Health Services Research, American Cancer Society, Atlanta, GA.
While the past decade has seen the development of multiple new interventions to diagnose and treat cancer, as well as to improve the quality of life for cancer patients, many of these interventions have substantial costs. This has resulted in increased scrutiny of the costs of care for cancer, as well as the costs relative to the benefits for cancer treatments. It is important for oncologists and other members of the cancer community to consider and understand how economic evaluations of cancer interventions are performed and to be able to use and critique these evaluations. This review discusses the components, main types, and analytic issues of health economic evaluations using studies of cancer interventions as examples. We also highlight limitations of these economic evaluations and discuss why members of the cancer community should care about economic analyses.
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Health Services Research, Department of Biostatistics, Division of Quantitative Sciences, and Department of Health Disparities Research, Division of Cancer Prevention & Population Sciences, The University of Texas M.D. Anderson Cancer Center, Houston, Texas, USA,1.
ABSTRACT We analyzed the utilization trend for immunotherapy as initial treatment in the United States and estimated the cost impact of three cancers for which the majority of newly approved immunotherapies were indicated: non-Hodgkin's lymphoma (NHL), metastatic breast cancer (MBC), and metastatic colorectal cancer (MCRC). Utilization was highly concentrated, with four cancers accounting for 70-80% of use. On the basis of the pattern in 2004 (22.9% NHL, 3.2% MCRC, and 3.3% MBC), immunotherapy was associated with $285,$73, and $12 million cost increase for NHL, MCRC, and MBC, respectively. Costs for these cancers would have exceeded $1.82 billion had 50% of eligible patients received immunotherapy.
Section of Health Services Research, Department of Biostatistics, Division of Quantitative Science, The University of Texas MD Anderson Cancer Center, Houston, Texas 77230, USA. yashih@mdanderson.org
CONTEXT: Immunotherapy represents a newer modality for cancer treatment with substantial health benefits. However, the high costs of immunotherapy may create access barriers. OBJECTIVE: To examine factors associated with the utilization of immunotherapy among newly diagnosed cancer patients. DESIGN AND SETTING: Analysis of a large national cancer registry data, the National Cancer Data Base, that represents approximately 75% of newly diagnosed cancer patients in the US. PATIENTS: Patient diagnosed with non-Hodgkin lymphoma (NHL), metastatic breast cancer (MBC), or metastatic colorectal cancer (MCRC). MAIN OUTCOME MEASURES: Factors associated with utilization of immunotherapy in NHL, MBC, or MCRC. RESULTS: The study included 207,581 and 42,804 patients diagnosed with NHL and MBC between 1998 and 2004, respectively (based on approval of rituximab in 1997 and trastuzumab in 1998); and 16,027 patients diagnosed with MCRC in 2004 (based on approval of cetuximab and bevacizumab in 2004). Immunotherapy use varied significantly by age, race, insurance status, year of diagnosis, and type of treatment facilities, with largest effects observed in years of diagnosis, followed by insurance status. Among young cancer patients, we found that compared with uninsured patients, the odds of receiving immunotherapy were 1.61, and 2.13 for privately insured MBC, and MCRC patients, respectively, and were in the range of 1.13 to 19.23 for privately insured NHL patients. For elderly NHL patients, those with secondary insurance in addition to Medicare were 1.26 to 8.24 times more likely to receive immunotherapy than those with Medicare alone. CONCLUSIONS: The disparities observed in this study suggest that additional efforts are required to ensure that all cancer patients receive timely and appropriate treatment.
Section of Health Services Research, Department of Biostatistics, Division of Quantitative Sciences, The University of Texas M.D. Anderson Cancer Center, Houston, Texas, USA.
