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16th DIA Japan Annual Meeting 2019

Delivering Rational Medicine for All People in the Globe

[S23] What Patient Registries Bring to Rare Disease Medicine Development

Session Chair(s)

Harumasa  Nakamura, MD, PhD

Harumasa Nakamura, MD, PhD

  • Director of Clinical Research Support Office, Translational Medical Center
  • 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.


Yoko  Aoi, PharmD

Current Situation and Future Perspectives of Review and Scientific Consultation about Orphan Drugs

Yoko Aoi, PharmD

  • Principal Reviewer, Office of New Drug V
  • Pharmaceuticals and Medical Devices Agency (PMDA), Japan
Yoshihisa  Yamano, MD, PhD

The Role of Patient Registry in the Drug Development of HTLV-1-Associated Myelopathy

Yoshihisa Yamano, MD, PhD

  • Director,Department of rare diseases reserch
  • Institute of Medical Science St. Marianna University Graduate School of Medicine, Japan
Hiroshi  Nagabukuro, PhD

Patient Registry Study in Novel Drug Development for Intractable Vascular Malformations

Hiroshi Nagabukuro, PhD

  • COO / CSO
  • ARTham Therapeutics, Inc, Japan