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Overview

Business Intelligence for the Pharmaceutical Industry

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Some key findings from this report

• The FDA and EMEA are asking for more data in drug applications that pose heightened safety concerns, although there have been no official changes in overall drug approval standards.
• The number of new molecular entities approved in the US each year has declined from 36 to 18 since 2004, despite a sustained increase in the amount of clinical trials conducted.
• The EMEA is approving fewer products than the FDA even though processing speed and volume have been significantly improved. System reforms include reduced review times, faster approvals for innovative drugs and relaxed requirements in pediatric trials.
• Information about new drug candidates is being increasingly shared by the FDA and EMEA as globalization continues throughout the pharma industry. Such convergence is prominent in the orphan drugs area, where evaluations require considerable resources.
• Biosimilar approval structures in the EMEA are significantly more comprehensive than those of the FDA. Europe is now becoming the global center for biosimilar development and production.

Drug Approval Trends at the FDA and EMEA

Drug safety has become a major industry concern following the market withdrawal of Merck’s Vioxx in 2004 and several other discontinuations of COX-2 inhibitors. Although adverse reactions were previously considered a relatively minor risk compared to a drug’s wider benefit, both newly approved and established drugs are now under intense scrutiny.

Drug approvals in the US and Europe have become subject to approval delays, rising requests for further clinical data, and greater chances of rejection. Regulators are currently attempting to reconfigure review processes in order to minimize delays in recognition of the potential mass benefit of new applications. Drug Approval Trends at the FDA and EMEA: Process improvements, heightened scrutiny and industry response is a new report published by Business Insights that provides a comprehensive review of current drug approval trends in the US and Europe with case studies highlighting successful and unsuccessful applications. Emerging regulatory developments and process improvements are examined and the strategic measures implemented by developers to enhance the approval potential of their drugs are assessed. This report also evaluates the future regulatory landscapes of the FDA and EMEA, with a review of anticipated developments through to 2012.

Top five reasons to order your copy today

• Understand the changing landscape for FDA and EMEA drug approvals, discover the implications of process changes upon your drug applications and anticipate future regulatory developments with this new report.
• Examine the latest developments in US and European drug approval processes with this report’s detailed analysis of the key regulatory issues surrounding new molecular entities, generic drug applications, indication expansions and drug safety.
• Understand the implications of rising adverse drug reactions by exploring the issues surrounding safety failures and measuring the potentially affected patient populations of recent discontinuations.
• Measure the future impact of drug approval trends on R&D programs using this report’s extensive review of anticipated changes to FDA and EMEA approval frameworks over the 2008-12 period.
• Evaluate the strategies that companies are using to accelerate drug approvals by understanding the latest industrial approaches to safety test improvements, outsourcing, repositioning and the management of clinical study results and data reporting.
• Benchmark successful drug applications and avoid approval delays with this report’s case studies featuring numerous profiles for issues such as risky drug applications, biosimilar approvals and Rx-to OTC switches.

Key issues examined in this report

• Delayed approvals. Regulators are requesting more data to validate or refute conclusions about product risks. This is creating a rise in the size and number of clinical trials as well as overall research budgets.
• Rising R&D costs. Drug developers are continually increasing their R&D expenditures to generate approvable drug applications, despite rising financial pressures in the industry. Emerging technologies such as stem cells, genomics and proteomics are among the most prevalent initiatives being used to identify appropriate drug candidates.
• US generics focus. The FDA is allocating resources to generic drug approvals in an attempt to speed these products to market. However, although Europe has recently developed a clear regulatory path for generic versions of biologicals, this is yet be achieved in the US.
• Regulatory process improvements. Regulators are enhancing their drug review processes with new technologies such as electronic drug applications and technology-based standards for drug review.

Your questions answered

• Why are new molecular entities experiencing growing approval delays?
• How is the FDA responding to allegations that reviewers are being influenced by the intense pressure to approve new products?
• In which therapy areas are the FDA currently showing the greatest flexibility for novel product approval?
• How will the FDA encourage Rx-to-OTC switches of novel medicines?
• What technologies are drug makers utilizing to help speed the drug development process?
• What new processes has the EMEA recently established to significantly speed drug approvals?
• What emerging trends are companies using to bypass early-stage drug testing?
• How has the convergence of FDA and EMEA approaches benefited European drug marketers?

