This chapter describes how functional nucleic acids, such as aptamers, antisense oligonucleotides (ASOs), small interfering (si) RNAs, and ribozymes are considered by some researchers as valuable tools to develop therapeutic agents. They have not been particularly fast in reaching the market as...

Increasing the participation of women and minorities in clinical trials is a challenge for the healthcare industry. The lack of diversity in clinical trials prevents the tailoring of healthcare interventions specifically for women and minorities. The purpose of this paper is to explore how...

An urn contains balls of d = 2 colors. At each time n = 1, a ball is drawn and then replaced together with a random number of balls of the same color. Let An =diag (An,1, . . . ,An,d) be the n-th reinforce matrix. Assuming EAn,j = EAn,1 for all n and j, a few CLT's are available for such urns....

This paper discusses how household panels in general - and the German Socio-Economic Panel (SOEP) in particular - can serve as reference data for researchers collecting datasets that do not represent the full universe of the population of interest (e.g., through clinical trials, intervention...

This paper discusses how household panels in general?and the German Socio- Economic Panel (SOEP) in particular?can serve as reference data for researchers collecting datasets that do not represent the full universe of the population of interest (e.g., through clinical trials, intervention...

Abstract We develop a transport formula for predicting overall cumulative vaccine efficacy through time t ( $$VE(t)$$ ) to prevent clinically significant infection with a genetically diverse pathogen (e. g., HIV infection) in a new setting for which a Phase III preventive vaccine efficacy...

Abstract Providing terminally ill patients with access to experimental treatments, as allowed by recent “right to try” laws and “expanded access” programs, poses a variety of ethical questions. While practitioners and investigators may assume it is impossible to learn the effects of...

Alemayehu and Levenstein offer an industry perspective on ghoswriting. They propose some general principles to prevent the types of egregious cases that have been reported, but more specifics are needed to understand how to combat this serious problem.

Two-stage predictor substitution (2SPS) and the two-stage residual inclusion (2SRI) are two approaches to instrumental variable (IV) analysis. While 2SPS and 2SRI with linear models are well-studied methods of causal inference, the properties of 2SPS and 2SRI for logistic binary outcomes have...