Improving the Cost-efficiency of Preventive Chemotherapy: Impact of New Diagnostics on Stopping Decisions for Control of Schistosomiasis.

BACKGROUND: Control of schistosomiasis (SCH) relies on the regular distribution of preventive chemotherapy (PC) over many years. For the sake of sustainable SCH control, a decision must be made at some stage to scale down or stop PC. These "stopping decisions" are based on population surveys that assess whether infection levels are sufficiently low. However, the limited sensitivity of the currently used diagnostic (Kato-Katz [KK]) to detect low-intensity infections is a concern. Therefore, the use of new, more sensitive, molecular diagnostics has been proposed. METHODS: Through statistical analysis of Schistosoma mansoni egg counts collected from Burundi and a simulation study using an established transmission model for schistosomiasis, we investigated the extent to which more sensitive diagnostics can improve decision making regarding stopping or continuing PC for the control of S. mansoni. RESULTS: We found that KK-based strategies perform reasonably well for determining when to stop PC at a local scale. Use of more sensitive diagnostics leads to a marginally improved health impact (person-years lived with heavy infection) and comes at a cost of continuing PC for longer (up to around 3 years), unless the decision threshold for stopping PC is adapted upward. However, if this threshold is set too high, PC may be stopped prematurely, resulting in a rebound of infection levels and disease burden (+45% person-years of heavy infection). CONCLUSIONS: We conclude that the potential value of more sensitive diagnostics lies more in the reduction of survey-related costs than in the direct health impact of improved parasite control.

Prophylactic anticoagulation after minimally invasive hysterectomy for endometrial cancer: a cost-effectiveness analysis.

OBJECTIVE: To determine our institutional rate of venous thromboembolism (VTE) following minimally invasive surgery for endometrial cancer and to perform a cost-effectiveness analysis of extended prophylactic anticoagulation after minimally invasive staging surgery for endometrial cancer. METHODS: All patients with newly diagnosed endometrial cancer who underwent minimally invasive staging surgery from January 1, 2017 to December 31, 2020 were identified retrospectively, and clinicopathologic and outcome data were obtained through chart review. Event probabilities and utility decrements were obtained through published clinical data and literature review. A decision model was created to compare 28 days of no post-operative pharmacologic prophylaxis, prophylactic enoxaparin, and prophylactic apixaban. Outcomes included no complications, deep vein thrombosis (DVT), pulmonary embolism, clinically relevant non-major bleeding, and major bleeding. We assumed a willingness-to-pay threshold of $100 000 per quality-adjusted life year (QALY) gained. RESULTS: Three of 844 patients (0.36%) had a VTE following minimally invasive staging surgery for endometrial cancer. In this model, no pharmacologic prophylaxis was less costly and more effective than prophylactic apixaban and prophylactic enoxaparin over all parameters examined. When all patients were assigned prophylaxis, prophylactic apixaban was both less costly and more effective than prophylactic enoxaparin. If the risk of DVT was ≥4.8%, prophylactic apixaban was favored over no pharmacologic prophylaxis. On Monte Carlo probabilistic sensitivity analysis for the base case scenario, no pharmacologic prophylaxis was favored in 41.1% of iterations at a willingness-to-pay threshold of $100 000 per QALY. CONCLUSIONS: In this cost-effectiveness model, no extended pharmacologic anticoagulation was superior to extended prophylactic enoxaparin and apixaban in clinically early-stage endometrial cancer patients undergoing minimally invasive surgery. This model supports use of prophylactic apixaban for 7 days post-operatively in select patients when the risk of DVT is 4.8% or higher.

Costs and Cost-Effectiveness of Malaria Control Interventions: A Systematic Literature Review.

OBJECTIVES: To systematically review the literature on the unit cost and cost-effectiveness of malaria control. METHODS: Ten databases and gray literature sources were searched to identify evidence relevant to the period 2005 to 2018. Studies with primary financial or economic cost data from malaria endemic countries that took a provider, provider and household, or societal perspective were included. RESULTS: We identified 103 costing studies. The majority of studies focused on individual rather than combined interventions, notably insecticide-treated bed nets and treatment, and commonly took a provider perspective. A third of all studies took place in 3 countries. The median provider economic cost of protecting 1 person per year ranged from $1.18 to $5.70 with vector control and from $0.53 to $5.97 with chemoprevention. The median provider economic cost per case diagnosed with rapid diagnostic tests was $6.06 and per case treated $9.31 or $89.93 depending on clinical severity. Other interventions did not share enough similarities to be summarized. Cost drivers were rarely reported. Cost-effectiveness of malaria control was reiterated, but care in methodological and reporting standards is required to enhance data transferability. CONCLUSIONS: Important information that can support resource allocation was reviewed. Given the variability in methods and reporting, global efforts to follow existing standards are required for the evidence to be most useful outside their study context, supplemented by guidance on options for transferring existing data across settings.

