scholarly journals Out-of-Pocket Healthcare Expenditures of Households Living With Rare Diseases

2021 ◽  
Author(s):  
Gülpembe Oğuzhan ◽  
Selin Ökçün ◽  
Mustafa Kurnaz ◽  
Zafer Çalışkan ◽  
Güvenç Koçkaya ◽  
...  
Keyword(s):  
Rare Diseases ◽  
Household Income ◽  
Online Survey ◽  
Metabolic Diseases ◽  
Healthcare Access ◽  
Productivity Loss ◽  
Annual Average ◽  
Access To Treatment ◽  
Average Household Income ◽  
Microsoft Office

Abstract Background: Economic impact of rare diseases for both individuals and family members, in addition to many expenditures such as disease-related medication, use of healthcare, access to treatment, special medical and nutritional requirements; it also causes indirect effects such as loss of productivity, loss of wages, health insurance and inability to find work. In this study, it is aimed to determine the out-of-pocket health expenditures and economic burden of households where individuals with rare diseases live in Turkey.Methods: The research population consists of registered members of associations that are members of the Rare Diseases Network. The questionnaire was developed based on literature review and expert opinion and the online survey was made available between 01.05.2020- 31.05.2020. After obtaining data on out-of-pocket expenditures in rare diseases, the necessary analysis was carried out through the Microsoft Office Excel application. In addition to the general analysis involving all participants, expenditures on the basis of disease group and disease were calculated. The costs calculated in Turkish Liras (₺/TL) have been converted to Euros (€) according to the average exchange rate for 2020. Results: 456 participants were included in the analysis. Accordingly, 233 patients were included in the metabolic diseases category, 178 in the neuromuscular system diseases category and 41 in the others category. It was determined that special nutrition were the highest expenditure group and emergency department were the lowest expenditure group. The lowest amount of out-of-pocket expenditure is in the metabolic diseases group. It has been observed that 47.35% of all participants allocate more than 10% of the annual average household income and 4.65% more than 100% of the annual average household income for medical expenses. It is seen that the average out-of-pocket expenses in DMD, SMA, cystinosis and cystic fibrosis diseases are close to each other.Conclusion: Since individuals with rare diseases are often unable to work, out-of-pocket expenditures related to the disease lead to financial difficulties and even impoverishment. Their already difficult lives get even more difficult by the financial sacrifices made for treatment. Treatments for rare diseases need to go beyond special exclusivity for patient access and reimbursement.

scholarly journals Stakeholder perspectives on clinical research related to therapies for rare diseases: therapeutic misconception and the value of research

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Kylie Tingley ◽  
◽  
Doug Coyle ◽  
Ian D. Graham ◽  
Pranesh Chakraborty ◽  
...  
Keyword(s):  
Focus Group ◽  
Clinical Research ◽  
Health Care Providers ◽  
Rare Diseases ◽  
Metabolic Diseases ◽  
Population Level ◽  
Qualitative Description ◽  
Therapeutic Misconception ◽  
Care Providers ◽  
Access To Treatment

Abstract Background For many rare diseases, few treatments are supported by strong evidence. Patients, family members, health care providers, and policy-makers thus have to consider whether to accept, recommend, or fund treatments with uncertain clinical effectiveness. They must also consider whether and how to contribute to clinical research that may involve receiving or providing the therapy being evaluated. Objective To understand why and how patients and families with rare metabolic diseases, specialist metabolic physicians, and health policy advisors choose whether to participate in studies and how they use and value research. Methods We conducted separate focus group interviews with each stakeholder group (three groups in total); two groups were conducted by telephone and the third was held in-person. Participants were recruited using purposive sampling. We analyzed each interview transcript sequentially using a qualitative description approach to inductively identify key themes. Several strategies to ensure credibility and trustworthiness were used including debriefing sessions after each focus group and having multiple team members review transcripts. Results Four patients/caregivers, six physicians, and three policy advisors participated. Our findings did not support conventional perspectives that therapeutic misconception (gaining access to treatment) is the main motivating factor for patients/caregivers to participate in clinical research. Rather, patients’/caregivers’ expressed reasons for participating in research included advancing science for the next generation and having an opportunity to share their experiences. Patients/caregivers and physicians described the difficulties in weighing risks versus benefits of accepting treatments not well-supported by evidence. Physicians also reported feeling conflicted in their dual role as patient advisor/advocate and evaluator of the evidence. Policy advisors were primarily focused on critically appraising the evidence to make recommendations for the health system. Conclusions Stakeholders differ in their perspectives on rare disease research but share concerns about the risks versus benefits of therapies when making individual- and population-level decisions.

