Economic Evaluations of Screening Programs for Chronic Kidney Disease: A Systematic Review.

OBJECTIVES: The aim of this review is to appraise and assimilate evidence from studies that have reported on the cost-effectiveness of screening programs for chronic kidney disease (CKD). METHODS: The study protocol was registered on International Prospective Register of Systematic Reviews (PROSPERO). The final search was conducted on 18 January 2023 using 7 databases. Screening of articles, data extraction, and quality assessment was performed by 2 independent reviewers. The ISPOR-AMCP-NPC checklist was used to assess the credibility of the included studies. RESULTS: From 4948 retrieved studies, a final total of 20 studies were included in the qualitative synthesis. Studies found that screening in diabetic populations was cost-effective (n = 8, 57%) or even cost-saving (n = 6, 43%). Four studies (67%) found that screening in hypertensive populations was also cost-effective. For the general population, findings were inconsistent across studies in which many found screening to be cost-effective (n = 11, 69%), some cost-saving (n = 2, 12%), and others not cost-effective (n = 3, 19%). The most influential parameters identified were prevalence of CKD and cost of screening. CONCLUSIONS: Screening for CKD in patients with diabetes or hypertension is recommended from a cost-effectiveness point of view. For the general population, despite some inconsistent findings, the majority of studies demonstrated that screening in this population is cost-effective, depending mainly on the prevalence and the costs of screening. Healthcare decision makers need to consider the prevalence, stratification strategies, and advocate for lower screening costs to reduce the burden on healthcare budgets and to make screening even more favorable from the health-economic perspective.

The PID Odyssey 2030: outlooks, unmet needs, hurdles, and opportunities - proceedings from the IPOPI global multi-stakeholders' summit (June 2022).

IPOPI held its first Global Multi-Stakeholders' Summit on 23-24 June 2022 in Cascais, Portugal. This IPOPI initiative was designed to set the stage for a stimulating forward-thinking meeting and brainstorming discussion among stakeholders on the future priorities of the PID community. All participants were actively engaged in the entire Summit, bringing provocative questions to ensure a high level of discussion and engagement, and partnered in identifying the outlooks, unmet needs, hurdles and opportunities of PIDs for 2030. The topics that were covered include diagnosis (e.g., newborn screening [NBS], genomic sequencing- including ethical aspects on the application of genomics on NBS, the role of more accurate and timely diagnostics in impacting personalized management), treatment (e.g., the therapeutic evolution of immunoglobulins in a global environment, new therapies such as targeted therapies, new approaches in curative therapies), the interactions of Primary ID with Secondary ID, Autoinflammatory Diseases and other diseases as the field experiences an incessant evolution, and also the avenues for research in the field of humanities and human sciences such as Patient-Reported Outcome Measures (PROMs), Patient-Reported Experience Measures (PREMs), and Health-Related Quality Of Life (HRQoL). During this meeting, all participants contributed to the drafting of recommendations based on our common understanding of the future opportunities, challenges, and scenarios. As a collection of materials, perspectives and summaries, they are succinct and impactful and may help determine some of the next key steps for the PID community.

An Updated Analysis of the Impact of HPV Vaccination Based on Long-term Effectiveness in the Netherlands.

