The economic impact of living with a rare disease for children and their families: a scoping review protocol.

Background: Rare diseases are an often chronic, progressive and life-limiting group of conditions affecting more than 30 million people in Europe. These diseases are associated with significant direct and indirect costs to a spectrum of stakeholders, ranging from individuals and their families to society overall. Further quantitative research on the economic cost for children and their families living with a rare disease is required as there is little known on this topic. This scoping review aims to document the extent and type of evidence on the economic impacts of living with a rare disease for children and their families. Methods: This scoping review will follow the PRISMA-ScR and Joanna Briggs Institute guidelines and follow the six-stage methodology for scoping reviews: (1) identifying the research question, (2) identifying relevant studies, (3) study selection, (4) charting the data, (5) collating, summarising and reporting results and (6) knowledge user consultation. Key inclusion criteria have been developed according to the Population-Concept-Context (PCC) framework. The databases EconLit, ABI/Inform, MEDLINE, PubMed, CINAHL, and Scopus will be searched for possible articles for inclusion. Two independent reviewers will screen titles and abstracts of potential articles using a dual review process to ensure all relevant studies are included. All included articles will be assessed using a validated quality appraisal tool. A panel of patient and public involvement representatives experiencing rare diseases and knowledge users will validate the review results. Conclusions: This scoping review will map the current literature on the economic impact of paediatric rare diseases to understand how these impacts affect children living with rare diseases and their families. This evidence has the potential to influence policy and future research in this area and will support further research on the economic impact of rare diseases on families.

The effect of local unemployment on health: A longitudinal study of Irish mothers 2001-2011.

The relationship between economic conditions and health is mixed, with some evidence from the most recent financial crisis finding a positive effect of periods of unemployment on heath behaviours. This study uses longitudinal data spanning the periods before, during and after the Irish crisis of 2007, to test the impact of economic expansion and contraction on mothers physical and mental health and health behaviours. Three waves of data from the Irish Lifeways Cohort Study for the period 2001-2011, and local area employment rates from the Irish Census, are used to capture the impact of a period of increased unemployment on health before, during and after the Irish recession, independent of individual employment status. The results from fixed effect linear probability models demonstrate that an additional unit of local area unemployment increases the probability of reporting poor self-rated health by 1-1.8 percentage points and of reporting poor mental well-being by 1.4 and 2.7 percentage points depending on the instrument used. There are decreases in the probability of consuming cigarettes by 3.3 percentage points, self-describing as a regular smoker by 2.9 percentage points and obesity by 0.9 percentage points. The probability of engaging in at least 20 minutes of strenuous or moderate exercise per week declines by 7.8 and 8.7 percentage points respectively, while the probability of engaging in at least 20 minutes of mild exercise rises by 10 percentage points. These results are largely consistent with the US literature, which is predominantly based on working men, thus demonstrating the universal impact of economic vulnerability on health.

Does maternal attachment to her infant mediate the link between perceptions of infant crying at 6 months and parenting stress at 24 months? A structural equation modelling approach.

BACKGROUND: Parenting stress is influenced by many factors including maternal attachment and excessive infant crying, yet the nature of these relationships is not well understood. For example, excessive infant crying despite maternal soothing may impact maternal attachment to the child, leading to higher stress. This paper explored whether maternal perception of excessive infant crying at 6 months was associated with higher maternal parenting stress at 24 months, and whether maternal attachment mediated this relationship. METHODS: All families, present at 24 months in a randomized controlled trial of a 5-year early intervention programme targeting school readiness skills in disadvantaged area of Ireland, were included. At 6 months, infant crying was assessed using a maternal reported measure of duration of infant crying, and maternal attachment to the infant was assessed using the Condon Maternal Attachment Scale. Parenting stress was assessed at 24 months using the childrearing stress subscale from the Parenting Stress Index. Structural equation modelling was used to explore the direct and indirect effects of maternal perceptions of excessive infant crying on parenting stress, controlling for infant, maternal, and environmental characteristics, and focusing on the mediating role of maternal attachment. RESULTS: Reporting excessive infant crying at 6 months was associated with lower maternal attachment at 6 months, which led to higher parenting stress at 24 months. In addition, vulnerable adult attachment style, previous maternal mental health difficulties, low paternal education, paternal involvement with the child, and not being married were associated with higher parenting stress. CONCLUSION: Findings suggest that the association between maternal perceptions of excessive crying at 6 months and later parenting stress may be mediated through maternal attachment to the infant. Interventions based on improving maternal attachment could be investigated to determine the effectiveness of supporting mothers with low attachment.

Population-Based System of Parenting Support to Reduce the Prevalence of Child Social, Emotional, and Behavioural Problems: Difference-In-Differences Study.

The quality of parenting is recognised as an important determinant of children's mental health. Parenting interventions typically target high-risk families rather than adopting a universal approach. This study examined the population impact of the Triple P Positive Parenting Programme on the prevalence of children's social, emotional, and behavioural problems. A propensity score matching difference-in-differences method was used to compare intervention and comparison regions matched on socio-demographic characteristics in midlands Ireland. The pre-intervention sample included 1501 and 1495 parents of children aged 4-8 years in the intervention and comparison regions respectively. The post-intervention sample included 1521 and 1544 parents respectively. The primary outcome measure was parental reports on the Strengths and Difficulties Questionnaire. There were some significant reductions in the prevalence rates of social, emotional, and behavioural problems in the intervention regions compared to the comparison regions. Children in the intervention sample experienced lower total difficulties, emotional symptoms, and conduct problems than children in the comparison sample, and they were less at risk of scoring within the borderline/abnormal range for total difficulties, conduct problems, and hyperactivity. The programme reduced the proportion of children scoring within the borderline/abnormal range by 4.7% for total difficulties, 4.4% for conduct problems, and 4.5% for hyperactivity in the total population. This study demonstrated that a universal parenting programme implemented at multiple levels using a partnership approach may be an effective population health approach to targeting child mental health.

