintile 3: HR 1.34 [95%CI: 1.18-1.53]; Quintile 4: HR 1.33 [95%CI: 1.17-1.52]; Quintile 5: HR 1.09 [95%CI: 0.96-1.23]). Among individuals without low income, individuals residing in the most deprived area had a higher mortality hazard than individuals residing in the least deprived area (HR: 1.22 [95%CI: 1.03-1.45]). In contrast, among individuals with low income, residing in a more deprived area, Quintile 2, was associated with a lower hazard of death than residing in the least deprived area, Quintile 1 (HR: 0.82 [95%CI: 0.67-0.99]), and there was no statistically significant difference between Quintile 1 and Quintiles 3, 4, and 5., Discussion: In this analysis, there was a statistically significant cross-level interaction between individual low-income status and area deprivation on mortality. More research is needed to fully understand the mechanism behind these associations, but the findings show that patients and their health should be considered in the context of where they live., Competing Interests: Declaration of Competing Interest JY has no competing interests to disclose. YDH is a contractor at the National Cancer Institute, employed by Kelly Services. This research was done prior to employment with Kelly Services. CDM reports receiving consulting fees from Bayer, Incyte, Merck, Pfizer, and Takeda and support for attending meetings/travel from Bayer. EO reports receiving grants/contracts from BeiGene and PhRMA and consulting fees from the PhRMA Foundation and Pfizer for work outside the scope of this project. EO serves on the Maryland Prescription Drug Affordability Board, Blue Health Intelligence Clinical Advisory Board, and the Professional Society for Health Economics and Outcomes Research (ISPOR) Board of Directors (all unpaid). ZZ reports receiving grant/research support from AstraZeneca for work outside the scope of this study. JFS reports receiving grants/contracts from Innovation and Value Initiative, PhRMA Foundation, PhRMA, National Health Council, and Genentech and support for attending meetings/travel from Academy of Managed Care Pharmacy. JFS serves on an advisory board for the COPD Foundation (unpaid), and holds leadership positions at ISPOR (unpaid)., (Copyright © 2022 Elsevier Ltd. All rights reserved.)

Background: Patients with Graves' disease treated with radioactive iodine report worse quality of life than those treated by thyroidectomy. However, radioactive iodine is often selected due to lower risk of complications and lower cost. The objective of this study was to estimate the cost-effectiveness of radioactive iodine versus total thyroidectomy for treatment of Graves' disease., Methods: A Markov decision-analytic model was created to simulate clinical outcomes and costs of medication-refractory Graves' disease treated with radioactive iodine or total thyroidectomy. Complication rates and utilities were derived from published data. Costs were extracted from national Medicare reimbursement rates. We conducted 1-way, 2-way, and probabilistic sensitivity analyses to identify factors that influence cost-effectiveness and reflect uncertainty in model parameters. The willingness-to-pay threshold was set at $100,000/quality-adjusted life-years., Results: Total thyroidectomy yielded 23.6 quality-adjusted life-years versus 20.9 quality-adjusted life-years for radioactive iodine. The incremental cost-effectiveness ratio was $2,982 per quality-adjusted life-years, indicating that surgery is highly cost-effective relative to radioactive iodine. Surgery was more cost effective than radioactive iodine in 88.2% of model simulations. Sensitivity analyses indicate that the model outcomes are driven predominantly by posttreatment quality of life, with contributing effects from rates of treatment complications and the impact of these complications on quality of life., Conclusion: For patients with Graves' disease who either cannot tolerate or are refractory to antithyroid drugs, thyroidectomy is more cost-effective than radioactive iodine. Future research should validate reported differences in quality of life between these 2 treatment modalities., (Copyright © 2022 Elsevier Inc. All rights reserved.)

Background: Attending healthcare appointments and participating in social activities are important for older adults, but these activities are often limited by transportation barriers. Public transportation may bridge these gaps, but little is known about older public transportation users. This study compares the characteristics of older adults who use public transportation to those who do not., Design: Cross-sectional analysis of data from Round 5 of the National Health and Aging Trends Study (NHATS). We identified 5696 urban community dwelling older adults, and calculated national estimates of those who reported public transportation use in the last month and those who used transit to see their regular doctor. We evaluated the age and sex-adjusted associations between economic and clinical characteristics and recent use of public transportation using survey-weighted logistic regression., Results: Nearly 1 in 10 (n = 555/5696, weighted n = 3,122,583) urban-dwelling older adults in the United States reported use of public transportation in the last month, and over 20% of users (weighted n = 658,850) relied on transit to see their regular doctor. Compared to non-users, those who reported using transit were significantly more likely to be younger and identify as non-Hispanic Black or Hispanic. Financially strained older adults were more likely to have recently relied on public transportation (adjusted odds ratio [aOR] 1.62, 95% confidence interval [CI] 1.07-2.44), but frailty (aOR = 0.61, 95% CI 0.41-0.91) and living in an area with cracked or broken sidewalks (aOR = 0.35, 95% CI 0.27-0.46) were both associated with lower odds of public transportation use., Conclusion: More than 3 million older adults in the United States reported recently using public transportation, with over 600,000 relying on these services to visit their doctor. With increasing investment in public infrastructure on the horizon, centering the unique medical, economic, and social needs of older transit users is critical to ensure urban communities remain age-friendly., (© 2022 The American Geriatrics Society.)

Background: The Japanese Gastric Cancer Association provided updated criteria for endoscopic local excision of early-stage gastric cancer in 2018. The purpose of this study was to evaluate utilization patterns for endoscopic local excision in the United States for resectable gastric adenocarcinoma., Methods: Patients with resectable gastric adenocarcinoma were identified from the National Cancer Database between 2010 and 2017. Patients were classified into strict appropriate criteria, expanded criteria, and inappropriate based on the Japanese Gastric Cancer Association guidelines. Factors associated with endoscopic local excision were identified using univariate and logistic multivariate regression., Results: Within the National Cancer Database, 46,334 patients were stratified into strict appropriate criteria (n = 1,405), expanded criteria (n = 727), and inappropriate (n = 43,675). Annual cases of local excision increased by 76.9% over the study period, from 273 in 2010 to 483 in 2017. Among patients who underwent local excision, 10.1% were classified as strict appropriate criteria, 1.6% were classified as expanded criteria, and 84.5% were classified as inappropriate. Among inappropriate patients, factors associated with endoscopic local excision were: more recent year of diagnosis, increasing age, female sex, tumor located in the cardia, smaller size, low-grade, absence of lymphovascular invasion, and treatment at an academic facility., Conclusion: The use of endoscopic local excision for gastric cancer has nearly doubled since 2010. However, most patients do not satisfy consensus criteria for endoscopic therapy., (Copyright © 2022. Published by Elsevier Inc.)

Background: Adjuvant therapy for most sentinel-node-positive (stage IIIA) melanoma may have limited clinical benefit for older patients given the competing risk of non-cancer death. The objective of this study is to model the clinical effect and cost of adjuvant therapy in stage IIIA melanoma across age groups., Study Design: A Markov decision analysis model simulated the overall survival of patients with resected stage IIIA melanoma treated with adjuvant therapy vs observation. In the adjuvant approach, patients are modeled to receive adjuvant pembrolizumab (BRAF wild type) or dabrafenib/trametinib (BRAF mutant). In the observation approach, treatment is deferred until recurrence. Transition variables were derived from landmark randomized trials in adjuvant and salvage therapy. The model was analyzed for age groups spanning 40 to 89 years. The primary outcome was the number needed to treat (NNT) to prevent one melanoma-related death at 10 years. Cost per mortality avoided was estimated using Medicare reimbursement rates., Results: Projections for NNT among BRAF wild type patients increased by age from 14.71 (age 40 to 44) to 142.86 (age 85 to 89), with patients in cohorts over the age of 75 having an NNT over 25. The cost per mortality avoided ranged from $2.75 million (M) (age 40 to 44) to $27.57M (age 85 to 89). Corresponding values for BRAF mutant patients were as follows: NNT 18.18 to 333.33; cost per mortality avoided ranged from $2.75M to $54.70M., Conclusion: Universal adjuvant therapy for stage IIIA melanoma is costly and provides limited clinical benefit in patients older than 75 years., (Copyright © 2022 by the American College of Surgeons. Published by Wolters Kluwer Health, Inc. All rights reserved.)

