Methodological Issues In Modeling Hemophilia Treatment - What We Have Learnt In the Course of a Decade and What We Must Learn In the Decade to Come

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4762-4762
Author(s):  
Martin Strandberg-Larsen
Keyword(s):  
Economic Evaluations ◽  
Treatment Regimens ◽  
Current State ◽  
To Come ◽  
Economic Analyses ◽  
Number Of Publications ◽  
Economic Constraints ◽  
The Cost ◽  
Year 2000 ◽  
Disease Specific

Abstract Abstract 4762 Introduction: The challenges faced by the hemophilia community because of economic constraints will increase in the future. Without disease specific pharmacoeconomic models to inform the decision process important decisions may be taken without taking hemophilia specific issues into account. Objectives: To investigate recent advances and current state-of-the-art knowledge in pharmacoeconomic modeling of hemophilia care we conducted a targeted literature review. Methods: Searches were conducted in MEDLINE®, and NHS Economic Evaluation Database using combinations of disease, and economic-related key words, limited to articles published in English between January 1970 and June 2010 (inclusive). A total of 127 articles (from 284 abstracts retrieved) were selected for detailed consideration. Results: Prior to the year 2000 the focus of the cost side of hemophilia treatment was not given much attention in the scientific literature. However in the last decade pharmacoeconomics have grown in importance and an increasing number of publications specific for this aspect of hemophilia reflect this trend. Still there is limited data on probabilities, costs and outcomes and remarkably few studies comparing QALY estimates of factor VIII prophylaxis in hemophilia. More studies are available specific for the limited inhibitor segment. Recommendations for reporting economic evaluations of hemophilia prophylaxis has been proposed and important ground clearing work has been published on theoretical aspects of hemophilia modeling. Conclusion: Economic analyses in hemophilia care are limited, but are increasingly in demand to examine the costs and outcomes associated with various treatment regimens. Unresolved questions critical to hemophilia remains such as the appropriate weighting of short term vs. future benefits and equity considerations associated with hemophilia being a rare genetic disease affecting children. Trials designed with pharmacoeconomic objectives in mind will be imperative. Data on generic and disease specific quality of life and resource use/cost data will provide whole new insights to how hemophilia impacts the lives of people living with this rare disease and will provide the data needed to assess the total value of different treatment regimens relevant for hemophilia. Disclosures: Strandberg-Larsen: Novo Nordisk A/S: Employment.

scholarly journals Engineered nanomaterials and human health: Part 1. Preparation, functionalization and characterization (IUPAC Technical Report)

2018 ◽  
Vol 90 (8) ◽  
pp. 1283-1324 ◽  
Author(s):  
Vladimir Gubala ◽  
Linda J. Johnston ◽  
Ziwei Liu ◽  
Harald Krug ◽  
Colin J. Moore ◽  
...  
Keyword(s):  
Close Contact ◽  
Consumer Products ◽  
Engineered Nanomaterials ◽  
Diverse Range ◽  
Size Dependent ◽  
Current State ◽  
Technical Report ◽  
Potential Benefits ◽  
To Come ◽  
Number Of Publications

Abstract Nanotechnology is a rapidly evolving field, as evidenced by the large number of publications on the synthesis, characterization, and biological/environmental effects of new nano-sized materials. The unique, size-dependent properties of nanomaterials have been exploited in a diverse range of applications and in many examples of nano-enabled consumer products. In this account we focus on Engineered Nanomaterials (ENM), a class of deliberately designed and constructed nano-sized materials. Due to the large volume of publications, we separated the preparation and characterisation of ENM from applications and toxicity into two interconnected documents. Part 1 summarizes nanomaterial terminology and provides an overview of the best practices for their preparation, surface functionalization, and analytical characterization. Part 2 (this issue, Pure Appl. Chem. 2018; 90(8): 1325–1356) focuses on ENM that are used in products that are expected to come in close contact with consumers. It reviews nanomaterials used in therapeutics, diagnostics, and consumer goods and summarizes current nanotoxicology challenges and the current state of nanomaterial regulation, providing insight on the growing public debate on whether the environmental and social costs of nanotechnology outweigh its potential benefits.