Variability of treatment modalities and intensity in patients with severe haemophilia A on prophylaxis: Results from the Italian national registry.

BACKGROUND: A shift from a standard to a personalized prophylaxis has been increasingly adopted in patients with severe haemophilia A (SHA). This approach has raised the likelihood of a significant variability in the prophylactic approaches and the relative Factor VIII (FVIII) consumptions. The aim of our study was to assess the treatment variability of SHA patients without inhibitors and on prophylaxis regimen in Italy. MATERIAL AND METHODS: Data reported in the National Registry of Congenital Coagulopathies (NRCC) were analysed to assess treatment distribution within SHA patients without inhibitors, focussing on FVIII consumption in 2017, associated with prophylaxis regimen. The analysis was stratified based on age groups and Italian regions to describe the variability of FVIII consumption in Italy. RESULTS: In 2017, the Registry reported the therapeutic plans of 1068 SHA patients without inhibitors on prophylaxis. The mean (95% CI) individual consumption ranges from 123 127 IU (99 736-146 518) in the age group 0-6 years to 345 000 IU (336 000-354 000) in the age group >20 years. A significant FVIII consumption variability was identified within the adult population. Regions with less than 50 patients reported the higher variability in mean FVIII consumption per patient-year within the different age groups. Similar difference in FVIII consumption variability was reported also in the age groups comparing "low," "middle" and "high" patient volume regions. DISCUSSION: A reliable estimation of FVIII consumption for patients' treatment is necessary to manage and plan the appropriate budget and keep treatment's costs affordable. However, without the implementation of a methodology aiming to assess the overall value produced by these FVIII consumptions, the scenario will keep driven by FVIII consumptions, its costs and the budget available. An effort by haemophilic community, haemophilia treatment centres and institutions is required to develop and share this cultural shift in improving haemophilia management and assessment.

Cost-effectiveness of district-wide seasonal malaria chemoprevention when implemented through routine malaria control programme in Kita, Mali using fixed point distribution.

BACKGROUND: Seasonal malaria chemoprevention (SMC) is a strategy for malaria control recommended by the World Health Organization (WHO) since 2012 for Sahelian countries. The Mali National Malaria Control Programme adopted a plan for pilot implementation and nationwide scale-up by 2016. Given that SMC is a relatively new approach, there is an urgent need to assess the costs and cost effectiveness of SMC when implemented through the routine health system to inform decisions on resource allocation. METHODS: Cost data were collected from pilot implementation of SMC in Kita district, which targeted 77,497 children aged 3-59 months. Starting in August 2014, SMC was delivered by fixed point distribution in villages with the first dose observed each month. Treatment consisted of sulfadoxine-pyrimethamine and amodiaquine once a month for four consecutive months, or rounds. Economic and financial costs were collected from the provider perspective using an ingredients approach. Effectiveness estimates were based upon a published mathematical transmission model calibrated to local epidemiology, rainfall patterns and scale-up of interventions. Incremental cost effectiveness ratios were calculated for the cost per malaria episode averted, cost per disability adjusted life years (DALYs) averted, and cost per death averted. RESULTS: The total economic cost of the intervention in the district of Kita was US $357,494. Drug costs and personnel costs accounted for 34% and 31%, respectively. Incentives (payment other than salary for efforts beyond routine activities) accounted for 25% of total implementation costs. Average financial and economic unit costs per child per round were US $0.73 and US $0.86, respectively; total annual financial and economic costs per child receiving SMC were US $2.92 and US $3.43, respectively. Accounting for coverage, the economic cost per child fully adherent (receiving all four rounds) was US $6.38 and US $4.69, if weighted highly adherent, (receiving 3 or 4 rounds of SMC). When costs were combined with modelled effects, the economic cost per malaria episode averted in children was US $4.26 (uncertainty bound 2.83-7.17), US $144 (135-153) per DALY averted and US $ 14,503 (13,604-15,402) per death averted. CONCLUSIONS: When implemented at fixed point distribution through the routine health system in Mali, SMC was highly cost-effective. As in previous SMC implementation studies, financial incentives were a large cost component.

Cost-effectiveness of taurolidine locks to prevent recurrent catheter-related blood stream infections in adult patients receiving home parenteral nutrition: a 2-year mirror-image study.

