The push for innovation in orphan disease research is driven by an accelerated research and development cycle, patient and family concerns for timely product access during the development process as well as following product registration, and the emergence of a regulatory and reimbursement environment facilitating innovation in program design. During this webinar, Worldwide Clinical Trial experts will be discussing these innovation drivers as well as emerging trends in clinical trial methodology for orphan disease drug development and ways to overcome challenges through the use of observational trials.
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