Background: There are limited data on economic aspects of the genetic variant of chronic obstructive pulmonary disease (COPD) in the context of the more prevalent form of COPD. The objective of this study was to isolate the healthcare resource utilization and economic burden attributable to the presence of a genetic factor among COPD patients with and without Alpha-1 Antitrypsin Deficiency (AATD), twelve months before and after their initial COPD diagnosis., Methods: Retrospective analysis of OptumLabs® Data Warehouse claims (OLDW; 2000-2017). The OLDW is a comprehensive, longitudinal real-world data asset with de-identified lives across claims and clinical information. AATD-associated COPD cases were matched with up to 10 unique non-AATD-associated COPD controls. Healthcare resource use and costs were assigned into the following categories: office (OV), outpatient (OP), and emergency room visits (ER), inpatients stays (IP), prescription drugs (RX), and other services (OTH). A generalized linear model was used to estimate total pre- and post-index (initial COPD diagnosis) costs from a third-party payer's perspective (2018 USD) controlling for confounders. Healthcare resource utilization was estimated using a negative binomial regression., Results: The study population consisted of 8881 patients (953 cases matched with 7928 controls). The AATD-associated COPD cohort had higher expenditures and use of office visits (OV) and other (OTH) services, as well as OV, outpatient (OP), emergency room (ER), and prescription drugs (RX) before and after the index date, respectively. Adjusted total all-healthcare cost ratios for AATD-associated COPD patients as compared to controls were 2.04 [95% CI: 1.60-2.59] and 1.98 [95% CI: 1.55-2.52] while the incremental cost difference totaled $6861 [95% CI: $3025 - $10,698] and $5772 [95% CI: $1940 - $9604] per patient before and after the index date, respectively., Conclusions: Twelve months before and after their initial COPD diagnosis, patients with AATD incur higher healthcare utilization costs that are double the cost of similar COPD patients without AATD. This study also suggests that increased costs of AATD-associated COPD are not solely attributable to augmentation therapy use. Future studies should further explore the relationship between augmentation therapy, healthcare resource use, and other AATD-associated COPD expenditures.

Background: Innovations in hepatitis C virus (HCV) therapy included in traditional comparative evaluations focus on sustained virologic response (SVR) without addressing challenges patients report beyond virologic cure. This study aims to evaluate the cost-effectiveness of HCV drug therapy with a patient-centered approach., Methods: An individual-based Markov model was constructed using guidance from a stakeholder advisory board (SAB), a patient Delphi panel, and published literature to evaluate direct-acting antivirals (DAAs) compared to no treatment. The United States (US) health sector and societal perspectives were considered for 10- and 20-year time horizons. Inputs for treatment costs and effectiveness reflect a generic regimen. Indirect costs used for the societal model included estimates from self-reported productivity in a matched-control sample. Beyond the traditional quality-adjusted life-year (QALY) health outcome, this study included two novel measures developed from the Delphi panel and SAB: infected life-years and workdays missed. All costs were measured in 2018 US dollars., Results: Health sector costs and QALYs were higher in the treatment group in both 10- and 20-year models. Total infected life-years and workdays missed were reduced in the treatment group for both models. When costs of absenteeism, presenteeism, and patient/caregiver time were included, the DAA intervention was cost-saving at both 10 and 20 years. Health sector results were sensitive to drug costs and utility estimates for post-SVR health states. Societal results were sensitive to presenteeism estimates and drug costs., Conclusion: Treatment was cost-effective from a health sector perspective and cost-saving when including non-health costs such as patient/caregiver time and productivity.

Historically, budget impact models have been used to demonstrate how a new drug or technology entering the market could influence payer budgets, with the hope of influencing coverage of the drug or technology. This article proposes the adaptation of a budget impact model framework to answer the payer question "What is the budget impact of our approaches to managing the utilization of drugs and technologies?" Throughout this article, we also describe lessons learned while collaborating with a U.S. payer to evaluate the budgetary impact of specific antidiabetic drug utilization management approaches. DISCLOSURES: The study discussed in this commentary was supported by grant number F32HS024857 from the Agency for Healthcare Research and Quality (AHRQ). The content is solely the responsibility of the authors and does not necessarily represent the official views of AHRQ, which had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; or design to submit the manuscript for publication. The findings discussed in this manuscript represent the views of the authors and do not necessarily reflect the views of the Department of Defense, the Defense Health Agency, nor the Departments of the Army, Navy, and Air Force. Hung reports personal fees from CVS Health and BlueCross BlueShield Association, outside the submitted work. Lugo has nothing to disclose. Mullins reports grants and other fees from AHRQ, during the conduct of the study; grants and personal fees from Bayer and Pfizer; and personal fees from Boehringer-Ingelheim, Janssen/J&J, Regeneron, and Sanofi, outside the submitted work. Parts of the study were presented as a poster presentation at the 2017 AMCP Managed Care & Specialty Pharmacy Annual Meeting; March 27-30, 2017; Denver, CO, and as poster and oral presentations at the 2017 AMCP Nexus Meeting; October 16-19, 2017; Dallas, TX. The study was also published as Hung's PhD dissertation.

Purpose: A hydrogel rectal spacer (HRS) is a medical device that is approved by the U.S. Food and Drug Administration to increase the separation between the prostate and rectum. We conducted a cost-effectiveness analysis of HRS use for reduction in radiation therapy (RT) toxicities in patients with prostate cancer (PC) undergoing external beam RT (EBRT)., Methods and Materials: A multistate Markov model was constructed from the U.S. payer perspective to examine the cost-effectiveness of HRS in men with localized PC receiving EBRT (EBRT alone vs EBRT + HRS). The subgroups analyzed included site of HRS placement (hospital outpatient, physician office, ambulatory surgery center) and proportion of patients with good baseline erectile function (EF). Data on EF, gastrointestinal and genitourinary toxicities incidence, and potential risks associated with HRS implantation were obtained from a recently published randomized clinical trial. Health utilities and costs were derived from the literature and the 2018 Physician Fee Schedule and were discounted 3% annually. Quality-adjusted life years (QALYs) and costs were modeled for a 5-year period from receipt of RT. Probabilistic sensitivity analysis and value-based threshold analyses were conducted., Results: The per-patient 5-year incremental cost for spacers administered in a hospital outpatient setting was $3578, and the incremental effectiveness was 0.0371 QALYs. The incremental cost-effectiveness ratio was $96,440/QALY for patients with PC undergoing HRS insertion in a hospital and $39,286/QALY for patients undergoing HRS insertion in an ambulatory facility. For men with good baseline EF, the incremental cost-effectiveness ratio was $35,548/QALY and $9627/QALY in hospital outpatient and ambulatory facility settings, respectively., Conclusions: Based on the current Medicare Physician Fee Schedule, HRS is cost-effective at a willingness to pay threshold of $100,000. These results contain substantial uncertainty, suggesting more evidence is needed to refine future decision-making., (Copyright © 2018. Published by Elsevier Inc.)

The learning healthcare system (LHS) model framework has three core, foundational components. These include an infrastructure for health-related data capture, care improvement targets and a supportive policy environment. Despite progress in advancing and implementing LHS approaches, low levels of participation from patients and the public have hampered the transformational potential of the LHS model. An enhanced vision of a community-engaged LHS redesign would focus on the provision of health care from the patient and community perspective to complement the healthcare system as the entity that provides the environment for care. Addressing the LHS framework implementation challenges and utilizing community levers are requisite components of a learning health care community model, version two of the LHS archetype.

Objective: The aim of this study was to assess the budget impact of introducing the proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i) alirocumab and evolocumab to market for the treatment of adults with heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular (CV) disease requiring additional lowering of low-density lipoprotein cholesterol (LDL-C)., Methods: A 3-year model estimated the costs of lipid-modifying therapy (LMT) and CV events to a hypothetical US health plan of 1 million members, comparing two scenarios-with and without the availability of PCSK9i as add-on therapy to statins. Proportions of patients with uncontrolled LDL-C despite receiving statins, and at risk of CV events, were estimated from real-world data. Total undiscounted annual LMT costs (2017 prices, including PCSK9i costs of $14,563.50), dispensing and healthcare costs, including the costs of CV events, were estimated for all prevalent patients in the target population, based on baseline risk factors. Maximum PCSK9i utilization of 1-5% over 3 years according to risk group (following the same pattern as current ezetimibe use), and 5-10% as a secondary scenario, were assumed., Results: Total healthcare budget impacts per target patient (and per member) per month for years 1, 2 and 3 were $3.62($0.10), $7.22($0.20) and $10.79($0.30), respectively, assuming 1-5% maximum PCSK9i utilization, and $15.81($0.44), $31.52($0.88) and $47.12($1.31), respectively, assuming 5-10% utilization. Results were sensitive to changes in model timeframe, years to maximum PCSK9i utilization and PCSK9i costs., Conclusions: The budget impact of PCSK9i as add-on therapy to statins for patients with hypercholesterolemia is relatively low compared with published estimates for other specialty biologics. Drug cost rebates and discounts are likely to further reduce budget impact.

