Residual Disease Burden After Neoadjuvant Therapy Among US Patients With High-Risk HER2-positive Early-Stage Breast Cancer: A Population Effectiveness Model.

BACKGROUND: Approximately half of patients with high-risk HER2-positive early-stage breast cancer (ESBC) do not have pathologic complete response (pCR) after neoadjuvant therapy. The residual burden of disease among this population has not been previously quantified. MATERIALS AND METHODS: We used decision-modeling techniques to simulate recurrence, progression from locoregional to distant cancer, breast cancer-related mortality, and mortality from other causes over a 10-year period in a hypothetical cohort. We derived progression probabilities primarily from the KATHERINE trial of T-DM1 (ado-trastuzumab emtansine) and mortality outcomes from the published literature. Modeled outcomes included recurrences, breast cancer deaths, deaths from other causes, direct medical costs, and costs due to lost productivity. To estimate the residual disease burden, we compared outcomes from a cohort of patients treated with T-DM1 versus a hypothetical cohort with no disease recurrence. RESULTS: We estimated that 9,300 people would experience incident high-risk HER2-positive ESBC in the United States in 2021 based on cancer surveillance databases, clinical trial data, and expert opinion. We estimated that, in this group, 2,118 would experience disease recurrence, including 1,576 distant recurrences, and 1,358 would experience breast cancer deaths. This residual disease burden resulted in 6,435 life-years lost versus the recurrence-free cohort, and healthcare-related costs totaling $644 million, primarily associated with treating distant cancers. CONCLUSION: Patients with HER2-positive ESBC who do not achieve pCR after neoadjuvant therapy are at ongoing risk of recurrence despite the effectiveness of neoadjuvant treatment. There is substantial clinical and economic value in further reducing the residual disease burden in this population.

Enhancing Health Equity Through Cancer Health Economics Research.

Cancer displays some of the largest health-equity concerns of all diseases. This paper draws attention to how health economics research can assess strategies to reduce or even eliminate health disparities and provides pivotal examples of existing research as well as areas for future contributions. The paper also highlights critical data limitations that currently restrain the impact health economics research could have. We then explore new areas of inquiry where economic research is sparse but could have an important impact on health equity, particularly in topics involving Medicare and Medicaid policies that expand reimbursement and generosity of coverage. Health economics studies are notably absent from policies and practices surrounding clinical trials, representing an opportunity for future research. We urge health economics researchers to consider experiments, interventions, and assessments through primary data collection; we further encourage the formulation of multidisciplinary teams to ensure that health economics skills are well melded with other areas of expertise. These teams are needed to maximize novelty and rigor of evidence. As policies are promulgated to address disparities in cancer, involvement of economics in a multidisciplinary context can help ensure that these policies do not have unintended impacts that may deepen inequities.

Financial Toxicity, Symptom Burden, Illness Perceptions, and Communication Confidence in Cancer Clinical Trial Participants.

PURPOSE: Cancer clinical trial (CCT) participants are at risk for experiencing adverse associations from financial toxicity, but these remain understudied. METHODS: From July 2015 to July 2017, we prospectively enrolled CCT participants referred for financial assistance and a group of patients matched by age, sex, cancer type, trial, and trial phase. We assessed financial burden of cancer care, cost concerns about CCTs, physical (Edmonton Symptom Assessment Scale [ESAS]) and psychologic (Patient Health Questionnaire-4 [PHQ-4]) symptoms, illness perceptions (Brief Illness Perception Questionnaire), and communication confidence (Perceived Efficacy in Patient-Physician Interactions). Adjusting for age, sex, race, performance status, marital status, income, insurance, and disease status, we examined associations of financial burden and cost concerns with patients' symptoms, illness perceptions, and communication confidence. RESULTS: Of 198 patients, 112 (56.6%) reported financial burden and 82 (41.4%) reported cost concerns. Higher ESAS-total (odds ratio [OR] = 1.03; 95% CI, 1.01 to 1.06; P = .001), PHQ-4 depression (OR = 1.58; 95% CI, 1.20 to 2.08; P < .001), PHQ-4 anxiety (OR = 1.26; 95% CI, 1.02 to 1.55; P = .025), and more negative illness perceptions (OR = 1.04; 95% CI, 1.00 to 1.07; P = .029) were associated with financial burden, but not communication confidence (OR = 0.98; 95% CI, 0.02 to 1.05; P = .587). Higher ESAS-total (OR = 1.03; 95% CI, 1.01 to 1.05; P = .004), PHQ-4 depression (OR = 1.36; 95% CI, 1.08 to 1.71; P = .03), PHQ-4 anxiety (OR = 1.26; 95% CI, 1.03 to 1.53; P = .018), more negative illness perceptions (OR = 1.06; 95% CI, 1.02 to 1.10; P = .001), and decreased communication confidence (OR = 0.93; 95% CI, 0.86 to 1.00; P = .029) were associated with cost concerns. CONCLUSION: In this study of CCT participants, greater symptom burden, more negative illness perceptions, and lower communication confidence were associated with financial toxicity, underscoring the importance of addressing these issues when seeking to alleviate adverse associations of financial toxicity.

