S. Altan, J. Kolassa
Apr 3, 2019
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Influential Citations
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Journal
Statistics in Biopharmaceutical Research
Abstract
This special issue on nonclinical statistical applications draws on invited and contributed presentations given at the NCB2017 conference held June 12–14, 2017 at the Piscataway campus of Rutgers University. The conference was the fourth biennial nonclinical conference in the US, alternating with a corresponding conference held in Europe on even numbered years. The conference was jointly sponsored by the ASA’s Biopharmaceutical Section and Rutgers University, Statistics Department. Of the nearly two dozen presentations and many posters, nine manuscripts were selected for publication in this special nonclinical issue. The papers represent a diverse range of statistical tools and highlight the rich and fertile ground that nonclinical scientific studies represent for innovative statistical applications. In setting the stage for the research papers chosen for this issue, we wanted to address concerns raised by some about the origin of the name “nonclinical statistics.” We note that it is unusual to name a discipline by a negative, in other words by what it’s not. In the field of statistics, it may be the only major area of application named in this way. For that matter, perhaps in all of science, for example, who has heard of nonclinical chemistry, or nonclinical biology? Much of the reason for this is that the term refers to statistical applications specific to the pharmaceutical industry. The industry has for convenience established a dichotomy between studies focused on human subjects, referred to as clinical studies, in contrast to scientific and engineering studies not involving human subjects, which are the nonclinical studies. These cover a wide variety of scientific disciplines. For convenience, we categorize these into three major areas of scientific studies: (1) discovery/pharmacology/-omics, (2) drug safety/toxicology, and (3) chemistry, manufacturing and controls (CMC). The discovery/pharmacology/-omics studies focus on chemical identification and biological and genetic properties of new molecular entities, NME for short. These can be “small” molecules in contrast to biologically derived molecules, referred to as new biological entities or NBE for short. The preclinical drug safety/toxicology area studies the toxicity and systemic effects in animal and in vitro models of the NME. CMC studies consist of formulating the NME into a drug product that has good drug performance characteristics (shelf stability, human absorption, and metabolism) and developing analytical tools for characterizing the quality and purity of the final drug product. The statistical applications in the nonclinical studies cover the entire life cycle of the drug development process, and play an important role in decision making, risk assessments, and improving the efficiency of the drug development process.