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Purpose: It is estimated that the PTEN tumor suppressor gene is functionally lost in 40%-50% of patients with metastatic castration-resistant prostate cancer (mCRPC). There is limited information on the prognostic significance of PTEN status identified with genomic testing. This real-world cohort study assessed PTEN as a genetic biomarker using data from US-based oncology practices., Methods: This retrospective real-world cohort study used a deidentified US-based metastatic prostate cancer clinicogenomic database linked to longitudinal clinical data derived from electronic health records. Patients were aged 18 years and older and diagnosed with mCRPC between January 1, 2018, and June 30, 2021. Comprehensive genomic profiling (CGP) of tumor specimens was performed using next-generation sequencing. First-line (1L) and second-line (2L) treatment patterns were assessed and stratified by PTEN status. Kaplan-Meier methods and a multivariable Cox model were used to compare the real-world overall survival by PTEN status among patients who received 1L novel hormone therapy or taxanes., Results: In patients with mCRPC who underwent CGP, PTEN loss of function (LOF) was associated with decreased survival compared with intact PTEN (hazard ratio, 1.61 [95% CI, 1.07 to 2.42]; P = .024). The results were not influenced by 1L treatment type. 1L treatment patterns were similar between intact PTEN and PTEN LOF subgroups, with abiraterone and enzalutamide being the two most common treatments in both groups. Patients with PTEN LOF were less likely to receive 2L treatments than patients with intact PTEN ., Conclusion: PTEN LOF, identified with genomic testing, was associated with decreased survival and negative prognoses in patients with mCRPC.

Aim: To assess the cost-effectiveness of definitive therapies for nonmetastatic oropharyngeal cancer (OPC). Materials & methods: Using the Surveillance, Epidemiology and End Results-Medicare dataset, patients diagnosed between 2000 and 2011 were identified. The cost-effectiveness of chemoradiation (CRT) versus radiotherapy (RT), cetuximab plus RT (cetuximab-RT) versus RT and cetuximab-RT versus CRT were estimated. Results: The incremental cost-effectiveness ratio for CRT compared with RT from 2000 to 2005 was US$56,650 (95% CI: US$4,522-$288,688) per additional year of survival. CRT was dominated by RT from 2006 to 2011. Cetuximab-RT was dominated by RT and CRT. Conclusion: CRT had a favorable value from 2000 to 2005 but was dominated by RT from 2006 to 2011. The value of cetuximab-RT compared with RT/CRT was not favorable with similar/inferior survival and substantial incremental costs.

BACKGROUND: The progressive nature of Parkinson disease (PD), together with a lack of curative treatments, contributes to its economic burden. OBJECTIVE: To estimate the longitudinal incremental costs attributable to PD among Medicare beneficiaries. METHODS: In this retrospective cohort study, we used data from the Chronic Conditions Data Warehouse to identify Medicare beneficiaries with and without PD-related claims identified from 2006 to 2014 with follow-up until 2015. We grouped PD cases and controls based on their survival profiles using a grouping algorithm that used the following baseline measures: age, race, sex, and comorbidity. We identified 3 survival groups and used them to stratify the descriptive annual cost estimates in the 9 years after the index date. We estimated the incremental 1-, 3-, and 5-year costs of PD using generalized linear models (GLM) that controlled for baseline factors. RESULTS: We identified 27,394 cases and controls who were grouped into 3 survival groups. The mean age of the full study sample was 73 years. No material differences were found in the incremental cost of PD across the survival groups. Based on the multivariable GLM, the 1-year incremental cost of PD was $9,625 (95% CI, $9,054-$10,197). The 3-year incremental cost of PD was $20,832 (95% CI, $19,390-$22,274). The 5-year incremental cost of PD was $27,466 (95% CI, 25,088-$29,844). CONCLUSIONS: Among Medicare beneficiaries, PD is associated with excess costs compared with controls. We did not identify substantial differences in the incremental cost of PD across the survival groups. DISCLOSURES: This study was funded by Pfizer Inc. The funding agreement did not impact the authors' independence in designing the study, collecting the data, interpreting the data, writing the manuscript, and submitting the manuscript for publication. Dr Onukwugha reports grants from Pfizer Inc for the conduct of this study and is an employee of University of Maryland, Baltimore, which received financial support from Pfizer Inc in connection with the development of this manuscript; Dr Shulman reports research funding from Pfizer Inc related to the current work, is an employee of University of Maryland, Baltimore, which received financial support from Pfizer Inc in connection with the development of this manuscript, and reports research funding from the NIH, The Rosalyn Newman Foundation, and the Eugenia and Michael Brin family unrelated to the current work and royalties from Oxford University Press and Johns Hopkins University Press; Ms Myers and Dr Alvir are employees and stockholders of Pfizer Inc; Dr Gray was an employee and stockholder of Pfizer Inc at the time of analysis.