INTRODUCTION: Vaccination against human papillomavirus (HPV) is considered the most effective strategy to protect women from cervical cancer. Three HPV vaccines are currently licensed in Europe and, although they are generally supported by favorable health economic outcomes, current models fall short in predicting vaccination benefits. Here, we aim to re-evaluate the health benefits of HPV vaccination, using updated long-term effectiveness data and emphasizing quality of life losses related to pre-cancer disease and treatment. METHODS: We used a static Markov model that compared "only screening" (includes unvaccinated girls) and "vaccination" (assumes 100% vaccination coverage with the bivalent HPV vaccine). A lifetime cohort of 100,000 uninfected 12-year-old girls was included, in which the number of cases with cervical intraepithelial neoplasia grade 2 or higher/3 (CIN2+, CIN3), cervical cancer, and cervical cancer deaths per scenario were determined. Furthermore, the reduction in major excisional procedures, the preterm deliveries averted, and the related gain in quality-adjusted life years (QALYs) due to vaccination were estimated. RESULTS: The bivalent vaccine showed larger reductions in CIN2+, CIN3, cervical cancer cases, cervical cancer deaths, and major excisional treatments, after including long-term efficacy and effectiveness data, compared to previous data. Moreover, we observed an increased amount of QALYs gained due to prevention of major excisional treatment and the negative side effects related to it. CONCLUSIONS: Updated health economic models for HPV vaccination, using updated and long-term effectiveness data and including prevention of treatment-related side effects, demonstrate a substantial additional positive effect on vaccination outcomes. Indeed, extrapolation of the bivalent HPV vaccine's updated long-term effectiveness data against HPV-related cervical diseases shows that the positive effects of vaccination may be more substantial than previously estimated. There is a graphical abstract available for this article.

Cervical cancer is one of the most common cancers among women, and the most effective strategy for its prevention is vaccination against HPV infection. Several studies have predicted the benefits of vaccination; however, most of them fall short due to a lack of long-term data and treatment impact. The aim of this study is to re-evaluate the benefits of vaccination with the bivalent vaccine in the Netherlands using updated longer-term data and benefits from preventing treatment.We used a cost-effectiveness model to compare two scenarios: only screening and vaccination plus screening. We included 100,000 12-year-old girls in the model and compared the following outcomes: number of individuals with benign cervical lesions, number of individuals with cervical cancer, number of deaths, reduction in treatment after vaccination, premature births avoided after vaccination, and quality of life gains.We found that the bivalent vaccine showed larger reductions in pre-cancerous lesions (CIN2+, CIN3), cervical cancer cases, cervical cancer deaths, and major excisional treatments, compared to the results of previously published cost-effectiveness analyses when new longer-term data were included. The prevention of treatment for the lesions represents a significant added value for vaccination.Our modeling study confirms the protective effect of the bivalent vaccine on cervical cancer. Moreover, it reflects a substantial additional value of vaccination compared to the benefits of vaccination that have been shown before.

Rationale and development of an e-health application to deliver patient-centered care during treatment for recently diagnosed multiple myeloma patients: pilot study of the MM E-coach.

BACKGROUND: Patients with multiple myeloma (MM) increasingly face complicated treatment regimens. E-health may support patients and healthcare providers in enhancing a patient-centered healthcare approach. Therefore, we aimed to develop a patient-centered multi-modality e-health application, to assess the application for usability and end-user experiences. METHODS: The application was developed following an iterative "action-based" methodology using the design thinking approach. Key end users participated, and relevant stakeholders were consulted in the development process. First, the care pathway was evaluated, the focus of development was determined, and a solution ideated during recurring multidisciplinary meetings. Second, a prototype was tested and improved. Third, a subsequent prototype was evaluated during a pilot study with patients and healthcare professionals on usability, usage, and experiences. RESULTS: The multi-modality application, named the "MM E-coach," consisted of a newly developed medication module, patient-reported outcome (PRO) questionnaire assessments, a messaging service, alerts, information provision, and a personal care plan. The median system usability score was 60 on a scale of 0-100. Patients appreciated the medication overview, healthcare professionals appreciated the outpatient clinic preparation module, and both appreciated the messaging service. Additional recommendations for improvement mostly revolved around the flexibility of functionalities and look and feel of the application. CONCLUSIONS: The MM E-coach has the potential to provide patient-centered care by supporting patients and caregivers during MM treatment and is a promising application to be implemented in the MM care pathway. A randomized clinical trial was initiated to study its clinical effectiveness.

Value-based health care in Western countries: a scoping review on the implementation of patient-reported-outcomes sets for hospital-based interventions.