Developing a Virtual Equity Hub: Adapting the Tumor Board Model for Equity in Cancer Care.

We define cancer equity as all people having as the same opportunity for cancer prevention, treatment, and survivorship care. However, marginalized populations continue to experience avoidable and unjust disparities in cancer care, access to clinical trials, and cancer survival. Racial and ethnic minorities, and individuals with low socioeconomic status, Medicaid insurance, limited health literacy, disabilities, and mental health disorders are more likely to experience delays to cancer diagnosis and less likely to receive guideline-concordant cancer care. These disparities are impacted by the social determinants of health including structural discrimination, racism, poverty, and inequities in access to healthcare and clinical trials. There is an urgent need to develop and adapt evidence-based interventions in collaboration with community partners that have potential to address the social determinants of health and build capacity for cancer care for underserved populations. We established the Virtual Equity Hub by developing a collaborative network connecting a comprehensive cancer center, academic safety net hospital, and community health centers and affiliates. The Virtual Equity Hub utilizes a virtual tumor board, an evidence-based approach that increases access to multi-specialty cancer care and oncology subspecialty expertise. We adapted the tumor board model by engaging person-centered teams of multi-disciplinary specialists across health systems, addressing the social determinants of health, and applying community-based research principles with a focus on populations with poor cancer survival. The virtual tumor board included monthly videoconferences, case discussion, sharing of expertise, and a focus on addressing barriers to care and trial participation. Specifically, we piloted virtual tumor boards for breast oncology, neuro-oncology, and individuals with cancer and serious mental illness. The Virtual Equity Hub demonstrated promise at building capacity for clinicians to care for patients with complex needs and addressing barriers to care. Research is needed to measure the impact, reach, and sustainability of virtual equity models for patients with cancer.

Assessment of patient navigation programs for breast cancer patients across the city of Boston.

PURPOSE: Healthcare systems contribute to disparities in breast cancer outcomes. Patient navigation is a widely cited system-based approach to improve outcomes among populations at risk for delays in care. Patient navigation programs exist in all major Boston hospitals, yet disparities in outcomes persist. The objective of this study was to conduct a baseline assessment of navigation processes at six Boston hospitals that provide breast cancer care in preparation for an implementation trial of standardized navigation across the city. METHODS: We conducted a mixed methods study in six hospitals that provide treatment to breast cancer patients in Boston. We administered a web-based survey to clinical champions (n = 7) across six sites to collect information about the structure of navigation programs. We then conducted in-person workflow assessments at each site using a semi-structured interview guide to understand site-specific implementation processes for patient navigation programs. The target population included administrators, supervisors, and patient navigators who provided breast cancer treatment-focused care. RESULTS: All sites offered patient navigation services to their patients undergoing treatment for breast cancer. We identified wide heterogeneity in terms of how programs were funded/resourced, which patients were targeted for navigation, the type of services provided, and the continuity of those services relative to the patient's cancer treatment. CONCLUSIONS: The operationalization of patient navigation varies widely across hospitals especially in relation to three core principles in patient navigation: providing patient support across the care continuum, targeting services to those patients most likely to experience delays in care, and systematically screening for and addressing patients' health-related social needs. Gaps in navigation across the care continuum present opportunities for intervention. TRIAL REGISTRATION: Clinical Trial Registration Number NCT03514433, 5/2/2018.

Disparities in phase 1 cancer clinical trial enrollment.

