Statistical modeling for Bayesian extrapolation of adult clinical trial information in pediatric drug evaluation

Children represent a large underserved population of “therapeutic orphans,” as an estimated 80% of children are treated off‐label. However, pediatric drug development often faces substantial challenges, including economic, logistical, technical, and ethical barriers, among others. Among many efforts trying to remove these barriers, increased recent attention has been paid to extrapolation; that is, the leveraging of available data from adults or older age groups to draw conclusions for the pediatric population. The Bayesian statistical paradigm is natural in this setting, as it permits the combining (or “borrowing”) of information across disparate sources, such as the adult and pediatric data. In this paper, authored by the pediatric subteam of the Drug Information Association Bayesian Scientific Working Group and Adaptive Design Working Group, we develop, illustrate, and provide suggestions on Bayesian statistical methods that could be used to design improved pediatric development programs that use all available information in the most efficient manner. A variety of relevant Bayesian approaches are described, several of which are illustrated through 2 case studies: extrapolating adult efficacy data to expand the labeling for Remicade to include pediatric ulcerative colitis and extrapolating adult exposure‐response information for antiepileptic drugs to pediatrics.     

Endless Pressure: Life on a Low Income in Austere Times

Much has been written that details the decline in living standards following the ‘credit crunch’ in the UK. It remains that we understand to a lesser degree the lived reality of poverty through the Great Recession and into the era of austerity. This article draws on testimonies of 62 participants from low income households conducted in three different areas of the UK during 2012–13 to document the pressures that this period brought to bear on these households. According to these testimonies, the experience of poverty intensified in key respects: first, participants reported feeling, more than ever before, that they were ‘existing, rather than living’ due to the meagre budgets they were forced to live on; second, the precarious nature of work and social security contributed to a sense of insecurity that was all pervasive in our participants' lives; third, due to the pejorative political rhetoric and media coverage of poverty, our participants felt that their lives were placed under increased scrutiny which deleteriously impacted their wider relationships and sense of belonging. Our analysis demonstrates the profound consequences for those living on low incomes of the continued shift to residual forms of state welfare and the increased reliance on the ‘Big Society’ as a means to deal with the pressures identified in this article.     

Medicare and Amyloid PET Imaging: The Battle Over Evidence

We examine a recent dispute regarding the Centers for Medicare and Medicaid Services’ (CMS) refusal to unconditionally pay for amyloid positron emission tomography (PET) imaging for Medicare beneficiaries being assessed for Alzheimer’s disease. CMS will only pay for amyloid PET imaging when patients are enrolled in clinical trials that meet certain criteria. The dispute reflects CMS’s willingness in certain circumstances to require effectiveness evidence that differs from the Food and Drug Administration’s standard for pre-market approval of a medical intervention and reveals how stakeholders with differing perspectives about evidentiary standards have played a role in attempting to shape the Medicare program’s coverage policies.     

Who does not cut down? Comparing characteristics and drinking motives of drinkers and abstainers during the exam periods

Objective: Drinking alcohol during the exams can affect academic performance and future career options, but is rarely investigated. Drinking motives, sociodemographics and personality characteristics are investigated in nonabstainers and weekly drinkers during the exams. Participants: 7,181 Belgian university students who anonymously responded to an email invitation to an online survey. Methods: Logistic regressions and mixed design analysis of variance on cross-sectional data. Results: One-third of the students continued drinking during the exams, with 40% drinking weekly. Nonabstainers were mainly men, elder, internally motivated when drinking, and housed with parents or independently. Weekly drinkers were similar, except mainly housed in student apartments or independently. Personality characteristics were nonsignificant. All drinking motives were less pronounced during the exams, with smaller differences for internal motives. Conclusions: Mainly linked to internal drinking motives, drinking during the exams in higher education is underestimated. The characteristics and motives of students doing so can be used in future interventions.     

A Systems Approach to Social Entrepreneurship

Currently, disabilities organizations are increasingly being challenged by the requirement for individualized service, expectations to show personal outcomes, and the need to base their services on evidence-based practices. Social entrepreneurship (SE) is being put forward as an innovative approach for dealing with these challenges. This article presents a systems approach to SE based on a program logic model. This model identifies the input factors (a strong social vision, exploiting opportunities, maximizing resources), throughput strategies (entrepreneurial orientation, critical thinking skills, networking, capacity building), and outcome components (improving people’s lives, community-building, improving society) of SE at the micro, meso, and macro level. Also, the importance of planning for contextual changes as a social entrepreneur is discussed. The article concludes with presenting three inspiring practices regarding SE in the field of disabilities organizations.     

Moroccan women in Madrid: between change and continuity

This article focuses on individual migration and acculturation processes experienced by Moroccan women in the area of Madrid, based on qualitative research conducted in the Spanish capital in 2008. The results show that Moroccan women restructure their daily practices through complex acculturation processes, orienting them towards both the society of origin and that of arrival. They experience a constant struggle in getting to know the traditions of Spanish society, reorienting their former traditions and inventing new solutions. Moroccan women develop new ways of adapting themselves to their situation in Madrid. This acculturation process bridges the continuity with their culture of origin and reflects changes related to the new context. The studied women move in a transnational space with rigid borders, which is intersected by several social categories such as their education, ethnicity and gender, influencing their relative position within this space.     

Poking a sleeping bear: the challenge of organizational recruitment for controversial topics

ABSTRACT

Researchers often approach employers to investigate employees’ work and family experiences. Organizational willingness to grant access to employees can vary, especially when the research topic is seen as controversial or contentious for the employer. This paper explores this methodological challenge using a research example from Manitoba, Canada, which explored the use of parental leave by male employees and the impact of managerial attitudes and corporate culture on usage. Sixty large employers were recruited with only seven of those organizations agreeing to participate. In this paper, the reasons organizations gave for declining to participate and the implications of their decisions for the research are examined. Although the final sample included 905 managers and employees, participating organizations tended to be employee-focused and family-friendly employers. Organizations declined participation for a variety of reasons: avoiding raising the issue with unions, awareness that their policies unfairly benefited female leave takers, and simply not seeing the relevance of a topic relating to men’s work–family experiences. A dialogue often absent from the literature, it is important to understand how employers can limit researchers’ access to employees on controversial topics. The existence of such barriers suggests alternative avenues to recruit participants directly when topics are contentious for employers.     

There is an app for that? The impact of community knowledge workers in Uganda

Rapidly expanding mobile network coverage in developing countries offers new ways to reach poor farmers in isolated places. This article explores the potential for Information and Communication Technology to provide agricultural information and extension services to smallholder farmers, and links this to empirical insights of an intervention in Uganda, where community knowledge workers rely on mobile devices to deliver context- and time-sensitive data to farmers. Consistent with theoretical insights related to information inefficiencies, we find that the intervention leads farmers to move away from low-risk low-return crops toward more commercially oriented commodities. Our analysis also suggests that, for the case of maize, the intervention causes farmers to sell less on the market, but at significantly higher prices.