INTRODUCTION: For implementation of the value-based health-care (VBHC) concept, use of patient-reported outcome measures (PROMs), patient-reported experience measures (PREMs), and clinical outcomes is crucial. The aim of this study was to summarize published studies on implemented PROMs, PREMs, and clinical-outcomes sets in health-care practice. AREAS COVERED: A scoping review was conducted by using PubMed and Embase. Our study focused on implementation examples of patient-reported outcome sets in Western countries' hospitals. Included papers were analyzed on content, in particular concerning PROMs, PREMs, and clinical outcomes. We also assessed differences between diseases, categorized as patient-reported outcomes in curative, chronic, and palliative treatments in the hospital. EXPERT OPINION: A total of 20 studies were found that presented VBHC implementation examples. Results illustrate the disconnection between the development of PROMs and PREMs and the implementation phase, with implementation still in infancy. Hospital organizations should enhance organization for the implementation of VBHC. It is crucial that leading examples of successful VBHC serve as blueprints for implementation, with the participation of all relevant stakeholders. Affordability and sustainability of health care can be enhanced by scaling up successful VBHC-interventions on population levels.

Economic evaluation of orphan drug Lutetium-Octreotate vs. Octreotide long-acting release for patients with an advanced midgut neuroendocrine tumour in the Netherlands.

OBJECTIVES: Multiple studies showed positive effects of Lutetium-Octreotate (LO) treatment in neuroendocrine tumours. LO has been used in the Netherlands since the 1980s and recently received the orphan status shortly after the acquisition by Novartis. Since then, the official list price has increased sixfold. From a value-based pricing perspective, we analysed the impact of the increase in price on the incremental cost-effectiveness ratio (ICER) of LO treatment compared to optimal best supportive care, a high dose of Octreotide long-acting release (O-LAR), using the clinical data of the NETTER-1 trial. METHODS: A Markov model was developed to evaluate the costs per quality-adjusted life-year (QALY) for LO treatment compared to O-LAR from the healthcare perspective. A scenario analysis was conducted to compare the cost-effectiveness with the initial and increased price level of the LO-treatment. RESULTS: At the increased price level, the cost-effectiveness analysis rendered a deterministic ICER of €53,500 per QALY, while at the initial pricing, the ICER was €19,000 per QALY. The probabilistic sensitivity analysis (PSA) showed that LO had a high probability of being cost-effective at both the increased and initial price level, considering a cost-effectiveness threshold of €80,000. CONCLUSIONS: Even at the increased price level, LO treatment can still be considered cost-effective using the applicable Dutch willingness-to-pay threshold of 80,000 euro per QALY. Considering the public scrutiny in relation to this price increase, these outcomes raise the question whether traditional cost-effectiveness methods are sufficient in fully capturing the societal acceptance of prices of new medicines.

Multi-criteria decision analysis to prioritize the introduction of new vaccines in Indonesia by using the framework of the strategic multi-attribute ranking tool for vaccines (SMART vaccines).

Background: Decision-making processes regarding new vaccine prioritizations are complex. The objective of this study was to prioritize the introduction of new vaccines in Indonesia.Methods: A multi-criteria decision analysis (MCDA) was applied in this study. A preliminary data collection form was developed to collect country-specific data in relation to 30 pre-defined attributes. In particular, an open-ended questionnaire was conducted among targeted respondents from global level, national level and vaccine manufacturers, which were involved in the financial flows of new vaccine procurement in Indonesia. For setting new vaccines priorities, targeted respondents were asked to assign weight on 10 selected criteria.Results: Top 3 attributes with the highest weight from respondents were premature deaths averted per year, incident cases prevented per year, and cost-effectiveness. Applying criteria scores and weight assessment, the result showed that PCV, rotavirus, HPV, and JE would be on the 1st, 2nd, 3rd, and 4th rank for setting new vaccine priority in Indonesia. There was a significant difference score (p value <0.05) between all these vaccines.Conclusions: PCV, rotavirus and HPV vaccines should be more prioritized than JE vaccine. This ranking is in line with the WHO's priority list, which potentially illustrates the validity and usefulness of our MCDA-approach.

Cost-effectiveness of hepatitis C virus screening, and subsequent monitoring or treatment among pregnant women in the Netherlands.