BACKGROUND: Phase 1 trials are increasingly important in the molecularly driven era of oncology, but few studies have examined phase 1 participation disparities. The authors of this study investigated factors associated with phase 1 versus phase 2/3 trial enrollment. METHODS: They authors conducted a cross-sectional study using serial samples of patients age ≥18 years enrolling on cancer trials from October 2011 to November 2014 at an academic cancer center. They used univariable and multivariable logistic regression models to analyze sociodemographic and clinical associations with phase 1 versus phase 2/3 trial enrollment. RESULTS: Among 3103 patients enrolled in cancer trials, 2657 unique patients participated in phase 1/2/3 trials. For patients enrolled in phase 1 (n = 1401) versus phase 2/3 (n = 1256) trials, we found no significant differences by age, insurance status, marital status, and income. Overall, 1216 (93%) White, 72 (6%) Asian, and 21 (2%) Black patients enrolled on phase 1 trials, whereas 1068 (93%) White, 40 (3%) Asian, and 43 (4%) Black patients enrolled on phase 2/3 trials. Adjusting for age, sex, race, ethnicity, insurance status, marital status, income, cancer type, disease status, travel distance, and trial year, compared with White patients, Black patients had lower phase 1 enrollment (odds ratio [OR], 0.46; 95% confidence interval [CI], 0.25-0.82), as did Hispanic/Latino (OR, 0.25; 95% CI, 0.08-0.79) and male patients (OR, 0.77; 95% CI, 0.62-0.94). Asian patients had higher phase 1 enrollment (OR, 1.38; 95% CI, 0.88-2.16). CONCLUSIONS: Disparities in phase 1 versus phase 2/3 cancer clinical trial enrollment underscore the urgent need for interventions addressing inequities in early-phase trial participation. LAY SUMMARY: Phase 1 trials are of increasing importance in oncology. The authors of the study analyzed all patients enrolling on cancer clinical trials at a large academic cancer center from October 2011 to November 2014. Among the 2657 trial participants, when age, sex, race, ethnicity, insurance status, marital status, income, cancer type, disease status, travel distance, and trial year were taken into account, Black, Hispanic/Latino, and male patients were less likely to enroll on phase 1 trials versus phase 2/3 trials. These findings suggest a need for targeted interventions to improve access to and education about phase 1 trials for Black and Hispanic/Latino patients.

Implementation of a Brief Screening Tool to Identify Needs of Breast Cancer Survivors.

BACKGROUND: Innovation in health care delivery is needed to improve care for cancer survivors. We report our experience with adapting screening questions from the National Comprehensive Cancer Network (NCCN) guideline to evaluate the needs of breast cancer survivors. MATERIALS AND METHODS: We adapted the NCCN-recommended screening questions into a plain language, self-administered 1-page intake questionnaire. The tool was administered to a convenience sample of female breast cancer survivors at follow-up oncology and primary care visits. Domains included symptoms, lifestyle concerns, and financial issues. Frequency of concerns was assessed as "never," "rarely," "sometimes," "very frequently," and "always." We evaluated feasibility and utility of administration and the prevalence and frequency of patient-reported concerns. RESULTS: The questionnaire was highly acceptable to patients and enhanced visits for clinicians. Clinicians reported that it led to discussion of issues that may not otherwise be addressed in the visit and did not find it burdensome. The most commonly endorsed patient concerns were desire to improve fitness or nutrition, worry about cancer recurrence, and insomnia. A majority also reported feeling anxiety and aches or pains in joints or extremities. Several issues known to be underreported in clinic visits were frequently endorsed, included sexual dissatisfaction and memory impairments. Clinicians suggested incorporating the tool into the electronic health record to increase utility and awareness. CONCLUSION: Screening for individual needs among breast cancer survivors is feasible, efficient, and may identify prevalent issues that otherwise can be missed in routine survivorship care.

Standardized activities for lay patient navigators in breast cancer care: Recommendations from a citywide implementation study.

BACKGROUND: There is a need for guidelines on patient navigation activities to promote both the quality of patient navigation and the standards of reimbursement for these services because a lack of reimbursement is a major barrier to the implementation, maintenance, and sustainability of these programs. METHODS: A broad community-based participatory research process was used to identify the needs of patients for navigation. A panel of stakeholders of clinical providers was convened to identify specific activities for navigators to address the needs of patients and providers with the explicit goal of reducing delays in the initiation of cancer treatment and improving adherence to the care plan. RESULTS: Specific activities were identified that could be generalized to all patient navigation programs for care during active cancer management to address the needs of vulnerable communities. CONCLUSIONS: Oncology programs that seek to implement lay patient navigation may benefit from the adoption of these activities for quality monitoring. Such activities are necessary as we consider reimbursement strategies for navigators without clinical training or licensure.