Designing culturally sensitive personalized interventions is essential to sustain patients' involvement in their treatment and encourage patients to take an active role in their own health and health care. We consider patient activation and empowerment as a cyclical process defined through patient accumulation of knowledge, confidence, and self-determination for their own health and health care. We propose a patient-centered, multilevel activation and empowerment framework (individual-, health care professional-, community-, and health care delivery system-level) to inform the development of culturally informed personalized patient activation and empowerment (P-PAE) interventions to improve population health and reduce racial and ethnic disparities. We discuss relevant Affordable Care Act payment and delivery policy reforms and how they affect patient activation and empowerment. Such policies include Accountable Care Organizations and value-based purchasing, patient-centered medical homes, and the community health benefit. Challenges and possible solutions to implementing the P-PAE are discussed. Comprehensive and longitudinal data sets with consistent P-PAE measures are needed to conduct comparative effectiveness analyses to evaluate the optimal P-PAE model. We believe the P-PAE model is timely and sustainable and will be critical to engaging patients in their treatment, developing patients' abilities to manage their health, helping patients express concerns and preferences regarding treatment, empowering patients to ask questions about treatment options, and building up strategic patient-provider partnerships through shared decision making., (© 2015 Society for Public Health Education.)

Purpose: Multidisciplinary physician care has increased for many cancers yet little evidence exists for hepatocellular carcinoma (HCC). The purpose of this study was to explore the association between multispecialist care and mortality in HCC., Methods: Treated patients with an HCC primary diagnosis from 2000 to 2007 were studied using Surveillance, Epidemiology, and End Results-Medicare data. A surrogate variable for multidisciplinary care was defined-multispecialist care-as the number of disciplines among surgeons, radiology oncologist, intervention radiologist, hematologist/medical oncologist, gastroenterologist, and generalist in the pretreatment period. Multivariate survival analysis was conducted and adjusted for selection and survival bias., Results: Of 3588 treated HCC patients, 1434 (40%) saw 1, 1343 (37%) saw 2, and 811 (23%) saw 3 or more specialists. Patients with multispecialist care received treatment that differed from patients who saw a single specialist. In propensity score-adjusted survival analysis, patients who saw 3 or more specialist types were associated with 10% (P=0.04) reduced mortality, compared with those who saw 1 specialist. When stratified by treatment received, patients on chemotherapy who saw 3 or more specialist types were associated with 28% (P=0.002) reduced mortality, compared with those who saw 1 specialist., Conclusions: Multispecialist care for treated HCC patients was associated with reduced mortality, particularly among chemotherapy recipients. While adjusting for selection and survival bias, our study is limited in capturing a causal relationship between coordinated multidisciplinary care and mortality. Our findings may provide support for the development of coordinated care delivery models but should be confirmed through more rigorous examination in future studies.

Background: Skeletal related events (SREs) are common in men with metastatic prostate cancer (mPC). Various methods have been used to identify SREs from claims data. The objective of this study was to provide a framework for measuring SREs from claims and compare SRE prevalence and cumulative incidence estimates based on alternative approaches in men with mPC., Methods: Several claims-based approaches for identifying SREs were developed and applied to data for men aged [greater than or equal to] 66 years newly diagnosed with mPC between 2000 and 2009 in the SEER-Medicare datasets and followed through 2010 or until censoring. Post-diagnosis SREs were identified using claims that indicated spinal cord compression (SCC), pathologic fracture (PF), surgery to bone (BS), or radiation (suggestive of bone palliative radiation, RAD). To measure SRE prevalence, two SRE definitions were created: 'base case' (most commonly used in the literature) and 'alternative' in which different claims were used to identify each type of SRE. To measure cumulative incidence, we used the 'base case' definition and applied three periods in which claims were clustered to episodes: 14-, 21-, and 28-day windows., Results: Among 8997 mPC patients, 46 % experienced an SRE according to the 'base case' definition and 43 % patients experienced an SRE according to the 'alternative' definition. Varying the code definition from 'base case' to 'alternative' resulted in an 8 % increase in the overall SRE prevalence. Using the 21-day window, a total of 12,930 SRE episodes were observed during follow up. Varying the window length from 21 to 28 days resulted in an 8 % decrease in SRE cumulative incidence (RAD: 10 %, PF: 8 %, SCC: 6 %, BS: 0.2 %)., Conclusions: SRE prevalence was affected by the codes used, with PF being most impacted. The overall SRE cumulative incidence was affected by the window length used, with RAD being most affected. These results underscore the importance of the baseline definitions used to study claims data when attempting to understand relevant clinical events such as SREs in the real world setting.

Background: Metastatic colon cancer (mCC) patients often receive multiple lines of chemotherapy/biological treatment (TX), yet subsequent TX lines have not been sufficiently examined using SEER-Medicare data. We developed an algorithm that identifies the number and type of TX lines received by mCC patients., Methods: The algorithm rules for detecting TX lines were developed a priori and applied to SEER-Medicare data for 7951 elderly mCC patients, diagnosed in 2003-2007 and followed through 2009. Statistical analysis estimated the relationship between the number of treatments received and patient characteristics. Sensitivity analyses examined how results changed when different algorithm rules were used., Results: Only 41% (3266) of mCC patients received any chemotherapy/biologics treatment; 1440 (18% of all, 44% of treated) and 274 (3% of all, 8% of treated) received second-line and third-line treatment, respectively. Initial and subsequent treatment regimens varied widely. Results were robust to alterations in the algorithm., Conclusions: The number of drugs used to treat cancer patients has increased during the past decade. Patients may have several TX lines with complex regimens. More guidance is needed with regard to identifying and studying these interventions using SEER-Medicare data. By proposing 1 approach to categorizing TX lines for mCC patients, we hope to empower the scientific community and to advance the use of SEER-Medicare data for health outcomes research.

Background: Dementia costs are critical for influencing healthcare policy, but limited longitudinal information exists. We examined longitudinal informal care costs of dementia in a population-based sample., Methods: Data from the Cache County Study included dementia onset, duration, and severity assessed by the Mini-Mental State Examination (MMSE), Clinical Dementia Rating Scale (CDR), and Neuropsychiatric Inventory (NPI). Informal costs of daily care (COC) was estimated based on median Utah wages. Mixed models estimated the relationship between severity and longitudinal COC in separate models for MMSE and CDR., Results: Two hundred and eighty-seven subjects (53% female, mean (standard deviation) age was 82.3 (5.9) years) participated. Overall COC increased by 18% per year. COC was 6% lower per MMSE-point increase and compared with very mild dementia, COC increased over twofold for mild, fivefold for moderate, and sixfold for severe dementia on the CDR., Conclusions: Greater dementia severity predicted higher costs. Disease management strategies addressing dementia progression may curb costs., (Copyright © 2015 The Alzheimer's Association. Published by Elsevier Inc. All rights reserved.)

The U.S. Patient Protection and Affordable Care Act (hence, Affordable Care Act, or ACA) was signed into law on March 23, 2010. Goals of the ACA include decreasing the number of uninsured people, controlling cost and spending on health care, increasing the quality of care provided, and increasing insurance coverage benefits. This manuscript focuses on how the ACA affects pharmacy benefit managers and consumers when they have prescriptions dispensed. PBMs use formularies and utilization control tools to steer drug usage toward cost-effective and efficacious agents. A logic model was developed to explain the effects of the new legislation. The model draws from peer-reviewed and gray literature commentary about current and future U.S. healthcare reform. Outcomes were identified as desired and undesired effects, and expected unintended consequences. The ACA extends health insurance benefits to almost 32 million people and provides financial assistance to those up to 400% of the poverty level. Increased access to care leads to a similar increase in overall health care demand and usage. This short-term increase is projected to decrease downstream spending on disease treatment and stunt the continued growth of health care costs, but may unintentionally exacerbate the current primary care physician shortage. The ACA eliminates limitations on insurance and increases the scope of benefits. Online health care insurance exchanges give patients a central location with multiple insurance options. Problems with prescription drug affordability and control utilization tools used by PBMs were not addressed by the ACA. Improving communication within the U.S. healthcare system either by innovative health care delivery models or increased usage of health information technology will help alleviate problems of health care spending and affordability., (Copyright © 2015 Elsevier Inc. All rights reserved.)

