NEW YORK, March 4 Reportlinker.com announces that a new market research report is available in its catalogue:
Opportunities in Orphan Drugs: Strategies for developing maximum returns from niche indications
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The US government introduced the Orphan Drugs Act (ODA) in 1983 as a means to provide incentives to drug developers so that they would develop treatments for diseases for which incidence is so low that that normal market forces are unable to deliver effective medication for these conditions. A 1984 modification to the ODA defined an Orphan Disease as any condition affecting fewer than 200,000 people in the US. The US was followed by both Japan and the EU which have also introduced their own orphan drug legislation in 1993 and 2000 respectively.
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Industry has responded to this call for action and the FDA has approved over 270 orphan drugs in the US whilst there are another 583 listed in the Medtrack database as being in ongoing development. This has been motivated by the special incentives provided by the FDA and the EU including enhanced market exclusivity, research grants and tax incentives.
Orphan drugs present opportunities to pharma companies looking to move into more niche indications and away from mass market indications that have very low levels of clinical unmet need. Although there are many successful orphan drugs that have made large profits for their developers (Gleevec, NovoSeven etc) the development of these products must be approached carefully. This report uses a mixture of case studies and data from secondary sources to elucidate the key issues in the development of orphan drugs and ways to navigate them.
Key features of this report
-- Overview of orphan drugs legislation in the US, EU, Japan and Australia including details of key incentives for developers and history of orphan designations and approvals.
-- Evaluation of clinical trial and approval strategies including data on current clinical trial designs and strategies to maximize the chances of clinical trial success through the use of adaptive clinical trials.
-- Overview of orphan drug reimbursement issues in both the US and the EU including details of the importance of the UK's NICE and the development of novel risk sharing agreements.
-- Examination of the role being played in orphan drug development by leading players in the pharmaceutical and biotech industry and the motivations behind this.
Scope of this report
-- Gain an understanding of key orphan drugs legislation and the incentives available to the developers of drugs which successfully gain orphan designation.
-- Get a quick overview of the overall orphan drug development pipeline in the EU and US.
-- Gain strategic insight into the key differences between the development of orphan drugs and non-orphan drugs including the importance of patient advocacy groups.
-- Discover key strategies to maximize revenues from orphan drugs through gaining market access and broadening the product label.
Key Market Issues
-- Gaining orphan designation for a product confers access to various incentives. Once a product is granted orphan designation its developers gain a variety of incentives including extended market exclusivity, tax credits and research grants. These incentives have motivated the development of hundreds of products for orphan indications in the US, EU and Japan.
-- The move away from reliance on blockbuster products towards niche indications is driving heightened interest in orphan drugs from companies of all sizes Orphan drugs lie at the opposite spectrum to blockbuster drugs and present Big Pharma with opportunities to enter specialist niche markets. Although on first analysis orphan drugs should not be particularly interesting to the pharma industry on account of their limited patient potential, by 2006 there were 50 orphan products with sales in excess of $200m of which 19 were blockbuster products and it is this which sustains the pharma industry's interest in developing orphan drugs.
-- Cancer continues to have the largest number of pipeline orphan drugs. This is largely motivated by the nature of cancer which allows it to be stratified into niche sub-diseases which qualify as orphan diseases. Developing orphan drugs for cancer indications makes additional sense as it presents opportunities to gain access to further orphan indications which have a similar disease mechanism.
Key findings from this report
-- The developers of orphan drugs receive several incentives from the governments of countries that grant orphan status including extended market exclusivity, fast track assessment, research grants and tax incentives.
-- There are 583 orphan drugs in active development with Cancer being the leading indication for which they are being developed with over 100 products in active development in the US alone.
-- The development of an orphan drug is often a strategy pursued by small biotech and pharma companies with a view to licensing the product out in the later stages of development as part of an exit strategy. One example of this is PharmaMar's development of Yondelis which has involved a complex network of partnerships as a source of both funding and expertise.
Key questions answered
-- What is the key legislation concerning orphan drugs in the US, Japan and the EU?
-- What incentives are available to the developers of orphan drugs in the US, Japan and the EU?
-- What is the involvement of leading pharmaceutical companies in the development of orphan drugs?
