Innovation in Drug Assessment Technology Based on Human Biology/CRDS-FY2007-SP-16
Executive Summary

In the current development of new drugs, there are serious problems such as a decline of the success rate and enormous research and development costs. Regarding new drugs represented by biotechnology-based drugs and molecularly-targeted drugs in recent years, the technology to assess their safety and efficacy has not been established yet, which has become one of the causes for prolongation of clinical development. Furthermore, despite that there are the wealth of fundamental technologies in Japan that can be applied to drug assessment such as a technology to create iPS cells and a measuring technology using optic engineering, many of them have not been fully utilized as the technology for drug assessment. In addition, safety and efficacy assessment data in the drug development phases from the pre-clinical to clinical studies have not been integrated and many of the technologies are not linked to acceleration of the entire drug development.

Based on the above situation, we propose to promote research and development to establish a “Drug Assessment Technology Based on Human Biology” as the integrated assessment technology to predict and prove the safety and efficacy of drugs in a highly accurate manner. The “Drug Assessment Technology Based on Human Biology” is the integrated assessment technology to predict and prove the safety and efficacy of drugs in a highly accurate manner based on recent development of life science taking advantage of deciphering the human genome. It develops an assessment system that integrates from pre-clinical to clinical phases in the drug development by standardizing human biological materials and human biological information and unifying them organically. When further amplified, it conducts research and development of the following elemental technologies.

[1] Development of cellular and organ materials mimicking human bodies and model animals
[2] Development of an objective clinical assessment method using molecular imaging, etc.
[3] Development of a technology to consolidate/integrate assessment experiment data by mathematical model, etc.

When promoting these research and development, it is desired to establish a virtual research and development platform to let the people be clearly aware of the exit of drug development and implement them under strong leadership by PO, etc. Specific targets should be laid out, for example, to establish an integrated assessment experiment system to predict toxicity of low-molecule compounds and an immunotherapy assessment system in the development of antibody drugs and to develop an assessment technology for the safety in the body in the development of nucleic-acid drugs, and the related assessment technology groups should be promoted in an integrated manner.
With this research and development, it will become possible to eliminate the difference in drug responsiveness that is attributed to species difference, which was difficult to evaluate with the conventional in vivo response assessment using model animals such as rodents. Furthermore, the establishment of a technology to consolidate the safety and efficacy assessment data will enable to build a system to accurately assess the efficacy and safety based on human materials from the clinical phase. In addition, this research and development can make a great contribution to the development of regulatory science to achieve quick review and approval of drugs in response to the current situation in which pharmaceutical companies, companies commissioned with drug assessment studies, governmental offices responsible for review and approval of drugs have been unable to share the latest technologies.