A research team at the University of Washington (UW) has been approved for more than $1 million in research funding by the Patient-Centered Outcomes Research Institute (PCORI) to develop statistical methods for analysis of platform trials.
Led by Ting Ye, an assistant professor of biostatistics at the UW School of Public Health, this three-year study aims to develop and apply a suite of robust and efficient statistical methods and software for estimating and inferring treatment effects in platform trials.
Platform trials are innovative clinical trials that use a master protocol (i.e., one overarching protocol) to evaluate multiple treatments in an ongoing manner, where new treatments can be added to the platform as they become available, and existing ones can leave the platform based on a finding of futility or efficacy.
“Platform trials represent an exciting advancement in clinical trial design to make the clinical trial process faster and more efficient. However, their complexity and adaptive nature introduce significant challenges to statistical analysis,” Ye explained.
“Issues that are straightforward in traditional parallel-group trials are now brought to the forefront. By addressing these critical statistical challenges, our research can lead to increased trust and, consequently, more adoption of platform trials, thereby accelerating clinical drug development and improving care for patients with unmet medical needs.”
In a recent preprint published on arXiv, Ye and co-authors Patrick Heagerty, a UW professor of biostatistics, and Nicole Mayer-Hamblett, a UW professor of pediatrics and adjunct professor of biostatistics, introduced a foundational framework for platform trials.The study addressed two fundamental challenges: clearly defining meaningful estimands and developing robust estimation methods.
“Achieving clarity on the estimand is crucial for addressing other key issues of platform trials in future research,” Ye emphasized.
“What makes this work particularly exciting is not only the innovation and statistical rigor we bring to platform trials but also our collaboration with a diverse stakeholder panel,” Ye added. “This panel includes patients, leading clinicians, and statisticians involved in landmark Phase II and III platform trials. Their perspectives and insights will be invaluable to our investigative team and the broader impact of this research.”
This study is among the latest methodology studies that PCORI has funded to address gaps in comparative clinical effectiveness research (CER) methods. These studies provide results that guide researchers in planning future studies and improve the strength and quality of evidence generated by CER.
“This study was selected for its potential to address a high-priority methodological gap in patient-centered comparative clinical effectiveness research,” said PCORI Executive Director Nakela L. Cook, M.D., MPH. “Improving methods for conducting comparative clinical effectiveness research helps ensure this research generates sound, trustworthy evidence to help patients and those who care for them become more empowered decision makers. We look forward to following the study’s progress and working with Professor Ye to share the results.”
Ye’s award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.
PCORI is an independent, nonprofit organization authorized by Congress with a mission to fund patient-centered comparative clinical effectiveness research that provides patients, their caregivers and clinicians with the evidence-based information they need to make better-informed health and health care decisions.
