Second APEC Bridging Studies Workshop: Summary and Next Steps

1
Second APEC Bridging Studies WorkshopSummary
and Next Steps

• Bert Spilker, PhD, MD
• 26 May 2001

2
A Recap

• ICH and the Global Cooperation Group
• The E5 guideline past, present and future
• Global drug development and E5
• Regulatory Environment
• Statistical methodology

3
ICH and GCG

• International leadership role in scientific
  harmonization
• Requirements for drug development must have a
  scientific basis
• Value of harmonization of regulations
• Brings innovative medicines to patients faster
• Reduces delays in drug development
• Minimizes duplication of clinical studies
• GCG Outreach to non-ICH countries
• ICH can add value in non-ICH countries

4
E5

• Prospective harmonization to
• Reduce duplicative clinical testing
• Minimize delays in drug development
• Aimed at facilitating registration of legacy
  medicines in ICH regions
• Implementation is complex!
• Focus today must be relevance of E5 to public
  health and global drug development

5
E5 and Global Development

• Large, multi-center, multi-ethnic clinical
  development programs
• Inter-individual gt inter-ethnic differences
• Data extrapolation often possible
• Bridging studies conducted less frequently
• Harmonized E5 implementation is critical

6
E5 Principles

• 3 ethnic groups Asian, Black, Caucasian
• Country-specific studies are not scientifically
  justified
• Internationally accepted approach is critical
• Scientific methodology
• Regulatory process
• Bridging studies should only be conducted if
  scientifically necessary

7
E5 Criteria

• Steps in assessing the need for bridging
• Can the existing data be extrapolated to the new
  population?
• If not, are the differences that exist clinically
  relevant?
• If they are, what study is required to allow the
  data to be extrapolated? This is the bridging
  study
• Steps 1 and 2 are bridging justification

8
E5 Implementation

• Must not cause disharmony among regulators
• Requires
• Sound science-based assessment of Clinical Data
  Package and relevance to new region (bridging
  justification)
• Well-defined regulatory process
• Statistics has a role, but must consider
• Statistical significance vs significance to
  patients and public health
• Questions that need to be answered before
  statistics are used
• Clinical relevance is key

9
Regulatory Environment

• Support innovation and public health
• Science based
• internationally harmonized scientific standards
• Well-structured, consistent and transparent
• E5 early binding consultations
• External advisory committee for appeals during
  approval process

10
Next Steps

• ICH will address implementation issues
• ICH activities will focus on how to determine if
  data can be extrapolated to the new population
• bridging justification
• Country-specific implementation programs will
  follow conclusions of ICH process
• We all have a public health responsibility to
  support harmonization