In the last twenty years, the environment of new drug development and regulatory review have significantly changed. Regarding the statistical guidance, “Statistical Principles for Clinical Trials” issued in 1998, which is internationally agreed in ICH framework, the preparation of the addendum is considered. New drug development is implemented internationally with multi-regional clinical trials, and simultaneous submission and approval in many regions are expected. How to investigate and consider the influence of ethnic factors to drug efficacy and safety based on the results of multi-regional clinical trials of the drug is also discussed internationally. To improve the efficiency of drug development, comprehensive utilization of the data obtained at each stage of development with modeling & simulation, and adaptive and efficient study design are actively discussed. Also discussion have been started on how to utilize the data from data source other than controlled clinical trials, such as real world data and patient registry data, for new drug application and post marketing surveillance. Under these circumstances, biostatisticians will deal with more kinds of data with various quality levels, and will cooperate with various areas of specialization. In this talk, several topics currently discussed internationally as well as in Japanese regulatory agency, and future expected roles of biostatisticians will be explained.
Dr. Yuki Ando is a Senior Scientist for Biostatistics of the Pharmaceuticals and Medical Devices Agency (PMDA), Japan.
She received a master’s degree in Engineering from Tokyo Science University, and PhD in Health Science from Osaka University.
In 1997, she joined the Pharmaceuticals and Medical Devices Evaluation Center (PMDEC), which was established that year and subsequently transformed into the current PMDA. Currently she is responsible for the biostatistics review and consultation in the new drug and device review offices in PMDA. Additionally, she works as a leader of business part of Advanced Review with Electronic Data Promotion Group, the group which is responsible for the use of patient level electronic study data that are submitted with new drug applications in Japan. She is responsible for promoting CDISC implementation and the use of submitted electronic data in new drug review in PMDA.