BACKGROUND & AIMS: The use of long-term taurolidine locks (LTTL) seems to be effective in preventing catheter-related blood stream infections (CRBSI), especially in patients on home parenteral nutrition (HPN). This work targets the cost-effectiveness of LTTL in a cohort of adult HPN patients. METHODS: A monocentric mirror-image design study was conducted in our referral centre among long-term HPN patients experiencing recurrent CRBSI. From 7th January 2011, LTTL were started after the third CRBSI episode within 12 months. CRBSI data was prospectively collected until 7th January 2013, in the same way as it had retrospectively been done before initiating LTTL. A cost-effective analysis was conducted to estimate the incremental costs and effects on CRBSI with LTTL. The efficacy of LTTL on CRBSI rate was assessed over 1000 days of catheter use. RESULTS: A total of 31,100 catheter days were analysed in 37 patients (median [interquartile range (IQR)]) aged 58 [42-68] years. The mean ± SD proven CRBSI rate was 3.18 ± 3.51 per 1000 catheter days before the introduction of LTTL and 0.39 ± 1.50 per 1000 catheter days after its introduction (p < 0.0001). Considering both proven and probable CRBSI requiring hospital management, LTTL reduced by (mean [bootstrap CI 95%]) -2.63 [-3.26 to -2.06] infections per patient (from 2.89 [2.31 to 3.49] before to 0.26 [0.13 to 0.41] after) as well as incremental costs by -7 258 [-10 450 to -4 016] € (from 11 176 [8 004 to 14 968] € before to 3 918 [2 390 to 5 445] € after). CONCLUSION: Implementing LTTL to prevent recurrent CRBSI is cost-effective by dramatically decreasing their incidence.

A cost-effectiveness analysis of the prophylaxis versus on-demand regimens in severe hemophilia A patients under 12 years old in southern Iran.

OBJECTIVE: This study aimed to compare the cost-effectiveness of these two regimens in hemophilia A patients, under-12-years-old in southern Iran. METHODS: A cost-effectiveness study comparing prophylaxis versus on-demand was conducted on 34 hemophilia patients (24 and 10 patients were on the prophylaxis and on-demand regimens respectively) in 2017. The Markov model was used to estimate the economic and clinical outcomes. The costs were collected from the societal perspective, and the utility criterion was the 'quality adjusted life year (QALY)' indicator. The required data were collected using a researcher-made cost checklist, the EQ5D standard questionnaire and Hemophilia Joint Health Score. The probabilistic sensitivity analysis (PSA) was performed to determine the robustness of the results. RESULTS: The means of costs, joint health score and QALY in the prophylaxis regimen were $478,963.1 purchasing power parity (PPP), 96.67, and 11.98 respectively, and in the on-demand regimen were $521,797.2 PPP, 93.46 and 10.99 respectively. The PSA confirmed the robustness of the model's results. The results of the scatter plots and acceptability curves showed that the prophylaxis regimen in 97% of the simulations for the thresholds below $20950 PPP was more cost-effective than on-demand regimen. CONCLUSION: Prophylaxis regimen showed the lower costs and higher effectiveness and utility in comparison with the on-demand regimen. It is recommended that prophylaxis should be considered as the standard care for treatment of hemophilic patients.

Assessing the cost and quality-of-life impact of on-demand-only medications for adults with hereditary angioedema.

Background: Novel subcutaneous (SC) prophylactic therapies are transforming the treatment landscape of hereditary angioedema (HAE). Although questions are being raised about their cost, little attention has been paid to the cost and quality of life (QoL) impact of using on-demand-only medications. Objective: We assessed the overall economic burden of on-demand-only treatment for HAE and compared patient QoL with patients who received novel SC prophylactic therapies. Methods: US Hereditary Angioedema Association members were invited to complete an anonymous online survey to profile attack frequency, treatment use, and the presence of comorbidities as well as economic and socioeconomic variables. We modeled on-demand treatment costs by using net pricing of medications in 2018, indirect patient and caregiver costs, and attack-related direct billed costs for emergency department admissions, physician office visits, and/or hospitalizations. QoL was assessed by using the Angioedema Quality of Life questionnaire. Results: A total of 1225 patients (31.4%) responded. Of these, 737 adults with HAE (type I or II) met the inclusion criteria and completed the survey. Per patient/year direct costs associated with modeled on-demand-only treatment totaled $363,795, with additional indirect socioeconomic costs of $52,576 per patient/year. The greatest improvement in QoL was seen in patients who used novel SC prophylactic therapies, with a 59.5% (p < 0.01) improvement in median impairment scores versus on-demand-only treatment. In addition, patients who used novel SC prophylactic therapies reported a 77% reduction in the number of attacks each year when compared with those who used on-demand-only treatment. Conclusion: Our real-world patient data showed the cost and QoL burden of HAE treatment with on-demand-only therapy. Use of novel SC prophylaxis can lead to sizeable reductions in attack frequency and statistically significant and clinically relevant improvements in QoL. These data could be useful to clinicians and patients as they consider therapy options for patients with HAE.

