Transferring care to enhance access to early-phase cancer clinical trials: Protocol to evaluate a novel program.

Involving diverse populations in early-phase (phase I and II) cancer clinical trials is critical to informed therapeutic development. However, given the growing costs and complexities of early-phase trials, trial activation and enrollment barriers may be greatest for these studies at healthcare facilities that provide care to the most diverse patient groups, including those in historically underserved communities (e.g., safety-net healthcare systems). To promote diverse and equitable access to early-phase cancer clinical trials, we are implementing a novel program for the transfer of care to enhance access to early-phase cancer clinical trials. We will then perform a mixed-methods study to determine perceptions and impact of the program. Specifically, we will screen, recruit, and enroll diverse patients from an urban, integrated safety-net healthcare system to open and active early-phase clinical trials being conducted in a university-based cancer center. To evaluate this novel program, we will: (1) determine program impact and efficiency; and (2) determine stakeholder experience with and perceptions of the program. To achieve these goals, we will conduct preliminary cost analyses of the program. We will also conduct surveys and interviews with patients and caregivers to elucidate program impact, challenges, and areas for improvement. We hypothesize that broadening access to early-phase cancer trials conducted at experienced centers may improve equity and diversity. In turn, such efforts may enhance the efficiency and generalizability of cancer clinical research.

Odds of Medical Comorbidities in Essential Tremor: Retrospective Analysis of a Large Claims Database in the United States.

INTRODUCTION: Essential tremor (ET) is the most common tremor disorder, estimated to affect 7 million individuals in the USA. There is little empirical evidence on comorbidities among this population beyond higher prevalence of brain-related and stress-related disorders. This study aims to examine differences in the prevalence of the 31 Elixhauser comorbidities among ET patients compared to statistically similar control patients. METHODS: An extract from Optum's de-identified Clinformatics® Data Mart Database (CDM) from 2018 to 2019 of adults aged 40-80 years with at least one claim with an ET diagnosis was propensity score matched to controls. Logistic regression was used to generate doubly robust adjusted odds ratios for each of the 31 Elixhauser comorbidities. RESULTS: In these analyses, ET patients had significantly greater adjusted odds of depression, alcohol abuse, and other neurological disorders, as well as chronic pulmonary disease, renal failure, hyperthyroidism, and cardiac arrhythmias relative to controls. They also had lower odds of uncomplicated diabetes, congestive heart failure, metastatic cancer, paralysis, peripheral vascular disease, and fluid and electrolyte disorders. CONCLUSION: A number of recent studies, including our own, suggest that psychiatric, neurologic, and stress-related disorders may be more prevalent among ET patients than controls. Additional differences in the prevalence of a range of medical comorbidities have also been variably reported across studies, suggesting that some combination of these might be more prevalent. Further studies would be of value in sorting through these associations.

Cost-Effectiveness Analysis of CAR T-Cell Therapies vs Antibody Drug Conjugates for Patients with Advanced Multiple Myeloma.

OBJECTIVES: Among advanced multiple myeloma (MM) patients, B-cell maturation antigen (BCMA) specific targets like Belantamab Mafodotin (belamaf) and CAR T-cell therapies have been shown to improve clinical outcomes, but at significant costs. To compare the expected costs per quality-adjusted life years (QALYs) gained among a hypothetical cohort of triple refractory MM patients treated with one of three BCMA-directed therapies: (1) idecabtagene vicleucel (ide-cel), (2) ciltacabtagene autoleucel (cilta-cel), and (3) belamaf for up to 20 months. METHODS: In this cost-effectiveness analysis, we built a Monte Carlo Markov Chain microsimulation model using estimates and parameters from the evidence on MM treatment for 10 000 hypothetical patients between the ages for 40 and 80. We assigned expected years of life remaining and made varying assumptions about survival beyond 5 years. RESULTS: We predicted total cost of treatment for CAR-T therapy to be six times greater than for belamaf, but the QALYs gained from treatment are 6 to 8 times greater. Ide-cel was weakly dominated by cilta-cel and our base-case incremental cost effectiveness ratio (ICER) comparing cilta-cel with belamaf was $109,497 per QALY gained, averaging $123,618 in probabilistic sensitivity analyses. CONCLUSIONS: These findings hinge on the assumption of longer-term survival but suggest that the use of CAR-T therapy is approaching standard ICER thresholds.

