Current usage and challenges of master protocols-based on survey results by ASA BIOP oncology methodology working group master protocol sub-team.

ABSTRACT

Background: Master protocol trials, such as basket trials, umbrella trials or platform trials, have the potential of increasing efficiency in modern drug development. Meanwhile, though the concept of master protocol is getting more and more accepted, many challenges exist from design to implementation of these trials. To understand current usage and challenges of master protocol trials in action, American Statistical Association (ASA) Biopharmaceutical Section (BIOP) Oncology Methods Scientific Working Group Master Protocol Sub-team conducted a survey with the goal of providing valuable information for the community to understand the current usage of master protocol, with the goal to identify the challenges. Methods: A total of 19 questions were included in an online survey that was distributed between April and May 2021. To avoid over-reporting within an organization, a pre-determined list of contacts from 37 organizations were reached out with the shared online link of the survey. Literature research and experience from the working group on challenges of master protocols are also summarized and discussed extensively. Results: A total of 39 responses were received from 37 organizations. Thirty-one (79%) respondents indicated that they had trials with master protocol(s) in planning or implementation in their organization with most applications (54%) in oncology. Self-reported challenges on trial design, regulatory engagement and trial implementations were further summarized in the report. Conclusions: The survey results were consistent with previous literatures and expectations of members from the Scientific Working Group Sub-team. Multiple stakeholders are called to work collaboratively to remove roadblocks for future usage of master protocol trials.