Abstract
Although a broad range of data resources have played a key role in the substantial achievements of cancer health economics research, there are now needs for more comprehensive data that ...represent a fuller picture of the cancer care experience. In particular, researchers need information that represents more diverse populations; includes more clinical details; and provides greater context on individual- and neighborhood-level factors that can affect cancer prevention, screening, treatment, and survivorship, including measures of financial health or toxicity, health-related social needs, and social determinants of health. This article highlights 3 critical topics for cancer health economics research: the future of the National Cancer Institute’s Surveillance, Epidemiology, and End Results-Centers for Medicare & Medicaid Services–linked data resources; use of social media data for cancer outcomes research; and multi-site–linked electronic health record data networks. These 3 topics represent different approaches to enhance data resources, linkages, and infrastructures and are complementary strategies to provide more complete information on activities involved in and factors affecting the cancer control continuum. These and other data resources will assist researchers in examining the complex and nuanced questions now at the forefront of cancer health economics research.
Abstract Background Economic evaluations often measure an intervention effect with mean overall survival (OS). Emerging types of cancer treatments offer the possibility of being “cured” in that ...patients can become long-term survivors whose risk of death is the same as that of a disease-free person. Describing cured and noncured patients with one shared mean value may provide a biased assessment of a therapy with a cured proportion. Objective The purpose of this article is to explain how to incorporate the heterogeneity from cured patients into health economic evaluation. Methods We analyzed clinical trial data from patients with advanced melanoma treated with ipilimumab (Ipi; n = 137) versus glycoprotein 100 (gp100; n = 136) with statistical methodology for mixture cure models. Both cured and noncured patients were subject to background mortality not related to cancer. Results When ignoring cured proportions, we found that patients treated with Ipi had an estimated mean OS that was 8 months longer than that of patients treated with gp100. Cure model analysis showed that the cured proportion drove this difference, with 21% cured on Ipi versus 6% cured on gp100. The mean OS among the noncured cohort patients was 10 and 9 months with Ipi and gp100, respectively. The mean OS among cured patients was 26 years on both arms. When ignoring cured proportions, we found that the incremental cost-effectiveness ratio (ICER) when comparing Ipi with gp100 was $324,000/quality-adjusted life-year (QALY) (95% confidence interval $254,000–$600,000). With a mixture cure model, the ICER when comparing Ipi with gp100 was $113,000/QALY (95% confidence interval $101,000–$154,000). Conclusions This analysis supports using cure modeling in health economic evaluation in advanced melanoma. When a proportion of patients may be long-term survivors, using cure models may reduce bias in OS estimates and provide more accurate estimates of health economic measures, including QALYs and ICERs.
Introduction Nationally, a greater proportion of American Indians and Alaska Natives (AI/ANs) are diagnosed with advanced-stage cancers compared with non-Hispanic whites. The reasons for observed ...differences in stage at diagnosis between AI/ANs and non-Hispanic whites remain unclear. Methods Medicaid, Indian Health Service Care Systems, and state cancer registry data for California, Oregon, and Washington (2001–2008, analyzed in 2014–2015) were linked to identify AI/ANs and non-Hispanic whites diagnosed with invasive breast, cervical, colorectal, lung, or prostate cancer. Logistic regression was used to estimate ORs and 95% CIs for distant disease versus local or regional disease, in AI/ANs compared with non-Hispanic white case patients. Results A similar proportion of AI/AN (31.2%) and non-Hispanic white (35.5%) patients were diagnosed with distant-stage cancer in this population (AOR=1.03, 95% CI=0.88, 1.20). No significant differences in stage at diagnosis were found for any individual cancer site. Among AI/ANs, Indian Health Service Care Systems eligibility was not associated with stage at diagnosis. Conclusions In contrast to the general population of the U.S., among Medicaid enrollees, AI/AN race is not associated with later stage at diagnosis. Cancer survival disparities associated with AI/AN race that have been observed in the broader population may be driven by factors associated with income and health insurance that are also associated with race, as income and insurance status are more homogenous within the Medicaid population than within the broader population.
