Xiaoyun Li1, Chengxing Lu2, Kristine Broglio3, Paul Bycott4, Jie Chen5, Qi Jiang6, Jianchang Lin7, Jingjing Ye8, Jun Yin9. 1. Global Statistics and Data Sciences, BeiGene, San Mateo, CA, USA. 2. Analytics and Data Sciences, Biogen Inc., Cambridge, MA, USA. 3. AstraZeneca, Gaithersburg, MD, USA. 4. Global Biometrics and Data Management, Pfizer Inc., La Jolla, CA, USA. 5. Department of Biometrics, Overland Pharmaceuticals, Dover, DE, USA. 6. Biometrics, Seagen, Bothell, WA, USA. 7. Statistical and Quantitative Science, Takeda, Cambridge, MA, USA. 8. Global Statistics and Data Sciences, BeiGene, Fulton, MD, USA. 9. Division of Clinical Trials and Biostatistics, Mayo Clinic, Rochester, MN, USA.
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. 2022 Annals of Translational Medicine. All rights reserved.
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. 2022 Annals of Translational Medicine. All rights reserved.
Authors: Michael Cecchini; Eric H Rubin; Gideon M Blumenthal; Kassa Ayalew; Howard A Burris; Michele Russell-Einhorn; Hildy Dillon; H Kim Lyerly; Gregory H Reaman; Scott Boerner; Patricia M LoRusso Journal: Clin Cancer Res Date: 2019-01-29 Impact factor: 12.531
Authors: Caroline Claire Morgan; Susan Huyck; Martin Jenkins; Li Chen; Alun Bedding; Christopher S Coffey; Brenda Gaydos; J Kyle Wathen Journal: Ther Innov Regul Sci Date: 2014-07 Impact factor: 1.778
Authors: Amy Burd; Richard L Schilsky; John C Byrd; Ross L Levine; Vassiliki A Papadimitrakopoulou; Roy S Herbst; Mary W Redman; Brian J Druker; David R Gandara Journal: Blood Adv Date: 2019-07-23
Authors: Jay J H Park; Ellie Siden; Michael J Zoratti; Louis Dron; Ofir Harari; Joel Singer; Richard T Lester; Kristian Thorlund; Edward J Mills Journal: Trials Date: 2019-09-18 Impact factor: 2.279
Authors: Elias Laurin Meyer; Peter Mesenbrink; Tobias Mielke; Tom Parke; Daniel Evans; Franz König Journal: Trials Date: 2021-03-04 Impact factor: 2.279