Objective: To compare differences in health care resource utilization (HcRU) over time between Medicare beneficiaries with and without Parkinson disease (PD)., Methods: This retrospective observational study used the Chronic Conditions Data Warehouse (5% Medicare sample) between 2005 and 2015. In a propensity score-matched (age, sex, race, and comorbidity adjusted) sample of beneficiaries with and without PD, we examined all-cause HcRU due to inpatient admissions, emergency department (ED) admissions, skilled nursing facility (SNF) admissions, health care provider encounters, neurologist visits, rehabilitation service visits, and non-PD medication fills. Relative to beneficiaries without PD, we reported adjusted incidence rate ratios (IRRs) and 95% confidence intervals (CIs) for beneficiaries with PD using generalized linear models with log link and negative binomial variance functions., Results: A total of 467,064 Medicare enrollees (unmatched sample) met the inclusion criteria. Of these, 3.3% had PD. In the matched sample and relative to beneficiaries without PD, beneficiaries with PD displayed higher rates of inpatient admissions (IRR 1.29, 95% CI 1.24-1.34), ED admissions (IRR 1.31, 95% CI 1.27-1.34), SNF admissions (IRR 2.00, 95% CI 1.92-2.09), health care provider encounters (IRR 1.18, 95% CI 1.16-1.20), neurologist visits (IRR 5.57, 95% CI 5.35-5.78), rehabilitation service visits (IRR 1.47, 95% CI 1.41-1.53), and non-PD medication fills (IRR 1.10, 95% CI 1.08-1.11) over time., Conclusion: These results reflect patterns of medical care among Medicare beneficiaries with PD. The findings can help clinicians, payers, and policy makers make evidence-based decisions for the allocation of scarce health care resources for PD management., Classification of Evidence: This study provides Class II evidence that Medicare beneficiaries with PD use more health care resources than matched controls without PD., (© 2021 American Academy of Neurology.)

The need for Health Economics and Outcomes Research (HEOR) has expanded globally, fueling demand for professionals trained in the discipline. By leveraging the expertise and perspectives of its members, the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) established a set of competencies for HEOR professionals. The resulting 41 competencies were organized into 13 topic domains that collectively comprise the ISPOR Health Economics and Outcomes Research Competencies Framework. In this article, we explain the collaborative process used by the ISPOR Institutional Council and Faculty Advisor Council to identify and validate the framework. This process entailed expertise from the council members combined with natural language processing to examine competencies included in ISPOR Career Center HEOR job postings, qualitative input from a focused Institutional Council-Faculty Advisor Council workgroup, and quantitative input from 3 surveys of mutually exclusive member groups: a general member survey to assess importance and relevance of each competency, a faculty member survey to assess the extent to which HEOR graduate degree programs cover each of the competencies, and a student member survey to assess exposure to each of the competencies. Organization of the competencies into topic domains was achieved by engaging the Education Council, which applied a taxonomy consistent with ISPOR's educational programming. The resulting ISPOR Health Economics and Outcomes Research Competencies Framework has the important potential of serving as a tool to guide academic curricula, fellowships, and continuing education programs, and assessment of job candidates. As the HEOR field advances, so do the job types and the breadth of topics in which professionals must demonstrate competence. Future work will entail revisiting the competencies to ensure their currency and comprehensiveness, and tailoring the framework according to major specialty areas., (Copyright © 2020 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

