We surveyed and accumulated a list of the major benefits gained when executing an adaptive clinical design strategy for their lead orphan product:
· Flexibility of clinical pathway and secondary endpoint in particular
· Prompt development
· Adaptively adjust power/size of a registration trial.
· Perceived higher chance of success
· Faster time to approval with lower cost
· Ability to use fewer subjects with more transparency of what is acceptable to regulatory agencies
· Cast a wide net during the clinical trial stage while conserving cost efficiencies & not breaking the bank or going belly up in the process
The World Orphan Drug Congress 2012 has a dedicated 2 day stream on orphan drug clinical development, and a morning session will be drilling down into the challenges and solutions available to drug developers during the design process.
Why not see how pioneering orphan biotech Prosensa are relentlessly driving their therapies through the clinic?
Or check out what our previous delegates view as the most significant challenges to designing a clinical trial for your orphan drug.