BACKGROUND: The prevalence of diagnosed chronic hepatitis C virus (HCV) infection among pregnant women in the Netherlands is 0.26%, yet many cases remain undiagnosed. HCV screening and treatment of pregnant HCV carriers could reduce the burden of disease and limit vertical transmission from mother to child. We assessed the impact of HCV screening and subsequent treatment with new direct-acting antivirals (DAAs) among pregnant women in the Netherlands. METHODS: An HCV natural history Markov transition state model was developed, to evaluate the public-health and economic impact of HCV screening and treatment. Besides all 179,000 pregnant women in the Netherlands (cohort 1), we modelled 3 further cohorts: all 79,000 first-time pregnant women (cohort 2), 33,000 pregnant migrant women (cohort 3) and 16,000 first-time pregnant migrant women (cohort 4). Each cohort was analyzed in various scenarios: i no intervention, i.e., the current practice, ii screen-and-treat, i.e., the most extensive approach involving treatment of all individuals found HCV-positive, and iii screen-and-treat/monitor, i.e., a strategy involving treatment of symptomatic (F1-F4) patients and follow-up of asymptomatic (F0) HCV carriers with subsequent treatment only at progression. RESULTS: For all cohorts, comparison between scenarios (ii) and (i) resulted in ICERs between €9,306 and €10,173 per QALY gained and 5 year budget impacts varying between €6,283,830 and €19,220,405. For all cohorts, comparison between scenarios (iii) and (i) resulted in ICERs between €1,739 and €2,749 per QALY gained and budget impacts varying between €1,468,670 and €5,607,556. For all cohorts, the ICERs (scenario iii versus ii) involved in delayed treatment of asymptomatic (F0) HCV carriers varied between €56,607 and €56,892, well above the willingness-to-pay (WTP) threshold of €20,000 per QALY gained and even above a threshold of €50,000 per QALY gained. CONCLUSION: Universal screening for HCV among all pregnant women in the Netherlands is cost-effective. However, it would be reasonable to consider smaller risk groups in view of the budget impact of the intervention.

Increasing the information provided by probabilistic sensitivity analysis: The relative density plot.

BACKGROUND: Results of probabilistic sensitivity analyses (PSA) are frequently visualized as a scatterplot, which is limited through overdrawing and a lack of insight in relative density. To overcome these limitations, we have developed the Relative Density plot (PSA-ReD). METHODS: The PSA-ReD combines a density plot and a contour plot to visualize and quantify PSA results. Relative density, depicted using a color gradient, is transformed to a cumulative probability. Contours are then plotted over regions with a specific cumulative probability. We use two real-world case studies to demonstrate the value of the PSA-ReD plot. RESULTS: The PSA-ReD method demonstrates proof-of-concept and feasibility. In the real-world case-studies, PSA-ReD provided additional visual information that could not be understood from the traditional scatterplot. High density areas were identified by color-coding and the contour plot allowed for quantification of PSA iterations within areas of the cost-effectiveness plane, diminishing overdrawing and putting infrequent iterations in perspective. Critically, the PSA-ReD plot informs modellers about non-linearities within their model. CONCLUSIONS: The PSA-ReD plot is easy to implement, presents more of the information enclosed in PSA data, and prevents inappropriate interpretation of PSA results. It gives modelers additional insight in model functioning and the distribution of uncertainty around the cost-effectiveness estimate.

A novel method for predicting the budget impact of innovative medicines: validation study for oncolytics.