scholarly journals Workplace nutrition habits in Slovenian small and middle sized enterprises

2020 ◽  
Vol 30 (Supplement_5) ◽  
Author(s):  
S Percic ◽  
M Stubelj
Keyword(s):  
Chronic Diseases ◽  
Online Survey ◽  
Healthy Lifestyle ◽  
Productivity Loss ◽  
Premature Death ◽  
Average Score ◽  
Workplace Health ◽  
Nutritional Habits ◽  
Healthy Nutrition ◽  
Nutrition Habits

Abstract Background Premature death, chronic diseases, productivity loss and other social and economic concerns can be influenced by healthy lifestyle workplace promotion programs, including healthy nutrition habits. In Slovenia a project called >>STAR-VITAL-healthy ageing at workplace << has been going on since September 2017. Methods The survey results are based on an analysis of 805 respondents from 20 different small and middle sized enterprises in Slovenia. The survey was carried out via both a paper questionnaire and an online survey during the period from June 2019 to February 2020. The screening questionnaire was completed before interventions. We estimated the average score for the eight questions concerning nutritional habits. Results Nutritional habits are the following: 62% respondents eat three or more meals a day, 64% respondents eat vegetables each day, 59,8% respondents eat fruits each day, 31,9% respondents eat fish or see fruits at least once a week, 45,7% respondents eat whole wheats at least once a week, 54,1% respondents eat red meat not more than twice a week, 62,1% respondents eat fried food not more than three times a month and 79,1% respondents drink sweet drinks less than three times a week. The results on question >>What is the most common form of meal you eat while at work? << are the following: purchase meal at restaurant 45,3%, bring from home 32,7%, delivery/carry out 6,2%, do not eat 5,2%, other 4,7%, employer cafeteria 3,9% and vending machine 1,2%. Conclusions The results of our survey show that a significant percentage of the workers in Slovenia still has unhealthy nutritional habits. Assessment of nutritional habits before workplace promotion program interventions allow us to compare which combinations of interventions are the most effective. The results will indicate the strength and weakness of each intervention and how it could be improved. Key messages The population is growing old and chronic diseases increases, which is part of the bad nutritional habits. Healthy lifestile promotion is needed to improve quality of life. The workplace health promoting programs have been shown to be an efficient way of improving workerś health. Implementing promotional program of nutritional habits are proper for Slvenian enterprises.

scholarly journals Association between parental socioeconomic status and offspring overweight/obesity from the China Family Panel Studies: a longitudinal survey

BMJ Open ◽  
2021 ◽  
Vol 11 (4) ◽  
pp. e045433
Author(s):  
Suqin Ding ◽  
Jingqi Chen ◽  
Bin Dong ◽  
Jie Hu
Keyword(s):  
High School ◽  
Socioeconomic Status ◽  
Regression Model ◽  
Household Income ◽  
Parental Education ◽  
Education Level ◽  
Longitudinal Survey ◽  
Average Household Income ◽  
Education Levels ◽  
Parental Socioeconomic Status