Seasonal malaria chemoprevention in the Sahel subregion of Africa: a cost-effectiveness and cost-savings analysis.

BACKGROUND: The intermittent administration of seasonal malaria chemoprevention (SMC) is recommended to prevent malaria among children aged 3-59 months in areas of the Sahel subregion in Africa. However, the cost-effectiveness and cost savings of SMC have not previously been evaluated in large-scale studies. METHODS: We did a cost-effectiveness and cost-savings analysis of a large-scale, multi-country SMC campaign with sulfadoxine-pyrimethamine plus amodiaquine for children younger than 5 years in seven countries in the Sahel subregion (Burkina Faso, Chad, Guinea, Mali, Niger, Nigeria, and The Gambia) in 2016. The financial and economic costs were analysed from the programmatic perspective and are reported in 2016 US$ for each country. The estimated numbers of averted malaria cases, deaths, and disability-adjusted life-years (DALYs) were based on numbers of SMC treatments administered and modelled malaria transmission. Cost savings were calculated from a programmatic perspective corresponding to the diagnostic and treatment costs for malaria cases averted. FINDINGS: The total cost of SMC for all seven countries was $22·8 million, and the weighted average economic cost of administering four monthly SMC cycles was $3·63 per child (ranging from $2·71 in Niger to $8·20 in The Gambia). Based on 80% modelled effectiveness of SMC, the incremental economic cost per malaria case averted ranged from $2·91 in Niger to $30·73 in The Gambia; the cost per severe case averted ranged from $119·63 in Niger to $506·00 in The Gambia; the cost per death averted ranged from $533·56 in Niger to $2256·92 in The Gambia; and the cost per DALY averted (discounted by 3%) ranged from $18·66 in Niger to $78·91 in The Gambia. The estimated total economic cost savings to the health systems in all seven countries were US$66·0 million and the total net economic cost savings were US$43·2 million. INTERPRETATION: SMC is a low-cost and highly cost-effective intervention that contributes to substantial cost savings by reducing malaria diagnostic and treatment costs among children. FUNDING: Unitaid.

Effectiveness of seasonal malaria chemoprevention at scale in west and central Africa: an observational study.