Health information technology offers a powerful tool to monitor the performance of a healthcare system. Advances in computer technology and capacity combined with lower start-up costs will allow developing countries to achieve greater impact when they initiate electronic health information systems. We focused on the integrated health information system that was established in Taiwan in conjunction with the launch of the National Health Insurance (NHI) programme. We used data from that health information system to conduct a cost-effectiveness analysis of chemotherapy use among breast cancer patients. We then used this analysis to discuss what policy makers can learn from this type of analysis. We identified a cohort of patients in the NHI Research Database who had been diagnosed with breast cancer in 2001 and had received chemotherapy following surgical removal of the tumour. We followed these patients for 3 years and conducted a cost-effectiveness analysis from the payer's perspective. Using the net benefit regression approach, we compared the cost effectiveness of the two most commonly prescribed first-line chemotherapy regimens for the treatment of breast cancer in 2001 in Taiwan. The dependent variable of the regression model was the individual-level net benefit, and the independent variables included a binary variable indicating the choice of chemotherapy regimen, the patients' age, co-morbidity, type of surgery, geographic region and type of treatment facility. We employed both frequentist and Bayesian approaches in our net benefit regression analyses. In the Bayesian analysis, we applied non-informative priors to all parameters in the base-case analyses. We then explored the use of informative priors in the sensitivity analysis, using cost-effectiveness data published in the literature to form the prior distributions for the relevant parameters. Over 60% of surgically treated breast cancer patients received either CMF (cyclophosphamide, methotrexate, fluorouracil) or CEF (cyclophosphamide, epirubicin, fluorouracil). A comparison of patient characteristics indicated that patients in the CEF group tended to be younger (47.8 vs 49.1 years; p = 0.016), and were significantly more likely to have undergone a mastectomy (84% vs 76%; p < 0.001) and to have been treated in a teaching hospital (26% vs 13%; p < 0.001). We also observed significant variations in geographic region of the location of facilities between treatment groups. On average, CEF was not cost effective in the treatment of patients with breast cancer in Taiwan, although analyses stratified by geographic region suggested a wide variation across regions. At a societal willingness to pay (WTP) of new Taiwanese dollar ($NT)1 500 000 ($US80 000), the probability that CEF was more cost effective than CMF was 0.0%, 0.0%, 0.0% and 3.9% for the Taipei metropolitan area, and the north, middle and the combined south and east region, respectively; the probability became 0.6%, 0.0%, 1.3% and 54.5%, respectively, at a WTP of $NT5 000 000 ($US270 000). After co-variate adjustments, the probabilities were 0.0%, 0.0%, 0.0% and 0.8%, respectively at a WTP of $NT1 500 000, and were 0.0%, 0.0%, 1.4% and 34.7% at $NT5 000 000. Sensitivity analyses showed that CEF potentially could have been more cost effective than CMF within a reasonable range of societal WTP (i.e.$NT1 000 000-3 000 000 or $US55 000-160 000) had the optimal dosage level for CEF been established for breast cancer patients in Taiwan. A population-based, fully integrated electronic health information system provides useful data to assess the cost effectiveness of competing treatments and interventions in current practice. This research may potentially inform policy makers of modifications that can be instituted to improve the cost effectiveness of a new therapy. However, findings from this study need to be interpreted with caution because the study provided information only on the short-term cost effectiveness (i.e. 3 years) of CEF compared with CMF. It is possible that a future analysis will reach a different conclusion when more years of follow-up data become available.
Exponent, 1800 Diagonal Road, Suite 355, Alexandria, VA 22314, USA. jschmier@exponent.com.
The reliability of recall patient reported outcomes, such as health-related quality of life, satisfaction and symptoms, varies substantially. The objectives of this special report are to identify key issues to consider in study design and provide suggestions for minimizing bias in studies including patient reported outcomes. A MEDLINE search identified several areas in which patient recall is subject to bias. Concordance between patient recall and baseline assessments (e.g., prior to an event or medical intervention) for these patient reported outcomes varies depending on the event being recalled, time since the event, and patient clinical and demographic characteristics. Symptom recall tends to be better than recall of health-related quality of life or pain intensity. Specific questionnaire techniques may help minimize the impact of recall bias. Further research is required to determine what factors or patient characteristics predict improved recall and what techniques minimize recall bias.
Section of Health Services Research, Unit 196, Department of Biostatistics, The University of Texas, MD Anderson Cancer Center, 1515 Holcombe Blvd., Houston, Texas 77030 4009, USA.
The Bayesian approach is becoming increasingly visible in pharmacoeconomics. This approach has been primarily applied to two decision problems commonly encountered in pharmacoeconomics: adoption and allocation. Acceptability curves generated from Bayesian cost-effectiveness analyses can be interpreted as the probability that the new treatment is cost-effective at a given level of willingness-to-pay. This probabilistic interpretation of study findings provides information that is more relevent and easier to understand for those who make adoption decisions. The Bayesian value of information analysis offers a decision-analytic framework to explore values of additional research and to set research priorities. It is a useful analytical framework for decision-makers who wish to achieve allocation efficacy.
