During this hour-long panel discussion, we teased out the opportunities for trial design in the future and discussed how to manage the flexibility of the new era.
The clinical trial landscape is radically changing – the implications for all aspects of drug and device development is substantial.
We are used to orphan programmes with adaptive study designs and often unvalidated clinical endpoints. These registrations have increased with FDA reporting that in 2020, 31 (58%) of CDER’s 53 novel drug approvals, were for treatment of rare or “orphan” diseases.
Now in 2020/21, the COVID-19 pandemic has led to the introduction of numerous platform trials (some highly adaptive), the use of new, barely established outcomes, challenge studies, rolling reviews and other approaches which are different to conventional licensing programmes.
This is stretching developers, regulators and politicians, alike, to understand best how to generate evidence for licensing new technologies.
- Edward Cox, Vice President, Regulatory Affairs at Regeneron Pharmaceuticals
- Giorgio Massimini, Vice President, External R&D at Merck KGaA
- Kevin Carroll, Expert Statistician & Owner of KJC Statistics
- Rene van der Merwe, Chief Medical Officer and Head of Clinical Development at Galvani Bioelectronics