Inference and Decision Making for 21st-Century Drug Development and Approval

ABSTRACT

The cost and time of pharmaceutical drug development continue to grow at rates that many say are unsustainable. These trends have enormous impact on what treatments get to patients, when they get them and how they are used. The statistical framework for supporting decisions in regulated clinical development of new medicines has followed a traditional path of frequentist methodology. Trials using hypothesis tests of “no treatment effect” are done routinely, and the p-value < 0.05 is often the determinant of what constitutes a “successful” trial. Many drugs fail in clinical development, adding to the cost of new medicines, and some evidence points blame at the deficiencies of the frequentist paradigm. An unknown number effective medicines may have been abandoned because trials were declared “unsuccessful” due to a p-value exceeding 0.05. Recently, the Bayesian paradigm has shown utility in the clinical drug development process for its probability-based inference. We argue for a Bayesian approach that employs data from other trials as a “prior” for Phase 3 trials so that synthesized evidence across trials can be utilized to compute probability statements that are valuable for understanding the magnitude of treatment effect. Such a Bayesian paradigm provides a promising framework for improving statistical inference and regulatory decision making.     

Secondary analysis of dangerousness among death sentenced capital murderers

The theory of incapacitation involves reducing an offender's ability or capacity to commit further crimes. Capital punishment accomplishes this goal. An executed murderer never murders again. However, we do not execute all murderers, only capital murderers. This policy produces several research questions. Do capital murderers present a special risk to society? Are capital murderers more likely to murder or commit other violent crimes again than other murderers or the average citizen? To answer these questions, many states require a prediction of future dangerousness of a newly convicted murderer. To what extent has the judgment of future dangerousness matched actuarial data of subsequent murders and serious crimes? Using a secondary analysis, this investigation attempted to assemble available data of postconviction dangerousness of death sentenced capital murderers to create a more comprehensive actuarial account of subsequent dangerousness and to present the data in a common format used by the Federal Bureau of Investigation and the Bureau of Justice Statistics. Across 14 studies identified with relevant data, there were 13 instances of subsequent murder and 462 serious crime or prison rule violations.     

Working children: the health and safety issue

Recent evidence has emerged that in Britain, like the US, many children below the official minimum school leaving age, are working. In many cases, the work is illegal. Research in the US suggests that many working children risk accidents and other hazards to health. Evidence from Britain on the health and safety aspects of child labour is of a more fragmentary nature, but enough exists to suggest a need for greater vigilance.     

The experience of sole mothers balancing paid work with care for a child with a disability

There are increasing numbers of families in Australia headed by sole parents, but little is known about the experiences of those who also care for a child with a disability. Additional caring responsibilities have previously been shown to impact on the work participation of parents. This study involved qualitative analyses of interview data conducted with sole mothers with school‐aged children with disability (N = 11). Thematic analysis revealed four themes that enabled and supported participation in the workforce: social support; managing appointments; characteristics of the workplace; and the role of the school. Results suggest the importance of practical help from family and friends in facilitating participation in paid employment. Additionally, the importance of flexible appointment scheduling on the part of service providers was highlighted, as well as the importance of workplace flexibility and supportive workplace cultures. Such factors were important in supporting sole mothers to balance work with care, with important implications for personal and family wellbeing.     

Preference for realistic art predicts support for Brexit

Following the UK’s EU referendum in June 2016, there has been considerable interest from scholars in understanding the characteristics that differentiate Leave supporters from Remain supporters. Since Leave supporters score higher on conscientiousness but lower on neuroticism and openness, and given their general proclivity toward conservatism, we hypothesized that preference for realistic art would predict support for Brexit. Data on a large nationally representative sample of the British population were obtained, and preference for realistic art was measured using a four‐item binary choice test. Controlling for a range of personal characteristics, we found that respondents who preferred all four realistic paintings were 15–20 percentage points more likely to support Leave than those who preferred zero or one realistic paintings. This effect was comparable to the difference in support between those with a degree and those with no education, and was robust to controlling for the respondent’s party identity.     

The Effect of Evaluator Masculinity on Dyadic Hiring Decisions

Gender Issues - Research examining gender disparities in the workplace tends to focus on individual behavior rather than the behavior of teams or dyads, yet important workplace decisions such as...     

The risks and incentives of disciplinary neoliberal feminism: the case of microfinance

This article develops an analysis of the concept of disciplinary neo-liberal feminism through a focus on gendered poverty alleviation strategies and illustrates the value of this through a discussion of microfinance. By locating this study within an analysis of the expansion of global capital accumulation, the article argues that the liberal frameworks of female empowerment and entrepreneurialism that are central to these programs and to feminism in this form, mask their underlying political, social and economic objectives. In contrast, a Marxist Feminist approach more adequately explains the interplay of class and gender that underpins poverty alleviation strategies. This article argues that in the context of financial crisis and reduced social provision, women living in poverty in the Global South were identified as targets for the expansion of global finance. Their integration into global financial networks via microfinance and other pro-poor strategies has facilitated the expansion of markets for credit while at the same time disciplining market participation through the twin forces of risk and incentive. Disciplinary neo-liberal feminism has underpinned this incorporation of women into global capital accumulation creating profound effects for households and families, with microfinance programs representing important sites of contestation for the politics of class and gender.