Table of Contents

Executive Summary 10
The shifting regulatory landscape 10
Drug approval trends at FDA 11
Drug approval trends at EMEA 12
Industry response 13
Global drug approval trends through 2012 14

Chapter 1 The shifting regulatory landscape 18
Summary 18
Drug usage in the U.S. and Europe 19
New molecular entities 20
Generic drugs 21
Rising usage of generics 21
Generic drug application trends 22
Indication expansions 23
Drug approval issues 26
Ensuring drug safety 27
Safety shortfalls: rising adverse drug reactions 29
Safety failures: drug withdrawals 30
The importance of time to market 31
Impact of cost constraints 34
Conclusion 35

Chapter 2 Drug approval trends at the FDA 38
Summary 38
Drug approval process 39
Pressure to approve drug candidates 42
Future directions in the FDA drug approval process 43
Ensuring drug safety 44
Evolution of drug safety testing 44
Pediatric safety testing 48
Future directions in drug safety reviews 49
Reducing time to market 50
Fast track approvals 52
Orphan drug approvals 53
Decreasing FDA drug approvals 54
Future directions in speeding drug approvals 56
Delaying risky applications 58
Rising data requirements 58
Case study: hormone replacement therapy 59
Case study: Galvus 60
The future for risky drug applications 62
Biosimilars 62
The future of biosimilar approvals 62
Canadian biosimilars legislation 63
Rx-to-OTC switches 64
New OTC drug categories 66
Antihistamines 67
Emergency contraceptives 69
Hyperosmotic laxatives 71
Ophthalmic allergy medications 72
Proton pump inhibitors 73
Weight loss medications 75
Switch rejections 76
Statins 77
Future directions in nonprescription drug approvals 78
Creation of a pharmacist dispensed class 79
Marketing withdrawals 80
Application withdrawals 81
Future directions in marketing and application withdrawals 82
Conclusion 82

Chapter 3 Drug approval trends at the EMEA 86
Summary 86
Drug approval process 87
History of European drug regulation 87
The formation of the EMEA and the MRP 88
The EMEA 89
The Mutual Recognition Procedure (MRP) 91
The National Competent Authorities 93
The Common Technical Document 93
Ensuring drug safety 96
Creation of Scientific Advisory Groups 97
Future directions in drug safety testing 98
Reducing time to market 98
Accelerated opinions 100
Pediatric testing exemptions 101
Decreasing EMEA approvals vs. FDA approvals 101
Current EMEA approval levels 104
Future directions in speeding drug approvals 104
Delaying risky applications 105
The future for risky drug applications 106
Biosimilars 107
New biosimilar guidelines 107
Future directions in biosimilar approvals 108
Rx-to-OTC switches 109
Future directions in nonprescription drug approvals 110
Marketing withdrawals 110
Application withdrawals 110
Future directions in marketing and application withdrawals 112
Conclusion 112

Chapter 4 Industry response 114
Summary 114
Background 115
Improving safety testing 115
Genomics 116
Stem cell approaches 117
Future drug safety testing improvements 118
Raising investment in R&D 118
Improving R&D productivity 119
Contract research organizations 119
Repositioning 121
Future directions in R&D investment 122
Public reporting of clinical study results 123
United States 123
Europe 124
Association of the British Pharmaceutical Industry 125
Future directions in clinical data reporting 126
Strengthening regulatory processes 126
Coalition for a Stronger FDA 126
Future directions in regulatory support 127
Conclusion 128

Chapter 5 Global drug approval trends to 2012 130
Summary 130
Background 131
Trends at the FDA 132
Focus on drug safety 132
New technology-based standards for drug application review 133
Decreasing application review times 135
Boosting integrity and accountability 136
Trends at the EMEA 137
Focus on drug safety 138
Product Information Management (PIM) Project 139
Cooperation with other regulators 140
Incentives for small drug developers 140
Convergence of regulatory approaches 141
Orphan drug approvals 143
Conclusion 144

Chapter 6 Appendix 146
Index 146

List of Figures
Figure 1.1: Drug safety risk spectrum 28
Figure 1.2: Growth in U.S. reported adverse drug reactions, 1995 – 2007 30
Figure 1.3: U.S. R&D spending versus new drug approvals, 1995 – 2007 36
Figure 2.4: U.S. drug approvals, 1995 – 2007 55
Figure 3.5: Average number of days for centralized procedure positive opinions, 2004 – 2006 99
Figure 3.6: Average number of days for orphan drug designation opinions, 2004 – 2006 100
Figure 3.7: EMEA marketing withdrawals, 2000 – 2007 111

List of Tables
Table 1.1: Selected indication expansions for U.S. commercialized drugs, 2002 – 2007 25
Table 1.2: Potential safety risks of popular drug classes 29
Table 1.3: Significant drug discontinuations and affected patient groups, 1997 – 2007 33
Table 2.4: Timeline of key FDA legislations 45
Table 2.5: CDER Advisory Committees, 2008 48
Table 2.6: U.S. New Molecular Entity (NME) Approvals, 2000 – 2007 56
Table 2.7: Key U.S. Rx-to-OTC switches, 1997 – 2007 66
Table 2.8: Key events in the approval of OTC nonsedating antihistamines 69
Table 2.9: Recent withdrawals of U.S. marketing applications, 2002 – 2007 82
Table 3.10: Leading European National Competent Authorities, 2008 94
Table 4.11: U.S. Clinical trials and R&D spending, 2000 – 2007 118
Table 4.12: Selected repositioning specialists 122