BACKGROUND: Seasonal malaria chemoprevention (SMC) aims to prevent malaria in children during the high malaria transmission season. The Achieving Catalytic Expansion of SMC in the Sahel (ACCESS-SMC) project sought to remove barriers to the scale-up of SMC in seven countries in 2015 and 2016. We evaluated the project, including coverage, effectiveness of the intervention, safety, feasibility, drug resistance, and cost-effectiveness. METHODS: For this observational study, we collected data on the delivery, effectiveness, safety, influence on drug resistance, costs of delivery, impact on malaria incidence and mortality, and cost-effectiveness of SMC, during its administration for 4 months each year (2015 and 2016) to children younger than 5 years, in Burkina Faso, Chad, The Gambia, Guinea, Mali, Niger, and Nigeria. SMC was administered monthly by community health workers who visited door-to-door. Drug administration was monitored via tally sheets and via household cluster-sample coverage surveys. Pharmacovigilance was based on targeted spontaneous reporting and monitoring systems were strengthened. Molecular markers of resistance to sulfadoxine-pyrimethamine and amodiaquine in the general population before and 2 years after SMC introduction was assessed from community surveys. Effectiveness of monthly SMC treatments was measured in case-control studies that compared receipt of SMC between patients with confirmed malaria and neighbourhood-matched community controls eligible to receive SMC. Impact on incidence and mortality was assessed from confirmed outpatient cases, hospital admissions, and deaths associated with malaria, as reported in national health management information systems in Burkina Faso and The Gambia, and from data from selected outpatient facilities (all countries). Provider costs of SMC were estimated from financial costs, costs of health-care staff time, and volunteer opportunity costs, and cost-effectiveness ratios were calculated as the total cost of SMC in each country divided by the predicted number of cases averted. FINDINGS: 12 467 933 monthly SMC treatments were administered in 2015 to a target population of 3 650 455 children, and 25 117 480 were administered in 2016 to a target population of 7 551 491. In 2015, among eligible children, mean coverage per month was 76·4% (95% CI 74·0-78·8), and 54·5% children (95% CI 50·4-58·7) received all four treatments. Similar coverage was achieved in 2016 (74·8% [72·2-77·3] treated per month and 53·0% [48·5-57·4] treated four times). In 779 individual case safety reports over 2015-16, 36 serious adverse drug reactions were reported (one child with rash, two with fever, 31 with gastrointestinal disorders, one with extrapyramidal syndrome, and one with Quincke's oedema). No cases of severe skin reactions (Stevens-Johnson or Lyell syndrome) were reported. SMC treatment was associated with a protective effectiveness of 88·2% (95% CI 78·7-93·4) over 28 days in case-control studies (2185 cases of confirmed malaria and 4370 controls). In Burkina Faso and The Gambia, implementation of SMC was associated with reductions in the number of malaria deaths in hospital during the high transmission period, of 42·4% (95% CI 5·9 to 64·7) in Burkina Faso and 56·6% (28·9 to 73·5) in The Gambia. Over 2015-16, the estimated reduction in confirmed malaria cases at outpatient clinics during the high transmission period in the seven countries ranged from 25·5% (95% CI 6·1 to 40·9) in Nigeria to 55·2% (42·0 to 65·3) in The Gambia. Molecular markers of resistance occurred at low frequencies. In individuals aged 10-30 years without SMC, the combined mutations associated with resistance to amodiaquine (pfcrt CVIET haplotype and pfmdr1 mutations [86Tyr and 184Tyr]) had a prevalence of 0·7% (95% CI 0·4-1·2) in 2016 and 0·4% (0·1-0·8) in 2018 (prevalence ratio 0·5 [95% CI 0·2-1·2]), and the quintuple mutation associated with resistance to sulfadoxine-pyrimethamine (triple mutation in pfdhfr and pfdhps mutations [437Gly and 540Glu]) had a prevalence of 0·2% (0·1-0·5) in 2016 and 1·0% (0·6-1·6) in 2018 (prevalence ratio 4·8 [1·7-13·7]). The weighted average economic cost of administering four monthly SMC treatments was US$3·63 per child. INTERPRETATION: SMC at scale was effective in preventing morbidity and mortality from malaria. Serious adverse reactions were rarely reported. Coverage varied, with some areas consistently achieving high levels via door-to-door campaigns. Markers of resistance to sulfadoxine-pyrimethamine and amodiaquine remained uncommon, but with some selection for resistance to sulfadoxine-pyrimethamine, and the situation needs to be carefully monitored. These findings should support efforts to ensure high levels of SMC coverage in west and central Africa. FUNDING: Unitaid.

Benchmarking the Cost-Effectiveness of Interventions Delaying Diabetes: A Simulation Study Based on NAVIGATOR Data.

OBJECTIVE: To estimate using the UK Prospective Diabetes Study Outcomes Model Version 2 (UKPDS-OM2) the impact of delaying type 2 diabetes onset on costs and quality-adjusted life expectancy using trial participants who developed diabetes in the NAVIGATOR (Nateglinide And Valsartan in Impaired Glucose Tolerance Outcomes Research) study. RESEARCH DESIGN AND METHODS: We simulated the impact of delaying diabetes onset by 1-9 years, utilizing data from the 3,058 of 9,306 NAVIGATOR trial participants who developed type 2 diabetes. Costs and utility weights associated with diabetes and diabetes-related complications were obtained for the U.S. and U.K. settings, with costs expressed in 2017 values. We estimated discounted lifetime costs and quality-adjusted life years (QALYs) with 95% CIs. RESULTS: Gains in QALYs increased from 0.02 (U.S. setting, 95% CI 0.01, 0.03) to 0.15 (U.S. setting, 95% CI 0.10, 0.21) as the imposed time to diabetes onset was increased from 1 to 9 years, respectively. Savings in complication costs increased from $1,388 (95% CI $1,092, $1,669) for a 1-year delay to $8,437 (95% CI $6,611, $10,197) for a delay of 9 years. Interventions costing up to $567-$2,680 and £201-£947 per year would be cost-effective at $100,000 per QALY and £20,000 per QALY thresholds in the U.S. and U.K., respectively, as the modeled delay in diabetes onset was increased from 1 to 9 years. CONCLUSIONS: Simulating a hypothetical diabetes-delaying intervention provides guidance concerning the maximum cost and minimum delay in diabetes onset needed to be cost-effective. These results can inform the ongoing debate about diabetes prevention strategies and the design of future intervention studies.

Leprosy post-exposure prophylaxis in the Indian health system: A cost-effectiveness analysis.