Background: Aligning with a national priority to bridge health disparities in disadvantaged populations, we explored contextual determinants of pretreatment quality of care and treatment receipt of Medicare disabled patients with hepatitis C virus (HCV) infection., Methods: We used Medicare claims (2006-2009) linked to the Area Health Resource Files. Ordinal partial proportional odds and weighted modified Poisson regressions were used to model the determinants of quality care receipt and interferon-based treatment, respectively., Results: We identified 1936 Medicare disabled HCV patients, of whom 10.4% were treated with peg-interferon. Despite the high comorbidity burden among HCV disabled patients, greater engagement in care correlated with greater likelihood of quality care and treatment receipt., Conclusion: Our study highlights the need for process and linkage to care in Medicare disabled HCV patients, but future research relevant to novel interferon-free agents is needed to assess patterns of quality of care and treatment receipt in this vulnerable population.

Background: Factors contributing to the lower likelihood of urologist follow-up among African American (AA) men diagnosed with prostate cancer may not be strictly related to patient factors. The authors investigated the relationship between crime, poverty, and poor housing, among others, and postdiagnosis urologist visits among AA and white men., Methods: The authors used linked cancer registry and Medicare claims data from 1999 through 2007 for men diagnosed with American Joint Committee on Cancer stage I to III prostate cancer. The USA Counties and County Business Patterns data sets provided county-level data. Variance components models reported the percentage of variation attributed to county of residence. Postdiagnosis urologist visits for AA and white men were investigated using logistic and modified Poisson regression models., Results: A total of 65,635 patients were identified; 87% of whom were non-Hispanic white and 9.3% of whom were non-Hispanic AA. Approximately 16% of men diagnosed with stage I to III prostate cancer did not visit a urologist within 1 year after diagnosis (22% of AA men and 15% of white men). County of residence accounted for 10% of the variation in the visit outcome (13% for AA men and 10% for white men). AA men were more likely to live in counties ranked highest in terms of poverty, occupied housing units with no telephone, and crime. AA men were less likely to see a urologist (odds ratio, 0.65 [95% confidence interval, 0.6-0.71]; rate ratio, 0.94 [95% confidence interval, 0.92-0.95]). The sign and magnitude of the coefficients for the county-level measures differed across race-specific regression models of urologist visits., Conclusions: Among older men diagnosed with stage I to III prostate cancer, the social environment appears to contribute to some of the disparities in postdiagnosis urologist visits between AA and white men., (© 2014 American Cancer Society.)

Background: In the past decade, many oncologic drugs have been approved that extend life and/or improve patients' quality of life. However, new cancer drugs are often associated with high price and increased medical spending. For example, in 2010, the average annual cost of care for breast cancer in the final stage of disease was reported to be $94,284, and the total estimated cost in the United States was $16.50 billion., Objective: To determine whether value threshold, as defined by the incremental cost-effectiveness ratio (ICER), differed between oncology and other therapeutic areas. , Methods: The PubMed database was searched for articles published between January 2003 and December 2013 with calculated ICER for therapeutic drug entities in a specific therapeutic area. The search term used was "ICER" and "United States." From 275 results, only those articles that reported ICERs using quality-adjusted life-years (QALY) were included. In addition, only those articles that used a U.S. payer perspective were retained. Among those, nondrug therapy articles and review articles were excluded. The mean ICER and value threshold for oncologic drugs and nononcologic drugs were evaluated for the analysis. , Results: From 54 articles selected for analysis, 13 pertained to drugs in oncology therapeutics, and the remaining 41 articles addressed ICER for drugs in other therapeutic areas. The mean and median of ICERs calculated for cancer-specific drug intervention was $138,582/QALY and $55,500/QALY, respectively, compared with $49,913/QALY and $31,000/QALY, respectively, for noncancer drugs. Among the cancer drugs, 45.0% had ICERs below $50,000/QALY and 70.0% below $100,000/QALY. In comparison, 72.0% of noncancer drugs showed ICERs below $50,000/QALY, and 90.0% had ICERs below $100,000/QALY. When a specific threshold was mentioned, it was in the range of $100,000-$150,000 in cancer drugs, whereas drugs in other therapeutic areas used traditional threshold value within the range of $50,000-$100,000., Conclusions: The average ICER reported for cancer drugs was more than 2-fold greater than the average ICER for noncancer drugs. In general, articles that addressed the relative value of oncologic pharmaceuticals used higher value thresholds and reported higher ICERs than articles evaluating noncancer drugs.

Objective: Skeletal-related events (SREs) are defined as a cluster of events including clinical diagnoses and treatment. Using claims data, the burden of SREs as a group has been reported among patients with cancer. We investigate the mortality impact of subcomponents of SREs, a topic that has received limited attention among older men., Materials and Methods: We analyzed prostate cancer (PCa) and all-cause mortality among men diagnosed with metastatic PCa from 2000 to 2007 using Surveillance, Epidemiology, and End Results data linked with 1999-2009 Medicare data. We created three measures of pathological fracture (PF), spinal cord compression (SCC), and bone surgery (BS) that differed in the use of claims-based bone metastasis information. We reported covariate-adjusted hazard ratios (HRs) using the full sample and a propensity score-matched sample (PSMS)., Results: Application of inclusion/exclusion criteria resulted in 7062 men in the full sample (1776 in the PSMS). PCa-specific (all-cause mortality) was 54% (80%) at a median follow-up of 609days. SRE prevalence ranged from 9.7% to 17.1% across the measures. In a PCa mortality model, the HR associated with an SRE ranged from 1.07 (0.98-1.16) to 1.31 (1.18-1.45). The HRs for SCC and PF were statistically significant and positively associated with PCa-specific mortality. The results for BS depended on the measure. Results for SCC and BS, but not for PF, were preserved using a PSMS., Conclusions: The relationship between SREs and mortality among older men with metastatic PCa was driven by SCC and depended on the definition used to measure SREs., (Copyright © 2014 Elsevier Inc. All rights reserved.)

Objective: To investigate hospital-level variation in short-term laparoscopic colectomy outcomes among stage I-III elderly colon cancer patients., Background: Surgical outcomes are associated with patient and surgeon characteristics. If outcomes are also impacted by the hospital where the surgery occurs, there is a hospital center effect (HCE)., Methods: Surveillance, Epidemiology, and End Results (SEER)-Medicare data was used to identify stage I-III colon cancer patients treated with laparoscopic colectomies. Multilevel regressions were utilized to study potential HCE for length of stay (LOS), 30-day rehospitalization, and in-hospital mortality, adjusting for patient, surgeon, and hospital-level characteristics. To quantify HCE, we calculated the median instantaneous rate ratio (MIRR) for LOS and median odds ratio (MOR) for in-hospital mortality and 30-day rehospitalization. Sensitivity analyses were conducted for high volume/medical school affiliated hospitals and colorectal surgeons., Results: The multilevel analyses based on 4617 patients from 465 hospitals documented statistically significant HCEs for LOS (MIRR = 1.35; P < 0.001) and in-hospital mortality (MOR = 1.69; P = 0.032), but no HCE for 30-day rehospitalization. Sensitivity analyses confirmed our findings. HCE was significant for LOS in all sensitivity analyses and was significant for in-hospital mortality for high volume/medical school affiliated hospitals., Conclusions: HCE exists for LOS and in-hospital mortality of laparoscopic colectomy, which suggests that the choice of hospital affects outcomes independently of other confounding variables. Reducing the variation in outcomes associated with HCE may improve the quality of cancer care.

Background and Objective: Advanced prostate cancer patients with bone metastasis are predisposed to skeletal complications termed skeletal-related events (SREs). There is limited information available on Medicare costs associated with treating SREs. The objective of this study was to ascertain SRE-related costs among older men with metastatic prostate cancer in the US., Methods: We analysed patients aged 66 years or older who were diagnosed with incident stage IV (M1) prostate cancer between 2000 and 2007 from the linked Surveillance, Epidemiology and End Results (SEER)-Medicare dataset. A propensity score for the incidence of an SRE was estimated using a logistic regression model including demographic and clinical baseline variables. Patients with SREs (cases) were matched to patients without SREs (controls) based on the propensity score, length of follow-up (i.e. date of prostate cancer diagnosis to last date of observation) and death. Health resource utilization cost differences between cases and controls over time were compared using generalized linear models. Healthcare costs were examined by type of SRE (pathological fracture only, pathological fracture with concurrent surgery, spinal cord compression only, spinal cord compression with concurrent surgery, and bone surgery only) and by source of care (inpatient, physician/non-institutional provider, skilled nursing facility, outpatient and hospice). All costs were adjusted to 2009 US dollars, using the medical care component of the Consumer Price Index., Results: Application of the inclusion criteria resulted in 1,131 metastatic prostate cancer patients with SREs and 6,067 patients without SREs during follow-up. The average age of the sample was 79 years, and 14 % were African American. A total of 928 patients with SREs were matched to 928 patients without SREs. The average health care utilization cost of patients with SREs was US$29,696 (95 % confidence interval [CI] US$24,730-US$34,662) higher than that of the controls. The most expensive SRE group was spinal cord compression with concurrent surgery (US$82,868: 95 % CI US$67,472-US$98,264) followed by bone surgery only (US$37,496: 95 % CI US$29,684-US$45,308), pathological fracture with concurrent surgery (US$34,169: 95 % CI US$25,837-US$ 42,501), spinal cord compression only (US$25,793: 95 % CI US$20,933-US$30,653) and pathological fracture only (US$14,649: 95 % CI US$6,537-US$22,761). The largest cost difference by source of care was observed for hospitalizations (p < 0.01)., Conclusion: Metastatic prostate cancer patients with SREs incur higher costs compared to similar patients without SREs. SRE costs among older stage IV (M1) prostate cancer patients vary by SRE type, with spinal cord compression and concurrent surgery costing at least twice as much as other SREs.

