Therapeutic properties of antibodies strongly depend on the composition of their glycans. Most of the currently approved antibodies are produced in mammalian cell lines, which yield mixtures of different glycoforms that are close to those of humans, but not fully identical. Glyco-engineering is being developed as a method to control the composition of carbohydrates and to enhance the pharmacolo...

Biomarkers play an integral part in conducting clinical trials and treating patients. In most instances, they help medical practitioners, researchers, and regulatory officials make well-informed, scientifically sound decisions. However, in clinical studies, there is often uncertainty in how much weight to place on biomarker results versus clinical outcomes. This uncertainty emanates from opposi...

An agent (such a pharmaceutical company) sequentially collects information (through clinical trials) to obtain a principal’s (the FDA) approval (to introduce a new drug). Principal and agent both pay the cost of information, but they derive different payoffs from adoption. We characterize how the principal can benefit from commitment to an approval standard and how the commitment solution compa...

Results More orphan drugs were developed in the US during the first ten years of the US Orphan Drug Act (1983-1992, N=73) and during the first ten years of the EU Regulation on Orphan Medicinal products (2000-2009, N=112) than in the EU (2000-2009, N=59). Orphan drug approval was strongly associated with previous experiences of the sponsor in obtaining approval for another orphan drug (OR=17.3,...

Cognitive impairment in schizophrenia is a core feature of the illness (i.e., not a result of clinical symptoms or drug treatments) that is related to the daily functioning of patients. Because schizophrenia is associated with poor community functioning, there is considerable interest in finding treatments to improve cognition in schizophrenia in the hopes that such improvement will yield funct...