Personalized Medication Response Prediction for Attention-Deficit Hyperactivity Disorder: Learning in the Model Space vs. Learning in the Data Space.

Attention-Deficit Hyperactive Disorder (ADHD) is one of the most common mental health disorders amongst school-aged children with an estimated prevalence of 5% in the global population (American Psychiatric Association, 2013). Stimulants, particularly methylphenidate (MPH), are the first-line option in the treatment of ADHD (Reeves and Schweitzer, 2004; Dopheide and Pliszka, 2009) and are prescribed to an increasing number of children and adolescents in the US and the UK every year (Safer et al., 1996; McCarthy et al., 2009), though recent studies suggest that this is tailing off, e.g., Holden et al. (2013). Around 70% of children demonstrate a clinically significant treatment response to stimulant medication (Spencer et al., 1996; Schachter et al., 2001; Swanson et al., 2001; Barbaresi et al., 2006). However, it is unclear which patient characteristics may moderate treatment effectiveness. As such, most existing research has focused on investigating univariate or multivariate correlations between a set of patient characteristics and the treatment outcome, with respect to dosage of one or several types of medication. The results of such studies are often contradictory and inconclusive due to a combination of small sample sizes, low-quality data, or a lack of available information on covariates. In this paper, feature extraction techniques such as latent trait analysis were applied to reduce the dimension of on a large dataset of patient characteristics, including the responses to symptom-based questionnaires, developmental health factors, demographic variables such as age and gender, and socioeconomic factors such as parental income. We introduce a Bayesian modeling approach in a "learning in the model space" framework that combines existing knowledge in the literature on factors that may potentially affect treatment response, with constraints imposed by a treatment response model. The model is personalized such that the variability among subjects is accounted for by a set of subject-specific parameters. For remission classification, this approach compares favorably with conventional methods such as support vector machines and mixed effect models on a range of performance measures. For instance, the proposed approach achieved an area under receiver operator characteristic curve of 82-84%, compared to 75-77% obtained from conventional regression or machine learning ("learning in the data space") methods.

Early intervention and child physical health: Evidence from a Dublin-based randomized controlled trial.

This article investigates the impact of an early intervention program, which experimentally modifies the parenting and home environment of disadvantaged families, on child physical health in the first 3 years of life. We recruited and randomized 233 (115 intervention, 118 control) pregnant women from a socioeconomically disadvantaged community in Dublin, Ireland into an intervention or control group. The treatment includes regular home visits commencing antenatally and an additional parenting course commencing at 2 years. Maternal reports of child health are assessed at 6, 12, 18, 24, and 36 months. Treatment effects are estimated using permutation testing to account for small sample size, inverse probability weighting to account for differential attrition, and both the stepdown procedure and an indices approach to account for multiple hypothesis testing. Following adjustment for multiple testing and attrition, we observe a positive and statistically significant main treatment effect for wheezing/asthma. The intervention group are 15.5 percentage points (pp) less likely to require medical attention for wheezing/asthma compared to the control group. Subgroup analysis reveals more statistically significant adjusted treatment effects for boys than girls regarding fewer health problems (d=0.63), accidents (23.9pp), and chest infections (22.8-37.9pp). Our results suggest that a community-based home visiting program may have favorable impacts on early health conditions.

Economics of childbirth.

BACKGROUND: Increasing birth rates have raised questions for policy makers and hospital management about the economic costs of childbirth. OBJECTIVE: The purpose of this article is to identify and review all existing scientific studies in relation to the economic costs of alternative modes of childbirth delivery and to highlight deficiencies in the existing scientific research. METHOD: We searched Cochrane, Centre for Reviews and Dissemination, EconLit, the Excerpta Medica Database, the Health Economic Evaluations Database, MEDLINE and PubMed. RESULTS: Thirty articles are included in this review. The main findings suggest that there is no internationally acceptable childbirth cost and clinical outcome classification system that allows for comparisons across different delivery modes. CONCLUSION: This review demonstrates that a better understanding and classification of the costs and associated clinical outcomes of childbirth is required to allow for valid comparisons between maternity units, and to inform policy makers and hospital management.

Investing in early human development: timing and economic efficiency.

Policy discussions to ameliorate socioeconomic (SES) inequalities are increasingly focused on investments in early childhood. Yet such interventions are costly to implement, and clear evidence on the optimal time to intervene to yield a high economic and social return in the future is meagre. The majority of successful early childhood interventions start in the preschool years. However socioeconomic gradients in cognitive skills, socio-emotional functioning and health can be observed by age three, suggesting that preventative programmes starting earlier in childhood may be even more effective. We discuss the optimal timing of early childhood intervention with reference to recent research in developmental neuroscience. We motivate the need for early intervention by providing an overview of the impact of adverse risk factors during the antenatal and early childhood periods on outcomes later in life. We provide a brief review of the economic rationale for investing early in life and propose the "antenatal investment hypothesis". We conclude by discussing a suite of new European interventions that will inform this optimal timing debate.

"... Take up thy bed, and vote." Measuring the relationship between voting behaviour and indicators of health.

In most countries health policy is an important part of the political agenda. Yet few studies have examined the relationship between the two. This study investigates the association between health and voter turnout in Britain using the National Child Development Study. Self-rated general health, the Malaise Inventory score and indicators of smoking and alcohol consumption, as measured at ages 23, 33 and 42, are regressed on voter turnout in the 1979, 1987 and 1997 general elections. The results indicate that individuals with poor general and mental health and smokers are less likely to vote at election time.