With new agents entering the market, the sequencing of first-line (Tx1), second-line (Tx2), and subsequent chemotherapy/biologics regimens are being examined. We examined how Tx1 regimens impacted the likelihood of receiving Tx2 among metastatic colon cancer (mCC) patients. Surveillance, Epidemiology and End Results (SEER)-Medicare data were used to identify elderly mCC patients between 2003 and 2007. The inverse probability weighting Cox regression method was utilized to study the relationship between receipt of Tx2 and Tx1 regimens, controlling for patient-level factors. Of the 7895 elderly patients identified, 3211 (41%) received Tx1 of which 1440 proceeded to Tx2. The impact of Tx1 on receipt of Tx2 varied by the specific regimens utilized. As compared to 5FU/LV users, IROX (Hazard Ratio [HR] = 0.03; P < 0.01) and IROX + Biologics (HR = 0.20; P < 0.01) users were less likely to receive Tx2; (oxaliplatin) OX + Biologics (HR = 1.26; P < 0.01) users were more likely to receive Tx2. Significant patient-level factors included: Hispanic ethnicity (HR = 0.67; P < 0.01); being married (HR = 0.87; P = 0.01); proxy for poor performance status (HR = 0.82; P = 0.05); each 10-year age increment (HR = 1.14; P < 0.01); and State buy-in status (HR = 1.21; P = 0.01). The specific first-line regimen does impact mCC patients' likelihood of receiving Tx2 in clinical practice. Elderly mCC patients, their health care providers, and policy makers will benefit from new evidence about the impact of sequencing of treatment lines., (© 2014 The Authors. Cancer Medicine published by John Wiley & Sons Ltd.)

Background: The incidence of hepatocellular carcinoma (HCC) is increasing in the USA and worldwide. Several treatments are available for patients diagnosed at any disease stage. It remains unclear how medical expenditures vary across patients who remain untreated or undergo different modes of therapy. We evaluate the comparative and cost effectiveness of treatment modalities for HCC from a Medicare perspective., Methods: The Surveillance, Epidemiology, and End Results (SEER) registries and linked Medicare database with claims from Parts A/B were used to identify Medicare enrollees with initial diagnosis of HCC between 2000 and 2007 and followed through 2009. Patients were assigned to treatment modalities based on HCC staging systems: transplant, resection, liver directed, radiation, chemotherapy or no treatment. Survival benefits and cumulative Medicare expenditures were estimated in multivariate models, stratified by initial disease stage, to control for confounding. Cost-effectiveness ratios compared costs and benefits of the modalities across initial stages., Results: Cancer stages I, II, III, IV and unstaged represented 24, 9, 14, 17 and 37 % of 11,047 patients, respectively. Fewer than 40 % received any treatment. Relative to no treatment, transplant was most effective in reducing mortality, followed by resection, liver directed, and radiation or chemotherapy. Resection tended to be most cost effective in early staged and unstaged patients; transplant was least cost effective. In stage IV patients, liver directed therapy was more cost effective than chemotherapy or radiation., Conclusions: Survival benefit was attributable to all treatment modalities. More effective treatments incurred greater Medicare expenditures, but resection patients incurred the least expenditures per year of life gained.

Among older individuals diagnosed with metastatic colon cancer (mCC) there is limited evidence available that describes the characteristics associated with advancing to second- and subsequent lines of treatment with chemotherapy/biologics. Our objective was to describe the trends and lines of treatment received among elderly mCC patients. Elderly beneficiaries diagnosed with mCC from 2003 to 2007 were identified in the Surveillance, Epidemiology and End Results (SEER)-Medicare dataset. Beneficiaries were followed up until death or censoring. Treatment lines were classified in combinations of chemotherapies and biologics. Modified Poisson regression was used to predict receipt of lines of treatment. Analyses controlled for age, race/ethnicity, gender, marital status, state buy-in during diagnosis year, SEER-registry site, Charlson comorbidity index (CCI), poor performance indicators, surgery of primary site, and surgery of regional/distal sites. Among 7951 Medicare beneficiaries identified with mCC, 3266 initiated therapy. Of these, 1440 advanced to second-line treatment. Of these, 274 advanced to a subsequent-line treatment. Surgeries of the primary tumor site and of the regional/distal sites and marital status were the most significant variables associated with advancing through second- and subsequent-line treatments. Greater than 80 years of age, African American race, SEER-registry area, less than 6 months state buy-in assistance in mCC diagnosis year, and having poor performance indicators were inversely associated with receipt of second- or subsequent-line treatments. Among elderly individuals diagnosed with mCC, we identified demographic, clinical, and regional factors associated with receipt of second- and subsequent-line chemotherapy/biologics. Additional research is warranted to understand the role of physician versus patient preferences as well as geographic differences explaining why patients advance through lines of chemotherapy., (© 2013 The Authors. Cancer Medicine published by John Wiley & Sons Ltd.)

Background: The Benefits Improvement and Protection Act (BIPA) expanded Medicare coverage for posttransplantation immunosuppresants for elderly patients and others eligible for Medicare beyond their end-stage renal disease (ESRD) status yet retained the 3-year limit for patients eligible solely because of ESRD status. Our objective was to determine BIPA's impact on renal transplantation among elderly patients (age ≥65 years) affected by BIPA., Methods: Medicare claims and the U.S. Renal Data System Standard Analysis Files were used to analyze the likelihood of transplantation among elderly patients, all of whom were affected by BIPA, versus the nonelderly, many of whom were unaffected by BIPA. A difference-in-differences approach and generalized logistic regressions were used to estimate BIPA's impact., Results: Analysis of data for 632,904 ESRD Medicare beneficiaries who met inclusion/exclusion criteria suggests that BIPA made elderly patients more likely (relative likelihood, 1.36; 95% confidence interval, 1.32-1.41) to have a transplant. The likelihood for nonelderly patients decreased following BIPA (relative likelihood, 0.93; 95% confidence interval, 0.92-0.94)., Conclusion: Transplantation rates increased among those elderly patients, all of whom were affected by BIPA by extending immunosuppressant coverage under BIPA. These results suggest that removing financial barriers to posttransplantation care may positively impact transplantation rates yet raise questions regarding whether the law shifted transplants from younger to older patients.

Background: Timely linkage to appropriate care after human immunodeficiency virus (HIV) diagnosis is critical to optimizing patient outcomes. Medicaid is the largest source of health care coverage for patients with HIV in the United States, yet no studies of linkage to appropriate HIV care have focused solely on the Medicaid population., Methods: This is a retrospective study using Medicaid claims data from 15 states. Study sample comprised patients aged 18 to 64 years with 1 or more HIV tests between January 1, 2003, to May 1, 2010, followed or accompanied by HIV diagnosis. The "Test Index" corresponded to the HIV test that was temporally proximate to first HIV diagnosis. Study end point was linkage to appropriate HIV care, defined as receipt of CD4 and viral load tests as per US treatment guidelines. Time-to-event analyses characterized patterns and correlates of linkage to appropriate care., Results: This study included 6684 patients, with a mean age of 35 years, 70% female, and 47% black race. Overall, 21.0% of patients linked to appropriate care within 1 year of the Test Index and 26.4% within 5 years. Compared with whites, blacks had a significantly shorter time to linkage to HIV appropriate care (hazard ratio, 2.034; P < 0.001)., Conclusions: These findings in Medicaid patients newly diagnosed with HIV contrast with prior research show disparities in access to HIV care favoring whites. Overall, the proportion of patients who linked to appropriate HIV care was very low given the availability of effective treatment, suggesting a need for more effective interventions promoting timely linkage to appropriate care after diagnosis.

