Eileen A. Sandberg

Systematic Overview of Cost-Utility Assessments in Oncology

PURPOSE Cost-utility analyses (CUAs) present the value of an intervention as the ratio of its incremental cost divided by its incremental survival benefit, with survival weighted by utilities to produce quality-adjusted life years (QALYs). We critically reviewed the CUA literature and its role in informing clinical oncology practice, research priorities, and policy. METHODS The English-language literature was searched between 1975 and1997 for CUAs. Two readers abstracted from each article descriptions of the clinical situation and patients, the methods used, study perspective, the measures of effectiveness, costs included, discounting, and whether sensitivity analyses were performed. The readers then made subjective quality assessments. We also extracted utility values from the reviewed papers, along with information on how and from whom utilities were measured. RESULTS Our search yielded 40 studies, which described 263 health states and presented 89 cost-utility ratios. Both the number and quality of studies increased over time. However, many studies are at variance with current standards. Only 20% of studies took a societal perspective, more than a third failed to discount both the costs and QALYs, and utilities were often simply estimates from the investigators or other physicians. CONCLUSION The cost-utility literature in oncology is not large but is rapidly expanding. There remains much room for improvement in the methodological rigor with which utilities are measured. Considering quality-of-life effects by incorporating utilities into economic studies is particularly important in oncology, where many therapies obtain modest improvements in response or survival at the expense of nontrivial toxicity.

Measuring costs in cost-utility analyses. Variations in the literature.

OBJECTIVES Although cost-utility analysis (CUA) has been recommended by some experts as the preferred technique for economic evaluation, there is controversy regarding what costs should be included and how they should be measured. The purpose of this study was to: a) identify the cost components that have been included in published CUAs; b) catalogue the sources of valuation used; c) examine the methods employed for estimating costs; and d) explore whether methods have changed over time. METHODS We conducted a comprehensive search of the published literature and systematically collected data on the cost estimation of CUAs. We audited the cost estimates in 228 CUAs. RESULTS In most studies (99%), analysts included some direct healthcare costs. However, the inclusion of direct non-healthcare and time costs (17%) was generally lacking, as was productivity costs (8%). Only 6% of studies considered future costs in added life-years. In general, we found little evidence of change in methods over time. The most frequently used source for valuation of healthcare services was published estimates (73%). Few studies obtained utilization data from RCTs (10%) or relied on other primary data (23%). About two-thirds of studies conducted sensitivity analyses on cost estimates. CONCLUSIONS We found wide variations in the estimation of costs in published CUAs. The study underscores the need for more uniformity and transparency in the field, and continued vigilance over cost estimates in CUAs on the part of analysts, reviewers, and journal editors.

A Comparison of HU12 and HU13 Utility Scores in Alzheimer's Disease

Purpose. The Health Utilities Index (HUI) is a generic, multiattribute, preference-based health-status classification system. The HUI Mark 3 (HU13) differs from the earlier HUI2 by modifying attributes and allowing more flexibility for capturing high levels of impairment. The authors compared HUI2 and HU13 scores of patients with Alzheimers disease (AD) and caregivers, and contrasted results of a cost-effectiveness analysis of new drugs for AD using the two systems. Methods. In a cross-sectional study of 679 AD patient/caregiver pairs, stratified by patients disease stage (questionable/mild/moderate/severe/profound/terminal) and setting (community/assisted living/nursing home), caregivers completed the combined HU12/HU13 questionnaire as proxy respondents for patients and for themselves. Results. Mean (SD) global utility scores for patients were lower on the HU13 (0.22[0.26]) than on the HU12 (0.53 [0.21]). Patient HU13 utility scores ranged from 0.47(0.24) for questionable AD to -0.23 (0.08) for terminal AD, compared with a range of 0.73 (0.15) to 0.14 (0.07) for the HU12. Among the 203 patients in the severe, profound, and terminal stages, 96 (48%) had negative global HU13 utility scores, while none had a negative HU12 score. The utility scores for caregivers were similar on the HUI3 (0.87 [0.14]) and HU12 (0.87 [0.11]). Cost-effectiveness analysis of a new medication to treat AD showed somewhat more favorable results using the HU13. Conclusions. The HU12 and HU13 discriminate well across AD stages. Compared with the HU12, the HUI3 yields lower global utility scores for patients with AD, and more scores for states judged worse than dead. The HUI3 may yield substantially different results from the HU12, particularly for persons who have serious cognitive impairments such as AD. Key words: Health Utility Index; Alzheimers disease ; utility assessment. (Med Decis Making 2000;20:413-422)

An Off-the-Shelf Help List A Comprehensive Catalog of Preference Scores from Published Cost-Utility Analyses

