Emicizumab prophylaxis for people with hemophilia A: Waste estimation and the Brazilian perspective.

Costs of hemophilia A treatment are increasing. Waste of clotting products should be avoided. To estimate the first-year waste of emicizumab prophylaxis for people with hemophilia A and inhibitors (PwHAi) who failed immune tolerance induction (ITI), in Brazil. We evaluated the manufacturer and the Brazilian Ministry of Health (MoH) protocol-recommended regimens in a budget impact model. The loading dose consisted of 3.0 mg/kg/Q1W for 4 weeks, for both recommendations. The manufacturer maintenance regimens comprised 1.5 mg/kg/Q1W, 3.0 mg/kg/Q2W, and 6.0 mg/kg/Q4W. The MoH protocol maintenance regimen encompassed a hybrid Q1W/Q2W administration, depending on the body weight. The Q4W regimen was not recommended by the MoH protocol. Analyses were performed to estimate waste given its expense based on the World Health Organization body weight range (percentiles [P] 15, 50, and 85). The first-year emicizumab waste was estimated individually and for the disclosed PwHAi who failed ITI (n = 114). The highest emicizumab waste was estimated for the lowest body weights and the Q1W regimen. The Q4W regimen resulted in the lowest emicizumab waste, followed by the MoH protocol regimen. The total reconstituted costs estimated for the PwHAi who failed ITI according to the hybrid MoH protocol ranged from US$32,858,777 (P15) to US$47,186,858 (P85), with emicizumab waste ranging from 7.9 % (US$2,594,515) to 3.7 % (US$1,738,750), respectively. Lost resources due to current protocols for emicizumab prophylaxis for PwHAi who failed ITI in Brazil are considerable. Waste was more pronounced due to lower body weight and shorter administration intervals.

Willingness to pay for a hypothetical malaria vaccine in Brazil: a cross-sectional study and the implications.

Aim: Malaria is an infection caused by protozoa of genus Plasmodium, considered the one associated with increasingly large outbreaks. Methods: A cross-sectional study was conducted with residents in the northern region of Brazil on the willingness to pay (WTP) for a hypothetical vaccine against malaria (effective protection of 80%). Results: Of 616 people interviewed, most interviewees were female (61%) and were employed (97%). The median individual maximum WTP for a hypothetical malaria vaccine was US$11.90 (BRL 50). Conclusion: The northern region of Brazil is one of the largest markets for a malaria vaccine due to its epidemiological relevance. Consequently, economic studies will be important to assist in the assessment of the potential price and value of new vaccines.

Consumer willingness to pay for a hypothetical Chagas disease vaccine in Brazil: a cross-sectional study and the implications.

Aim: Chagas disease is a serious public health problem, endemic in 21 countries in Latin America. A future vaccine can contribute to decreasing the number of cases and its complications. Methods: A cross-sectional study was conducted with residents of the northern region of Brazil, on the willingness to pay for a hypothetical vaccine against Chagas disease (effective protection of 80%). Results: We interviewed 619 individuals and seven were excluded from the analysis and the value of willingness to pay was US$23.77 (100.00 BRL). Conclusion: The Northern region of Brazil is one of the largest markets for this vaccine, due to its epidemiological relevance, so economic studies with this vaccine will be important to assist in the assessment of technologies.

Avoiding Radical Surgery in Elderly Patients With Rectal Cancer Is Cost-Effective.

BACKGROUND: Radical surgery is associated with significant perioperative mortality in elderly and comorbid populations. Emerging data suggest for patients with a clinical complete response after neoadjuvant chemoradiotherapy that a watch-and-wait approach may provide equivalent survival and oncological outcomes. OBJECTIVE: The purpose of this study was to compare the cost-effectiveness of watch and wait and radical surgery for patients with rectal cancer after a clinical complete response following chemoradiotherapy. DESIGN: Decision analytical modeling and a Markov simulation were used to model long-term costs, quality-adjusted life-years, and cost-effectiveness after watch and wait and radical surgery. Sensitivity analysis was used to investigate the effect of uncertainty in model parameters. SETTINGS: A third-party payer perspective was adopted. PATIENTS: Patients included in the study were a 60-year-old male cohort with no comorbidities, 80-year-old male cohorts with no comorbidities, and 80-year-old male cohorts with significant comorbidities. INTERVENTIONS: Radical surgery and watch-and-wait approaches were studied. MAIN OUTCOME MEASURES: Incremental cost, effectiveness, and cost-effectiveness ratio over the entire lifetime of the hypothetical patient cohorts were measured. RESULTS: Watch and wait was more effective (60-year-old male cohort with no comorbidities = 0.63 quality-adjusted life-years (95% CI, 2.48-3.65 quality-adjusted life-years); 80-year-old male cohort with no comorbidities = 0.56 quality-adjusted life-years (95% CI, 0.52-1.59 quality-adjusted life-years); 80-year-old male cohort with significant comorbidities = 0.72 quality-adjusted life-years (95% CI, 0.34-1.76 quality-adjusted life-years)) and less costly (60-year-old male cohort with no comorbidities = $11,332.35 (95% CI, $668.50-$23,970.20); 80-year-old male cohort with no comorbidities = $8783.93 (95% CI, $2504.26-$21,900.66); 80-year-old male cohort with significant comorbidities = $10,206.01 (95% CI, $2762.014-$24,135.31)) independent of patient cohort age and comorbidity. Consequently, watch and wait was more cost-effective with a high degree of certainty (range, 69.6%-89.2%) at a threshold of $50,000/quality-adjusted life-year. LIMITATIONS: Long-term outcomes were derived from modeled cohorts. Analysis was performed for a United Kingdom third-party payer perspective, limiting generalizability to other healthcare contexts. CONCLUSIONS: Watch and wait is likely to be cost-effective compared with radical surgery. These findings strongly support the discussion of organ-preserving strategies with suitable patients.

Addressing challenges for sustainable healthcare in Central and Eastern Europe.

All European countries face increasing challenges in the provision of equitable and comprehensive healthcare for their citizens in view of a number of factors. These include changing demographics and the continual launch of new premium priced medicines. The challenges are even more difficult among Central and Eastern European healthcare systems. Consequently, there is a need for countries to learn from each other to help address some of these challenges and to maintain sustainable systems. This was the basis of the 2-day conference, The Fifth International Conference: Challenges for Efficient Healthcare in Central and Eastern Europe, 9-10 October 2015, Belgrade, Serbia.