Deputy Chairperson, PMDA Regulatory Science Research Support Committee Specially Appointed Professor, University of Tsukuba Tsukuba Clinical Research and Development Organization Yoshihiro Arakawa The candidates for future research are listed below. I hope these could serve as useful suggestions for research. multifactorial diseases such as neurological diseases; shift from symptomatic therapies to disease-modifying therapies / 26 In the review for approval of drugs and other products, it is essential to continuously gather information on the latest technological trends, and to understand both the possibilities and the potential risks of the products. For potential risks, in particular, it is important not only to consult experts, but also to proactively identify risks based on their own research experience. Currently, PMDA staff are overwhelmed with daily review duties and have limited time for research and self-improvement. However, in order to maintain and improve the quality of reviews, it is strongly expected to promote their research activities. [Regional characteristics] PMDA is a regulatory agency that serves as a key role in the ICH’s three major regions and is expected to evaluate the efficacy and safety in the region on behalf of East Asia. In particular, now that international clinical trials are becoming the standard, it is crucial to independently analyze the regional characteristics (genetic background, lifestyle, and medical care system) in East Asia and consider these factors in discussions regarding the reliance in East Asia, as well as in ordinary Regulatory Science (RS) consultations. [Impact of changes in disease structure] As the focus of research and development shifts from lifestyle-related diseases and cancers to rare diseases and intractable diseases, the profile of required drugs, etc. has been changing. There are many issues to be studied, and it has become necessary to discuss even the medical care system itself. These issues include: methods for identifying target molecules in preemptive medicine before disease onset; shift towards preventive medicine based on genomic information; development of clinically applicable biomarkers and clinical evaluation methods for slowly progressive diseases. [Changes in modality] For biological drugs, cell / gene therapies and other new modalities, the development of guidelines based on scientific evidence is required. There still remains a large gap between in vitro and in vivo studies and in the extrapolation from animal models to humans. In this regard, it is essential for reviewers to constantly update their knowledge, including by the interviews with both domestic and overseas experts and the continuous discussions with other regulatory agencies such as FDA. 4. Message from external expert
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