Enhancing access to and diversity in cancer clinical trials through a financial reimbursement program: Protocol to evaluate a novel program.

As clinical trials have become more complex, with increasing numbers of required procedures and clinic visits, gaining access to promising new treatments has become even more challenging for many individuals. To address these barriers, we implemented a financial reimbursement and outreach program designed to increase the number and diversity of participants in cancer clinical trials at centers in Dallas, Houston, and Philadelphia. As endorsed by U.S. Food and Drug Administration (FDA) and the Texas and Pennsylvania State Legislatures, the program provides financial reimbursement for non-clinical costs (e.g., travel, lodging) to patients on cancer clinical trials with household income up to 700% the Federal poverty rate. The research study described here, centered at the Dallas site, evaluates program impact by assessing (1) numbers and diversity of patients enrolled to cancer clinical trials before and after program implementation; (2) characteristics of patients offered participation in the program who do versus do not enroll; (3) characteristics of patients enrolled in the program who do versus do not complete the reimbursement process. To evaluate perceived barriers and facilitators of program participation, we will conduct semi-structured interviews and administer the Comprehensive Score for Financial Toxicity Patient Reported Outcome Measure (COST PROM) and the Short Assessment of Health Literacy (SAHL). This program will examine how reimbursement of non-clinical costs can improve access to cancer clinical trials, with the eventual goal of increasing trial enrollment, diversity, representativeness, and generalizability.

Brief Cost Analysis of Surgical Personal Protective Equipment During the COVID-19 Pandemic.

OBJECTIVES: This study aimed to compare the costs incurred and saved from universal use of N95 respirators with surgical masks for operating room providers in the United States during the COVID-19 pandemic. METHODS: We built a decision analytic model to compare direct medical costs of healthcare workers (HCWs) infected with COVID-19 during operating room procedures from expected transmission when using an N95 respirator relative to a surgical mask. We also examined quarantine costs. RESULTS: Results varied depending upon prevalence and false-negative rates of tests, but if N95 respirators reduce transmission by 2.8%, prevalence is at 1%, and testing yields 20% false negatives, providers should be willing to pay an additional $0.64 per HCW for the additional protection. Under this scenario, approximately 11 COVID-19 cases would be averted among HCWs per day. CONCLUSIONS: Potential savings depend on disease prevalence, rate of asymptomatic patients with COVID-19, accuracy of testing, the marginal cost of respirators, and the quarantine period. We provide a range of calculations to show under which conditions N95 respirators are cost saving.

Cost Effectiveness of Mailed Outreach Programs for Colorectal Cancer Screening: Analysis of a Pragmatic, Randomized Trial.

BACKGROUND & AIMS: Clinical guidelines for colorectal cancer (CRC) screening suggest use of either stool-based tests or colonoscopy - modalities that differ in recommended screening intervals, adherence, and costs. We know little about the long-term cost differences in population-health outreach strategies to promote these strategies. METHODS: We conducted a cost-effectiveness analysis to compare 2 mailed outreach strategies to increase CRC screening from a pragmatic, randomized clinical trial: mailed fecal immunochemical test (FIT) kits vs invitations to complete a screening colonoscopy. We built a 10-year Markov chain Monte Carlo microsimulation model to account for differences in screening intervals, adherence, and costs. RESULTS: Mailed FIT kits had a lower 10-year average per-person cost of screening relative to colonoscopy invitations ($1139 vs $1725) but with 10.89 fewer months of compliance and 60 fewer advanced neoplasia detected (37 advanced adenomas and 23 CRC). Incremental cost effectiveness ratios for colonoscopy invitations compared with mailed FIT kits were $55.23, $15.84, and $25.48 per additional covered month, advanced adenoma, and CRC, respectively. Although FIT was the preferred strategy at low willingness-to-pay thresholds, the 2 strategies were equal at a willingness-to-pay threshold of $41.31 per covered month gained. CONCLUSION: Mailed FIT or colonoscopy invitations are both options to improve CRC screening completion and advanced neoplasia detection, and the choice of outreach strategy may differ by a health system's willingness-to-pay threshold. Mailed FIT kits are less expensive than colonoscopy invitations but result in fewer months of screening compliance and advanced neoplasia detected.

Colorectal Cancer Screening and State Health Insurance Mandates.