Purpose: To provide recommendations to trialists and sponsors that guide the design and implementation of prospective postapproval clinical trials for oncology drugs used outside US Food and Drug Administration-labeled indications for treatment of late-stage cancers., Methods: A meeting was hosted by the Center for Medical Technology Policy in Baltimore, MD, on November 12, 2009. Discussions during the meeting and key informant interviews were conducted before and after this stakeholder meeting. Peer review by multidisciplinary stakeholders was followed by a public comment period. Input was received from patient advocacy groups, medical oncologists, pharmaceutical companies, the US Food and Drug Administration, Centers for Medicare and Medicaid Services, the National Cancer Institute, foreign government agencies involved in health technology assessment, public and private payers, drug compendia, clinical research entities, statisticians, academics, and the American Society of Clinical Oncology., Results: To address the needs of patients and their clinical providers, compendia, payers, and policy makers, recommendations are proposed to guide the design of future prospective trials for off-label use of oncology drugs across four areas: trial design and data analysis, patient and site recruitment, comparators, and outcomes., Conclusion: The US Food and Drug Administration provides guidance to the pharmaceutical industry and others designing randomized clinical trials for regulatory approval. However, a gap exists for postregulatory decision makers, including patients, prescribers, and payers, because regulatory trials do not answer the questions most relevant to them. Therefore, guidance is needed for trials performed in the postapproval environment for these postapproval decision makers.

Aim: The prognostic effects of chemotherapy and various lymph node measures [positive nodes, total node count and the positive lymph node ratio (PLNR)] have been established. It is unknown whether the cancer-specific survival benefit of chemotherapy differs across these nodal prognostic categories., Method: This retrospective analysis of linked Surveillance, Epidemiology and End Results (SEER) data and Medicare data (SEER-Medicare)included patients ≥ 65 years of age with a diagnosis of stage III colon cancer between 1997 and 2002. We grouped patients according to the number of positive nodes (N1 and N2), total node count (≥ 12 and < 12 total nodes) and PLNR (below the 75th percentile and at least at the 75th percentile of the PLNR). The end point was colon cancer-specific mortality., Results: Fifty-one per cent (3701) of the 7263 patients received adjuvant therapy during the time period 1997-2002. The mean (standard deviation) number of total nodes examined was 13 (9) and the number of positive nodes identified was 3 (3). Patients with N2 disease, < 12 total nodes examined and a high PLNR had a worse survival at 2, 3 and 5 years following colectomy. Utilization of chemotherapy demonstrated a colon cancer-specific survival benefit (hazard ratio at median follow up = 0.7; P < 0.001) that was consistent and statistically significant across the three nodal prognostic categories examined., Conclusion: The benefit of chemotherapy did not vary based on N stage, total node count or PLNR. The results favour a broad-based approach towards increasing the chemotherapy treatment rates in stage III patients of ≥ 65 years of age, rather than an approach that targets clinical subgroups., (© 2011 The Authors. Colorectal Disease © 2011 The Association of Coloproctology of Great Britain and Ireland.)

Background: The improvement in survival rates for patients with colon cancer has shifted the focus from examining cancer-specific mortality to exploring all-cause mortality. Adverse events such as venous thromboembolism (VTE) affect overall survival times and the net clinical benefit of cancer management strategies., Methods: This retrospective study used Surveillance, Epidemiology and End Results (SEER) Medicare data to examine VTE incidence and mortality rates for elderly patients with stage III colon cancer who were diagnosed in 2004 or 2005 and followed through 2007. The impact of VTE on mortality was estimated using multivariable Cox proportional hazards regression., Results: In all, 20.7% of 4,985 elderly patients with stage III colon cancer had clinically diagnosed VTE following diagnosis. All-cause mortality risk was higher for patients with a VTE diagnosis (hazard ratio [HR]: 1.15, 95% confidence interval [CI]: 1.04-1.27), greater comorbidity burden, more advanced tumor depth and nodal involvement within stage III, advanced age, and male sex; the risk was lower for patients treated with chemotherapy. VTE was associated with higher mortality hazards (HR: 1.41, 95% CI: 1.21-1.64) for patients treated with adjuvant chemotherapy but not for untreated patients., Conclusions: A new diagnosis of VTE significantly reduced survival rates for elderly patients with stage III colon cancer and further reduced survival rates for patients treated with chemotherapy. Improved prevention and management of VTE for elderly patients with stage III colon cancer who are at risk for VTE is warranted, particularly for patients treated with chemotherapy.

Background: The Patient Protection and Affordable Care Act brought considerable attention to comparative effectiveness research (CER).  , Objectives: To (a) suggest best practices for conducting and reporting CER using "real-world data" (RWD), (b) describe some of the data and infrastructure requirements for conducting CER using RWD, (c) identify statistical challenges with the analysis of nonrandomized studies and suggest appropriate techniques to address those challenges, (d) recognize the value of patient-reported outcomes in CER, (e) encourage the incorporation of observational data into randomized controlled studies, and (f) highlight the importance of incorporating payers in industry-sponsored research. , Summary: The first article in this supplement, "Something old, something new…" provides a policy perspective on the recent evolution of CER. It reviews the historical context, discusses the "promise and fear" of CER, and then describes the new role of the Patient-Centered Outcomes Research Institute (PCORI) in defining and sponsoring CER. The second paper, "Ten Commandments," proposes a series of tenets for planning, conducting, and reporting CER done with RWD. Oriented for basic-to-intermediate researchers, it combines standard scientific research principles with considerations specific to nonrandomized, RWD studies. The third article, "Infrastructure Requirements," points out that effective use of secondary data requires addressing major methodological and infrastructural issues, including development of analytical tools to readily access and analyze data, formulation of guidelines to enhance quality and transparency, establishment of data standards, and creation of data warehouses that respect the privacy and confidentiality of patients. It identifies gaps that must be filled to address the underlying issues, with emphasis on data standards, data quality assurance, data warehouses, computing environment, and protection of privacy and confidentiality. The fourth paper, "Statistical Issues," discusses how the validity of analytic results from observational studies is adversely impacted by biases that may be introduced due to lack of randomization. It reviews some of the methodological challenges that arise in the analysis of data from nonrandomized studies, with particular emphasis on the limitations of traditional approaches and potential solutions from recent methodological developments. The fifth paper, "Considerations on the Use of Patient Reported Outcomes (PROs)," describes how PRO data can play a critical role in guiding patients, health care providers, payers, and policy makers in making informed decisions regarding patient-centered treatment from among alternative options and technologies and have been noted as such by PCORI. However, collection and interpretation of such data within the context of CER have not yet been fully established. It discusses some challenges with including PROs in CER initiatives, provides a framework for their effective use, and proposes several areas for future research. Lastly, "Developing a Collaborative Study Protocol…" indicates that there is the potential, the desire, and the capability for payers to be involved in CER studies, combining elements of their own observational data with prospective studies. It describes a case example of a payer, a pharmaceutical company, and a research organization collaborating on a prospective study to examine the effect of prior authorization for pregabalin on health care costs to the payer.  , Conclusion: Researchers at Pfizer routinely conduct CER-type studies. In this supplement, we have proposed some approaches that we believe are useful in developing certain kinds of evidence and have described some of our experiences. Our experiences also make us acutely aware of the limitations of approaches and data sources that have been used for CER studies and suggest that there is a need to further develop methods that are most useful for answering CER questions.

To address the historically low rate of minority participation in clinical trials, the NIH and others have provided incentives to increase the diversity of patients and study sites involved in NIH-funded research. An example of the efforts to achieve this aim was the creation of the Partnerships Program to Reduce Cardiovascular Health Disparities," whereby a health care system that serves a predominantly minority patient population partners with a research-intensive medical center that has a track record of NIH-supported research. In the city of Baltimore, Maryland, the Bon Secours Baltimore Health System partnered with the University of Maryland and was awarded 1 of 7 U01 partnerships within cardiovascular health. This commentary describes the qualitative experiences of the participating physicians and not a quantitative analysis of the study data or findings. It describes the lessons learned by Bon Secours that can address patient and physician barriers to clinical trial participation. Based upon the views of participating physicians, it is easiest to engage physicians when there is a supportive principal investigator and appropriate infrastructure to do research. Patient recruitment requires cultural competence and sensitivity, and using minority physicians, nurses, and staff may greatly assist in building the trust that is necessary for patients to be willing to participate in research. Clarity of the study's purpose and aims at the outset is critical, and reinforcement with educational sessions helped with physician and patient retention throughout the study.