Ya-Chen Tina Shih,
Ying Xu,
Janice N Cormier,
Sharon Giordano,
Sheila H Ridner,
Thomas A Buchholz,
George H Perkins,
Linda S Elting
Section of Health Services Research, Department of Biostatistics, Division of Quantitative Sciences; Department of Surgical Oncology; Department of Breast Medical Oncology, Division of Cancer Medicine; and Department of Radiation Oncology, Division of Radiation Oncology, Breast Service, The University of Texas M. D. Anderson Cancer Center, Houston, TX; and Vanderbilt University School of Nursing, Nashville, TN.
PURPOSE: This study estimated the economic burden of breast cancer-related lymphedema (BCRL) among working-age women, the incidence of lymphedema, and associated risk factors. METHODS: We used claims data to study an incident cohort of breast cancer patients for the 2 years after the initiation of cancer treatment. A logistic regression model was used to ascertain factors associated with lymphedema. We compared the medical costs and rate of infections likely associated with lymphedema between a woman with BCRL and a matched control. We performed nonparametric bootstrapping to compare the unadjusted cost differences and estimated the adjusted cost differences in regression analysis. RESULTS: Approximately 10% of the 1,877 patients had claims indicating treatment of lymphedema. Predictors included treatment with full axillary node dissection (odds ratio [OR]= 6.3, P <.001) and chemotherapy (OR = 1.6, P =.01). A geographic variation was observed; women who resided in the West were more likely to have lymphedema claims than those in the Northeast (OR = 2.05, P =.01). The matched cohort analysis demonstrated that the BCRL group had significantly higher medical costs ($14,877 to $23,167) and was twice as likely to have lymphangitis or cellulitis (OR = 2.02, P =.009). Outpatient care, especially mental health services, diagnostic imaging, and visits with moderate or high complexity, accounted for the majority of the difference. CONCLUSION: Although the use of claims data may underestimate the true incidence of lymphedema, women with BCRL had a greater risk of infections and incurred higher medical costs. The substantial costs documented here suggest that further efforts should be made to elucidate reduction and prevention strategies for BCRL.
The Center for Healthcare Research and Reform, The Dartmouth Institute for Health Policy and Clinical Practice, Dartmouth Medical School, Hanover, New Hampshire., Department of Community and Family Medicine, Dartmouth Medical School, Hanover, New Hampshire., The Norris Cotton Cancer Center, Dartmouth Hitchcock Medical Center, Lebanon, New Hampshire.
Abstract Objectives: End-of-life care is increasingly recognized as an important part of cancer management for many patients. Current methods to measure end-of-life care are limited by difficulties in identifying cancer cohorts with administrative data. We examined several techniques of identifying end-of-life cancer cohorts with claims data that is population-based, geographically scalable, and amenable to routine updating. Methods: Using Medicare claims for patients 65 years of age and older, four techniques for identifying end-of-life cancer cohorts were compared; one based on Part A data using a broad primary or narrow secondary diagnosis of cancer, two based on Part B data, and one combining the Part A and B methods. We tested the performance of each definition to ascertain an appropriate end-of-life cancer population. Results: The combined Part A and B definition using a primary or secondary diagnosis of cancer within a window of 180 days prior to death appears to be the most accurate and inclusive in ascertaining an end-of-life cohort (78.7% attainment). Conclusion: Combining inpatient and outpatient claims data, and identifying cases based upon a broad primary or a narrow secondary cancer definition is the most accurate and inclusive in ascertaining an end-of-life cohort.
Emory University, United States.
