Remote Monitoring and Data Collection for Decentralized Clinical Trials.

Importance: Less than 5% of patients with cancer enroll in a clinical trial, partly due to financial and logistic burdens, especially among underserved populations. The COVID-19 pandemic marked a substantial shift in the adoption of decentralized trial operations by pharmaceutical companies. Objective: To assess the current global state of adoption of decentralized trial technologies, understand factors that may be driving or preventing adoption, and highlight aspirations and direction for industry to enable more patient-centric trials. Design, Setting, and Participants: The Bloomberg New Economy International Cancer Coalition, composed of patient advocacy, industry, government regulator, and academic medical center representatives, developed a survey directed to global biopharmaceutical companies of the coalition from October 1 through December 31, 2022, with a focus on registrational clinical trials. The data for this survey study were analyzed between January 1 and 31, 2023. Exposure: Adoption of decentralized clinical trial technologies. Main Outcomes and Measures: The survey measured (1) outcomes of different remote monitoring and data collection technologies on patient centricity, (2) adoption of these technologies in oncology and all therapeutic areas, and (3) barriers and facilitators to adoption using descriptive statistics. Results: All 8 invited coalition companies completed the survey, representing 33% of the oncology market by revenues in 2021. Across nearly all technologies, adoption in oncology trials lags that of all trials. In the current state, electronic diaries and electronic clinical outcome assessments are the most used technology, with a mean (SD) of 56% (19%) and 51% (29%) adoption for all trials and oncology trials, respectively, whereas visits within local physician networks is the least adopted at a mean (SD) of 12% (18%) and 7% (9%), respectively. Looking forward, the difference between the current and aspired adoption rate in 5 years for oncology is large, with respondents expecting a 40% or greater absolute adoption increase in 8 of the 11 technologies surveyed. Furthermore, digitally enabled recruitment, local imaging capabilities, and local physician networks were identified as technologies that could be most effective for improving patient centricity in the long term. Conclusions and Relevance: These findings may help to galvanize momentum toward greater adoption of enabling technologies to support a new paradigm of trials that are more accessible, less burdensome, and more inclusive.

Health Care Policy and Disparities in Health.

ABSTRACT: The United States has seen a 33% decline in age-adjusted cancer mortality since 1991. Despite this achievement, the United States has some of the greatest health disparities of any developed nation. US government policies are increasingly directed toward reducing health disparities and promoting health equity. These policies govern the conduct of research, cancer prevention, access, and payment for care. Although implementation of policies has played a significant role in the successes of cancer control, inconsistent implementation of policy has resulted in divergent outcomes; poorly designed or inadequately implemented policies have hindered progress in reducing cancer death rates and, in certain cases, exacerbated existing disparities. Examining policies affecting cancer control in the United States and realizing their unintended consequences are crucial in addressing cancer inequities.

Disparities in Breast Cancer Outcomes and How to Resolve Them.

There has been a 40% decline in breast cancer age-adjusted death rate since 1990. Black American women have not experienced as great a decline; indeed, the Black-White disparity in mortality in the United States is greater today than it has ever been. Certain states (areas of residence), however, do not see such dramatic differences in outcome by race. This latter finding suggests much more can be done to reduce disparities and prevent deaths. Interventions to get high-quality care (screening, diagnostics, and treatment) involve understanding the needs and concerns of the patient and addressing those needs and concerns. Patient navigators are 1 way to improve outcomes.

On the Potential for Optimizing Colorectal Screening Outcomes.

The development of colorectal cancer screening is a cancer control success. It is preventing thousands of deaths, but it has the potential of preventing thousands more. This can be achieved through offering all eligible patients high quality screening, diagnostics, and treatment. Let us educate and encourage colorectal screening among all average risk Americans beginning at 45. Let us not allow a recommendation to start at 45 to deemphasize screening those older persons who are most likely to benefit from colorectal cancer screening. See related article by Liu et al., p. 1701.

