ORPHAN DRUGS IN ASIA 2017 Guidelines and Regulatory Requirements To Help Orphan Drug Products Enter the Asian Market Pacific Bridge Medical 7315 Wisconsin Avenue, Suite 609E Bethesda, MD 20814 Tel: (301) 469-3400 Fax: (301) 469-3409 Email: [email protected] Copyright © 2017 Pacific Bridge Medical. All rights reserved. This content is protected by US and International copyright laws and may not be copied, reprinted, published, translated, resold, hosted, or otherwise distributed by any means without explicit permission. Disclaimer: the information contained in this report is the opinion of Pacific Bridge Medical, a subsidiary of Pacific Bridge, Inc. It is provided for general information purposes only, and does not constitute professional advice. We believe the contents to be true and accurate at the date of writing but can give no assurances or warranties regarding the accuracy, currency, or applicability of any of the contents in relation to specific situations and particular circumstances. TABLE OF CONTENTS 1. INTRODUCTION ................................................................................................................................ 1 1.1 GLOBAL OUTLOOK ON RARE DISEASES ............................................................................................. 1 1.2 DEVELOPMENT OF ORPHAN DRUG LEGISLATION ............................................................................... 3 1.3 WELL-KNOWN RARE DISEASES ......................................................................................................... 4 1.4 US FDA CONTACT INFORMATION ..................................................................................................... 5 1.5 US ORPHAN DRUG ASSOCIATIONS ..................................................................................................... 5 1.6 US FDA ORPHAN DRUG APPROVALS (JANUARY 2010 TO JULY 2017) .............................................. 7 2. OVERVIEW OF ASIA ...................................................................................................................... 24 2.1 THE ASIAN ECONOMY ...................................................................................................................... 24 2.2 THE PHARMACEUTICAL MARKETS IN ASIA ...................................................................................... 24 3. ORPHAN DRUGS IN ASIA .............................................................................................................. 26 3.1 DO ORPHAN DRUGS HAVE POTENTIAL IN ASIA? ............................................................................... 26 3.2 WHY SEEK ORPHAN DRUG STATUS? ................................................................................................. 26 4. ISSUES TO CONSIDER PRIOR TO ORPHAN DRUG REGISTRATION ............................... 27 4.1 INTRODUCTION ................................................................................................................................. 27 4.2 ARE TREATMENT OPTIONS ALREADY AVAILABLE IN THE COUNTRY? ............................................... 27 4.3 IS YOUR DRUG SUPERIOR TO THOSE ALREADY ON THE MARKET? ..................................................... 27 4.4 CONCLUSION .................................................................................................................................... 28 5. JAPAN ................................................................................................................................................. 29 5.1 INTRODUCTION ................................................................................................................................. 29 5.2 MINISTRY OF HEALTH, LABOR AND WELFARE ................................................................................ 29 5.3 PHARMACEUTICALS AND MEDICAL DEVICES AGENCY .................................................................... 29 5.4 ORPHAN DRUGS IN JAPAN ................................................................................................................ 29 5.5 HEALTH AUTHORITY CONTACT INFORMATION ................................................................................ 46 5.6 ORPHAN DRUG ASSOCIATIONS ........................................................................................................ 46 5.7 ORPHAN DRUGS APPROVED IN JAPAN .............................................................................................. 48 5.8 ORPHAN DRUGS DESIGNATED (NOT YET APPROVED AND DESIGNATION NOT REVOKED) IN JAPAN .. 74 6. TAIWAN ............................................................................................................................................. 79 6.1 OVERVIEW ....................................................................................................................................... 79 6.2 TAIWANESE HEALTH AUTHORITIES ................................................................................................. 79 6.3 HEALTH INSURANCE SCHEME .......................................................................................................... 80 6.4 ORPHAN DRUGS IN TAIWAN ............................................................................................................. 80 6.5 HEALTH AUTHORITY CONTACT INFORMATION ................................................................................ 86 6.6 ORPHAN DRUG ASSOCIATIONS ........................................................................................................ 87 6.7 ORPHAN DRUGS APPROVED IN TAIWAN .......................................................................................... 88 7. KOREA ............................................................................................................................................... 96 7.1 OVERVIEW ....................................................................................................................................... 96 7.2 DRUG REGISTRATION OVERVIEW .................................................................................................... 96 7.3 ORPHAN DRUGS IN KOREA ............................................................................................................... 97 7.4 HEALTH AUTHORITY CONTACT INFORMATION .............................................................................. 103 7.5 ORPHAN DRUG ASSOCIATIONS ...................................................................................................... 103 7.6 ORPHAN DRUGS APPROVED IN KOREA .......................................................................................... 104 8. HONG KONG .................................................................................................................................. 120 8.1 OVERVIEW ..................................................................................................................................... 120 Copyright © 2017 Pacific Bridge Medical. i 8.2 HONG KONG HEALTH AUTHORITY ................................................................................................ 120 8.3 ORPHAN DRUGS IN HONG KONG .................................................................................................... 120 8.4 HEALTH AUTHORITY CONTACT INFORMATION .............................................................................. 125 9. CHINA ............................................................................................................................................... 126 9.1 OVERVIEW ..................................................................................................................................... 126 9.2 PHARMACEUTICAL MARKET .......................................................................................................... 126 9.3 CHINA HEALTH AUTHORITY .......................................................................................................... 126 9.4 DRUG REGISTRATION PROCESS ...................................................................................................... 127 9.5 DRUG PRICING ............................................................................................................................... 134 9.6 ORPHAN DRUGS IN CHINA ............................................................................................................. 134 9.7 HEALTH AUTHORITY CONTACT INFORMATION .............................................................................. 137 10. SINGAPORE .................................................................................................................................... 138 10.1 OVERVIEW ................................................................................................................................. 138 10.2 SINGAPORE HEALTH AUTHORITY .............................................................................................. 138 10.3 ORPHAN DRUG REGISTRATION ................................................................................................. 138 10.4 HEALTH AUTHORITY CONTACT INFORMATION ......................................................................... 139 11. SOUTHEAST ASIA INTRODUCTION ........................................................................................ 140 11.1 OVERVIEW ................................................................................................................................. 140 11.2 DRUG REGISTRATION REQUIREMENTS FOR SOUTHEAST ASIA .................................................. 141 12. PHILIPPINES