Introduction: We evaluated patient-level factors associated with the initial management of older adults diagnosed with follicular lymphoma (FL)., Materials and Methods: Using linked Surveillance, Epidemiology, and End Results-Medicare (SEER-Medicare) data; we identified 11,500 beneficiaries aged ≥ 66 years, diagnosed with FL between 2000 and 2013. A logistic regression model was used to estimate adjusted odds ratios (AORs) for factors associated with the receipt of active treatment versus watchful waiting (WW) as an initial management strategy. A multinomial logistic regression model was used to predict factors associated with receipt of specific active treatments, namely chemoimmunotherapy, rituximab monotherapy, chemotherapy, or radiation as compared with WW., Results: Overall, the initial management strategies adopted were WW (49%), chemoimmunotherapy (25%), radiation (10%), rituximab monotherapy (9%), and chemotherapy (7%). In reference to WW, grade III FL (AOR, 2.21; 95% confidence interval [CI], 1.99-2.46), increasing disease stage (Stage IV AOR, 1.80; 95% CI, 1.62-2.00), and use of preventive services (AOR, 1.18; 95% CI, 1.07-1.30) were associated with increased odds of active treatment receipt. Age > 80 years (AOR, 0.79; 95% CI, 0.71-0.87), Non-Hispanic African-American race (AOR, 0.64; 95% CI, 0.50-0.80), and state buy-in coverage (AOR, 0.81; 95% CI, 0.70-0.94) were associated with decreased odds of active treatment receipt. In reference to WW, the multinomial logistic regression model displayed differences in the receipt of rituximab-based therapies by age and comorbidity burden. Non-Hispanic African-American race and state buy-in coverage were associated with decreased odds of receiving rituximab-based therapies., Conclusion: The present analysis identifies disparities in the initial management of older adults with FL owing to race and socioeconomic status. Future research should examine implications for subsequent treatment and health outcomes., (Copyright © 2019 Elsevier Inc. All rights reserved.)

Aim: Characterize follicular lymphoma (FL) treatment patterns among elderly patients using a dataset with longer follow-up time. Materials & methods: Using the linked Surveillance, Epidemiology and End Results-Medicare data, we identified patients diagnosed with FL between 2000 and 2013 with claims data until 2014. We investigated the treatments received and assigned them to lines of treatment. Results: We identified 10,238 elderly patients. Over a 4.7-year median follow-up, 78% of the patients received at least first-line treatment. Fewer individuals received second-line (47%) and third-line (30%) treatments. RCHOP (rituximab, cyclophosphamide, doxorubicin, vincristine and prednisolone), RCVP (rituximab, cyclophosphamide, vincristine and prednisolone) and rituximab monotherapy were the most common treatment regimens. Conclusion: One in five elderly patients did not receive FL-directed therapy. The most common treatment regimens were limited to RCHOP, RCVP and rituximab monotherapy.

There is limited information on the cost burden associated with follicular lymphoma (FL) and how it compares to other non-Hodgkin lymphoma (NHL) subtypes. We examined the direct medical costs associated with FL and estimated the incremental 3-year cost of FL compared to other NHL subtypes. Using the linked Surveillance, Epidemiology and End Results-Medicare dataset, we identified 16,691 NHL patients aged 66 years or older who were diagnosed with NHL between 2007 and 2013. The mean 3-year cost among the full NHL sample was $120,120 (standard error (SE) 839). The mean 3-year cost per patient was $114,443 (SE 1738) for FL and $121,402 (SE 950) for non-FL subtypes. The incremental 3-year cost of FL compared to non-FL was US$-5458 (95% confidence interval: US$-9325 to US$-1590). Longitudinally, FL was less costly than other NHL subtypes in the first year only, and became more expensive in the second and third years.

Objectives: To evaluate the overall survival benefit associated with follicular lymphoma (FL)-directed therapy among patients diagnosed with FL at 80+ years., Patients and Methods: This retrospective cohort study utilized the linked Surveillance, Epidemiology and End Results-Medicare dataset to identify patients 80+ years, diagnosed with FL between 2000 and 2013. We identified FL-directed treatments based on published guidelines. We utilized a propensity-score matched sample to compare treated and untreated groups who had similar observed characteristics. We reported the median overall survival time and the 3-year restricted mean survival time (RMST) of the study groups as well as the hazard ratio (HR) of death associated with treatment receipt., Results: We identified 3705 older patients with FL (mean [SD] age, 84 [3.6] years). Over a median follow-up of 2.9 years, 68% of the sample received FL-directed therapy and the most common regimen was rituximab monotherapy (N = 768, 21%). The matched sample included 2306 patients. The median overall survival for the treated group was 4.31 years (95% confidence interval [CI], 4.00-4.61) compared to 2.86 years (95% CI, 2.59-3.16) for the untreated group. The 3-year RMST for the treated group was 2.36 years (95% CI, 2.30-2.41), while it was 2.05 years (95% CI, 1.98-2.11) for the untreated group. Treatment was associated with a 23% reduction in the hazards of death (HR: 0.77, 95% CI: 0.70-0.85; p < .001)., Conclusion: FL-directed therapy was associated with improved survival among patients diagnosed with FL at 80+ years. These findings can support treatment decision-making for individuals diagnosed with FL at older ages., (Copyright © 2019 Elsevier Inc. All rights reserved.)

