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Publication Date: February 2010
"I was very impressed by the report's usefulness and comprehensiveness"
Senior Official, Health Insurance Bureau, Ministry of Health, Labor and Welfare, Japan
It is estimated that 6-10% of the world’s population will suffer from a rare disease at one point in life. On this basis, the markets of Asia-Pacific should offer great potential to companies developing orphan drugs. The region’s population is 4 billion, nearly two-thirds of the global total, so even the rarest disease will have many patients seeking treatment.
But clinical need rarely correlates with affordability, which dictates what is actually spent on healthcare. A major new report Orphan Drugs in Asia-Pacific: From designation to pricing, funding and market access focuses on five developed markets in the region: Japan, South Korea, Taiwan, Singapore and Australia. Each has passed specific measures to promote the development, registration and/or commercialisation of orphan drugs. Singapore’s Medicines (Orphan Drug) Exemption Order of 1991 was the first legislation anywhere to follow the US Orphan Drug Act, with the other four Asia-Pacific countries also implementing orphan-promoting measures ahead of the EU.
This report describes the processes for obtaining orphan product designation, what can be omitted from the standard dossier to obtain marketing authorisation, and provides full details of the regulatory bodies and timelines involved. The main emphasis though is not on registration but on market access, which for products like orphan drugs that often carry premium prices, means eligibility for reimbursement or other sources of public funds.
Based on extensive research, including fieldwork in Tokyo and Seoul in November 2009 to capture detail, understanding and all the latest trends, the report includes:
- The incentives on offer to orphan drug sponsors.
- How P&R processes work specifically for orphan drugs.
- The role of HTA and risk sharing as levers to obtain reimbursement.
- Where special funding schemes operate and how to access them.
- 13 case studies on the P&R history of 12 different orphan drugs in 5 countries.
- How much patients are expected to contribute to the cost of their treatment.
- Listings of designated and approved orphan drugs.
- Comparisons between countries with orphan drug policies and those without.
- Contact details of the main rare disease patient organisations.
Learn which of the Asia-Pacific countries ...offers the most comprehensive programme for the management of rare disease…often enjoys higher orphan drug prices than the US...has the most orphan drugs on the market and reimbursed…promotes the development of rare disease medical devices as well as drugs…where ‘named patient’ imports are discouraged by rare disease patient representatives.
About the Author
With well over 50 major titles to his name, Donald Macarthur is one of the world’s most experienced and widely read commercial biopharmaceutical writers. Known for his clear, succinct and eminently readable style, where the emphasis is on imparting understanding. Every report deals with the subject comprehensively in a neutral, fact-based manner. Each is based on up-to-the-minute research by the author himself - largely primary research in the field - among government officials,industry and other experts on the topic concerned. Rather than a rehash of the published literature readers will find detail, insight and forecasts not found elsewhere.
For two years up to June 2007, Don Macarthur was senior consultant with the strategic international pharmaceutical pricing, reimbursement and market access consultancy PriceSpective. From 2001-2005, Mr Macarthur was Secretary General of the European Association of Euro-Pharmaceutical Companies, the grouping of national associations and individual firms involved in parallel trading of medicines with membership from 16 EEA countries.
His current role as independent consultant and analytical writer also occupied the bulk of the 16 years preceding 2001, with a focus on drug cost containment policy worldwide, pricing and reimbursement, hospital access, European integration and enlargement, wholesale and retail distribution, mail order, homecare, orphan drugs, generics, parallel trade, Rx-to-OTC switching, and several aspects of the Japanese market and industry.
He has written over 50 major reports. Published by Scrip, IMS Health, Financial Times/Informa, Taylor Nelson Sofres Healthcare, Decision Resources, Droit & Pharmacie, PPR Communications and himself, several have become industry standards. He also founded, edited and published for four years the world’s first periodical on drug pricing and reimbursement, Pharma Pricing Review.
Consultancy clients have included the European Federation of Pharmaceutical Industries and Associations - he was the very first consultant ever used by EFPIA - Pharmaceutical Research and Manufacturers of America (PhRMA), the European grouping of national full-line pharmaceutical wholesaling associations (GIRP), the UK’s Office of Fair Trading, other government agencies, and many major multinational biopharmaceutical manufacturers, legal and financial firms.
Numerous presentations have been given at international conferences, and he has testified both at a US Senate Committee hearing and at the HHS Secretary’s task force hearing on prescription drug importation. His articles have appeared in many English language pharmaceutical business journals, with some translated into Japanese, German and Turkish.
Qualifying as a pharmacist from the University of London, Mr Macarthur’s early career involved community and hospital pharmacy practice, 16 years in development, regulatory affairs and medical department functions in the pharmaceutical industry in the UK (Fisons, 1969-72; Parke-Davis, 1972-75; Roche, 1976-78; Serono, 1978-82; Lundbeck 1982-84), one year in Japan, and four years with PJB Publications (publishers of Scrip).
