Clinical Trial Enrollment in Rare Diseases A Company Perspective

1
Clinical Trial Enrollment in Rare Diseases- A
Company Perspective

• Karin Ludwig, MD
• Sr. VP Clinical Research
• Amicus Therapeutics

NORD Conference 30 September 2006
2
Rigorous Environment

• Regulatory requirements
• Drug development process
• ICH/GCP guidelines
• The roles and responsibilities of sponsors and
  investigators
• Patient rights and privacy protection
• Strict study protocols
• Amendments
• Selected patient populations

3
Drug Development Process
Preclinical
IND
Clinical Phase 1
Clinical Phase 2
Clinical Phase 3
NDA
4
Challenging Environment

• Small numbers in rare diseases
• Few patients and few investigators
• Referrals
• Variability
• Multi-national studies
• Operational complexity
• Enrollment timelines

5
Potential for Collaboration

• Information about clinical studies
• Clinical centers
• Patient organizations
• Public study registry clinicaltrials.gov
• General education about drug development