India has the highest burden of leprosy in the world. Following a recent WHO guideline, the Indian National Leprosy Programme is introducing post-exposure prophylaxis with single-dose rifampicin (SDR-PEP) in all high-endemic districts of the country. The aim of this study is to estimate the long-term cost-effectiveness of SDR-PEP in different leprosy disability burden situations. We used a stochastic individual-based model (SIMCOLEP) to simulate the leprosy new case detection rate trend and the impact of implementing contact screening and SDR-PEP from 2016 to 2040 (25 years) in the Union Territory of Dadra Nagar Haveli (DNH) in India. Effects of the intervention were expressed as disability adjusted life years (DALY) averted under three assumption of disability prevention: 1) all grade 1 disability (G1D) cases prevented; 2) G1D cases prevented in PB cases only; 3) no disability prevented. Costs were US$ 2.9 per contact. Costs and effects were discounted at 3%. The incremental cost per DALY averted by SDR-PEP was US$ 210, US$ 447, and US$ 5,673 in the 25th year under assumption 1, 2, and 3, respectively. If prevention of G1D was assumed, the probability of cost-effectiveness was 1.0 at the threshold of US$ 2,000, which is equivalent to the GDP per capita of India. The probability of cost-effectiveness was 0.6, if no disability prevention was assumed. The cost per new leprosy case averted was US$ 2,873. Contact listing, screening and the provision of SDR-PEP is a cost-effective strategy in leprosy control in both the short (5 years) and long term (25 years). The cost-effectiveness depends on the extent to which disability can be prevented. As the intervention becomes increasingly cost-effective in the long term, we recommend a long-term commitment for its implementation.

Cost of interventions to control schistosomiasis: A systematic review of the literature.

BACKGROUND: Schistosomiasis, a disease caused by blood flukes of the genus Schistosoma, belongs to the neglected tropical diseases. Left untreated, schistosomiasis can lead to severe health problems and even death. An estimated 800 million people are at risk of schistosomiasis and 250 million people are infected. The global strategy to control and eliminate schistosomiasis emphasizes large-scale preventive chemotherapy with praziquantel targeting school-age children. Other tools are available, such as information, education, and communication (IEC), improved access to water, sanitation, and hygiene (WASH), and snail control. Despite available evidence of the effectiveness of these control measures, analyses estimating the most cost-effective control or elimination strategies are scarce, inaccurate, and lack standardization. We systematically reviewed the literature on costs related to public health interventions against schistosomiasis to strengthen the current evidence-base. METHODOLOGY: In adherence to the PRISMA guidelines, we systematically searched three readily available electronic databases (i.e., PubMed, WHOLIS, and ISI Web of Science) from inception to April 2019 with no language restrictions. Relevant documents were screened, duplicates eliminated, specific rules on studies to consider were defined, and the eligible studies fully reviewed. Costs of schistosomiasis interventions were classified in three groups: (i) preventive chemotherapy; (ii) preventive chemotherapy plus an individual diagnostic test to identify at-risk population; and (iii) test-and-treat interventions. PRINCIPAL FINDINGS: Fifteen articles met our inclusion criteria. In general, it was hard to compare the reported costs from the different studies due to different approaches used to estimate and classify the costs of the intervention assessed. Costs varied considerably from one study to another, ranging from US$ 0.06 to US$ 4.46 per person treated. The difference between financial and opportunity costs only played a minimal role in the explanation of the costs' variation, even if delivery costs were two times higher in the analyses including economic costs. Most of the studies identified in our systematic review focused on sub-Saharan African countries. CONCLUSIONS/SIGNIFICANCE: The degree of transparency of most of the costing studies of schistosomiasis interventions found in the current review was limited. Hence, there is a pressing need for strategies to improve the quality of cost analyses, and higher reporting standards and transparency that should be fostered by peer-review journal policies. Cost information on these interventions is crucial to inform resource allocation decisions and those regarding the affordability of scaling-up interventions.

Real-world annualized healthcare utilization and expenditures among insured US patients with acute intermittent porphyria (AIP) treated with hemin.