The Development of Rural Water Supply in the Province of Fayoum, Egypt

1993 ◽  
Vol 27 (9) ◽  
pp. 37-46 ◽  
Author(s):  
Abdel Rahman Abu Hamed ◽  
A. M. H. Sannen
Keyword(s):  
Water Supply ◽  
Production Capacity ◽  
Cost Recovery ◽  
Rural Water Supply ◽  
Rural Water ◽  
To Come ◽  
State Of Affairs ◽  
The Cost ◽  
Year 2000

The paper provides a case study of rural water supply in Fayoum. First, the present state-of-affairs is described and analyzed with respect to production, consumption and coverage. A water balance is drawn up and the projected trends up to the year 2000 are indicated. Nearly full coverage has been achieved already but still more than 50% of the population is served by public standposts. Considerable demand for house connections is apparent. Based on the present trend it is expected that by the year 2000, 75% of the population could be served by private connections, provided sufficient production capacity becomes available. Secondly, the focus is on organization and finance with special attention to cost recovery. Estimates for present billing efficiency and the Unaccounted For Water (UFW) percentages are provided. With the perceived trend in the growth of house connections and decline of public taps there is ample potential to reduce UFW, provided an efficient billing and fee collection system is introduced and leakage is controlled. With the reduction of UFW by “technical” measures half of the cost recovery target can be achieved. The other half has to come from tariff increases. Experience is drawn from the Netherlands-supported Fayoum Drinking Water and Sanitation Project (FADWS) which started in 1990.

Gezondheidseconomische evaluaties binnen een preventief beleid: een kritische introductie

2021 ◽  
Author(s):  
A. WERBROUCK ◽  
L. ANNEMANS ◽  
N. VERHAEGHE ◽  
S. SIMOENS
Keyword(s):  
Health Economic ◽  
Cost Effectiveness ◽  
Health Effects ◽  
Quality Adjusted Life Year ◽  
Cost Utility ◽  
Economic Evaluations ◽  
Decision Analytic Model ◽  
To Come ◽  
Economic Analyses ◽  
Preventive Policy

Health-economic evaluations in preventive policy: a critical introduction. The Flemish prevention decree specifies that policy initiatives should maximize health gains at a socially acceptable cost. As health-economic evaluations aim to evaluate the expected health effects as well as the expected costs of interventions, this type of studies can be considered indispensable to come to evidence-based policy. The goal of this paper is to introduce the basic aspects of health-economic evaluations, with a focus on preventive interventions. Although the term cost-effectiveness is often used as an umbrella term, technically speaking there is a distinction between cost-effectiveness studies and cost-utility studies. In the latter case, Belgium and many other countries often use the quality-adjusted life year (QALY) to assess health effects. Health-economic analyses can either be performed alongside a clinical trial, or by the use of a decision-analytic model in which different sources are combined to estimate long-term costs and health effects. How do we handle the effects of prevention in the (sometimes far) future? What is the optimal target group? What about the uncertainty within cost-effectiveness analyses? Can health inequity be incorporated? Several aspects of health-economic evaluations require sufficient attention when analysing preventive policy.

scholarly journals Cost-effectiveness evidence for strategies to promote or support breastfeeding: a systematic search and narrative literature review

2020 ◽  
Vol 20 (1) ◽  
Author(s):  
Elizabeth M. Camacho ◽  
Hannah Hussain
Keyword(s):  
Cost Effectiveness ◽  
Current Knowledge ◽  
Cost Effective ◽  
Systematic Search ◽  
Current Evidence ◽  
Economic Evaluations ◽  
Breastfeeding Promotion ◽  
Published Evidence ◽  
Economic Analyses ◽  
The Cost

Abstract Background Global health policy recommends exclusive breastfeeding until infants are 6 months. Little is known about the cost-effectiveness of breastfeeding promotion strategies. This paper presents a systematic search and narrative review of economic evaluations of strategies to support or promote breastfeeding. The aim of the review is to bring together current knowledge to guide researchers and commissioners towards potentially cost-effective strategies to promote or support breastfeeding. Methods Searches were conducted of electronic databases, including MEDLINE and Scopus, for economic evaluations relevant to breastfeeding, published up to August 2019. Records were screened against pre-specified inclusion/exclusion criteria and quality was assessed using a published checklist. Costs reported in included studies underwent currency conversion and inflation to a single year and currency so that they could be compared. The review protocol was registered on the PROSPERO register of literature reviews (ID, CRD42019141721). Results There were 212 non-duplicate citations. Four were included in the review, which generally indicated that interventions were cost-effective. Two studies reported that breastfeeding promotion for low-birth weight babies in critical care is associated with lower costs and greater health benefits than usual care and so is likely to be cost-effective. Peer-support for breastfeeding was associated with longer duration of exclusivity with costs ranging from £19–£107 per additional month (two studies). Conclusions There is limited published evidence on the cost-effectiveness of strategies to promote breastfeeding, although the quality of the current evidence is reasonably high. Future studies should integrate evaluations of the effectiveness of strategies with economic analyses.

