ON-DEMAND WEBINAR

The Impetus for Observational Research in Orphan Drug Development


DR. MICHAEL MURPHY
Chief Medical and
Scientific Officer

BARBARA ZUPANCIC
Director, Global Patient
Recruitment and Retention

LORNA GRAHAM
Associate Director, Project
Management, Evidence

JOSIE MEASURES
Vice President
Biostatistical Operations

 

Summary


During this free on-demand webinar, Worldwide Clinical Trial experts discuss emerging trends in clinical trial methodology for orphan disease drug development, innovation drivers in orphan disease research, and ways to overcome challenges through the use of observational trials by:

  • Providing an impetus for further enhancements in the drug development processes in orphan disease research
  • Describing both benefits and limitations of program innovation from the perspective of patient and family engagement
  • Expanding the portfolio of observational research methods which can complement product registration programs
  • Highlighting techniques that have been considered for data analyses and methods of presentation to assist in informing healthcare decisions