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.     

Flood Risk Perceptions and Insurance Choice: Do Decisions in the Floodplain Reflect Overoptimism?

In the presence of rare disasters, risk perceptions may not always align with actual risks. These perceptions can nevertheless influence an individual's willingness to mitigate risks through activities such as purchasing flood insurance. In a survey of Maryland floodplain residents, we find that stated risk perceptions predict voluntary flood insurance take‐up, while perceptions themselves varied widely among surveyed residents, owing in large part to differences in past flood experience. We use a formal test for overoptimism in risk perceptions and find that, on aggregate, floodplain residents are overly optimistic about flood risks.     

From Privilege to Prevalence: Contextual Effects of Women’s Schooling on African Marital Timing

In Africa and elsewhere, educated women tend to marry later than their less-educated peers. Beyond being an attribute of individual women, education is also an aggregate phenomenon: the social meaning of a woman’s educational attainment depends on the educational attainments of her age-mates. Using data from 30 countries and 246 birth cohorts across sub-Saharan Africa, we investigate the impact of educational context (the percentage of women in a country cohort who ever attended school) on the relationship between a woman’s educational attainment and her marital timing. In contexts where access to education is prevalent, the marital timing of uneducated and highly educated women is more similar than in contexts where attending school is limited to a privileged minority. This across-country convergence is driven by uneducated women marrying later in high-education contexts, especially through lower rates of very early marriages. However, within countries over time, the marital ages of women from different educational groups tend to diverge as educational access expands. This within-country divergence is most often driven by later marriage among highly educated women, although divergence in some countries is driven by earlier marriage among women who never attended school.     

Identity Development in a Transracial Environment: Racial/Ethnic Minority Adoptees in Minnesota

It has been argued that transracially adopted children have increased risk of problems related to self-esteem and ethnic identity development. We evaluated this hypothesis across four groups of transracial adoptees: Asian (n = 427), Latino (n = 28), Black (n = 6), mixed/other (n = 20), and same-race White adoptees (n = 126) from 357 adoptive families. No mean differences were found in adoptees’ ratings of affect about adoption or of curiosity about birth parents. Some differences were found in general identity development and adjustment. There were notable differences in communication about race/ethnicity across groups and between parent and child report.     

Solving Current Social Challenges: Engaging Undergraduates in Policy Practice

This article explores an innovation in teaching policy practice at the undergraduate level. It analyzes an experiential learning model that uses current social challenges as a compelling way to generate student learning about policy practice. Over the course of the semester, students examine antecedents, current issues, and potential solutions to a contemporary social issue through a series of assignments that build knowledge and skills and link the topic to larger social policy concerns. Using quantitative and qualitative evidence from seven semesters, the article explores benefits and challenges of this approach to teaching, and derives lessons for social work education.     

Assessing the contribution of fallen autumn leaves to airborne fungi in an urban environment

Urban Ecosystems - Many street trees in urban areas are deciduous and drop leaves during autumn. These leaves are a potential growing substrate for fungi, which when aerosolized and inhaled, can...     

Disabilities and sexual expression: A review of the literature

Since the late 1980s and early 1990s, scholars have sought to advance a more nuanced and robust understanding of the sexual lives of disabled people. In doing so, these scholars have identified some of the barriers to sexual expression that disabled people face as well as some of the positive and pleasurable aspects of disabled people's sexual lives. This article reviews research regarding the sexual expression and activity of disabled people as well as some of the challenges to sexual expression that people with disabilities experience. Persons with disabilities—whether they are acquired, congenital, intellectual, physical, and/or sensorial impairments—continue to encounter alienation, stigmatization, and discrimination, particularly in terms of their sexuality. However, I argue that if proper laws, policies, and adequate supports are in place, people with disabilities can further challenge and push past these barriers to engage in a wide array of sexual and erotic acts. Furthermore, although the amount of disability‐sexuality‐related research has increased over past decades, I argue that many intersections of disability and sexuality remain under‐studied and that further research in this field is necessary.