Resource efficiency in deinked pulp production: a review

TAPPI Journal ◽  
2014 ◽  
Vol 13 (11) ◽  
pp. 37-43 ◽  
Author(s):  
LIISA KOTANEN ◽  
MIKA KÖRKKÖ ◽  
ARI ÄMMÄLÄ ◽  
JOUKO NIINIMÄKI
Keyword(s):  
Waste Disposal ◽  
Resource Efficiency ◽  
Raw Material ◽  
Ecological Strategy ◽  
Recycled Paper ◽  
Paper Production ◽  
Recycled Pulp ◽  
Current State ◽  
The Cost ◽  
Pulp Production

The use of recovered paper as a raw material for paper production is by far the most economical and ecological strategy for the disposal of waste paper. However, paper production from recovered paper furnish generates a great amount of residues, and the higher the demand requirements for the end product, the higher the amount of rejected material. The reason for this is that the selectivity of the deinking process is limited; therefore, some valuable components are also lost in reject streams. The rejection of usable components affects the economics of recycled paper production. As the cost of waste disposal continues to increase, this issue is becoming more and more severe. This paper summarizes the current state of the resource efficiency in recycled pulp production and provides information on the volumes of rejected streams and the usable material within them. Various means to use these reject streams are also discussed, including the main findings of a recent thesis by the main author. This review summarizes current internal and external use of reject streams generated in the deinking operations.

scholarly journals Olfaction and Aging: A Review of the Current State of Research and Future Directions

i-Perception ◽  
2021 ◽  
Vol 12 (3) ◽  
pp. 204166952110203
Author(s):  
Jonas K. Olofsson ◽  
Ingrid Ekström ◽  
Maria Larsson ◽  
Steven Nordin
Keyword(s):  
Warning Signs ◽  
Future Directions ◽  
Current State ◽  
Challenging Tasks ◽  
State Of Research ◽  
To Come ◽  
Health Cognition ◽  
Olfactory Abilities ◽  
And Training

Olfaction, the sense of smell, is characterized by a notable age-dependency such that aging individuals are more likely to have poor olfactory abilities. These impairments are considered to be mostly irreversible and as having potentially profound effects on quality of life and food behavior, as well as constituting warning signs of mortality, cognitive dysfunction, and dementia. Here, we review the current state of research on aging and olfaction, focusing on five topics which we regard to be of particular relevance for the field: nutrition and health, cognition and dementia, mortality, environment and genetics, and training-based enhancement. Under each of these headlines, we provide a state-of-the-art overview and discuss gaps in our knowledge which might be filled by further research. Understanding how olfactory abilities are diminished in aging, and how they may be alleviated or recovered, involves a set of challenging tasks for researchers in the years to come.

scholarly journals CAR-NK Cells in the Treatment of Solid Tumors

2021 ◽  
Vol 22 (11) ◽  
pp. 5899
Author(s):  
Ewa Wrona ◽  
Maciej Borowiec ◽  
Piotr Potemski
Keyword(s):  
Nk Cells ◽  
Solid Tumors ◽  
Hematological Malignancies ◽  
Toxicity Profile ◽  
Multiple Sources ◽  
Eligibility Criteria ◽  
Hematological Neoplasms ◽  
Current State ◽  
Car T ◽  
The Cost

CAR-T (chimeric antigen receptor T) cells have emerged as a milestone in the treatment of patients with refractory B-cell neoplasms. However, despite having unprecedented efficacy against hematological malignancies, the treatment is far from flawless. Its greatest drawbacks arise from a challenging and expensive production process, strict patient eligibility criteria and serious toxicity profile. One possible solution, supported by robust research, is the replacement of T lymphocytes with NK cells for CAR expression. NK cells seem to be an attractive vehicle for CAR expression as they can be derived from multiple sources and safely infused regardless of donor–patient matching, which greatly reduces the cost of the treatment. CAR-NK cells are known to be effective against hematological malignancies, and a growing number of preclinical findings indicate that they have activity against non-hematological neoplasms. Here, we present a thorough overview of the current state of knowledge regarding the use of CAR-NK cells in treating various solid tumors.