OBJECTIVES: Rheumatoid arthritis (RA) causes significant disability and often results in loss of work capacity and productivity. We evaluated the impact of adalimumab, a tumour necrosis factor antagonist with demonstrated efficacy in RA, on long-term employment. METHODS: Data from an open-label extension study (DE033) of 486 RA patients receiving adalimumab monotherapy who previously failed at least 1 disease-modifying antirheumatic drug (DMARD) and had baseline work status information were compared with data from 747 RA patients receiving DMARD therapy in a Norway-based longitudinal registry. Primary outcomes included the time patients continued working at least part-time and the likelihood of stopping work. Secondary outcomes included American College of Rheumatology (ACR) and EULAR responses and disease remission. Outcomes were compared 6, 12, and 24 months after enrollment. RESULTS: During a 24-month period, the 158 adalimumab-treated patients who were working at baseline worked 7.32 months longer (95% confidence interval [CI]: 4.8, 9.1) than did the 180 DMARD-treated patients, controlling for differences in baseline characteristics. Regardless of baseline work status, patients receiving adalimumab worked 2.0 months longer (95% CI: 1.3, 2.6) and were significantly less likely to stop working (hazard ratio [HR], 0.36, 95% CI:-0.30, 0.42] for all patients and 0.36, 95% CI: 0.15, 0.85] for patients working at baseline, respectively) than those receiving DMARDs. Adalimumab-treated patients also were significantly more likely to achieve ACR responses and disease remission than DMARD-treated patients. Patients who achieved EULAR good response and remission were less likely to stop working, but this relationship was only seen in in patients receiving DMARDs. CONCLUSIONS: Patients with RA receiving adalimumab experienced significantly longer periods of work and continuous employment, as well as greater rates of clinical responses, than did those receiving DMARDs. The mechanism by which adalimumab decreases likelihood of stopping work appears different from that of DMARD therapy and independent of clinical responses.
Health Services Research, American Cancer Society, Atlanta, Georgia 30303, USA. michael.halpern@cancer.org
Little is known regarding the prevalence of outpatient cancer treatment in the U.S. We analyzed nationally-representative data from the 2000-2004 Medical Expenditure Panel Survey to estimate the number of U.S. cancer patients receiving outpatient chemotherapy and/or radiation therapy annually. Each year, over 1.1 million individuals are estimated to receive chemotherapy or radiation therapy for cancer. Cancer patients younger than 65 receiving treatment who were uninsured were less likely to receive chemotherapy or combined chemotherapy/radiation therapy than were those with public or private insurance. These estimates may be useful for understanding the burden of cancer care and development of programs for cancer survivors.
University of Texas M. D. Anderson Cancer Center.
Background. Over 11% of the US population in 2002 was foreign born. Studies that have examined disparities of cancer screening often focus on racial/ethnicity differences without considering their origins. This study examines the disparities in colorectal cancer (CRC) screening between US- and foreign-born groups and explores factors associated with such disparities. Methods. Data were obtained from the 2000 National Health Interview Survey Cancer Control Module. Multivariate logistic models were used to compare the use of CRC screening among the foreign born and 4 US-born race/ethnicity subgroups while controlling for other factors that may affect such screening. Appropriate weighting procedures were employed to account for the complex design of the survey. Results. Compared with the US-born non-Hispanic Whites, the odds ratio of ever having had a colorectal screening for the US-born Hispanic, non-Hispanic Black, non-Hispanic other races groups, and the foreign-born group with 15 or more years of residency was 0.65, 0.77, 0.98, and 0.58, respectively. An even lower odds ratio (0.46) was found among the foreign-born group with a shorter duration of residency. We found that the rate of screening for foreign-born individuals with good socioeconomic status and/or with health insurance was significantly lower than that of the US-born with similar status, and the rate was not much different from that of US-born individuals in lower socioeconomic levels. Having a usual source of care was found to be the most important protective factor for the foreign-born group. Conclusions. Foreign birth was associated with a lower rate of CRC screening. Future studies of health disparities should also consider immigration status. J Cancer Educ. 2008; 23:18-25.
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Affiliations of authors: The Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA (DG, C-HF, PJN); Department of Health Systems Management, Ben-Gurion University of the Negev, Beer-Sheva, Israel (DG); Institute for Clinical Evaluative Sciences, Sunnybrook Health Sciences Centre, Toronto, ON, Canada (CE); Department of Community and Preventive Medicine, University of Rochester School of Medicine and Dentistry, Rochester, NY (AE-L).
