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[S23] What Patient Registries Bring to Rare Disease Medicine Development
Session Chair(s)
Harumasa Nakamura, MD, PhD
Director of Department of Clinical Research Support,
National Center of Neurology and Psychiatry, Japan
In drug development for rare diseases, the small number of target patients often makes it difficult to collect the number of patients required to verify the drug’s effectiveness in a clinical trial. Therefore, pharmaceutical companies often do not encourage drug development for rare diseases. But in recent years, development of patient registries [for rare diseases?] has been vigorously promoted. This session will introduce practical use of, and current utilization problems with, patient registries for drug development from different expert points of view.
Speaker(s)
Current Situation and Future Perspectives of Review and Scientific Consultation about Orphan Drugs
Yoko Aoi, PhD
Pharmaceuticals and Medical Devices Agency (PMDA), Japan
Deputy Review Director, Office of New Drug V
The Role of Patient Registry in the Drug Development of HTLV-1-Associated Myelopathy
Yoshihisa Yamano, MD, PhD
Institute of Medical Science St. Marianna University Graduate School of Medicine, Japan
Director,Department of rare diseases reserch
Patient Registry Study in Novel Drug Development for Intractable Vascular Malformations
Hiroshi Nagabukuro, PhD
ARTham Therapeutics, Inc, Japan
COO / CSO
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