Development of Orphan Drugs in Japan: Effects of a Support System for Development of Orphan Drugs in Japan


In order to promote development of orphan drugs, the Japanese Ministry of Health and Welfare introduced support measures for drug development in 1985 and 1993. To see if these support measures were really effective for drug development, we compared the number of orphan drugs and review periods required for their approval in three different periods. The number of approved drugs in a year of Term 3 (after 1993) was approximately 3.3 times that of Term 1 (before 1985). The median value of the review period required for approval of drugs in Term 3 was 15.5 months, shorter than that in Term 1 by 10.5 months. It was clearly shown that the number of drugs developed was increased and the review period was significantly reduced because of the support measures. Now it is possible for patients suffering from orphan diseases to get access to new and more effective drugs sooner.

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  1. 1.

    Notification No. 2. Notification of Director of Pharmaceuticals and Cosmetics Division I, Director of Safety Division and Director of Biologics and Antibiotics Division, Pharmaceutical Affairs Bureau, MHW, Data should be attached in case of Application for manufacturing and import of orphan drug. June 29, 1985.

  2. 2.

    Hirai T, Urayama T. Intractable diseases and orphan drugs. Farumashia. 1995;31(7):770–778.

    Google Scholar 

  3. 3.

    Shiragami M. Forefront of orphan drugs—Overview of orphan drugs development supporting system. Dispensing Drug Inf. 1999;5(7).

    Google Scholar 

  4. 4.

    Medical Economics Division, Health Insurance Bureau, MHW ed. NHI Drug Price System. Tokyo: The Yakujinippo Limited; 1998;88.

    Google Scholar 

  5. 5.

    Ueki A. Development promotion measure of orphan drugs newly introduced. Bio Tech. 1993;7:206.

    Google Scholar 

  6. 6.

    Research Division, Pharmaceutical and Medical Bureau, MHW (supervised). Guideline for Drug Manufacture. 1997 edition. Tokyo: Jiho, Inc.; 1997;582–632.

    Google Scholar 

  7. 7.

    Research and Development Division, Pharmaceutical Affairs Bureau, MHW (supervised). Handbook of Orphan Drugs. Second edition. Tokyo: Jiho, Inc.; 1996:3–17.

    Google Scholar 

  8. 8.

    Organization for Pharmaceutical Safety and Research ed. Orphan Drug Development Promotion System. Tokyo, Organization for Pharmaceutical Safety and Research; 1999.

    Google Scholar 

  9. 9.

    Asbury CH. The Orphan Drug Act. JAMA. 1991;265:893–897.

    CAS  Article  Google Scholar 

  10. 10.

    Holmer AF. Evidence by the Chairman of Pharmaceutical Research and Manufacturers of America in Budget Committee of the US Senate, PhRMA Statement 1998.

    Google Scholar 

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Correspondence to Dr. Makoto Shiragami PhD.

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The views expressed in this article are solely those of the authors, and official endorsement by the Ministry of Health and Welfare or The Organization for Pharmaceutical Safety and Research should not be inferred.

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Shiragami, M., Nakai, K. Development of Orphan Drugs in Japan: Effects of a Support System for Development of Orphan Drugs in Japan. Ther Innov Regul Sci 34, 829–837 (2000).

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Key Words

  • Orphan drug
  • Support system for orphan drugs
  • Approval of orphan drugs
  • Review period