ObjectiveTo examine the association between parental socioeconomic status (SES) and the risk of offspring overweight/obesity and the changes of the association that occur as children grow older.DesignWe used data from the nationally representative longitudinal survey of the China Family Panel Studies of 2010 and its three follow-up waves in 2012, 2014 and 2016.ParticipantsA total of 6724 children aged 0–15 years old were included.Primary and secondary outcome measuresAverage household income and paternal and maternal education levels were used as SES indicators. Logistic regression model for panel data was used to examine the associations between SES indicators and child overweight/obesity. A restricted cubic spline linear regression model was used to estimate body mass index (BMI) trajectories with child growth across parental SES levels.ResultsCompared with the lowest education level (primary school or less), the ORs for fathers who had completed junior high school, senior high school and junior college or higher were 0.85 (95% CI 0.75 to 0.97), 0.77 (95% CI 0.64 to 0.92) and 0.72 (95% CI 0.55 to 0.93), respectively. The corresponding ORs for mothers were 0.76 (95% CI 0.67 to 0.86), 0.59 (95% CI 0.47 to 0.72) and 0.45 (95% CI 0.34 to 0.60), respectively. A negative association between parental education and offspring overweight/obesity was observed in the first 10 years but not in children 11–15 years old. BMI differences across parental education levels emerged from birth and widened before 6–7 years old, but decreased before adolescence. High average household income was related to a low risk of offspring overweight/obesity but not when parental education level was adjusted for.ConclusionHigh parental education levels were associated with a low risk of offspring overweight/obesity, especially before adolescence. Effective approaches need to be adopted in early childhood to reduce socioeconomic differences in overweight/obesity.

scholarly journals ANALISIS PENDAPATAN DAN TINGKAT KESEJAHTERAAN RUMAH TANGGA MASYARAKAT SEKITAR TAMAN NASIONAL BUKIT BARISAN SELATAN KABUPATEN PESISIR BARAT

2021 ◽  
Vol 9 (3) ◽  
pp. 418
Author(s):  
M. Reza Fachrezy ◽  
Zainal Abidin ◽  
Adia Nugraha
Keyword(s):  
Household Income ◽  
National Park ◽  
Linear Regression Analysis ◽  
Multiple Linear Regression Analysis ◽  
Farm Income ◽  
Labor Costs ◽  
The Third ◽  
Average Household Income ◽  
Average Education ◽  
On Farm

This study aims to determine the socio-economic characteristics of farmers, analyze the income structure, the factors that affect the income of farmers, and the level of household welfare of farmers who live around Bukit Barisan Selatan National Park, Pesisir Barat District. This research is a survey research involving 99 respondents who were randomly selected. Respondents were spread across four subdistricts, namely Bengkunat Belimbing, Ngambur, Pesisir Selatan, and Karya Penggawa Districts. The study was conducted from July to August 2018. The first objective was analyzed qualitatively, the second objective was analyzed using household income analysis, the third objective used multiple linear regression analysis, and the fourth objective was using Sajogyo's criteria (1997). The study suggested that the socio-economic characteristics of the farmers around TNBBS were spread from 15 to 64 years of age with an average age of 43.74 years. The average education level was elementary school, the number of majority of family sizes was from 4 to 6 people (67.68%), and the average land was 1.41 ha. The average household income of farmers is IDR 36,946,883.94 per year, which consists of on-farm income 63.68%, off-farm income 11.00%, and non-farm income 25.33%.. Factors that affect farmers' income are fertilizer costs and labor costs. The welfare of farmers around TNBBS is in quite prosperous category.Key words: characteristics, household income, welfare

scholarly journals Loss of work productivity in a warming world: Differences between developed and developing countries

2020 ◽  
Author(s):  
Shuang Yu ◽  
Zhongwei Yan ◽  
Jiangjiang Xia ◽  
Alcide Zhao ◽  
Anzhi Zhang ◽  
...  
Keyword(s):  
Developing Countries ◽  
Online Survey ◽  
Climate Models ◽  
Heat Waves ◽  
Productivity Loss ◽  
Work Productivity ◽  
Developed Countries ◽  
Point Of View ◽  
The World ◽  
Warming World

