Developing orphan drugs can be a highly profitable activity for the pharmaceutical industry when legislation provides the right incentives. Laws to encourage the development of orphan drugs have already been passed in the US and in Japan, and it is expected that similar EU regulations will be adopted before long.
Orphan Drugs: Medical breakthroughs and commercial opportunities is a timely Scrip report presenting the latest EU developments in this field, and comparing and contrasting these with schemes already operating in other countries.
The report provides a step-by-step guide through the processes involved in obtaining orphan designation for R&D candidates, to those companies interested in their development, and includes detailed analyses of experiences from companies already in the field.
Orphan Drugs: Medical breakthroughs and commercial opportunities will answer all the key questions on the issue of orphan drugs, including:
PUBLICATION: February 2000
REFERENCE: BS1037C
PAGES: 140
PRICE: �395/$830/�95,000
CONTENTS
LIST OF TABLES
ACKNOWLEDGEMENTS
EXECUTIVE SUMMARY
ABBREVIATIONS
REPORT OVERVIEW
CHAPTER 1 INTRODUCTION
1.1 Orphan drugs
1.2 Orphan diseases
1.3 Classification
1.3.1 Reclassification
1.3.2 Orphan dosage forms
1.3.3 Orphan products for tropical diseases
1.4 Other distinguishing characteristics
CHAPTER 2 THE DEMANDS OF PATIENTS
2.1 'Named-patient' use
2.2 Development of the US Act
2.2.1 Origins
2.2.2 Progress
2.2.3 Concluding steps
2.3 Patient access
2.3.1 US patient assistance programmes
2.3.2 Other countries
2.4 Paediatric labelling
2.5 Patient advocacies today
2.5.1 National Organisation for Rare Diseases
2.5.2 European Organisation for Rare Disorders
CHAPTER 3 US ORPHAN DRUG ACT
3.1 Scope
3.2 Designation process
3.3 Incentives
3.3.1 Marketing exclusivity
3.3.2 Protocol assistance
3.3.3 Tax credits
3.3.4 Research grants
CHAPTER 4 SITUATION IN OTHER NON-EUROPEAN
COUNTRIES
4.1 Countries with orphan drug policies
4.1.1 Japan
4.1.2 Australia
4.1.3 Singapore
4.1.4 South Korea
4.2 Situation in other major countries
4.2.1 Canada
4.2.2 New Zealand
CHAPTER 5 SITUATION IN EUROPE
5.1 Research programmes
5.2 Current national situation
5.2.1 Denmark
5.2.2 France
5.2.3 Germany
5.2.4 Italy
5.2.5 Netherlands
5.2.6 Spain
5.2.7 Sweden
5.2.8 UK
5.3 Evolution of EU position
5.4 EU Regulation
5.4.1 Timetable
5.4.2 Canvassing views
5.4.3 Contents of final version
5.4.4 Implementing regulations
5.4.5 National incentives
5.4.6 Review
5.4.7 Comments
CHAPTER 6 WHAT HAS BEEN ACHIEVED
6.1 US
6.1.1 Orphan designations and approvals
6.1.2 Sponsor profile
6.1.3 Grants awarded
6.2 Japan
6.3 Australia
6.4 Singapore
CHAPTER 7 ALLEGED ABUSES
7.1 'Blockbuster' orphans
7.1.1 Growth hormone
7.1.2 Zidovudine
7.1.3 Erythropoietin
7.1.4 Alglucerase
7.2 Non-exclusivity
7.2.1 Human growth hormone
7.2.2 Erythropoietin
7.2.3 Interferon beta
7.3 Attempts to amend the Orphan Drug Act
CHAPTER 8 PROFILES
8.1 Case studies of orphan drugs
8.1.1 Penicillamine
8.1.2 Thalidomide
8.1.3 Cysteamine
8.1.4 Alglucerase
8.1.5 Tobramycin
8.1.6 Factor IX
8.2 Specialist orphan development companies
8.2.1 Orphan Europe
8.2.2 Orphan Medical
8.2.3 Swedish Orphan
8.3 Named-patient suppliers
8.3.1 International Drug Information Service
8.3.2 Penn Pharmaceutical Services
CHAPTER 9 BENEFITS TO INDUSTRY
9.1 Views on US Act
9.1.1 Rare disease prevalence/non-profitability
9.1.2 Incentives
9.2 Views on Japanese regulations
9.2.1 Regulatory issues
9.2.2 Pricing issues
CHAPTER 10 INTO THE FUTURE
10.1 Benefits of collaboration
10.2 Pricing and cost issues
10.3 Achieving the balance
10.4 Need for EU legislation
10.5 Industrial strategy
10.6 Remaining barriers
CHAPTER 11 CONTACT DETAILS
11.1 Regulatory and other government bodies
11.2 Umbrella groupings of rare disease patient advocacies
11.3 Named-patient suppliers
11.4 Specialist orphan drug companies
REFERENCES
APPENDIX 1
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