Abstract Clinical trials evaluating medicines, medical devices, and procedures now commonly assess the economic value of these interventions. The growing number of prospective clinical/economic ...trials reflects both widespread interest in economic information for new technologies and the regulatory and reimbursement requirements of many countries that now consider evidence of economic value along with clinical efficacy. As decision makers increasingly demand evidence of economic value for health care interventions, conducting high-quality economic analyses alongside clinical studies is desirable because they broaden the scope of information available on a particular intervention, and can efficiently provide timely information with high internal and, when designed and analyzed properly, reasonable external validity. In 2005, ISPOR published the Good Research Practices for Cost-Effectiveness Analysis Alongside Clinical Trials: The ISPOR RCT-CEA Task Force report. ISPOR initiated an update of the report in 2014 to include the methodological developments over the last 9 years. This report provides updated recommendations reflecting advances in several areas related to trial design, selecting data elements, database design and management, analysis, and reporting of results. Task force members note that trials should be designed to evaluate effectiveness (rather than efficacy) when possible, should include clinical outcome measures, and should obtain health resource use and health state utilities directly from study subjects. Collection of economic data should be fully integrated into the study. An incremental analysis should be conducted with an intention-to-treat approach, complemented by relevant subgroup analyses. Uncertainty should be characterized. Articles should adhere to established standards for reporting results of cost-effectiveness analyses. Economic studies alongside trials are complementary to other evaluations (e.g., modeling studies) as information for decision makers who consider evidence of economic value along with clinical efficacy when making resource allocation decisions.
Abstract Objective To collect disease-specific and generic preference values for three populations. Methods Prostate cancer-specific health states were developed with attributes that varied across ...five health domains: sexual function, urinary function, bowel function, pain, and fear of the future. Men with prostate cancer, men at risk for prostate cancer, and a sample of the general population assigned value to 18 disease-specific health states using standard gamble (SG) methodology. Study participants also completed the Health Utilities Index (HUI) to obtain generic, community-based preference values to capture their current health rating. Results A total of 136 participants were enrolled (n = 43 prostate cancer; n = 40 at risk for prostate cancer; n = 49 general population). Mean HUI mark 3 current health ratings: men with prostate cancer 0.75 ± 0.260; men at risk for prostate cancer 0.77 ± 0.238; general population 0.84 ± 0.178. Mean SG preference values ranged from 0.46 to 0.85 among men with prostate cancer, 0.37 to 0.75 among men at risk for prostate cancer, and 0.32 to 0.81 among the general population group. Conclusions In general, preference values for disease-specific health states using the patient perspective were higher than those for the general population. Generic preference values calculated from the HUI were higher than disease-specific preference values calculated from the SG. The higher values calculated from the HUI, from all three perspectives, indicate that a generic measure may not be sensitive enough to capture the disutility of prostate cancer symptoms, specifically sexual dysfunction, urinary dysfunction, and bowel dysfunction, which are being directly measured in the disease-specific health states.
Comparative effectiveness research (CER) has emerged as an approach to improve quality of care and patient outcomes while reducing healthcare costs by providing evidence to guide healthcare ...decisions. Randomized controlled trials (RCTs) have represented the ideal study design to support treatment decisions in head-and-neck (H&N) cancers. In RCTs, formal chance (randomization) determines treatment allocation, which prevents selection bias from distorting the measure of treatment effects. Despite this advantage, only a minority of patients qualify for inclusion in H&N RCTs, which limits the validity of their results to the broader H&N cancer patient population seen in clinical practice. Randomized controlled trials often do not address other knowledge gaps in the management of H&N cancer, including treatment comparisons for rare types of H&N cancers, monitoring of rare or late toxicity events (eg, osteoradionecrosis), or in some instances an RCT is simply not feasible. Observational studies, or studies in which treatment allocation occurs independently of investigators' choice or randomization, may address several of these gaps in knowledge, thereby complementing the role of RCTs. This critical review discusses how observational CER studies complement RCTs in generating the evidence to inform healthcare decisions and improve the quality of care and outcomes of H&N cancer patients. Review topics include a balanced discussion about the strengths and limitations of both RCT and observational CER study designs; a brief description of design and analytic techniques to handle selection bias in observational studies; examples of observational studies that inform current clinical practices and management of H&N cancers; and suggestions for relevant CER questions that could be addressed by an observational study design.