Cancer in sub-Saharan Africa: a Lancet Oncology Commission.

In sub-Saharan Africa (SSA), urgent action is needed to curb a growing crisis in cancer incidence and mortality. Without rapid interventions, data estimates show a major increase in cancer mortality from 520 348 in 2020 to about 1 million deaths per year by 2030. Here, we detail the state of cancer in SSA, recommend key actions on the basis of analysis, and highlight case studies and successful models that can be emulated, adapted, or improved across the region to reduce the growing cancer crises. Recommended actions begin with the need to develop or update national cancer control plans in each country. Plans must include childhood cancer plans, managing comorbidities such as HIV and malnutrition, a reliable and predictable supply of medication, and the provision of psychosocial, supportive, and palliative care. Plans should also engage traditional, complementary, and alternative medical practices employed by more than 80% of SSA populations and pathways to reduce missed diagnoses and late referrals. More substantial investment is needed in developing cancer registries and cancer diagnostics for core cancer tests. We show that investments in, and increased adoption of, some approaches used during the COVID-19 pandemic, such as hypofractionated radiotherapy and telehealth, can substantially increase access to cancer care in Africa, accelerate cancer prevention and control efforts, increase survival, and save billions of US dollars over the next decade. The involvement of African First Ladies in cancer prevention efforts represents one practical approach that should be amplified across SSA. Moreover, investments in workforce training are crucial to prevent millions of avoidable deaths by 2030. We present a framework that can be used to strategically plan cancer research enhancement in SSA, with investments in research that can produce a return on investment and help drive policy and effective collaborations. Expansion of universal health coverage to incorporate cancer into essential benefits packages is also vital. Implementation of the recommended actions in this Commission will be crucial for reducing the growing cancer crises in SSA and achieving political commitments to the UN Sustainable Development Goals to reduce premature mortality from non-communicable diseases by a third by 2030.

Inclusion of the US Preventive Services Task Force Recommendation for Mammography in State Comprehensive Cancer Control Plans in the US.

Importance: The recommendations for the age and frequency that women at average risk for breast cancer should undergo breast cancer mammography screening have been a matter of emotional, political, and scientific debate over the past decades. Multiple national organizations provide recommendations for breast cancer screening age and frequency. US Centers for Disease Control and Prevention (CDC) funding for state comprehensive cancer control (CCC) planning requires compliance with stated objectives for attaining goals. US Preventive Services Task Force (USPSTF) recommendations on cancer prevention and control are currently used to require coverage of prevention services. Objectives: To evaluate the consistency of state CCC plan objectives compared with the most current (2016) USPSTF recommendations for the age and frequency that individuals should undergo mammography screening and to make recommendations for improvement of state CCC plans. Design, Setting, and Participants: This cross-sectional study used a descriptive, point-in-time evaluation and was conducted from November 1, 2019, to June 30, 2021. In November 2019, the most recent CCC plans from 50 US states and the District of Columbia were downloaded from the CDC website. The recommended ages at which to begin and end mammography examinations and the frequency of mammography examinations were extracted from plan objectives. Main Outcomes and Measures: The recommendations found in CCC plan objectives regarding the ages at which to begin and end mammography examinations and the frequency of mammography examinations for women with average risk for breast cancer were compared with USPSTF recommendations. Results: Of the 51 CCC plans, 16 (31%) were consistent with all USPSTF recommendations for age and frequency that women at average risk should undergo mammography. Twenty-six plans (51%) were partially consistent with recommendations, and 9 plans (18%) were not consistent with any of the 3 guideline components. Conclusions and Relevance: Compared with the USPSTF recommendation, state CCC plans are not homogenous regarding the age and frequency that women at average risk for breast cancer should undergo mammography. This variation is partially due to differences in state-specific planning considerations and discretion, variations in recommendations among national organizations, and publication of plans prior to the most current USPSTF recommendation (2016). Specifying the concept that high-risk populations need different age and frequency of screening recommendations than the general population may reduce heterogeneity among plans.