<p>Comparable estimates of the heat-related work productivity loss (WPL) in different countries over the world are difficult partly due to the lack of exact measures and comparable data for different counties. In this study, we analysed 4363 responses to a global online survey on the WPL during heat waves in 2016. The participants were from both developed and developing countries, facilitating estimates of the heat-related WPL across the world for the year. The heat-related WPL for each country involved was then deduced for increases of 1.5, 2, 3 and 4 °C in the global mean surface temperature under the representative concentration pathway scenarios in climate models. The average heat-related WPL in 2016 was 6.6 days for developing countries and 3.5 days for developed countries. The estimated heat-related WPL was negatively correlated with the gross domestic product per capita. When global surface temperatures increased by 1.5, 2, 3 and 4 °C, the corresponding WPL was 9 (19), 12 (31), 22 (61) and 33 (94) days for developed (developing) countries, quantifying how developing countries are more vulnerable to climate change from a particular point of view. Moreover, the heat-related WPL was unevenly distributed among developing countries. In a 2°C-warmer world, the heat-related WPL would be more than two months in Southeast Asia, the most influenced region. The results are considerable for developing strategy of adaptation especially for developing countries.</p>

Enzyme Replacement Therapy for Genetic Disorders Associated with Enzyme Deficiency

2021 ◽  
Vol 28 ◽  
Author(s):  
Marialaura Marchetti ◽  
Serena Faggiano ◽  
Andrea Mozzarelli
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Rare Diseases ◽  
Mammalian Cells ◽  
Metabolic Diseases ◽  
Genetic Disorders ◽  
Life Quality ◽  
Lysosomal Storage Diseases ◽  
Lysosomal Storage ◽  
Enzyme Replacement

: Mutations in human genes might lead to loss of functional proteins, causing diseases. Among these genetic disorders, a large class is associated with the deficiency in metabolic enzymes, resulting in both an increase in the concentration of substrates and a loss in the metabolites produced by the catalyzed reactions. The identification of therapeutic actions based on small molecules represents a challenge to medicinal chemists because the target is missing. Alternative approaches are biology-based, ranging from gene and stem cell therapy, CRISPR/Cas9 technology, distinct types of RNAs, and enzyme replacement therapy (ERT). This review will focus on the latter approach that since the 1990s has been successfully applied to cure many rare diseases, most of them being lysosomal storage diseases or metabolic diseases. So far, a dozen enzymes have been approved by FDA/EMA for lysosome storage disorders and only a few for metabolic diseases. Enzymes for replacement therapy are mainly produced in mammalian cells and some in plant cells and yeasts and are further processed to obtain active, highly bioavailable, less degradable products. Issues still under investigation for the increase in ERT efficacy are the optimization of enzymes interaction with cell membrane and internalization, the reduction in immunogenicity, and the overcoming of blood-brain barrier limitations when neuronal cells need to be targeted. Overall, ERT has demonstrated its efficacy and safety in the treatment of many genetic rare diseases, both saving newborn lives and improving patients’ life quality, and represents a very successful example of targeted biologics.

Facebook support groups for pediatric rare diseases: opportunities, limitations and privacy concerns (Preprint)

2021 ◽  
Author(s):  
Sarah Catrin Titgemeyer ◽  
Christian Patrick Schaaf
Keyword(s):  
Rare Disease ◽  
Support Groups ◽  
Support Group ◽  
Rare Diseases ◽  
Health Professionals ◽  
Online Survey ◽  
Privacy Rights ◽  
Cross Sectional ◽  
Privacy Concerns ◽  
Group Members

BACKGROUND Due to the nature of rare diseases with affected individuals being widely geographically dispersed, finding an in-person/offline support group itself can be a challenge. Affected individuals therefore turn to social networking platforms such as Facebook for online support groups. OBJECTIVE We aim to put into perspective the opportunities Facebook offers as a tool for pediatric rare disease support groups by investigating its use, advantages and limitations including privacy concerns. We analyze group accessibility and usage, advantages specific to rare diseases, perceived privacy and views on using Facebook for communication between health professionals and parents, pharmaceutical companies and for study recruitment. METHODS We contacted twelve Facebook support groups for twelve respective rare diseases with pediatric onset and invited group members to participate in a cross-sectional online survey. RESULTS Of 231 respondents, 87.0% (n=201) respondents were female, 12.6% (n=29) were male and 1 respondent reported another sex (0.4%, n=1). Respondents’ mean age was 41.56 years (SEM=0.621, SD=9.375). 91.3% (n=211) respondents were parents (183 mothers, 27 fathers, 1 other sex). 59.7% (n=138) reported a self-initiated search for the Facebook group, 24.2% (n=56) received recommendations from their health professionals, 12.6% (n=29) recommendations from someone else affected by the disease. On average, support group members visited Facebook at least once a day, visited and passively participated (read/liked posts) several times a week and participated actively (commented/posted) once a month. 79,6% agreed that they would like to have health professionals as members of the respective Facebook group. Group members expressed more concern about privacy issues on Facebook in general than in their respective Facebook support groups, with concerns mostly related to Facebook itself and non-group-members. CONCLUSIONS Our study confirmed that Facebook enhances support group accessibility for parents of children with rare diseases. Group participants perceive a reduction and elimination of distance, a common challenge in rare disease, and Facebook support groups create an environment of perceived privacy. The group’s privacy setting can be a critical factor for active support group participation. Sharing personal information and pictures on Facebook is very common among group participants, which shows the importance of discussing and protecting children’s privacy rights in this context. CLINICALTRIAL DRKS00016067