New cancer treatments pose a substantial financial burden on health-care systems, insurers, patients, and society. Cost-utility analyses (CUAs) of cancer-related interventions have received increased attention in the medical literature and are being used to inform reimbursement decisions in many health-care systems. We identified and reviewed 242 cancer-related CUAs published through 2007 and included in the Tufts Medical Center Cost-Effectiveness Analysis Registry (www.cearegistry.org). Leading cancer types studied were breast (36% of studies), colorectal (12%), and hematologic cancers (10%). Studies have examined interventions for tertiary prevention (73% of studies), secondary prevention (19%), and primary prevention (8%). We present league tables by disease categories that consist of a description of the intervention, its comparator, the target population, and the incremental cost-effectiveness ratio. The median reported incremental cost-effectiveness ratios (in 2008 US $) were $27 000 for breast cancer,$22 000 for colorectal cancer,$34 500 for prostate cancer,$32 000 for lung cancer, and $48 000 for hematologic cancers. The results highlight the many opportunities for efficient investment in cancer care across different cancer types and interventions and the many investments that are inefficient. Because we found only modest improvement in the quality of studies, we suggest that journals provide specific guidance for reporting CUA and assure that authors adhere to guidelines for conducting and reporting economic evaluations.
Centre for the Evaluation of Vaccination,Epidemiology and Community Medicine, University of Antwerp,Universiteitsplein 1,2610 Antwerp, Belgium. pbeutels@uia.ua.ac.be.
We performed an extensive search for full economic evaluations on pneumococcal vaccination in adults. We included 11 North American and 9 European studies for review. There was a clear preference for cost-effectiveness and cost-utility versus cost-benefit analysis. In general, pneumococcal vaccination of the elderly <75 years and of particular risk groups is found to be relatively cost-effective and potentially cost-saving to the health care sector and to society. However, there is substantial uncertainty about the effectiveness of the intervention in these target groups. From 1997 on most analysts have taken this partly into account by distinguishing between invasive and non-invasive pneumococcal disease. In order to be relevant, future analyses should use quality adjusted life-years, adapt life-expectancy for people at risk of infection, narrow the targeted age groups, include realistic programme costs and discuss the potential influence of unrelated future costs. Finally, estimating the impact of antimicrobial resistance (not included in any of the analyses) seems particularly challenging for the future.
Division of Cardiovascular & Rare Diseases, Korean Centers for Disease Control and Prevention, Korea.
OBJECTIVES: The purposes of this study is to estimate the cost of cancer care after its diagnosis and to identify factors that can influence the cost of cancer care. METHODS: The study subjects were patients with an initial diagnosis one of four selected tumors and had their first two-years of cancer care at a national cancer center. The data were obtained from medical records and patient surveys. We classified cancer care costs into medical and nonmedical costs, and each cost was analyzed for burden type, medical service, and cancer stage according to cancer types. Factors affecting cancer care costs for the initial phase included demographic variables, socioeconomic status and clinical variables. RESULTS: Cancer care costs for the initial year following diagnosis were higher than the costs for the following successive year after diagnosis. Lung cancer (25,648,000 won) had higher costs than the other three cancer types. Of the total costs, patent burden was more than 50% and medical costs accounted for more than 60%. Inpatient costs accounted for more than 60% of the medical costs for stomach and liver cancer in the initial phase. Care for late-stage cancer was more expensive than care for early-stage cancer. Nonmedical costs were estimated to be between 4,500,000 to 6,000,000 won with expenses for the caregiver being the highest. The factors affecting cancer care costs were treatment type and cancer stage. CONCLUSIONS: The cancer care costs after diagnosis are substantial and vary by cancer site, cancer stage and treatment type. It is useful for policy makers and researchers to identify tumor-specific medical and nonmedical costs. The effort to reduce cancer costs and early detection for cancer can reduce the burden to society and improve quality of life for the cancer patients.
Uppsala University/Akademiska Sjukhuset, Department of Oncology, Akademiska Hospital, 751 85 Uppsala, Sweden.
The rapid development of the care makes it important to have relevant cost information for cost-effectiveness assessments. The aim of this study is to estimate the health care cost of a disseminated breast cancer relapse in Sweden. A retrospective cohort study of women with disseminated breast cancer in Sweden was done. The individual case records were reviewed and all data concerning treatments, hospitalisation, examinations and palliative care were collected. The study included 53 patients with a total mean cost of euro93,700 (95% Confidence Interval (CI): euro78,500-euro109,600). Drugs and hospitalisations were the largest single cost sources. HER2-positive patients had slightly higher mean costs (euro123,300), while triple negative patients had slightly lower mean costs (euro70,600). The current costs for patients with disseminated breast cancer are considerably higher than those previously shown, which may have important consequences for economic evaluations of interventions aimed at reducing the risk of disseminated breast cancer.