Purpose. The Panel on Cost-Effectiveness in Health and Medicine recommends an organized collection of preference measure values for health states that can be used in cost-utility analyses (CUAs). The authors sought to construct a catalog of preference scores from published CUAs, organize the catalog by clinical categories, and identify methods of preference score assessment. Method. The authors systematically searched Medline and other databases to identify original CUAs published through 1997. Information was abstracted on the health state descriptions, corresponding preference scores, method of preference score elicitation, and the source of the estimate. Results. Two hundred twenty-eight CUAs were appraised. The authors found 949 health states and corresponding preference scores. Most frequently, health states pertained to the circulatory system (21.7%), health states were valued by experts (35.8%), and values were derived through community-based preference scores (23.5%). Conclusion. A catalog of preference scores for health states can be constructed. The catalog (http://www.hsph.harvard.edu/organizations/hcra/cuadatabase/intro.html) may provide a useful reference tool for producers and consumers of CUAs but also underscores the methodologic variation and inconsistencies present in the field.

A Systematic Review of Cost-Effectiveness Studies Reporting Cost-per-DALY Averted.

Introduction Calculating the cost per disability-adjusted life years (DALYs) averted associated with interventions is an increasing popular means of assessing the cost-effectiveness of strategies to improve population health. However, there has been no systematic attempt to characterize the literature and its evolution. Methods We conducted a systematic review of cost-effectiveness studies reporting cost-per-DALY averted from 2000 through 2015. We developed the Global Health Cost-Effectiveness Analysis (GHCEA) Registry, a repository of English-language cost-per-DALY averted studies indexed in PubMed. To identify candidate studies, we searched PubMed for articles with titles or abstracts containing the phrases “disability-adjusted” or “DALY”. Two reviewers with training in health economics independently reviewed each article selected in our abstract review, gathering information using a standardized data collection form. We summarized descriptive characteristics on study methodology: e.g., intervention type, country of study, study funder, study perspective, along with methodological and reporting practices over two time periods: 2000–2009 and 2010–2015. We analyzed the types of costs included in analyses, the study quality on a scale from 1 (low) to 7 (high), and examined the correlation between diseases researched and the burden of disease in different world regions. Results We identified 479 cost-per-DALY averted studies published from 2000 through 2015. Studies from Sub-Saharan Africa comprised the largest portion of published studies. The disease areas most commonly studied were communicable, maternal, neonatal, and nutritional disorders (67%), followed by non-communicable diseases (28%). A high proportion of studies evaluated primary prevention strategies (59%). Pharmaceutical interventions were commonly assessed (32%) followed by immunizations (28%). Adherence to good practices for conducting and reporting cost-effectiveness analysis varied considerably. Studies mainly included formal healthcare sector costs. A large number of the studies in Sub-Saharan Africa addressed high-burden conditions such as HIV/AIDS, tuberculosis, neglected tropical diseases and malaria, and diarrhea, lower respiratory infections, meningitis, and other common infectious diseases. Conclusion The Global Health Cost-Effectiveness Analysis Registry reveals a growing and diverse field of cost-per-DALY averted studies. However, study methods and reporting practices have varied substantially.

Assessing the Value of Treatment to Address Various Symptoms Associated with Multiple Sclerosis: Results from a Contingent Valuation Study.

BackgroundAlthough it is well recognized that people with multiple sclerosis (MS) may experience impairments in addition to limited mobility, there has been little effort to study their relative importance to patients with the condition. The objective of this study was to assess patient preferences for addressing various MS symptoms.MethodsThis study was conducted at Tufts Medical Center, Boston, Massachusetts. We developed a national online survey of MS patients and neurologists to estimate the value each group places on treating specific MS symptoms. Each respondent was presented with two randomly selected scenarios with different symptoms and treatments. MS patients were asked about their own preferences, whereas neurologists were asked to consider what a patient of theirs would do or think in each scenario. We used a bidding game approach to elicit respondents’ willingness to pay (WTP) for the treatments.ResultsTo treat mobility alone, WTP for MS patients averaged US

The quality of reporting in published cost-utility analyses, 1976-1997.

410–US

Are pharmaceuticals cost-effective? A review of the evidence

520 per month, depending on the scenario. For paired symptoms, MS patients would pay most to treat mobility and upper limb function (US

Modeling for Health Care and Other Policy Decisions: Uses, Roles, and Validity

525/month) or mobility and cognition (US

A Comprehensive League Table of Cost-Utility Ratios and a Sub-table of "Panel-worthy" Studies

514/month), somewhat less to treat mobility and eyesight (US