-- Why are orphan drugs an attractive proposition for pharmaceutical companies looking to expand their product portfolios
Companies mentioned
Actelion, Bayer, Eli Lilly, Genzyme, GSK, Johnston & Johnston, Merck & Co, Novartis, Pfizer, Roche and Sanofi-Aventis
Table of Contents
Opportunities in orphan drugs
Executive summary 8
Orphan drug legislation 8
The orphan drug pipeline 9
Orphan drug development trends and strategies 10
Orphan drug strategies 11
Chapter 1 Orphan drug legislation 14
Summary 14
Introduction 15
Orphan drug legislation in the US 15
Orphan drugs act 1983 15
Orphan drug designation 16
Orphan grant program 18
Orphan drug legislation in the EU 19
The Committee for Orphan Medicinal Products 19
Orphan designation process 20
EU incentives for orphan medicinal product developers 20
Orphan drug legislation in Japan 21
Incentives for orphan drug developers in Japan 22
Orphan drug legislation in Australia 23
Incentives for orphan drug developers in Australia 23
Chapter 2 The orphan drug pipeline 26
Summary 26
Introduction 27
Oncology 29
Istodax - romidepsin 29
Yondelis - trabectedin 30
Omapro - omacetaxine 33
Clolar - clofarabine 34
Onrigin - laromustine 35
TM601 36
EGEN - 001 37
Central nervous system 38
Zenas - amifampridine 38
H P Acthar Gel 39
ITI111 - midazolam 42
Respiratory and pulmonary systems 43
Surfaxin 43
Anti-infectives 44
Cayston - aztreonam lysine 44
ABthrax - raxibacumab 46
Autoimmune and inflammation 48
EN 101 48
Genetic diseases and dysmorphic syndromes 49
Uplyso - taliglucerase alfa 49
Chapter 3 Orphan drug development trends and issues 54
Summary 54
Introduction 55
Clinical trial and approval strategies for orphan drugs 55
Adaptive clinical trials in orphan drug development 58
The role of patient advocacy groups 62
Patient group/industry R&D partnerships 63
Orphan drug reimbursement issues 64
Orphan drug access in the US 65
Orphan drug access in the EU 65
Pharmaceutical risk-sharing agreements 70
Chapter 4 Orphan drug strategies 74
Summary 74
Introduction 75
The move towards niche indications 75
Opportunities in orphan drugs 76
Small pharma and biotech 77
Big pharma and established biotech 78
Pfizer 78
GlaxoSmithKline 82
Novartis 83
Sanofi-Aventis 85
Roche 86
Johnson & Johnson 88
Merck & Co 89
Eli Lilly 90
Bayer 91
Orphan drug specialists 92
Genzyme 92
Actelion 94
Strategies for sales growth for orphan drugs 96
Expansion into non-orphan indications 97
Expansion into further orphan indications 98
Using biomarkers to develop orphan drugs 101
Conclusion 104
Glossary 105
Index 106
List of Figures
Figure 1.1: Number of products receiving orphan designation in the US, 1984-December 2009 16
Figure 1.2: Number of Orphan drug approvals in the US, 1990-2008 17
Figure 1.3: Average NDA approval time, (months), 1995-2008 18
Figure 2.4: Therapeutic focus of products given orphan designation in the EU, 2009 27
Figure 2.5: Therapeutic focus of products given orphan designation in the US, 2009 28
Figure 2.6: Yondelis deals and alliances structure 32
Figure 2.7: EGEN-001 mode of action 38
Figure 2.8: Licensing deals for H P Acthar Gel 41
Figure 3.9: Blinding in US orphan drug trials, 2009 57
Figure 3.10: Randomization in US orphan drug trials, 2009 57
Figure 3.11: Comparators in US orphan drug trials, 2009 58
Figure 3.12: Reasons for adopting adaptive clinical trials 60
Figure 3.13: Overview of strategic considerations when designing adaptive clinical trials 61
Figure 3.14: Overview of operational considerations when designing adaptive trials 62
Figure 3.15: Relationship between annual cost of treatment (EUR) per patient and prevalence 69
Figure 3.16: The relationship between cost-effectiveness and societal value 70
Figure 4.17: Blockbuster to orphan drugs 75
Figure 4.18: Required conditions for stratified medicine 102
List of Tables
Table 1.1: Comparison of orphan drug systems 24
Table 2.2: Yondelis orphan designations 30
Table 2.3: Surfaxin's US orphan designations by date 43
Table 3.4: Recent deals involving CFF and pharma companies 64
Table 4.5: Pfizer's orphan designations since 2003 79
Table 4.6: Pfizer's active orphan programs 79
Table 4.7: GlaxoSmithKline's marketed US orphan drug portfolio 82
Table 4.8: GlaxoSmithKline's active orphan programs 82
Table 4.9: Novartis' marketed US orphan drug portfolio 84
Table 4.10: Novartis's active orphan programs 84
Table 4.11: Sanofi-Aventis's active orphan programs 86
Table 4.12: Roche's marketed US orphan drug portfolio 86
Table 4.13: Roche's active orphan programs 87
Table 4.14: J&J's marketed US orphan drug portfolio 88
Table 4.15: J&J's active orphan programs 89
Table 4.16: Merck & Co's active orphan programs 90
Table 4.17: Eli Lilly's marketed US orphan drug portfolio 90
Table 4.18: Eli Lilly's active orphan programs 90
Table 4.19: Bayer's marketed US orphan drug portfolio 91
Table 4.20: Genzyme's global marketed orphan product portfolio ($m), 2008 92
Table 4.21: Genzyme's lysosomal storage disorder product history 94
Table 4.22: Actelion's global marketed portfolio ($m), 2008 95
Table 4.23: Actelion's product pipeline 96
Table 4.24: Gleevec's US orphan indications 99
Table 4.25: Nutropin's US orphan indications 100
Table 4.26: Orphan designated products with at least one associated clinically validated biomarker 103
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Drug and Medication Industry: Opportunities in Orphan Drugs: Strategies for developing maximum returns from niche indications
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