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.     

The Persistent Problem of Colorism: Skin Tone, Status, and Inequality

Colorism is a persistent problem for people of color in the USA. Colorism, or skin color stratification, is a process that privileges light-skinned people of color over dark in areas such as income, education, housing, and the marriage market. This essay describes the experiences of African Americans, Latinos, and Asian Americans with regard to skin color. Research demonstrates that light-skinned people have clear advantages in these areas, even when controlling for other background variables. However, dark-skinned people of color are typically regarded as more ethnically authentic or legitimate than light-skinned people. Colorism is directly related to the larger system of racism in the USA and around the world. The color complex is also exported around the globe, in part through US media images, and helps to sustain the multibillion-dollar skin bleaching and cosmetic surgery industries.     

Pleasing the students: teachers' orientaton to classroom life in adult education

It is noted that existing sociological research on classrooms has focused too narrowly on compulsory schooling with the result that we have no data showing the rituals and routines of Adult Classrooms or the ways that teachers cope and resolve them. The paper presents fieldnote and interview data from a small ethnographic study of the two Adult Evening classes. The data suggest that in contrast to the teacher of school aged pupils, who seeks control and discipline, ‘pleasing students’ is the major orientation to classroom life of the evening class teacher. Do not say, the people must be educated when after all you only mean amused, refreshed, soothed put into good spirits and good humour or kept from vicious excesses. (Newman, 1873).     

2½‐Year‐Old Children Use Animacy and Syntax to Learn a New Noun

We examine how attention to animacy information may contribute to children's developing knowledge of language. This research extends beyond prior research in that children were shown dynamic events with novel entities, and were asked not only to comprehend sentences but to use sentence structure to infer the meaning of a new word. In a 4 × 3 design, animacy status (e.g., animate agent, inanimate patient) and labeling syntax (agent, patient, nonlabel control) were varied. Across most events, 2 1/2‐year‐old participants responded as if they expected animate entities to be named. However, in a prototypical (animate agent‐inanimate patient) event condition, children responded differentially across different syntactic structures. Thus, the clearest evidence for attention to syntactic cues was found in the prototypical event condition. These results suggest that young children attend to the animacy status of unfamiliar entities, that they have expectations about animacy relations in events, and that these expectations support emerging syntactic knowledge.