BACKGROUND: High budget impact (BI) estimates of new drugs have led to decision-making challenges potentially resulting in restrictions in patient access. However, current BI predictions are rather inaccurate and short term. We therefore developed a new approach for BI prediction. Here, we describe the validation of our BI prediction approach using oncology drugs as a case study. METHODS: We used Dutch population-level data to estimate BI where BI is defined as list price multiplied by volume. We included drugs in the antineoplastic agents ATC category which the European Medicines Agency (EMA) considered a New Active Substance and received EMA marketing authorization (MA) between 2000 and 2017. A mixed-effects model was used for prediction and included tumor site, orphan, first in class or conditional approval designation as covariates. Data from 2000 to 2012 were the training set. BI was predicted monthly from 0 to 45 months after MA. Cross-validation was performed using a rolling forecasting origin with e^|Ln(observed BI/predicted BI)| as outcome. RESULTS: The training set and validation set included 25 and 44 products, respectively. Mean error, composed of all validation outcomes, was 2.94 (median 1.57). Errors are higher with less available data and at more future predictions. Highest errors occur without any prior data. From 10 months onward, error remains constant. CONCLUSIONS: The validation shows that the method can relatively accurately predict BI. For payers or policymakers, this approach can yield a valuable addition to current BI predictions due to its ease of use, independence of indications and ability to update predictions to the most recent data.

Affordability of oncology drugs: accuracy of budget impact estimations.

Background: In many countries, Budget Impact (BI) informs reimbursement decisions. Evidence has shown that decision-makers have restricted access based on high BI estimates but studies show that BI estimates are often inaccurate. Objective: To assess the accuracy of BI estimations used for informing access decisions on oncology drugs in the Netherlands. Study Design: Oncology products for which European Medicines Agency Marketing Authorisation was granted between 1-1-2000 and 1-10-2017 were selected. Observed BI data were provided by FarmInform. BI estimates were extracted from the reimbursement dossiers of the Dutch Healthcare Institute. Products without an estimated BI in the reimbursement dossier were excluded. Accuracy is defined as the ratio observed BI/estimated BI. Setting: General community, the Netherlands. Results: Ten products were included in the base case analysis. Mean accuracy was 0.64 and observed BI deviated by more than 40% and 100% from the estimated BI for 4 and 5 products, respectively. For all products together, €141 million BI was estimated and €82 million BI was observed, a €59 million difference. Conclusions: The findings indicate that BI estimates for oncology drugs in the Netherlands are inaccurate. The role and use of BI in reimbursement decisions for these potentially life-saving drugs should therefore be considered carefully, as well as BI estimation methodology.

A Genotype-Guided Strategy for Oral P2Y12 Inhibitors in Primary PCI.

BACKGROUND: It is unknown whether patients undergoing primary percutaneous coronary intervention (PCI) benefit from genotype-guided selection of oral P2Y12 inhibitors. METHODS: We conducted a randomized, open-label, assessor-blinded trial in which patients undergoing primary PCI with stent implantation were assigned in a 1:1 ratio to receive either a P2Y12 inhibitor on the basis of early CYP2C19 genetic testing (genotype-guided group) or standard treatment with either ticagrelor or prasugrel (standard-treatment group) for 12 months. In the genotype-guided group, carriers of CYP2C19*2 or CYP2C19*3 loss-of-function alleles received ticagrelor or prasugrel, and noncarriers received clopidogrel. The two primary outcomes were net adverse clinical events - defined as death from any cause, myocardial infarction, definite stent thrombosis, stroke, or major bleeding defined according to Platelet Inhibition and Patient Outcomes (PLATO) criteria - at 12 months (primary combined outcome; tested for noninferiority, with a noninferiority margin of 2 percentage points for the absolute difference) and PLATO major or minor bleeding at 12 months (primary bleeding outcome). RESULTS: For the primary analysis, 2488 patients were included: 1242 in the genotype-guided group and 1246 in the standard-treatment group. The primary combined outcome occurred in 63 patients (5.1%) in the genotype-guided group and in 73 patients (5.9%) in the standard-treatment group (absolute difference, -0.7 percentage points; 95% confidence interval [CI], -2.0 to 0.7; P<0.001 for noninferiority). The primary bleeding outcome occurred in 122 patients (9.8%) in the genotype-guided group and in 156 patients (12.5%) in the standard-treatment group (hazard ratio, 0.78; 95% CI, 0.61 to 0.98; P = 0.04). CONCLUSIONS: In patients undergoing primary PCI, a CYP2C19 genotype-guided strategy for selection of oral P2Y12 inhibitor therapy was noninferior to standard treatment with ticagrelor or prasugrel at 12 months with respect to thrombotic events and resulted in a lower incidence of bleeding. (Funded by the Netherlands Organization for Health Research and Development; POPular Genetics ClinicalTrials.gov number, NCT01761786; Netherlands Trial Register number, NL2872.).