Table of Contents
2.1 Healthcare and Health Insurance System
2.2 Orphan Drug Legislation
2.2.1 Orphan Drug Development Programme, 1993
2.2.2 Qualifying Criteria
22.214.171.124 R&D Support
126.96.36.199 Protocol Assistance
188.8.131.52 Expedited Regulatory Review
184.108.40.206 Lower Regulatory Fees
220.127.116.11 Market Exclusivity
2.3.2 Marketing Approval
2.4 Pricing & Reimbursement
2.4.1 Process for New Drugs
18.104.22.168 Price Comparators
22.214.171.124 Price Premiums
126.96.36.199 Cost Calculation
188.8.131.52 Foreign Price Adjustment
2.4.2 Health Economic Considerations
2.4.4 Price Revision Process
2.5 Funding for Orphan Drugs
2.6 Prescribing Controls
2.7 Patient Co-payment
2.9 Patient Access
2.9.1 Unapproved Products
2.9.2 Marketed Products
2.10 Case Studies
2.11 Rare Disease Organisations
2.12 Pending Reforms
3.1 Healthcare and Health Insurance System
3.2 Orphan Drug Legislation
3.2.1 Qualifying Criteria
184.108.40.206 R&D Support
220.127.116.11 Protocol Assistance
18.104.22.168 Expedited Regulatory Review
22.214.171.124 Lower Regulatory Fees
126.96.36.199 Market Exclusivity
3.4 Pricing & Reimbursement
3.4.1 Process for New Drugs
3.4.2 Health Economic Considerations
3.4.4. Price Revision Process
3.5 Funding for Orphan Drugs
3.6 Prescribing Controls
3.7 Patient Co-payment
3.8 Patient Access
3.9 Korea Orphan Drug Center
3.10 Case Studies
3.11 Rare Disease Organisations
3.11.1 Genetic and Rare Diseases Center
3.11.2 Korean Organisation for Rare Diseases
4.1 Healthcare and Health Insurance System
4.2 Orphan Drug Legislation
4.2.1 Qualifying Criteria
4.3.1 R&D Support
4.3.2 Protocol Assistance
4.3.3 Expedited Regulatory Review
4.3.4 Lower Regulatory Fees
4.3.5 Market Exclusivity
4.5 Pricing & Reimbursement
4.5.1 Process for New Drugs
4.5.2 Health Economic Considerations
4.5.3 Price Revision Process
4.6 Funding for Orphan Drugs
4.7 Prescribing Controls
4.8 Patient Co-payment
4.9 Patient Access
4.10 Case Study: Zavesca in Niemann-Pick Disease Type C
4.11 Rare Disease Organisations
5.1 Healthcare and Health Insurance System
5.2 Orphan Drug Legislation
5.2.1 Qualifying Criteria
5.4 Pricing & Reimbursement
5.6 Compassionate Use
6. Other Asian Countries
6.2.1 Case Study: Glivec
7.1 Healthcare and Health Insurance System
7.2 Orphan Drug Legislation
7.2.1 Qualifying Criteria
7.3.1 R&D Support
7.3.2 Protocol Assistance
7.3.3 Expedited Regulatory Review
7.3.4 Lower Regulatory Fees
7.3.5 Market Exclusivity
7.5 Pricing & Reimbursement
7.5.1 Process for New Drugs
7.5.2 Criteria Employed
7.5.3 Health Economic Considerations
7.5.4 Price Revision Process
7.6 Funding for Orphan Drugs
7.6.1 Highly Specialised Drugs Programme
7.6.2 Life Saving Drug Programme
7.7. Patient Co-payment
7.8 Patient Access
7.9 Case Studies
7.10 Rare Disease Organisations
8. New Zealand
8.1 Healthcare and Health Insurance System
8.2 Orphan Drug Provisions
8.3 Pricing & Reimbursement
8.4 Funding for Orphan Drugs
8.5 Patient Co-payment
8.6 Patient Access
8.7 Rare Disease Organisations
List of Tables
1.1 Basic statistics on selected countries in Asia-Pacific
1.2 Results of first five years of orphan drug legislation by country/region
2.1 Orphan drug/device designations and approvals in Japan (1993-2008)
2.2 Orphan drug designation in Japan by origin of sponsor
2.3 Orphan drug designation in Japan by main disease area target
2.4 Authorised orphan drugs by leading therapeutic area, US, EU
2.5 Orphan drugs granted marketing approval in Japan, FY 2005-08
2.6 Average time from first marketing in world to local marketing
2.7 Regulatory approval dates with orphan drugs, Japan, US, EU
2.8 Price premiums for new drugs in Japan
2.9 Best-selling orphan drugs in Japan, 2008
2.10 Diseases in Japan with patient co-payment ceiling
3.1 Reimbursement listing success rates in Korea, pre- and post-DERP
3.2 Patient co-payments in Korea, 2009
3.3 Products procured/supplied by KODC, first half 2009
3.4 P&R history of Glivec in Korea, 2001-2009
Glossary of Abbreviations and Acronyms
1. Cross-national comparison of orphan drug policies
2. Korean-designated orphan drugs with marketing authorisations
3. Taiwan-designated orphan drugs with marketing authorisations
4. Australian-designated orphan drugs and those with marketing authorisations
5. International comparison of manufacturer selling prices of selected orphan drugs