Drug Approval Trends at the FDA and EMEA: Process improvements, heightened scrutiny and industry response

Table of Contents

• The shifting regulatory landscape
• Drug approval trends at FDA
• Drug approval trends at EMEA
• Industry response
• Global drug approval trends through 2012

• Summary
• Drug usage in the U.S. and Europe
• New molecular entities

• Generic drugs
• Rising usage of generics
• Generic drug application trends
• Indication expansions
• Drug approval issues
• Ensuring drug safety
• Safety shortfalls: rising adverse drug reactions
• Safety failures: drug withdrawals
• The importance of time to market
• Impact of cost constraints
• Conclusion

• Summary
• Drug approval process
• Pressure to approve drug candidates
• Future directions in the FDA drug approval process
• Ensuring drug safety
• Evolution of drug safety testing
• Pediatric safety testing

• Future directions in drug safety reviews
• Reducing time to market
• Fast track approvals
• Orphan drug approvals
• Decreasing FDA drug approvals
• Future directions in speeding drug approvals
• Delaying risky applications
• Rising data requirements
  • Case study: hormone replacement therapy
  • Case study: Galvus
• The future for risky drug applications
• Biosimilars
• The future of biosimilar approvals
• Canadian biosimilars legislation

• Rx-to-OTC switches
• New OTC drug categories
  • Antihistamines
  • Emergency contraceptives
  • Hyperosmotic laxatives
  • Ophthalmic allergy medications
  • Proton pump inhibitors
  • Weight loss medications
• Switch rejections
• Statins

• Future directions in nonprescription drug approvals
• Creation of a pharmacist dispensed class
• Marketing withdrawals
• Application withdrawals
• Future directions in marketing and application withdrawals
• Conclusion

• Summary
• Drug approval process
• History of European drug regulation
• The formation of the EMEA and the MRP

• The EMEA
• The Mutual Recognition Procedure (MRP)
• The National Competent Authorities
• The Common Technical Document
• Ensuring drug safety
• Creation of Scientific Advisory Groups

• Future directions in drug safety testing
• Reducing time to market
• Accelerated opinions
• Pediatric testing exemptions
• Decreasing EMEA approvals vs. FDA approvals
• Current EMEA approval levels
• Future directions in speeding drug approvals

• Delaying risky applications
• The future for risky drug applications

• Biosimilars
• New biosimilar guidelines
• Future directions in biosimilar approvals

• Rx-to-OTC switches
• Future directions in nonprescription drug approvals
• Marketing withdrawals
• Application withdrawals
• Future directions in marketing and application withdrawals
• Conclusion

• Summary
• Background
• Improving safety testing
• Genomics
• Stem cell approaches
• Future drug safety testing improvements

• Raising investment in R&D
• Improving R&D productivity
  • Contract research organizations
• Repositioning
• Future directions in R&D investment
• Public reporting of clinical study results
• United States
• Europe
• Association of the British Pharmaceutical Industry

• Future directions in clinical data reporting
• Strengthening regulatory processes
• Coalition for a Stronger FDA
• Future directions in regulatory support
• Conclusion

• Summary
• Background
• Trends at the FDA
• Focus on drug safety
  • New technology-based standards for drug application review
• Decreasing application review times
• Boosting integrity and accountability

• Trends at the EMEA
• Focus on drug safety
• Product Information Management (PIM) Project
• Cooperation with other regulators
• Incentives for small drug developers
• Convergence of regulatory approaches
• Orphan drug approvals
• Conclusion

Index

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

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