On discounting of health gains from human papillomavirus vaccination: effects of different approaches.

OBJECTIVES: Discounting has long been a matter of controversy in the field of health economic evaluations. How to weigh future health effects has resulted in ongoing discussions. These discussions are imminently relevant for health care interventions with current costs but future benefits. Different approaches to discount health effects have been proposed. In this study, we estimated the impact of different approaches for discounting health benefits of human papillomavirus (HPV) vaccination. METHODS: An HPV model was used to estimate the impact of different discounting approaches on the present value of health effects. For the constant discount approaches, we varied the discount rate for health effects ranging from 0% to 4%. Next, the impact of relevant alternative discounting approaches was estimated, including hyperbolic, proportional, stepwise, and time-shifted discounting. RESULTS: The present value of health effects gained through HPV vaccination varied strongly when varying discount rates and approaches. The application of the current Dutch guidelines resulted in a present value of health effects that was eight or two times higher than that produced when using the proportional discounting approach or when using the internationally more common 4% discount rate for health effects, respectively. Obviously, such differences translate into large variations in corresponding incremental cost-effectiveness ratios. CONCLUSION: The exact discount rate and approach chosen in an economic evaluation importantly impact the projected value of health benefits of HPV vaccination. Investigating alternative discounting approaches in health-economic analysis is important, especially for vaccination programs yielding health effects far into the future. Our study underlines the relevance of ongoing discussions on how and at what rates to discount.

Screen-and-treat strategies for albuminuria to prevent cardiovascular and renal disease: cost-effectiveness of nationwide and targeted interventions based on analysis of cohort data from the Netherlands.

BACKGROUND: Albuminuria is a marker for renal and cardiovascular (CV) risk, allowing early diagnosis of subjects with elevated renal and CV risk. OBJECTIVE: This study aimed to estimate the cost-effectiveness and budget impact of various population-based screen-and-treat scenarios for elevated albuminuria levels (ie, microalbuminuria) in the Netherlands. METHODS: A multistate transition Markov model was developed to simulate the natural course of albuminuria-based disease progression to dialysis and occurrence of CV events. Several population-based strategies directed at screening for elevated albuminuria were evaluated. These strategies depended on urinary albumin concentration (UAC), urinary albumin excretion (UAE), and age. Transition probabilities were derived from the observational community-based Prevention of Renal and Vascular End Stage Disease (PREVEND) cohort study. Health care costs (in year-2008 euros) and life-years gained were calculated over an 8-year period. In the base-case analysis, we analyzed screening for and treatment of microalbuminuria. Screening for microalbuminuria involved prescreening for UAC >or=20 mg/L, followed by a confirmation test for UAE >or=30 mg/d. Other options based on combinations of albuminuria for UAC prescreening (no prescreening, and >or=10, >or=20, >or=100, and >or=200 mg/L) and UAE confirmation test (>or=15, >or=30, and >or=300 mg/d) for treatment were investigated in scenario analyses. Furthermore, these various strategies based on UAC and UAE values were analyzed in different subgroups based on age (all ages, aged >or=50 years, and aged >or=60 years). RESULTS: The PREVEND study included 8592 Dutch residents aged 28 to 75 years at the time of initial screening. Among a hypothetical cohort of 1000 subjects identified and treated in the base-case analysis, it was estimated (based on PREVEND follow-up data) that, in the screening/treatment and no-screening scenarios, 76 versus 124 CV events occurred, 16 versus 27 CV deaths, and 3 versus 5 dialysis cases, respectively. The per-person difference in net costs for screening was calculated at euro926 (euro2003 vs euro1077), and prevention of CV deaths was estimated to gain 0.0421 discounted life-year per person. Correspondingly, the cost-effectiveness was estimated at euro22,000 per life-year gained. In the base-case analysis, probabilistic sensitivity analysis indicated that the likelihood of cost-effectiveness of a screen-and-treat strategy was 54%, 90%, and 95% for a maximum acceptable cost-effectiveness threshold of euro20,000, euro50,000, and euro80,000 per life-year gained, respectively. Higher albuminuria thresholds for screening and start of treatment further improved the cost-effectiveness but reduced the overall health gains achieved. Limiting screening to those subjects aged >or=50 and >or=60 years resulted in more favorable cost-effectiveness compared with population-based screening without age restriction. CONCLUSIONS: Our analyses suggest the potentially favorable cost-effectiveness of population-based screening for albuminuria in the general Dutch population. The results offer health care decision-makers new tools for considering actual implementation of such screening.