Abstract Objectives Bevacizumab is approved for treatment of advanced non-small cell lung cancer (NSCLC) in combination with chemotherapy based on a 2-month median survival benefit demonstrated in ...one randomized trial. The cost-utility of adding bevacizumab to chemotherapy in advanced NSCLC remains unknown. We evaluated the cost-utility of bevacizumab added to chemotherapy in patients with advanced NSCLC. Methods We developed a Markov model to estimate quality-adjusted life years (QALYs) and direct medical costs from the US payer perspective in patients treated with bevacizumab plus chemotherapy and compared these outcomes with patients treated with chemotherapy alone. We populated the model with survival and toxicity data from the clinical trial that compared the two strategies. We obtained utilities from a literature search and unit costs from Medicare. We discounted QALYs and costs at 3% per year. We addressed uncertainty with one-way and probabilistic sensitivity analyzes. Results Compared with chemotherapy alone, bevacizumab and chemotherapy increased mean QALYs by 0.13, at an incremental life-time cost of US$72,000 per patient. The incremental cost-utility ratio (ICUR) was US$560,000/QALY. The ICUR was most sensitive to the survival on bevacizumab treatment, the drug costs of bevacizumab, and the utility of stable disease on treatment. At a threshold of US$100,000/QALY, the addition of bevacizumab had a 0.2% probability of being cost-effective. Conclusions Bevacizumab does not appear to be cost-effective when added to chemotherapy in patients with advanced NSCLC, based on approximate cost-effectiveness thresholds that have been identified in the United States. These results may inform decision-makers about resource allocation for NSCLC care.
Abstract The recent acceleration of scientific discovery has led to greater choices in health care. New technologies, diagnostic tests, and pharmaceuticals have widely varying impact on patients and ...populations in terms of benefits, toxicities, and costs, stimulating a resurgence of interest in the creation of frameworks intended to measure value in health. Many of these are offered by providers and/or advocacy organizations with expertise and interest in specific diseases (e.g., cancer and heart disease). To help assess the utility of and the potential biases embedded in these frameworks, we created an evaluation taxonomy with seven basic components: 1) define the purpose; 2) detail the conceptual approach, including perspectives, methods for obtaining preferences of decision makers (e.g., patients), and ability to incorporate multiple dimensions of value; 3) discuss inclusions and exclusions of elements included in the framework, and whether the framework assumes clinical intervention or offers alternatives such as palliative care or watchful waiting; 4) evaluate data sources and their scientific validity; 5) assess the intervention’s effect on total costs of treating a defined population; 6) analyze how uncertainty is incorporated; and 7) illuminate possible conflicts of interest among those creating the framework. We apply the taxonomy to four representative value frameworks recently published by professional organizations focused on treatment of cancer and heart disease and on vaccine use. We conclude that each of these efforts has strengths and weaknesses when evaluated using our taxonomy, and suggest pathways to enhance the utility of value-assessing frameworks for policy and clinical decision making.
An abstract of a study by McDermott et al evaluating the association between depression occurring in the year prior to a diagnosis of stage 3B/4 non-small cell lung cancer (NSCLC), hospice enrollment ...after diagnosis, and utilization of emergency room services, hospitalization, and chemotherapy in the last 30 days of life is presented. Older adults with depression prior to a diagnosis of advanced stage NSCLC were more likely to utilize hospice services compared to those without depression, were less likely to die in the hospital, and were no more likely to use high-intensity end of life care.
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