Colorectal cancer (CRC) is the third most deadly cancer in the USA. CRC screening is the most effective way to prevent CRC death, but compliance with recommended screenings is very low. In this study, we investigate whether CRC screening behavior changed under state mandated private insurance coverage of CRC screening in a sample of insured adults from the 1997 to 2008 Behavioral Risk Factor Surveillance Survey (BRFSS). We present difference-in-difference-in-differences (DDD) estimates that compare insured individuals age 51 to 64 to Medicare age-eligible individuals (ages 66 to 75) in mandate and non-mandate states over time. Our DDD estimates suggest endoscopic screening among men increased by 2 to 3 percentage points under mandated coverage among 51 to 64 year olds relative to their Medicare age-eligible counterparts. We find no clear evidence of changes in screening behavior among women. DD estimates suggest no evidence of a mandate effect on either type of CRC screening for men or women.

Affordable Care Act Provision Lowered Out-Of-Pocket Cost And Increased Colonoscopy Rates Among Men In Medicare.

Colorectal cancer screening is one of the few cancer screenings with an "A" rating from the US Preventive Services Task Force, meaning that the procedure confers a substantial health benefit. However, 40 percent of people who should receive colorectal cancer screenings do not receive them. Colonoscopies are the most thorough method of screening because they allow physicians to view the entire length of the colon and remove polyps as needed. Billing methods that distinguish between screening and therapeutic procedures have kept expected colonoscopy costs high. However, the Affordable Care Act partially closed the so-called colonoscopy loophole and reduced expected out-of-pocket expenses for all Medicare beneficiaries. Using data from the Behavioral Risk Factor Surveillance System, we found that annual colonoscopy rates among men ages 66-75 increased significantly (by 4.0 percentage points) after the Affordable Care Act policy change, and we found some evidence of even larger increases among socioeconomically disadvantaged men. We found no significant increases among women, a result that may be explained by health behavior and other factors and that requires further study. Our research indicates that cost may be an important barrier to colorectal cancer screening, at least among men, and that making further policy changes to close remaining loopholes may improve screening rates.

Mandated coverage of preventive care and reduction in disparities: evidence from colorectal cancer screening.

OBJECTIVES: We identified correlates of racial/ethnic disparities in colorectal cancer screening and changes in disparities under state-mandated insurance coverage. METHODS: Using Behavioral Risk Factor Surveillance System data, we estimated a Fairlie decomposition in the insured population aged 50 to 64 years and a regression-adjusted difference-in-difference-in-difference model of changes in screening attributable to mandates. RESULTS: Under mandated coverage, blood stool test (BST) rates increased among Black, Asian, and Native American men, but rates among Whites also increased, so disparities did not change. Endoscopic screening rates increased by 10 percentage points for Hispanic men and 3 percentage points for non-Hispanic men. BST rates fell among Hispanic relative to non-Hispanic men. We found no changes for women. However, endoscopic screening rates improved among lower income individuals across all races and ethnicities. CONCLUSIONS: Mandates were associated with a reduction in endoscopic screening disparities only for Hispanic men but may indirectly reduce racial/ethnic disparities by increasing rates among lower income individuals. Findings imply that systematic differences in insurance coverage, or health plan fragmentation, likely existed without mandates. These findings underscore the need to research disparities within insured populations.

Environmental influences on young adult weight gain: evidence from a natural experiment.

OBJECTIVES: This study investigated the importance of environmental influences in explaining weight gain and related behaviors among freshman college students. METHODS: We exploited a natural experiment that takes place on most college campuses in the United States--randomized dormitory assignments. We estimated the effects of living in dormitories with varying physical environment characteristics on weight gain and related behaviors (daily number of meals and snacks, weekly frequency of exercise) among randomly assigned freshman students. RESULTS: We found strong evidence linking weight and related behaviors to individual dormitories, as well as to specific characteristics of the dormitories. On average, students assigned to dormitories with on-site dining halls gained more weight and exhibited more behaviors consistent with weight gain during the freshman year as compared with students not assigned to such dormitories. Females in such dormitories weighed .85 kg (p = .03) more and exercised 1.43 (p < .01) times fewer; males consumed .22 (p = .02) more meals and .38 (p = .01) more snacks. For female students, closer proximity of the dormitory to a campus gym led to more frequent exercise (.54, p = .03), whereas living closer to central campus reduced exercise (-.97, p = .01). CONCLUSIONS: Using a natural experiment to deal with the potential endogeneity of the living environment, this study found that the physical environment affects both students' weight changes and weight-related behaviors.