Cost-effectiveness of screening for albuminuria with subsequent fosinopril treatment to prevent cardiovascular events: A pharmacoeconomic analysis linked to the prevention of renal and vascular endstage disease (PREVEND) study and the prevention of renal and vascular endstage disease intervention trial (PREVEND IT).

OBJECTIVE: This study estimated the cost-effectiveness,from the Dutch health care perspective, of screening for albuminuria in the general Dutch population to prevent cardiovascular events (CVEs) with subsequent angiotensin-converting enzyme inhibitor treatment, using data from the Prevention of REnal and Vascular ENdstage Disease Intervention Trial (PREVEND IT). METHODS: PREVEND IT was a single-center, double-blind, randomized, placebo-controlled trial with a 2 x 2 factorial design within the larger observational Prevention of REnal and Vascular ENdstage Disease (PREVEND) study. The PREVEND IT study was conducted to assess the effects of fosinopril 20 mg and pravastatin 40 mg on CVEs in subjects with specific inclusion criteria: urinary albumin excretion (UAE) rate in the range from 15 to 300 mg/d, blood pressure <160/100 mm Hg, and plasma cholesterol level <8.0 mmol/L. Cost-effectiveness estimates for the Dutch population were expressed in euros (2002; 1 euro = US 1.01 dollars) as net costs per life-year gained (LYG) in the baseline and sensitivity (stochastic) analyses. RESULTS: Data were assessed for 864 subjects, with a mean (SD) follow-up of 46 (7) months. CVEs occurred in 45 (5.2%) subjects. Subjects who received fosinopril had a 40% lower incidence of CVEs than subjects in the placebo group (3.9% vs 6.5%, respectively; P = NS). The cost-effectiveness of screening for albumnuria was determined to be euro 16,700/LYG for the study population. Stochastic analysis indicated that the probability of the cost-effectiveness being below the suggested Dutch threshold of euro 20,000/LYG was 59% in the baseline analysis. The probability of cost-effectiveness below euro 20,000/LYG would increase to 91% if only subjects with UAE >50 mg/d were treated with fosinopril. Limiting the screening to subjects aged >50 years and >60 years also improved cost-effectiveness. CONCLUSIONS: The results of our study suggest that screening the general Dutch population for albuminuria and subsequently treating those found positive with fosinopril may be cost-effective compared with no screening and adopting the Dutch health care perspective. However, confirmation from larger multicenter trials is needed.

Pharmacoeconomic review of recombinant human DNase in the management of cystic fibrosis.

For the treatment of patients with cystic fibrosis, recombinant human deoxyribonuclease I is widely used. Deoxyribonuclease I has a positive effect on lung function and the number of hospitalizations. Deoxyribonuclease I is currently administered by nebulization, which is an inefficient administration method. For expensive drugs, such as deoxyribonuclease I, dry powder inhalation would be advantageous due to increased deposition efficiency, patient mobility and compliance. Furthermore, a significant cost reduction may be obtained. The current status of deoxyribonuclease I in the management of cystic fibrosis was investigated and special attention given to the developments in delivery systems, such as dry powder inhalation. It is estimated that if dry powder inhalation of deoxyribonuclease I could be used, a reduction in the cost-effectiveness ratio of approximately 40% can be obtained as compared with nebulization.