We are in the midst of a rapid evolution in clinical trial design, aiming to develop treatments that best meet the needs of all patients. Trials that are more efficient, more adaptive, more inclusive, and ultimately more informative. This has led to a need for a multi-stakeholder debate to discuss the wide range of key topics that underpin future trial design and performance. This 1.5-day hybrid workshop is a neutral and collaborative platform for the evaluation and discussion of innovation in these areas. It will include attendees from all relevant stakeholders (academics, regulators, patient advocates, pharmaceutical industry).
The workshop will focus on a range of topics, from novel (and surrogate) endpoints, patient-reported outcomes and harnessing real-world data, to novel statistical designs and methods. Specific attention will be given to the practicalities and regulatory environment around these approaches, and in the increasingly common scenarios of rare cancer (sub)populations, combination therapies, and utilization of master protocols.
Informative lectures and panel discussions will be open to all attendees, to formulate how novel techniques can be best implemented in drug development-related trials, based on insights from all relevant stakeholders. The workshop outcome will be published by CDDF and is expected to provide a valuable resource for the clinical trial industry.
- How to use novel endpoints and include them in novel trial designs.
- How to use integrated real-world data or more pragmatic trial designs.
- How to best describe patient-reported evidence generation.
- To familiarize with the new statistical methods and designs now in use.
- How to address the practicalities of running trials in small (sub)populations.
The target is a multidisciplinary audience of academia representatives, EU and US regulatory bodies (EMA, FDA, National Agencies), the pharmaceutical industry, HTAs, and patient advocates.