scholarly journals Addressing Household Income Distribution in Air Travel Demand Models: Case Study of the Baltimore–Washington Region

2019 ◽  
Vol 2673 (1) ◽  
pp. 491-502 ◽  
Author(s):  
Geoffrey D. Gosling ◽  
David Ballard
Keyword(s):  
Income Distribution ◽  
Household Income ◽  
Travel Demand ◽  
Metropolitan Region ◽  
Annual Data ◽  
Demand Model ◽  
Passenger Demand ◽  
Average Household Income ◽  
Demand Models ◽  
The Future

The paper describes the development of an air passenger demand model for the Baltimore–Washington metropolitan region that was undertaken as part of a recently concluded ACRP project that explored the use of disaggregated socioeconomic data in air passenger demand studies. The model incorporated a variable reflecting the change in household income distribution, together with more traditional aggregate causal variables: population, employment, average household income, and airfares as measured by the average U.S. airline yield, as well as several year-specific dummy variables. The model was estimated on annual data for the period 1990 to 2010 and obtained statistically significant estimated coefficients for all variables, including both the average household income and the household income distribution variable. Including household income distribution in the model resulted in a significant change to the estimated coefficient for average household income, giving a much higher estimated elasticity of demand with respect to average household income compared with a model that does not consider changes in household income distribution. This has important implications for the use of such demand models for forecasting, as household income distribution and average household income may change in the future in quite different ways, which would affect the future levels of air passenger travel projected by the models.

scholarly journals Factors Related to the Number of Existing Teeth among Korean Adults Aged 55–79 Years

2019 ◽  
Vol 16 (20) ◽  
pp. 3927 ◽  
Author(s):  
Jung-Ha Lee ◽  
Seung-Kyoo Yi ◽  
Se-Yeon Kim ◽  
Ji-Soo Kim ◽  
Han-Na Kim ◽  
...  
Keyword(s):  
Metabolic Syndrome ◽  
Oral Health ◽  
Household Income ◽  
Elderly Population ◽  
Metabolic Diseases ◽  
Income Group ◽  
Korean Adults ◽  
Health And Nutrition ◽  
Region Of Residence ◽  
Health Related

This study aimed to determine the association between the number of existing teeth (NET) and socioeconomic status (SES), oral health-related behaviours, and metabolic syndrome in Korean adults aged 55–79 years. The study included 3255 adults who underwent oral health examinations and answered questionnaires regarding SES, oral health-related behaviours, and metabolic diseases in the Sixth Korea National Health and Nutrition Examination Survey (2013–2015). The dependent variable was the binary status based on the median NET in each age group. The independent variables were based on SES, oral health-related behaviours, and the presence of metabolic syndrome. The study findings showed that the factors associated with the NET were sex, household income, education level, region of residence, daily toothbrushing frequency, dental visit within 1 year, smoking, and metabolic syndrome. NET was lower in males (adjusted OR: 0.74), in low household income group (adjusted OR: 0.77), in primary school graduates (adjusted OR: 0.53) and in rural residents (adjusted OR: 0.78). The interventions aimed at preserving existing teeth in elderly population should consider their SES, oral health-related behaviours, and metabolic syndrome and overhauling current oral healthcare system and redefining the roles of oral health professionals.

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