Annals of Internal Medicine. jenwilson@acponline.org
Robert M. La Follette School of Public Affairs, University of Wisconsin-Madison, Madison, WI, USA.
BACKGROUND: Alzheimer's disease (AD) is a progressive neurodegenerative disease that places substantial burdens on those who provide support for family members with declining cognitive and functional abilities. Many AD patients eventually require formal long-term care services because of the absence, exhaustion, or inability of family members to provide care. The costs of long-term care, and especially nursing home care, often deplete private financial resources, placing a substantial burden on state Medicaid programs. Current evidence suggests that pharmacological treatments and caregiver interventions can delay entry into nursing homes and potentially reduce Medicaid costs. However, these cost savings are not being realized because many patients with AD are either not diagnosed or diagnosed at late stages of the disease, and have no access to Medicare-funded caregiver support programs. METHODS AND RESULTS: A Monte Carlo cost-benefit analysis, based on estimates of parameters available in the medical literature, suggests that the early identification and treatment of AD have the potential to result in large, positive net social benefits as well as positive net savings for states and the federal government. CONCLUSIONS: These findings indicate that the early diagnosis and treatment of AD are not only socially desirable in terms of increasing economic efficiency, but also fiscally attractive from both state and federal perspectives. These findings also suggest that failure to fund effective caregiver interventions may be fiscally unsound.
Department of Medicine, Chief, Division of Cardiovascular Medicine, Medical University of Ohio, Toledo, Ohio, USA.
NYU Medical Center, New York, NY 10282, USA. mmalach2@gmail.com
The explosion of medical knowledge has deluged the medical community with a plethora of new medicines, new tests, and new procedures. This creates a serious need to carefully evaluate the definable benefits from these new developments, which promise to increase the quality of medical care beyond older, established, and usually less-costly methods. In addition, more recent information has clearly identified genetic variation in an individual's response to medications. As such, conventional wisdom may now prove to be wrong or subject to question. Examples of this, which we present, include situations where medication may more safely provide benefits to asymptomatic, stable patients than surgical interventions with high potential for complications that counteract predicted benefits. We argue that preventive medicine offers an unusual and comprehensive promise of disease prevention and treatment. The issues we cite may be effective in the future cost reduction of medical care.
Aalborg Universitet, Institut for Erhvervsstudier. rs@rikkesogaard.dk
This article summarizes the current level of evidence for cost-effectiveness in lumbar spinal fusion. Several economic evaluations have recently been conducted alongside randomized controlled trials, but choices of populations and comparators are diverse. Overall, cost-effectiveness depends on clinical outcomes, but there is some evidence that the best technology leads to extra-hospital cost-savings. The optimal technique, therefore, remains a clinical research question.
Universitätsklinikum Hamburg-Eppendorf, Zentrum für Psychosoziale Medizin, Institut für Medizin-Soziologie.
OBJECTIVES: This article primarily reflects on the economic benefits of self-help associations, and the problems of their adequate evaluation. METHODS: We present a critical discussion of the concepts in the economic evaluation and empiricism by secondary analysis of data available based on a review of published studies as well as the re-analysis of primary data from our Hamburg study in 2003 (n=345 or 52.4% of the self-help associations registered in Hamburg). RESULTS: Most approaches to evaluate the economic benefits of self-help groups were inspired by the idea of output-related, cost-benefit analysis, by which activities of members were monetarily estimated. Projected for Hamburg, the productive performance amounts to nearly 1.8 million Euro per month, and 21.5 million per year. Furthermore, additional voluntary commitment can be mobilized at 300,000 Euro per month. CONCLUSION: All calculations are based on some assumptions (e.g., coefficient of productivity, wages) which cannot be finally verified. In view of the state of research reviewed we strongly recommend to bring forward both outcome-oriented approaches of economic evaluation, and new representative studies of self-help groups for Germany as a whole.
