Patient Database and Orphan Drug Development

Mizushima Hiroshi, Tanabe Mai, Kanatani Yasuhiro

doi:10.1248/yakushi.13-00256-4

There is no concept of rare disease (RD) but Nambyo (intractable disease) since 1972. In 1995 the definition of Nambyo included the concept of rareness and the frequency for a Nambyo is less than 50000 in Japanese population. Currently Nambyo are selected for special research support, and special treatment of medical expenses. The budget for research is 10 billion yen. The approximate number of medical recipients is estimated to be 700000. The measures already developed for Nambyo are not comprehensive, therefore currently several additional measures are being envisaged. We are now planning to join the Orphanet. The expectations for Orphanet Japan are to: Enhance international collaboration of RD, providing international up-to-date information of RD in Japanese, inform historical and up-to-date research of Nambyo, and promote information exchange, joint research and network establishment. It is necessary to make a Patient Registry for rare disease, and hopefully have a structure to integrate worldwide registry with same concept. Recently “International Rare Disease Research Consortium (IRDiRC)” was formed. The purpose of this consortium is to make an international coordination of the rare disease research, and to integrate the knowledge of the rare disease research. We will also talk about the Patient Registry by Patient Advocacy group, including Patient Reported Outcome (PRO).<br>

Patient Database and Orphan Drug Development

Bibliographic Information

Search this article

Abstract

Journal

Citations (1)*help

Keywords

Details 詳細情報について

Export

Report a problem