Background and aims: Patients with acute intermittent porphyria (AIP) may suffer from acute non-specific attacks that often result in hospitalizations or emergency room (ER) visits. Prior to the recent approval of givosiran (November 2019), hemin was the only FDA-approved therapy for AIP attacks in the US. Our aim was to estimate the annual healthcare utilization and expenditures for AIP patients treated with hemin using real-world data.Methods: Patients with ≥1 hemin claim and confirmed AIP diagnosis - 1 inpatient claim or 2 outpatient claims ≥30 d apart for AIP (2015-2017) or acute porphyria (prior to 2015) - were identified in MarketScan administrative claims dataset between 2007 and 2017. Continuous enrolment for ≥6 months from confirmed diagnosis was required. A secondary analysis ("active disease population") limited the sample to adult patients with ≥3 attacks or 10 months of prophylactic use of hemin within a 12-month pre-index period. AIP-related care was defined by hemin use during an attack (daily glucose and/or hemin use) or prophylaxis (non-attack hemin use). Outcomes were annualized and expenditures were inflated to 2017.Results: Across 10 years, patients with a confirmed AIP diagnosis (N = 8,877) and ≥1 hemin claim (N = 164) were restricted by ≥6 months continuous follow-up (N = 139). AIP patients were mostly female (N = 112; 81%), had median age of 40 and 3 years average follow-up. Annualized average total expenditures for AIP-related care were $113,477. Annualized average all-cause (any diagnosis) hospitalizations were statistically significantly lower for patients treated with hemin prophylaxis vs. acute treatment (1.0 vs. 2.1; p < .001). In the secondary analysis (N = 27), annualized average total expenditures for AIP-related care were higher ($187,480).Conclusions: For AIP patients treated with hemin, patients treated for acute attacks may use a greater number of resources compared to patients treated prophylactically.

Clinical and economic burden of pre-emptive therapy of cytomegalovirus infection in hospitalized allogeneic hematopoietic cell transplant recipients.

Cytomegalovirus (CMV) infection remains a major complication after allogeneic hematopoietic cell transplantation (allo-HCT). We conducted a retrospective study to determine the clinical and economic burden of pre-emptive therapy (PET) for CMV infection in 100 consecutive hospitalized adult CMV positive serostatus allo-HCT recipients and compared their hospitalization cost with allo-HCT recipients hospitalized with graft vs host disease without CMV infection (control group) and across 19 US cancer centers for hospitalized patients with CMV infection between 2012 and 2015 (Vizient database). A total of 192 CMV episodes of PET for CMV infection occurred within 1 year post-HCT. PET consisted of ganciclovir (41% of episodes), foscarnet (40%), and valganciclovir (38%) with the longest average length of stay in foscarnet-treated patients (41 days). The average direct cost per patient admitted for PET was $116 976 (range: $7866-$641 841) compared with $12 496 (range: $2004-$43 069) in the control group (P < .0001). The total direct cost per encounter was significantly higher in patients treated with foscarnet and had nephrotoxicity ($284 006) compared with those who did not ($112 195). The average cost amongst the 19 US cancer centers, including our institution, was $42 327 with major disparities in cost and clinical outcomes. PET for CMV infection is associated with high economic burden in allo-HCT recipients.

Cost analysis of ganciclovir and foscarnet in recipients of allogeneic hematopoietic cell transplant with cytomegalovirus viremia.

BACKGROUND: Ganciclovir (GCV) and foscarnet (FOS) are the most commonly used antivirals for preemptive treatment of cytomegalovirus (CMV) viremia in recipients of allogeneic hematopoietic cell transplantation (alloHCT). The current literature indicates similar efficacy between these agents. Thus, the primary consideration for choice of initial anti-CMV treatment is the safety profile, time period after alloHCT, and concern of myelosuppression or renal dysfunction. METHODS: Herein, we retrospectively reviewed medical records of 124 alloHCT recipients who received GCV or FOS between April 27, 2014, and December 31, 2015, during the first year post-transplant. Healthcare resource use included drug, hospitalization, home health, dialysis, and growth factor costs. RESULTS: Total duration of therapy was longer in the GCV group (37 days vs 28 days, P = .21) but hospitalization days were similar (9 days) in both groups. The total treatment cost was significantly lower in the GCV group ($38 100 vs $59 400, P < .05). CONCLUSION: Preemptive anti-CMV therapy is associated with major healthcare resource costs, which were greater in patients who required FOS than those who were treated with GCV.

Cost-Benefit Limitations of Extended, Outpatient Venous Thromboembolism Prophylaxis Following Surgery for Crohn's Disease.

