Mark S. Thompson

Auranofin therapy and quality of life in patients with rheumatoid arthritis. Results of a multicenter trial

In a six-month, randomized, double-blind study at 14 centers, auranofin (3 mg twice daily) was compared with placebo in the treatment of patients with classic or definite rheumatoid arthritis. All patients had unremitting disease for at least the previous six months and at least three months of therapy with nonsteroidal anti-inflammatory drugs (NSAIDs). NSAIDs, oral steroids, and analgesics were allowed throughout the trial. Efficacy was analyzed in 154 patients who received auranofin and 149 who received placebo. To reflect an expanded view of outcome assessment, the measures used included some 20 nontraditional measures of functional performance, pain, global impression, and utility (worth or value) in addition to five standard clinical measures of rheumatoid synovitis (e.g., number of tender joints). The nontraditional measures were mainly in the form of structured questionnaires administered by trained interviewers. To minimize the statistical problem of multiple comparisons, most of the measures were grouped into four composites--clinical (standard measures), functional, global, and pain--and the treatment effect for each composite was tested at the 0.0125 level of significance. Auranofin was superior to placebo in the clinical (p = 0.003), functional (p = 0.001), and global (p = 0.007) composites and trended similarly in the pain composite (p = 0.021). Individual measures within the composites consistently favored auranofin. Other measures, not part of the composites, also favored auranofin, including a patient utility measure designed for this study, the PUMS (p = 0.002). Results confirm the hypothesis that the favorable effect of auranofin on clinical synovitis is accompanied by improvements across a range of outcomes relevant to the patients quality of life.

Feasibility of willingness-to-pay measurement in chronic arthritis.

Quantification of nonmonetary aspects of disease is a major challenge for economic analysts. Using the amounts of money recipients are willing to pay for nonmonetary benefits has theoretical appeal, but it has proven difficult to implement. Difficulties encountered include noncomprehension by subjects, misrepresentation of preferences, extraneous determinants of answers, and ethical concerns. In a preliminary exploration of feasibility, 184 patients with osteoarthritis and rheumatoid arthritis were asked their willingness to pay (WTP) for hypothetical complete cure. With minimal pressure put on the patients to respond, 27 percent gave plausible answers. People with more schooling, with paid employment, or who were having more treatments for their arthritis were more likely to respond. Patients were willing on average to pay 17 percent of family income for arthritis cure. Methods for measuring WTP are being strengthened and may soon play an important role in health services research.

A randomized controlled trial of quality assurance in sixteen ambulatory care practices

A crossover randomized controlled trial of cycles of quality assurance in 16 primary care (8 medical, 8 pediatric) group practices was conducted. Of four medical and four pediatric tasks important to patient outcome, two were randomly assigned to experimental intervention (a quality assurance cycle), and two were also measured and used as blinded controls for each medical or pediatric group practice. Task performance was measured in each group for 12 months prior to, 9 months during, and 9 months after the experimental intervention, using as a performance score the percentage of evaluation criteria failed of those applicable to a case. As a result of quality assurance intervention, quality of performance was significantly improved in two of the tasks (P<0.0001, with 6.7, and 9.8 percentage points improvement), and marginally improved in one task (P=0.06, 5.7 percentage points improvement). Surprisingly, tasks with lower perceived effect on patient health (low physician motivation) had greater improvement in quality. Unimproved tasks were associated with the perceived need for delivery system changes beyond the immediate control of the individual practitioner.

Quality Assurance in Eight Adult Medicine Group Practices

Four evaluations of ambulatory medical care tasks were developed for use in quality assurance. The evaluations used medical records data and explicit criteria incorporating branching logic. They were implemented in eight general medicine provider groups in two teaching hospitals and six related health centers. Agreement with criteria among 316 provider responses to questionnaires varied from 57% to 100%. The percentage of cases with one or more variation from evaluation criteria, confirmed on peer review to have a deficiency in care, ranged by task from 6% to 42%, with substantial variation between sites. Physician reviewers from each site varied in leniency. Numbers of actions taken to correct deficiencies ranged by site and task from zero to six. Multisite evaluations revealed differences in performance and efforts to improve that are not apparent when each site conducts its own evaluations. More uniformly effective and impartial quality assurance is needed to correct some important deficiencies in care observed in this study.

Decision-Analytic Determination of Study Size The Case of Electronic Fetal Monitoring

There is uncertainty about the efficacy of electronic fetal monitoring (EFM) in lowering perinatal mortality among births at low prospective risk. A randomized controlled trial offers the greatest promise of reducing this uncertainty. Haphazard methods of evaluating decision making have prevailed in the past and have led to study sizes too small to estimate effects on perinatal mortality. Statistical methods can determine the study size necessary to meet statistical parameters. Choice of these parameters is, however, somewhat arbitrary. Decision-analytic methods calculate the expected value of information (EVI) as the likely worth of future decision guidance. The optimal size, cost, and focus for an evaluation study can then be taken as those maximizing the net EVI after consideration of study cost. This methodology indicates that, in evaluating EFM, two randomly-assigned groups of roughly 180,000 births each should be studied. This would achieve net expected societal benefits estimated at

Cost-Effectiveness Analysis in Health Program Evaluation

118 million at a cost of roughly

Health Versus Money: Value Judgments in the Perspective of Decision Analysis

22 million. The optimal study size is somewhat sensitive to analytic parameters. If feasible, a superior dynamic strategy is to allow study findings to determine the ultimate study size.

The Cost of Quality Assurance in Medicine

The growing demand for prospective evaluation enhances the popularity of cost-effective ness analysis, a technique for identifying best uses of scarce resources. Defined in diverse ways during its short history, cost-effectiveness analysis is now seen as the evaluative comparison of monetary and nonmonetary dimensions of impact. The cost-effectiveness ratio for health programs divides monetary effects by health effects. Decisions on com peting alternative programs should be resolved by regarding cost-effectiveness ratios on the differences between programs.

A cost-effectiveness analysis of screening for hepatitis B surface antigen in India.

An important, but largely uninvestigated, value trade-off balances marginal nonhealth consumption against marginal medical care. Benefit-cost analysts have traditionally, if not fully satisfactorily, dealt with this issue by valuing health gains by their effects on productivity. Cost-effectiveness analysts compare monetary and health effects and leave their relative valuations to decision makers. A decision-analytic model using the satisfaction or utility gained from nonhealth consumption and the level of health enables one to calculate willingness to pay--a theoretically superior way of assigning monetary values to effects for benefit-cost analysis-and to determine minimally acceptable cost-effectiveness ratios. Examples show how a decision-analytic model of utility can differentiate medical actions so essential that failure to take them would be considered negligent from actions so expensive as to be unjustifiable, and can help to determine optimal legal arrangements for compensation for medical malpractice.

Cost-effectiveness of screening for hypo- and hyperthyroidism in India.

Cost analysis has beenfrequently neglected in program evaluations but is currently of high relevance in policy decisions on quality assurance in medicine. The Ambulatory Care Medical Audit Demonstration (A CMAD) Project implemented and evaluated a program of medical record-based quality assurance in eleven sites for nine medical topics. Total direct costs for the project were