Treatment benefit measures

In CEA, the total cost and the total benefits, measured in terms of an efficacy parameter, associated with two or more treatment pathways are added, and the increment is calculated. The incremental costs are then compared (in a ratio) with incremental outcomes (as measured in physical or natural emits). Physical and natural units can include both intermediate (surrogate) clinical endpoints (e.g. millimetres of mercury blood pressure reduction, changes in FEVi) or final endpoints (e.g. deaths averted or life-years gained). In a study that assessed the cost per deaths due to pulmonary embolism averted, Hull and associates reported that subcutaneous administration of... 

One potential difficulty of cost-benefit analysis is that it requires researchers to express an intervention s costs and outcomes in the same units. Thus, monetary values must be associated with years of life lost and morbidity due to disease and with years of life gained and morbidity avoided due to intervention. Expressing costs in this way is obviously difficult in health care analyses. Outcomes (treatment benefits) may be difficult to measure in units of currency. Translating disease and treatment outcomes into monetary measures may be more difficult than translating them into clinical outcome measures, such as years of life saved or years of life saved adjusted for quality. 

The importance of this effort is illustrated in the following hypothetical example. A new therapy is under development that reduces the absolute risk of dying from a chronic disease by 50% as measured in a one-year trial. However, this therapy is not curative. A four-year trial was initiated at the same time as the one-year trial. The first-year results were the same in both the four-year trial and the one-year trial. However, there was an increased risk of death for treatment patients in the second and third year of the four-year trial, and by the end of the third year of the trial the survival rate was identical in the treatment and control arms of the four-year trial. While there was a clear benefit to the new therapy in terms of postponing events from the first year of treatment to later years, the economic assessment of the therapy would suggest a greatly reduced treatment benefit from the four-year trial as compared with the one-year trial. 

In projecting results of short-term trials over patients lifetimes, it is typical to present at least two of the many potential projections of lifetime treatment benefit. A one-time effect model assumes that the clinical benefit observed in the trial is the only clinical benefit received by patients. Under this model, after the trial has ended, the conditional probability of disease progression for patients is the same in both arms of the trial. Given that it is unlikely that a therapy will lose all benefits as soon as one stops measuring them, this projection method generally is pessimistic compared to the actual outcome. A continuous-benefit effect model assumes that the clinical benefit observed in the trial is continued throughout the patients lifetimes. Under this model, the conditional probability of disease progression for treatment and control patients continues at the same rate as that measured in the clinical trial. In contrast to the one-time model, this projection of treatment benefit most likely is optimistic compared to the treatment outcome. 

Measures of treatment benefit for categorical and ordinal data... 

Radiation victims exposed to doses greater than 10-12 Gy have virtually no chance for survival, and are therefore not candidates for definitive care. These patients should receive comfort measures rather than aggressive definitive treatment. Comfort measures should include analgesia, antiemetic agents and antidi-arrheal agents. In addition, these patients, their families and their friends would benefit from psychological support and spiritual care. 

The results of the 2008 European Cooperative Acute Stroke Study (ECASS 111) expanded the 3-h time window for IV thrombolysis and revealed that although safe and effective up to 4.5 h after stroke onset, treatment benefits roughly one-half as many patients as those treated within 3 h [2, 158, 159]. Hence, the ratio between the hemorrhagic risk of treatment and the potential clinical benefit of treatment becomes a more critical consideration as the time window for therapy is expanded with newer IV and lA techniques. It is the mismatch between the size of the infarct core (proportional to hemorrhagic risk) and the size of the ischemic penumbra (proportional to potentially salvageable tissue), as determined by CTP, that provides an imaging measure of this risk-to-benefit ratio. Evidence suggests that core/penumbra mismatch may persist up to 24 h in some patients [160,161]. 

Implementation of cleaner production processes and pollution prevention measures can yield both economic and environmental benefits. The primary treatment technologies afforded to this manufacturing include the following ... 

Your process may produce wastes that cannot be treated on-site, and so must be transported off-site for treatment and disposal. Wastes of this type are usually non-aqueous liquids, sludge, or solids. Often, wastes for off-site disposal are costly to transport and to treat, and represent a third-party liability. Therefore, minimization of these wastes yields a direct cost benefit, both present and future. Measure the quantity and note the composition of any wastes associated with your process that need to be sent for off-site disposal. Record your results in a table or an appropriate spreadsheet. 

Cost-benefit analysis (CBA) is unique among economic evaluations in that it addresses the extent to which a particular course of action, such as a drug treatment or a hospital admission, is economically or socially worth while in the broadest sense. A CBA measures... 

Knapp MR) (1998). Measuring the economic benefit of treatment with atypical antipsychotics. Eur Psychiatry (suppl. 1), 37-4 5s. 

No clinical treatments other than supportive measures are currently available to enhance elimination of trichloroethylene following exposure. Studies designed to assess the potential risks or benefits of increasing ventilation to enhance pulmonary elimination or of stimulating excretion of trichloroethylene and its decomposition products are needed. 

Nonpharmacologic Therapy Pruritus associated with CKD is difficult to alleviate. It is important to evaluate other potential dermatologic causes of pruritus to maximize the potential for relief. Adequate dialysis is generally the first line of treatment in patients with pruritus. However, this has not been shown to decrease the incidence of pruritus significantly. Maintaining proper nutritional intake, especially with regard to dietary phosphorus and protein intake, may lessen the degree or occurrence of pruritus. Patients who do not attain relief from other measures may benefit from ultraviolet B phototherapy. 

Hypotension may be related to alterations in levocarnitine levels during dialysis. Patients who have low levels of levocarnitine may benefit from supplementation. Levocarnitine is administered as doses of 20 mg/kg intravenously at the end of each dialysis session. However, levocarnitine should not be used as a first-line agent for the treatment of hypotension because of the significant cost associated with the treatment. Patients receiving levocarnitine should be evaluated every 3 months for response to therapy.47 Other preventive measures that have not been well studied include caffeine, sertraline, or fludrocortisone. 

Although increases in bone mineral density have been reported at other sites, most of the clinically significant fractures occur in the hip or spine, and these sites have become clinically important measures in the trials. These increases in bone mineral density are an important marker of treatment effects and are related to the benefits found in larger trials of decreased fracture risk. 

Recent advances in the treatment of cancer of the colon and rectum now offer the potential to improve patient survival, but for many patients, improved disease- and progression-free survival represent equally important therapeutic outcomes. In the absence of the ability of a specific treatment to demonstrate improved survival, important outcome measures should include the effects of the treatment on patient symptoms, daily activities, performance status, and other quality-of-life indicators. Individualized patient care to balance the risks associated with treatment with the benefits of a specific treatment regimen is necessary to optimize patient outcomes. 

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