Cost-Effectiveness of Systemic Treatments for Metastatic Castration-Sensitive Prostate Cancer: An Economic Evaluation Based on Network Meta-Analysis.

OBJECTIVES: To assess the cost-effectiveness of systemic treatments for metastatic castration-sensitive prostate cancer from the US healthcare sector perspective with a lifetime horizon. METHODS: We built a partitioned survival model based on a network meta-analysis of 7 clinical trials with 7287 patients aged 36 to 94 years between 2004 and 2018 to predict patient health trajectories by treatment. We tested parameter uncertainties with probabilistic sensitivity analyses. We estimated drug acquisition costs using the Federal Supply Schedule and adopted generic drug prices when available. We measured cost-effectiveness by an incremental cost-effectiveness ratio (ICER). RESULTS: The mean costs were approximately $392 000 with androgen deprivation therapy (ADT) alone and approximately $415 000, $464 000, $597 000, and $959 000 with docetaxel, abiraterone acetate, enzalutamide, and apalutamide, added to ADT, respectively. The mean quality-adjusted life-years (QALYs) were 3.38 with ADT alone and 3.92, 4.76, 3.92, and 5.01 with docetaxel, abiraterone acetate, enzalutamide, and apalutamide, added to ADT, respectively. As add-on therapy to ADT, docetaxel had an ICER of $42 069 per QALY over ADT alone; abiraterone acetate had an ICER of $58 814 per QALY over docetaxel; apalutamide had an ICER of $1 979 676 per QALY over abiraterone acetate; enzalutamide was dominated. At a willingness to pay below $50 000 per QALY, docetaxel plus ADT is likely the most cost-effective treatment; at any willingness to pay between $50 000 and $200 000 per QALY, abiraterone acetate plus ADT is likely the most cost-effective treatment. CONCLUSIONS: These findings underscore the value of abiraterone acetate plus ADT given its relative cost-effectiveness to other systemic treatments for metastatic castration-sensitive prostate cancer.

Health inequity drives disease biology to create disparities in prostate cancer outcomes.

Prostate cancer exerts a greater toll on African American men than on White men of European descent (hereafter referred to as European American men): the disparity in incidence and mortality is greater than that of any other common cancer. The disproportionate impact of prostate cancer on Black men has been attributed to the genetics of African ancestry, to diet and lifestyle risk factors, and to unequal access to quality health care. In this Review, all of these influences are considered in the context of the evolving understanding that chronic or recurrent inflammatory processes drive prostatic carcinogenesis. Studies of inherited susceptibility highlight the contributions of genes involved in prostate cell and tissue repair (BRCA1/2, ATM) and regeneration (HOXB13 and MYC). Social determinants of health appear to accentuate these genetic influences by fueling prostate inflammation and associated cell and genome damage. Molecular characterization of the prostate cancers that arise in Black versus White men further implicates this inflammatory microenvironment in disease behavior. Yet, when Black and White men with similar grade and stage of prostate cancer are treated equally, they exhibit equivalent outcomes. The central role of prostate inflammation in prostate cancer development and progression augments the impact of the social determinants of health on disease pathogenesis. And, when coupled with poorer access to high-quality treatment, these inequities result in a disparate burden of prostate cancer on African American men.

Disparities in Breast Cancer.

In the western world, breast cancer is the most common lethal cancer in women and the second leading cause of cancer death behind lung cancer. When assessing registry data, incidence and mortalty vary significantly by race or ethnicity and by socioeconomic status. There are a number of established risk factors, that effect risk of not just risk of breast cancer overall but the risk of certain molecular subtypes of breast cancer. Other factors in the disparity in outcomes include certain populations experiencing lower quality of care; prevention, screening, diagnosis and treatment.

Race, ethnicity, and gender reporting in North American clinical trials for BCG-unresponsive non-muscle invasive bladder cancer.