Addressing the Financial Burden of Cancer Clinical Trial Participation: Longitudinal Effects of an Equity Intervention.

BACKGROUND: The financial burden experienced by patients with cancer represents a barrier to clinical trial participation, and interventions targeting patients' financial concerns are needed. We sought to assess the impact of an equity intervention on clinical trial patients' financial burden. MATERIALS AND METHODS: We developed an equity intervention to reimburse nonclinical expenses related to trials (e.g., travel and lodging). From July 2015 to July 2017, we surveyed intervention and comparison patients matched by age, sex, cancer type, specific trial, and trial phase. We longitudinally assessed financial burden (e.g., trial-related travel and lodging cost concerns, financial wellbeing [FWB] with the COmprehensive Score for financial Toxicity [COST] measure) at baseline, day 45, and day 90. We used longitudinal models to assess intervention effects over time. RESULTS: Among 260 participants, intervention patients were more likely than comparison patients to have incomes under $60,000 (52% vs. 24%, p < .001) and to report travel-related (41.0% vs. 6.8%, p < 0.001) and lodging-related (32.5% vs. 2.0%, p < .001) cost concerns at baseline. Intervention patients were more likely to report travel to appointments as their most significant financial concern (24.0% vs. 7.0%, p = .001), and they had worse FWB than comparison patients (COST score: 15.32 vs. 23.88, p < .001). Over time, intervention patients experienced greater improvements in their travel-related (-10.0% vs. +1.2%, p = .010) and lodging-related (-3.9% vs. +4.0%, p = .003) cost concerns. Improvements in patients reporting travel to appointments as their most significant financial concern and COST scores were not statistically significant. CONCLUSION: Cancer clinical trial participants may experience substantial financial issues, and this equity intervention demonstrates encouraging results for addressing these patients' longitudinal financial burden. IMPLICATIONS FOR PRACTICE: Clinical trials are critical for developing novel therapies for patients with cancer, yet financial barriers may discourage some patients from participating in cancer clinical trials. This study found that patients who received financial assistance from an equity intervention experienced significant improvements over time in their concerns about the cost of travel and lodging associated with clinical trials compared with comparison patients who did not receive financial assistance from the equity intervention. Among cancer clinical trial participants, an equity intervention shows potential for addressing patients' concerns regarding clinical trial-related travel and lodging expenses.

Addressing Financial Barriers to Patient Participation in Clinical Trials: ASCO Policy Statement.

Research conducted through clinical trials is essential for evaluating new treatment modalities, establishing new standards of cancer care, and ultimately improving and prolonging the lives of patients with cancer. However, participation in trials has been low, and this is attributable to various factors including patient financial barriers. Such financial barriers include the rising cost of cancer care; a lack of transparency in coverage policy; and the perception of ethical, compliance, or institutional impediments to patient financial support. ASCO convened a roundtable discussion with a variety of stakeholders to define the scope of the problem, as well as to identify clinical practice and policy solutions applicable at the institutional and system-wide levels. This statement summarizes key discussions from the ASCO Roundtable, as well as findings from the literature, and provides ASCO's recommendations for overcoming financial barriers that may otherwise prevent participation in clinical trials. These recommendations broadly address the following key areas: (1) improving the policy environment for coverage of clinical trials; (2) facilitating transparency among providers, patients, and payers for trial-related out-of-pocket costs; (3) refuting the specter of inducement to enable targeted financial support for patients; and (4) improving the available data on costs of cancer clinical trials.

Patterns in Health Care Access and Affordability Among Cancer Survivors During Implementation of the Affordable Care Act.