Introduction: Men diagnosed with metastatic prostate cancer (PCa) are at increased risk for skeletal complications which are associated with significant morbidity and mortality. Although both the urologist and the medical oncologist play important roles in the management of patients with advanced PCa, there is limited information regarding their role in the context of skeletal complications. The current study investigated these relationships among newly diagnosed metastatic patients with PCa., Methods and Materials: This retrospective cohort study used Surveillance, Epidemiology and End Results cancer registry data for incident stage IV metastatic (M1) cases diagnosed from 2000 to 2007 with linked Medicare claims. Postdiagnosis urologist and medical oncologist visits were identified using billing codes. We considered skeletal-related events (SREs) that occurred after the urologist or medical oncologist visit. We used Cox proportional hazards models to examine the relationship between a physician visit and the timing of the first SRE with and without propensity-score matching to account for observable selection., Results: The sample included 5,572 patients with stage IV M1 prostate cancer. Seventy-six percent of the patients were non-Hispanic White, 16% were non-Hispanic African American, and 8% were of other races; 75% of patients saw a urologist (median time to first visit = 19 days) and 44% saw an oncologist (median = 80 days), whereas 41% experienced at least one SRE (median = 309 days). Covariate-adjusted Cox models showed a longer time to an SRE for patients with only a medical oncologist visit (hazard ratio [HR] = 0.53, 95% CI: 0.45-0.61), only a urologist visit (HR = 0.35, 95% CI: 0.31-0.39) or both a urologist and medical oncologist visit (HR = 0.34, 95% CI: 0.31-0.38), compared to individuals without these visits. Among men with a urologist visit, a medical oncologist visit was not associated with the time to the first SRE (HR = 0.97, 95% CI: 0.90-1.05). Among those without a urologist visit a medical oncologist visit was associated with a longer time to an SRE (HR = 0.54, 95% CI: 0.46-0.64). Results were comparable using propensity-score matched samples., Conclusion: Among men newly diagnosed with metastatic PCa, 4 of 10 patients experienced an SRE. Patients experienced a delay in skeletal complications when managed by a urologist or a medical oncologist compared to patients who did not see either specialist., (Copyright © 2018 Elsevier Inc. All rights reserved.)

Importance: Potential research participants may assume that randomized trials comparing new interventions with older interventions always hypothesize greater efficacy for the new intervention, as in superiority trials. However, antibiotic trials frequently use "noninferiority" hypotheses allowing a degree of inferior efficacy deemed "clinically acceptable" compared with an older effective drug, in exchange for nonefficacy benefits (eg, decreased adverse effects). Considering these different benefit-harm trade-offs, proper informed consent necessitates supplying different information on the purposes of superiority and noninferiority trials., Objective: To determine the degree to which the study purpose is explained to potential participants in randomized clinical trials of antibiotics and the degree to which study protocols justify their selection of noninferiority hypotheses and amount of "clinically acceptable" inferiority., Design and Setting: Cross-sectional analysis of study protocols, statistical analysis plans (SAPs), and informed consent forms (ICFs) from clinical study reports submitted to the European Medicines Agency. The ICFs were read by both methodologists and patient investigators., Main Outcomes and Measures: Protocols and SAPs were used as the reference standard to determine prespecified primary hypothesis and record rationale for selection of noninferiority hypotheses and noninferiority margins. This information was cross-referenced against ICFs to determine whether ICFs explained the study purpose., Results: We obtained trial documents from 78 randomized trials with prespecified efficacy hypotheses (6 superiority, 72 noninferiority) for 17 antibiotics conducted between 1991 and 2011 that enrolled 39 407 patients. Fifty were included in the ICF analysis. All ICFs contained sections describing study purpose; however, none consistently conveyed study hypothesis to both methodologists and patient investigators. Methodologists found that 1 of 50 conveyed a study purpose. Patient investigators found that 11 of 50 conveyed a study purpose, 7 accurately and 4 inaccurately compared with the reference standard. Seventy-one of 72 noninferiority trial protocols or SAPs provided no rationale for selection of noninferiority hypothesis. None provided a clinical rationale for the chosen amount of decreased efficacy., Conclusions and Relevance: Patients were not accurately informed of study purpose, which raises questions regarding the ethics of informed consent in antibiotic trials. Noninferiority and superiority trials entail different benefit-harm trade-offs that must be conveyed for ethical informed consent.