Unlabelled: To investigate the survival benefit associated with chemotherapy receipt in older women with estrogen receptor-negative (ER-) Stage IV breast cancer., Design: Observational, retrospective cohort study using Cox proportional hazards regression to determine effect of chemotherapy on hazard of all-cause mortality. The two samples were an overall sample (n=1,519) and a propensity score-matched sample (n=580) to control for selection to treatment receipt. Hazard ratios (HRs) and 95% confidence intervals (CIs) were obtained for regression models., Setting: U.S. women within the National Cancer Institute Surveillance, Epidemiology and End Results cancer registries (SEER) linked to Medicare enrollment and claims database., Participants: Female Medicare beneficiaries aged 66 and older with Stage IV ER- breast cancer diagnosed between 1999 and 2005., Measurements: Outcome measure was all-cause death during the follow-up period. Survival was measured as time from breast cancer diagnosis until death or last follow-up date. Information on receipt of chemotherapy, defined as chemotherapy received within 6 months after diagnosis, was obtained from linked Medicare claims., Results: One thousand five hundred nineteen ER- women diagnosed with metastatic breast cancer were identified; 494 (33%) received chemotherapy. Chemotherapy was associated with a statistically significant survival benefit (HR=0.61, 95% CI=0.54-0.70). Age did not modify the survival benefit of chemotherapy., Conclusion: Chemotherapy received within 6 months after diagnosis was associated with a 39% lower hazard of death within the time period for the study. These findings reflect chemotherapy use outside of the clinical trial setting and have important clinical and policy implications for the study of treatments in older women with advanced ER- breast cancer., (© 2011, Copyright the Authors. Journal compilation © 2011, The American Geriatrics Society.)

Objective: To assess the national impact of the March 2004 Food and Drug Administration (FDA) antidepressant suicidality warning on the outpatient treatment of new-onset depression in youth., Method: A repeated measures, longitudinal design in a cohort of youth diagnosed with new-onset depression was used to assess pre- and post-FDA warning effects. US commercial insurance enrollees in the i3 INNOVUS database from January 2003 through December 2006 were examined. The study population included youth 2- to 17-years old with a new-onset depression diagnosis from July 2003 through June 2006 (N = 40,309). The main independent variables were the warning period (post- vs. pre-FDA warning) and age group (children vs. adolescents). The main outcome measures were youth with antidepressant dispensings and psychotherapy visits measured in 30-day intervals across 36 months following a new-onset diagnosis of any depressive disorder (N = 40,309) and specifically major depressive disorder (MDD) (N = 11,532)., Results: Compared to youth with a new-onset diagnosis of depression in the pre-FDA warning period, youth with new-onset diagnosis of depression during the postwarning period had (1) A significantly lower likelihood of antidepressant use: (odds ratio [OR] = 0.85 [0.81-0.89]); When youth with the diagnosis of depression were separated into those with MDD and those with less severe depression diagnoses, only the latter had a significant postwarning antidepressant decline. (2) A significant increase in the odds of a psychotherapy visit (children, OR = 1.31 [1.23-1.40]; adolescents OR = 1.19 [1.15-1.24])., Conclusions: The FDA suicidality warning was associated with an overall decrease in antidepressant treatment for youth with a clinician-reported diagnosis of depression, but not for those with MDD. Also, following the warning, psychotherapy without medication increased.

Background: There is limited information in the literature about reasons for discharges against medical advice (DAMA) as supplied by patients and providers. Information about the reasons for DAMA is necessary for identifying workable strategies to reduce the likelihood and health consequences of DAMA. The objective of this study is to identify the reasons for DAMA based on patient and multicategory provider focus-group interviews (FGIs)., Methods: Patients who discharged against medical advice between 2006 and 2008 from a large, academic medical centre along with hospital providers reporting contact with patients who left against medical advice were recruited. Three patient-only groups, one physician-only group and one nurse/social worker group were held. Focus-group interviews were transcribed, and a thematic analysis was performed to identify themes within and across groups. Participants discussed the reasons for patient DAMA and identified potential solutions., Results: Eighteen patients, five physicians, six nurses and four social workers participated in the FGIs. Seven themes emerged across the separate patient, doctor, nurse/social worker FGIs of reasons why patients leave against medical advice: (1) drug addiction, (2) pain management, (3) external obligations, (4) wait time, (5) doctor's bedside manner, (6) teaching hospital setting and (7) communication. Solutions to tackle DAMA identified by participants revolved mainly around enhanced communication and provider education., Conclusions: In a large, academic medical centre, the authors find some differences and many similarities across patients and providers in identifying the causes of and solutions to DAMA, many of which relate to communication.

Objectives: To examine whether race or age disparities affected the odds of being staged among prostate cancer (PC) patients. Accurate staging is critical for determining treatment for PC., Methods: Multivariable logistic regression models examined race and age disparities with respect to the odds of being staged among PC patients using Surveillance, Epidemiology, and End Results-Medicare data. Similar analyses were performed to estimate the adjusted odds of being staged with distant metastatic vs in situ or local/regional disease., Results: The proportion of patients without staging ranged between 3% and 16% by age and between 6% and 8% by race. Adjusted results demonstrated statistically significant lower odds ratios (P <.05) for 70-74, 75-79, and 80+-year-olds of 0.76, 0.52, and 0.23, respectively, relative to PC patients aged 65-69. The odds of being staged for African Americans are 0.78 times that of non-Hispanic Whites (95% confidence interval = 0.72-0.86). The adjusted probability of distant metastatic disease at initial diagnosis is higher for African Americans (odds ratio = 1.61; 95% confidence interval = 1.47-1.76) and older men with odds ratios of 1.25, 1.85, and 4.33 for ages 70-74, 75-79, and 80+, respectively, compared with 65-69-year-olds (all P <.05)., Conclusions: Even though the overall odds of being staged increased over time, race and age disparities persisted. When staging did occur, the probability of distant metastatic disease was high for African Americans, and there were increasing odds of metastatic disease for all men with advanced age., (Copyright © 2010 Elsevier Inc. All rights reserved.)

Objective: To determine whether there would be racial and ethnic disparities in meeting eligibility criteria for medication therapy management (MTM) services implemented in 2006 for Medicare beneficiaries., Data Sources/study Setting: Secondary data analyses of the Medical Expenditure Panel Survey (2004-2005)., Study Design: Logistic regression and recycled predictions were used to test the disparities in meeting eligibility criteria across racial and ethnic groups. The eligibility thresholds used by health plans in 2006 and new thresholds recommended for 2010 were examined. Racial and ethnic disparities were examined by comparing non-Hispanic blacks (blacks) with non-Hispanic whites (whites) and comparing Hispanics with whites, respectively. Disparities were also examined among individuals with severe health problems., Principal Findings: According to 2006 thresholds, the adjusted odds ratios for meeting eligibility criteria for blacks and Hispanics to whites were 0.36-0.60 (p<.05) and 0.13-0.46 (p<.05), respectively. Blacks and Hispanics would be 21-34 and 32-38 percent, respectively, less likely to be eligible than whites according to recycled predictions. Similar patterns were found using the 2010 eligibility thresholds and among individuals with severe health problems., Conclusions: There would be racial and ethnic disparities in meeting MTM eligibility criteria. Future research is warranted to confirm the findings using data after MTM implementation.

The application of cost-effectiveness analysis in healthcare has become commonplace in the US, but the validity of this approach is in jeopardy unless the proverbial $US50,000 per QALY benchmark for determining value for money is updated for the 21st century. While the initial aim of this article was to review the arguments for abandoning the $US50,000 threshold, it quickly turned to questioning whether we should maintain a fixed threshold at all. Our consideration of the relevance of thresholds was framed by two important historical considerations. First, cost-effectiveness analysis was developed for a resource allocation exercise where a threshold would be determined endogenously by maximizing a fixed budget across all possible interventions and not for piecemeal evaluation where a threshold needs to be set exogenously. Second, the foundations of the $US50,000 threshold are highly dubious, so it would be unacceptable merely to adjust for inflation or current clinical practice. Upon consideration of both sides of the argument, we conclude that the arguments for abandoning the concept for maintaining a fixed threshold outweigh those for keeping one. Furthermore, we document a variety of reasons why a threshold needs to vary in the US, including variations across payer, over time, in the true budget impact of interventions and in the measurement of the effectiveness of interventions. We conclude that while a threshold may be needed to interpret the results of a cost-effectiveness analysis, that threshold must vary across payers, populations and even procedures.