INTRODUCTION: The National Institutes of Health (NIH) Revitalization Act of 1993 established guidelines for the inclusion of racial/ethnic minorities and women in clinical research. However, the reporting rate of such patient demographic data in clinical trials for BCG-unresponsive non-muscle invasive bladder cancer is not well characterized. METHODS: We identified published clinical trials of all phases (I -III) for BCG-unresponsive non-muscle invasive bladder cancer conducted in the US and/or Canada. We calculated the proportion of studies reporting patient gender and race/ethnicity, tabulating these data when present. We compared reported trial participant race, ethnicity and gender with the number of new bladder cancer cases and deaths using the Centers for Disease Control and Prevention (CDC) and National Cancer Institute (NCI) U.S. Cancer Statistics data from 2013 -2017. RESULTS: We identified 27 trials published from 1998 -2021 enrolling a total of 1673 patients. While all trials included data on patient gender (22% women overall), only 40.7% included any data on patient race/ethnicity. Among those that did, trial participants were reported as white (94%), Black (2.1%), Hispanic (0.6%), Asian (0.9%), and Other (2.3%). Racial/ethnic minorities were underrepresented in clinical trials relative to their proportion of new bladder cancer cases and deaths. CONCLUSION: Most clinical trials that have been conducted for BCG-unresponsive non-muscle invasive bladder cancer do not report data on patient race or ethnicity despite NIH guidelines advocating for inclusion of such data. Racial/ethnic minorities remain underrepresented in these trials relative to the burden of bladder cancer prevalence and mortality faced by these groups.

The 50 years' war: The history and outcomes of the National Cancer Act of 1971.

The National Cancer Act of 1971 instigated 50 years of momentum that raised the federal investment in cancer research from $500 million in 1972 to $6.5 billion in 2021. This investment has fueled basic, translational, and clinical research that has had a tremendous impact on our understanding of cancer and our ability to prevent, diagnose, and treat it. It has also affected many other diseases.

Disparities in Tumor Mutational Burden, Immunotherapy Use, and Outcomes Based on Genomic Ancestry in Non-Small-Cell Lung Cancer.

PURPOSE: In patients with advanced non-small-cell lung cancer (aNSCLC), tumor mutational burden (TMB) may vary by genomic ancestry; however, its impact on treatment outcomes is unclear. This retrospective, observational study describes treatment patterns of patients with aNSCLC by genomic ancestry and electronic health record (EHR)-reported race and/or ethnicity and evaluates differences in TMB, cancer immunotherapy (CIT) access, and treatment outcomes across racial and ancestral groups. METHODS: Patients diagnosed with aNSCLC after January 1, 2011, were selected from a real-world deidentified clinicogenomics database and EHR-derived database; continuously enrolled patients were evaluated. Race and/or ethnicity was recorded using variables from the EHR database; genomic ancestry was classified by single-nucleotide polymorphisms on a next-generation sequencing panel. A threshold of 16 mutations per megabase was used to categorize TMB status. RESULTS: Of 59,559 patients in the EHR-derived database and 7,548 patients in the clinicogenomics database, 35,016 (58.8%) and 4,392 (58.2%) were continuously enrolled, respectively. CIT use was similar across EHR-reported race groups, ranging from 34.4% to 37.3% for non-Hispanic Asian and non-Hispanic Black patients, respectively. TMB levels varied significantly across ancestry groups (P < .001); patients of African ancestry had the highest median TMB (8.75 mutations per megabase; interquartile range, 4.35-14.79). In patients who had received CIT, high TMB was associated with improved overall survival compared with low TMB (20.89 v 11.83 months; hazard ratio, 0.60; 95% CI, 0.51 to 0.70) across genomic ancestral groups. CONCLUSION: These results suggest that equitable access to next-generation sequencing may improve aNSCLC outcome disparities in racially and ancestrally diverse populations.