**3.3 Evaluation cases of medical value**

This section introduces reports that discuss the socio-economic significance of the spread of lifesaving medical devices and the research and development (R&D) of expensive pharmaceuticals (at the time of 2010).

First, a case of microeconomic valorization of end-stage renal failure is discussed [39]. With the progression of renal impairment in patients with chronic kidney disease, the dysregulation of electrolyte and water metabolism and retention of uremic toxins can significantly impact health status and even threaten life [40]. Treatment with hemodialysis (HD) should target maintaining the amount and composition of body fluids within the normal range. The study subjects were aged >20 years and had received HD for at least 6 months. HD patients were prospectively observed for 36 months, and patient utility was assessed based on the EQ-5D, from which qualityadjusted life years (QALYs) were estimated. Medical costs were calculated based on the medical service fees. Cost-effectiveness, defined as the incremental cost-utility

#### **Figure 11.**

*Utility values (EQ-5D score) during the first 4 weeks of observation and the 36th week. Four-week interval after the classification of primary diseases for end-stage kidney disease (glomerulonephritis, diabetes mellitus, and the whole) [39]. \*p < 0.05, \*\*p < 0.01.*

*Socio-Economic Considerations of Universal Health Coverage: Focus on the Concept… DOI: http://dx.doi.org/10.5772/intechopen.104798*

#### **Figure 12.**

*Change in cost-effectiveness (ICUR) between the first 4 weeks of observation and the 36th four-week interval. \*p < 0.05, \*\*p < 0.01 [39].*

ratio (ICUR), was analyzed socially. A total of 29 patients (mean age; 59.9 13.1 years) undergoing 437 dialysis sessions were analyzed.

Utility-based EQ-5D score was 0.75 0.21, and the estimated total medical cost for 1 year of maintenance HD (MHD) treatment was 45,200 8800 USD. On average, the ICUR was 68,800 44,700 USD/QALY (**Figure 11**). When comparing the ICUR based on the causes of kidney failure, the value for diabetic nephropathy was higher than that for glomerulonephritis (81,700 62,800 vs. 68,200 40,700). The ICUR after 36 months of observation increased mainly in patients below 65 years of age (all P < 0.05; <65, P < 0.01; ≥ 65, not significant) (**Figure 12**). MHD could improve the socio-economic status of older-adult patients with end-stage kidney disease; however, the ICUR for diabetic nephropathy was higher than that for glomerulonephritis (**Table 2**). However, the ICUR does not deteriorate in older-adult patients. Therefore, measures to prevent malnutrition and establish the optimum time per session and frequency of dialysis (i.e., optimal dialysis volume) are necessary to further improve MHD's cost-effectiveness.

The present findings may contribute to the reexamination of the socio-economic value of MHD therapy, which is a lifesaving medical treatment.

Subsequently, a case of socio-economic valuation of a (then) new drug for the refractory nephrotic syndrome was discussed [41]. Nephrotic syndrome is the generic name for the pathological conditions associated with proteinuria (≥3.5g/day), hypoproteinemia, and generalized edema. The disorder is further classified as a


*\*p<0.05. \*\*p<0.01. The data source for this analysis was the mean value over 4 weeks in 2011. BUN. blood urea nitrogen: CI. Confidence interval: Cr. creatinine: SD. Standard deviation: QALYs. Quality-adjusted life years. These values were analyzed by distinguishing between the primary disease of end-stage kidney disease (ESKD), glomerulonephritis, diabetic nephropathy, and others during the first 4 weeks of observation [39].*

#### **Table 2.**

*Cost-effectiveness by utility and cost in patients on maintenance hemodialysis (MHD).*

primary nephrotic syndrome (caused by primary glomerular disease) or secondary nephrotic syndrome (caused by systemic disorders). The syndrome rapidly improves with steroid (e.g., prednisolone) and immunosuppressant (e.g., cyclosporine) treatment. Refractory cases (frequent relapse type, steroid dependence, or steroid resistance) may also occur, requiring steroid therapy for prolonged periods, for which side effects become a major issue. Therefore, there is a need for novel medical strategies to *Socio-Economic Considerations of Universal Health Coverage: Focus on the Concept… DOI: http://dx.doi.org/10.5772/intechopen.104798*

suppress relapse while reducing reliance on steroids. The regimen has not been clinically verified regarding the use of rituximab in patients with steroid-dependent nephrotic syndrome and frequently relapsing nephrotic syndrome. Still, there is a lack of evidence in health economics [42].

Therefore, we conducted a prospective clinical study of 30 patients before (with steroids and immunosuppressants) and after introducing rituximab therapy (**Figure A3**). Relapse rates and total medical expenses were selected as the primary endpoints for treatment effectiveness and treatment costs, respectively. As a secondary endpoint, cost-effectiveness was compared before and after rituximab administration in relation to previous pharmacotherapy. The observation period was 24 months before and after rituximab initiation. The authors demonstrated a statistically significant improvement in the relapse rate, from a mean of 4.30 events before administration to a mean of 0.27 events after administration. Furthermore, a significantly better prognosis emerged in the cumulative avoidance of relapse rate by Kaplan–Meier analysis (p < 0.01) (**Figure 13**). Finally, the total medical costs

**Figure 13.** *Kaplan–Meier curves of the cumulative avoidance rate of the first relapse [41].*

**Figure 14.** *Mutual relationship between urinary protein levels and total medical cost (before and after rituximab therapy) [41].*

decreased from 2923USD to 1280USD per month, and pre-post cost-effectiveness was confirmed to be dominant (**Figure 14**). Thus, treatment with rituximab may be superior to previous pharmacological treatments from a health economics perspective (**Table 3**). Although this study did not directly observe patient utility, the excellent results in recurrence rates suggest an improvement in HRQOL.

As this study indicates the superior cost-effectiveness of rituximab against refractory nephrotic syndrome, health economics is expected to be actively applied to the valuation of technical innovations such as drug discovery.