Patients, clinicians, payers, and policymakers face an environment of significant evidentiary uncertainty as they attempt to achieve maximum value, or the greatest level of benefit possible at a given level of cost in their respective health care decisions. This is particularly true in the area of oncology, for which published evidence from clinical trials is often incongruent with real-world patient care, and a substantial portion of clinical use is for off-label indications that have not been systematically evaluated. It is this uncertainty in the knowledge of the clinical harms and benefits associated with oncology treatments that prevents postregulatory decision makers from making accurate assessments of the value of these treatments. Because of the incentives inherent in the clinical research enterprise, randomized control trials (RCTs) are designed for the specific purpose of regulatory approval and maximizing market penetration. The pursuit of these goals results in RCT study designs that achieve maximal internal validity at the expense of generalizability to diverse real-world patient populations that may have significant comorbidities and other clinically mitigating factors. As such, systematic reviews for the purposes of coverage and treatment decisions often find relevant and high-quality evidence to be limited or nonexistent. For a number of reasons, including frequent off-label use of medications and the expedited approval process for cancer drugs by the U.S. Food and Drug Administration, this situation is exacerbated in the area of oncology. This paper investigates the convergence of incentives and circumstances that lead to widespread uncertainty in oncology and proposes new paradigms for clinical research, including pragmatic clinical trials, methodological guidance, and coverage with evidence development. Each of these initiatives would support the design of clinical research that is more informative for postregulatory decision makers, and would therefore reduce uncertainty and provide greater confidence in conclusions about the value of these treatments.

Objectives: Public programs finance a large share of the US pharmaceutical expenditures. To date, there are not guidelines for estimating the cost of drugs financed by US public programs. The objective of this study was to provide standards for estimating the cost of drugs financed by US public programs for utilization in pharmacoeconomic evaluations., Methods: This report was prepared by the ISPOR Task Force on Good Research Practices-Use of Drug Costs for Cost-Effectiveness Analysis Medicare, Medicaid, and other US Government Payers Subgroup. The Subgroup was convened to assess the methodological and practical issues confronted by researchers when estimating the cost of drugs financed by US public programs, and to propose standards for more transparent, accurate and consistent costing methods., Results: The Subgroup proposed these recommendations: 1) researchers must consider regulation requirements that affect the drug cost paid by public programs; 2) drug cost must represent the actual acquisition cost, incorporating any rebates or discounts; 3) transparency with respect to cost inputs must be ensured; 4) inclusion of the public program's perspective is recommended; 5) high cost drugs require special attention, particularly when drugs represent a significant proportion of health-care expenditures for a specific disease; and 6) because of variations across public programs, sensitivity analyses for actual acquisition cost, real-world adherence, and generics availability are warranted. Specific recommendations also were proposed for the Medicare and Medicaid programs., Conclusions: As pharmacoeconomic evaluations for coverage decisions made by US public programs grows, the need for precise and consistent estimation of drug costs is warranted. Application of the proposed recommendations will allow researchers to include accurate and unbiased cost estimates in pharmacoeconomic evaluations.

Objective: To explore the effect of a prior authorization (PA) policy restricting access to pregabalin for the management of diabetic peripheral neuropathy (DPN) or postherpetic neuralgia (PHN) on the overall utilization of pharmacologic therapy and healthcare services among fee-for-service Medicaid plan beneficiaries., Study Design: Retrospective claims data were obtained for 2005 and 2006 from 6 state Medicaid programs. Two states that had implemented pregabalin PAs beginning in 2006 were compared in terms of drug utilization and costs with 4 states having no such restrictions., Methods: Patients at least 18 years old in a Medicaid fee-for-service program having a diagnosis of DPN or PHN and at least 1 claim for DPN- or PHN-specific pain medication were selected. Pharmacologic therapy, healthcare utilization, and expenditures were analyzed using bivariate statistics and generalized linear models in a difference-in-difference approach for comparing outcomes between cohorts year over year., Results: The 2 cohorts included 424 patients in the restricted states and 5153 patients in the unrestricted states. Compared with the use in the unrestricted states, the probability of pregabalin use in the restricted states decreased by 4.0 percentage points (P = .02) from 2005 to 2006, while the probability of opioid use increased by 6.5 percentage points (P <.01).The DPN- or PHN-related total healthcare costs were $418 higher for the restricted states versus the unrestricted states (P <.001)., Conclusion: Although the PA was shown to effectively control access to pregabalin, the overall effect was an increase in the use of opioids and alternative pain management therapies associated with increased disease-related healthcare costs.

Objectives: To estimate the modifying effect of age on the survival benefit associated with adjuvant chemotherapy receipt in elderly patients with a diagnosis of Stage III colon cancer., Design: Observational, retrospective cohort study using two samples: an overall sample of 7,182 patients to provide externally valid analyses and a propensity score-matched sample of 3,016 patients to provide more internally valid analyses by reducing the presence of treatment endogeneity. An interval-censored survival model with a complementary log-log link was used. Hazard ratios and 95% confidence intervals were obtained for all regressions., Settings: Data from the National Cancer Institute's Surveillance, Epidemiology and End Results database and the linked Medicare enrollment and claims database were used., Participants: Selected patients were aged 66 and older and had a diagnosis of Stage III colon cancer. Patients were followed from surgery to time of death or censorship., Measurements: The outcome was colon cancer-specific death during the follow-up period. Receipt of adjuvant chemotherapy was measured according to the presence of a claim for 5-fluorouracil or leucovorin within 6 months after surgery., Results: All elderly patients had a significant survival benefit associated with adjuvant chemotherapy receipt, although the survival benefit of adjuvant chemotherapy was not uniform across all age groups., Conclusion: These findings have important clinical and policy implications for the risk-benefit calculation induced by treatment in older patients with Stage III colon cancer. The results suggest that there is a benefit from chemotherapy, but the benefit is lower with older age.

Objective: To employ results from a recent US population-based survey to calculate disease-specific total costs of overactive bladder (OAB)., Study Design: Disease-specific total cost-of-illness method using population prevalence estimates., Methods: Cases were identified as community-dwelling adults reporting the presence of urinary urgency or urgency urinary incontinence. Two OAB classifications were used based on Likert scale responses of OAB symptoms: "often" (base case) or "sometimes" (alternative). The study estimates disease-specific total costs of OAB from the societal perspective and using an average costing method. A population-based survey, a claims data analysis, and the published literature provided the prevalence and resource utilization data., Results: The prevalence of OAB as defined in the base case (alternative) was 18.6% (28.7%) in the adult US population, accounting for 42.2 million (65.1 million) community-dwelling adults. The disease-specific total cost of OAB is estimated at $24.9 billion for the base case and $36.5 billion for the alternative case. Total direct costs were $22.3 billion in the base case and $33.5 billion in the alternative case. Costs were higher among adults younger than 65 years of age, compared with adults 65 years or older. This relative cost burden was lower for the base case compared with the alternative case in the full sample, with a larger gap among men., Conclusion: The total cost of OAB among community-dwelling adults is significant and varies with demographic groups. Future research is needed to determine whether the differential cost burden is robust to alternate cost-of-illness estimation methods.

Background: Despite the importance of equal access to prescription drugs by racial and ethnic groups, studies on access to prescription drugs have been incomprehensive., Objective: To describe the Medical Expenditure Panel Survey (MEPS) as a valuable data source to study racial and ethnic disparities in prescription drug use., Methods: Use of the MEPS database to study disparities in prescription drug use is detailed. This includes strengths and limitations of the database, as well as key methodological, sampling, and statistical issues to consider when using it., Results: Using MEPS allows researchers to control for sociodemographic and health status information when studying racial and ethnic disparities in prescription drug use. MEPS has taken measures to improve reliability of the information on the utilization of prescription drugs. An additional benefit of MEPS is that it has oversampled Hispanics, Blacks, and Asians; as such, it affords the statistical power to examine these minority groups. One limitation of MEPS is the inability of researchers to study some drug categories, because of limitations in statistical power. Moreover, the names of the prescription medications are not standardized in MEPS, and some information is not publicly available in MEPS databases. When conducting studies requiring information not publicly available, researchers may commute to the MEPS Data Center or may request the Data Center to run statistical programs for them., Conclusions: Using MEPS to study racial and ethnic disparities in prescription drug use has significant benefits. Nonetheless, researchers need to keep in mind the limitations of using MEPS.

Many pharmacy schools have increased the amount of economics coursework to which pharmacy students are exposed in their prepharmacy and pharmacy curriculums. Students obtain competencies aimed at understanding the basic concepts of microeconomic theory, such as supply and demand. However, pharmacy students often have trouble applying these principles to real world pharmaceuticals or healthcare markets. Our objective is to make economics more relevant for pharmacy students. Specifically, we detail and provide pharmacy-relevant examples of the effects of monopoly power, barriers to marketplace entry, regulatory environment, third party insurance, information asymmetry and unanticipated changes in the marketplace on the supply and demand for pharmaceuticals and healthcare services.