BACKGROUND: Patients with Crohn's disease are at increased risk of postoperative venous thromboembolism. Historically, extended outpatient prophylaxis has not met conventional measures of societal cost-benefit advantage. However, extended prophylaxis for patients with Crohn's disease may be more cost-effective because of the patients' high thrombotic risk and long life expectancy. OBJECTIVE: This study aimed to assess the cost-effectiveness of extended prophylaxis in patients with Crohn's disease after abdominal surgery. DESIGN: A decision tree model was used to assess the incremental cost-effectiveness and cost per case averted with extended-duration venous thromboembolism prophylaxis following abdominal surgery. SETTING: The risk of a postdischarge thrombotic event, age at surgery, type of thrombotic event, prophylaxis risk reduction, bleeding complications, and mortality were estimated by using existing published sources. PATIENTS: Studied were patients with Crohn's disease versus routine care. INTERVENTION: We constructed a decision analysis to compare costs and outcomes in patients with Crohn's disease postoperatively with and without extended prophylaxis over a lifetime horizon. MAIN OUTCOME MEASURES: Productivity costs ($) and benefits (quality-adjusted life-year) were used to reflect a societal perspective and were time discounted at 3%. Multivariable probabilistic sensitivity analysis accounted for uncertainty in probabilities, costs, and utility weights. RESULTS: With the use of reference parameters, the individual expected societal total cost of care was $399.83 without and $1387.95 with prophylaxis. Preventing a single mortality with prophylaxis would cost $43.00 million (number needed to treat: 39,839 individuals). The incremental cost was $1.90 million per quality-adjusted life-year. Adjusting across a range of scenarios upheld these conclusions 88% of the time. With further sensitivity testing, subpopulations with postdischarge thrombosis rates greater than 4.9% favors postoperative extended-duration venous thromboembolism prophylaxis. LIMITATIONS: Further investigation is needed to determine if specific high-risk individuals can be preemptively identified in the Crohn's surgical population for targeted prophylaxis. CONCLUSION: Extended prophylaxis in patients with Crohn's disease postoperatively is not cost-effective when the cumulative incidence of posthospital thrombosis remains less than 4.9%. These findings are driven by the low absolute risk of thrombosis in this population and the considerable cost of universal treatment. See Video Abstract at http://links.lww.com/DCR/A998. LIMITACIONES DE COSTO-BENEFICIO DE LA PROFILAXIS AMBULATORIA PROLONGADA DEL TROMBOEMBOLISMO VENOSO DESPUÉS DE CIRUGÍA EN CASOS DE ENFERMEDAD DE CROHN:: Los pacientes con enfermedad de Crohn tienen un mayor riesgo de tromboembolismo venoso postoperatorio. Históricamente, la profilaxis ambulatoria prolongada no ha cumplido con las medidas convencionales de ventajas en costo-beneficio para la sociedad. Sin embargo, la profilaxis prolongada en los pacientes con Crohn puede ser más rentable debido al alto riesgo trombótico y a una larga esperanza de vida en estos pacientes.Evaluar la rentabilidad de la profilaxis prolongada en pacientes postoperados de un Crohn.Se utilizó un modelo de árbol de decisión para evaluar el incremento de rentabilidad y el costo por cada caso evitado con la profilaxis prolongada de tromboembolismo venoso después de cirugía abdominal.Se calcularon utilizando fuentes publicadas el riesgo de evento trombótico posterior al alta, la edad del paciente al momento de la cirugía, el tipo de evento trombótico, la reducción del riesgo de profilaxis, las complicaciones hemorrágicas y la mortalidad.Se estudiaron los pacientes de atención rutinaria versus aquellos portadores de Crohn.Construimos un arbol de análisis decisional para comparar costos y resultados de pacientes portadores de Crohn, con y sin profilaxis prolongada en el postoperatorio en un horizonte de por vida.Los costos de productividad ($) y los beneficios (año de vida ajustado por calidad) se utilizaron para reflejar la perspectiva social y se descontaron en el tiempo de un 3%. El análisis de sensibilidad probabilística multivariable dió cuenta de la incertidumbre en las probabilidades, costos y peso de utilidades.Usando parámetros de referencia, el costo total social esperado de la atención individual fue de $ 399.83 sin y $ 1,387.95 con profilaxis. La prevención del deceso de un paciente con profilaxis costaría $ 43.00 millones (valor requerido para tratar: 39,839 individuos). El costo incrementado fue de $ 1.90 millones por año de vida ajustado por la calidad. El ajuste a través de una gama de escenarios confirmó estas conclusiones el 88% del tiempo. Con pruebas de sensibilidad adicionales, las subpoblaciones con tasas de trombosis posteriores al alta fueron superiores al 4,9% y favorecían la profilaxis prolongada del tromboembolismo venoso en el postoperatorio.Se necesita más investigación para determinar si se puede identificar de manera preventiva los individuos específicos de alto riesgo en la población quirúrgica de Crohn en casos de profilaxis dirigida.La profilaxis prolongada en pacientes postoperados de un Crohn no es rentable cuando la incidencia acumulada de trombosis posthospitalaria sigue siendo inferior al 4,9%. Estos hallazgos son impulsados por el bajo riesgo absoluto de trombosis en esta población y el costo considerable del tratamiento universal. Vea el resumen del video en http://links.lww.com/DCR/A998.