Economic impact of the use of the National Surgical Quality Improvement Program

2021 ◽  
Vol 37 (1) ◽  
Author(s):  
Alvine Fansi ◽  
Angela Ly ◽  
Julie Mayrand ◽  
Maggy Wassef ◽  
Aldanie Rho ◽  
...  
Keyword(s):  
Quality Improvement ◽  
Cost Analysis ◽  
Economic Impact ◽  
Quality Improvement Program ◽  
Economic Evaluations ◽  
Complication Rates ◽  
Improvement Program ◽  
Surgical Quality ◽  
Acs Nsqip ◽  
The Cost

Objectives The American College of Surgeons National Surgical Quality Improvement Program (ACS NSQIP®) is a validated, risk-adjusted database for improving the quality and security of surgical care. ACS NSQIP can help participating hospitals target areas that need improvement. The aim of this study was to systematically review the literature analyzing the economic impact of using NSQIP. This paper also provides an estimation of annual cost savings following the implementation of NSQIP and quality improvement (QI) activities in two hospitals in Quebec. Methods In June 2018, we searched in seven databases, including PubMed, Embase, and NHSEED for economic evaluations based on NSQIP data. Contextual NSQIP databases from two hospitals were collected and analyzed. A cost analysis was conducted from the hospital care perspective, comparing complication costs before and after 1 year of the implementation of NSQIP and QI activities. The number and the cost of complications are measured. Costs are presented in 2018 Canadian dollars. Results Out of 1,612 studies, 11 were selected. The level of overall evidence was judged to be of moderate to high quality. In general, data showed that, following the implementation of NSQIP and QI activities, a significant decrease in complications and associated costs was observed, which improved with time. In the cost analysis of contextual data, the reduction in complication costs outweighed the cost of implementing NSQIP. However, this cost analysis did not take into account the costs of QI activities. Conclusions NSQIP improves complication rates and associated costs when QI activities are implemented.

scholarly journals Analysis of Non-Idealities on CMOS Passive Mixers

Electronics ◽  
2021 ◽  
Vol 10 (9) ◽  
pp. 1105
Author(s):  
Antonio D. Martinez-Perez ◽  
Francisco Aznar ◽  
Guillermo Royo ◽  
Santiago Celma
Keyword(s):  
Final Section ◽  
Cmos Technology ◽  
Monte Carlo Analysis ◽  
Image Rejection ◽  
Structure Approach ◽  
Quadrature Scheme ◽  
Current State ◽  
The Cost ◽  
Image Rejection Ratio ◽  
The Ideal

In the current state of the art, WiFi-alike standards require achieving a high Image Rejection Ratio (IRR) while having low power consumption. Thus, quadrature structures based on passive ring mixers offer an attractive and widely used solution, as they can achieve a high IRR while being a passive block. However, it is not easy for the designer to know when a simple quadrature scheme is enough and when they should aim for a double quadrature structure approach, as the latter can improve the performance at the cost of requiring more area and complexity. This study focuses on the IRR, which crucially depends on the symmetry between the I and Q branches. Non-idealities (component mismatches, parasitics, etc.) will degrade the ideal balance by affecting the mixer and/or following/previous stages. This paper analyses the effect of imbalances, providing the constraints for obtaining a 40 dB IRR in the case of a conversion from a one-hundred-megahertz signal to the five-gigahertz range (upconversion) and vice versa (downconversion) for simple and double quadrature schemes. All simulations were carried out with complete device models from 65 nm standard CMOS technology and also a post-layout Monte Carlo analysis was included for mismatch analysis. The final section includes guidelines to help designers choose the most adequate scheme for each case.

Computing Robust Principal Components by A* Search

2018 ◽  
Vol 27 (07) ◽  
pp. 1860013 ◽  
Author(s):  
Swair Shah ◽  
Baokun He ◽  
Crystal Maung ◽  
Haim Schweitzer
Keyword(s):  
State Of The Art ◽  
Principal Component ◽  
Low Rank ◽  
A Algorithm ◽  
Running Time ◽  
Current State ◽  
Dimensionality Reduction Technique ◽  
Related Variant ◽  
Low Rank Representation ◽  
The Cost

Principal Component Analysis (PCA) is a classical dimensionality reduction technique that computes a low rank representation of the data. Recent studies have shown how to compute this low rank representation from most of the data, excluding a small amount of outlier data. We show how to convert this problem into graph search, and describe an algorithm that solves this problem optimally by applying a variant of the A* algorithm to search for the outliers. The results obtained by our algorithm are optimal in terms of accuracy, and are shown to be more accurate than results obtained by the current state-of-the- art algorithms which are shown not to be optimal. This comes at the cost of running time, which is typically slower than the current state of the art. We also describe a related variant of the A* algorithm that runs much faster than the optimal variant and produces a solution that is guaranteed to be near the optimal. This variant is shown experimentally to be more accurate than the current state-of-the-art and has a comparable running time.

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