Abstract
For the selection of clinical candidate-compound in the early stage of drug discovery, it is important to estimate the human prediction from the results of efficacy and safety studies in experimental animals. The research and development studies by using human tissues are generally to be done for the purpose of screening the model system development, effective target defining as a medicine, and estimating the target distribution and function regarding efficacy and safety. Recently it has been clearly articulated that insufficient curative-medicine is existing in some disease areas such as the therapeutic medication of cancer and central nervous system, despite considerable diseases become curable or improvable in these ten years. Tremendous progress of science and technology in molecular biology and other scientific disciplines has led to have a better understanding the disease-biology and its mechanism. As a recent trend, following approaches have become an urgent issue in order to improve or get cured of obstinate diseases. ①Understanding the disease itself based on molecular biological genome-science and technology. ②Efficient biomarker setting for translation from pre/non clinical experimental data to clinical development research ③Selection of the right patient by using precise diagnosis. Basic consideration for improving/curing disease should take a direction toward the questions as follows : “Right target?” ; to identify right target and its role of cellular pathways, “Right molecule?” ; to identify molecule and optimize their properties, “Right patient?” ; to prove molecule efficacy and safety in indicated/selected patients. These processes would lead to precision medicine from one-size-fits-all through personalized medicine.