Importance: Cancer survivors face ongoing health issues and need access to affordable health care, yet studies examining health care access and affordability in this population are lacking. Objectives: To evaluate health care access and affordability in a national sample of cancer survivors compared with adults without cancer and to evaluate temporal trends during implementation of the Affordable Care Act. Design, Setting, and Participants: We used data from the National Health Interview Survey from 2010 through 2016 to conduct a population-based study of 30 364 participants aged 18 years or older. We grouped participants as cancer survivors (n = 15 182) and those with no reported history of cancer, whom we refer to as control respondents (n = 15 182), matched on age. We excluded individuals reporting a cancer diagnosis prior to age 18 years and those with nonmelanoma skin cancers. Main Outcomes and Measures: We compared issues with health care access (eg, delayed or forgone care) and affordability (eg, unable to afford medications or health care services) between cancer survivors and control respondents. We also explored trends over time in the proportion of cancer survivors reporting these difficulties. Results: Of the 30 364 participants, 18 356 (57.4%) were women. The mean (SD) age was 63.5 (23.5) years. Cancer survivors were more likely to be insured (14 412 [94.8%] vs 13 978 [92.2%], P < .001) and to have government-sponsored insurance (7266 [44.3%] vs 6513 [38.8%], P < .001) compared with control respondents. In multivariable models, cancer survivors were more likely than control respondents to report delayed care (odds ratio [OR], 1.38; 95% CI, 1.16-1.63), forgone medical care (OR, 1.76; 95% CI, 1.45-2.12), and/or inability to afford medications (OR, 1.77; 95% CI, 1.46-2.14) and health care services (OR, 1.46; 95% CI, 1.27-1.68) (P < .001 for all). From 2010 to 2016, the proportion of survivors reporting delayed medical care decreased each year (B = 0.47; P = .047), and the proportion of those needing and not getting medical care also decreased each year (B = 0.35; P = .04). In addition, the proportion of cancer survivors who reported being unable to afford prescription medication decreased each year (B=0.66; P = .004) and the proportion of those unable to afford at least 1 of 6 services decreased each year (B = 0.51; P = .01). Conclusions and Relevance: Despite higher rates of insurance coverage, cancer survivors reported greater difficulties accessing and affording health care compared with adults without cancer. Importantly, the proportion of survivors reporting these issues continued a downward trend throughout our observation period in the years following the implementation of the Affordable Care Act. Our findings suggest incremental improvement in health care access and affordability after recent health care reform and provide an important benchmark as additional changes are likely to occur in the coming years.

Financial Burden of Cancer Clinical Trial Participation and the Impact of a Cancer Care Equity Program.

INTRODUCTION: Cancer clinical trial (CT) participation rates are low and financial barriers likely play a role. We implemented a cancer care equity program (CCEP) to address financial burden associated with trial participation. We sought to examine the impact of the CCEP on CT enrollment and to assess barriers to participation. METHODS: We used an interrupted time series design to determine trends in CT enrollment before and after CCEP implementation. Linear regression models compared trial enrollment before and after the CCEP. We also compared patient characteristics before and after the CCEP and between CCEP and non-CCEP participants. We surveyed CCEP and non-CCEP participants to compare pre-enrollment financial barriers. RESULTS: After accounting for increased trial availability and the trends in accrual for prior years, we found that enrollment increased after CCEP implementation (18.97 participants per month greater than expected; p < .001). A greater proportion of CCEP participants were younger, female, in phase I trials, lived farther away, had lower incomes, and had metastatic disease. Of 87 participants who completed the financial barriers survey, 49 CCEP and 38 matched, non-CCEP participants responded (63% response rate). CCEP participants were more likely to report concerns regarding finances (56% vs. 11%), medical costs (47% vs. 14%), travel (69% vs. 11%), lodging (60% vs. 9%), and insurance coverage (43% vs. 14%) related to trial participation (all p < .01). CONCLUSION: CT participation increased following implementation of the CCEP and the program enrolled patients experiencing greater financial burden. These findings highlight the need to address the financial burden associated with CT participation. IMPLICATIONS FOR PRACTICE: Financial barriers likely discourage patients from participating in clinical trials. Implementation of a cancer care equity program (CCEP) seeking to reduce financial barriers by assisting with travel and lodging costs was associated with increased trial accrual. The CCEP provided assistance to patients particularly in need, including those living farther away, those with lower incomes, and those reporting financial barriers related to trial participation. These findings suggest that financial concerns represent a major barrier to patient participation in clinical trials and underscore the importance of efforts to address these concerns.

Value of cancer care: ethical considerations for the practicing oncologist.

The value of cancer care has emerged at the center of a national discourse on fiscal responsibility and resource allocation. The cost of cancer care is rising at a higher pace than any other area of health care. As we struggle to address this unsustainable rise in cancer expenditures, oncology providers are forced to examine our practice patterns and our contributions to the overall health care cost burden. This article provides an oncologist-centered examination of our duties to individual patients and how they may seem at odds with our duties to society. It also discusses how oncology providers can do their part to contain health care costs while honoring their professional obligation to do their best for each patient.