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Orphan drugs : regulatory framework, market positioning and major development challenges and strategies
| Content Provider | Semantic Scholar |
|---|---|
| Author | Amaral, I. |
| Copyright Year | 2016 |
| Abstract | ............................................................................................................. VII Resumo ................................................................................................................ IX Abbreviations ...................................................................................................... XI List of Figures .................................................................................................... XV List of Tables .................................................................................................... XVI Thesis Organization ....................................................................................... XVII Chapter 1 General Introduction ....................................................................... 1 1.1. Rare Diseases ....................................................................................................................................... 3 1.2. Orphan Drugs and global efforts to improve their development ............................................ 6 Chapter 2 Regulatory Framework for the development of orphan drugs . 11 2.1 Legal Framework and orphan drug designation application in the European Union .......... 13 2.1.1 Orphan Medical Product Designation in the EU ............................................................. 14 2.1.2 Post-Orphan Medicinal Product Designation – incentives and supporting programmes ....................................................................................................................................... 18 2.1.3 Post-Orphan Medicinal Product Designation – the road until Marketing Authorisation ..................................................................................................................................... 20 2.2 Legal Framework and orphan drug designation application in the USA ................................ 23 2.2.1 Orphan Medicinal Product Designation in the USA ....................................................... 25 2.2.2 Post-Orphan Medicinal Product Designation................................................................... 28 2.3 Common EMA/FDA Application for Orphan Medicinal Product Designation .................... 31 2.4 Legal Framework and orphan drug designation application in Japan ...................................... 32 2.4.1 Orphan Medicinal Product Designation in Japan ............................................................. 34 2.4.2 Post-Orphan Medicinal Product Designation in Japan ................................................... 36 2.5 Common EMA/Japan Application for Orphan Medicinal Product Designation ................... 39 2.6 Comparison of OD-based legislations in Europe, USA and Japan .......................................... 40 Chapter 3 Pharmaceutical market: positioning of orphan drugs and focus on biotechnology-derived orphan drugs ................................................................ 43 3.1 Pharmaceutical market: general overview .................................................................................... 45 3.1.1 Pharmaceutical market by therapy area ............................................................................ 45 3.1.2 Pharmaceutical market by type of drug developed ........................................................ 46 3.1.3 Pharmaceutical market: orphan drugs versus non-orphan drugs ................................ 47 3.2 Biotechnology providing powerful tools to diagnostics and treatments for orphan diseases ....................................................................................................................................................... 48 |
| File Format | PDF HTM / HTML |
| Alternate Webpage(s) | https://estudogeral.sib.uc.pt/bitstream/10316/37141/1/In%C3%AAs%20Amaral.pdf |
| Language | English |
| Access Restriction | Open |
| Content Type | Text |
| Resource Type | Article |
